Department of BMT, Level 4 Westlink, Great Ormond Street Hospital for Children NHS Trust, Great Ormond Street, London, WC1N 3JH, UK.
Immunol Allergy Clin North Am. 2010 Feb;30(1):103-24. doi: 10.1016/j.iac.2009.11.003.
Many advances have been made since the first successful hematopoietic cell transplants (HCT) in children with primary immunodeficiency disorders (PID) were reported 40 years ago, and many children with PID can now be cured from their otherwise lethal disorders through well-matched HCT procedures. Preexisting morbidity and infection remain the principal adverse factors for poor outcomes with HCT. To improve current results, earlier diagnosis, well-tolerated pretransplant conditioning regimens, and promotion of immune reconstitution need to be considered. This article addresses modifications in the conditioning regimen that might lead to further improvement in HCT outcomes.
自 40 年前首例原发性免疫缺陷疾病(PID)患儿成功进行造血细胞移植(HCT)以来,已经取得了许多进展,现在许多 PID 患儿可以通过匹配良好的 HCT 程序治愈其致命疾病。HCT 不良预后的主要不良因素仍然是先前存在的发病率和感染。为了提高目前的结果,需要考虑早期诊断、可耐受的移植前预处理方案和促进免疫重建。本文讨论了预处理方案的改变,这可能会进一步改善 HCT 的结果。