Intrapulmonary Recombinant Factor VIIa for Diffuse Alveolar Hemorrhage in Children
Intrapulmonary Recombinant Factor VIIa for Diffuse Alveolar Hemorrhage in Children
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DOI：
10.1542/peds.2014-1782
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发表时间：
2015-01-01
期刊：
影响因子：
8
通讯作者：
Kim, Byoung-Ju
中科院分区：
文献类型：
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作者：
Park, Jeong A.;Kim, Byoung-Ju
弥漫性肺泡出血(DAH)是恶性血液病或自身免疫性疾病患者的一种危及生命的肺部并发症，死亡率高。目前皮质类固醇、输血和免疫抑制剂的治疗选择有限，而且大多不成功，而且它们可能伴随着多种并发症。重组激活因子VII(RFVIIa)在成人肺内给药已有报道，但关于其在儿童中的使用数据很少。本文回顾了我们在肺内应用rFVIIa治疗儿童DAH的机构经验。这项研究包括2011至2013年间治疗的6名经支气管镜确诊的急性DAH儿童患者。中位年龄11岁，诊断为急性髓系白血病2例，骨髓增生异常综合征1例，噬血细胞淋巴组织细胞增生症1例，T细胞淋巴母细胞性淋巴瘤1例，特发性肺含铁血黄素沉着症1例。这些患者接受了肺内rFVIIa联合甲基强的松龙、新鲜冰冻血浆和维持血小板计数&50000/mm的治疗。在rFVIIa治疗后，所有患者都观察到了完全和持续的止血，没有不良事件发生。动脉血氧分压(PaO2)/吸氧分数显著升高，临床改善迅速。接受造血干细胞移植的2例患者死于随后的呼吸道合胞病毒和鲍曼不动杆菌感染，但其余4例病情迅速好转，成功脱离呼吸机，并获得长期生存。我们的研究结果表明，肺内给药rFVIIa是治疗DAH儿童的有效和安全的选择；然而，还需要进一步的临床研究。
Diffuse alveolar hemorrhage (DAH) is a life-threatening pulmonary complication in patients with hematologic malignancies or autoimmune disorders, and it has a high mortality rate. The current treatment options of corticosteroids, transfusions, and immunosuppressants have been limited and largely unsuccessful, and they can be accompanied by multiple complications. Intrapulmonary administration of recombinant activated factor VII (rFVIIa) has been reported in adults, but there are scarce data on its use in children. The present article reviews our institutional experience with intrapulmonary rFVIIa for the treatment of DAH in children. The study included 6 pediatric patients with acute, bronchoscopically confirmed DAH treated between 2011 and 2013. The median age was 11 years, and patient diagnoses were as follows: acute myeloid leukemia (2 patients), myelodysplastic syndrome (1 patient), hemophagocytic lymphohistiocytosis (1 patient), T-cell lymphoblastic lymphoma (1 patient), and idiopathic pulmonary hemosiderosis (1 patient). These patients were treated with intrapulmonary rFVIIa concurrent with methylprednisolone, fresh-frozen plasma, and maintenance of the platelet count >50 000/mm(3). Complete and sustained hemostasis after rFVIIa treatment and an absence of adverse events were observed in all patients. The Pao(2)/fraction of inspired oxygen ratio increased significantly, and rapid clinical improvements were observed. Two patients who received hematopoietic stem cell transplantation died of subsequent respiratory syncytial virus and Acinetobacter baumannii infections, but the other 4 patients exhibited rapid improvement, were successfully weaned from ventilators, and experienced long-term survival. Our findings indicate that intrapulmonary administration of rFVIIa is an effective and safe treatment option for children with DAH; however, further clinical studies are needed.