Hematology and Oncology - Cook Children's

Hematology
and Oncology
2005-2009
Hematology and Oncology 1

Knowing that every child’s life is sacred, it is the
promise of Cook Children’s to improve the health of
every child in our region through the prevention and
treatment of illness, , disease and injury.
2 Hematology and Oncology

table of contents
From the Desk of Gretchen Eames, M.D., M.P.H.,
Medical Director, Hematology and Oncology Center,
Stem Cell Transplant Program ................................................1
Message from Rick W. Merrill, President and CEO ................3
Hematology and Oncology Center Overview ..........................5
From Research to Patient-Centered Clinical Care ..................7
Acute Lymphocytic Leukemia ............................................... 11
Bone Marrow and Stem Cell Transplant
Program Overview ................................................................15
Hematology
and Oncology
2005-2009
Transplant Experience with Acute
Lymphocytic Leukemia ..........................................................19
Neuro-Oncology Program .....................................................23
Radiation Therapy .................................................................27
Neuroblastoma Program........................................................29
Acute Myeloid Leukemia Update ..........................................33
Support Services ...................................................................37
Cancer Registry Operations ..................................................41
Quality Improvement Initiatives .............................................43
Hematology Services ............................................................45
Hematology and Oncology Research ...................................49
Physician Profiles ..................................................................54
Locations ...............................................................................59
In 2009, U.S.News & World Report named Cook Children’s to its list of
America’s Best Children’s Hospitals, ranking it in the top 30 for Cancer.
Lobby of Cook Children’s Grapevine Hematology and Oncology Clinic
Hematology and Oncology 3

4 Hematology and Oncology

From the Desk of Gretchen Eames, M.D., M.P.H., Medical Director,
Hematology and Oncology Center, Stem Cell Transplant Program
Dear friends and colleagues,
For more than 27 years, Cook Children’s Hematology and Oncology Center has been providing top quality
clinical care to patients with cancer and blood disorders. The Hematology and Oncology Center is one of the
larger programs in the Southwest and we have a distinguished reputation of excellent care and outcomes.
Additionally, we are ranked in the top 5 percent of the nation’s hospitals for our nursing excellence.
The Hematology and Oncology Center’s mission is to provide high-quality, familyand
patient-centered care to children and adolescents with cancer and blood
disorders, as well as to provide access to leading-edge therapy and research.
Our goal is to cure those with these devastating diseases through discovery and
implementation of innovative research and treatment advances.
Through the following pages you will read our stories and learn of our successes toward this goal over the last
few years. It has been a time of many changes and exciting growth. It has also been a time of preparation and
planning for a very exciting future, which includes a new expansive center incorporating both our outpatient
and inpatient clinical care services and teams essentially under one roof by late 2011. We also opened an
innovative, full-service clinic in Grapevine, an area located between Dallas and Fort Worth, this spring, which
allows our patients to receive evaluations and therapies closer to home.
After many years of great leadership under the helm of W. Paul Bowman, M.D., and then Tim Griffin, M.D., I
took the reins in late 2006 and it has been a wonderful ride. Our staff roster has grown tremendously as we
strive to provide the safest and best care for the patients we serve. I am pleased to announce the addition of
four superb physicians to our center since 2006, recruitment of two additional physicians who joined our team
in the summer of 2009, and the hiring of three additional nurse practitioners. We also formally established
many of our sub-programs, such as leukemia and lymphoma, neuroblastoma, neuro-oncology and many new
hematology initiatives, including a thrombophilia program and a stroke clinic. These are just a few of our many
accomplishments over the last few years.
The challenges are great as we reach to serve patients in more areas, carry out complex care and perform
more stem cell transplants. With the excellent collaboration amongst our staff, and with all of our ancillary
services and other subspecialists, it is a task we can achieve with pride. We have also reached beyond our
walls and collaborated with great institutions within our region, as well as nationally, to expand our research
opportunities and bring the best therapies back home.
It is with great pleasure and enthusiasm that I present this report of our activities in the Hematology and
Oncology Center within Cook Children’s Health Care System. It is our privilege to serve our patients and their
families.
Gretchen Eames, M.D., MPH
Medical Director
Cook Children’s Hematology and Oncology Center, Stem Cell Transplant Program
Hematology and Oncology 1

2 Hematology and Oncology

Message from Rick W. Merrill, President and CEO
We are immensely proud of the distinguished reputation for excellent care and outcomes achieved
by our Hematology and Oncology Center as demonstrated by its inclusion in U.S.News & World
Report’s 2009 edition of America’s Best Children’s Hospitals, ranking it as No. 29 for Cancer. Since
1986, Cook Children’s Bone Marrow and Stem Cell Transplant Program has performed more than 700
autologous and allogeneic transplants, making it one of the more diverse and experienced pediatric
transplant programs in the Southwest.
Through our medical center, specialty clinics, neighborhood clinics, physicians’ offices, outpatient
settings, health plan and home care services, we are uniquely poised to care for children throughout
the region and improve the health of every child. Our Magnet-designated Cook Children’s Medical
Center places us in the top 5 percent of the nation’s hospitals for nursing excellence and we are
blessed to have an extraordinary team of caregivers and physicians who share an unwavering
commitment to our patients and families.
As you read this report, you’ll learn more about the comprehensive cancer care we
provide and no doubt will be impressed by the rich history and success of
this program.
Sincerely,
Rick W. Merrill
President and CEO
Cook Children’s Health Care System
About Cook Children’s
Cook Children’s is one of the country’s leading integrated pediatric
health care delivery systems. Based in Fort Worth, Texas, the nonprofit
organization includes a nationally recognized Medical Center,
Physician Network, Home Health company, Northeast Hospital,
Health Plan and Health Foundation.
Knowing that every child’s life is sacred, it is the promise of
Cook Children’s to improve the health of every child in our region
through the prevention and treatment of illness, disease and injury.
Cook Children’s Medical Center
Hematology and Oncology 3

4 Hematology and Oncology

Hematology and Oncology
Center Overview
When parents discover that their child is severely
sick and requires treatment for cancer or a blood
disorder, it is one of life’s most difficult moments.
patientcentered
therapy
Most parents never consider the possibility their child might be touched by these tragic
circumstances. However, childhood cancer is the leading cause of non-accidental death in
children in the United States. Although cancer remains a serious threat to the health of children,
great strides have been made in treatment. Statistics show that by the year 2010, approximately one in every 250
adults under age 55 will have survived childhood cancer, which provides much hope for both pediatric cancer patients
and their parents.
At Cook Children’s, we are constantly driven to cure our pediatric patients with cancer and blood disorders through
implementation of innovative research and treatment advances. We are dedicated to providing the highest level of
care because we know every child’s life is sacred.
Comprehensive Cancer and Hematology Program
Cook Children’s Hematology and Oncology Center treats infants, children and adolescents with a broad spectrum of
oncologic and hematologic disorders, including:
• Leukemia (acute lymphocytic, acute myelocytic and chronic myelocytic)
• Lymphoma (Hodgkin’s and non-Hodgkin’s)
• Solid tumors of soft tissue (neuroblastoma, Wilms’ tumor, rhabdomyosarcoma and other soft tissue sarcomas,
neuroectodermal tumors, retinoblastoma and a variety of rare childhood tumors)
• Central nervous system tumors
• Bone tumors (osteogenic and Ewing’s sarcoma and rare tumors of the skeletal system)
• Cytopenias (anemia, neutropenia and thrombocytopenia)
• Bone marrow failure syndromes
• Thrombocytopenia (sickle cell disease, thalassemia)
• Bleeding disorders (hemophilia and von Willebrand’s disease)
• Thrombophilia disorders (inherited and acquired clot disorders, stroke)
Patient Statistics
Patient Encounters 2006 2007 2008
Outpatient encounters (includes satellite clinics) 12,357 13,201 13,097
Inpatient days 7,713 8,079 8,016
Hematology and Oncology 5

6 Hematology and Oncology

From Research to Patient-
Centered Clinical Care
If you look at the value of the years you can save
by curing a pediatric patient, you can easily see the
value of pediatric cancer research.
leading
research
Cook Children’s is not a traditional academic center tied to a university, but we are academic in spirit in how we
practice day in and day out. In fact, it was the amount of research being carried out at Cook Children’s that attracted
me to this integrated system.
We build a relationship with the patients’ parents starting day one of the diagnosis, and it is only in the context of
that relationship that we introduce the subject of research. Parents do not want to expose their children to risks, but
most of them come to understand the concept of clinical trials. We explain in this report how our clinical research
has advanced the field of medicine and how it contributes to our cure rates here, as well as advances in oncology
and hematology.
The Hematology and Oncology Center performs much of the clinical research at Cook Children’s, a pattern that
is true for other pediatric institutions. Oncology is making tremendous strides, with new medicines and treatments
developed constantly. Sometimes, the only way for patients to take advantage of leading-edge drugs and therapies
is to participate in a clinical research study. Oncology is benefiting from a veritable explosion of new products as
scientists are starting to unravel the mysteries of cancer.
We always tell our patients and their families that if they should decide not to participate in a research study, their
decision will in no way affect our relationship or change the fact that we will provide them with the best possible care.
Many patients participate in studies, but some patients do not for a variety of reasons. One of the biggest hurdles
can be fitting into the study’s inclusion criteria. In other words, some patients may not qualify medically for studies
that they might have otherwise been willing to join.
We have highly skilled clinical research associates here. They are involved in patient care day-to-day and have a
great deal of interaction with our doctors and nurses. They sometimes identify patients who are good candidates
for specific research studies before we do, and then work through our rigorous system with the doctors to get the
investigation done right.
Gretchen Eames, M.D., MPH
Medical Director, Hematology and Oncology
Center, Stem Cell Transplant Program
Hematology and Oncology 7

Sponsor
Clinical trials and registries between
Jan. 2004 - Jan. 2008
Children’s Oncology Group (COG) 153
Multiple Institutional Collaboration* 13
International Studies (Histiocyte Society) 12
Private Pharmaceutical Industry 9
Pediatric Blood and Marrow Transplant Consortium (PBMTC) 7
Center for Disease Control for Hemophilia 4
Cook Children’s Medical Center (in-house projects) 4
National Marrow Donor Program (NMDP) 4
Hemophilia & Thrombosis Research Society 2
Bone Marrow Transplant Clinical Trials Network (BMT-CTN) 1
Center for International Blood & Marrow Transplant Research (CIBMTR) 1
* (including institutions: Texas Children’s Cancer Center, St. Jude Children’s Research Hospital, University of Oklahoma, University of Colorado, Children’s Hospital of Los Angeles,
Columbia Presbyterian College of Physicians and Surgeons, UT Southwestern Dallas, Children’s Medical Center Dallas and Fred Hutchinson Cancer Center)
8 Hematology and Oncology

From Research to Patient-Centered Clinical Care (continued)
The Bone Marrow and Stem Cell Transplant Program at
Cook Children’s, a key area of clinical research, is one of the
largest in the Southwest. In 1995, we performed just under 18
transplants. By 2007, that rate had tripled to between 50 to 55
transplants per year and in early 2009, the 700th transplant was
performed. Currently, the Bone Marrow Transplant Program
actively participates in clinical trials through the Center for
International Bone Marrow Transplant Research (CIBMTR), the
Blood & Marrow Transplant Clinical Trials Network (BMT-CTN),
the Pediatric Blood and Marrow Transplant Consortium (PBMTC)
and the Children’s Oncology Group (COG).
While the Hematology and Oncology group has gained
prominence in North Texas, I hope ongoing and future research
will give the group and Cook Children’s a greater national profile.
In the future, more pharmaceutical trials will be conducted
by partnering with industry. These studies are often smaller
and more feasible, but there are not as many opportunities as
compared to trials for adults. Yet, if you look at the value of the
years you can save by curing a pediatric patient, you can easily
see the value of pediatric cancer research. Our Hematology
division is also launching more studies, including those for sickle
cell anemia, hemophilia and clotting disorders. I’d also like to see
our physicians more involved in research steering councils on a
national level.
One of the greatest challenges to any clinician conducting
research at Cook Children’s is to strike a balance between
required patient care and key investigations. We don’t have
protected time dedicated to solely pursue research interests.
By taking the time to grow and adding the support necessary to
conduct excellent clinical research, Cook Children’s will be the
best it can possibly be.
The sponsor table on the previous page demonstrates
Cook Children’s diverse experience in clinical research over the
past five years. In order to be accepted and maintain privileges,
Cook Children’s must meet criteria specific to each group, as
well as successfully pass routine audits and site visits.
Year Enrollments in research studies
2004 191
2005 264
2006 289
2007 285
2008 334
* 760 patients resulting in 1363 enrollments
In order to provide clinical trials to patients with blood disorders,
malignancies or those undergoing stem cell transplantation,
Cook Children’s Hematology and Oncology Center participates
in projects that include biology, classification and/or banking
of diagnostic and re-evaluation tissue; diagnosis and multidisciplinary
treatment options, as well as epidemiology, quality of
life, supportive care and registry studies.
Yearly enrollment in research studies continues to grow as is
evident in the table above. As of January 2009, more than 760
patients were enrolled in a clinical trial.
Participation in clinical research is overseen internally by the
Cook Children’s Institutional Review Board (IRB). The IRB
is an ethical oversight committee required by the Food and
Drug Administration and the National Institutes of Health for
organizations that participate in clinical trials using human
subjects. The IRB’s primary purpose is to protect the safety and
rights of pediatric research participants.
Clinically applied research is the medical standard of care for
pediatric oncology at Cook Children’s. In the past four years,
the IRB has overseen the initiation of 118 clinical trial projects
proposed by Cook Children’s Hematology and Oncology
Center. Yearly review of the protocols and monitoring of events
associated with each protocol is used to maintain the continuum
of quality.
Hematology and Oncology 9

