Pediatric Hematology Oncology & BMT Unit, Department of Pediatrics, Sir Ganga Ram Hospital, Delhi, India.
Indian J Pediatr. 2011 Aug;78(8):961-8. doi: 10.1007/s12098-011-0364-6. Epub 2011 Feb 17.
To determine the safety and efficacy of off label usage of Recombinant activated factor VII (rFVIIa) in children with severe bleeding in non-hemophiliac children with diverse etiologies like leukemia, post hematopoietic stem cell transplantation, dengue shock syndrome and Glanzmann thrombasthenia.
Medical records of 16 non-hemophiliac children with 20 bleeding episodes where rFVIIa was administered only after failure of standard measures to control bleeding were retrospectively reviewed and data collected regarding patient demographics, diagnosis and location of bleeding. Blood counts, coagulation and other lab parameters, both pre and post rFVIIa, were also noted. Severity of bleeding was assessed using a scoring system used previously by Nevo S et al. A record of usage of the amount of blood components (red blood cells, platelets, fresh frozen plasma, and cryoprecipitate) 24 h pre and post rFVIIa was also made. The dose of rFVIIa and any adverse side effects were recorded.
Bleeding sites were gastrointestinal (13/20), pulmonary hemorrhage (6/20) and intracranial hemorrhage (1/20). Thrombocytopenia (platelet count <50,000/cumm) was present in 50% episodes and five had refractory low platelets. Usage of packed red cells and platelets was significantly less after usage of rFVIIa (p value 0.001 and 0.006, respectively). Mean dosage of rFVIIa was 77 μgm/kg/dose (range 60 to 90 μgm/kg/dose). The bleeding stopped completely in 11(55%), decreased to minimal in 2 (10%), reduced but transfusions needed in 4 (20%) and did not stop in 3 (15%) episodes. Five patients (31%) did not survive the bleeding episode. None of the patients developed thrombosis.
rFVIIa appears to be safe and effective in controlling life-threatening bleed in non-hemophiliac children.
确定在患有白血病、造血干细胞移植后、登革热休克综合征和 Glanzmann 血小板功能不全等不同病因的非血友病儿童中，超适应证使用重组活化因子 VII（rFVIIa）治疗严重出血的安全性和疗效。
回顾性分析了 16 名非血友病儿童的病历，这些儿童共发生 20 次出血事件，在标准止血措施失败后仅给予 rFVIIa 治疗。收集患者的人口统计学、诊断和出血部位的数据。还记录了 rFVIIa 前后的血液计数、凝血和其他实验室参数。使用 Nevo S 等人之前使用的评分系统评估出血的严重程度。还记录了 rFVIIa 前后 24 小时内使用的血液成分（红细胞、血小板、新鲜冷冻血浆和冷沉淀）的数量。记录 rFVIIa 的剂量和任何不良反应。
出血部位为胃肠道（13/20）、肺出血（6/20）和颅内出血（1/20）。血小板减少症（血小板计数<50,000/cumm）在 50%的病例中存在，5 例血小板计数低且难治。使用 rFVIIa 后，使用红细胞和血小板的数量明显减少（p 值分别为 0.001 和 0.006）。rFVIIa 的平均剂量为 77 μgm/kg/剂量（范围 60 至 90 μgm/kg/剂量）。11 例（55%）出血完全停止，2 例（10%）出血减少至最小，4 例（20%）出血减少但仍需要输血，3 例（15%）出血未停止。5 名患者（31%）在出血事件中死亡。没有患者发生血栓形成。
rFVIIa 似乎可安全有效地控制非血友病儿童的危及生命的出血。
