OBJECTIVES: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood condition, caused by complement-mediated hemolysis with persistent anemia and fatigue, which has a negative impact on patients. Currently approved treatments include complement 5 inhibitors (C5i). The study aim was to assess the humanistic burden of PNH from the patient perspective.

METHODS: Data were drawn from the Adelphi PNH Disease Specific Program™, a cross-sectional survey of PNH-treating physicians and their patients from Canada, France, Germany, Italy, Japan, Spain, and the USA (collected Jan-July 2022). Patients completed a survey comprising demographics, PNH-related symptoms, FACIT-Fatigue (recall over 7 days, score ranging 0-52), Short Form Survey (SF-36) allowing the calculation of mental and physical component summary scores (MCS, PCS), EQ-5D-5L. Lower scores indicate worse outcomes for all instruments. EQ-5D utility scores were calculated using UK tariffs. Physicians reported patient’s current treatment, dates of diagnosis and treatment initiation. Descriptive analyses were conducted on data from patients treated with C5i.

RESULTS: Overall, 143 PNH patients on C5i were analyzed; mean (SD) age 48.2 (14.8) years (median 47.0) and 52% were male. Mean (SD) disease duration was 2.5 (2.8) years and duration on C5i therapy was 1.8 (2.1) years. The majority of patients (60%) reported their most bothersome symptom as tiredness/lack of energy. The FACIT-Fatigue score was 36.3 (10.1), the MCS 44.0 (8.7), PCS 43.9 (9.7), EQ-5D-VAS of 72.1 (15.4) and utility of 0.77 (0.2). In the EQ-5D-5L, 30% of patients reported moderate-severe anxiety/depression and 22% reported moderate-severe problems doing their usual activities.

CONCLUSIONS: Despite current treatment with C5i, many PNH patients continue to experience symptoms including tiredness/lack of energy, with low mental, physical status and utility scores reported. Better therapies capable of providing comprehensive control of PNH may address the remaining clinical and humanistic burden.