10 Hematology and Oncology
treatment
advances

Acute Lymphocytic Leukemia
Twenty-five Years Experience with Relapsed Acute Lymphocytic
Leukemia at Cook Children’s Medical Center
Acute lymphocytic leukemia (ALL) is the most common pediatric malignancy and represents 85 percent of childhood
leukemias. Long-term remission and overall survival approaches 80 percent using contemporary protocols. However,
approximately 25 percent of children diagnosed with ALL are expected to suffer a relapse of their disease. The purpose of
this patient care evaluation presented here is to analyze the outcomes of children diagnosed with ALL at Cook Children’s
Medical Center who have experienced a relapse of their disease. We seek to identify our successes and the major
challenges to improving outcomes while gaining a better understanding of the disease. Patients considered in
long-term remission before relapse, defined as bone marrow relapses greater than 36 months since diagnosis, or
extramedullary relapses occurring greater than 18 months since diagnosis, have a much greater chance of survival
than early relapses. We plan to determine the influence of different treatment regimens and factors that affect
response to therapy after relapse of ALL.
W. Paul Bowman, M.D.
Chairman of Cook Children’s Leukemia and Lymphoma
program, Medical Director, Academic Pediatrics,
Hematology and Oncology Center
W. Paul Bowman, M.D., continues legacy as pediatric hematologist and oncologist
As a fourth year medical student, W. Paul Bowman, M.D., saw a little girl with leukemia. He was inspired by how his
mentoring physician treated the little girl. That experience began an interest in treating children with cancer that continues
to this day.
In addition to his current role as senior pediatric hematologist and oncologist, as well as chairman of Cook Children’s
Leukemia and Lymphoma program, Dr. Bowman serves as the medical director of academic pediatrics for Cook Children’s
Medical Center. He is also a department chair of pediatrics for the University of North Texas Health Science Center and the
Texas College of Osteopathic Medicine (TCOM), Fort Worth, Texas.
These appointments tie together the teaching and research capabilities of the Health Science Center with the renowned
patient care of Cook Children’s and its clinic network to bring leading-edge research and care to pediatric patients in North
Texas.
Under Dr. Bowman’s guidance, TCOM student physicians can take advantage of the knowledge of Cook Children’s
physicians when they instruct and oversee the student interns.
Dr. Bowman, a native of Winnipeg, Canada, graduated from the University of Manitoba as class valedictorian before
becoming a fellow in pediatric hematology-oncology at St. Jude Children’s Research Hospital in Memphis, Tenn. In 1982,
he came to what is now known as Cook Children’s as co-director of the Hematology and Oncology department, making Fort
Worth his home.
He has served on numerous boards and committees in Tarrant County, Texas, including the American Cancer Society,
American Society of Clinical Oncology and the Legislative Task Force on Cancer in Texas. Dr. Bowman won the
Cook Children’s Distinguished Physician Award and was presented with the Gold Headed Cane from the Cook Children’s
Physician Network in 2003. Dr. Bowman was the first subspecialty physician to receive the award, voted on by his peers.
In the following pages, you will find recent research and work written by Dr. Bowman – an important academic presence at
Cook Children’s.
Hematology and Oncology 11

ALL Diagnosis
• B Precursor ....................... 86/106
• T-cell ................................ 14/106
• B-cell .................................. 3/106
• Infant................................... 3/106
Ethnicity
• Caucasian.......................... 64/106
• Hispanic............................ 34/106
• African American................. 5/106
• Asian .................................. 3/106
Sites of Relapse
• BM ................................... 68/106
• CNS .................................. 29/106
• BM/CNS ............................. 3/106
• BM/Testicular....................... 2/106
• Testicular............................. 2/106
• BM & mediastinal relapse..... 1/106
• Nodal.................................. 1/106
The study population is derived from patients whose initial diagnosis of ALL and
relapse of their disease occurred at Cook Children’s from 1982 through 2007. A
total of 617 children have been diagnosed with ALL at Cook Children’s in the last
25 years. Of these patients, 110 (17.8 percent) have suffered a relapse. Four of the
110 patients are not included in this study due to insufficient diagnostic information.
The male to female ratio is 65:41 and the range of age at diagnosis is 3 months to
19 years (median: 6 years, 1 month). Primary sites of relapse for our patients are
consistent with national data.
A total of 43 patients underwent bone marrow or stem cell transplantation to manage
their relapsed disease, 41 allogeneic and two autologous. The majority of these
patients were transplanted during second complete remission (n=29), although 14
were conducted after a second relapse. Twenty-three (23) of the 43 patients are alive
in remission, following transplant.
Among the 106 patients who experienced relapse, 47 patients are alive; this
includes the 23 patients who survived after stem cell transplant, 41 in second
complete remission and six in third complete remission. Although these results
are encouraging, further studies are necessary to determine the best therapies for
relapsed patients. This patient care evaluation will include an analysis of clinical,
biological and treatment factors that influence outcome after relapse of childhood ALL
in a single institution.
The basic demographics of the 617 patients diagnosed with ALL at Cook Children’s
are consistent with national statistics for median age at diagnosis and male to female
ratio (57 percent:43 percent). The racial distribution (60 percent Caucasian, 32
percent Hispanic, 5 percent African American, and 3 percent other) is characteristic
of the population of our referral region of North-central and West Texas. As
expected from published experience, the proportion of patients relapsing that are
male, older or high risk is high relative to their frequency at diagnosis. The race
breakdown is proportional to that of the initial patient population: 64 Caucasian, 34
Hispanic, five African American, and three Asian. The distribution of the patients by
immunophenotype is as follows: B Precursor (n=86), T-cell (n=14) and Mature B-cell
(n=3).There are three patients with ALL who are not classified by immunophenotype,
but by age; they are patients with infant ALL. The most common site of relapse was
the bone marrow (n=68), followed by central nervous system (CNS) (n=29), bone
marrow and CNS (n=3), testicular (n=2), bone marrow and testicular (n=2), with one
bone marrow and mediastinal relapse and one nodal relapse. Seventy-four (74) of
the observed relapses occurred following remission of less than 36 months and 32
relapses took place after remission of greater than 36 months.
Of the isolated extramedullary relapses, 13 took place after initial remission of
less than 18 months and 19 after remission of greater than 18 months. As stated
previously, a total of 43 relapsed patients have undergone marrow transplantation
in an effort to achieve long-term complete remission. Twenty-three (23) of the 43
patients are currently alive in complete remission. Philadelphia chromosome positive
ALL is associated with poor prognosis and a high-relapse rate. All five of these
patients had a bone marrow relapse and a subsequent bone marrow transplant.
Three (3) patients have expired, two of which had subsequent bone marrow relapses.
Two (2) patients with Philadelphia chromosome positive are currently surviving in
second complete remission. Only three (3) patients with mature B-cell ALL had
relapses at Cook Children’s. It is important to note that all of the relapses occurred
early between one to six months. Two (2) patients expired after a subsequent
relapse and the one survivor maintained remission after receiving bone marrow
12 Hematology and Oncology

transplantation. There are three infants with high-risk disease
who all had early bone marrow relapses. The two (2) surviving
had bone marrow transplants in second complete remission
(CR). Among the 106 relapsed patients, 47 are currently
surviving; 41 in second complete remission, six in third complete
remission. In our series, 25 of 51 (49 percent) standard risk
patients remain alive, 22 in second CR and three in third CR.
There are 22 of 55 (40 percent) high-risk patients surviving in
CR, 19 in second CR and three in third CR.
Our review of patients with recurrent ALL brings sharply
into focus the problems associated with managing this
heterogeneous group. Patients with standard risk classification
as defined by the National Cancer Institute (NCI) appear to have
a better chance for long-term remission after relapse than the
high-risk patients.
Conclusion/Summary:
The following conclusions appear to be justified based on our
patient experiences. It is possible to identify a subset of patients
with a favorable outcome following appropriate chemotherapy
and possible bone marrow or stem cell transplantation. This
group is comprised of patients who experience a late bone
marrow relapse (greater than 36 months) and are clinically
standard risk at diagnosis and relapse. Although we can
approach this group with optimism for their overall survival, they
comprise a minority of the relapsing patients.
Second, we can identify a subset of patients whose prognosis
following relapse remains very poor. This group includes those
patients with early bone marrow relapse (less than 36 months),
patients who are classified as high risk at diagnosis and
those with biological features predicting a poor outcome after
relapse. These include patients with T-cell ALL and Philadelphia
chromosome positive ALL. Of the 14 patients with relapsed T-cell
ALL, only the patients with extramedullary relapses are currently
surviving. This confirms that survival after an early bone marrow
relapse in a patient with T-cell ALL is unlikely. There were no
late relapses among the patients with T-cell ALL. Our data is
consistent with national data that a Philadelphia chromosome
positive ALL has very high relapse rates. Three of our five
Philadelphia chromosome positive patients have had multiple
bone marrow relapses and all three have succumbed to their
disease. In our patient population, only 20 of 59 early relapsing
patients are currently surviving. This is consistent with data that
the early relapse is associated with a lower overall survival rate
than the late-relapsing patient.
Third, bone marrow or stem cell transplantation is increasing in
its applicability due to broader donor availability and improved
supportive care. Transplantation may provide the only prospect
for long-term remission and cure among patients that have
Cumulative Survival Percentage
1.0
0.8
0.6
0.4
0.2
0.0
Figure 1
Isolated Extramedullary Relapse:
Survival Functions
Duration of
1st Remission =< 18 M
0 2000 4000 6000 8000
Overall Survival in Days
=< 18M
> 18M
=< 18M-censored
> 18M-censored
early bone marrow relapse or those with a second relapse.
Stem cell transplant provides a better relapse-free survival rate
than chemotherapy alone, but it is also associated with higher
treatment-related morbidity and mortality rates. Of the twenty
(20) early relapsing patients surviving, 16 had bone marrow
transplants. This suggests that stem cell transplant is crucial
to survival for early relapsing patients who otherwise would
have a very poor outcome. Because morbidity and mortality
following transplant is so high, it is important to identify transplant
candidates before the toxicities of other intensive treatment
therapies increase the chances of transplant complications.
It is important to note that in our patient population, there were
no significant differences in outcomes of early versus late
extramedullary relapses (Figure 1). Although there has been
dramatic progress in the treatment of ALL in the last 50 years,
15 to 20 percent of patients may still suffer relapse, increasing
their risk of death from the disease. The observations from our
experience indicate that the approach to treatment should be
stratified according to patient features at original diagnosis and at
the time of relapse. The use of chemotherapy alone or combined
with bone marrow transplantation should be carefully considered,
especially for the early relapsed patient. Future progress in
treating relapse ALL lies in a greater understanding of the
biology and molecular classification of the disease and devising
individualized patient treatment plans.
Analysis done by Lindsay Adkins, undergraduate student Texas Christian University
and W. Paul Bowman, M.D., edited by Teresa Clark, RN, MS HCA, CPON.
Hematology and Oncology 13

