Luspatercept
Also known as: ACE-536, ACE‑536, Luspatercept, luspatercept-aamt, Reblozyl
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Summary
Luspatercept is a recombinant fusion protein administered subcutaneously to treat anemia in adult patients with beta‑thalassemia requiring transfusion and in lower‑risk myelodysplastic syndromes with ring sideroblasts, by enhancing erythroid maturation and reducing transfusion burden.
Mechanism of Action
Acts as a ligand trap for select TGF‑β superfamily members (e.g., GDF11, activin B), reducing SMAD2/3 signaling and promoting late‑stage erythroid maturation
Routes of Administration
Goals & Uses
- Treatment of anemia in lower-risk MDS (non-RS)Hematologic / InvestigationalModerate
- Reduction of transfusion burden in beta-thalassemiaHematologic / Anemia ManagementHigh
- Reduce transfusion burden in β‑thalassemiaHematologyHigh
- Improve anemia in lower‑risk MDS with ring sideroblastsHematologyHigh
- Treatment of anemia in MDS with ring sideroblastsHematologic / Anemia ManagementHigh
- Improvement of erythropoiesis in myelofibrosis-associated anemiaHematologic / InvestigationalModerate
Contraindications
- PregnancyPopulationModeratePotential fetal risk or insufficient safety data
- Hypersensitivity to luspatercept or any component of the formulationAllergyHigh
- Known hypersensitivity to luspatercept or excipientsAllergyHigh
Adverse Effects
- Bone painMusculoskeletalCommon
- HypertensionCardiovascularCommonHigh blood pressure
- HeadacheNeurologicCommonPain in the head or upper neck
- Thromboembolic eventsCardiovascularUncommon
- Musculoskeletal pain (bone pain, arthralgia, myalgia)MusculoskeletalCommon
- FatigueGeneralCommonLow energy or tiredness
- DizzinessNeurologicCommonFeeling faint, lightheaded, or unsteady
Drug Interactions
- Erythropoiesis-stimulating agents (ESAs)Moderate
- Antihypertensive agentsLow
- Erythropoiesis‑stimulating agents (ESAs)Moderate
Population Constraints
- Pediatric patients (<18 years)AgeRelative
- Pediatric patientsAgeRelative
- Patients with uncontrolled hypertensionCardiovascularAbsolute
- Patients with severe hepatic impairmentHepaticRelative
- Patients with severe renal impairmentOrgan ImpairmentRelative
- Splenectomized patientsSurgical HistoryRelative
Regulatory Status
- European UnionApprovedApproved: Transfusion‑dependent β‑thalassemia, Lower‑risk MDS with ring sideroblastsMarketing authorisation granted in 2021.
- United StatesApprovedApproved: Transfusion‑dependent β‑thalassemia, Lower‑risk myelodysplastic syndromes with ring sideroblastsPrescribed under brand name Reblozyl.
- United KingdomApprovedApproved: Anemia in adults with transfusion-dependent beta-thalassemia, Anemia in adults with MDS with ring sideroblastsMHRA approved; NICE technology appraisals have evaluated use in MDS and beta-thalassemia with conditional recommendations
FDA approved (2020 for transfusion‑dependent β‑thalassemia; 2023 for MDS with ring sideroblasts). EMA approval for same indications in 2021.
Evidence & Sources
No sources recorded yet.
Frequently Asked Questions
What is Luspatercept?
Luspatercept is a recombinant fusion protein administered subcutaneously to treat anemia in adult patients with beta‑thalassemia requiring transfusion and in lower‑risk myelodysplastic syndromes with ring sideroblasts, by enhancing erythroid maturation and reducing transfusion burden.
What is Luspatercept used for?
Luspatercept is educationally associated with: Treatment of anemia in lower-risk MDS (non-RS), Reduction of transfusion burden in beta-thalassemia, Reduce transfusion burden in β‑thalassemia, Improve anemia in lower‑risk MDS with ring sideroblasts, Treatment of anemia in MDS with ring sideroblasts, Improvement of erythropoiesis in myelofibrosis-associated anemia. Educational only — not medical advice.
How is Luspatercept administered?
Recorded routes of administration: Subcutaneous.
What are the potential side effects of Luspatercept?
Reported adverse effects include: Bone pain, Hypertension, Headache, Thromboembolic events, Musculoskeletal pain (bone pain, arthralgia, myalgia), Fatigue, Dizziness. This list is not exhaustive — consult a qualified clinician.
Who should avoid Luspatercept?
Recorded contraindications: Pregnancy, Hypersensitivity to luspatercept or any component of the formulation, Known hypersensitivity to luspatercept or excipients. Consult a qualified clinician before use.