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1
Chronic transfusion and iron chelation remain the mainstay of TDT management, while allogeneic HCT and gene therapy are the only curative treatments described.
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2
The EHA-EBMT consensus updates 2021 guidance by incorporating newer treatments, transplantation data, and clinical evidence on gene addition and gene editing.
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3
The consensus algorithm makes liver disease assessment central to gene therapy selection, using hepatic fibrosis as a marker of cumulative oxidative injury.
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4
The panel also considered cardiac, endocrine, renal, and thrombotic complications, and distinguished ineligible patients from possible future candidates by organ damage, iron overload, and age.
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5
Long-term gene therapy follow-up remains limited, and the authors call for more data on treated patient numbers, observation duration, and survival compared with healthy populations and HCT.
