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Lessin SR , Duvic M , Guitart J , Pandya AG , Strober BE , Olsen EA , Hull CM , Knobler EH , Rook AH , Kim EJ , Naylor MF , Adelson DM , Kimball AB , Wood GS , Sundram U , Wu H , Kim YH
Topical chemotherapy in cutaneous T-cell lymphoma: positive results of a randomized, controlled, multicenter trial testing the efficacy and safety of a novel mechlorethamine, 0.02%, gel in mycosis fungoides
JAMA Dermatol. 2013 Jan;149(1) :25-32
PMID: 23069814    PMCID: PMC 3662469   
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Abstract
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< .01). No drug-related serious adverse events were seen. Approximately 20.3% of enrolled patients in the gel treatment arm and 17.3% of enrolled patients in the ointment treatment arm withdrew because of drug-related skin irritation. No systemic absorption of the study medication was detected. CONCLUSION: The use of a novel mechlorethamine, 0.02%, gel in the treatment of patients with mycosis fungoides is effective and safe. TRIAL REGISTRATION: clinicaltrials.gov Identifier:NCT00168064.
Notes
Lessin, Stuart R Duvic, Madeleine Guitart, Joan Pandya, Amit G Strober, Bruce E Olsen, Elise A Hull, Christopher M Knobler, Elizabeth H Rook, Alain H Kim, Ellen J Naylor, Mark F Adelson, David M Kimball, Alexa B Wood, Gary S Sundram, Uma Wu, Hong Kim, Youn H R01 FD003017/FD/FDA HHS/United States Research Support, Non-U.S. Gov't Research Support, U.S. Gov't, P.H.S. United States JAMA dermatology (Chicago, Ill.) JAMA Dermatol. 2013 Jan;149(1):25-32. doi: 10.1001/2013.jamadermatol.541.