Real-world data in patients with congenital hemophilia and inhibitors: final data from the FEIBA Global Outcome (FEIBA GO) study.

IF 3.4 3区医学 Q2 HEMATOLOGY

Therapeutic Advances in Hematology Pub Date : 2023-01-01 DOI:10.1177/20406207231184323

Carmen Escuriola Ettingshausen, Cedric Hermans, Pål A Holme, Ana R Cid, Kate Khair, Johannes Oldenburg, Claude Négrier, Jaco Botha, Aurelia Lelli, Jerzy Windyga

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引用次数: 1

Abstract

Background: The bypassing agent, activated prothrombin complex concentrate [aPCC, FEIBA (factor VIII inhibitor bypass activity); Baxalta US Inc, a Takeda company, Lexington, MA, USA], is indicated for the treatment of bleeding episodes, perioperative management, and routine prophylaxis in patients with hemophilia A or B with inhibitors. In certain countries, aPCC is also indicated for the treatment of bleeding episodes and perioperative management in patients with acquired hemophilia A.

Objectives: To describe long-term, real-world effectiveness, safety, and quality-of-life outcomes for patients with congenital hemophilia A or B and high-responding inhibitors receiving aPCC treatment in routine clinical practice.

Design: FEIBA Global Outcome (FEIBA GO; EUPAS6691) was a prospective, observational study.

Methods: Investigators determined the treatment regimen and clinical monitoring frequency. The planned patient observation period was 4 years. Data are from the safety analysis set (patients who received ⩾1 aPCC infusion).

Results: Overall, 50 patients received either aPCC prophylaxis (n = 37) or on-demand therapy (n = 13) at screening [hemophilia A, n = 49; hemophilia B, n = 1; median (range) age, 16.5 [2-71] years). Mean ± standard deviation overall annualized bleeding rate and annualized joint bleeding rate for patients receiving prophylaxis were 6.82 ± 11.52 and 3.77 ± 5.71, respectively, and for patients receiving on-demand therapy were 10.94 ± 11.27 and 6.94 ± 7.39, respectively. Overall, 177 and 31 adverse events (AEs) were reported in 28 of 40 and 10 of 13 patients receiving prophylaxis or on-demand therapy, respectively. Two serious AEs were considered possibly related to aPCC: acute myocardial infarction due to coronary artery embolism in one patient receiving prophylaxis. No thrombotic microangiopathy was reported. No AEs resulted in death.

Conclusion: This study demonstrated the long-term, real-world effectiveness and consistent safety profile of aPCC as on-demand therapy and prophylactic treatment in patients with hemophilia and high-responding inhibitors.

Trial registry: FEIBA Global Outcome Study; EUPAS6691 https://www.encepp.eu/encepp/viewResource.htm?id=32774.

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先天性血友病和抑制剂患者的真实世界数据:FEIBA全球结局(FEIBA GO)研究的最终数据

背景:旁路剂，活化凝血酶原复合物浓缩物[aPCC, FEIBA(因子VIII抑制剂旁路活性);Baxalta美国公司(武田公司，Lexington, MA, USA)适用于使用抑制剂治疗血友病a或B患者的出血发作、围手术期管理和常规预防。在某些国家，aPCC也被用于治疗获得性血友病A患者的出血发作和围手术期管理。目的:描述在常规临床实践中接受aPCC治疗的先天性血友病A或B患者和高反应抑制剂的长期、现实世界的有效性、安全性和生活质量。设计:FEIBA Global Outcome (FEIBA GO;EUPAS6691)是一项前瞻性观察性研究。方法:研究人员确定治疗方案和临床监测频率。计划患者观察期为4年。数据来自安全分析集(接受大于或等于1 aPCC输注的患者)。结果:总体而言，50例患者在筛查时接受了aPCC预防(n = 37)或按需治疗(n = 13)[血友病A, n = 49;血友病B, n = 1;年龄中位数(范围)为16.5岁[2-71]。预防组总体年化出血率和关节年化出血率的平均值±标准差分别为6.82±11.52和3.77±5.71，按需治疗组分别为10.94±11.27和6.94±7.39。总体而言，接受预防或按需治疗的40名患者中有28名和13名患者中分别报告了177和31起不良事件(ae)。两种严重的ae被认为可能与aPCC有关:一名接受预防治疗的患者因冠状动脉栓塞而急性心肌梗死。无血栓性微血管病变报告。没有ae导致死亡。结论:本研究证明了aPCC作为血友病患者和高反应抑制剂的按需治疗和预防性治疗的长期、实际有效性和一致的安全性。试验注册:FEIBA全球结局研究;EUPAS6691 https://www.encepp.eu/encepp/viewResource.htm?id=32774。

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来源期刊

Therapeutic Advances in Hematology HEMATOLOGY-

CiteScore

4.30

自引率

0.00%

发文量

审稿时长

7 weeks

期刊介绍： Therapeutic Advances in Hematology delivers the highest quality peer-reviewed articles, reviews, and scholarly comment on pioneering efforts and innovative studies across all areas of hematology. The journal has a strong clinical and pharmacological focus and is aimed at clinicians and researchers in hematology, providing a forum in print and online for publishing the highest quality articles in this area.