Phase I study of human chorionic gonadotropin given subcutaneously to patients with acquired immunodeficiency syndrome-related mucocutaneous Kaposi's sarcoma

Background: In vitro and in vivo clinical studies have shown that certain preparations of human chorionic gonadotropin have antitumor activity against Kaposi's sarcoma, the most common tumor in patients infected with human immunodeficiency virus type 1 (HIV-1). Methods: A phase I trial was conducted in 18 male patients with acquired immunodeficiency syndrome-related Kaposi's sarcoma, Successive cohorts of six patients each received human chorionic gonadotropin (A.P.L.; Wyeth-Ayerst, Radnor, PA) subcutaneously at doses of 5000 IU daily (level I), 10000 IU three times a week (level II), or 10 000 IU daily (level III), Toxic effects, changes in reproductive hormone levels, HIV-1 RNA plasma levels, and response to therapy were evaluated, Results: A.P.L. treatment was well tolerated at all dose levels, and no maximum-tolerated, dose-defined toxic effects were observed at the highest dose tested, The most common side effects were weight gain, increased libido, and increased energy, A persistent increase in testosterone level and a persistent decline in luteinizing hormone and follicle-stimulating hormone levels were seen over time, Major responses were observed in six patients, Partial remissions (greater than or equal to 50% decrease in lesion numbers, volume, or surface area) were observed at dose level I and dose level II (two patients each); biopsy-confirmed complete remissions (resolution of all lesions) were observed at dose level III (two patients), All but one major response have persisted from 207 to more than 515 days, Nine patients had stable disease lasting 10 weeks or longer, Conclusions: A.P.L. given at daily doses ranging from 5000 to 10000 IU has antitumor activity in patients with acquired immunodeficiency syndrome-related Kaposi's sarcoma, A.P.L. can be given for more than 1 year with minimal side effects, Larger efficacy studies are warranted.