Patient-Reported Meaningful Change in Symptoms and Impacts of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Three Phase III Clinical Trials of Iptacopan.

Background: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematological disease, with symptoms including fatigue, difficulty breathing, and hemoglobinuria, which negatively affect health-related quality of life.

Objective: In-trial interviews with patients with PNH, enrolled across three clinical trials, were conducted to understand patient experiences and satisfaction after receiving iptacopan, a novel treatment for PNH.

Methods: Adult participants with PNH were recruited and consented into the qualitative interviews across three phase II/III iptacopan trials (NCT04558918, NCT04820530, and NCT04747613). Interview documents were developed and approved by the relevant ethics authorities in target countries. Trained qualitative interviewers used a semi-structured interview guide to elicit information on PNH-related symptoms and impacts participants experienced prior to the trial, how these changed after receiving iptacopan, and their treatment experience and satisfaction with iptacopan.

Results: Interviews were conducted with 61 participants in eight countries. Participants reported a broad range of symptoms and impacts associated with PNH, including fatigue, tiredness, frustration, difficulty doing usual activities, and limitation to social activities. Interview participants reported improvements in the signs, symptoms, and impacts of PNH after receiving iptacopan, and the majority of participants considered those improvements to be meaningful. Participants reported being either "very satisfied" or "satisfied" with their treatment experience of iptacopan.

Conclusion: Findings from these interviews provide valuable patient-reported data on the positive treatment experience and meaningful improvements in PNH symptoms and health-related quality-of-life impacts patients reported after receiving iptacopan. These qualitative reports from patients support and contextualize the positive efficacy results demonstrated in the three iptacopan clinical trials. NCT04558918 (16 Sep 2020), NCT04820530 (25 Mar 2021), NCT04747613 (09 Feb 2021).