Department of Pediatrics, Graduate School of Medicine, Kyoto University, 54 Kawahara-cho, Shogoin, Sakyo-ku, Kyoto, 606-8507, Japan.
Int J Hematol. 2022 Jul;116(1):41-47. doi: 10.1007/s12185-022-03361-5. Epub 2022 May 14.
Allogeneic hematopoietic cell transplantation (HCT) can be curative for a variety of non-malignant diseases (NMDs) as well as hematological malignancies. However, there are several fundamental differences between HCT for NMDs and hematological malignancies, which may necessitate the use of alternative HCT strategies. For example, these diseases differ in the intensity of conditioning regimen sufficient to improve disease. In addition, patients with NMDs are at higher risk of graft failure or mixed chimerism following HCT, and gain no or little survival benefit from graft-versus-host disease. Because more than 80% of patients with NMDs become long-term survivors, greater attention has been paid to late adverse effects and decreased of quality of life after HCT. This review addresses several unresolved issues in allogeneic HCT for patients with NMDs, such as (1) stem cell source, (2) conditioning regimen, (3) use of serotherapy or low-dose irradiation, and (4) therapeutic intervention for mixed chimerism. Resolving these issues may improve transplant outcomes in patients with NMDs.
异基因造血细胞移植（HCT）可治愈多种非恶性疾病（NMD）和血液系统恶性肿瘤。然而，NMD 和血液系统恶性肿瘤的 HCT 之间存在一些根本差异，可能需要采用替代的 HCT 策略。例如，这些疾病在改善疾病所需的预处理方案强度方面存在差异。此外，NMD 患者在 HCT 后发生移植物失败或混合嵌合体的风险较高，并且不会从移植物抗宿主病中获得生存获益或获益甚微。由于超过 80%的 NMD 患者成为长期幸存者，因此人们更加关注 HCT 后晚期不良事件和生活质量下降的问题。本文针对 NMD 患者异基因 HCT 中的几个未解决的问题进行了讨论，例如（1）干细胞来源，（2）预处理方案，（3）使用血清疗法或低剂量照射，以及（4）混合嵌合体的治疗干预。解决这些问题可能会改善 NMD 患者的移植结果。
