OBJECTIVES: PNH is a rare, chronic, and potentially fatal disease. It is caused by uncontrolled complement activation resulting in hemolysis, which clinically manifests as anemia, fatigue, and in severe cases, thrombosis. Current treatments (e.g., C3/C5 inhibitors) inhibit complement activation to manage complement-driven hemolysis and associated symptoms. In this analysis, we assessed the clinical effectiveness of pegcetacoplan for real-world PNH patients in the USA.

METHODS: Data were drawn from the Adelphi PNH Disease Specific Programme™, a real-world point-in-time survey of hematologists and oncologists, and their next consulting PNH patients in the USA (January-November 2022). Patients were eligible for inclusion if prescribed pegcetacoplan for ≥6 months. Physicians recorded data on patient demographics, clinical markers, and treatment satisfaction. Descriptive statistics were reported.

RESULTS: Five physicians provided data on 22 PNH patients receiving pegcetacoplan for a median (SD) 10.3 (3.0) months. 90.9% had switched from a C5 inhibitor. Median (SD) age was 35.0 (10.8) and 59.1% were male. Median (SD) time since diagnosis was 2.4 (2.4) years. From pegcetacoplan initiation to point of study, physicians reported a 3.4 g/dL improvement in hemoglobin (8.2 g/dL vs. 11.6 g/dL, respectively), improvement in lactate dehydrogenase (LDH) (27.3% vs. 68.2% reporting LDH <1.5x ULN when ULN set to 250µL, respectively) and an increased proportion of patients experiencing no fatigue (0.0% vs. 59.1%, respectively). All patients were considered by their physicians to have ‘well/very well controlled’ disease at time of survey and were ‘satisfied/completely satisfied’ with their patients’ treatment. Physicians reported 86.4% of patients had ‘excellent/very good’ overall QoL.

CONCLUSIONS: These findings demonstrate the real-world effectiveness of pegcetacoplan through improvement in endpoints, in line with clinical trial findings. Satisfaction with treatment and disease control were reported favorably, highlighting the potential for pegcetacoplan to improve outcomes of current PNH patients. Further research is required to understand the long-term effectiveness of pegcetacoplan in the real-world.