Synopsis
Deliver phenotype-matched regular transfusion, prevent ineffective-erythropoiesis complications, quantify tissue iron and tailor chelation while protecting endocrine, hepatic, cardiac and reproductive health.
- Transfusion-dependent beta-thalassaemia requires regular red cells to sustain growth, activity and organ oxygenation and suppress harmful marrow expansion and extramedullary haematopoiesis.
- Before the first elective transfusion, record an extended red-cell phenotype or genotype and antibody screen; use leucocyte-depleted, appropriately antigen-matched units through the specialist programme.
- Pre-transfusion haemoglobin targets and intervals are individualised, commonly maintaining about 90–105 g/L, with higher targets for selected cardiac or extramedullary indications.
- Each red-cell unit delivers iron that the body cannot actively excrete, so chronic transfusion inevitably requires a measured iron-removal strategy unless definitive cure intervenes.
- Ferritin is useful as a trend but changes with infection, inflammation, liver injury and ascorbate; liver iron concentration by validated MRI better quantifies storage burden.
- Cardiac T2-star MRI identifies myocardial iron before symptoms and guides chelator intensity; normal liver iron does not guarantee a normal heart in every patient.
- Chelator selection considers liver and cardiac iron, transfusion rate, renal and liver function, age, pregnancy, adverse effects, lifestyle and likely adherence.
- Deferasirox is oral and requires renal, hepatic and gastrointestinal monitoring; deferiprone penetrates cardiac iron well but requires strict neutrophil surveillance.
- Desferrioxamine is administered subcutaneously or intravenously and remains important for severe cardiac loading, but infusion burden, auditory and ocular toxicity affect use.
- Long-term care includes annual or risk-based endocrine, bone, liver, cardiac, viral, fertility and psychosocial assessment, plus early discussion of transplantation or advanced therapies where appropriate.
Key red flags
Fever or painful throat while taking deferiprone requires stopping the drug and obtaining an urgent neutrophil count.
Investigation priorities
Guide dose and detect new compatibility risk.
Management branches
A patient meets specialist criteria for transfusion-dependent thalassaemia care.
- Record genotype, extended red-cell antigens, antibody and reaction history and obtain baseline viral, iron and organ assessments before repeated exposure.
- Give leucocyte-depleted, appropriately antigen-matched red cells, commonly 10–15 mL/kg every 2–4 weeks, to maintain a pre-transfusion haemoglobin of about 90–105 g/L; use a higher specialist target only for a defined cardiac or extramedullary indication.
- Investigate increasing requirements through bleeding, hypersplenism, alloantibody, haemolysis, unit characteristics and adherence before altering the long-term target.
Key medicines
Leucocyte-depleted red cellsA common maintenance prescription is 10–15 mL/kg every 2–4 weeks, adjusted to maintain pre-transfusion haemoglobin around 90–105 g/L and suppress ineffective erythropoiesis. Use appropriately antigen-matched units and calculate actual annual volume per kilogram.Check the complete antibody and reaction history before every exposure. Alloimmunisation, delayed haemolysis, volume overload, infection and iron input require observation, haemovigilance and long-term review.
DeferasiroxFor film-coated tablets, a common starting dose with frequent transfusion is 14 mg/kg orally once daily; 7 mg/kg may suit a lower iron input and 21 mg/kg a high input needing reduction. Adjust by 3.5–7 mg/kg every 3–6 months to ferritin and MRI response, up to 28 mg/kg/day. Dispersible-tablet doses are about 30% higher and are not interchangeable.Confirm duplicate baseline creatinine and avoid treatment when creatinine clearance is below the licensed threshold. Monitor eGFR, urine protein, liver tests, full count and gastrointestinal bleeding; interrupt for significant dehydration or organ injury and review pregnancy, interacting nephrotoxins and over-chelation risk.