Bodsworth N J, Bloch M, Bower M, Donnell D, Yocum R
Taylor Square Private Clinic, Darlinghurst, New South Wales, Australia.
Am J Clin Dermatol. 2001;2(2):77-87. doi: 10.2165/00128071-200102020-00004.
This randomized, double-blind and vehicle-controlled phase III study was conducted to evaluate the efficacy and safety of alitretinoin gel 0.1% for the topical treatment of the cutaneous lesions of AIDS-related Kaposi's sarcoma (KS).
Patients received treatment with alitretinoin gel (n = 62) or vehicle gel (n = 72) twice daily for 12 weeks. The primary efficacy endpoint was the cutaneous KS tumor response rate according to AIDS Clinical Trials Group (ACTG) objective criteria applied to topical therapy, with the patient as the unit of analysis.
Treatment of patients with alitretinoin gel resulted in a significant antitumor effect. The overall patient response rate (complete plus partial response) was 37% (23 of 62) for the alitretinoin-treated patients and 7% (5 of 72) for the vehicle-treated patients (p = 0.00003). The difference in response rates for the 2 treatment groups remained significant even after taking into consideration numerous variables, including age (p = 0.00001), Eastern Cooperative Oncology Group (ECOG) status (p = 0.00002), CD4+ cell count (p = 0.00002), history of opportunistic infection (p = 0.00002), aggregate area of indicator lesions (p = 0.00005), number of raised indicator lesions (p = 0.00002), prior therapy for KS (p = 0.00003), and number of drugs (p = 0.00002) used in concomitant antiretroviral therapy. Generally, treatment with alitretinoin gel was well tolerated. The overall incidence of adverse events was similar for the 2 treatment groups. Adverse events related to treatment with alitretinoin gel tended to be mild to moderate in severity and limited to the site of application. The most frequent adverse event occurring at the application site following alitretinoin gel treatment was irritation coded as rash (32%).
The results of this study provide convincing evidence of the superiority of alitretinoin gel over vehicle gel for the treatment of the cutaneous lesions of AIDS-related KS.
本随机、双盲、赋形剂对照的III期研究旨在评估0.1%阿利维A酸凝胶局部治疗艾滋病相关卡波西肉瘤（KS）皮肤病变的疗效和安全性。
患者接受阿利维A酸凝胶治疗（n = 62）或赋形剂凝胶治疗（n = 72），每日两次，共12周。主要疗效终点是根据应用于局部治疗的艾滋病临床试验组（ACTG）客观标准得出的皮肤KS肿瘤反应率，以患者作为分析单位。
阿利维A酸凝胶治疗患者产生了显著的抗肿瘤效果。阿利维A酸治疗组患者的总体反应率（完全缓解加部分缓解）为37%（62例中的23例），赋形剂治疗组为7%（72例中的5例）（p = 0.00003）。即使考虑了众多变量，包括年龄（p = 0.00001）、东部肿瘤协作组（ECOG）状态（p = 0.00002）、CD4 + 细胞计数（p = 0.00002）、机会性感染病史（p = 0.00002）、指示病变的总面积（p = 0.00005）、隆起指示病变的数量（p = 0.00002）、KS的既往治疗（p = 0.00003）以及联合抗逆转录病毒治疗中使用的药物数量（p = 0.00002），两个治疗组的反应率差异仍然显著。总体而言，阿利维A酸凝胶治疗耐受性良好。两个治疗组不良事件的总体发生率相似。与阿利维A酸凝胶治疗相关的不良事件严重程度往往为轻至中度，且局限于用药部位。阿利维A酸凝胶治疗后在用药部位发生最频繁的不良事件是被编码为皮疹的刺激反应（32%）。
本研究结果提供了令人信服的证据，表明阿利维A酸凝胶在治疗艾滋病相关KS皮肤病变方面优于赋形剂凝胶。
