JMIR Publications

ABSTRACT

Background:

Extra-Mammary Paget's disease of the Vulva (EMPV) is a rare skin disorder, which is commonly seen in postmenopausal caucasian females and appears clinically as red, eczematous, pruriginous and sometimes painful lesions. Although most cases are non- invasive, EMPV may be associated with an underlying or distant adenocarcinoma. EMPV has a chronic and relapsing course. The reference treatment is based on local surgical excision with negatives margins. However, disease frequently extends far from the visible lesion and surgical margins are frequently positive. Topical photodynamic therapy (PDT) is an established treatment modality for various dermato-oncologic conditions. For example, the red light irradiation with the Aktilite® CL128 and Metvixia® as PS (Galderma SA, Lausanne, Switzerland) is a conventional protocol approved and widely used in Europe for PDT treatment of actinic keratosis but this treatment is not yet widely used for the EMPV because it has never clearly been demonstrated and it is very painful.

Objective:

The aim of the study is to investigate the efficacy and safety relating to the medical device “PAGETEX®” as a new photodynamic therapy device using Metvixia® in the treatment of vulvar Paget disease. The primary endpoint is the disease control rate at 3 months in 30% of the patients included, defined as stability, partial response or total response, considering the extent of the lesion. Secondary endpoints are the disease control rate at 6 months, the patient quality of life, the level of pain experienced by the patient at each PDT session, the severity of erythema, the presence of PpIX in Paget’s cells after each PDT session and the overall satisfaction level of the patient.

Methods:

The trial is an interventional, exploratory, simple group, non-randomized and single center (Lille University Hospital) study. Twenty four patients will be included according to Simon's optimal plan. Therapeutic procedure is based on a cycle of two PDT sessions with the PAGETEX® medical device at 15-days intervals (Metvixia incubation during 30 minutes and 635 nm red light illumination with a low irradiance during 2h30 for a total fluence of 12 J/cm²). At the assessment session, 3 months after inclusion, if the control of the disease is partial or null, the patient will complete another cycle of two PDT sessions. A final evaluation will be performed in all patients at 6 months. Analyzes will be performed using the SAS software version 9.4 (SAS Institute Inc). The characteristics of the patients at baseline will be described; qualitative variables will be described by numbers and percentages, and quantitative variables will be described by the mean and standard deviation for Gaussian distribution, or by the median and interquartile (i.e. 25th and 75th percentiles) if not. The normality of the distributions will be tested by a Shapiro-Wilk test and checked graphically by histograms.

Results:

Clinical investigations are planned for July 2019 and concluding in August 2022. The final results of this study are expected to be available in January 2023.

Conclusions:

This phase II clinical trial aims to evaluate the efficacy and security of a new photodynamic therapy protocol for the treatment of the EMPV. PAGETEX® device could become the treatment of choice if it is effective and easy to implement and use in hospitals Clinical Trial: N° RCB 2018-A01873-52 & N° EudraCT : 2018-002604-1 Clinicaltrials.gov NCT03713203 Protocol version n°1.0 (20/06/2018)