The PROVe Study: US Real-World Experience with Chlormethine/Mechlorethamine Gel in Combination with Other Therapies for Patients with Mycosis Fungoides Cutaneous T-Cell Lymphoma
- PMID: 33656660
- PMCID: PMC8068681
- DOI: 10.1007/s40257-021-00591-x
The PROVe Study: US Real-World Experience with Chlormethine/Mechlorethamine Gel in Combination with Other Therapies for Patients with Mycosis Fungoides Cutaneous T-Cell Lymphoma
Erratum in
-
Correction to: The PROVe Study: US Real-World Experience with Chlormethine/Mechlorethamine Gel in Combination with Other Therapies for Patients with Mycosis Fungoides Cutaneous T-Cell Lymphoma.Am J Clin Dermatol. 2022 May;23(3):425. doi: 10.1007/s40257-022-00676-1. Am J Clin Dermatol. 2022. PMID: 35292936 Free PMC article. No abstract available.
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.
Conflict of interest statement
E.J. Kim has received research support from and/or acted as principal investigator for Actelion, Galderma, MedImmune, and Soligenix; has received consultant, speaking, or travel support from Actelion, Galderma, Helsinn, and Soligenix; and has served on a scientific advisory board for Helsinn and Kyowa Kirin. J. Guitart has received research support from Galderma and Soligenix and served on a scientific advisory board for Helsinn, Kyowa Kirin, miRagen, and Leo Pharma. C. Querfeld has received a research grant from Celgene; acted as clinical investigator for Celgene, Trillium, miRagen, Bioniz, and Kyowa Kirin; and served on a steering committee or advisory board for Helsinn/Actelion, miRagen, Bioniz, Trillium, and Kyowa Kirin. M. Girardi has served on an advisory board for Helsinn and Mallinckrodt and received research support and/or acted as principal investigator for AbbVie and Soligenix. A. Musiek has acted as investigator for Elorac, Soligenix, miRagen, Pfizer, Menlo, and Connect and served on an advisory board for Kyowa and Helsinn. O.E. Akilov has received research support from Trillium Therapeutics, Kyowa Kirin, and Pfizer; served as a clinical investigator for Celgene, Trillium Therapeutics, Bioniz, Kyowa, Soligenix, Galderma, and Innate Pharma; and received consulting fees or honorarium from Kyowa Kirin, Bioniz, and Dr. Reddy’s. J.T. Angello and W.L. Bailey are employed by Helsinn Therapeutics (U.S.), Inc. L.J. Geskin has received a research grant from Actelion; consulting fees or honorarium from Actelion, Helsinn, Mallinckrodt, Sanofi, and Regeneron; and served on a speakers’ bureau for Helsinn.
Figures
![Fig. 1](https://cdn.statically.io/img/www.ncbi.nlm.nih.gov/pmc/articles/instance/8068681/bin/40257_2021_591_Fig1_HTML.gif)
Similar articles
-
Management of Mycosis Fungoides with Topical Chlormethine/Mechlorethamine Gel: A Columbia University Cutaneous Lymphoma Center Experience.Acta Derm Venereol. 2021 Sep 8;101(9):adv00544. doi: 10.2340/00015555-3911. Acta Derm Venereol. 2021. PMID: 34436621 Free PMC article.
-
Evaluating the Treatment Patterns of Chlormethine/Mechlorethamine Gel in Patients With Stage I-IIA Mycosis Fungoides: By-time Reanalysis of a Randomized Controlled Phase 2 Study.Clin Lymphoma Myeloma Leuk. 2021 Feb;21(2):119-124.e4. doi: 10.1016/j.clml.2020.11.022. Epub 2020 Dec 3. Clin Lymphoma Myeloma Leuk. 2021. PMID: 33358692
-
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. doi: 10.1001/2013.jamadermatol.541. JAMA Dermatol. 2013. PMID: 23069814 Free PMC article. Clinical Trial.
-
Clinical potential of mechlorethamine gel for the topical treatment of mycosis fungoides-type cutaneous T-cell lymphoma: a review on current efficacy and safety data.Drug Des Devel Ther. 2018 Jan 31;12:241-254. doi: 10.2147/DDDT.S137106. eCollection 2018. Drug Des Devel Ther. 2018. PMID: 29440874 Free PMC article. Review.
-
Topical chlormethine gel in the treatment of mycosis fungoides: A single-center real-life experience and systematic review of the literature.Dermatol Ther. 2022 Sep;35(9):e15683. doi: 10.1111/dth.15683. Epub 2022 Jul 27. Dermatol Ther. 2022. PMID: 35778940 Review.
Cited by
-
Complete Remission of Tumour-Stage Mycosis Fungoides with Combined Chlormethine Gel and Local Low-Dose Radiotherapy in a Patient Under Extracorporeal Photopheresis.Indian J Dermatol. 2023 Nov-Dec;68(6):700-702. doi: 10.4103/ijd.ijd_709_23. Epub 2024 Jan 9. Indian J Dermatol. 2023. PMID: 38371586 Free PMC article. No abstract available.
-
Expert opinions and clinical experiences with chlormethine gel as maintenance treatment for patients with mycosis fungoides.Front Med (Lausanne). 2024 Jan 18;10:1298988. doi: 10.3389/fmed.2023.1298988. eCollection 2023. Front Med (Lausanne). 2024. PMID: 38304309 Free PMC article.
-
Chlormethine gel in combination with other therapies for treatment of mycosis fungoides: a review with patient cases.Front Med (Lausanne). 2024 Jan 11;10:1308491. doi: 10.3389/fmed.2023.1308491. eCollection 2023. Front Med (Lausanne). 2024. PMID: 38274457 Free PMC article. Review.
-
Effectiveness and tolerability of chlormethine gel for the management of mycosis fungoides: a multicenter real-life evaluation.Front Oncol. 2024 Jan 4;13:1298296. doi: 10.3389/fonc.2023.1298296. eCollection 2023. Front Oncol. 2024. PMID: 38239642 Free PMC article.
-
Advances in the treatment of mycoses fungoides and Sézary syndrome: a narrative update in skin-directed therapies and immune-based treatments.Front Immunol. 2023 Oct 5;14:1284045. doi: 10.3389/fimmu.2023.1284045. eCollection 2023. Front Immunol. 2023. PMID: 37868986 Free PMC article. Review.
References
-
- Willemze R, Hodak E, Zinzani PL, Specht L, Ladetto M, ESMO Guidelines Committee. Primary cutaneous lymphomas: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2018;29(suppl 4):iv30–40. - PubMed
-
- National Comprehensive Cancer Network. NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®). Primary cutaneous lymphomas. Version 1.2020. 2020. https://www.nccn.org/professionals/physician_gls/default_nojava.aspx. Accessed 25 Jun 2020.
Publication types
MeSH terms
Substances
Grants and funding
LinkOut - more resources
Full Text Sources
Other Literature Sources
Medical