Unrelated umbilical cord blood transplantation for children with hereditary leukodystrophy: A retrospective study.

ABSTRACT:

Objective

To analyze the efficiency of unrelated umbilical cord blood transplantation (UCBT) in the treatment of hereditary leukodystrophy following busulfan- and cyclophosphamide-based myeloablative chemotherapy.

Methods

A retrospective study was performed in patients with hereditary leukodystrophy who underwent UCBT after myeloablative chemotherapy between April 2015 and March 2020.

Results

The study cohort included 12 pediatric patients (ten males), nine with cerebral adrenoleukodystrophy (ALD) and three with juvenile globoid cell leukodystrophy (GLD). All received HLA-matched or partially mismatched unrelated UCBT. There were no cases of graft rejection. Median neutrophil engraftment time was 20 days [12-33 days] and median platelet engraftment time was 29 days [1

SUBMITTER: Wang P 

PROVIDER: S-EPMC9561100 | biostudies-literature | 2022

REPOSITORIES: biostudies-literature