Qian Xiaowen, Wang Ping, Wang Hongsheng, Jiang Wenjin, Sun Jinqiao, Wang Xiaochuan, Zhai Xiaowen
Department of Hematology/Oncology, Children's Hospital of Fudan University, Shanghai 201102, China.
Department of Clinical Immunology, Children's Hospital of Fudan University, Shanghai 201102, China.
Transl Pediatr. 2020 Feb;9(1):34-42. doi: 10.21037/tp.2020.01.06.
This study aims to investigate the efficacy and safety of umbilical cord blood transplantation (UCBT) without serotherapy for treating children with leukocyte adhesion deficiency type I (LAD-I).
Clinical characteristics and data of five children with LAD-I who underwent UCBT at our hospital between September 2016 and September 2018 were retrospectively analyzed.
Five (two boys and three girls) patients with LAD-I were included. The median age at UCBT was 9 months (range, 8 to 32 months). The same myeloablative conditioning regimen was administered for each patient and included busulfan, fludarabine, and cyclophosphamide. HLA matching of patients and umbilical cord blood was 8/10 to 10/10. The median dose of total nucleated cells (TNC) infused was 10.2×10/kg (range, 4.5×10 to 20.6×10/kg) and the median dose of CD34+ cells was 3.2×10/kg (range, 1.9×10 to 5.7×10/kg). The median time of neutrophil engraftment was 20 days (range, 13 to 28 days). The median time of platelet engraftment was 36 days (range, 32 to 56 days). All patients received complete donor chimerism (CDC). Four of the five patients developed grade II-IV acute graft-versus-host disease (GvHD). The median follow-up time after transplantation was 19 months (range, 8 to 38 months). Four of the patients survived and achieved complete clinical remission. The other patient died of bronchiolitis obliterans 8 months after UCBT.
UCBT is an effective treatment method for LAD-I patients. Also, severe LAD-I patients should undergo stem cell transplantation as early as possible.
本研究旨在探讨无血清疗法的脐带血移植（UCBT）治疗Ⅰ型白细胞黏附缺陷症（LAD-Ⅰ）患儿的疗效和安全性。
回顾性分析2016年9月至2018年9月在我院接受UCBT的5例LAD-Ⅰ患儿的临床特征和数据。
纳入5例（2例男孩和3例女孩）LAD-Ⅰ患者。UCBT时的中位年龄为9个月（范围8至32个月）。每位患者均采用相同的清髓预处理方案，包括白消安、氟达拉滨和环磷酰胺。患者与脐带血的HLA配型为8/10至10/10。输注的总核细胞（TNC）中位剂量为10.2×10⁸/kg（范围4.5×10⁸至20.6×10⁸/kg），CD34⁺细胞中位剂量为3.2×10⁶/kg（范围1.9×10⁶至5.7×10⁶/kg）。中性粒细胞植入的中位时间为20天（范围13至28天）。血小板植入的中位时间为36天（范围32至56天）。所有患者均获得完全供体嵌合（CDC）。5例患者中有4例发生Ⅱ-Ⅳ级急性移植物抗宿主病（GvHD）。移植后的中位随访时间为19个月（范围8至38个月）。4例患者存活并实现完全临床缓解。另1例患者在UCBT后8个月死于闭塞性细支气管炎。
UCBT是治疗LAD-Ⅰ患者的有效方法。此外，重度LAD-Ⅰ患者应尽早进行干细胞移植。
