Hematopoietic stem cell transplantation for thalassemia major using HLA fully-matched and mismatched donor grafts

Until now, allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the only effective method to cure Thalassemia major. However, it has not been determined whether similar results can be obtained with the same conditioning regimen for both fully-matched and mismatched donors grafts. We hypothesized that using modified NF-08-TM conditioning regimen could achieve similar results for both fully and mismatched donors grafts.

This retrospective cohort study included patients with β-thalassemia major who underwent HSCT with modified NF-08-TM conditioning regimen at Guangzhou Women and Children's Medical Centre between January 2013 and January 2019.

Among the 257 patients (172 males) included in this study, 3 had two transplantations. Totally 193 and 67 had fully-matched and mismatched donors were examined, respectively. The median follow-up was 29 months; 6-year overall survival (OS), thalassemia-free survival (TFS), graft rejection (GR) and transplantation-related mortality (TRM) were 92.08%, 90.89%, 1.24% and 8.01%, respectively. Multivariate analysis showed that human leukocyte antigen (HLA) compatibility between patient and donor was not independently associated with OS, TFS, GR or TRM. Mismatched donor graft transplantation for β-thalassemia major is associated with similar survival outcomes and incidences of complications (except for acute GVHD) to fully-matched donor graft transplantation based on modified NF-08-TM conditioning regimen.

In conclusion, based on the modified NF-08-TM regimen, certain mismatched donor transplantations for thalassemia major patients could achieve similar results as fully-matched donor transplantations.