The International Prospective Glanzmann Thrombasthenia Registry: Pediatric Treatment and Outcomes

R. Zotz, M. Poon, G. Di Minno, R. d’Oiron
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引用次数: 9

Abstract

Abstract Background Standard treatment for Glanzmann thrombasthenia (GT), a severe inherited bleeding disorder, is platelet transfusion. Recombinant activated factor VII (rFVIIa) is reported to be effective in GT with platelet antibodies and/or refractoriness to platelet transfusions. Methods We evaluated rFVIIa effectiveness and safety for the treatment and prevention of surgical and nonsurgical bleeding in children <18 years old, with or without platelet antibodies and/or refractoriness, as reported in the GT Registry (GTR). Data were used from the GTR, an international, multicenter, observational, postmarketing study of rFVIIa that prospectively collected data on the treatment and outcomes of bleeds in patients with GT. Only patients with a diagnosis of congenital GT were included in the registry. Results Between 2007 and 2011, 27 children were treated for 44 surgical procedures (minor: 36; major: 8); nonsurgical bleeds occurred in 104 patients (599 episodes: severe, 145; moderate, 454; spontaneous, 423; posttraumatic, 176). The effectiveness of treatment for minor procedures, major procedures, nonsurgical bleeds was 6/6, 1/1, and 75/84 for rFVIIa, 6/6, 2/2, and 64/76 for rFVIIa + antifibrinolytics (AF), 11/12, 1/1, and 162/214 for platelets ± AF, and 5/6, 0/3, and 33/45 for rFVIIa + platelets ± AF. In all, 25 adverse events were reported in children; no thromboembolic events were reported. Conclusion For all patients, regardless of platelet antibody or refractoriness status, rFVIIa, administered with or without platelets (± AF), provided effective hemostasis with a low frequency of adverse events in surgical, as well as nonsurgical, bleeding in patients with GT. clinicaltrials.gov identifier: NCT01476423.
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国际前瞻性Glanzmann血栓缺失登记:儿童治疗和结果
背景:格兰兹曼血栓减少症(GT)是一种严重的遗传性出血性疾病,其标准治疗是输注血小板。据报道,重组活化因子VII (rFVIIa)对血小板抗体和/或血小板输注难治性GT有效。方法:根据GT Registry (GTR)的报告,我们评估了rFVIIa治疗和预防18岁以下儿童手术和非手术出血的有效性和安全性,无论是否有血小板抗体和/或难治性。数据来自GTR,这是一项国际、多中心、观察性的rFVIIa上市后研究,该研究前瞻性地收集了GT患者出血的治疗和结果数据。只有诊断为先天性GT的患者被纳入注册表。结果2007 - 2011年共收治27例患儿44例外科手术(未成年36例;主要:8);104例患者发生非手术出血(599次,重度145次;温和,454;自发的,423;创伤后,176)。小手术、大手术、非手术出血的治疗效果rFVIIa为6/6、1/1和75/84,rFVIIa +抗纤溶药物(AF)的治疗效果为6/6、1/ 2和64/76,血小板±AF的治疗效果为11/12、1/1和262 /214,rFVIIa +血小板±AF的治疗效果为5/6、0/3和33/45。总共报告了25例儿童不良事件;无血栓栓塞事件报道。结论:对于所有患者,无论血小板抗体或难治性,在有或没有血小板(±AF)的情况下,rFVIIa均能有效止血,手术和非手术出血患者的不良事件发生率均较低。
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