1. Hematopoietic stem cell transplantation for thalassemia major using HLA fully-matched and mismatched donor grafts
- Author
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Hua Jiang, Yuhua Qu, Shushan Nie, Sha Liu, and Chuwen Huang
- Subjects
medicine.medical_specialty ,Graft rejection ,business.industry ,Thalassemia ,medicine.medical_treatment ,Retrospective cohort study ,Human leukocyte antigen ,Hematopoietic stem cell transplantation ,medicine.disease ,Gastroenterology ,Transplantation ,Regimen ,surgical procedures, operative ,Donor graft ,Internal medicine ,Pediatrics, Perinatology and Child Health ,medicine ,Original Article ,business - Abstract
Background 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. Methods 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. Results 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. Conclusions 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.
- Published
- 2020