Activity of Thalidomide in AIDS-Related Kaposi’s Sarcoma

• DOI: 10.1200/jco.2000.18.13.2593
• Publication Date: 2017-02-24T09:08:21Z
• Authors: Richard F. Little; Kathleen M. Wyvill; James M. Pluda; Lauri Welles; Vickie Marshall; William D. Figg; Fonda M. Newcomb; Giovanna Tosato; Ellen Feigal; Seth M. Steinberg; Denise Whitby; James J. Goedert; Robert Yarchoan
• Keywords: Adult; Male; 0301 basic medicine; Acquired Immunodeficiency Syndrome; Anti-HIV Agents; Administration, Oral; Middle Aged; Thalidomide; 3. Good health; 03 medical and health sciences; Disease Progression; Humans; Sarcoma, Kaposi

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 patients with biopsy-confirmed KS that progressed over the 2 months before enrollment received an initial dose of 200 mg/d of oral thalidomide in a phase II 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/mm3(range, 14 to 646 cells/mm3) were enrolled. All patients were assessable for toxicity, and 17 for response. Drowsiness in nine and depression in seven patients were the most frequent toxicities observed. Eight (47%; 95% confidence interval [CI], 23% to 72%) of the 17 assessable patients achieved a partial response, and an additional two patients had stable disease. 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 was 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 to 1,000 mg/d for as long as 12 months and was found to induce clinically meaningful anti-KS responses in a sizable subset of the patients. Additional studies of this agent in KS are warranted.