Patient Experience Interviews with a Novel Treatment Iptacopan for Paroxysmal Nocturnal Hemoglobinuria

Introduction: Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired clonal and complex disorder characterized by hemolytic anemia, thrombosis, smooth muscle dystonia, hemoglobinuria, and chronic kidney disease. Individuals with PNH experience severe and chronic fatigue, which can significantly impact their quality of life. While two anti-C5 antibody therapies have been approved for treating PNH (eculizumab and ravulizumab), the majority of patients who use these therapies still do not achieve normal hemoglobin levels. Iptacopan, the first oral proximal complement inhibitor developed to fill this critical need, has shown in two Phase III trials (APPLY-PNH [NCT04558918] and APPOINT PNH [NCT04820530]) to achieve a comprehensive control of hemolysis. As part of the trials, patient experience interviews were conducted with patients entering an iptacopan extension study (CLNP023C12001B) to explore their experience with the treatment.

Objectives: To conduct patient experience interviews across 5 countries to understand and document how patients' PNH disease experience changed in response to treatment, the meaningfulness of any reported changes, patients' experience receiving the iptacopan treatment, patients' preference for iptacopan compared to other PNH treatments, and their satisfaction with treatment.

Methodology: Patients were recruited by clinical sites in the United States, United Kingdom, France, Italy, and Germany enrolled in the iptacopan extension study. Patients enrolled in the iptacopan extension study were eligible to consent to and participate in an optional 30-minute qualitative interview. Interviews were conducted by independent, trained interviewers in the patient's local language. A semi-structured interview guide was used to ask patients open-ended questions to elicit spontaneous descriptions of their disease, and treatment experience, including satisfaction and preferences. Interviews were audio-recorded, transcribed verbatim, translated into English (if applicable), anonymized, coded, and then analyzed using qualitative data analytic methods.

Results: Interviews were completed with 22 patients diagnosed with PNH across 5 countries. Following treatment with iptacopan, patients reported improvements in their PNH-related signs, symptoms, and impacts that they experienced before treatment. The most common symptoms that patients reported improvements in were fatigue (n=13/13, 100.0%), tiredness (n=11/12, 91.7%), and low energy (n=10/10, 100.0%). Patients most commonly reported their ability to work (n=5/8, 62.5%), ability to walk (n=4/8, 50.0%), and feelings of unhappiness (n=7/8, 87.5%) improved with treatment. Patients commented that these improvements in PNH-related signs, symptoms, and impacts were meaningful to them.

When discussing their treatment experience with iptacopan, patients reported that they liked the convenience of the oral treatment (n=18, 81.8%), treatment efficacy (n=13, 59.1%), and reduced medical appointments (n=8, 36.4%). Additionally, patients described the oral administration as convenient (n=18, 81.8%) and the treatment schedule easy to follow (n=17, 77.3%). Some patients disliked the frequent treatment schedule in the trial (n=7, 31.8%). Of the 18 patients who were asked, all (n=18/18, 100.0%) reported they preferred iptacopan over previous PNH treatments overall, including eculizumab and ravulizumab (Table 1). Compared to previous anti-C5 PNH treatments, most patients preferred the iptacopan treatment administration (n=20/20, 100.0%) and treatment schedule (n=16/20, 80.0%). Overall, patients reported being very satisfied (n=14, 63.6%) or satisfied (n=8, 36.3%) with their iptacopan treatment experience (Table 2).

Conclusion: These qualitative interviews provide evidence that patients were satisfied with their iptacopan treatment experience, and prefer iptacopan over other PNH treatments. Patients preferred iptacopan for the convenience of treatment administration and treatment schedule, the ease of following the treatment regimen, and liked the treatment for the oral administration, treatment efficacy, and reduced medical appointments. Additionally, this data demonstrates significant and meaningful improvement in patients' PNH-related signs, symptoms, and impacts with iptacopan.