摘要
重型再生障碍性贫血（severeaplasticanemia，SAA）是一种以全血细胞减少为特征的骨髓衰竭性疾病。目前通常推荐缺乏HLA全相合亲缘供者的患儿接受免疫抑制治疗，包括抗胸腺细胞／淋巴细胞球蛋白和环孢菌素等。对于免疫抑制治疗无效或复发的患儿，可以选择重复免疫抑制、异基因造血干细胞移植以及新型药物或临床试验等治疗。由于移植技术的提高以及艾曲泊帕等药物的应用，患儿预后得到明显改善。该文就近年来复发或难治性SAA患者治疗方案的研究进展进行综述，旨在指导临床治疗方式的选择。
Severe aplastic anaemia is a marrow failure syndrome characterized by pancytopenia and hypocellular bone marrow. Immunosuppressive therapy（IST） consisting of antithymocyte or antilymphocyte globulin （ATG）and cyclosporine has been recommended for children lacking HLA-matched sibling donor. For children unresponsive to initial immunosuppressive therapy or those who relapsed after first immunosuppressive therapy, they can choose repeat course of immunosuppressive therapy, allogeneic hematopoietic stem cell transplantation, new drugs or clinical trials. And the prognosis has been greatly improved in recent years, dueing to the develop- ment of allogeneic hematopoietic stem cell transplantation and the application of new drugs such as eltrombopag. This article reviews researches advance in different therapies for children with relapsed or refractory severe aplastic anaemia, aimed at guaiding treatment methods.
出处
《国际儿科学杂志》
2017年第5期339-343,共5页
International Journal of Pediatrics
基金
中国医学科学院医学科学创新基金（2016-12M-1-002）
