[Clinical Observation on Haploid Hematopoietic Stem Cells Combined with Umbilical Cord Blood Double Transplantation for Aplastic Anaemia in Children].

Q4 Medicine 中国实验血液学杂志 Pub Date : 2024-06-01 DOI:10.19746/j.cnki.issn.1009-2137.2024.03.037

Huan-Jun Liu, Xing-Hua Chen, Wen-Bang Zhang, Shu-Xia Guo

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Abstract

Objective: To explore the efficacy and safety of haploidentical hematopoietic stem cell transplantation combined with umbilical cord blood infusion for the treatment of aplastic anaemia in children.

Methods: Nine cases of children with aplastic anaemia treated with umbilical cord blood combined with haploidentical hematopoietic stem cell transplantation at the People's Hospital of Henan University of Chinese Medicine from January 1, 2021 to September 15, 2023 with a median age of 11(2-13) years and a median follow up of 18(7.5-21) months were included, and the clinical data were retrospectively analyzed. Hematopoiesis reconstitution, the incidence of graft-versus-host disease(GVHD), infections and survival of the patients were analyzed.

Results: All 9 children were successfully implanted. The median time to neutrophil and platelet implantation was 11.11±1.27 d and 12.44±3.36 d, respectively. One case developed acute gastrointestinal GVHD of degree I, which was improved after treatment, and the patient developed superficial gastritis and chronic gastrointestinal GVHD at a later stage, which is currently under clinical follow-up. Acute GVHD of II-IV degree was 0%. Hemorrhagic cystitis in 3 cases, CMV infection in 5 cases and bacterial and fungal infections in 5 cases improved with symptomatic treatment.All 9 children demonstrated complete donor chimerism within 1 month after transplantation, at two years of follow-up, all nine children survived without recurrence or development of grade II-IV GVHD, and there were no children with transplant-related deaths.

Conclusion: Haploidentical hematopoietic stem cell transplantation combined with umbilical cord blood transfusion for aplastic anaemia in children has a low incidence and mild degree of GVHD, with significant efficacy, and can be used as a therapeutic option for children without an HLA full donor chimeric match.

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[单倍体造血干细胞联合脐带血双移植治疗儿童再生障碍性贫血的临床观察]。

目的探讨单倍体造血干细胞移植联合脐带血输注治疗儿童再生障碍性贫血的有效性和安全性：纳入2021年1月1日至2023年9月15日在河南中医药大学附属人民医院接受脐带血联合单倍体造血干细胞移植治疗的再生障碍性贫血患儿9例，中位年龄11（2-13）岁，中位随访18（7.5-21）个月，回顾性分析临床资料。对患者的造血重建、移植物抗宿主病（GVHD）发生率、感染和存活率进行了分析：结果：9名患儿均成功移植。中性粒细胞和血小板植入的中位时间分别为（11.11±1.27）d和（12.44±3.36）d。1例患儿出现急性胃肠道GVHD（Ⅰ度），治疗后好转，该患儿后期出现浅表性胃炎和慢性胃肠道GVHD，目前正在临床随访中。Ⅱ-Ⅳ度急性 GVHD 为 0%。3例出血性膀胱炎、5例CMV感染、5例细菌和真菌感染经对症治疗后好转。所有9名患儿均在移植后1个月内显示出完全的供体嵌合，在两年的随访中，所有9名患儿均存活，没有复发或出现II-IV度GVHD，也没有患儿因移植相关原因死亡：结论：同种异体造血干细胞移植联合脐带血输注治疗儿童再生障碍性贫血的GVHD发生率低、程度轻、疗效显著，可作为无HLA完全供体嵌合匹配儿童的治疗选择。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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