Department of Pediatrics, Division of Pediatric Hematology and Oncology and Bone Marrow Transplantation, King Hussein Cancer Center, Amman, Jordan.
Department of Pharmacy, King Hussein Cancer Center, Amman, Jordan.
J Clin Immunol. 2021 Nov;41(8):1754-1761. doi: 10.1007/s10875-021-01113-4. Epub 2021 Aug 6.
Haploidentical hematopoietic cell transplantation (HCT) is a valuable curative option for children with non-malignant diseases. Haploidentical HCT using post-transplant cyclophosphamide (PTCy) is a readily available option in the absence of an HLA-matched donor. We conducted a retrospective single-center study on the outcome of haploidentical HCT in children with non-malignant diseases. We gathered data from 44 patients underwent HCT in the period 2015 to 2020. The indications for HCT were bone marrow failure, primary immunodeficiency, metabolic disorders, and hemoglobinopathy. Median age at HCT was 4 years (range 0.7-20). The conditioning regimens were myeloablative (n = 17) or reduced intensity (n = 27). After a median follow-up of 20 months (range 4-71), 2-year overall survival was 89% and 2-year GvHD-free relapse-free survival (GRFS) was 66%. Incidence of primary graft failure was 13.6%. Cumulative incidence of grade II-IV acute and moderate/severe chronic GvHD were 20% and 6.4%, respectively. Younger age at HCT (< 4 years) and primary immunodeficiency were significantly associated with better GRFS (p < 0.05). In conclusion, haploidentical HCT using PTCy is feasible and curative in children with non-malignant diseases lacking an HLA-matched donor. Early diagnosis and referral in addition to timely treatment can further improve outcomes.
单倍体造血细胞移植（HCT）是一种有价值的治疗选择，可用于治疗非恶性疾病的儿童。在缺乏 HLA 匹配供体的情况下，使用移植后环磷酰胺（PTCy）的单倍体 HCT 是一种现成的选择。我们对在缺乏 HLA 匹配供体的情况下，患有非恶性疾病的儿童进行单倍体 HCT 的结果进行了回顾性单中心研究。我们从 2015 年至 2020 年期间接受 HCT 的 44 名患者中收集数据。HCT 的适应证为骨髓衰竭、原发性免疫缺陷、代谢紊乱和血红蛋白病。HCT 时的中位年龄为 4 岁（范围 0.7-20）。预处理方案为清髓性（n=17）或强度降低（n=27）。中位随访 20 个月（范围 4-71）后，2 年总生存率为 89%，2 年无 GvHD 无复发生存率（GRFS）为 66%。原发性移植物失败的发生率为 13.6%。Ⅱ-Ⅳ级急性和中/重度慢性 GvHD 的累积发生率分别为 20%和 6.4%。HCT 时年龄较小（<4 岁）和原发性免疫缺陷与更好的 GRFS 显著相关（p<0.05）。总之，在缺乏 HLA 匹配供体的情况下，使用 PTCy 的单倍体 HCT 对患有非恶性疾病的儿童是可行且有效的。早期诊断和转介以及及时治疗可以进一步改善结局。
