Busulfan–fudarabine- or treosulfan–fudarabine-based myeloablative conditioning for children with thalassemia major
[ X ]
Tarih
2021
Dergi Başlığı
Dergi ISSN
Cilt Başlığı
Yayıncı
Annals of Hematology
Erişim Hakkı
info:eu-repo/semantics/closedAccess
Özet
Signifcant advances in supportive care for patients with transfusion-dependent thalassemia major (TDT) have improved
patients’ life expectancy. However, transfusion-associated iron overload remains a signifcant 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–
fudarabine-based and treosulfan–fudarabine-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 frst
HSCT between 2010 and 2018. Four hundred ten patients received busulfan–fudarabine-based conditioning (median age
8.6 years) and 362 patients received treosulfan–fudarabine-based conditioning (median age 5.7 years). Patient outcomes
were retrospectively compared by conditioning regimen. Two-year overall survival was 92.7% (95% confdence interval:
89.3–95.1%) after busulfan–fudarabine-based conditioning and 94.7% (95% confdence interval: 91.7–96.6%) after treosulfan–fudarabine-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.
Açıklama
Anahtar Kelimeler
Hematopoietic Stem Cell Transplantation, Conditioning, Thalassemia Major, Busulfan, Treosulfan
Kaynak
Annals of Hematology
WoS Q Değeri
Scopus Q Değeri
Q2
Cilt
Sayı
Künye
Lüftinger, R., Zubarovskaya, N., Galimard, J. E., Cseh, A., Salzer, E., Locatelli, F., ... & Corbacioglu, S. (2022). Busulfan–fludarabine-or treosulfan–fludarabine-based myeloablative conditioning for children with thalassemia major. Annals of Hematology, 1-11.
