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luspatercept

TGFβ Inhibitor · Hematopoietic Agent

TGFβ InhibitorHematopoietic Agent
CDSCO approved
EXCRETION
not curated
INTERACTIONS
none in our sources
PREGNANCY
not curated

Mechanism

Luspatercept is a recombinant fusion protein consisting of a human IgG1 Fc domain linked with a modified extracellular domain of the activin receptor 2B (ActRIIB). It binds and traps select TGFβ family ligands (GDF8, GDF11, and activin B), decreasing SMAD2 and SMAD3 signaling, which enables erythroblast differentiation and erythroid maturation.

Indications

beta thalassemia (adults requiring regular red cell transfusions)myelodysplastic syndromes (adults with very low- to intermediate-risk MDS with ring sideroblasts or myelodysplastic/myeloproliferative neoplasms with ring sideroblasts and thrombocytosis, with anemia despite ESA and requiring 2 or more red blood units over an 8-week period)

Dosing

Adult
Beta Thalassemia: Starting dose is 1 mg/kg once every 3 weeks by subcutaneous injection. Delay dosing for hemoglobin levels of 11.5 g/dL or greater (if not influenced by recent transfusion). If no reduction in red blood cell transfusion burden after at least 6 weeks, increase dose to 1.25 mg/kg (maximum dose).…

Side effects

Common
transient bone painarthralgiasdizzinesshypertensionhyperuricemiamild fatiguediarrheaasthenianauseaback pain
Serious
  • thromboembolic events

Related guidelines

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Sources: Goodman & Gilman 14e, Harrison 22e·Verified: 2026-05-13 · House clinical team