Therapeutic Management and Healthcare Resource Use (HCRU) of Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) in France Between 2018 and 2022: Results of the Hogan Study

OBJECTIVES: Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired clonal disease of hematopoietic stem cells. The availability of C5 inhibitors (anti-C5) has considerably improved patients' overall survival and quality of life. The aim of this study is to describe hospital management and HCRU of patients with PNH in France.

METHODS: This real-life retrospective study was based on the French hospital database (PMSI). Pediatric and adult patients hospitalized at least once for PNH (ICD-10 code D595), between 2018 and 2022, were included. Exposure to anti-C5 drugs (ravulizumab, eculizumab) was observed over the entire extraction period (2013-2022). Insufficient responders to anti-C5 were defined by an increased number of transfusions more than 6 months after anti-C5 initiation or by a switch to anti-C3 (pegcetacoplan).

RESULTS: A total of 910 patients were identified with at least one stay for PNH between 2018 and 2022, including 531 exposed to anti-C5, of whom 23% were insufficient responders (defined as an intensification of transfusions after 6 months of treatment or a switch to anti-C3). Among the 183 patients newly exposed to anti-C5 with minimum of 6 months of follow-up, the average number of hospitalizations over the study period was 48,2 (±32,7), with a mean duration of 1.4 (±3,5) days, mainly related to anti-C5 administrations (82,3%). Fifteen percents (N=66) of incident patients (N=446) were diagnosed with bone marrow failure during follow-up period with a mean time (sd) of 141 (±323) days following the first PNH hospital stay. The average annualized cost of all hospitalizations for incident patients with PNH was €233 622,59 (±123 311,81) for patients exposed to anti-C5 and €297 809,80 (±94 103,73) for patients with insufficient response to anti-C5.

CONCLUSIONS: This study, based on real-life data from 2018 up to 2022, provides updated knowledge on the therapeutic management and HCRU for PNH patients in France.