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\begin{document}

\title{Encouraging outcome of children with beta-thalassemia major who
underwent fresh cord blood transplantation from an HLA-matched sibling
donor}



\author[1]{Jianyun Wen}%
\author[2]{Xiaodong Wang}%
\author[1]{libai Chen}%
\author[3]{Yuelin He}%
\author[1]{Xiaoqin  Feng}%
\author[3]{Chunfu  Li}%
\author[1]{Yongsheng Ruan}%
\author[2]{Sixi Liu}%
\author[1]{Xuedong Wu}%
\affil[1]{Southern Medical University Nanfang Hospital}%
\affil[2]{Shenzhen Children's Hospital}%
\affil[3]{Nanfang-Chunfu Children's Institute of Hematology \& Oncology}%


\vspace{-1em}



  
  \date{May 29, 2024}


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\selectlanguage{english}
\begin{abstract}
Background: Allogeneic hematopoietic stem cell
transplantation(allo-HSCT) is currently the only curative treatment for
thalassemia major (TM). Cord blood (CB) from a sibling has different
characteristics from marrow and has potential advantages and
disadvantages as a stem cell source. Methods: We retrospectively
analysed 68 children with \selectlanguage{greek}β-\selectlanguage{english}TM who underwent fresh cord blood
transplantation (F-CBT) from an human leukocyte antigen (HLA)-matched
sibling donor (MSD) between June 2010 and July 2018 in the Department of
Pediatrics, Nanfang Hospital and Haematology-Oncology, Shenzhen
Children's Hospital. Results: The median infused doses of total
nucleated cells (TNCs) and CD34+ cells were 8.51\selectlanguage{ngerman}×107/kg and
3.16×105/kg,respectively. The median time to neutrophil and platelet
engraftment were respectively 27 days and 31 days. The cumulative
probability of acute and chronic graft- versus-host disease (GVHD) were
very low after F-CBT (7.8\% and 0.0\%, respectively). Of the 68
paediatric patients, 67 patients survived during a median follow-up
period of 61 months.The estimated 5-year probability of overall survival
(OS) and disease-free survival (DFS) were 98.5\% and 92.4\%,
respectively. Three patients experienced graft rejection (GR) (4.5\%) ,
and this study found that GR was higher in the thiotepa (TT)-free
regimen group than that in the TT-based regimen group (0\%
vs.10.7\%,P=0.038). Multivariable prognostic analysis, a conditioning
regimen including TT, improved the DFS of patients with \selectlanguage{greek}β-\selectlanguage{english}TM receiving
F-CBT (P=0.032). Conclusions: The above results indicate that patients
with \selectlanguage{greek}β-\selectlanguage{english}TM have excellent outcomes after F-CBT from an HLA-MSD.%
\end{abstract}%



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