OBJECTIVES: Using patient-level data from an Adelphi Disease Specific Programme (DSP), we describe physician-reported and self-reported patient satisfaction with eculizumab or ravulizumab for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).

METHODS: The PNH Adelphi DSP is a point-in-time survey of hematologists and oncologists, and their consulting patients with PNH conducted in Australia, Austria, Canada, France, Germany, Italy, Spain, UK and USA between December 2021 and July 2022. Physicians provided data on patients’ demographics, symptoms, laboratory values, disease control and satisfaction with eculizumab or ravulizumab. Data quality checks were performed to exclude records with missing or incorrectly reported information, however, no source data verification was performed. The same patients were invited to provide self-reported data on their satisfaction with treatment. The current analysis matched patient and physician satisfaction responses, with satisfaction defined as a report of ‘completely satisfied’ or ‘satisfied’.

RESULTS: Of the 164 patients, majority were White/Caucasian (91.5%), 55.5% were males and the mean age (standard deviation) was 47.7 (14.5) years. Mean time (range) since PNH diagnosis was 2.3 (0.1–16.9) years and 24 patients (14.6%) had aplastic anemia or bone marrow disorder. For 93.2% and 97.9% of patients their physician was satisfied with eculizumab (n = 104) and ravulizumab (n = 47), respectively, whereas the patient-reported satisfaction was 84.6% and 91.5%, respectively. In patients treated for over 12 months, for 90.0% and 96.6% their physician was satisfied with eculizumab (n = 50) and ravulizumab (n = 29), respectively, whereas patient-reported satisfaction was 86.0% and 93.1%, respectively.

CONCLUSIONS: Most physicians and patients were satisfied with eculizumab and ravulizumab treatment for PNH in a real-world setting, and satisfaction was higher with ravulizumab than eculizumab. These results demonstrate perceptions and experiences of physicians who treat PNH and patients with PNH treated with eculizumab or ravulizumab and may improve the treatment decision process.