Encouraging the outcomes of children with beta-thalassaemia major who underwent fresh cord blood transplantation from an HLA-matched sibling donor

Autor: Jianyun Wen, Xiaodong Wang, Libai Chen, Yuelin He, Xiaoqin Feng, Chunfu Li, Yongshen Ruan, Sixi Liu, Xuedong Wu
Jazyk: angličtina
Rok vydání: 2022
Předmět:
Zdroj: Hematology, Vol 27, Iss 1, Pp 310-317 (2022)
Druh dokumentu: article
ISSN: 16078454
1607-8454
DOI: 10.1080/16078454.2022.2038402
Popis: Background Allogeneic haematopoietic stem cell transplantation (allo-HSCT) is currently the only curative treatment for thalassaemia 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 analyzed 68 children with beta-thalassaemia major (β-TM) who underwent fresh cord blood transplantation (F-CBT) from an 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 8.51×107/kg and 3.16×105/kg, respectively. The median time to neutrophil and platelet engraftment were, respectively, 27 and 31 days. The cumulative probabilities 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 87.9%, respectively. Three patients experienced graft rejection (GR) (4.5%), and we identified CD34 + cell dose as a significant risk factor for graft failure (p = 0.036) in stratify analysis.Conclusions The above results indicate that patients with β-TM have excellent outcomes after F-CBT from an HLA-MSD.
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