Nusrat Sanober, Popuri Niveditha, Nagalapuram Vishnu, Khan Osman
Hematology-Oncology Section, Department of Internal Medicine, University of Oklahoma Health Sciences Center, Oklahoma City, OK 73104, USA.
Department of Internal Medicine, University of Oklahoma Health Sciences Center, Oklahoma City, OK 73104, USA.
Res Pract Thromb Haemost. 2024 Nov 28;9(1):102637. doi: 10.1016/j.rpth.2024.102637. eCollection 2025 Jan.
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.
Can immune tolerance induction be used for patients with NSHA who develop inhibitors?
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.
More research specific to persons with NSHA with inhibitors is warranted to develop guidelines regarding indications and strategies for inhibitor eradication therapy.
甲型血友病患者反复接触凝血因子（F）VIII浓缩物有产生抑制物的风险。非重度甲型血友病（NSHA）患者产生抑制物时，有出现与重度甲型血友病（SHA）患者类似的严重出血表现的风险。与产生抑制物的SHA患者相比，指导该人群抑制物清除的证据有限。因此，NSHA患者的抑制物清除策略基于观察性和回顾性数据，并且很大程度上借鉴了有抑制物的SHA患者的证据。
免疫耐受诱导能否用于产生抑制物的NSHA患者？
在本病例系列中，我们描述了我们单机构对5例产生FVIII抑制物的NSHA患者的管理经验，这些患者出现了严重出血并发症，并通过成功的免疫耐受诱导清除了FVIII抑制物。
有必要针对有抑制物的NSHA患者开展更多专门研究，以制定抑制物清除治疗的适应证和策略指南。
