Haematologica 2003 - Supplements

We have transplanted 125 patients with AL. 118 had a
monoclonal light chain in serum or urine. The remaining 7 all
had clonal plasma cells in the bone marrow with an overall kappa
to lambda ratio of 1:2.6. At the time of diagnosis signs of
amyloid were seen in the kidney in 83 (66%), the heart in 60
(48%), peripheral nerves in 17 (14%) and the liver in 21 (17%).
The characteristics of the patient are given in the table.
Characteristics of Patients with AL (n=125) before ASCT
Stem cell mobilization was accomplished with
Cyclophosphamide 1.5 g/m 2 on two consecutive days followed
by GM-CSF 5 mcg/kg/d in 33. The other 92 patients received G-
CSF 10 mcg/kg/d. The CD34 yield in 119 patients ranged from
2.01 to 51 million cells/kg with a median yield of 5.9 x 10 6 /kg.
The number of apheresis sessions required to collect the stem
cells ranged from 1 to 10, median 3. The goal was to collect a
minimum of 5 x 10 6 stem cells.
Conditioning was with Melphalan and total body radiation in 17,
Melphalan 200/m 2 in 59, Melphalan 140/m 2 in 29 and Melphalan
100/m 2 in 11. Reduced dose conditioning was given on a riskadjusted
basis, considering the serum creatinine, the degree of
cardiac function, age and number of organs involved.
Characteristics Number (%)
or Median
(Range)
Number (%)
Abnormal
Abnormal
Value
Male gender 68 (54)
Age 55 (31-71)
Albumin 2.8 (1.0-4.4) 54 (43)
35 (28)
3 g/d
>1 g/d
Urine M protein 0.19 (0.002- 48 (38) >0.25 g/d
(N=114) 2.85)
% BM plasma
cells
8 (0.4-49) 22 (18)
Echo IVS (mm) 12 (7-25) 29 (23) ≥15
EF% 65 (28-84) 25 (20) 3
Number of
organs involved
1
2
>2
64 (51)
42 (34)
19 (15)
survival has only been reached for those patients who had greater
than two organs involved, 16.5 months. We have 4 patients with
single organ involvement that now have been followed for greater
than 5 years and 2 patients with two organ involvement that have
been followed in excess of 5 years. The presence of long-term
survival should not be equated with a proven advantage of stem
cell transplant since these patients are highly selected. However,
the overall response of 64% exceeds anything that we have
previously achieved with conventional chemotherapy. This is a
technique worth pursuing, although a phase III trial is necessary.
POEMS syndrome:
We have transplanted six patients with POEMS syndrome, all of
whom had lambda light chains, three G λ, two A λ, one free λ.
The age range of the six was from 20 to 62. Two of the patients
were previously treated with radiation therapy and progressed.
One patient died of graft failure and was inevaluable for
response. Of the remaining five patients four had complete
eradication of the monoclonal protein and one had a greater than
50% reduction in the monoclonal protein. Four of the five had
definite improvement in their neuropathy and neuropathic
symptomatology, all of whom had both sensory and motor
neuropathy. The use of high dose therapy for patients with
POEMS syndrome can produce both hematologic and
symptomatic responses. Five patients are alive with survivals
ranging from 8 to 48 months.
Eight of the 125 patients died prior to day 30 and were not
evaluable for neutrophil engraftment. Of the remaining 117
patients all achieved a neutrophil count of 500/uL ranging from 7
to 116 days (median 14) following transplantation. There were 15
patients who died without having achieved a platelet count of
20,000/uL. The time to 20,000 platelets for the remaining 110
patients ranged from 7 to 406 days (median 17). There was one
mobilization-related death and one patient died on day 0 during
stem cell infusion. Treatment related mortality for the entire
group was 15% with death due to a wide variety of complications
including intractable intestinal bleeding, acute treatment-related
renal failure, cardiac arrhythmias, disseminated fungus infection,
etc.
There are two major predictors of survival which include the
serum creatinine at the time of transplantation and the number of
organs involved at the time of transplantation (p=0.001). Median
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