先天性血友病伴抑制物患者的真实世界数据：FEIBA全球结局（FEIBA GO）研究的最终数据。
Real-world data in patients with congenital hemophilia and inhibitors: final data from the FEIBA Global Outcome (FEIBA GO) study.
作者信息
Ettingshausen Carmen Escuriola, Hermans Cedric, Holme Pål A, Cid Ana R, Khair Kate, Oldenburg Johannes, Négrier Claude, Botha Jaco, Lelli Aurelia, Windyga Jerzy
机构信息
Hämophilie-Zentrum Rhein Main - HZRM GmbH, Mörfelden-Walldorf, Germany.
Hemostasis and Thrombosis Unit, Division of Haematology, Cliniques Universitaires Saint-Luc, UCLouvain, Brussels, Belgium.
出版信息
Ther Adv Hematol. 2023 Jul 24;14:20406207231184323. doi: 10.1177/20406207231184323. eCollection 2023.
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 ( = 37) or on-demand therapy ( = 13) at screening [hemophilia A, = 49; hemophilia B, = 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.
背景
旁路制剂活化凝血酶原复合物浓缩物[aPCC，FEIBA（因子VIII抑制物旁路活性）；美国马萨诸塞州列克星敦市武田公司旗下的百特国际有限公司]适用于治疗患有抑制剂的甲型或乙型血友病患者的出血发作、围手术期管理和常规预防。在某些国家，aPCC还适用于治疗获得性甲型血友病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治疗的先天性甲型或乙型血友病及高反应性抑制剂患者的长期、实际疗效、安全性和生活质量结果。
设计
FEIBA全球结果（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。计划的患者观察期为4年。数据来自安全性分析集（接受≥1次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）[甲型血友病，n = 49；乙型血友病，n = 1；中位（范围）年龄，16.5 [2 - 71]岁]。接受预防治疗的患者的总体年化出血率和年化关节出血率的均值±标准差分别为6.82±11.52和3.77±5.71，接受按需治疗的患者分别为10.94±11.27和6.94±7.39。总体而言，接受预防治疗或按需治疗的40例患者中的28例和13例患者中的10例分别报告了177例和31例不良事件（AE）。2例严重AE被认为可能与aPCC有关：1例接受预防治疗的患者因冠状动脉栓塞导致急性心肌梗死。未报告血栓性微血管病。没有AE导致死亡。
结论
本研究证明了aPCC作为按需治疗和预防性治疗在血友病及高反应性抑制剂患者中的长期、实际疗效和一致的安全性。
试验注册
FEIBA全球结果研究；EUPAS6691 https://www.encepp.eu/encepp/viewResource.htm?id=32774
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