Patients with transfusion-dependent β-thalassemia who have access to an HLA-identical donor represent a specific clinical population for whom a curative strategy is considered the priority, ideally before complications from chronic transfusion arise.
Clinical scenario: Transfusion-dependent β-thalassemia (TDT) in a patient with an available HLA-identical donor. Current evidence supports offering a transplant-based approach to eligible patients and families at an early stage, before iron overload-related complications develop.
The structured regimen centers on hematopoietic cell transplantation, with defined protocols governing donor source selection, HLA compatibility criteria, and the conditioning strategy used.
HCT should be offered to TDT patients and their parents at an early age, before complications due to iron overload develop, if an HLA identical donor is available (Grade B, Class I).
Either bone marrow or cord blood from an HLA-identical sibling can be used (Grade B, Class I).
A matched unrelated donor can be used, provided that high compatibility criteria for both HLA class I and II loci are met (Grade B, Class I).
Myeloablative conditioning regimens should always be used for standard transplantation (Grade B, Class I).
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