Across the Myeloablative Spectrum: Hematopoietic Cell Transplant Conditioning Regimens for Pediatric Patients with Sickle Cell Disease.
allogeneic transplant
conditioning intensity
myeloablative conditioning
non-myeloablative conditioning
reduced intensity conditioning
sickle cell disease
Journal
Journal of clinical medicine
ISSN: 2077-0383
Titre abrégé: J Clin Med
Pays: Switzerland
ID NLM: 101606588
Informations de publication
Date de publication:
03 Jul 2022
03 Jul 2022
Historique:
received:
07
05
2022
revised:
18
06
2022
accepted:
22
06
2022
entrez:
9
7
2022
pubmed:
10
7
2022
medline:
10
7
2022
Statut:
epublish
Résumé
One out of every five hundred African American children in the United States has sickle cell disease (SCD). While multiple disease-modifying therapies are available, hematopoietic cell transplantation (HCT) remains the only curative option for children with SCD. HLA-matched sibling HCT has demonstrated excellent efficacy, but its availability remains limited; alternative donor strategies are increasingly explored. While Busulfan-Cyclophosphamide has become the most widespread conditioning regimen employed in HCT for pediatric SCD, many other regimens have been examined. This review explores different conditioning regimens across the intensity spectrum: from myeloablative to non-myeloablative. We describe survival and organ function outcomes in pediatric SCD patients who have received HCT and discuss the strengths and weaknesses of the various conditioning intensities. Finally, we posit novel directions in allogeneic HCT for SCD.
Identifiants
pubmed: 35807140
pii: jcm11133856
doi: 10.3390/jcm11133856
pmc: PMC9267729
pii:
doi:
Types de publication
Journal Article
Review
Langues
eng
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