Haematologica 2003 - Supplements

296
PERIPHERAL BLOOD STEM CELL TRANSPLANT
(PBSCT) IN ELEVEN PATIENTS WITH POEMS
SYNDROME
Angela Dispenzieri1, Alvaro Moreno-Aspitia2, Martha Q.
Lacy1, Gerardo Colon-Otero2, Ayalew Tefferi1, Mark R.
Litzow1, Robert A. Kyle1 and Morie A. Gertz1
Division of Hematology, 1Mayo Clinic, Rochester, MN. 2Mayo
Clinic, Jacksonville, FL
Background: The POEMS syndrome is characterized by peripheral
neuropathy (PN), clonal plasma cell disorder (PCD), organomegaly,
endocrinopathy, skin changes, edema, sclerotic bone lesions, and
thrombocytosis. Patients have a low bone marrow (BM) plasma cell
(PC) burden and a survival superior to patients with multiple
myeloma (MM). Based on the improved response rates observed
with PBSCT in patients with other PCD, autologous PBSCT may be
an attractive treatment option for patients with POEMS syndrome.
Methods: Between March 1999 and February 2003, 11 patients with
POEMS syndrome have undergone PBSCT at Mayo Clinic
Rochester and Jacksonville. Seven received melphalan 200 mg/m2,
three melphalan 140 mg/m2, and one BEAM as conditioning
regimens. All received a minimum of 4.4 x 10(6) CD34 autologous
PBSC/kg. Standard supportive care with prophylactic antibiotics and
growth factor support was provided. Results: All but one had a severe
rapidly progressive sensorimotor PN involving both upper and lower
extremities, 7 wheelchair confined. One patient had sensory
neuropathy without significant motor involvement. All but one was
male. A monoclonal lambda PCD was documented in all patients,
and two had biopsy proven Castleman Disease. Median age was 50
years (range 19-62). Seven had organomegaly (5 splenomegaly; 2
hepatomegaly; and 3 lymphadenopathy). Both endocrinopathy and
skin changes were present in 9 patients. Seven had thrombocytosis
and/or erythrocytosis, and 8 had sclerotic bone lesions (diffuse in 5,
solitary lesion in 3). Using the Bardwick Criteria, the median number
of POEMS features was 5 (range 2-5). Using the Mayo Criteria
which includes the five criteria of the Bardwick acronym, but also
sclerotic bone lesions, Castleman Disease, extravascular volume
overload, and papilledema, the median number of features was 6
(range 3-8). The median number of therapeutic regimens prior to
PBSCT was 3 (range 0-6); two of the three patients with a solitary
bone lesion were progressing despite external beam radiation. From
first symptoms and from diagnosis of POEMS the median times to
transplant were 27 and 10 months (ranges 4-180 and 2-180),
respectively. Two patients had had prior cerebrovascular accidents
and one a history of transient ischemic attacks. All but one patient
had significantly abnormal pre-transplant pulmonary function tests.
There was one transplant-related death in a patient who did not
engraft after an episode of a culture negative systemic inflammatory
response syndrome on day +8; he died 115 days after his transplant.
This same patient and another had temporary renal failure, requiring
dialysis. Four of the 11 patients spent time in the intensive care unit,
and 3 required intubation for respiratory compromise. One patient
required biphasic positive airway pressure throughout his transplant
course. Of the 8 evaluable patients, all have had a complete
hematologic response and 7 have had neurologic improvement at a
median follow-up of 6 months. Other symptoms including fatigue,
organomegaly, pulmonary compromise, hyperpigmentation and
extravascular volume overload have improved substantially in
affected patients. Conclusions: PBSCT for POEMS syndrome may
result in a high hematologic response rate and improvement in
peripheral neuropathy and systemic symptoms, but may also be
associated with significant morbidity.
297
Autologous stem cell transplantation (ASCT) for
primary amyloidosis (AL): a single-center experience
J. Esteve, J. Bladé, S. Rives, L. Rosiñol, C. Martínez, M.
Rovira, A. Urbano-Ispizua, P. Marín*, E. Carreras, E.
Montserrat.
Hematology Department, Hemato-Oncology Institute,
Hemotherapy Department*, Institut d’Investigacions Biomèdiques
August Pi i Sunyer (IDIBAPS), Hospital Clínic, Barcelona, Spain.
Primary amyloidosis is a poor-prognosis disease with limited
response to conventional chemotherapy. High-dose melphalan
followed by autologous hematopoetic stem-cell rescue results in a
significant response rate, although it is hampered by a significant
toxicity.
Aim: To analyze the long-term results of a series of 17
consecutive patients with AL undergoing peripheral blood ASCT
at our institution.
Patients and methods: 17 patients (53% female; median age, 53,
range: 33 – 66) diagnosed with systemic AL who underwent
ASCT during a 5-year period (11/97 – 11/02) were included in
the analysis. Median interval from diagnosis to ASCT was 9
months (2 – 63) and seven patients had received previous
chemotherapy (melphalan/prednisone, 4; VBMCP +
melphalan/prednisone, 1; VAD, 1; fludarabine, 1). The light
chain was of lambda type in 82% of cases. One patient presented
with a concomitant Waldenström’s macroglobulinemia and a
non-clonally related follicular lymphoma. The median number of
involved organs was 2, including renal (14 patients), heart (13),
liver (7), and peripheral nervous system (5). All patients were
mobilized with G-CSF (dose ranging from 10 to 24 µg/kg) and
conditioned with high-dose melphalan (200 mg/m2 in 13 pts, 140
mg/m2 in 4).
Results: Six patients (35%) died in the peritransplant period due
to multiorgan failure (n=2), hemorrhage (n=1), arrhythmia (n=2),
or splenic rupture (n=1). With regard to organ-based responses,
seven among 9 assessable patients (78%) achieved a response,
while no significant improvement was obtained in the remaining
two cases. Additionally, six hematological responses, including
three complete responses, were obtained. During follow-up, one
of the responders had a clinical progression at 48 months after
transplant, and underwent a second transplant procedure. Overall,
5-year survival was 57% + 12 and event-free survival at 2-year
was 57%+ 18 (figures 1 & 2).
Conclusion: Due to its benefit in a significant proportion of
patients, ASCT constitutes the treatment of choice for younger
patients with AL. Nonetheless, given the high toxicity related to
the systemic nature of the amyloid deposition, an accurate
selection of patients is crucial in order to minimize the high
mortality associated with to the procedure.
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