Busulfan-fludarabine- or treosulfan-fludarabine-based myeloablative conditioning for children with thalassemia major.
Adolescent
Antineoplastic Agents
/ therapeutic use
Busulfan
/ analogs & derivatives
Child
Child, Preschool
Female
Hematopoietic Stem Cell Transplantation
Humans
Immunosuppressive Agents
/ therapeutic use
Infant
Male
Myeloablative Agonists
/ therapeutic use
Retrospective Studies
Transplantation Conditioning
Vidarabine
/ analogs & derivatives
beta-Thalassemia
/ therapy
Busulfan
Conditioning
Hematopoietic stem cell transplantation
Thalassemia major
Treosulfan
Journal
Annals of hematology
ISSN: 1432-0584
Titre abrégé: Ann Hematol
Pays: Germany
ID NLM: 9107334
Informations de publication
Date de publication:
Mar 2022
Mar 2022
Historique:
received:
20
06
2021
accepted:
26
11
2021
pubmed:
10
1
2022
medline:
11
2
2022
entrez:
9
1
2022
Statut:
ppublish
Résumé
Significant advances in supportive care for patients with transfusion-dependent thalassemia major (TDT) have improved patients' life expectancy. However, transfusion-associated iron overload remains a significant barrier to long-term survival with good quality of life. Today, allogeneic hematopoietic stem cell transplantation (HSCT) is the current curative standard of care. Alongside selection of the best available donor, an optimized conditioning regimen is crucial to maximize outcomes for patients with TDT undergoing HSCT. The aim of this retrospective analysis was to investigate the role of busulfan-fludarabine-based and treosulfan-fludarabine-based conditioning in TDT patients undergoing HSCT. We included 772 patients registered in the European Society for Blood and Marrow Transplantation (EBMT) database who underwent first HSCT between 2010 and 2018. Four hundred ten patients received busulfan-fludarabine-based conditioning (median age 8.6 years) and 362 patients received treosulfan-fludarabine-based conditioning (median age 5.7 years). Patient outcomes were retrospectively compared by conditioning regimen. Two-year overall survival was 92.7% (95% confidence interval: 89.3-95.1%) after busulfan-fludarabine-based conditioning and 94.7% (95% confidence interval: 91.7-96.6%) after treosulfan-fludarabine-based conditioning. There was a very low incidence of second HSCT overall. The main causes of death were infections, graft-versus-host disease, and rejection. In conclusion, use of busulfan or treosulfan as the backbone of myeloablative conditioning for patients with TDT undergoing HSCT resulted in comparably high cure rates. Long-term follow-up studies are warranted to address the important issues of organ toxicities and gonadal function.
Identifiants
pubmed: 34999929
doi: 10.1007/s00277-021-04732-4
pii: 10.1007/s00277-021-04732-4
doi:
Substances chimiques
Antineoplastic Agents
0
Immunosuppressive Agents
0
Myeloablative Agonists
0
treosulfan
CO61ER3EPI
Vidarabine
FA2DM6879K
Busulfan
G1LN9045DK
fludarabine
P2K93U8740
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
655-665Informations de copyright
© 2021. The Author(s), under exclusive licence to Springer-Verlag GmbH Germany, part of Springer Nature.
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