Successful umbilical cord blood transplantation in children with leukocyte adhesion deficiency type I.
Leukocyte adhesion deficiency type I (LAD-I)
primary immunodeficiency disease
umbilical cord blood transplantation (UCBT)
Journal
Translational pediatrics
ISSN: 2224-4344
Titre abrégé: Transl Pediatr
Pays: China
ID NLM: 101649179
Informations de publication
Date de publication:
Feb 2020
Feb 2020
Historique:
entrez:
11
3
2020
pubmed:
11
3
2020
medline:
11
3
2020
Statut:
ppublish
Résumé
This study aims to investigate the efficacy and safety of umbilical cord blood transplantation (UCBT) without serotherapy for treating children with leukocyte adhesion deficiency type I (LAD-I). Clinical characteristics and data of five children with LAD-I who underwent UCBT at our hospital between September 2016 and September 2018 were retrospectively analyzed. Five (two boys and three girls) patients with LAD-I were included. The median age at UCBT was 9 months (range, 8 to 32 months). The same myeloablative conditioning regimen was administered for each patient and included busulfan, fludarabine, and cyclophosphamide. HLA matching of patients and umbilical cord blood was 8/10 to 10/10. The median dose of total nucleated cells (TNC) infused was 10.2×10 UCBT is an effective treatment method for LAD-I patients. Also, severe LAD-I patients should undergo stem cell transplantation as early as possible.
Sections du résumé
BACKGROUND
BACKGROUND
This study aims to investigate the efficacy and safety of umbilical cord blood transplantation (UCBT) without serotherapy for treating children with leukocyte adhesion deficiency type I (LAD-I).
METHODS
METHODS
Clinical characteristics and data of five children with LAD-I who underwent UCBT at our hospital between September 2016 and September 2018 were retrospectively analyzed.
RESULTS
RESULTS
Five (two boys and three girls) patients with LAD-I were included. The median age at UCBT was 9 months (range, 8 to 32 months). The same myeloablative conditioning regimen was administered for each patient and included busulfan, fludarabine, and cyclophosphamide. HLA matching of patients and umbilical cord blood was 8/10 to 10/10. The median dose of total nucleated cells (TNC) infused was 10.2×10
CONCLUSIONS
CONCLUSIONS
UCBT is an effective treatment method for LAD-I patients. Also, severe LAD-I patients should undergo stem cell transplantation as early as possible.
Identifiants
pubmed: 32154133
doi: 10.21037/tp.2020.01.06
pii: tp-09-01-34
pmc: PMC7036647
doi:
Types de publication
Journal Article
Langues
eng
Pagination
34-42Informations de copyright
2020 Translational Pediatrics. All rights reserved.
Déclaration de conflit d'intérêts
Conflicts of Interest: The authors have no conflicts of interest to declare.
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