The burden of illness of patients with paroxysmal nocturnal haemoglobinuria receiving C5 inhibitors in France, Germany and the United Kingdom: Patient‐reported insights on symptoms and quality of life

摘要

Abstract Objectives To assess the clinical, humanistic and economic burden of paroxysmal nocturnal haemoglobinuria (PNH) among C5 inhibitor (C5i)‐treated patients with PNH. Methods This was a web‐based, cross‐sectional survey (01FEB2021‐31MAR2021) of adults with PNH treated with eculizumab (France, Germany, United Kingdom) or ravulizumab (Germany). Self‐reported outcomes included: patient characteristics; patient‐reported symptoms; and standardised patient‐reported outcomes (e.g. Functional Assessment of Chronic Illness Therapy FACIT‐Fatigue, European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 EORTC QLQ‐C30). Results Among 71 included patients, 98.6 were C5i‐treated for ≥3?months (88.7 ≥12?months); among those with self‐reported haemoglobin (Hb) levels (n?=?63), most (85.7) were anaemic (defined as ≤12.0?g/dL). Fatigue was the most common symptom at both diagnosis (73.2) and survey time (63.4); there were no statistically significant differences in symptom prevalence between treatment subgroups (eculizumab vs. ravulizumab). Total FACIT‐Fatigue and EORTC QLQ‐C30 scores were substantially lower than European general population references, but there were no statistically significant differences between treatment subgroups. Hb‐level subgroups (<10.5?g/dL vs. ≥10.5?d/dL) followed similar trends for all measures, with few significant subgroup differences. Conclusions Results suggest that there remains a considerable burden and unmet need among C5i‐treated patients with PNH that requires improved therapies.