Drug reference
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
Blood transfusion in obstetrics and gynaecology
FOGSI · Obstetrics & Gynaecology · 2024
Empirical antimicrobial use in common syndromes
ICMR · Infectious Diseases · 2019
Sickle cell disease
ASH · Haematology · 2020
Hypertension in adults
ICMR · Cardiology · 2019
Venous thromboembolism prophylaxis
ASH · Haematology · 2018
Ask House about luspatercept
Continue into a citation-backed clinical answer with the drug context already attached.
Sources: Goodman & Gilman 14e, Harrison 22e·Verified: 2026-05-13 · House clinical team