14 Hematology and Oncology

Bone Marrow and Stem Cell
Transplant Program Overview
high-quality
care
Since 1986, Cook Children’s Bone Marrow and Stem Cell Transplant
program has performed more than 700 pediatric transplants.
Stem cell transplantation has become an established,
viable treatment modality for many patients suffering
from hematologic malignancies, non-hematologic
malignancies, nonmalignant hematologic disorders,
metabolic disorders and immunodeficiencies.
Unfortunately, approximately 70 percent of candidates
for an allogeneic transplant will not have a suitably
matched related donor and thus require a search for
alternative donor.
In 1986, a group of physicians and patient family
members began a grass roots national campaign
to organize a registry of volunteer marrow donors
now known as the National Marrow Donor Program
(NMDP). To date, the NMDP has facilitated more than
35,000 transplants from its nearly 8 million registered
donors. Marrow registries like the NMDP, in which
Cook Children’s Hematology and Oncology Center is
an active participant, have given children a chance for
a cure where none was previously possible.
Since 1986, Cook Children’s Bone Marrow and Stem
Cell Transplant program has performed more than
700 autologous and allogeneic transplants in children
with cancer, blood disorders or inherited conditions,
making it one of the more diverse and experienced
pediatric transplant programs in the Southwest.
Primary diseases treated 1986 – 2008
Total transplants ....................................... 696
Acute lymphoblastic leukemia (ALL) ......... 297
Chronic myelogenous leukemia (CML) ........ 14
Myelodysplastic syndrome (MDS) ............... 40
Lymphoma .................................................. 36
Neuroblastoma ......................................... 131
Other malignancy........................................ 75
Anemia/hemoglobinopathy ......................... 49
Immune deficiency ..................................... 24
Inherited disorders of metabolism ............... 30
Median length of inpatient stays
Autologous ........................................... 28 days
Matched related ................................... 36 days
Matched unrelated ............................... 39 days
Cord blood ........................................... 51 days
Gretchen Eames, M.D., MPH
Medical Director, Hematology and Oncology
Center, Stem Cell Transplant Program
Hematology and Oncology 15

Breakout Parties
At the time of the patient’s discharge from the Stem Cell Transplant Unit (SCTU), the Child Life, nursing and
physician staff hosts a party for the patient and their family. The patient, family and staff come together on the
balcony outside the transplant unit to wish the patient well and celebrate their discharge home.
16 Hematology and Oncology

Our program is a member of the Center for International Blood
and Marrow Transplant Research (CIBMTR), Pediatric Blood
and Marrow Transplant Consortium (PBMTC) and the Children’s
Oncology Group (COG) stem cell transplant section. We are also
accredited through the Foundation for Accreditation of Cellular
Therapy (FACT). Over the last three years, 30 to 40 transplants
were performed annually for a variety of diseases, with leukemia
being the most common primary diagnosis.
Following this overview is a report by Richard Howrey, M.D.,
regarding our center’s experience with stem cell transplant for
acute lymphoblastic leukemia.
A key focus in our program is patients receiving transplants from
unrelated marrow or cord blood unit donors. After successful
development of a bone marrow transplant program at
Cook Children’s in 1986, the Unrelated Donor (URD) program
began in 1993 with two transplants that year. This number
increased to 20 URD transplants in 1997, just four years later
and over the last 10 years, that volume has maintained. In fact,
URD transplants now account for approximately 50 percent of the
transplants performed at Cook Children’s Medical Center.
The program continues to gain vast experience in the area of
umbilical cord blood (UCB) stem cell transplant with 60 UCB
transplants performed over the last 4 and a half years. Early
analysis shows a 60 to 70 percent overall survival rate for these
patients, which is a very favorable outcome in comparison
to living unrelated donor transplant outcomes (approximate
overall survival 40 to 45 percent). This improved survival is
mostly attributed to a decreased incidence and severity of acute
graft-versus-host disease (GVHD). However, the use of cord
blood units for transplant is limited in the larger adolescent and
young adult population due to limited cell dose available in each
unit. To help overcome the cell dose issue, Cook Children’s
is participating in a national trial utilizing double cord blood
infusions in leukemia patients to assess engraftment, overall
survival and graft-versus-host disease incidence in relapsed
leukemia patients.
The goal of the program is to provide a stem cell or marrow
transplant to any child who needs one and to improve the
outcomes for these patients who do not have better therapy
options. We work to achieve this goal through multidisciplinary,
excellent clinical care, quality initiatives and ongoing
benchmarking against large academic transplant centers and
international data.
Hematology and Oncology 17

18 Hematology and Oncology

Transplant Experience with
Acute Lymphocytic Leukemia
Utilization of uUCB as the source for stem cells
has not only contributed to improved outcomes,
but it has given patients who lack a matched donor
the opportunity to have a life-saving transplant that
otherwise would not be possible.
innovative
treatment
Acute lymphocytic leukemia (ALL) accounts for approximately one-fourth of all childhood cancers. While the cure rate
for children with ALL has steadily improved, there continues to be a small group of patients who will not survive with
conventional therapy alone. For these children, stem cell transplantation may hold the only hope for cure.
Cook Children’s Bone Marrow and Stem
Cell Transplant program has provided more
than 170 stem cell transplants for patients
with acute lymphocytic leukemia since the
program’s inception in 1986.
The most common indication for stem cell
transplant in this group of patients has been
relapsed disease. A smaller proportion
of patients will receive transplants in first
remission either due to very high-risk
characteristics (20 percent) or failure to
achieve remission following initial induction
(5 percent).
In the first 12 years of the program’s
existence, 66 patients with ALL received
transplants with an overall survival rate of 38
percent. In the next half decade, 50 patients
received transplants with an overall survival
rate of 38 percent. In the past five years, 54
patients with ALL have received transplants
with an overall survival rate of 57 percent.
(Figure 1)
Survival Percent
Figure 1
100
80
60
40
20
0
Cook Children’s Medical Center
BMT Survival Curves 1986-2007
0 2 4 6 8 10 12 14 16 18 20
Number of Years
Groups
1986-1997
1998-2002
2003-2007
Richard Howrey, M.D.
Hematology and Oncology Center
Cook Children’s Medical Center
BMT Survival Curves 1986-2007
30
ntage
25
Hematology and Oncology 19
25

20 Hematology and Oncology

0
Transplant Experience 0 2 4with 6Acute 8 Lymphocytic 10 12 14Leukemia 16 18(continued)
20
Number of Years
While the primary indication for stem cell transplant has remained
fairly constant, the source for stem cells has shifted significantly
since 1986. Matched related donors continue to be the first
choice for patients with ALL who need a transplant. During the
early years of our program, patients tended to have an unrelated
bone marrow donor as their source of stem cells, with less than
2 percent of patients receiving an unrelated umbilical cord blood
transplant (uUCB). Between 1998 and 2002, utilization of uUCB
cells gradually increased, but unrelated marrow continued to
be the preferred choice for patients who lack a matched related
donor. In the past five years, the trend toward using uUCB as
the source of stem cells has continued. The gradual shift in stem
cells source can be seen in Figure 2.
One of the great benefits for utilizing umbilical cord blood is
that the degree of human leukocyte antigen (HLA) tissue type
matching is less of an issue. While sibling and unrelated bone
marrow stem cells need to be closely matched, umbilical cord
blood transplants can be successful with less stringent matching.
In fact, less than 20 percent of our cord blood units have been
fully matched, with 30 percent having one mismatch and around
50 percent having two or more mismatches.
Utilization of uUCB as the source for stem cells has not only
contributed to improved outcomes, but it has given patients
who lack a matched donor the opportunity to have a life-saving
transplant that otherwise would not be possible.
and Stem Cell Transplant program steadily improve outcomes.
As newer techniques and agents are developed, the program will
continue to strive for improvement while working toward the best
possible outcome for all children.
Stem Cell Source Percentage
30
25
20
15
10
5
0
Cook Children’s Medical Center
BMT Survival Curves 1986-2007
1
18
5
19
25
10
1986-1997 1998-2002 2003-2007
• UCB
Years
• Unrelated Bone Marrow
The experience utilizing all sources of stem cells is one of the
many factors that have helped Cook Children’s Bone Marrow
Figure 2
Hematology and Oncology 21

22 Hematology and Oncology

Neuro-Oncology Program
Central nervous system (CNS) tumors are the most common
type of solid tumor in children and rank second only to
leukemias as the most common malignancy in children.
Brain and spinal cord tumors account for approximately 20 percent of all pediatric
cancers, with 2,500 to 3,000 children diagnosed in the United States each year. As for
the vast majority of childhood cancers, the cause of central nervous system (CNS) tumors
is not known. Despite progress in successful therapy for all types of childhood cancers in
the past decades, CNS tumors remain the leading cause of cancer death in youth.
collaboration
Cook Children’s Neurosciences, in conjunction with the Hematology and Oncology Center, provides leading-edge
treatment for children, adolescents and select young adults with all types and stages of CNS tumors. The Neuro-
Oncology program participates in national and international research studies and clinical trials, providing the most
current available therapies. Our team includes board certified pediatric neurologists, neurosurgeons and oncologists
who represent some of the top experts in clinical care and research.
Cook Children’s dedicated social workers and nursing staff are available to provide emotional support to families
as their child undergoes treatment. In addition, social workers can assist families in locating community resources
and address concerns that may arise including financial issues, transportation, lodging and educational needs. The
Ronald McDonald House and several hotels with reduced medical rates are available near the medical center and
offer free shuttle service to the medical center.
The Neuro-Oncology program is seamlessly joined with our Life After Cancer program, which is staffed by experienced
physicians who continue to monitor children treated for CNS tumors long after the treatments have been completed.
Jeffrey C. Murray, M.D.
Medical Director, Neuro-Oncology,
Hematology and Oncology Center
A Collaborative Effort
The optimal treatments for CNS neoplasms are developed by an integrated team of specialists as part of Cook Children’s
advanced Neuro-Oncology program. Our team uses the best-available neurosurgical and radiotherapeutic techniques
and equipment, proper chemotherapy regimens, along with access to leading-edge clinical trials.
Cook Children’s Neuro-Oncology program is staffed by the following team members:
• Pediatric Neuro-Oncologist
• Pediatric Neurologists
• Pediatric Neurosurgeons
• Pediatric Radiation Oncologists
• Pediatric Pathologists
• Pediatric Neuroradiologists
• Pediatric Neuropsychologists
• Pediatric Nurses
• Pediatric Endocrinologists
• Pediatric Nurse Practitioners
• Pediatric Social Services
• Child Life Specialists
• Life After Cancer
Program Specialists
Hematology and Oncology 23

There are many different types of CNS tumors in children and
adolescents. Distinct histologic patterns and neuroimaging, varied
constellations of presenting signs and association with several
genetic syndromes make the CNS tumors an extremely complex
group of neoplasms.
The more common types of childhood CNS tumors are:
• Low-grade gliomas (pilocytic astrocytoma, infiltrative
astrocytoma)
• High-grade gliomas (anaplastic astrocytoma, glioblastoma
multiforme)
• Medulloblastoma
• Ependymoma
• Germ cell tumors
• Optic pathway tumor (glioma)
• Intrinsic brainstem glioma
• Pineoblastoma
• Supratentorial primitive neuroectodermal tumors
• Choroid plexus papilloma and carcinoma
• Spinal cord glioma
• Spinal cord ependymoma
Most CNS tumors in children are low-grade gliomas
(e.g., astrocytoma) and primitive neuroectodermal tumors
(e.g., medulloblastoma), whereas in adults, meningioma and
high-grade gliomas (glioblastoma multiforme) predominate.
The diagnosis of CNS tumors in children involves a thorough
history and physical examination, computed tomography,
magnetic resonance imaging and surgical biopsy or tumor
resection. The complexity of healthy neural tissue and the varied
pathologic changes that can occur with neoplasia make accurate
neuropathologic diagnoses challenging.
Moreover, the therapy for children with CNS tumors is very
complicated, based on:
• Immaturity of the child’s CNS
• Propensity for some tumors to spread via the
cerebrospinal fluid
• Inability to neurosurgically remove some tumors
• Blood-brain barrier
• Long-term side effects of therapy: growth, neurocognitive
development, etc.
• Inability to use radiation therapy in very young children,
based on age and tumor location
• Underlying genetic conditions (e.g., neurofibromatosis)
The general principles of therapy for pediatric CNS tumors
involve one of several modalities or a combination thereof:
• Neurosurgery (biopsy or resection of tumor)
• Chemotherapy (many different drugs)
• External beam radiation therapy (different techniques)
• Stereotactic radiosurgery (e.g., CyberKnife ® ,
GammaKnife ® )
The combined five-year survival rate for childhood CNS tumors
has increased slowly over the past decades, from 55 percent
to approximately 60 percent. However, for some tumors (e.g.,
intrinsic brainstem glioma, atypical teratoid/rhabdoid tumor and
glioblastoma multiforme), long-term survival rates remain well
below 20 percent.
Table 1, on the following page, summarizes the 262 children and
adolescents with CNS tumors cared for by the Cook Children’s
Neuro-Oncology program from 1997-2007.
The distribution of CNS tumors seen in the Neuro-Oncology
program during the last 11 years has been consistent with
the pattern seen across the United States. Glial neoplasms,
including the most common astrocytoma histology (as
well as ependymoma, a type of glial tumor) and primitive
neuroectodermal tumors (medulloblastoma and supratentorial
PNET) represent the majority. Of note, meningiomas and highgrade
gliomas (e.g., W.H.O. grade IV, or glioblastoma multiforme),
the types typically seen in adults, were rarely seen.
During the time period of these analyses, researchers witnessed
changes in neurosurgery technologies, radiotherapeutic techniques
and changes in clinical trials. Hence, precise survival statistics are
not comparable from year-to-year. Regardless, the overall survival
results of all histologic categories are remarkably good, meeting
and in some cases exceeding reported national results.
Compared to other childhood cancers, national progress
in the cure of CNS tumors has not been as dramatic. This
reflects the complex and diverse nature of CNS tumors and the
limitations in the ability to uniformly apply ‘aggressive therapy’
to the child’s immature brain. Fortunately, relatively recent
advents in the understanding of tumor cell genetics, enhanced
neuroimaging techniques, neurosurgical capabilities, novel
radiotherapy approaches (e.g., proton beam therapy) and unique
chemotherapy strategies promise significant progress in the
coming years.
continued on page 27
24 Hematology and Oncology

