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

Achieving clinically meaningful response in endometriosis pain symptoms is associated with improvements in health-related quality of life and work productivity: analysis of 2 phase III clinical trials

“…This is an important finding, given the subjectivity of clinical endpoints for endometriosis treatments and is in line with patient-centered drug development. 10 Interventions should address the patient experience, and these results provide further evidence that the EHP-30 is well suited for monitoring patient treatment goals.…”

“…3,5,15,16 This post hoc analysis provided substantial evidence that demonstrated that significantly lowering moderate to severe endometriosis-related pain levels results in significant improvements in HRQoL and productivity. A greater proportion of women who were clinical responders to treatment, as defined by DYS and NMPP in the EM-I and EM-II studies, achieved the thresholds for clinical meaningfulness 10 for the EHP-30 domains and the sexual relationship module when compared with women who were not clinical responders, indicating treatment efficacy across all the domains. While some DYS or NMPP nonresponders meet the thresholds for clinical meaningfulness, the group LS means for the EHP-30 domain scores did not indicate a change in domain score that met the threshold for clinical meaningfulness.…”

“…A study based on EM-I and EM-II recently established the score changes, by domain, that indicates a treatment response for the EHP-30 in this patient population. 10 To arrive at responder thresholds for the EHP-30 domains, a 3method approach, or triangulation approach, 11 was used. Specifically, anchor-based, distribution-based, and endpoints that are clinically relevant data were utilized to recommend the threshold score changes that indicate an EHP-responder.…”

Clinically Meaningful Reduction in Dyspareunia Is Associated with Significant Improvements in Health-Related Quality of Life Among Women with Moderate to Severe Pain Associated with Endometriosis: A Pooled Analysis of Two Phase III Trials of Elagolix

“…Based on a previously reported EHP-30 responder threshold, 17 all improvements in EHP-30 domain scores obtained by dyspareunia responders at months 3 and 6 relative to baseline were clinically meaningful ( Table 4). The months 3 and 6 decreases for dyspareunia responders were greatest for the control and powerlessness (42.7 and 43.6 points, respectively) and pain (34.7 and 35.4 points, respectively) domains.…”

“…For sexual intercourse, there was a 29.3-and 30.8-point decrease at months 3 and 6, respectively, for responders, which was greater than the 20-point threshold for clinical meaningfulness. 17 None of the Table 2. Baseline EHP-30 domain scores for responders and non-responders at month 3 and month 6 changes in EHP-30 scores at months 3 and 6 for non-responders reached threshold levels.…”