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

Abstract

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 conducted. Neurologic function scores (NFS) were assessed. The conditioning regimen was busulfan 9.6 mg/kg, cyclophosphamide 200 mg/kg, and fludarabine 90 mg/m2 (BFC). Graft-versus-host disease prophylaxis consisted of anti-human thymocyte globulin, cyclosporine A, mycophenolate mofetil, and short course of methotrexate.

RESULTS:

Among the 29 cases, 14 cases (NFS = 0) were asymptomatic, and 15 (NFS ≥ 1) were symptomatic. The median age at SCT was 8 years (range: 4-16 years); the median follow-up time was 1058 days (range: 398-3092 days); 28 cases were father donors and 1 case was a grandfather donor. Hematopoietic reconstitution was successful in all patients, and all of them achieved complete donor chimerism at the time of engraftment. The leading cause of death was still primary disease progression (n = 4). Survival free of major functional disabilities was 100% in asymptomatic patients versus 66.67% in the symptomatic group (p = .018).

CONCLUSION:

BFC regimen used in haploidentical SCT was administered safely without major transplant-related complications even in symptomatic patients, and neurological symptoms were stabilized after SCT.

• Authors: Yao Chen, Lan-Ping Xu, Xiao-Hui Zhang, Huan Chen, Kai-Yan Liu, Jiong Qing, Yan-Ling Yang, Xiao-Jun Huang
• Journal: Pediatric transplantation (Pediatr Transplant) Vol. 28 Issue 3 Pg. e14735 (May 2024) ISSN: 1399-3046 [Electronic] Denmark
• PMID: 38602169 (Publication Type: Journal Article)
• Copyright: © 2024 Wiley Periodicals LLC.

Chemical References

• Busulfan
• fludarabine
• Cyclophosphamide
• Antilymphocyte Serum
• Vidarabine

Topics

• Humans
• Child
• Child, Preschool
• Adolescent
• Busulfan (therapeutic use)
• Retrospective Studies
• Graft vs Host Disease (etiology)
• Transplantation Conditioning (adverse effects)
• Hematopoietic Stem Cell Transplantation (adverse effects)
• Cyclophosphamide (therapeutic use)
• Antilymphocyte Serum (therapeutic use)
• Adrenoleukodystrophy (therapy, complications)
• Vidarabine (analogs & derivatives)