Division of Pediatric Hematology, Oncology and Stem Cell Transplantation, Columbia University Irving Medical Center, Vagelos College of Physicians and Surgeons, Columbia University, New York, New York, USA.
Curr Opin Pediatr. 2024 Dec 1;36(6):653-658. doi: 10.1097/MOP.0000000000001407. Epub 2024 Sep 25.
Allogeneic hematopoietic cell transplantation (HCT) is a curative option for many for inborn errors of immunity (IEI). This review highlights recent progress in the field of HCT for IEI.
Alternative donor transplantation continues to expand donor options for patients with IEI. Reduced intensity and reduced toxicity conditioning approaches are being investigated and optimized. Immunomodulatory bridging therapies are yielding impressive progress in outcomes for primary immune regulatory disorders (PIRD) but require further study in prospective trials. Single-institution, multicenter and consortium studies have improved our understanding of factors that affect overall outcomes in IEI and outcomes in Wiskott-Aldrich syndrome (WAS), chronic granulomatous disease (CGD) and PIRD in particular. Data show that second HCT offers a viable chance of cure to some IEI patients. Late effects in IEI HCT survivors are being better characterized. Preclinical studies of chemo(radiation)-free HCT strategies hold promise for decreasing HCT toxicity.
Improvements in our understanding of HCT donor choice, conditioning regimen, immunomodulatory bridging therapies, diagnostic and post-HCT surveillance testing and late effects continue to yield advancements in the field of HCT for IEI.
异基因造血细胞移植（HCT）是许多遗传性免疫缺陷（IEI）的一种治疗选择。本综述强调了 HCT 在 IEI 领域的最新进展。
替代供体移植继续扩大了 IEI 患者的供体选择。正在研究和优化强度降低和毒性降低的预处理方案。免疫调节桥接治疗在原发性免疫调节障碍（PIRD）的结果方面取得了令人印象深刻的进展，但需要在前瞻性试验中进一步研究。单中心、多中心和联盟研究提高了我们对影响 IEI 总体结果以及 Wiskott-Aldrich 综合征（WAS）、慢性肉芽肿病（CGD）和 PIRD 特定结果的因素的理解。数据表明，第二次 HCT 为一些 IEI 患者提供了可行的治愈机会。IEI HCT 幸存者的晚期效应正在得到更好的描述。无化疗（放）疗 HCT 策略的临床前研究有望降低 HCT 毒性。
对 HCT 供体选择、预处理方案、免疫调节桥接治疗、诊断和 HCT 后监测检测以及晚期效应的理解的提高，继续推动了 IEI 的 HCT 领域的进展。
