Thiotepa-containing conditioning for allogeneic hematopoietic stem cell transplantation in children with inborn errors of immunity: a retrospective clinical analysis.
10.7499/j.issn.1008-8830.2504147
- Author:
Xiao-Jun WU
1
;
Xia-Wei HAN
1
;
Kai-Mei WANG
1
;
Shao-Fen LIN
1
;
Li-Ping QUE
1
;
Xin-Yu LI
1
;
Dian-Dian LIU
1
;
Jian-Pei FANG
1
;
Ke HUANG
1
;
Hong-Gui XU
1
Author Information
1. Department of Hematopoietic Stem Cell Transplantation, Children's Medical Center, Sun Yat-sen Memorial Hospital, Sun Yat-sen University, Guangzhou 510220, China.
- Publication Type:Journal Article
- Keywords:
Allogeneic hematopoietic stem cell transplantation;
Child;
Inborn error of immunity;
Thiotepa;
Umbilical cord blood
- MeSH:
Humans;
Retrospective Studies;
Transplantation Conditioning/methods*;
Thiotepa/therapeutic use*;
Hematopoietic Stem Cell Transplantation/adverse effects*;
Male;
Female;
Child, Preschool;
Infant;
Child;
Transplantation, Homologous;
Graft vs Host Disease;
Adolescent
- From:
Chinese Journal of Contemporary Pediatrics
2025;27(10):1240-1246
- CountryChina
- Language:Chinese
-
Abstract:
OBJECTIVES:To evaluate the safety and efficacy of thiotepa (TT)-containing conditioning regimens for allogeneic hematopoietic stem cell transplantation (HSCT) in children with inborn errors of immunity (IEI).
METHODS:Clinical data of 22 children with IEI who underwent HSCT were retrospectively reviewed. Survival after HSCT was estimated using the Kaplan-Meier method.
RESULTS:Nine patients received a traditional conditioning regimen (fludarabine + busulfan + cyclophosphamide/etoposide) and underwent peripheral blood stem cell transplantation (PBSCT). Thirteen patients received a TT-containing modified conditioning regimen (TT + fludarabine + busulfan + cyclophosphamide), including seven PBSCT and six umbilical cord blood transplantation (UCBT) cases. Successful engraftment with complete donor chimerism was achieved in all patients. Acute graft-versus-host disease occurred in 12 patients (one with grade III and the remaining with grade I-II). Chronic graft-versus-host disease occurred in one patient. The incidence of EB viremia in UCBT patients was lower than that in PBSCT patients (P<0.05). Over a median follow-up of 36.0 months, one death occurred. The 3-year overall survival (OS) rate was 100% for the modified regimen and 88.9% ± 10.5% for the traditional regimen (P=0.229). When comparing transplantation types, the 3-year OS rates were 100% for UCBT and 93.8% ± 6.1% for PBSCT (P>0.05), and the 3-year event-free survival rates were 100% and 87.1% ± 8.6%, respectively (P>0.05).
CONCLUSIONS:TT-containing conditioning for allogeneic HSCT in children with IEI is safe and effective. Both UCBT and PBSCT may achieve high success rates.
