Objective

The Hemraude study was conducted to describe the profile of patients with HA, disease management, and economic burden in a collective perspective.

Methods

This retrospective study was conducted using the French administrative healthcare claims database SNIIRAM/SNDS. Male patients treated for hemophilia A with a long-term illness (ALD) status or invalidity were included in the study between January 1, 2016 and December 31, 2017. Patients were classified in six treatment groups: no treatment, on-demand FVIII, prophylactic FVIII, FVIII in immune tolerance induction (ITI) protocol, on-demand bypassing agents, and prophylactic bypassing agents. Patients treated with FVIII in ITI protocol and those treated with bypassing agents are deemed to have developed inhibitors. HA patients were compared to a control population without coagulation disorder and matched (ratio 1:3) on age and sex.

Results

A total of 4172 patients were included in the analysis, aged on average 35.2 years, 5.3% had HIV infection, and 8.8% had hepatitis B or C. In 2017, half of the patients received no treatment for HA, 46.7% were treated with FVIII (25% on demand, 20.6% with prophylaxis, and 1.1% ITI), 1.5% with bypassing agents. Patients treated with prophylactic treatments, either inhibitor or non-inhibitor, were less likely to be hospitalized for severe bleeding compared to patients receiving on-demand treatments. The average annual costs for HA management per patient were 72,209.60 €. The highest costs were observed in patients treated with FVIII in ITI protocol and those receiving prophylactic bypassing agents.

Conclusion

Direct costs of HA treatments for HA may be very high especially in the small percentage of patients developing inhibitors or treated with ITI protocol.

中文翻译：

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法国血友病 A 患者的费用和管理：Hemraude 研究

客观的

Hemraude 研究旨在从集体角度描述 HA 患者的概况、疾病管理和经济负担。

方法

这项回顾性研究是使用法国行政医疗保健索赔数据库 SNIIRAM/SNDS 进行的。2016 年 1 月 1 日至 2017 年 12 月 31 日期间，接受治疗的患有长期疾病 (ALD) 状态或无效的 A 型血友病男性患者被纳入研究。患者分为六个治疗组：不治疗、按需 FVIII、预防性 FVIII、免疫耐受诱导 (ITI) 方案中的 FVIII、按需旁路药物和预防性旁路药物。在 ITI 方案中使用 FVIII 治疗的患者和使用旁路药物治疗的患者被认为已产生抑制剂。将 HA 患者与没有凝血障碍的对照人群进行比较，并在年龄和性别上匹配（比例 1:3）。

结果

分析共纳入 4172 名患者，平均年龄 35.2 岁，5.3% 感染 HIV，8.8% 患有乙型或丙型肝炎。2017 年，一半的患者未接受 HA 治疗，46.7% 接受了治疗FVIII（按需使用 25%，预防使用 20.6%，ITI 使用 1.1%），旁路药物使用 1.5%。与接受按需治疗的患者相比，接受预防性治疗（抑制剂或非抑制剂）的患者因严重出血而住院的可能性较小。每名患者的 HA 管理年平均费用为 72,209.60 欧元。在 ITI 方案中接受 FVIII 治疗的患者和接受预防性旁路药物的患者中观察到的成本最高。

结论

HA 治疗的直接成本可能非常高，特别是在开发抑制剂或接受 ITI 方案治疗的一小部分患者中。