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首页> 外文期刊>Pediatrics: Official Publication of the American Academy of Pediatrics >Intrapulmonary Recombinant Factor VIIa for Diffuse Alveolar Hemorrhage in Children
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Intrapulmonary Recombinant Factor VIIa for Diffuse Alveolar Hemorrhage in Children

机译：小儿弥漫性肺泡出血的肺内重组因子VIIa

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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.
机译：弥漫性肺泡出血（DAH）是血液恶性肿瘤或自身免疫性疾病患者的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，而且基本上没有成功，并且可能伴有多种并发症。成年人中已有肺内施用重组活化因子VII（rFVIIa）的报道，但在儿童中使用它的数据很少。本文回顾了我们在肺内rFVIIa治疗儿童DAH方面的机构经验。该研究纳入了2011年至2013年间接受支气管镜检查证实为DAH的6例儿科患者。中位年龄为11岁，患者诊断如下：急性髓细胞性白血病（2例），骨髓增生异常综合症（1例），吞噬性淋巴细胞组织细胞增生症（ 1例），T细胞淋巴母细胞淋巴瘤（1例）和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，新鲜冷冻血浆的治疗，并维持血小板计数> 50000 / mm（3）。在所有患者中均观察到rFVIIa治疗后完全和持续的止血，并且没有不良事件。 Pao（2）/吸入氧气比率的分数显着增加，并且观察到快速的临床改善。两名接受造血干细胞移植的患者死于随后的呼吸道合胞病毒和鲍曼不动杆菌感染，但其他4例患者表现出快速的好转，已成功地从呼吸机断奶，并经历了长期生存。我们的研究结果表明，rFVIIa的肺内给药是治疗DAH儿童的一种有效且安全的选择。但是，还需要进一步的临床研究。

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