Savaşan Süreyya
Division of Hematology/Oncology, Pediatric Blood and Marrow Transplant Program, Children's Hospital of Michigan, Barbara Ann Karmanos Cancer Center, Wayne State University School of Medicine, 3901 Beaubien Boulevard, Detroit, MI 48201, USA.
Pediatr Clin North Am. 2018 Jun;65(3):597-606. doi: 10.1016/j.pcl.2018.02.006.
Acquired aplastic anemia (aAA) characterized by peripheral pancytopenia and bone marrow aplasia is a rare and serious disorder. Differential diagnosis includes constitutional bone marrow failure syndromes and myelodysplastic disorders. Autoimmune reaction to altered hematopoietic stem cells highlights the underlying mechanism. Matched related donor allogeneic hematopoietic stem cell transplantation is the ideal pediatric treatment; alternative approaches include immunosuppressive therapy and use of eltrombopag. Progression to clonal disorders can occur. Recently, alternative donor hematopoietic stem cell transplantation outcomes have significantly improved. Despite advances, aAA continues to be a challenge for hematologists.
获得性再生障碍性贫血（aAA）以全血细胞减少和骨髓再生障碍为特征，是一种罕见且严重的疾病。鉴别诊断包括先天性骨髓衰竭综合征和骨髓增生异常综合征。对改变的造血干细胞的自身免疫反应是其潜在机制。匹配的相关供体异基因造血干细胞移植是理想的儿科治疗方法；其他方法包括免疫抑制治疗和使用艾曲泊帕。可能会进展为克隆性疾病。最近，替代供体造血干细胞移植的结果有了显著改善。尽管取得了进展，但aAA对血液学家来说仍然是一个挑战。
