DISEASE BACKGROUND
TDT is a severe genetic disease caused by impaired β-globin production that requires lifelong transfusion and chelation therapy3
Chronic supportive care can be a long-term challenge for patients with TDT3
- Current management for most TDT patients consists of lifelong transfusions of packed red blood cells (pRBCs)3
- This approach may alleviate anaemia and suppress the symptoms of TDT, but it does not address the genetic cause of the disease3
- Lifelong pRBC transfusions can lead to unavoidable iron overload, which can cause serious complications and lead to organ damage, particularly in the hepatic, cardiac, and endocrine systems3
- Chelation therapy helps to reduce iron overload caused by lifelong transfusions3
Allogenic haematopoietic stem cell transplant (HSCT) can address the genetic cause of TDT and is only available to a limited number of patients:
- Younger paediatric patients <17 years of age3
- Patients with a human leukocyte antigen (HLA)-matched sibling donor3
Transfusions provide temporary relief of anaemia symptoms and thus do not address the underlying genetic cause of TDT3
*Patients with TDT are recommended to maintain a pre-transfusion haemoglobin level of 9–10.5 g/dL.3