Morris Emma C, Albert Michael H
Institute of Immunity and Transplantation, University College London, London, United Kingdom.
University College London Hospital and Royal Free London Hospitals, London, United Kingdom.
Front Pediatr. 2019 Oct 24;7:437. doi: 10.3389/fped.2019.00437. eCollection 2019.
Significant advances in hematopoietic transplantation over the past 20 years, have facilitated the safe transplantation of older adults with higher co-morbidities. In pediatric practice these advances have simultaneously improved outcomes for sicker children with complex, rare diseases including the primary immunodeficiencies, PID. With more widespread adoption of genetic sequencing, older patients with disease-causing mutations restricted to the hematopoietic system can be identified who may benefit from allogeneic hematopoietic stem cell transplantation (Allo-HSCT). Here we discuss the evidence for Allo-HSCT in adolescent and younger adults (AYAs) with PID.
在过去20年里，造血移植取得了重大进展，这使得合并症较多的老年人能够安全地接受移植。在儿科实践中，这些进展同时改善了患有复杂罕见疾病（包括原发性免疫缺陷病，PID）的病情较重儿童的治疗效果。随着基因测序的更广泛应用，可以识别出那些造血系统存在致病突变的老年患者，他们可能从异基因造血干细胞移植（Allo-HSCT）中获益。在此，我们讨论PID青少年及年轻成人（AYAs）接受Allo-HSCT的证据。
