Introduction: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired condition characterized by complement-mediated hemolysis that can lead to anemia and thrombosis. Pegcetacoplan is a C3/C3b inhibitor approved for PNH that can be self-administered at home with a subcutaneous (SC) infusion pump or with a newly approved on-body, single-use, SC injector. Injector advantages over manual injections include hands-free administration, constant pressure delivery, and hidden/retractable needle. This study aimed to assess user experience with the new pegcetacoplan injector quantitatively and qualitatively.

Methods: Participants for this cross-sectional mixed method (survey/interviews) study were recruited in the United States, between November 2023 and February 2025, if they were adult (≥21 years) with PNH diagnosed for ≥6 months, who self-administered pegcetacoplan for >3 months before switching to the new injector and using it for ≥6 weeks, and gave informed consent to participate. After completing a 5-minute web-based survey, participants took part in a qualitative, 45-minute semi-structured telephone interview to discuss and evaluate their experience using the new injector. Interview results were summarized quantitatively using Likert-scale rating questions (from 1 [worst] to 7 [best]) about patients' perceptions, experience, confidence, and satisfaction with the new pegcetacoplan Injector. Qualitative findings and supportive quantitative data were reported as captured for each respondent and assessed in aggregate using descriptive statistics.

Results: Twelve patients with PNH (mean age, 46 years) previously treated with C5is (eculizumab, n=5; ravulizumab, n=7) and on pegcetacoplan for a mean of 18.2 months were recruited; 1 patient who had limited experience with the injector did not complete the interview.

Average ratings (n=11) were 6.5 for overall satisfaction with the injector, 6.6 for overall confidence in administration, and 6.9 for likelihood to recommend injector to another patient (1 [worst] to 7 [best] rating scale). Patients' reported primary value of the injector was its convenience, achieved through straightforward set-up/ease of use and portability owing to the compact size and lack of loose parts. High ratings on ease of use were given because using the injector reduced administration burden, decreased medication errors, and built patients' confidence. The injector portability ratings were reflected by patients' reported perceptions of the injector improving mobility, protecting patient privacy, and mitigating the risks of needle puncture during administration.

Conclusions: Most patients with PNH were satisfied with multiple aspects of their experience with the pegcetacoplan injector. Patients perceived the injector positively as it provides significant benefits, improving their quality of life and reducing treatment burden. Similar on-body delivery systems used by patients with relapsed/refractory multiple myeloma (phase 2 IZALCO and phase 3 IRAKLIA trials) were associated with low infusion-reaction rates, potentially leading to improved long-term adherence, patient quality of life, and practice efficiency [Parmar G, et al. 2025 ASCO Annual Meeting; Ailawadhi S, et al. J Clin Oncol. 2025 Jun 3:JCO2500744].

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2025
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