BACKGROUND: Multiple myeloma (MM) is the second most common hematologic malignancy, affecting approximately 14,000 new patients in the United State per year. The median overall survival is 5 years, and cure is a realistic goal for only a small minority of patients. METHODS: A review of the literature was conducted that focused on treatment strategies for MM involving administration of high doses of chemotherapy followed by autologous or allogeneic hematopoietic stem cell transplant. RESULTS: For over three decades, the standard treatment for MM has been a regimen of melphalan and prednisone (MP). Complete responses (CRs) have been rare, and 50 of patients have had disease that was resistant to treatment with MP. Attempts have been made to improve the outcome of MM by administering other combinations of standard doses of chemotherapy, but these treatments are equivalent in terms of overall survival. For patients who are candidates, high-dose therapy followed by autologous stem cell transplantation results in higher CR rates and improved long-term survival compared to treatment with standard doses of chemotherapy alone. While this strategy represents an advance in the treatment of MM, evidence-based reviews indicate that there are a number of issues to consider regarding the induction therapy, the collection of stem cells, and the timing, type, and number of high-dose therapies to use in this type of treatment strategy. CONCLUSIONS: Advances have been made in autologous transplantation, allogeneic transplantation, anti-MM agents, and immunotherapy for MM. Combining these different strategies to achieve synergistic responses is an exciting possibility.
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