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A verdict on the value of stem-cell
transplantation in multiple myeloma awaits the results of ongoing
studies
Multiple myeloma (MM) accounts for 1 percent of all cancers and
occurs when certain blood plasma cells infiltrate the normal bone
marrow causing multiple bone marrow tumors. These tumors result
in widespread bone lesions, pain, and fractures.
The feasibility of peripheral blood stem-cell
transplantation in conjunction with high-dose chemotherapy (HDCT)
has been documented in hundreds of MM patients in all stages of
disease. The rationale for this combined approach is that
stem-cell support would circumvent the blood toxicity associated
with HDCT. According to proponents, it currently is the only
regimen that offers the potential for cure or long-term survival
in selected patients. However, randomized controlled trials are
ongoing, and 4 to 5 years of followup will be required to
evaluate the trials, concludes Harry Handelsman, D.O., of the
Center for Health Care Technology, Agency for Health Care Policy
and Research.
Dr. Handelsman recently conducted a review of the
literature on use of stem-cell transplantation for treating MM.
He points out that accurate assessment of the efficacy of
treatments for myeloma is difficult because most trials have
involved limited numbers of patients and non-uniform treatment
regimens confounded by varied prognostic factors. Also, similar
median survivals seen after any form of current treatment makes
the identification of optimal therapy difficult.
Research shows that HDCT regimens with autologous
stem-cell support achieve a cure rate of 20-30 percent of
patients, with the best results seen in good-risk patients,
defined as young patients (less than age 50) with good
performance status, a low tumor burden, and response to previous
chemotherapy. Treatment-related deaths in such patients at
experienced centers is less than 5 percent, and reported 4-year
progression-free and overall survival ranges from 50 to 70
percent. Whether specific treatments can overcome the unfavorable
influence of factors such as high tumor mass, or whether
high-risk patients are the best candidates for HDCT remains
unproven. Until the completion of randomized controlled trials,
the use of stem-cell transplantation should be regarded as a
technology that requires further clinical study to determine its
role in treatment of MM, concludes Dr. Handelsman.
Details are in "Haematopoietic stem-cell transplantation in
multiple myeloma," by Dr. Handelsman, in
Cancer Treatment Reviews 22, pp. 119-125, 1996. Reprints (AHCPR
Publication No. 96-R132) are available from the AHCPR Publications Clearinghouse.
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