Clinical Profile, Humanistic and Economic Burden of Paroxysmal Nocturnal Hemoglobinuria in Patients Treated With C5...

To evaluate disease burden among paroxysmal nocturnal hemoglobinuria (PNH) patients prescribed C5 inhibitors (C5i).

Data were drawn from the Adelphi Real World PNH Disease Specific Programme, a cross-sectional survey of physicians and PNH patients in Australia, Canada, France, Germany, Italy, Spain, the United Kingdom, and Japan from January-December 2022. Physicians reported patient demographics, laboratory parameters, and treatments. Patients completed the EQ-5D-5L, Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-Fatigue), and Work Productivity and Activity Impairment (WPAI) questionnaire. Analyses were descriptive.

Overall, 81 physicians reported on 288 patients receiving C5i. Median (IQR) age was 50.0 (38.3-65.0) years, 79.2% were white, and 54.5% were male. Median (IQR) C5i duration was 1.0 (0-3.0) years; 77.4% received eculizumab, 22.6% ravulizumab. At time of survey, median (IQR) hemoglobin (Hb) was 11.0 (9.9-12.0) g/dL, 73.5% of patients had Hb < 12 g/dL. Lactate dehydrogenase was >1.5× upper limit of normal for 16.7% of patients. Mean (SD) EQ-5D-5L was 0.76 (0.20), FACIT-Fatigue was 36.1 (9.7), WPAI work impairment was 27.5% (22.3) and activity impairment was 35.3% (24.6).

Despite C5i treatment, a notable proportion of patients remained anemic and reported impaired quality of life, indicating the need for novel, efficacious therapies.