Topical Chemotherapy in Cutaneous T-cell Lymphoma

作者：Stuart R. Lessin, Madeleine Duvic, Joan Guitart, Amit G. Pandya, Bruce E Strober, Elise A. Olsen, Christopher M. Hull, Elizabeth H. Knobler, Alain H. Rook, Ellen J. Kim, Mark F. Naylor, David M. Adelson, Alexa B. Kimball, Gary S. Wood, Uma N. Sundram, Hong Gyun Wu, Youn H. Kim · 发表于：JAMA Dermatology · 年份：2012 · DOI：10.1001/2013.jamadermatol.541 · 被引用次数：190 · 研究领域：Cutaneous lymphoproliferative disorders research、Lymphoma Diagnosis and Treatment、Nail Diseases and Treatments

OBJECTIVE: To evaluate the efficacy and safety of a novel mechlorethamine hydrochloride, 0.02%, gel in mycosis fungoides. DESIGN Randomized, controlled, observer-blinded, multicenter trial comparing mechlorethamine, 0.02%, gel with mechlorethamine, 0.02%, compounded ointment. Mechlorethamine was applied once daily for up to 12 months. Tumor response and adverse events were assessed every month between months 1 and 6 and every 2 months between months 7 and 12. Serum drug levels were evaluated in a subset of patients. SETTING: Academic medical or cancer centers. PATIENTS: In total, 260 patients with stage IA to IIA mycosis fungoides who had not used topical mechlorethamine within 2 years and were naive to prior use of topical carmustine therapy. MAIN OUTCOME MEASURES: Response rates of all the patients based on a primary clinical end point (Composite Assessment of Index Lesion Severity) and secondary clinical end points (Modified Severity-Weighted Assessment Tool and time-to-response analyses). RESULTS: Response rates for mechlorethamine gel vs ointment were 58.5% vs 47.7% by the Composite Assessment of Index Lesion Severity and 46.9% vs 46.2% by the Modified Severity-Weighted Assessment Tool. By the Composite Assessment of Index Lesion Severity, the ratio of gel response rate to ointment response rate was 1.23 (95% CI, 0.97-1.55), which met the prespecified criterion for noninferiority. Time-to-response analyses demonstrated superiority of mechlorethamine gel to ointment (P< ....