Responsiveness Evaluation and Recommendation for Responder Thresholds for Endometriosis Health Profile-30: Analysis of Two Phase III Clinical Trials.

Abstract

Objective: To evaluate the responsiveness of the Endometriosis Health Profile-30 (EHP-30) and ascertain score changes that are indicative of response to treatment. A post hoc analysis of two Phase III, double-blind, placebo-controlled, randomized clinical trials among women with moderate-to-severe endometriosis-associated pain (Elaris Endometriosis I and II [EM-I and EM-II]). Materials and Methods: EHP-30 core items and sexual relationship module were administered at day 1, month 3 (M3), and month 6 (M6) to monitor patient-reported impacts of endometriosis-related pain. A seven-response level Patient Global Impression of Change (PGIC) was administered at M3 and M6. Dysmenorrhea (DYS), nonmenstrual pelvic pain (NMPP), and dyspareunia (DYSP) were collected using a daily diary. Three psychometric approaches, triangulation, were used to suggest responder thresholds for the EHP-30 domains. The three approaches were anchor- and distribution-based analyses and use of clinically relevant indicators (DYS, NMPP, DYSP). Results: EM-I and EM-II enrolled 871 and 815 women, respectively. All EHP-30 domains improved during the trials (M3, M6). Differences (p\u2009<\u20090.001) for all EHP-30 domains were found among the PGIC responses at M3 and M6, indicating greater change was associated with greater EHP-30 improvements. Large effect sizes were noted for all EHP-30 domains (EM-I range -0.59 to -1.80; EM-II range -0.52 to -1.59). EHP-30 thresholds of meaningful change ranged from -20 to -35, with greater changes indicating greater improvement in health status. Conclusion: Responder thresholds by EHP-30 domain are recommended to evaluate treatment efficacy. Clinicians can individualize goals of treatment by EHP-30 domain and track changes using the EHP-30.