Cook Children’s Neuro-Oncology program’s CNS tumor cases (1997-2007)
CNS tumor Total Percent living
Low-grade glioma (JPA) (a) 51 98
Other low-grade gliomas (b) 47 87
Brainstem glioma 37 41
Medulloblastoma 36 83
Ependymoma 13 69
Supratentorial PNET (c) 8 38
High-grade gliomas (d) 7 43
Craniopharyngioma 7 86
Meningioma 6 83
AT/RT (e) 6 50
Choroid plexus tumors (f) 5 100
Germ cell tumors (g) 4 100
Miscellaneous brain tumors (h) 24 N/A
Spinal cord tumors (i) 11 64
All CNS tumors 262
(a) World Health Organization (W.H.O.) grade I glioma = juvenile pilocytic astrocytoma
(b) Other W.H.O. grades I and II gliomas (e.g., fibrillary astrocytoma)
(c) Supratentorial primitive neuroectodermal tumors (e.g., pineoblastoma)
(d) W.H.O. grades III and IV gliomas (e.g., glioblastoma multiforme)
(e) Atypical teratoid/rhabdoid tumors (rare tumor of infants and young children)
(f) Choroid plexus papilloma and choroids plexus carcinoma
(g) (Intracranial) germinoma and non-germinomatous germ cell tumors
(h) Ganglioglioma, astroblastoma, others
(i) Astrocytoma, ependymoma, others
Hematology and Oncology 25

Dual room Intraoperative MRI
The Cook Children’s Hematology and Oncology Center and
Neurosciences are committed to offering premier neuro-oncology
care as the sub-specialty advances in the years to come.
This commitment is evidenced by our investment in the latest
neuroimaging/neurosurgery technology, the iMRIS dual room
intraoperative MRI. Cook Children’s is one of only a handful of
children’s hospitals in the United States to house this technology.
One component of the iMRI suite is the diagnostic imaging area;
the second part is the surgical area, where imaging can be
performed during the surgical procedure. The iMRI increases
accuracy in complex neurosurgical procedures, allowing
neurosurgeons to detect any residual tumor in patients during the
operation, which may prevent the need for a second surgery.
The Neuro-Oncology program continues to be committed to
advancement. The program includes a full-time pediatric neurooncologist
and three pediatric neurosurgeons, plus ongoing
participation in Children’s Oncology Group clinical trials and
interinstitutional consortiums in Texas. The next decade of care
for children, adolescents and young adults with CNS tumors at
Cook Children’s promises innovative and leading-edge treatment.
Cook Children’s
houses the revolutionary
intraoperative MRI (iMRI).
The IMRIS technology, of which
Cook Children’s is one of a small
handful of children’s hospitals in
the country to acquire, provides
real-time intraoperative
images.
26 Hematology and Oncology

Radiation Therapy
Radiation is essential in a child’s fight against many types of
cancer. Cook Children’s Hematology and Oncology Center has
maintained a close relationship with Texas Oncology to ensure
that patients have access to this important form of therapy.
Through this relationship, Cook Children’s offers specialized
types of radiation therapy including:
• Total body irradiation (TBI), which is the use of radiation
therapy to delete a patient’s bone marrow and suppress the
immune system prior to stem cell transplantation, treating the
malignancy and preventing the rejection of donor stem cells.
• Radiation therapy for solid tumors, including Wilms’ tumor,
Hodgkin’s disease and soft tissue sarcomas. Radiation therapy
is used to treat involved areas of a patient’s body before, after
or instead of surgical resection of malignant tumors.
• Stereotactic radiosurgery is an advanced technology that
delivers precise radiation beams that target tumors sparing
surrounding normal tissue. This targeted radiation treatment
is invaluable in treating children and minimizing late effects of
radiation therapy.
On average, Texas Oncology is involved with treating
approximately 60 children each year with pediatric malignancies.
These patients range from age 11 months to 21 years and many of
the patients are enrolled in Children’s Oncology Group protocols.
The radiation oncologists work closely with Cook Children’s
staff to determine and provide treatment for each patient. They
participate in the weekly tumor boards and brain tumor clinics at
Cook Children’s and lead a team of oncology nurses, physicists,
dosimetrists, radiation therapists and support staff at Texas
Oncology. The center has three linear accelerators and a CT
scanner for treatment simulation and complex treatment planning.
Texas Oncology is part of the nation’s US Oncology Cancer Care
Network. Its relationship with Cook Children’s Hematology and
Oncology Center reflects a shared commitment to the treatment
of children with cancer.
Hematology and Oncology 27

28 Hematology and Oncology

Neuroblastoma Program
Cook Children’s Neuroblastoma program strives to grow
and offer new approaches to therapy for this complex
patient population.
discovery of
treatment
Neuroblastoma is a cancer of the sympathetic nervous system and can occur in almost any region of the body. It
is the most common extracranial solid tumor in children, with about 600 children diagnosed annually in the United
States. The most frequent presenting location is an adrenal mass. The cause of neuroblastoma is unknown. Initial
symptoms are often vague, and may include fatigue and loss of appetite, but can also include symptoms at locations
where the tumor has spread. A tumor in the abdomen may cause swelling, constipation and pain. A tumor in the chest
may cause breathing problems. Tumors pressing on the spinal cord cause a feeling of weakness, difficulty walking
or bladder dysfunction. Unfortunately, half of all neuroblastomas will spread to other parts of the body by the time
suspicions are raised and a diagnosis is made. In some cases this is a factor of time; in others, it may be due to the
inherit biology of the tumor cells themselves.
Because neuroblastoma’s symptoms are vague and can be similar to symptoms of other more common diseases,
there is often a delay in making the diagnosis. A biopsy of the tumor and the results of urine and bone marrow tests
are necessary to make the diagnosis. The experience of a pediatric pathologist in reviewing the biopsy material is
essential and masses suspicious for cancer should always be biopsied at a tertiary care center.
Screening is possible by looking for elevation of urine catecholamines. However, large population studies which
employed neonatal screening for neuroblastoma did not show improvement in outcome because many infants may
have tumors that regress or do well with limited therapy.
Neuroblastomas are highly diverse in their behavior. Some will go away without any treatment and others can be
cured by surgery alone. However, half of these tumors spread quickly to other parts of the body, including bone
and bone marrow, and require intense multimodality therapy such as chemotherapy, radiation therapy, stem cell
transplantation and targeted or biologic therapy. The right treatment for each child depends mainly on the child’s age,
the tumor location, where the tumor has spread and the biology of the tumor. Intensive induction chemotherapy has
been shown to be successful in helping achieve complete response in patients with metastatic disease. Most children
with neuroblastoma will undergo surgery at some point during their treatment, either before or after chemotherapy.
High dose chemotherapy and stem cell rescue is used to consolidate good responses after induction chemotherapy
and surgery. This therapy is effective against tumor cells that may have developed drug resistant patterns. Radiation
therapy is used to kill cancer cells that remain after surgery and chemotherapy; this is usually delivered to the site of
the primary tumor and any remaining bone lesions.
continued on page 31
Meaghan Granger, M.D.
Hematology and Oncology Center
Hematology and Oncology 29

A heart felt thanks
Cook Children’s Neuroblastoma program is
grateful for the tremendous financial support
received from a number of reputable organizations
and groups. The St. Baldrick’s Foundation has
awarded $50,000 for the development of the
Neuroblastoma program, which will increase
necessary personnel support as we offer more
clinical trials and complex therapies. Community
members of Fort Worth organized “A Walk for
the Cure” which raised almost $20,000 in its first
year with plans to become an annual fundraising
event. The Children’s Oncology Group (COG) will
continue to provide partial funding for clinical trials
through National Cancer Institute (NCI) funds and
grants from the St. Baldrick’s Foundation. Thanks
to this support Cook Children’s Neuroblastoma
program can continue to grow and offer new
approaches to therapy for this complex patient
population.
Cook Children’s
Neuroblastoma program
was selected to become a
member of the New Approaches
to Neuroblastoma Therapy (NANT)
consortium because of its record
of clinical care and commitment
to clinical research and
clinical trials.
30 Hematology and Oncology

Neuroblastoma Program (continued)
Neuroblastoma is the most common type of extracranial solid
tumor in children and can be one of the most aggressive forms
of pediatric cancer. Unfortunately, in these cases, most children
succumb to the disease. To prevent such a devastating outcome,
changes in strategies to combat this deadly disease are needed.
On average, approximately 20 patients are diagnosed with
neuroblastoma annually at Cook Children’s. Their demographics
are spread broadly over all of North and West Texas, and
surrounding states. We have a growing number of therapeutic
options for patients with newly diagnosed and relapsed disease,
and are pleased to offer leading-edge treatment in the area of
clinical trials, surgery and neurosurgery. Additionally, superb
supportive care is available for the patient and family.
In the past year, Cook Children’s Neuroblastoma program
has experienced tremendous growth. We were selected to
become a member of the New Approaches to Neuroblastoma
Therapy (NANT) consortium due to our record of clinical care
and commitment to clinical research and clinical trials. NANT is
a group of 15 hospitals and universities strongly committed to
neuroblastoma research in patients with relapsed or refractory
disease, cases where new agents are most needed. Through our
collaboration with NANT, we are currently enrolling patients in
new trials focused on pursuing these agents, and thereby offering
our patients the leading edge of care.
These trials are unique in that they specifically target cancer
cells. Targeted therapy is aimed at disrupting cancer cell growth
and comes in a variety of forms. It is often used to specifically
regulate neuroblastoma cells that remain after multimodality
intensive therapy. Some of our trials use a type of “targeted
radiation” based on an I 13I MIBG (metaiodobenzylguanidine)
compound, which delivers radiation directly to cancer cells.
Cook Children’s is delighted to announce the construction of a
leading-edge, patient-and family-centered MIBG facility expected
in 2011. Additional types of targeted therapy may include
retinoids, such as retinoic acid, small molecules or types of
immunotherapy, which uses an antibody and the body’s immune
system (including white blood cells) to fight the cancer.
Cook Children’s continues to be a very active member of the
Children’s Oncology Group (COG) and is involved in clinical trial
design and conduct of research. We have trials available for low-,
intermediate-, and high-risk patients. We have been privileged to
see advances made in the survival of high-risk patients through
these trials using monoclonal antibody targeting neuroblastoma
cells. Cook Children’s was pleased to be one of the few
participating sites in Texas and around the country to offer this
type of treatment.
Our neuroblastoma team, comprised of a clinical, surgical and
research team, is able to meet the many needs of patients with
neuroblastoma and their family. Our clinical team is headed
by Meaghan Granger, M.D., a pediatric oncologist with special
emphasis in neuroblastoma. Our surgical team is headed by
Jim Miller, M.D., a technically proficient surgeon with extensive
neuroblastoma experience. We have a dedicated clinical
research associate, neuroblastoma research interns and other
research professionals. Our RN coordinator, Tracey Easley, acts
as a liaison between the clinical team and research members and
works with families during the referral process. We look forward
to providing hope to patients and their families through the
continued growth of our Neuroblastoma program as we continue
to pursue new advances for patients with this disease.
Hematology and Oncology 31

