Haploidentical Stem Cell Transplantation with Post-Transplant Cyclophosphamide in Fanconi Anemia: Improving Outcomes with Improved Supportive Care in India.
Fanconi anemia
Ferritin
Haploidentical stem cell transplantation
N-acetyl cysteine
Post-transplant cyclophosphamide
Ruxolitinib
Journal
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
ISSN: 1523-6536
Titre abrégé: Biol Blood Marrow Transplant
Pays: United States
ID NLM: 9600628
Informations de publication
Date de publication:
12 2020
12 2020
Historique:
received:
09
06
2020
revised:
24
07
2020
accepted:
16
08
2020
pubmed:
25
8
2020
medline:
24
6
2021
entrez:
25
8
2020
Statut:
ppublish
Résumé
Fanconi anemia is the most common inherited bone marrow failure syndrome, and hematopoietic stem cell transplantation (HSCT) is the only curative option. Post-transplant cyclophosphamide (PTCy) is challenging in this group of children, given their increased sensitivity to chemotherapy. We performed a retrospective analysis of the data on children diagnosed with Fanconi anemia who underwent a haploidentical HSCT with PTCy from January 2014 to December 2019. Nineteen children (male/female, 0.75:1) underwent 21 haplo-HSCTs with PTCy. Fludarabine, low-dose cyclophosphamide, and 200 centi-gray total body irradiation were included in the conditioning regimen with 25 mg/kg PTCy on days +3 and +4. Haplo-graft was from a sibling in 38% and father in 57% of transplants. The source of stem cells was peripheral blood stem cells in 81% and bone marrow in 19% of transplants, with a median CD34 dose of 5.0 × 10
Identifiants
pubmed: 32835780
pii: S1083-8791(20)30511-5
doi: 10.1016/j.bbmt.2020.08.019
pii:
doi:
Substances chimiques
Cyclophosphamide
8N3DW7272P
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
2292-2298Informations de copyright
Copyright © 2020 American Society for Transplantation and Cellular Therapy. Published by Elsevier Inc. All rights reserved.