Groomes Charles L, Gianferante David M, Crouch Gary D, Parekh Dina S, Scott David W, Lieuw Kenneth
Department of Pediatrics, Uniformed Services University of the Health Sciences, Bethesda, Maryland.
Walter Reed National Military Medical Center, Bethesda, Maryland.
Pediatr Blood Cancer. 2016 May;63(5):922-4. doi: 10.1002/pbc.25874. Epub 2016 Jan 6.
The development of inhibitors toward factor VIII (FVIII) is a common and serious complication of hemophilia A (HA) therapy. Patients with hemophilia who develop inhibitors often undergo time- and resource-intensive immune tolerance induction (ITI) protocols. We report a 15-month-old male with severe HA and a high-titer inhibitor that occurred while receiving prophylactic treatment with recombinant FVIII (rFVIII), in whom significant inhibitor titer reduction was achieved with thrice weekly infusions of a new, prolonged half-life rFVIII-Fc fusion protein product (trade name Eloctate). Further studies are warranted to explore the potential of Eloctate in ITI protocols.
针对凝血因子VIII（FVIII）的抑制剂的产生是A型血友病（HA）治疗中常见且严重的并发症。发生抑制剂的血友病患者通常要接受耗时且资源密集的免疫耐受诱导（ITI）方案。我们报告了一名15个月大的重度HA男性患者，其在接受重组FVIII（rFVIII）预防性治疗时出现了高滴度抑制剂，该患者通过每周三次输注一种新的、半衰期延长的rFVIII-Fc融合蛋白产品（商品名Eloctate），实现了抑制剂滴度的显著降低。有必要开展进一步研究以探索Eloctate在ITI方案中的潜力。
