Treatment of Waldenstrom's macroglobulinemia with thalidomide

Purpose: We performed a prospective phase II study to assess the activity o f thalidomide in patients with Waldenstrom's macroglobulinemic (WM). Patients and Methods: Twenty patients with WM were treated with thalidomide at a starting dose of 200 mg daily with dose escalation in 200-mg incremen ts every 14 days as tolerated to a maximum of 600 mg. All patients were sym ptomatic, their median age was 74 years, and 10 patients were previously un treated. Results. On an intent-to-treat basis, five (25%) of 20 patients achieved a partial response after treatment. Responses occurred in three of 10 previou sly untreated and in two of 10 pretreated patients. None of the patients tr eated during refractory relapse or with disease duration exceeding 2 years responded to thalidomide. Time to response was short, ranging between 0.8 m onths to 2.8 months. Adverse effects were common but reversible and consist ed primarily of constipation, somnolence, fatigue, and mood changes. The da ily dose of thalidomide was escalated to 600 mg in only five patients (25%) , and in seven patients (35%), this agent was discontinued within 2 months because of intolerance. Conclusion: Our data indicate that thalidomide has activity in WM but only low doses were tolerated in this elderly patient population. Confirmatory s tudies as well as studies that will combine thalidomide with chemotherapy o r with rituximab may be relevant. J Clin Oncol 79:3596-3607. (C) 2001 by American Society of Clinical Oncolog y.