ArticleTranslational pediatrics2021
Hematopoietic stem cell transplantation for thalassemia major using HLA fully-matched and mismatched donor grafts.
Article in Translational pediatrics, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.
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Who cites it
7 citing papers in PubMed, 19 citations in OpenAlex.
- Bringing the rare into sight: applications of haploidentical stem cell transplantation in the treatment of rare diseases.Bone marrow transplantation · 2026Review
- A novel conditioning regimen with pre-transplantation immunosuppression reduces the complication rates in hematopoietic stem cell transplantation in transfusion-dependent β-thalassemia.Stem cell research & therapy · 2024Article
- Finding a balance in reduced toxicity hematopoietic stem cell transplantation for thalassemia: role of infused CD3+ cell count and immunosuppression.Bone marrow transplantation · 2024Article
- Nutritional and Body Composition Changes in Paediatric β-Thalassemia Patients Undergoing Hematopoietic Stem Cell Transplantation: A Retrospective Study Using Bioelectrical Impedance Analysis.Journal of multidisciplinary healthcare · 2024Article
- Case report: Curing a rare, unstable hemoglobin variant Hb Bristol-Alesha using haploidentical hematopoietic stem cell transplantation.Frontiers in immunology · 2023Article
- Health-related quality of life in pediatric patients with β-thalassemia major after hematopoietic stem cell transplantation.Bone marrow transplantation · 2022Article
- Genetic Manipulation Strategies for β-Thalassemia: A Review.Frontiers in pediatrics · 2022Review
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Authors and funding
5 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundUntil 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.
methodsThis 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.
resultsAmong 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.
conclusionsIn 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.
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