A novel conditioning regimen with pre-transplantation immunosuppression reduces the complication rates in hematopoietic stem cell transplantation in transfusion-dependent β-thalassemia

Allo-HSCT is a curative therapy for patients with transfusion-dependent thalassemia (TDT). The high incidence of transplant-related complications is becoming an obstacle to safe and effective unrelated donor (URD) transplantation. In this retrospective study, we reported the survival outcomes and co...

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Published in:Stem cell research & therapy 2024-12, Vol.15 (1), p.495-11
Main Authors: Yang, Huaqing, Li, Xinyu, Que, Liping, Chen, Han, Zhan, Liping, Zhou, Dunhua, Li, Yang, Lin, Shaofen, Wang, Yin, Wu, Xiaojun, Han, Xiawei, Wu, Zhengzhou, Zhong, Danping, Huang, Ke, Xu, Honggui, Fang, Jianpei
Format: Article
Language:English
Subjects:
Adolescent
Adult
beta-Thalassemia - therapy
Blood Transfusion
Busulfan - administration & dosage
Busulfan - therapeutic use
Child
Child, Preschool
children
Cyclophosphamide - administration & dosage
Cyclophosphamide - therapeutic use
Female
hematopoietic stem cell transplantation
Hematopoietic Stem Cell Transplantation - adverse effects
Hematopoietic Stem Cell Transplantation - methods
Humans
Immunosuppression Therapy - methods
Immunosuppressive Agents - therapeutic use
Infant
Male
pre-transplantation immunosuppression
Retrospective Studies
transfusion-dependent β-thalassemia
Transplantation Conditioning - methods
unrelated donor transplantation
Vidarabine - administration & dosage
Vidarabine - analogs & derivatives
Vidarabine - therapeutic use
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Summary:Allo-HSCT is a curative therapy for patients with transfusion-dependent thalassemia (TDT). The high incidence of transplant-related complications is becoming an obstacle to safe and effective unrelated donor (URD) transplantation. In this retrospective study, we reported the survival outcomes and complications of transplantation in thalassemia patients using a novel regimen consisting of pre-transplantation immunosuppression (PTIS) and modified myeloablative conditioning based on intravenous busulfan, cyclophosphamide, fludarabine, and rabbit anti-human thymocyte immunoglobulin. A total of 88 thalassemia patients received the novel conditioning regimen (NCR group), while 118 patients received the conventional conditioning regimen (CCR group). The median age at HSCT in the NCR group was older (7 years vs. 4 years, p 
ISSN:1757-6512
1757-6512
DOI:10.1186/s13287-024-04103-6