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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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