严重血友病 A 患者的免疫耐受诱导：英国血友病临床医生组织抑制剂工作组和儿科工作组共识更新。
Immune tolerance induction in severe haemophilia A: A UKHCDO inhibitor and paediatric working party consensus update.
机构信息
Royal London Hospital Haemophilia Centre, Barts and The London School of Medicine and Dentistry, QMUL, London, UK.
Evelina London Children's Hospital, London, UK.
出版信息
Haemophilia. 2021 Nov;27(6):932-937. doi: 10.1111/hae.14381. Epub 2021 Aug 17.
INTRODUCTION
In good risk patients (historic inhibitor peak < 200BU), the International Immune Tolerance Study demonstrated equal efficacy to induce tolerance between high (200iu/kg/day) and low dose (50iu/kg ×3 times/week) immune tolerance induction (ITI) regimens. However, the trial stopped early on account of the excessive bleed rate in the low dose ITI arm.
METHODS
United Kingdom Haemophilia Centre Doctors' Organization (UKHCDO) Paediatric and Inhibitor working parties considered available ITI data alongside the bi-phenotypic antibody emicizumab (Hemlibra®) efficacy and safety data to develop a consensus guideline for the future UK ITI guideline.
RESULTS
This revision of UKHCDO ITI guidance incorporates the recommendation to use emicizumab as a prophylaxis haemostatic agent to reduce bleeding rates and to facilitate low dose and reduced frequency of FVIII CFC for ITI in the majority of children.
CONCLUSION
This consensus protocol will facilitate future evaluation of ITI outcomes in the evolving landscape of haemophilia therapeutics and ITI strategies.
简介
在低风险患者（历史抑制剂峰值 < 200BU）中，国际免疫耐受研究表明，高剂量（200iu/kg/天）和低剂量（50iu/kg×3 次/周）免疫耐受诱导（ITI）方案在诱导耐受方面具有同等疗效。然而，由于低剂量 ITI 组出血率过高，该试验提前停止。
方法
英国血友病中心医生组织（UKHCDO）儿科和抑制剂工作组考虑了可用的 ITI 数据以及双表型抗体emicizumab（Hemlibra®）的疗效和安全性数据，以制定未来英国 ITI 指南的共识指南。
结果
对 UKHCDO ITI 指南的修订纳入了使用 emicizumab 作为预防出血的止血剂的建议，以降低出血率，并促进大多数儿童使用低剂量和降低 FVIII CFC 的频率进行 ITI。
结论
该共识方案将促进未来在血友病治疗和 ITI 策略不断发展的背景下对 ITI 结果的评估。
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