Alpha-Thalassaemia Major (Haemoglobin Bart’s Hydrops Foetalis): Management When Iron Chelation Does Not Achieve the Target Liver Iron Concentration
Infants with alpha-thalassaemia major (haemoglobin Bart’s hydrops foetalis) who survive the neonatal period become lifelong transfusion-dependent unless a curative intervention is pursued. When first-line iron chelation therapy fails to achieve the target liver iron concentration, an escalated approach is indicated.
Alpha-thalassaemia major (haemoglobin Bart’s hydrops foetalis) — a severe condition in which neonatal survivors will face lifelong transfusion dependence unless they receive a curative intervention.
Iron chelation therapy — with deferasirox, deferiprone, or deferoxamine — was the prior treatment. The clinical target was a liver iron concentration below 7 mg/g dry weight (ideally 2–5 mg/g dry weight). Failure to reach this target is the trigger for escalation to the protocol described here.
A curative transplant-based approach is the option addressed at this stage, and the full protocol specifies when it should be offered and to whom. Access the complete structured guidance below.
Once an infant with α-thalassaemia major (haemoglobin Bart’s hydrops foetalis) survives the neonatal period, they will become lifelong transfusion-dependent, unless they are cured by haematopoietic stem cell transplantation.
Allogeneic haematopoietic stem cell transplantation is the only curative option currently available for long term survivors of haemoglobin Bart’s hydrops foetalis.
Transplant for thalassaemia should be offered as early as possible as outcomes are superior prior to the onset of organ dysfunction secondary to iron overload.
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