32 Hematology and Oncology

Acute Myeloid Leukemia Update
Cook Children’s Leukemia and Lymphoma program
partnered with St. Jude Children’s Research
Hospital and a small number of other institutions
in conducting an innovative clinical trial, known as
AML 2002.
leading
research
The strategy of this protocol is to adapt the intensity and
components of therapy to risk factors recognized through
molecular genetic analysis and response to therapy. A
total of five courses of chemotherapy are given beginning
with the standard combination of ara-C (Cytarabine),
daunomycin and etoposide (ADE). A randomization
was included, which compared standard versus high
dose ara-C (Cytarabine) (results not yet complete for
presentation). In contrast to the contemporary study of
the Children’s Oncology Group (COG), which offers a
new agent, gemtuzumab ozogamicin, on a randomized
basis, this agent was offered selectively to patients
with poor response as defined by minimal residual
disease (MRD). The protocol was modified to reduce
the threshold for poor response definition from 5 percent
down to 1 percent blast cells. Bone marrow specimens
with companion blood samples were sent to St. Jude for
MRD analysis at the end of induction and prior to each
course of consolidation therapy.
Patients are further classified according to criteria
defining three categories of risk. These are outlined in
Figure 2 on page 34.
W. Paul Bowman, M.D.
Chairman of Cook Children’s Leukemia and Lymphoma
program, Medical Director, Academic Pediatrics,
Hematology and Oncology Center
Research Efforts
The Cook Children’s experience with acute myeloid leukemia (AML) was first presented in the Cook Children’s
Cancer Program Annual Report 1995-1996 by Timothy Griffin, M.D. At that time, Cook Children’s experience
of cases diagnosed between 1982 and 1995, totaling 46, was summarized. Complete remission was achieved
by 36, or 80 percent, with overall long-term survival of 17, or 37 percent. These results were consistent with
national experience and those of contemporary clinical trials. We are working diligently to enhance and continually
improve results.
Hematology and Oncology 33

AML 2002 Risk Assignment
Low Risk
Presence of t(8;21)/AML1-ETO, inv(16)/CBFβ-MYH11, or t(9;11)/MLL-AF9.
Standard Risk
High Risk
Absence of low-risk or high-risk features or the presence of both a high-risk and a low-risk feature.
Presence of one of the following: monosomy 7, t(6;9), FAB M6 or M7 morphology, treatmentrelated
AML, AML arising from prior MDS, FLT3 ITD or RAEB-T. In addition, patients who have no
response to Induction I or who do not achieve CR after two courses of induction therapy will be
considered high-risk.
Table 1
During the conduct of the protocol initially, an early problem was
the emergence of isolated central nervous system (CNS) relapse.
Intrathecal therapy was modified from the single adjacent ara-C
(cytarabine) to triple intrathecal therapy that include methotrexate
and hydrocortisone along with ara-C. The emergence of CNS
relapse subsided with the addition of triple intrathecal therapy.
From 2002 to 2008, 29 consecutively diagnosed patients
with AML have been registered on the AML 2002 trial at
Cook Children’s and the data presented here represents our
institution’s experience on this trial. Our results indicate a
considerable improvement in outcome for this group of patients
compared to that reported in the mid 1990s, with survival rates
now more than 70 percent (Figure 1). Complete remission has
been achieved by all patients after the risk stratified induction
chemotherapy. In contrast to previous protocols, they were
then separated into three categories of risk for post-induction
management. The low risk group (see Figure 2) had a uniformly
successful outcome with intensive chemotherapy alone.
However, the standard and high-risk groups have not fared as
well. An important observation which has emerged recently is
the dire negative prognostic implications for leukemia cells that
harbor the FLT-3 internal tandem mutation. Of the five such
patients in our AML 2002 series, only one presently survives.
The AML 2002 protocol has further clarified the important
influence of biological factors on response to treatment and
durability of remission. Considerable challenges exist to better
balance risks versus benefits of intensive chemotherapy
treatment and to improve outcomes for those in the standard
and high-risk categories through chemotherapeutic and/or
transplant related innovations. Such plans were introduced in
the following AML 2008 trial, the second in this series, in which
Cook Children’s Hematology and Oncology Center participates.
AML ’02 Overall Survival
Figure 1 Figure 2
AML ’02 Overall Survival by Risk Category
100
100
Cumulative
Cumulative
Percent
Percent
Survival
Survival
80
60
40
20
Cumulative
Cumulative
Percent
Percent
Survival
Survival
80
60
40
20
0
0
0 20 40 60 80
0 20 40 60 80
Months Since Diagnosis
Months Since Diagnosis
Groups
Survival Function
Censored
Risk
High
Low
Standard
High-censored
Low-censored
Standard-censored
34 Hematology and Oncology

Cook Children’s
Hematology and Oncology
Clinic-Grapevine location is one
of the few freestanding pediatric
Hematology and Oncology clinics
in the nation with its own
pediatric pharmacy.
The
Cook Children’s
Hematology and Oncology
Clinic-Grapevine has an infusion
clinic with a dedicated full-time
pharmacist to prepare chemotherapy
compounds.The USP797 compliant
pharmacy provides a controlled
environmentfor sterile preparation
of drugs ensuring the highest level
of quality assurance.
Hematology and Oncology 35

36 Hematology and Oncology

Support Services
Illness can be stressful for children and their family
members, so Cook Children’s offers a variety of ways
to meet the emotional, psychological and spiritual
needs of our patients and families.
familyand
patientcentered
care
Cook Children’s Support Services include Child Life specialists who provide therapeutic, educational and
recreational activities, licensed play therapists, psychologists and social workers, as well as Pastoral Care
services dedicated to caring for the hematology, oncology and stem cell transplant patients. Translation
services are also provided for non-English speaking patients and families.
Other means of support are offered by providing patients and siblings an opportunity to attend medical
camps. Camp Sanguinity, a camp for patients and patient siblings with an oncology diagnosis, Camp
Jubilee, for patients with a diagnosis of sickle cell disease, and Camp Ailihpomeh, for patients diagnosed
with hemophilia, are just three of the medical camps offered. Other support services include Celebrate Life,
an annual reunion for cancer survivors, Candlelighters Childhood Cancer Foundation of Greater Fort Worth,
a family support group, and an annual Remembrance Ceremony for those families that have lost a child. We
also have a Ronald McDonald House, which offers a home away from home for families in treatment.
Additionally, educational support is offered by two full-time RN educators who provide disease-specific
information to patients and families. The educators also provide group, as well as one-on-one, training on
how to care for the patient’s central line prior to discharge.
Adolescent Oncology program
The Adolescent Oncology program recognizes the special needs of teenagers and is designed to address
their concerns throughout cancer treatment. Teenagers diagnosed with cancer face emotional and
psychological challenges regarding changes in appearance, school activities and interactions with friends.
The goal of Cook Children’s teen support group is to help the patient maintain as much normalcy as
possible as they undergo treatment for cancer.
Orthopedic Oncology program
Children and adolescents with tumors of the bones or soft tissues of the extremities represent a
relatively small, but uniquely challenging population of oncology patients. The Orthopedic Oncology
program at Cook Children’s combines orthopedic surgery and oncology expertise in a comprehensive,
multidisciplinary team approach to care for children with cancers such as osteogenic sarcoma, Ewing’s
sarcoma, peripheral primitive neuroectodermal tumor and rhabdomyosarcoma. The team uses National
Cancer Institute-sponsored clinical trial chemotherapy protocols and special surgical techniques aimed
at limb preservation (non-amputation local tumor control). Orthopedic oncology specialists also care for
children with benign tumors or diseases that affect bones and the skeletal system.
Hematology and Oncology 37

Palliative Care program
Life After Cancer program
In the last 20 years, more than 1,500 cancer survivors have been
treated at Cook Children’s. Despite the positive outcome, cancer
survivors today still experience a range of physical and emotional
issues, educational handicaps and fear of recurrence or second
cancers. The Life After Cancer program (LACP) at Cook Children’s
works to benefit children and young adults by providing medical
care and other services for childhood, adolescent and young adult
cancer survivors. The program was the first of its kind funded by
the Lance Armstrong Foundation and serves as a model for other
survivorship programs.
Services provided through the Life After Cancer program
include:
• Comprehensive medical and psychosocial assessments by
experts in survivorship
• Consultants in survivorship, including endocrinology, neurology,
pulmonology, cardiology, orthopedics and other onsite subspecialists
• Complete review of treatment and summary
• Educational and vocational counseling
• Neuropsychological testing and counseling
LACP Support Services:
• Camp O’Hana– a family/survivor weekend retreat
• Project Survivor– adult and young adult survivor group which
meets monthly; facilitated by a social worker who is an adult
cancer survivor
• Project Survivor Retreat– for adult and young adult survivors of
childhood cancer
LACP Initiatives:
• Reproductive counseling when requested
• Fertility preservation
• Research related to cancer survivorship
• Health practices of the adult and young adult population
• Quality of life studies for cancer survivors and their families
• Other behavioral health research initiatives
Cook Children’s has joined the ranks of other national, elite
pediatric hospitals in developing a Palliative Care program.
Palliative Care is a holistic multidisciplinary team approach to
the relief of suffering in all its dimensions — physical, emotional,
psychosocial and spiritual. The program focuses on enhancing
quality of life and support for children living with life-threatening
conditions and their families. The program espouses an
integrative approach to care. Components of Palliative Care may
be offered at the time of diagnosis and continued throughout the
course of the patient’s illness, in conjunction with curative and/or
life prolonging treatments.
The Cook Children’s Palliative Care team:
• Facilitates collaboration, coordination and continuity of care
and communication across care settings.
• Acknowledges the practical concerns of the child/family and
helps to access important resources and services that address
those unmet needs.
• Assists with care conferences and family meetings.
• Helps guide open, honest communication and difficult
conversations between care givers, family members, and
(when appropriate) the ill child or young adult.
• Attends to the emotional effects of the child’s illness on the
family and to the special needs of siblings. This includes
attending to the family’s grief and bereavement needs.
• Provides timely information and support in decision making
regarding quality of life concerns and treatment decisions.
• Appreciates the role of spirituality in finding meaning, providing
guidance in making difficult medical decisions and in coping
with the child’s illness.
• Helps children and families establish goals of care based on
their values, beliefs, preferences and framework of meaning.
For children and families faced with uncertainty and/or life
limiting conditions, this includes help establishing goals of care
for their child’s end-of-life care to ensure comfort, dignity and
integrity.
• Supports the primary care team with managing distressing
symptoms.
The Palliative Care team appreciates the qualities of care
that make the provision of pediatric Palliative Care so unique.
These qualities include important factors such as the resiliency
of children and the uncertainty in predicting prognosis, the
varying developmental levels in children and their capacity to
comprehend their illness, and the focus on family-centered care –
with parents acting as surrogates and being active participants in
shared decision making with the child’s medical team.
The Palliative Care program under the direction of Todd
Pearson, M.D., has developed a collaborative interdisciplinary
team that includes a nurse practitioner, Social Work, Pastoral
Care, Psychology, Pharmacy, Pain Management and Child Life
specialists. The Palliative Care team often becomes involved
early – at the time of diagnosis — in order to know the child and
family and to help plan for any future concerns. An integrated
model of Palliative Care can be of benefit and service to a patient
and family pursuing curative and life-prolonging treatment.
38 Hematology and Oncology

