382 PHASE-III Randomized monocentric controlled trial comparing clinical and cost-effectiveness analysis of botulinum toxin uterine injections versus placebo for severe dysmenorrhea and chronic pelvic pain from uterine origin after standard therapeutic failure

Abstract

Introduction and aims of the study: Severe dysmenorrhea and chronic pelvic pain (CPP) from uterine origin may be followed by psycho-social consequences and professional absenteism. Despite a negative magnetic resonance imaging and laparoscopy, current available treatments (analgesics, anti-inflammatory drugs and hormones) are ineffective with low quality of life gains. As published previously, injections of botulinum toxin (BT) type A under hysteroscopy into uterine myometrium revealed significant decrease of patient-reported symptoms and improvement of global quality of life scores at 8 and 12-week post-injection. The aims of this study are to evaluate clinical and cost-effectiveness of BT use for severe dysmenorrhea/CPP after treatment failure. Methods: Phase-III randomized monocentric controlled trial comparing BT versus placebo among 100 participants. Randomization will be centralized. Study investigators, analysis team, and subjects will remain blinded. Participants will be informed and will sign a written informed consent. Data will be collected and registered in a secure electronic platform before enrollment, at inclusion, 8-week and 4-month follow-up. Main clinical outcome: Patient Global Impression of Improvement. Secondary clinical outcomes: Health-Related Quality of Life (HRQoL) by Endometriosis Health Profile, Generic HRQoL EQ-5D-3L, Female Sexual Function Index, dysmenorrhea and dyspareunia numerical rating scale, pain visual analogue scale, side effects, drug tolerance, surgical complications, global patient satisfaction. Effectiveness and costs outcomes: quality-adjusted life years (QALYs), direct and indirect costs, net social benefit, incremental cost-effectiveness ratios. Discussion: This study will provide the most reliable evidence on the effectiveness of a novel therapy for patients suffering from severe dysmenorrhea and CPP from uterine origin, in the event of standard therapeutic failure. The health economic evaluation will give evidence to guide collective decision-making on marketing authorization for this clinical indication. Conclusions: Evidence-based clinical and cost-effectiveness of BT will guide the proposal and implementation of a new treatment for severe dysmenorrhea and CPP from uterine origin.