Liu Huan-Jun, Chen Xing-Hua, Zhang Wen-Bang, Guo Shu-Xia
The Fifth Clinical College Affiliated to Henan University of Chinese Medicine (Zhengzhou People's Hospital).
Department of Hematology, People's Hospital of Henan University of Chinese Medicine, Zhengzhou 450003, Henan Province, China.
Zhongguo Shi Yan Xue Ye Xue Za Zhi. 2024 Jun;32(3):890-895. doi: 10.19746/j.cnki.issn.1009-2137.2024.03.037.
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.
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.
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.
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.
探讨单倍型造血干细胞移植联合脐血输注治疗儿童再生障碍性贫血的疗效及安全性。
纳入2021年1月1日至2023年9月15日在河南中医药大学人民医院接受脐血联合单倍型造血干细胞移植治疗的9例儿童再生障碍性贫血患者，中位年龄11（2 - 13）岁，中位随访时间18（7.5 - 21）个月，对临床资料进行回顾性分析。分析患者的造血重建、移植物抗宿主病（GVHD）发生率、感染情况及生存情况。
9例患儿均成功植入。中性粒细胞和血小板植入的中位时间分别为11.11±1.27天和12.44±3.36天。1例发生I度急性胃肠道GVHD，治疗后好转，后期患者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例发生出血性膀胱炎，5例发生巨细胞病毒感染，5例发生细菌和真菌感染，经对症治疗后好转。9例患儿移植后1个月内均表现为完全供体嵌合，随访2年时，9例患儿均存活，无II - IV度GVHD复发或进展，无移植相关死亡患儿。
单倍型造血干细胞移植联合脐血输注治疗儿童再生障碍性贫血GVHD发生率低、程度轻，疗效显著，可作为无HLA全相合供体儿童的治疗选择。
