Batty P, Honke A, Bowles L, Hart D P, Pasi K J, Uprichard J, Austin S K
St George's University Hospitals NHS Foundation Trust, London, UK.
Barts and The London School of Medicine & Dentistry, The Royal London Hospital, QMUL, London, UK.
Haemophilia. 2015 Jul;21(4):490-5. doi: 10.1111/hae.12682. Epub 2015 Apr 9.
Factor XI (FXI) deficiency is the commonest of the rare bleeding disorders, affecting 2079 individuals in the United Kingdom. Treatment options for bleeding or surgery include antifibrinolytics, fresh frozen plasma or plasma-derived (pd) FXI concentrates. There were a number of reports of thrombosis following treatment with FXI concentrates prior to changes in their manufacturing processes made in the mid-1990's.
The aim of the study was to determine the occurrence of adverse events (haemorrhagic and thrombotic) following usage of pd-FXI concentrates at two large UK haemophilia centres. Retrospective chart review of all consecutively treated patients with BPL Factor XI(®) or Hemoleven(®) over a 5-year period (11/06-11/11) was performed.
Twenty-nine patients (median age = 57.1 years) received treatment over 64 treatment episodes (surgery = 56, bleeding = 5, other = 3), using 126 000 U of concentrate. Median baseline FXI:C was 9 U dL(-1) (range = <1-51), with 21 having severe and eight partial deficiency. BPL Factor XI(®) was used in 39 episodes (79 110 U) and Hemoleven(®) 25 episodes (46 890 U). There were six clinically significant bleeding events, managed either with a single additional dose of FXI concentrate (n = 4) or requiring no further intervention (n = 2). One patient required blood transfusion and one oral iron replacement. Two thrombotic events (transient ischaemic attack and pulmonary emboli), occurred in two patients with severe FXI deficiency, despite cautious FXI concentrate usage in the perioperative period.
FXI concentrate use is efficacious and safe in the majority of cases although physicians should remain mindful of the possibility of thrombotic complications.
因子 XI（FXI）缺乏症是最常见的罕见出血性疾病，在英国影响了2079人。出血或手术的治疗选择包括抗纤溶药物、新鲜冷冻血浆或血浆源性（pd）FXI浓缩物。在20世纪90年代中期其生产工艺改变之前，有许多关于使用FXI浓缩物治疗后发生血栓形成的报告。
本研究的目的是确定在英国两个大型血友病中心使用pd-FXI浓缩物后不良事件（出血性和血栓性）的发生情况。对在5年期间（2006年11月至2011年11月）连续接受BPL因子XI（®）或Hemoleven（®）治疗的所有患者进行回顾性病历审查。
29名患者（中位年龄 = 57.1岁）接受了64次治疗（手术 = 56次，出血 = 5次，其他 = 3次），使用了126000 U浓缩物。基线FXI:C的中位数为9 U dL（-1）（范围 = <1 - 51），其中21例为严重缺乏，8例为部分缺乏。39次治疗（79110 U）使用了BPL因子XI（®），25次治疗（46890 U）使用了Hemoleven（®）。发生了6例具有临床意义的出血事件，其中4例通过额外单次使用FXI浓缩物处理，2例无需进一步干预。1例患者需要输血，1例需要口服铁剂替代治疗。尽管在围手术期谨慎使用FXI浓缩物，但2例严重FXI缺乏的患者发生了2次血栓事件（短暂性脑缺血发作和肺栓塞）。
尽管医生应始终留意血栓并发症的可能性，但在大多数情况下，使用FXI浓缩物是有效且安全的。
