Activity of thalidomide in AIDS-related Kaposis's sarcoma

Purpose: To assess the toxicity and activity of oral thalidomide in Kaposi' s sarcoma (KS) in a phase II dose-escalation study. Patients and Methods: Human immunodeficiency virus (HIV)-seropositive patie nts with biopsy-confirmed KS that progressed over the 2 months before enrol lment received an initial dose of 200 mg/d of oral thalidomide in a phase I I study. The dose was increased to a maximum of 1,000 mg/d for up to 1 year . Anti-HIV therapy was maintained during the study period. Toxicity, tumor response, immunologic and angiogenic factors, and virologic parameters were assessed. Results: Twenty patients aged 29 to 49 years with a median CD4 count of 246 cells/mm(3) (range, 14 to 646 cells/mm(3)) were enrolled, All patients wer e assessable for toxicity, and 17 for response, Drowsiness in nine and depr ession in seven patients were the most frequent toxicities observed. Eight (47%; 95% confidence interval [CI], 23% to 72%) of the 17 assessable patien ts achieved a partial response, and an additional two patients had stable d isease, Based on all 20 patients treated, the response rate was 40% (95% CI , 19% to 64%). The median thalidomide dose at the time of response wets 500 mg/d (range, 400 to 1,000 mg/d). The median duration of drug treatment was 6.3 months, and the median time to progression was 7.3 months. Conclusion: Oral thalidomide was tolerated in this population at doses up t o 1,000 mg/d for as long as 12 months and was found to induce clinically me aningful anti-KS responses in a sizable subset of the patients. Additional studies of this agent in KS are warranted.