City of Hope National Medical Center, Duarte, CA, USA.
HZRM - Hemophilia Centre Rhein Main, Moerfelden-Waldorf, Germany.
Haemophilia. 2015 May;21(3):358-364. doi: 10.1111/hae.12602. Epub 2015 Jan 21.
The benefits shown with factor VIII (FVIII) prophylaxis relating to joint health and quality of life (QoL) provide the rationale for FEIBA prophylaxis in haemophilia A patients with persistent FVIII inhibitors. FEIBA has previously shown efficacy in preventing bleeds in inhibitor patients who failed to respond to, or were ineligible for immune tolerance induction (ITI). The study examined the outcome of paediatric patients undergoing long-term FEIBA prophylaxis. A retrospective chart review included severe haemophilia A patients with persistent inhibitors aged ≤13 years at the start of FEIBA prophylaxis. Baseline characteristics captured dose, frequency of prophylaxis, history of inhibitor development, including baseline titre, historical peak titre and history of ITI. Outcome measurements included annual bleed rate before and during FEIBA prophylaxis, joint status and school days missed. Sixteen cases of FEIBA prophylaxis from two centres are presented. The mean age of subjects at prophylaxis initiation was 7.5 ± 3.6 years and median baseline inhibitor titre was 23 (range 3.1-170) BU. Prior to prophylaxis initiation, median annual joint bleeds among all patients was 4 (0-48), which dropped significantly after the first year of prophylaxis, to a median annual joint bleed rate of 1 (0-7; P = 0.0179). Subsequent years (median = 9) of prophylaxis therapy demonstrated similarly low annual joint bleed rates. There were no life-threatening bleeds, no viral seroconversions or thrombotic events during FEIBA prophylaxis treatment. FEIBA prophylaxis was effective for preventing joint bleeds and subsequent joint damage, delaying arthropathy and improving outcomes in children with haemophilia A and inhibitors to FVIII, who failed or were ineligible for ITI.
VIII 因子（FVIII）预防治疗在关节健康和生活质量（QoL）方面显示出的益处为存在持续性 FVIII 抑制剂的血友病 A 患者使用 FEIBA 预防治疗提供了依据。FEIBA 此前已显示出在对免疫耐受诱导（ITI）无反应或不适合 ITI 的抑制剂患者中预防出血的疗效。该研究检查了接受长期 FEIBA 预防治疗的儿科患者的结果。回顾性图表审查包括在开始 FEIBA 预防治疗时年龄≤13 岁的持续性抑制剂的严重血友病 A 患者。基线特征包括剂量、预防治疗的频率、抑制剂发展史，包括基线滴度、历史最高滴度和 ITI 史。结果测量包括 FEIBA 预防治疗前和治疗期间的年出血率、关节状况和缺课天数。来自两个中心的 16 例 FEIBA 预防治疗病例。预防治疗开始时受试者的平均年龄为 7.5±3.6 岁，中位基线抑制剂滴度为 23（范围 3.1-170）BU。在开始预防治疗之前，所有患者的中位数年关节出血数为 4（0-48），在预防治疗的第一年显著下降，中位数年关节出血率为 1（0-7；P=0.0179）。随后几年（中位数=9）的预防治疗也显示出类似的低年关节出血率。在 FEIBA 预防治疗期间，没有发生危及生命的出血、病毒血清转换或血栓事件。FEIBA 预防治疗对预防关节出血和随后的关节损伤、延迟关节炎以及改善对 ITI 无反应或不适合 ITI 的血友病 A 和 FVIII 抑制剂患者的结局是有效的。
