Clinical and Economic Burden of Patients With Haemophilia A and B in France: Analysis of a Nationwide Claims Database

IF 3 2区医学 Q2 HEMATOLOGY

Haemophilia Pub Date : 2025-03-28 DOI:10.1111/hae.70041

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

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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.

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法国血友病A和B患者的临床和经济负担：全国索赔数据库的分析。

关于法国血友病A （HA）和B （HB）患者的医疗资源使用和相关费用的数据很少。目的：本研究旨在描述HA和HB患者的概况，目前的疾病管理，临床负担和成本。方法：从法国全国索赔数据库（SNDS）中提取2022年1月1日存活的所有年龄段血友病患者的相关数据。患者分为四个治疗组：按需治疗组或有或无抑制剂的预防治疗组。采用风险比（RR[95%置信区间]）将血友病患者与年龄、性别和地区相匹配的对照组（比例为1:3）进行比较。估计了人均年度直接医疗保健费用。结果：共纳入5577例HA患者和1332例HB患者（平均年龄：36.4岁）。大多数患者按需治疗(HA: 72.8%；HB: 76.6%)，少数有抑制剂(HA: 3.6%；HB: 1.1%)。总体而言，血友病患者的临床负担显著高于对照组，特别是死亡率（RR:1.42[1.04-1.92]）、工作残疾（RR: 2.71[2.22-3.30]）、因大出血住院（RR:12.06[8.67-16.80]）、骨科手术（RR: 2.97[2.65-3.32]）和全因住院（RR: 2.44[2.31-2.58]）。这种负担在使用抑制剂或接受预防治疗的患者中更为重要，并且在HA和HB患者中接近。在无抑制剂预防中，HA和HB的年人均成本分别为282,560欧元和181,566欧元。使用抑制剂的人群虽然数量有限，但费用更高。结论：血友病治疗的临床负担和费用可能非常高，特别是预防和/或抑制剂治疗的患者。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Haemophilia 医学-血液学

CiteScore

6.50

自引率

28.20%

发文量

226

审稿时长

3-6 weeks

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.