Prognosis of childhood adrenoleukodystrophy with cognitive disorder after haploidentical allogenic hemato-poietic stem cell transplantation
10.3760/cma.j.cn101070-20221205-01367
- VernacularTitle:伴认知障碍的儿童肾上腺脑白质营养不良单倍体异基因造血干细胞移植的预后分析
- Author:
Yu′ang AN
1
;
Ying ZHANG
;
Yijia ZHANG
;
Ya′nan CUI
;
Jingjing LIANG
;
Qinrui LI
;
Jie FU
;
Na FU
;
Yao CHEN
;
Yanling YANG
;
Jiong QIN
Author Information
1. 北京大学人民医院儿科，北京 100044
- Keywords:
Adrenoleukodystrophy;
Haploidentical allogenic hematopoietic stem cell transplantation;
Cognitive disorder
- From:
Chinese Journal of Applied Clinical Pediatrics
2023;38(8):566-570
- CountryChina
- Language:Chinese
-
Abstract:
Objective:To investigate the prognosis of childhood adrenoleukodystrophy (ALD) with cognitive disorder after haploidentical allogenic hematopoietic stem cell transplantation (haplo-HSCT), and to identify risk factors affecting the prognosis.Methods:It was a single-center retrospective study involving 31 ALD children receiving haplo-HSCT in Peking University People′s Hospital from January 2014 to October 2022.Survival analysis was performed by Kaplan-Meier method. Cox regression analysis was performed to identify risk factors for the prognosis of childhood ALD following haplo-HSCT. Results:Among the 31 children with ALD, 1 case died of cardiogenic shock during the transplantation, and the remaining had a successful haplo-HSCT.Ten children with ALD had cognitive disorder before haplo-HSCT, including 3 cases with the minimal LOES score ≥10 points and 8 cases with the Neurologic Function Score (NFS)>0 point before haplo-HSCT.Six children had major functional disability (MFD) and 2 cases died due to progression of ALD after haplo-HSCT.Twenty children did not have cognitive disorder before haplo-HSCT, of whom 3 cases had the LOES score≥10 points and 6 cases had NFS>0 before haplo-HSCT.Four children had MFD and 2 cases died due to progression of ALD after haplo-HSCT.For ALD patients without cognitive disorder after haplo-HSCT, the 3-year and 5-year survival rate were 100.0% and 72.9%, respectively, and the 5-year MFD-free survival was 61.6%.For ALD patients with cognitive disorder after haplo-HSCT, the 3-year survival rate was 83.3%.Compared with ALD patients with the LOES score<10 points before haplo-HSCT, those with the LOES score≥10 points had 9.243 times the risk of developing MFD after haplo-HSCT ( P=0.024, 95% CI: 1.332-64.127). Compared with ALD patients without cognitive disorder before haplo-HSCT, ALD patients with cognitive disorder had 9.749 times the risk of developing MFD after haplo-HSCT ( P=0.023, 95% CI: 1.358-66.148). Conclusions:Cognitive disorder and LOES score≥10 points before haplo-HSCT are risk factors for developing MFD in children with ALD following haplo-HSCT.
