How Do Erythroid Maturation Agents Work?

Erythroid maturation agents are used to treat anemia in patients with beta-thalassemia, myelodysplastic syndromes with ring sideroblasts (MDS-RS), or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T). Luspatercept is the only drug approved by the U.S. Food and Drug Administration in this drug class for the treatment of anemia in adults who need to have regular red blood cell transfusions.

Patients with beta-thalassemia have a defective beta-globin gene that leads to oxidative stress and premature apoptosis (programmed cell death) of erythroblasts (precursors of red blood cells [RBCs]), leading to ineffective erythropoiesis (formation of RBCs).

This drug prevents the activation of various tumor growth factor-beta superfamily members involved in causing ineffective erythropoiesis in the late stage and thus improves erythropoiesis.

However, luspatercept is not a substitute for RBC transfusions in patients who require immediate correction of anemia.

Erythroid maturation agents are given subcutaneously to treat anemia caused by the following:

• Beta-thalassemia
• Myelodysplastic syndromes (MDS-RS)
• Primary myelofibrosis

Side effects of erythroid maturation agents may include:

• Nausea
• Diarrhea
• Dizziness
• Headache
• Fatigue
• Cough
• Elevated liver enzymes
• Increased bilirubin
• Muscle and joint pains
• Abdominal pain
• Renal impairment
• Hyperuricemia (high blood uric acid)
• Hypertension (Increased blood pressure)
• Injection site reactions
• Upper respiratory tract infections
• Anaphylaxis (life-threatening allergic reactions)

The information contained herein is not intended to cover all possible side effects, precautions, warnings, drug interactions, allergic reactions, or adverse effects. Check with your doctor or pharmacist to make sure these drugs do not cause any harm when you take them along with other medicines. Never stop taking your medication and never change your dose or frequency without consulting your doctor.

Generic and brand names of erythroid maturation agents include:

• Luspatercept (Reblozyl)
• Luspatercept-aamt