Abstract
Background: Chlormethine/mechlorethamine gel is a skin-directed therapy for patients with mycosis fungoides cutaneous T-cell lymphoma. Currently, real-world data on chlormethine gel are lacking. Objective: Our objective was to analyze the effect of chlormethine gel in combination with other therapies on efficacy, safety, and health-related quality of life in a real-world setting. Methods: This prospective, observational study enrolled adult patients actively using chlormethine gel. Patients were monitored for up to 2 years during standard-of-care clinic visits. No specific visit schedules or clinical assessments, with the exception of patient-completed questionnaires, were mandated because of the expected variability in practice patterns. The primary efficacy endpoint was the proportion of patients with stage IA–IB disease receiving chlormethine + topical corticosteroids + other with ≥ 50% decrease in body surface area from baseline to 12 months. Response was assessed at each visit using by-time analysis, which investigates the trend to treatment response and allows assessment of response over time. Health-related quality of life was assessed with the Skindex-29 questionnaire. Results: In total, 298 patients were monitored. At 12 months post-treatment initiation, 44.4% (chlormethine + topical corticosteroids + other) and 45.1% (patients receiving chlormethine + other treatment) of efficacy-evaluable patients were responders. By-time analysis demonstrated that peak response occurred (chlormethine + other; 66.7%) at 18 months. There was a significant correlation between responder status and lower post-baseline Skindex-29 scores. Conclusions: This real-world study confirmed that chlormethine gel is an important therapeutic option for patients with mycosis fungoides and contributes to reducing the severity of skin lesions and improving health-related quality of life.
| Original language | English (US) |
|---|---|
| Pages (from-to) | 407-414 |
| Number of pages | 8 |
| Journal | American Journal of Clinical Dermatology |
| Volume | 22 |
| Issue number | 3 |
| DOIs | |
| State | Published - May 2021 |
Funding
The authors acknowledge and thank the volunteers, investigators, and the study teams at the centers participating in this study. The PROVe investigators collected data and provided and cared for study patients. Expert advice was provided by Prof. Youn H. Kim from Stanford Cancer Institute, Stanford, CA, USA. Statistical programming and analysis were overseen by Michael J. Williams, PhD, and David Mink, MS, from ICON Commercialization & Outcomes, Dublin, Ireland. Editorial and medical writing assistance was provided by Judith Land, PhD, from Aptitude Health, The Hague, The Netherlands, funded by Helsinn Healthcare SA. The authors are fully responsible for all content and editorial decisions for this manuscript.
ASJC Scopus subject areas
- Dermatology