Immune tolerance induction for inhibitor eradication in nonsevere hemophilia A: a case series

IF 3.4 3区医学 Q2 HEMATOLOGY

Research and Practice in Thrombosis and Haemostasis Pub Date : 2025-01-01 DOI:10.1016/j.rpth.2024.102637

Sanober Nusrat , Niveditha Popuri , Vishnu Nagalapuram , Osman Khan

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Abstract

Background

Persons with hemophilia A are at risk of inhibitor development with repeated exposures to factor (F)VIII concentrates. When persons with nonsevere hemophilia A (NSHA) develop inhibitors, they are at risk of developing severe bleeding manifestations like persons with severe hemophilia A (SHA). Evidence to guide inhibitor eradication in this population is limited as opposed to persons with SHA who develop inhibitors. Hence, inhibitor eradication strategies in NSHA are based on observational and retrospective data and are largely adopted from evidence derived from SHA with inhibitors.

Key Clinical Question

Can immune tolerance induction be used for patients with NSHA who develop inhibitors?

Clinical Approach

In this case series, we describe our single institutional experience with the management of 5 persons with NSHA who developed FVIII inhibitors, leading to significant bleeding complications, and underwent successful immune tolerance induction with eradication of FVIII inhibitor.

Conclusion

More research specific to persons with NSHA with inhibitors is warranted to develop guidelines regarding indications and strategies for inhibitor eradication therapy.

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免疫耐受诱导抑制剂根除在非严重血友病A：一个病例系列。

背景：血友病A患者在反复暴露于因子(F)VIII浓缩物时有抑制剂发展的危险。当非严重血友病A （NSHA）患者使用抑制剂时，他们有发展为严重出血表现的风险，就像严重血友病A （SHA）患者一样。与发展出抑制剂的SHA患者相比，指导在这一人群中根除抑制剂的证据有限。因此，NSHA的抑制剂根除策略基于观察性和回顾性数据，并且主要采用来自具有抑制剂的SHA的证据。关键的临床问题：免疫耐受诱导是否可以用于NSHA抑制剂患者？临床方法：在这个病例系列中，我们描述了我们的单一机构管理经验，5名NSHA患者出现FVIII抑制剂，导致严重的出血并发症，并通过根除FVIII抑制剂成功诱导免疫耐受。结论：有必要对NSHA抑制剂患者进行更多的研究，以制定有关抑制剂根除治疗的适应症和策略的指南。

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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