Haploidentical hematopoietic stem cell transplantation with busulfan, cyclophosphamide, and fludarabine conditioning for X‐linked adrenal cerebral leukodystrophy

• Published in: Pediatric transplantation Vol. 28; no. 3; pp. e14735 - n/a
• Main Authors: Chen, Yao, Xu, Lan‐ping, Zhang, Xiao‐hui, Chen, Huan, Liu, Kai‐yan, Qing, Jiong, Yang, Yan‐ling, Huang, Xiao‐jun
• Format: Journal Article
• Language: English
• Published: Denmark Wiley Subscription Services, Inc 01.05.2024
• Subjects: Adrenoleukodystrophy; Asymptomatic; Busulfan; Chimerism; Cyclophosphamide; Cyclosporins; engraftment; Fludarabine; Globulins; graft‐versus‐host disease; haploidentical; Hematopoietic stem cells; Leukodystrophy; Methotrexate; Mycophenolate mofetil; Mycophenolic acid; Pediatrics; Prophylaxis; Stem cell transplantation; engraftment; graft‐versus‐host disease; haploidentical; adrenoleukodystrophy; stem cell transplantation

Objective We investigated the safety and efficacy of haploidentical stem cell transplantation (SCT) in pediatric patients with X‐linked adrenoleukodystrophy (ALD). Methods A retrospective analysis of transplantation data from 29 cases of ALD, treated between December 2014 and April 2022, was conduct...