What makes
Cook Children’s
Hematology and Oncology
program different is
the focus on patientand
family-centered
care.
Happy Campers - Camp Sanguinity marks 30th anniversary
This year marked the 30th anniversary of Camp Sanguinity, for
Cook Children’s patients with cancer and blood disorders and
their siblings, which takes place at Camp John Marc in Bosque
County, Texas. This also is the final year for Asha Anderson to
attend as a camper. But it will not end her relationship with the
camp that has meant so much to her since she began going
seven years ago after being diagnosed with leukemia at 5 and
receiving a bone marrow transplant at 7.
Asha still remembers her first day at camp and how afraid she
was of the rope course. She wasn’t sure if she could climb the wall
and then take the zip line down to the ground. “The first time I was
really scared,” Anderson said. “They told me don’t look down. So
naturally I looked down. I freaked and climbed down.”
Now it’s what she looks forward to the most. “I just want to keep
doing it.”
“I think about all the things we’ve done,” Anderson said. “Some
memories are good. Some are sad. The first day we always light a
candle for everyone who’s died and that’s really sad.”
Like Asha, Scott Odom remembers being scared his first time at
camp. He said it took quite a bit of convincing for him to even go.
He was self-conscious about losing his hair and had just recently
had his leg amputated. Odom has now been at camp for nine
consecutive years as either a camper or in his current role of
counselor.
“But I came out and it basically changed my life,” Odom said. “I
came out with no self-esteem and very down on myself and then
at the end of the week, I didn’t want to leave. I was sad to go
because out here the kids get to have fun and there is none of the
negativity. At home, I was the guy that had cancer or the kid with
one leg, but here I just got to be Scott.”
“One time out and I was hooked.”
For campers, Camp Sanguinity is an opportunity to spend time
with others who do not have to be told how something feels or
what it is like. They know.
“These kids understand each other. They have been there. They
all have someone here with the same problems whether it is
baldness or scars or easily fatigued or just looking different from
surgery or disease,” said Jill Koss, director of Cook Children’s
Family Services.
That understanding is what brings many former campers back as
adults. Ashley Cook was a camper from 6 to 11 and has been a
counselor for five years. “I love working with kids. I know what they
are going through and feel like I can connect with them,” said Cook.
Along with the emotional support offered by the staff, patients
are treated by physicians, nurses and clinical staff from
Cook Children’s.
Like Koss, Joann Sanders, M.D., a physician in the Hematology
and Oncology Center at Cook Children’s, volunteers her time at
Camp Sanguinity and finds great reward while doing so.
“These are kids we don’t see smile a lot in the hospital. So it’s
great for us. But for them, it’s something they take home with
them and they keep that going through the year,” Sanders said.
Camp Sanguinity is unique from the standpoint that it allows not
only patients to go, but their siblings as well. Veronica Kubiak was
a sibling camper for two years and has been a counselor since
2002. She said camp changed her perspective.
“The first year it was more shocking than anything to see how
many kids were sick, but the biggest thing was knowing that it
was OK,” she said. “Your feelings were OK and being afraid was
OK. It can be traumatizing just watching your sister or brother go
through this, so to be in a big group going through the same thing
is comforting.”
Hematology and Oncology 39

40 Hematology and Oncology

Cancer Registry Operations
Cook Children’s Medical Center maintains an accurate and ongoing database of children and
young adults diagnosed with cancer. The registry data is reported to the Texas Department
advancements
of Health, Cancer Registry Division and the National Cancer Institute’s, Physician Data Query ®
(PDQ ® ).
Since the program’s accession date of Jan. 1992-Dec. 2008, 2,136 cases have been entered into the
registry.
The cancer registry data includes demographic information, cancer identification, treatment and staging and yearly follow
up. Data from the registry is used to aid in research studies, patient case conferences/review and quality improvement
initiatives for Cook Children’s Hematology and Oncology Center.
2008
181
2007
165
2006
163
2005
146
2004
139
2003
152
Year
2002
2001
2000
141
138
158
1999
117
1998
110
1997
111
1996
105
1995
78
1994
84
1993
92
1992
56
0 20
40 60 80 100 120 140 160 180 200
registry data
Cook Children’s Cancer Committee
The Cook Children’s Cancer Committee is a multidisciplinary team composed of physicians and non-physicians
meeting quarterly to ensure the quality of the cancer registry data and the cancer program. The cancer committee
develops and evaluates program goals and objectives related to cancer care, promotes clinical research and
patient management through an educational and consultative case conference format. In addition, the Cancer
Committee oversees programs in which hematology and oncology patients are involved while receiving treatment
at Cook Children’s.
Hematology and Oncology 41

42 Hematology and Oncology

Quality Improvement Initiatives
Cook Children’s Hematology and Oncology Center
utilizes integrated processes for quality assurance,
monitoring and improvement to evaluate outcomes,
determine accuracy and reliability and identify
opportunities for improvement.
high-quality
The Plan Do Study Act (PDSA) model is utilized for the identification and documentation of our quality program.
Our process improvement initiatives are identified in collaboration with the medical director, physician staff, program
director, management team, quality RN coordinator and team members/staff involved in patient care.
The following is a listing of quality improvement projects from 2005-2009
• Documentation of start and stop times of high-risk drugs (i.e., chemotherapy) during preparative regimens during
stem cell transplant.
• Implementation of Alaris ® Smart Pumps: compliance with the use of guardrails on defined high-risk medications.
• Implementation of enteral nutrition via feeding tube in acute stem cell transplant patients.
• Implementation of primary nursing model in stem cell transplantation.
• Documentation of appropriate work-up indicators for autologous and allogeneic transplant patients.
• Rounding for success: concepts from Quint Studer’s “Hardwiring Excellence.”
• Implementation of monthly tracer activities to validate ongoing readiness for The Joint Commission survey and to
monitor compliance with National Patient Safety Goals.
• Relocation of patients undergoing diagnostic procedures requiring sedation to special procedure area to facilitate
flow of patients through the outpatient clinic and provide a safer environment for the patient.
• Implementation of Lean Process Improvement in the outpatient clinic to enhance patient safety and flow through
the outpatient clinic.
• Monitoring of blood stream infections in the inpatient and outpatient settings.
Hematology and Oncology 43

44 Hematology and Oncology

Hematology Services
Cook Children’s Hematology services directs the care
of children and adolescents with diseases of the blood
that are non-malignant.
leading-edge
therapy and
research
Due to the fact that the blood system and clotting system are so complex, there is a broad range of disorders
and diseases evaluated and treated by our hematology physicians. These include many types of anemia,
neutropenia and thrombocytopenia, thrombocytosis, bone marrow failure syndromes, hemoglobinopathies,
as well as hemophilia and other inherited or acquired bleeding disorders. Our team also cares for an evergrowing
population of patients with thrombophilic disorders. Each patient is provided individualized treatments
and guidelines to maximize healthy participation in school and minimize hospitalization. The team includes four
physicians, a nurse practitioner, dedicated nursing staff, a hematology data coordinator and other dedicated
ancillary services such as Child Life, Social Work, Physical Therapy and Neuropsychology.
Program Highlights
• Programs to treat patients with sickle cell disease, hemophilia and von Willebrand disease, as well as those
with history of strokes
• Dedication to clinical research – participation in 16 clinical research protocols or disease registries in the last
four years; presentation by both physicians and nursing staff at national meetings
• Many family education and support programs
• Outreach clinics throughout Texas – Grapevine, Waco, Midland, Odessa, Abilene, San Angelo – and the
capability to provide telemedicine services
• Hemophilia Twinning program with Lima, Peru
• Camp Ailihpomeh for hemophilia patients and Camp Jubilee for sickle cell disease patients
Marcella Torres, M.D.
Medical Director, Hematology Services,
Hematology and Oncology Center
Hematology and Oncology 45

Patients seen in the Hematology and Oncology Center Jan. 2008 – April 2009
Bleeding Disorders (hemophilia, von Willebrand disease, others)..............................................250
Neutropenia..............................................................................................................................100
Stroke..........................................................................................................................................75
Thrombophilia Disorders............................................................................................................30
Sickle Cell Disease....................................................................................................................275
46 Hematology and Oncology

Sickle Cell Comprehensive program
Sickle cell disease is a chronic blood disease that affects about
70,000 people nationwide. Cook Children’s currently treats
275 children with this hemoglobinopathy from Tarrant County,
Texas, as well as many areas of North and West Texas. The
comprehensive program provides new diagnosis evaluations,
genetic counseling and individualized treatment plans on an
ongoing basis. The team collaborates with the Sickle Cell Disease
Association of America to host important family and patient
support groups, as well as education events. Collaborative efforts
also extend to regional academic partners including recent
initiatives with Texas Children’s Hospital in Houston regarding
care of patients with sickle cell disease and other hematologic
disorders.
Comprehensive Hemophilia Treatment
Center
The Cook Children’s Comprehensive Hemophilia Treatment
Center is part of a nationwide, federally funded network that
specializes in the diagnosis, evaluation and treatment of
hemophilia, von Willebrand disease and other bleeding disorders.
Cook Children’s program is affiliated with the Center for Disease
Control’s Region VI, which includes Texas, Oklahoma, Louisiana
and Arkansas. The table to the right illustrates the number of
patients followed in our comprehensive hemophilia treatment
center by specific diagnosis.
Publications
Immune Tolerance Therapy Utilizing Factor VIII/von Willebrand
Factor Concentrate in Hemophilia A patients with High Titer Factor
VIII Inhibitors. Kurth M.A.H, DiMichelle D., Sexauer C., Sanders
J.M., Torres M., Zappa S.C., Ragni M., Leaonard N. Hemophilia.
2008 Jan:14(1):50-5. Epub 2007 Oct. 18.
Isolation of Oligodendrocyte-like Cells from Human Umbilical Cord
Blood. Tracy E., Aldrink J., Panosian J., Beam D., Thacker J.,
Reese M., Kurtzberg J. Cytotherapy, 2008 Jun. 3:1-8.
Internal Exchange, WFH’s Twinning Program Improves Care
Worldwide, Hemaware Sept./Oct. 2008. Featured – Susan Zappa,
RN.
Hemophilia Thrombosis Research Society President’s Message.
Featured – 2008 HTC “Twin of the Year” between Fort Worth and
Lima, Peru.
Diagnosis
Number of Patients
as of March 2008
Hemophilia A 75
Hemophilia B 14
von Willebrand disease 114
Platelet function defect 25
Rare bleeding disorders
(i.e., Glanzmann’s, thrombasthenia,
factor XI, factor II, factor VII, factor X,
afibrinogenemia, etc.)
Special Hemophilia Center
International Project
The Cook Children’s Lima–Fort Worth Twinning program
was named the 2006 Twin of the Year Award by the World
Federation of Hemophilia.
This special program developed in 2004 paired a hemophilia
treatment center in a developed country with a center in
a developing country. The goal for the “twin centers” is
collaboration through visits, educational presentations,
shared supplies and clinical care to provide the best
support, treatments and education to patients and families in
underserved areas.
Many great initiatives have resulted from this innovative
partnership:
• Increased education and awareness regarding hemophilia to
medical staff
• Designed multi-discipline team to care for hemophilia
patients in a comprehensive manner
• Medical treatments updated and incorporated into standards
of care
• Better relationships established with the Health Ministry in
Peru and a contract with the Peruvian government to provide
more hemophilia treatment factor to the hospital for patients
• Increased education for families including psychosocial
component
• A Young Adult Leadership program for 18-30 year old
patients to take control of their situation and become the next
leaders of the patient association
• The first camp for patients in Peru
26
Hematology and Oncology 47

48 Hematology and Oncology

esearch
initiatives
Hematology and Oncology Research
If you look at the value of the years you can save by curing a pediatric patient, you can easily see the value of pediatric
cancer research. Cook Children’s is not a traditional academic center tied to a university, but we are academic in spirit in
how we practice day in and day out.
Research Studies, 2005-2007
Hematology
• Epidemiology and Treatment of Circulating
Anticoagulants in Patients with Hemophilia and von
Willebrand Disease The HTRS Registry
• A Retrospective Study to Describe the Incidence of
Moderate to Severe Allergic Reactions to Factor IX in
Patients with Hemophilia B
• Examining The Immune Tolerance Induction in Patients
with Hemophilia A
• Single Patient Treatment IND for the Use of Protein C
Concentrate
• Compassionate Use of Deferiprone in Patients with
Thalassemia
• A Randomized, Open-label, Multi-center, Phase II
Study to Evaluate the Safety and Efficacy of Oral
ICL670 (deferasirox) 20 mg/kg/day Relative to
Subcutaneous Deferoxamine in Sickle Cell Disease
Patients with Iron Overload
• A Retrospective Study to Capture Dosing and
Treatment Outcome Data in Subjects with Severe
Congenital Protein C Deficiency Who Were Treated
with Protein C Concentrate Under an Emergency Use
• Prospective Multi-center Evaluation of the Duration of
Therapy for Thrombosis in Children (The “Kids-DOTT”
Trial)
• Cultural Adaptation and Psychometirc Validation of the
Haemo-QoL into American English
• Fludarabine-based Conditioning for Allogeneic Marrow
Transplantation from HLA-compatible Unrelated
Donors in Severe Aplastic Anemia BMT-CTN0301
• ThromboEmbolism Registry
• The Epidemiology of Diamond Blackfan: The Diamond
Blackfan Anemia Registry
• Investigation of the Modulation of Phospholipase
in Acute Chest Syndrome (Dose Escalation Study:
Varespladib Infusion [A-001] for the Prevention of Acute
Chest Syndrome in At-risk Patients with Sickle Cell
Disease and Vaso-occlusive Crisis)
• The Universal Data and Serum Specimen Collection
System for Hemophilia
• Severe Chronic Neutropenia International Registry
Leukemia/Lymphoma
• A Pilot Study to Determine the Toxicity of the Addition of
Rituximab to the Induction and Consolidation Phases
and the Addition of Rasburicase to the Reduction
Phase in Children with Newly Diagnosed Advanced
B-cell Leukemia/Lymphoma Treated with LMB/FAB
Therapy ANHL01P1
• A Phase II/III Study of Immunomodulation after High
Dose Myeloablative Therapy with Autologous Stem Cell
Rescue for Refractory/Relapsed Hodgkin’s Disease
AHOD0121
• Classification of Acute Lymphoblastic Leukemia
AALL03B1
• High-risk B-precursor Acute Lymphoblastic Leukemia
AALL0232
• Single Patient Treatment IND for the Special Exception
Use of Carboxypeptidase G2 for MTX Toxicity
Hematology and Oncology 49

