Prospective Pilot Evaluation of the Efficacy and Safety of Topical Ingenol Mebutate Gel for Localized Patch/Plaque Stage Mycosis Fungoides



Eve Lebas1, Charlotte Castronovo1, Jorge E. Arrese2, Florence Libon1, Nazli Tassoudji1, Laurence Seidel3, Arjen F. Nikkels1, *
1 Dermatology (Dermato-oncology unit), University Hospital Centre, CHU du Sart Tilman, Liège, Belgium
2 Dermatopathology, University Hospital Centre, CHU du Sart Tilman, Liège, Belgium
3 Biostatistics University Hospital Centre, CHU du Sart Tilman, Liège, Belgium


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© 2017 Lebas et al.

open-access license: This is an open access article distributed under the terms of the Creative Commons Attribution 4.0 International Public License (CC-BY 4.0), a copy of which is available at: (https://creativecommons.org/licenses/by/4.0/legalcode). This license permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.

* Address correspondence to this author at the Department of Dermatology, CHU of Sart Tilman, University of Liège, B-4000, Liège, Belgium; Tel: +32-43667232; Fax: +32-43667234; E-mail: af.nikkels@chu.ulg.ac.be


Abstract

Background:

Mycosis Fungoides (MF) is the most frequent type of the primary cutaneous NK/T-cell lymphomas. Ingenol mebutate (IM) displays in vitro pro-apoptotic properties on neoplastic lymphocytes.

Objectives:

To evaluate the efficacy and safety of IM gel as topical treatment for MF.

Materials and Methods:

Ten male patients with longstanding classic type MF (n=9) and follicular MF (FMF; n=1), T2bN0M0B0, stage Ib, resistant to systemic methotrexate or acitretin therapies for at least 3 months, were included in this pilot study. In these patients, 11 target patch/plaque stage lesions with an area ≤ 25 cm2 were selected for IM therapy (0,05%, 2 weekly applications). The primary endpoint was the improvement of the CAILS scores. Biopsies were performed before and after treatment from 10 target lesions. Relapse rates were evaluated at 6 months.

Results:

The mean CAILS score of treated target lesions was reduced by 58.2%. The mean erythema, scaling and plaque elevation scores were improved by 73.6%, 93.9% and 97.9% (p<0.0001), respectively, while the lesion size remained unchanged (p=0.34). A complete or partial clearance of histological and immunohistochemical features was observed in 6/10 (60%) and 4/10 (40%) of the MF or FMF target lesions, respectively. Monoclonal TCR rearrangement was evidenced in 100% (7/7) of the patients and in 3/7 (43%) after treatment. The relapse rate at 6 months was 18%. All the patients experienced burning sensations, oozing and crusting.

Conclusion:

IM gel warrants further investigation and development as a potential topical treatment for localized patch/plaque stage MF and FMF.

Keywords: Ingenol Mebutate, Mycosis Fungoides, PCNKTCL, Alternative Topical Treatment Option.