{"title":"Clinical and Economic Burden of Patients with Haemophilia A and B in France: Analysis of a Nationwide Claims Database.","authors":"Laurent Frenzel, Stéphane Bouée, Hervé Lilliu, Véronique Cahoreau, Nicolas Giraud, Stéphanie Delienne, Francis Fagnani, Juliette Cottin, Isabelle Bureau, Anaïs Reynaud, Yasmine Fahfouhi, Amélie Coumert, Maud Beillat, Nadir Mammar, Jérémie Rudant, Aurélien Lebreton","doi":"10.1111/hae.70041","DOIUrl":null,"url":null,"abstract":"<p><strong>Introduction: </strong>There are few data on healthcare resource use and related costs of French haemophilia A (HA) and B (HB) patients.</p><p><strong>Aims: </strong>This study aimed to describe the profile of HA and HB patients, current disease management, clinical burden and costs.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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.</p><p><strong>Conclusion: </strong>The clinical burden and costs of haemophilia treatments may be very high especially in patients in prophylaxis and/or with inhibitors.</p>","PeriodicalId":12819,"journal":{"name":"Haemophilia","volume":" ","pages":""},"PeriodicalIF":3.0000,"publicationDate":"2025-03-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Haemophilia","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1111/hae.70041","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"HEMATOLOGY","Score":null,"Total":0}
引用次数: 0
Abstract
Introduction: There are few data on healthcare resource use and related costs of French haemophilia A (HA) and B (HB) patients.
Aims: This study aimed to describe the profile of HA and HB patients, current disease management, clinical burden and costs.
Methods: 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.
Results: 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.
Conclusion: The clinical burden and costs of haemophilia treatments may be very high especially in patients in prophylaxis and/or with inhibitors.
期刊介绍:
Haemophilia is an international journal dedicated to the exchange of information regarding the comprehensive care of haemophilia. The Journal contains review articles, original scientific papers and case reports related to haemophilia care, with frequent supplements. Subjects covered include:
clotting factor deficiencies, both inherited and acquired: haemophilia A, B, von Willebrand''s disease, deficiencies of factor V, VII, X and XI
replacement therapy for clotting factor deficiencies
component therapy in the developing world
transfusion transmitted disease
haemophilia care and paediatrics, orthopaedics, gynaecology and obstetrics
nursing
laboratory diagnosis
carrier detection
psycho-social concerns
economic issues
audit
inherited platelet disorders.