Topical Treatment of Cutaneous Lesions of Acquired Immunodeficiency Syndrome–Related Kaposi Sarcoma Using Alitretinoin Gel: Results of Phase 1 and 2 Trials

OBJECTIVE To evaluate the efficacy and safety of topical alitretinoin gel (9-cis-retinoic acid [LGD1057], Panretin gel; Ligand Pharmaceuticals, Inc, San Diego, Calif) in cutaneous Kaposi sarcoma (KS). DESIGN Open-label, within-patient, controlled, dose-escalating phase 1 and 2 clinical trials. In all patients, 1 or more cutaneous KS lesions were treated with alitretinoin gel, and at least 2 other lesions served as untreated controls for up to 16 weeks. Alitretinoin (0.05% or 0.1% gel) was applied twice daily for the first 2 weeks and up to 4 times daily thereafter, if tolerated. SETTING Nine academic clinical centers. PATIENTS One hundred fifteen patients with biopsy-proven acquired immunodeficiency syndrome (AIDS)–related KS. MAIN OUTCOME MEASURES AIDS Clinical Trials Group response criteria. RESULTS Statistically significant clinical responses were observed in 31 (27%) of 115 patients for the group of treated index lesions compared with 13 (11%) for the group of untreated control lesions (P