Abstract：
Acquired aplastic anemia (AA) is a rare heterogeneous disorder characterized by pancytopenia and hypoplastic bone marrow. The incidence is 2-3 per million population per year in the Western world, but 3 times higher in East Asia. Survival in severe aplastic anemia (SAA) has improved significantly due to advances in hematopoietic stem cell transplantation (HSCT), immunosuppressive therapy, biologic agents, and supportive care. In SAA, HSCT from a matched sibling donor (MSD) is the first-line treatment. If a MSD is not available, options include immunosuppressive therapy (IST), matched unrelated donor, or haploidentical HSCT. The purpose of this guideline is to provide health care professionals with clear guidance on the diagnosis and management of pediatric patients with AA. A preliminary evidence-based document prepared by a group of pediatric hematologists of the Bone Marrow Failure Study Group of the Italian Association of Pediatric Hemato-Oncology (AIEOP) was discussed, modified and approved during a series of consensus conferences that started online during COVID 19 and continued in the following years, according to procedures previously validated by the AIEOP Board of Directors.
摘要：
获得性再生障碍性贫血（AA）是一种罕见的异质性疾病，其特征是全血细胞减少和增生性骨髓。在西方世界，发病率为每年每百万人口2-3，但在东亚高3倍。由于造血干细胞移植（HSCT）的进步，重度再生障碍性贫血（SAA）的生存率显着提高。免疫抑制治疗,生物制剂，和支持性护理。在SAA,来自匹配的同胞供体（MSD）的HSCT是一线治疗。如果MSD不可用，选择包括免疫抑制治疗（IST），匹配的无关捐赠者，或单倍体HSCT。本指南的目的是为医疗保健专业人员提供有关AA儿科患者诊断和管理的明确指导。讨论了由意大利小儿血液肿瘤协会（AIEOP）骨髓衰竭研究组的一组小儿血液学家编写的基于证据的初步文件，在COVID19期间开始的一系列共识会议上进行了修改和批准，并在接下来的几年中继续进行，根据AIEOP董事会先前验证的程序。
