Treatment of Beta Thalassemia Major in Transfusion-Dependent Adults (Age ≥18)

This protocol addresses beta thalassemia major in adults aged 18 years or older whose disease is transfusion-dependent — a population where reducing the ongoing transfusion burden is the central clinical objective.

Clinical Scenario

Transfusion-dependent β-thalassemia (TDT) in an adult patient aged 18 years or older. The need for regular transfusions characterises this population and defines the primary therapeutic target.

Treatment Approach

A specific subcutaneously administered biologic agent is indicated for this population, with a structured dose escalation pathway based on the patient’s transfusion response over the initial treatment period.

The complete regimen, escalation criteria, and dosing schedule are available in the full protocol.

Treatment Goal

Reduction in transfusion burden, assessed after an initial defined treatment period.

Instant Access to Structured Evidence-Based Regimens

References

DOI: 10.1002/hem3.70095

Luspatercept is recommended in TDT adults (≥18 years) to achieve transfusion burden reduction (Grade B, Class I).
Starting dose of 1 mg/kg subcutaneously every 3 weeks.
Dose increase to 1.25 mg/kg if patient has no reduction in transfusion burden after at least 2 consecutive doses (6 weeks) of 1 mg/kg.

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