Unmet medical needs in paroxysmal nocturnal hemoglobinuria treatment: Insights from the SATURNO Study on C5 inhibitor therapy in Spain

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematological disorder. Benefits of current C5 inhibitor (C5i) treatments are indisputable, but some symptoms and impairment of daily quality of life persist. Objective : To assess satisfaction of PNH patients and hematologists with C5i treatment. Study Design : Spanish observational, cross-sectional, retrospective study. Demographic, clinical, biochemical, and treatment information was collected. Patients’ and physicians’ treatment satisfaction, fatigue, Health-Related Quality of Life (HRQoL), work productivity and activity impairment were assessed using standardized questionnaires. Results : Fifty-six adult PNH patients under C5i treatment were included. Median time since diagnosis was 11.7 years, and 6.6 years on C5i treatment. At the time of the study entry, 58.9% of patients were on eculizumab treatment and 41.1% of patients were on ravulizumab standard dosing. Of these, 50% switched to another C5 inhibitor, and 62.5% increased the dose. Most patients (94.6%) and hematologists (90.6%) were satisfied with the treatment, despite persistence of fatigue (55.2%), dyspnea (20.7%) and hemoglobinuria (13.8%). Hemoglobin (Hb) levels were below the lower limit of normal (LLN) in 67.9% of patients and 14.8% of patients still had elevated lactate dehydrogenase (LDH) levels > 1.5 times the upper limit of normal (ULN). The scores on HRQoL and fatigue questionnaires revealed a high impact of symptoms on their HRQoL. Conclusions : Despite improvements in PNH management, C5i do not completely normalize laboratory values related to hemolysis and anemia. The persistence of some symptoms and daily quality of life impairment reveals unmet needs. Future therapies should address the medical needs highlighted by our study.