Stein J, Ratnoff O D
Department of Pediatrics, Rainbow Babies and Children's Hospital, Cleveland, Ohio.
Am J Pediatr Hematol Oncol. 1993 Aug;15(3):346-50.
Inhibitors of factor VIII occur in < or = 20% of severely affected patients with classic hemophilia, but are unusual in nonhemophilic individuals, and have not been reported in very young children. We treated a child with a "high-responding" inhibitor.
Our patient was an 18-month-old boy who had experienced several episodes of life-threatening hemorrhage. The techniques we used to decrease production of factor VIII in our patient were prolonged small doses of alternate day corticosteroids and continued administration of factor VIII.
We controlled the acute bleeding with porcine factor VIII or with recombinant human factor VIIa (rFVIIa). Immune tolerance was successfully achieved using a combination of corticosteroids and daily factor VIII infusions.
Multimodal therapy aimed at inducing long-term remission, along with stop-gap measures for hemostasis, may be effective for treating children with this acquired coagulopathy.
因子VIII抑制剂见于≤20%的重度典型血友病患者，但在非血友病个体中不常见，且在幼儿中未见报道。我们治疗了一名有“高反应性”抑制剂的儿童。
我们的患者是一名18个月大的男孩，曾经历数次危及生命的出血事件。我们用于减少患者体内因子VIII生成的技术是隔日小剂量长期使用皮质类固醇并持续输注因子VIII。
我们用猪源性因子VIII或重组人因子VIIa（rFVIIa）控制了急性出血。通过联合使用皮质类固醇和每日输注因子VIII成功实现了免疫耐受。
旨在诱导长期缓解的多模式治疗，以及止血的权宜措施，可能对治疗患有这种获得性凝血病的儿童有效。
