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, *
Identifiers and Pagination:Year: 2017
First Page: 98
Last Page: 107
Publisher Id: TODJ-11-98
Article History:Received Date: 29/09/2017
Revision Received Date: 05/12/2017
Acceptance Date: 14/12/2017
Electronic publication date: 28/12/2017
Collection year: 2017
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.
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.
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.
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.
IM gel warrants further investigation and development as a potential topical treatment for localized patch/plaque stage MF and FMF.