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Erythroid Maturation Agent (TGF-β Ligand Trap) Pregnancy: Contraindicated — thrombotic risk and animal embryotoxicity data; effective contraception required during and for 3 months after

Luspatercept

Brand names: Reblozyl

Adult dose

Dose: 1 mg/kg SC every 3 weeks; titrate up to 1.33 mg/kg then 1.75 mg/kg if no response
Route: Subcutaneous injection
Frequency: Every 21 days
Max: 1.75 mg/kg per dose
MDS with ring sideroblasts (SF3B1 mutation or ≥15% ring sideroblasts); beta-thalassaemia requiring transfusions; minimum 24-week trial before assessing response

Paediatric dose

Dose: Not established N/A/kg
Route: N/A
Frequency: N/A
Max: N/A
Not licensed in paediatrics

Dose adjustments

Renal

No dose adjustment required; limited data in severe renal impairment

Hepatic

No dose adjustment required

Paediatric weight-based calculator

Not licensed in paediatrics

Clinical pearls

  • MEDALIST trial (Fenaux et al. NEJM 2020): luspatercept achieved transfusion independence in 38% of MDS patients with ring sideroblasts vs 13% placebo — first new treatment for lower-risk MDS in a decade
  • BELIEVE trial (Cappellini et al. NEJM 2020): significantly reduced transfusion burden in adults with beta-thalassaemia — median 33% reduction in transfusion units
  • MHRA 2020 approved; NICE TA663 for MDS-RS; mechanism: traps late-stage TGF-β superfamily ligands (GDF11, activin B) that impair late-stage erythroid differentiation — distinct from EPO stimulation
  • Thromboembolic events reported; risk highest in splenectomised patients with beta-thalassaemia — consider thromboprophylaxis in high-risk patients
  • Luspatercept vs EPO: luspatercept is indicated specifically for ring sideroblast MDS (SF3B1-mutated) where EPO has often failed — serum EPO level <500 IU/L associated with better response

Contraindications

  • Pregnancy (thrombotic risk and foetal effects)
  • Platelet count <75 × 10⁹/L at treatment initiation

Side effects

  • Hypertension
  • Fatigue
  • Diarrhoea
  • Thrombosis (arterial and venous)
  • Bone/muscle pain
  • Dizziness
  • Headache

Interactions

  • Erythropoiesis-stimulating agents — additive erythropoietic effect; combination not recommended
  • Antihypertensives — monitor blood pressure closely; luspatercept can raise BP significantly

Monitoring

  • Haemoglobin (before each dose)
  • Blood pressure (every visit)
  • Platelet count
  • Thrombotic events (DVT/PE/arterial)
  • Iron stores (ferritin, transferrin saturation)

Reference: BNFc; BNF 90; MEDALIST trial (Fenaux et al. NEJM 2020); BELIEVE trial (Cappellini et al. NEJM 2020); NICE TA663; MHRA SPC Reblozyl. Verify against your local formulary and the latest BNF before prescribing.

Related

Curated clinical cross-links plus same-class fallbacks.

Pathways