• A Phase II Study of Amifostine in Children with Myelodysplastic
Syndrome AAML0121
• Treatment of Late Isolated Extramedullary Relapse from Acute
Lymphoblastic Leukemia (ALL) (Initial CR1 >/= 18 months
AALL02P2
• Standard Risk B-Precursor Acute Lymphoblastic Leukemia: A
Phase III Group Wide Study AALL0331
• Treatment of Children with Newly Diagnosed Low Stage
Lymphocyte Predominant Hodgkin’s Disease (LPHD)
AHOD03P1
• A Phase III Study for the Treatment of Children and Adolescents
with Newly Diagnosed Low Risk Hodgkin’s Disease AHOD0431
• Renal Tumors Classification, Biology, And Banking Study
AREN03B2
• A Study of Rasburicase Treatment for Chemotherapy or
Malignancy-induced Hyperuricemia in Patients with a History of
Asthma or Allergies
• Rare and Cutaneous Non-Hodgkin’s Lymphoma Registry
ANHL04B1
• Epidemiology of Infant Leukemia AE24
• Patient Care Evaluation Study of Institutional Acute Myeloid
Leukemia
• Patient Care Evaluation Study of Institutional Acute
Lymphoblastic Leukemia (ALL) Relapse
• Hodgkin’s Disease (HD) Banking Study AHOD04B1
• A Phase II Study of Bortezomib (Velcade, PS-341, IND# 58443)
in Combination with Ifosfamide/Vinorelbine in Pediatric Patients
and Young Adults with Refractory/Recurrent Hodgkin’s Disease
AHOD0521
• A Feasibility Pilot and Phase II Study of Chemoimmunotherapy
with Epratuzumab for Children with Relapsed CD22-Positive
Acute Lymphoblastic Leukemia ADVL04P2
• The Treatment of Down Syndrome Children with Acute Myeloid
Leukemia (AML) and Myelodysplastic Syndrome (MDS) under
the Age of 4 Years AAML0431
50 Hematology and Oncology

• Induction Intensification and Allogeneic Bone Marrow Transplant
in Infant ALL P9407
• Randomized Phase III Study for the Treatment of Newly
Diagnosed Disseminated Lymphoblastic Lymphoma or Localized
Lymphoblastic Lymphoma A5971
• The Use of Modified BFM+/- Compound 506U78 (NSC#
686673) in an Intensive Chemotherapy Regimen for Treatment
of T-cell Leukemia AALLOOP2
• A Pilot Study of Re-induction Chemotherapy with Ifosamide and
Vinorelbine (IV) in Children with Refractory/Relapsed Hodgkin’s
Disease AHOD00P1
• Phase II Window Evaluation of the Farnesyl Transferase
Inhibitor (R115577) Followed by 13-Cis Retinoc Acid, Cytosine
Arabinoside and Fludarabine Plus Hematopoietic Stem Cell
Transplantation in Children with Juvenile Myelomonocytic
Leukemia AAML0122
• Database for the North American Juvenile Myelomonocytic
Leukemia (JMML) Project and Reference Laboratory
• Treatment of Patients with Newly Diagnosed Acute Myeloid
Leukemia or Myelodysplasia. A Collaborative Trial of St. Jude
Children’s Research Hospital, Stanford University Medical
Center, Cook Children’s Medical Center and Children’s Hospital
of Michigan AML02
• A Phase III, Groupwide Study of Dose-intensive Response
based Chemotherapy and Radiation Therapy for Children and
Adolescents with Newly Diagnosed Intermediate-risk Hodgkin’s
Disease AHOD0031
• A COG Pilot Study for the Treatment of Very High-risk Acute
Lymphoblastic Leukemia in Children and Adolescents [STI571
(GLEEVEC) NSC#716051] AALL0031
• Intensive Induction Therapy for Children with ALL who
Experience a Bone Marrow Relapse AALL01P2
• Clinical and Biological Predictors of Therapy-related Leukemia
AB9804
• Total Therapy Study XV for Newly Diagnosed Patients with Acute
Lymphoblastic Leukemia
• A Phase II Study of Rituximab and Ifosfamide Carboplatin and
Etoposide (ICE) chemotherapy in children with Recurrent/
Refractory B-cell (CD 20+) Non-Hodgkin’s Lymphoma and B-cell
Acute Lymphoblastic Leukemia ANHL0121
• 97 Bank Protocol for Collecting, Archiving and Distributing
Human Tissue Specimens
• Advanced Stage Anaplastic Large Cell Lymphoma (ALCL)
with Standard APO (Doxorubicin, Prednisone, VCR) vs.
Consolidation with a Regimen Including Vinblastine ANHL0131
• A Phase II Study of Weekly Gemcitabine and Vinorelbine in
Children with Recurrent Hodgkin’s Disease AHOD0321
• Compassionate Use of Lestaurtinib (CEP-701) in Combination
with Chemotherapy in a Young Child with Relapsed or Refractory
FLT3-mutant Acute Myeloid Leukemia
• Intensified Methotrexate, Nelarabine (Compound 506U78:
IND# 52611) and Augmented BFM Therapy for Children and
Young Adults with Newly Diagnosed T-cell Acute Lymphoblastic
Leukemia AALL0434
Neuroblastoma
• A Phase III, Randomized Trial of Intravenous Gammaglobulin
Therapy for Patients with Neuroblastoma Associated
Opsoclonus - Myoclonus-Ataxia Syndrome Treated with
Chemotherapy and Prednisone ANBL00P3
• A Phase II study of hu14.18-IL2 (BB-IND-9728) in Children with
Recurrent or Refractory Neuroblastoma ANBL0322
• Perinatal Neuroblastoma: Expectant Observation ANBL00P2
• A Phase II Study of Irinotecan + Temozolomide in Children with
Recurrent Neuroblastoma ANBL0421
• Expanded Access Protocol for Purging Peripheral Blood
Stem Cells or Bone Marrow from Patients with High-risk
Neuroblastoma Prior to Autologous Transplantation Under FDA
Investigational Device Exemption
• A Phase II Study of ABT-751, an Orally Bioavailable Tubulin
Binding Agent, in Children with Relapsed or Refractory
Neuroblastoma ANBL0621
• A Randomized Study of Purged vs Unpurged Peripheral Blood
Stem Cell Transplant Following Dose Intensive Induction
Therapy for High-risk Neuroblastoma A3973
• Neuroblastoma Biology Studies ANBL00B1
• Phase III Randomized Study of Chimeric Antibody 14.18
(Ch14.18) in High-risk Neuroblastoma Following Myeloablative
Therapy and Autologous Stem Cell Rescue ANBL0032
Neuro-Oncology
• A Phase II Study of R11577 (ZARNESTRA) (NSC#702818,
IND 58359) in Children with Recurrent or Progressive High
grade Glioma, Medulloblastoma/PNET, or Brainstem Glioma
ACNS0226
• A Phase II Trial of Conformal Radiation Therapy for Pediatric
Patients with Localized Ependymoma, Chemotherapy Prior
to Second Surgery for Incompletely Resected Ependymoma
and Observation for Completely Resected, Differentiated,
Supratentorial Ependymoma ACNS0121
• A Phase II Study to Assess the Ability of Neoadjuvant
Chemotherapy +/- Second Look Surgery to Eliminate All
Measurable Disease Prior to Radiotherapy for NGGCT
ACNS0122
• A COG Protocol for Collecting and Banking Pediatric Brain
Tumor Research Specimens ACNS02B3
• Concurrent Radiation and Temozolomide and Lomustine in the
Treatment of High-grade Gliomas ACNS0423
• A Phase III Randomized Trial for the Treatment of Pediatric
Hematology and Oncology 51

• High-grade Gliomas at First Recurrence with a Single Highdose
Chemotherapy and Autologous Stem Cell Transplant
Versus Three Courses of Intermediate-dose Chemotherapy with
Peripheral Blood Stem Cell Transplant ACNS0231
• High-dose Temozolomide, Thiotepa and Carboplatin with
Autologous Stem Cell Rescue (ASCR) Followed by Continuation
Therapy with 13-cis-Retinoic Acid in Patients with Recurrent/
1refractory Malignant Brain Tumors ONC-032P
• A Phase I/II Study of Topotecan with G-CSF and Radiation
Therapy in Children with Malignant Intrinsic Pontine Brainstem
Gliomas of Childhood ACNS0224
• A Pilot Study Using Carboplatin, Vincristine and Temozolomide
for Children < 10 Years of age with Progressive/ Symptomatic
Low-grade Gliomas ACNS0223
• A Phase II Study of Conformal Radiotherapy in Patients with
Low-grade Gliomas ACNS0221
• Pre-clinical Pharmacology in Surgical Brain Tumor Specimens
ACNS02B1
• A COG Protocol for Collecting and Banking Pediatric Research
Specimens Including Rare Pediatric Tumors ABTR01B1
• A Phase II Study of Motexafin-Gadolinium (NSC 695238, IND #
55583) and Involved Field Radiation Therapy for Intrinsic Pontine
Glioma of Childhood ACNS0222
• Systemic Chemotherapy, Second Look Surgery and Conformal
Radiation Therapy Limited to the Posterior Fossa and Primary
Site for Children > 8 months and < 3 years with Non-metastatic
Medulloblastoma P9934
• A Study Evaluating Limited Target Volume Boost Irradiation
and Reduced Dose Craniospinal Radiotherapy (18.00 Gy) and
Chemotherapy in Children with Newly Diagnosed Standard-risk
Medulloblastoma ACNS0331
• Efficacy of Carboplatin Administered Concomitantly with
Radiation and Isotretinoin as a Pro-apoptotic Agent in Other
Than Average Risk Medulloblastoma/PNET Patients ACNS0332
Oncology
• A Phase II Study of Oxaliplatin in Children with Recurrent Solid
Tumors ADVL0421
• Vincristine, Dactinomycin and Lower Doses of
Cyclophosphamide with or without Radiation Therapy for
Patients with Newly Diagnosed Low-risk Embryonal/ Botryoid/
Spindle Cell Rhabdomyosarcoma ARST0331
• Pilot Study of Cisplatin, Etoposide, Bleomycin and Escalating
Dose Cyclophosphamide Therapy for Children with High-risk
Malignant Germ Cell Tumors AGCT01P1
• Key Adverse Events after Childhood Cancer ALTE03N1
• A Pilot Study Combining Temozlomide, Oncovin, Camptosar
(irinotecan) and Oral Antibiotic in Children and Adolescents with
Recurrent Malignancy
• Hemophagocytic Lymphohistiocytosis Study Group HLH 2004
• A Pilot Study of Low-dose Antiangiogenic Chemotherapy in
Combination with Standard Multi-agent Chemotherapy for
Patients with Newly Diagnosed Metastatic Ewing Sarcoma
Family of Tumors AEWS02P1
• A Phase II Study of Aerosolized GM-CSF (NSC# 613795,
IND#11042) in Patients with First Pulmonary Recurrence of
Osteosarcoma AOST0221
• A Study of Unilateral Retinoblastoma With and Without
Histopathologic High-risk Features and the Role of Adjuvant
Chemotherapy ARET0332
• A Phase II Study of Sulindac and Tamoxifen in Patients with
Desmoid Tumors that are Recurrent or Not Amendable to
Standard Therapy ARST0321
• Low Birth Weight and Other Risk Factors for Hepatoblastoma
AEPI04C1
• A Randomized Trial of the European and American
Osteosarcoma Study Group to Optimize Treatment Strategies
for Resectable Osteosarcoma Based on Histological Response
to Pre-operative Chemotherapy AOST0331
• An International Phase II Study Evaluating the Combination
of Cladribine (2 CdA) + Cytarabine Cytosine-arabinoside in
Refractory Multisystem Langerhans Cell Histiocytosis (LCH)
LCH-S-2005
• A Phase II Trial of Ixabepilone, an Epothilone B Analog, in
Children and Young Adults with Refractory Solid Tumors
ADVL0524
• Treatment of High-risk Renal Tumors AREN0321
• Intensive Multi-agent Therapy, Including Dose-compressed
Cycles of Ifosfamide/Etoposide (IE) and Vincristine/Doxorubicin/
Cyclophosphamide (VDC) for Patients with High-risk
Rhabdomyosarcoma ARST0431
• A Pilot Phase II Study for Children with Infantile Fibrosarcoma
ARST03P1
• Treatment of Childhood Nasopharyngeal Carcinoma with
Neoadjuvant Chemotherapy and Concomitant Chemoradio
Therapy ARAR0331
• A Phase III, Open-label, Single-arm Study of Tenecteplase for
the Restoration of Function in Dysfunctional Central Venous
Access Devices N3699g
• Treatment for Very Low, Low and Standard Risk Favorable
Histology Wilms Tumor AREN0532
• Randomized Study of Vincristine, Dactinomycin and
Cyclophosphamide (VAC) vs. VAC Alternating with Vincristine
and Irinotecan (VI) for Patients with Intermediate-risk
Rhabdomyosarcoma (RMS) ARST0531
• Treatment of Newly Diagnosed Higher-risk Favorable Histology
Wilms Tumors AREN0533
• Hemophagocytic Lymphohistiocytosis Study Group HLH 94
• Hepatoblastoma Biology and Tissue Bank P9346
• Role of Dose-intensive Chemotherapy with Peripheral Blood
Stem Cell Rescue in Patients with Osteosarcoma with High-risk
Features
• Intergroup Protocol for Treatment of Children with
Hepatoblastoma 9645
52 Hematology and Oncology

