Multiple myeloma is typically incurable, with a median survival of 3-4 years.1-3 Conventional chemotherapy with melphalan and prednisone (MP) produces a response rate of approximately 50%, with complete responses (CRs) in less than 10% of patients.4 Combination chemotherapy with additional cytotoxic agents improves response rates (60-70%), but without significant survival benefit compared to MP.4-6

The careful evaluation of the efficacy of the various therapeutic options and their impact on patient outcome has implications for both practice and research. In this chapter, we review the current criteria for evaluating response to therapy in myeloma, summarize established and novel prognostic factors for the disease, and briefly outline the recommended follow-up for patients.

Strict remission (response) criteria are required to monitor effectiveness of therapy in patients and to evaluate new drugs and interventions. Response criteria are also required to compare various therapeutic alternatives, both in clinical practice and in prospective trials. In some, but not all instances, response to therapy also serves as a marker for a good clinical outcome. In addition to response rates, other estimates of successful therapy in myeloma include measurements such as progression-free (PFS) and overall survival (OS).

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