HAEMFIX: Impact of Switching From SHL-FIX to EHL-FIX in Patients With Haemophilia B.

IF 3 2区医学 Q2 HEMATOLOGY

Haemophilia Pub Date : 2025-10-25 DOI:10.1111/hae.70157

Jasmin Lonardi, Susan Halimeh, Sylvia von Mackensen, Lisa Kleinlein, Juliet Fleischer, Henri Funk, Julia Hölz, Johannes Holzapfel, Sabrina Juranek, Victoria Lieftüchter, Christoph Bidlingmaier, Martin Olivieri

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引用次数: 0

Abstract

Introduction: Haemophilia B is an X-linked recessive bleeding disorder caused by coagulation factor IX (FIX) deficiency. Treatment involves intravenous replacement of FIX. Recently, extended half-life (EHL) FIX products have been introduced alongside standard half-life (SHL) products to optimize therapy.

Aim: This study evaluated bleeding rates, joint health, factor consumption, dosage, and health-related quality of life (HRQoL) in patients switching from SHL- to EHL-FIX products, as well as in those exclusively treated with EHL-FIX.

Methods: Retrospective data from the medical records of 37 children with haemophilia B treated between 2010 and 2023 at two German Haemophilia Care Centres were analysed. HRQoL was assessed cross-sectionally using haemophilia-specific and generic questionnaires.

Results: Twenty-seven patients (median age: 12 years, range 2-19 years) switched from SHL- to EHL-FIX, while 10 received EHL-FIX from the start of prophylaxis. The mean annual bleeding rate (ABR) improved from 6.01 ± 7.01 (SHL) to 2.85 ± 3.42 (EHL). Factor consumption (159,577.8 ± 99,817.9 IU/year), dosage (118.9 ± 50.3 IU/kg/week) and infusion frequency (145 ± 35.6 infusions/year) decreased after switching (100,247.7 ± 46,268.6 IU/year; 56.4 ± 23.7 IU/kg/week; 55.1 ± 9.8 infusions/year). HRQoL improved in both self-reports and parent reports. No severe adverse events occurred.

Conclusion: Switching from SHL-FIX to EHL-FIX in children with haemophilia B is safe and may improve outcomes by reducing bleeding rates, infusion frequency, and factor consumption while enhancing joint health and HRQoL.

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血友病B患者从SHL-FIX切换到EHL-FIX的影响

血友病B是一种由凝血因子IX （FIX）缺乏引起的x连锁隐性出血性疾病。治疗包括静脉注射FIX。最近，延长半衰期（EHL） FIX产品与标准半衰期（SHL）产品一起被引入以优化治疗。目的：本研究评估从SHL-转换为EHL-FIX产品的患者以及仅接受EHL-FIX治疗的患者的出血率、关节健康、因子消耗、剂量和健康相关生活质量（HRQoL）。方法：回顾性分析2010年至2023年在德国两家血友病护理中心治疗的37名B型血友病儿童的医疗记录。采用血友病特异性问卷和一般问卷对HRQoL进行横断面评估。结果：27例患者（中位年龄：12岁，范围2-19岁）从SHL-切换到EHL-FIX， 10例患者从预防开始就接受EHL-FIX。平均年出血率（ABR）由6.01±7.01 （SHL）改善至2.85±3.42 （EHL）。转换后因子消耗（159,577.8±99,817.9 IU/年）、剂量（118.9±50.3 IU/kg/周）和注射频率（145±35.6次/年）降低（100,247.7±46,268.6 IU/年；56.4±23.7 IU/kg/周；55.1±9.8次/年）。HRQoL在自我报告和父母报告中都有所改善。未发生严重不良事件。结论：血友病B患儿从SHL-FIX切换到EHL-FIX是安全的，并且可以通过减少出血率、输注频率和因子消耗来改善预后，同时增强关节健康和HRQoL。

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.