Treatment of Beta Thalassemia Major in Transfusion-Dependent Patients Aged 14 Years or Older
Clinical Scenario
This protocol addresses transfusion-dependent β-thalassemia (TDT) in patients who are 14 years of age or older, or who lack an HLA-identical family donor. In these circumstances, matched sibling transplantation is unavailable or not applicable, and an alternative curative strategy is indicated.
Relevant Condition — Age ≥14 or No HLA-Identical Donor
For patients aged 14 years and older, or for those who do not have an HLA-identical family donor, gene therapy constitutes an optimal therapeutic option (Grade C, Class IIa).
Treatment Approach (partial)
The protocol is built around a gene therapy strategy; agents in this class have received regulatory approvals across multiple jurisdictions for eligible TDT patients.
The complete regimen — including specific agents, eligibility criteria, and sequencing — is available via the link below.
References
DOI: 10.1002/hem3.70095
- For patients aged 14 years and older or for those who do not have an HLA‐identical family donor, gene therapy instead constitutes an optimal therapeutic option (Grade C, Class IIa):
- Beti‐cel is currently available only in the USA, where it is FDA‐approved for pediatric and adult subjects with TDT, irrespective of the patient's age and genotype.
- Exa‐cel has been recently approved by regulatory agencies in Europe (EMA), North America (FDA), the UK, Bahrain, and Saudi Arabia for TDT patients aged 12 years and above, without an upper age limit.
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