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儿童血友病中依库珠单抗的应用:一项单中心回顾性研究中的出血和手术结局。

Emicizumab in pediatric hemophilia: Bleeding and surgical outcomes from a single-center retrospective study.

机构信息

Department of Pediatrics, Section of Hematology-Oncology, Texas Children's Cancer and Hematology Centers, Baylor College of Medicine, Houston, Texas, USA.

出版信息

Pediatr Blood Cancer. 2021 Nov;68(11):e29325. doi: 10.1002/pbc.29325. Epub 2021 Sep 7.

Abstract

Real-world data describing emicizumab prophylaxis in pediatric hemophilia A is limited with current literature providing a heterogenous approach to procedural management. We performed a retrospective review on the 28 patients who have been treated with emicizumab prophylaxis at Texas Children's Hospital from 2018 to 2021. After starting emicizumab, the mean annualized bleeding rate reduced from 3.61 to 0.44. Seven surgical procedures were performed in the cohort, and all treated with pre- and postoperative factor replacement resulting in one minor bleeding event. We demonstrate a successful experience with emicizumab prophylaxis and safe perioperative approach with a focus on minimizing postoperative bleeding.

摘要

目前的文献对儿童甲型血友病依库珠单抗预防治疗的描述主要基于真实世界数据,且其对手术管理的方法各异。我们对 2018 年至 2021 年期间在德克萨斯儿童医院接受依库珠单抗预防治疗的 28 例患者进行了回顾性研究。开始使用依库珠单抗后,年出血率从 3.61 降至 0.44。该队列中有 7 例患者接受了手术治疗,所有患者均在术前和术后进行了因子替代治疗,仅发生 1 例轻微出血事件。我们展示了依库珠单抗预防治疗的成功经验和安全的围手术期处理方法,重点是尽量减少术后出血。

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