Encouraging outcome of children with beta-thalassemia major who
underwent fresh cord blood transplantation from an HLA-matched sibling
donor
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 β-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 8.51×107/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 β-TM receiving
F-CBT (P=0.032). Conclusions: The above results indicate that patients
with β-TM have excellent outcomes after F-CBT from an HLA-MSD.