Luspatercept
ESAs
Erythropoiesis-stimulating agents (ESAs)Pregnancy: contraindicated
It may harm the fetus: a pregnancy test is done before starting, contraception is used during treatment and for at least 3 months after, and breastfeeding is avoided for 3 months.
A ligand trap that lets red-cell precursors mature, reducing transfusion needs in beta thalassaemia and some myelodysplastic syndromes.
Also called: luspatercept-aamt
Quick facts
- Group
- Erythropoiesis-stimulating agents (ESAs)
- Target
- TGF-β superfamily ligands that brake red-cell maturation (GDF11, GDF8, activin B)
- Used for
- Anaemia in adults
- Key effects
- Bone · Headache · Flu-like symptoms
- Monitor
- Haemoglobin before each dose · Signs of thrombosis
- ATC
- B03XA06
No doses: the clinician sets them by the current guideline.
Mechanism of action
No structure with luspatercept is available; the figure shows human GDF11 held by the extracellular domain of activin receptor type 2B (from rat) and ALK5. Luspatercept is built from a modified human version of that receptor domain.
Luspatercept is a fusion protein: a modified extracellular domain of activin receptor type 2B joined to the Fc part of human IgG1. It traps GDF11, GDF8 and activin B, which reduces SMAD2/3 signalling in late erythroid precursors. Freed from that brake, the cells mature into red cells. It acts on a different step from erythropoietin.
- TGF-β family ligands brake late red-cell maturation
- Luspatercept traps these ligands in the blood
- SMAD2/3 signalling in erythroblasts falls
- More precursors mature; fewer transfusions are needed
Indications
- Anaemia in adults with transfusion-dependent beta thalassaemia Thalassemias (α and β) →
- Anaemia in lower-risk myelodysplastic syndromes needing transfusion, after or instead of erythropoiesis-stimulating agents Myelodysplastic syndromes →
Adverse effects
Common
- Bone, joint or muscle pain
- Headache, dizziness, tiredness
- Flu-like symptoms, cough
- Diarrhoea, nausea, abdominal pain
- Injection-site pain, redness or itching
Contraindications
- Hypersensitivity to luspatercept
- Caution: previous clots, splenectomy, hypertension, smoking, extramedullary haematopoietic masses
- Pregnancy (may harm the fetus)
Key interactions
- Oestrogen-containing hormone therapy or contraceptives
- Add to the clot risk; reviewed before starting
What is monitored
- Haemoglobin before each dose
- Blood pressure regularly
- Transfusion needs to judge response
- Signs of thrombosis
Administration and safety
- Subcutaneous injection by a health professional in a clinic
- The dose may be delayed, changed or stopped according to haemoglobin and side effects
- A missed appointment is rescheduled at once
Pregnancy and breastfeeding
It may harm the fetus: a pregnancy test is done before starting, contraception is used during treatment and for at least 3 months after, and breastfeeding is avoided for 3 months.
Nursing care
- Check haemoglobin and blood pressure before each injection
- Ask about leg swelling, chest pain or neurological symptoms
- Teach contraception during treatment and for at least 3 months after
- Ask about splenectomy and smoking
Pharmacokinetics
- Absorption
- Subcutaneous injection every few weeks
Test yourself
Think of the answer, then tap the question to see it.
How does luspatercept differ from epoetin?
It traps TGF-β family ligands to allow late red-cell maturation, instead of stimulating the erythropoietin receptor.
In which thalassaemia patients is thrombosis most likely?
Those who have had a splenectomy and have other risk factors.
Which two diseases is it used in?
Transfusion-dependent beta thalassaemia and lower-risk myelodysplastic syndromes.
Connections
← Back to the class: Erythropoiesis-stimulating agents (ESAs)
Educational content on how drugs work; not a prescribing guide and no substitute for a health professional's advice. No doses are given.
Sources: Luspatercept-aamt Injection: MedlinePlus Drug Information (MedlinePlus (U.S. National Library of Medicine), 2025); ATCDDD - ATC/DDD Index (WHO Collaborating Centre for Drug Statistics Methodology, 2026).
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Representative model, for educational purposes only.