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Umbilical cord blood transplantation in children with beta-thalassemia diseases
Preeda Vanichsetakul1, Teera Wacharaprechanont, Rachanee O-Charoen
1Department of Pediatrics, Faculty of Medicine, Chulalongkorn University, Bangkok 10330, Thailand.
Insights
Umbilical cord blood transplantation (CBT) is a viable option for Thai children with beta-thalassemia disease. This study shows promising survival rates and transfusion independence following CBT.
Area of Science:
- Hematology
- Pediatric Oncology
- Transplantation Immunology
Background:
- Beta-thalassemia disease is a severe inherited blood disorder requiring lifelong transfusions.
- Hematopoietic stem cell transplantation (HSCT) offers a potential cure but donor availability can be a challenge.
- Umbilical cord blood (UCB) is an alternative stem cell source for HSCT.
Purpose of the Study:
- To evaluate the outcomes of UCB transplantation (CBT) in Thai children with beta-thalassemia.
- To identify factors influencing the success of CBT from sibling and unrelated donors.
Main Methods:
- A case-series study involving six children with beta-thalassemia major undergoing CBT.
- Analysis of donor types (HLA-identical sibling, mismatched sibling, unrelated UCB) and infused cell counts (nucleated and CD34+ cells).
- Assessment of engraftment, chimerism, graft-versus-host disease (GVHD), survival, and transfusion independence.
Main Results:
- Five of six children survived with transfusion independence at a median follow-up of 7 months.
- Two patients achieved complete donor engraftment, and three showed mixed chimerism (MC).
- Two of three MC patients were clinically cured; GVHD was manageable with immunosuppression.
Conclusions:
- Umbilical cord blood is a feasible stem cell source for treating beta-thalassemia in children.
- CBT demonstrated favorable outcomes in this cohort, suggesting its potential as a therapeutic option in Thailand.
- Further research can explore optimizing CBT protocols for improved engraftment and reduced complications.
Abstract:
To evaluate factors affecting the outcome of sibling and unrelated donor umbilical cord blood transplantation (CBT) in Thai children with beta-thalassemia diseases. The case-series study of all children undergoing such transplants in our institute was conducted Six children with thalassemia major were diagnosed at a median age of 1.5 years and CBT was performed at a median age of 5.5 years (range 2-15). Six donors consisted of three HLA-identical siblings, one two-allele, one three-antigen mismatched sibling, and one one-allele mismatched unrelated cord blood. The median number of nucleated cells infused was 2.83 x 10(7)/kg (range 1.49-5.3); the median number of CD34+ cells infused was 1.94 x 10(5)/kg (range 0.2-5.3). In all, two patients had complete donor engraftment; three had mixed chimerism (MC); one patient died of cerebral thrombosis and neutropenic septicemia. Of the two complete donor-engrafted patients, two developed grade 2 acute graft-versus-host disease (GVHD) which responded well to immunosuppressive therapy. Of the three mixed-chimeric patients, two were clinically cured. With a median follow-up of 7 months (range 2-30), five children survived and have done well with transfusion-independent. Umbilical cord blood provides a reasonable option for hematopoietic stem cell source to transplant for beta-thalassemia diseases and the outcome in the present study was good.
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