Context
A two-year-old girl became a blood stem cell donor for her elder brother suffering from thalassemia major after being identified as a 12/12 Human Leukocyte Antigen (HLA) match. The case highlights the importance of HLA matching and hematopoietic stem cell transplantation in treating severe thalassemia.
Explanation
- Thalassemia is an inherited autosomal recessive blood disorder caused by reduced or absent synthesis of alpha or beta globin chains of haemoglobin.
- Beta-thalassemia major is a severe form in which patients may require regular blood transfusions and iron-chelation therapy to prevent iron overload.
- Hematopoietic stem cells (HSCs) are multipotent cells that produce red blood cells, white blood cells and platelets.
- They occur mainly in bone marrow and can also be obtained from peripheral blood and umbilical-cord blood.
- Hematopoietic stem cell transplantation (HSCT) can provide a potential cure for eligible thalassemia patients by replacing defective blood-forming stem cells with healthy ones.
- Human Leukocyte Antigen (HLA) molecules help the immune system distinguish self from non-self.
- Close HLA matching between donor and recipient improves transplant success and reduces graft-versus-host disease (GVHD).
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