Division of Pediatric Hematology/Oncology, Washington University in St. Louis, St. Louis, Missouri.
Pediatr Blood Cancer. 2018 Sep;65(9):e27263. doi: 10.1002/pbc.27263. Epub 2018 May 24.
Sickle cell disease (SCD) presents challenges to hematopoietic stem cell transplantation (HSCT), including donor availability and morbidity with age/disease severity. However, severe SCD causes irreversible organ damage that HSCT can mitigate. This benefit must be balanced against preparative regimen toxicity, graft-versus-host disease, and mortality risk. We review efforts to balance HSCT complications with the promise of cure, and knowledge gaps that warrant further investigation. We highlight the burden of SCD, HSCT risks and benefits, and SCD families' approach to this balance. We emphasize the necessity for information exchange to ensure a joint decision-making process between providers and patients.
镰状细胞病（SCD）给造血干细胞移植（HSCT）带来了挑战，包括供体的可用性和随年龄/疾病严重程度而增加的发病率。然而，严重的 SCD 会导致不可逆转的器官损伤，而 HSCT 可以减轻这种损伤。这种益处必须与预处理方案的毒性、移植物抗宿主病和死亡率风险相平衡。我们回顾了平衡 HSCT 并发症与治愈希望的努力，以及需要进一步研究的知识空白。我们强调 SCD 的负担、HSCT 的风险和益处，以及 SCD 家庭在这方面的平衡方法。我们强调需要信息交流，以确保提供者和患者之间的共同决策过程。
