Results of a randomized study of IM862 nasal solution in the treatment of AIDS-related Kaposi's sarcoma

Purpose: Although advances have been made in the treatment of AIDS-related Kaposi's sarcoma (AIDS-KS) with systemic chemotherapy, less toxic therapies are needed. lM862 is a naturally occurring peptide with antiangiogenic pro perties and was thus studied in patients with AIDS-KS. Patients and Methods: lM862 was given as intranasal drops at a dose of 5 mg . patients were randomized to two dosing schedules given in repeated cycles until disease progression or unacceptable toxicity: 5 days of therapy foll owed by 5 days off (n = 18) and every other day dosing (n = 26). Results: Forty-two male patients and two female patients with a median age of 38 years (range, 22 to 53 years) were accrued. Twenty-one patients (47%) had more than 50 mucocutaneous lesions, 14 (32%) had lymphedema, and none had visceral involvement. Thirty-three patients (75%) had received prior sy stemic chemotherapy. Twenty-four patients (55%) had CD4(+) lymphocyte count less than or equal to 200/mm(3). All but five patients were being treated with concurrent protease inhibitor(s), for a median of 10 months (range, 0 to 24 months). Major responses were documented in 36%, with five complete a nd 11 partial remissions, occurring after a median of 6 weeks (range, 3 to 26 weeks) and lasting a median of 33+ weeks (range, 12+ to 95+ weeks). Twen ty one patients had stable disease for periods of 7 to 72+ weeks. Adverse e ffects to IM862 were limited to mild and transient headache, fatigue, tingl ing, and nausea. No hematologic adverse effects attributed to treatment wer e reported. Conclusion: lM862 given as intranasal drops is well tolerated and has antit umor activity in patients with AIDS-KS. A randomized double-blinded study t o define the activity of lM862 in patients with AIDS-KS is in progress. (C) 2000 by American Society of Clinical Oncology.