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Abstract
There are few data on healthcare resource use and related costs of French haemophilia A (HA) and B (HB) patients. This study aimed to describe the profile of HA and HB patients, current disease management, clinical burden and costs. Data related to haemophilia patients of all ages alive on 1/1/2022 were extracted from the nationwide French claims database (SNDS). Patients were divided into four treatment groups: on-demand or prophylaxis with or without inhibitors. Haemophilia patients were compared with a control group (ratio 1:3) matched for age, gender and region using risk ratios (RR [95% confidence interval]). The annual direct health care costs per person were estimated. A total of 5,577 (HA) and 1,332 (HB) patients were included (mean age: 36.4 years). Most patients were treated on-demand (HA: 72.8%; HB: 76.6%) and a few had inhibitors (HA: 3.6%; HB: 1.1%). Overall, haemophilia clinical burden was significantly higher than among controls, in particular, mortality (RR:1.42 [1.04-1.92]), work disability (RR: 2.71 [2.22-3.30]), hospitalisation for major bleeding (RR:12.06 [8.67-16.80]), orthopaedic surgery (RR: 2.97 [2.65-3.32]) and hospitalisation all causes (RR: 2.44 [2.31-2.58]). This burden was more important in patients with inhibitors or treated in prophylaxis and was close for HA and HB patients. The annual per-person costs were €282,560 and €181,566 for HA and HB in prophylaxis without inhibitors, respectively. The population with inhibitors, although limited, had even much higher costs. The clinical burden and costs of haemophilia treatments may be very high especially in patients in prophylaxis and/or with inhibitors.
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