Impact of Treosulfan Exposure on Early and Long-Term Clinical Outcomes in Pediatric Allogeneic Hematopoietic Stem Cell Transplantation Recipients: A Prospective Multicenter Study.
Conditioning regimen
Nonmalignant diseases
Pediatrics
Pharmacokinetics
Treosulfan
Journal
Transplantation and cellular therapy
ISSN: 2666-6367
Titre abrégé: Transplant Cell Ther
Pays: United States
ID NLM: 101774629
Informations de publication
Date de publication:
02 2022
02 2022
Historique:
received:
18
06
2021
revised:
22
09
2021
accepted:
22
09
2021
pubmed:
5
10
2021
medline:
21
4
2022
entrez:
4
10
2021
Statut:
ppublish
Résumé
Treosulfan-based conditioning has gained popularity in pediatric allogeneic hematopoietic stem cell transplantation (HSCT) because of its presumed favorable efficacy and toxicity profile. Treosulfan is used in standardized dosing regimens based on body surface area. The relationships between systemic treosulfan exposure and early and long-term clinical outcomes in pediatric patients undergoing allogeneic HSCT for nonmalignant diseases remain unclear. In this a multicenter, prospective observational study, we assessed the association between treosulfan exposure and early and, in particular, long-term clinical outcomes. Our study cohort comprised 110 pediatric patients with nonmalignant diseases who underwent HSCT between 2011 and 2019 in Leiden, The Netherlands and Rome, Italy. Blood samples were collected, and treosulfan area under the receiver operating characteristic curve (AUC
Identifiants
pubmed: 34607071
pii: S2666-6367(21)01259-8
doi: 10.1016/j.jtct.2021.09.018
pii:
doi:
Substances chimiques
treosulfan
CO61ER3EPI
Busulfan
G1LN9045DK
Types de publication
Journal Article
Multicenter Study
Observational Study
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
99.e1-99.e7Informations de copyright
Copyright © 2021 The American Society for Transplantation and Cellular Therapy. Published by Elsevier Inc. All rights reserved.