Diagnosis and management of acquired aplastic anemia in childhood. Guidelines from the Marrow Failure Study Group of the Pediatric Haemato-Oncology Italian Association (AIEOP)
Diagnosis and management of acquired aplastic anemia in childhood. Guidelines from the Marrow Failure Study Group of the Pediatric Haemato-Oncology Italian Association (AIEOP)
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DOI：
10.1016/j.bcmd.2015.03.007
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发表时间：
2015-06-01
影响因子：
2.3
通讯作者：
Saracco, Paola
中科院分区：
文献类型：
--
作者：
Barone, Angelica;Lucarelli, Annunziata;Saracco, Paola
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骨髓再生不良。欧洲的发病率为2-3/百万居民/年，但东亚较高。由于造血干细胞移植、免疫抑制和生物药物以及支持治疗的进步，在过去的20年中，重型再生障碍性贫血（SAA）的生存率得到了显著改善。在SAA中，来自匹配的同胞供体（MSD）的造血干细胞移植（HSCT）是治疗的选择。如果MSD不可用，则选择包括免疫抑制治疗（IST）或无关供体HSCT。本指南的目的是为医疗保健专业人员提供关于AA儿科患者诊断和管理的明确指导。根据AIEOP委员会先前验证的程序，在一系列“共识会议”期间讨论、修改和批准了一组儿科血液学家发布的初步循证文件。该指南强调了将患有AA的儿科患者转诊到在AA的诊断、鉴别诊断、管理、支持性护理和随访方面具有长期经验的儿科中心的重要性。(C)2015 Elsevier Inc. All rights reserved.
Acquired aplastic anemia (AA) is a rare heterogeneous disease characterized by pancytopenia and hypoplastic bone marrow. The incidence is 2-3/million inhabitants/year, in Europe, but higher in East Asia. Survival in severe aplastic anemia (SAA) has markedly improved in the past 2 decades because of advances in hematopoietic stem cell transplantation, immunosuppressive and biologic drugs, and supportive care. In SAA hematopoietic stem cell transplant (HSCT) from a matched sibling donor (MSD) is the treatment of choice. If a MSD is not available, the options include immunosuppressive therapy (IST) or unrelated donor HSCT. The objective of this guideline is to provide healthcare professionals with clear guidance on the diagnosis and management of pediatric patients with AA. A preliminary, evidence-based document issued by a group of pediatric hematologists was discussed, modified and approved during a series of "Consensus Conferences" according to procedures previously validated by the AIEOP Board. The guidelines highlight the importance of referring pediatric patients with AA to pediatric centers with long experience in diagnosis, differential diagnosis, management, supportive care and follow-up of AA. (C) 2015 Elsevier Inc. All rights reserved.