• A Groupwide Protocol for Collecting and Banking
Pediatric Cancer Research Specimens. An Intergroup
Rhabdomyosarcoma Study Group Protocol D9902
• Randomized Study of Vincristine, Actinomycin-D and
Cyclophosphamide (VAC) versus VAC Alternating with
Vincristine, Topotecan and Cyclophosphamide for Patients with
Intermediate-risk Rhabdomyosarcoma D9803
• Trial of Chemotherapy Intensification Through Interval
Compression in Ewing Sarcoma and Related Tumors
AEWS0031
• Treatment Protocol of the Third International Study for
Langerhans Cell Histiocytosis LCH III
• Second Malignant Neoplasms Following Childhood Cancer
AS9801
• A Groupwide, Randomized, Phase II Window Study of Two
Difference Schedules of Irinotecan in Combination with
Vincristine and Pilot Assessment of Safety and Efficacy of
Tirapazamine Combined with Multiagent Chemotherapy for First
Relapse or Progressive Disease ARST0121
• International Pediatric Adrenocortical Tumor Registry (IPACTR)
Data Collection and Use of Tissue for Banking and Research
• A Groupwide Biology and Banking Study for Ewing Sarcoma
AEWS02B1
• European Ewing Tumor Working Initiative of National Groups
Ewing Tumor Studies AEWS0331
• Gene Expression in Langerhans Cell Histiocytosis
• A Phase III Study of Reduced Therapy in the Treatment of
Children with Low and Intermediate-risk Extracranial Germ Cell
Tumors AGCT0132
• Osteosarcoma Biology Protocol. A Companion to Groupwide
Therapeutic Studies P9851
• Phase III Trial of High Dose INF Alpha-2b versus Cisplatin,
Vinblastine, Dacarbazine plus IL-2 and INF Alpha-2b in Patients
with High-risk Melanoma - A SWOG Coordinated Intergroup Trial
S0008
Stem Cell Transplant Supportive Protocols
• A Phase II Study of Pentostatin for the Treatment of High-risk or
Refractory Chronic GVHD in Children GVH0313
• A Randomized Double Blind Placebo Controlled Clinical Trial
to Assess the Efficacy of Traumeel ® S (IND # 66649) for the
Prevention and Treatment of Mucositis in Children Undergoing
Hematopoietic Stem Cell Transplantation ACCL0331
• Soluble Tumor Necrosis Factor Receptor: Enbrel ® (Etanercept)
for the Treatment of Acute Non-infectious Pulmonary Dysfunction
(Idiopathic Pneumonia Sydrome) Following Allogeneic Stem Cell
Transplantation ASCT0521
• Compassionate Use of Defibrotide in Patient with Veno-occlusive
Disease
• Blood Collection for National Marrow Donor Program
An Environment That Encompasses Compassion
The open infusion room at the new Hematology and Oncology Clinic – Grapevine invites interaction. Patients and families gather to catch
up on each other’s lives while waiting to see the physician or receive chemotherapy. Additionally, a Child Life specialist engages patients in
activities which use the imagination.
Hematology and Oncology 53

Profiles
Akers, Lauren, D.O.
Education: Nova Southeastern University, College of Osteopathic Medicine,
Fort Lauderdale, Fla.
Residency: Children’s Hospital of Austin, Austin, Texas
Fellowship: Children’s Cancer Hospital at MDA Cancer Center, Houston, Texas
Board Certified: Pediatrics, Pediatric Hematology and Oncology
Beam, Donald, M.D.
Education: Louisiana State University Health Sciences Center School of
Medicine, New Orleans, La.
Residency: Louisiana State University Health Sciences Center School of
Medicine, New Orleans, La.
Fellowship: Duke University Medical Center, Durham, N.C.
Board Certified: Pediatrics, Internal Medicine, Pediatric Hematology and Oncology
Bowman, W. Paul, M.D., Chairman of Cook Children’s
Leukemia and Lymphoma program, Medical Director,
Academic Pediatrics
Education: University of Manitoba, Winnipeg, Manitoba, Canada
Residency: University of Manitoba, Winnipeg, Manitoba, Canada
Fellowship: St. Jude Children’s Research Hospital, Memphis, Tenn.
Board Certified: Pediatrics, Pediatric Hematology and Oncology
Davis, Samuel, M.D.
Education: Swarthmore College, Swarthmore, Pa.
Residency: University of Texas, Austin, Texas
Fellowship: University of Texas Southwestern Medical School, Dallas, Texas
Board Certified: Pediatric Hematology and Oncology
54 Hematology and Oncology

Eames, Gretchen, M.D., MPH, Medical Director,
Hematology and Oncology Center,
Stem Cell Transplant Program
Education: University of Iowa, Iowa City, Iowa
Residency: University of Minnesota, Duluth, Minn.
Fellowship: University of Minnesota, Duluth, Minn.
Board Certified: Pediatrics, Pediatric Hematology and Oncology
Granger, Meaghan, M.D.
Education: University of Arkansas for Medical Sciences, Little Rock, Ark.
Residency: Vanderbilt University, Vanderbilt Children’s Hospital,
Nashville, Tenn.
Fellowship: Northwestern University, Chicago, Ill.
Board Certified: Pediatrics, Pediatric Hematology and Oncology
Heym, Kenneth, M.D., Medical Director,
Oncology Services
Education: Vanderbilt University School of Medicine, Nashville, Tenn.
Residency: Children’s Hospital of New York-Presbyterian, New York, N.Y.
Fellowship: Baylor College of Medicine, Houston, Texas
Board Certified: Pediatrics, Pediatric Hematology and Oncology
Howrey, Richard, M.D.
Education: University of Michigan Medical School, Ann Arbor, Mich.
Residency: Children’s Medical Center, Dallas, Texas
Fellowship: Duke University Medical Center, Durham, N.C.
Board Certified: Pediatrics, Pediatric Hematology and Oncology
Johnson, Clarissa, M.D.
Education: Case Western Reserve University School of Medicine,
Cleveland, Ohio
Residency: Cincinnati Children’s Hospital Medical Center, Cincinnati, Ohio
Fellowship: Duke University Medical Center, Durham, N.C.
Board Certified: Pediatrics
Board Eligible: Pediatric Hematology and Oncology
Hematology and Oncology 55

Murray, Jeffrey C., M.D., Medical Director,
Neuro-Oncology
Education: Baylor College of Medicine, Houston, Texas
Residency: Texas Children’s Hospital, Baylor College of Medicine, Houston,
Texas
Fellowship: Texas Children’s Hospital, Baylor College of Medicine, Houston,
Texas
Board Certified: Pediatric Hematology and Oncology
Richie-Gillespie, Mayme, M.D., Medical Director,
Orthopedic Oncology
Education: Vanderbilt University School of Medicine, Nashville, Tenn.
Residency: University of Texas Southwestern Medical Center, Dallas, Texas
Fellowship: Children’s Orthopedic Hospital and Medical Center, Seattle,
Wash.; University of Texas Southwestern Medical Center, Dallas, Texas
Board Certified: Orthopedic Surgery
Sanders, Joann, M.D., Medical Director,
Clinical Excellence
Education: Saint Louis University School of Medicine, St. Louis, Mo.
Residency: Saint Louis University Cardinal Glennon Children’s Hospital,
St. Louis, Mo.
Fellowship: University Texas Southwestern Medical Center, Dallas, Texas
Board Certified: Pediatrics, Pediatric Hematology and Oncology
Torres, Marcela, M.D., Medical Director,
Hematology Services
Education: Universidad Peruana Cayetano Heredia, Lima, Peru
Residency: Children’s Hospital of Wisconsin, Medical College of Wisconsin,
Milwaukee, Wis.
Fellowship: Children’s Hospital of Wisconsin, Medical College of Wisconsin,
Milwaukee, Wis.
Board Certified: Pediatrics, Pediatric Hematology and Oncology
Vallance, Kelly, M.D.
Education: Tulane University School of Medicine, New Orleans, La.
Residency: Massachusetts General Hospital, Boston, Mass.
Fellowship: St. Jude Children’s Research Hospital, Memphis, Tenn.
Board Certified: Pediatrics
Board Eligible: Pediatric Hematology and Oncology
56 Hematology and Oncology

Bashore, Lisa, MS, RN, CPNP, CPON
Education: The State University of New York,
Buffalo, N.Y.
Bayles, Amy Elizabeth, RN, CPNP, CPON
Education: University of Texas Medical Branch,
Galveston, Texas
Bennett, Penny R., RN, MSN, CPNP
Education: University of Texas at Arlington,
Arlington, Texas
Braly, Emily, RN, MSN, CPNP
Education: Texas Christian University, Fort Worth,
Texas; Pediatric Nurse Practitioner, University of
Texas at Arlington, Arlington, Texas
Brentlinger, Angela Kay, RN, MSN, CPNP
Education: Texas Woman’s University,
Dallas, Texas
Dalton, Lisa M., RN, MSN, FNP
Education: Yale University School of Nursing,
New Haven, Conn.
Hensley, Rebecca, RN, MSN, CPNP
Education: University of Texas at Arlington,
Arlington, Texas
Hematology and Oncology 57

58 Hematology and Oncology

accessibility
Hematology and Oncology Locations
Hematology and Oncology Center
901 Seventh Ave., Ste. 220
Fort Worth, TX 76104
682-885-4007 phone
682-885-3914 fax
Hematology and Oncology Clinic – Grapevine
1600 W. Northwest Hwy., Ste. 500
Grapevine, TX 76051
817-310-0024 phone
817-310-0618 fax
Hematology and Oncology offices across Texas
Abilene
1401 Ambler Ave., Ste. 101
Abilene, TX 79601
325-670-3910 phone
325-670-3919 fax
Denton
209 N. Bonnie Brae, Ste. 100
Medical Building 3
Denton, TX 76201
866-614-8991 toll-free
940-243-0104 phone
940-320-3680 fax
Midland
4214 Andrews Hwy., Ste. 305
Midland, TX 79701
432-570-5693 phone
432-570-5696 fax
Odessa
701 West Fifth, Ste. 2106
Odessa, TX 79764
432-570-5693 phone
432-570-5696 fax
San Angelo
201 E. Harris
San Angelo, TX 76903
432-570-5693 phone
432-570-5696 fax
Waco
2201 MacArthur Dr., Ste. 101
Waco, TX 76708
254-757-6775 phone
254-757-1912 fax
Hematology and Oncology 59

60 Hematology and Oncology