Long-term Results of Single-Agent Thalidomide as Initial Therapy for Asymptomatic (Smoldering or Indolent) Myeloma

摘要

We report the long-term follow-up results of a phase II trial of thalidomide for early stage multiple myeloma (MM). Patients were eligible if they had smoldering multiple myeloma (SMM) or indolent MM without need for immediate therapy. Thalidomide was initiated at a dose of 200 mg/day and adjusted as tolerated. Disease progression was defined using modified American Society of Hematology/Food and Drug Administration consensus panel criteria for SMM. Thirty-one patients were enrolled; 29 (19 SMM, 10 indolent MM) were eligible. The median age was 61 years. Median follow up of living patients was 10.2 years (range, 7.5–11.0 years). Ten patients (34%) had a partial response (PR) and nine had minimal response (MR) for an MR plus PR rate of 66%. The median time to progression (TTP) to symptomatic myeloma was 35 months. Median TTP was 61 months in those achieving PR, 39 months with MR, and 9 months among those failing to achieve either MR or PR, p=0.005. Median overall survival from diagnosis was 86 months; median survival from onset of symptomatic myeloma was 49 months. Grade 3–4 non-hematologic adverse events were noted in 55% of patients. Randomized trials are needed to determine the role of early therapy in SMM.