Low-dose thalidomide for multiple myeloma: Interim analysis of a compassionate use program

Background: Despite recent advances in systemic and supportive therapies, multiple myeloma remains an incurable plasma cell malignancy. Novel therapeutic approaches are thus needed. Thalidomide has recently been recognized as an effective new agent for previously untreated, refractory or relapsed myeloma. Patients and Methods: To evaluate the efficacy and tolerability of thalidomide in myeloma, we performed a retrospective analysis of 21 consecutive patients receiving thalidomide alone or in combination with dexamethasone and/or intermittent cyclophosphamide as first-line, maintenance or salvage therapy within a compassionate use program. Results: Of the 21 patients, 16 (76.2%) had refractory or relapsed disease, including 7 (33.3%) patients relapsing after autologous stem cell transplantation. Three patients received thalidomide as maintenance therapy after having achieved a partial remission following autologous stem cell transplantation or conventional chemotherapy. Two patients were given thalidomide as first-line treatment for indolent disease. During long-term treatment ( median 12 months, range 1-27 months), patients tolerated only low doses of thalidomide (50-150 mg/day) due to cumulative neurotoxicity. At a median follow-up of 16 months (range 1.5-28 months), we observed an overall response rate of 61.9% (50% for the subgroup receiving thalidomide alone; 77.8% for combination therapy) consisting of 1 complete response, 2 near-complete responses, 8 partial responses and 2 minor responses. Median progression-free survival was 20 months. Conclusions: We conclude that low-dose thalidomide (50-100 mg/day) alone or in combination is a safe, well-tolerated and effective form of therapy for patients with myeloma at various stages of disease.