How it is administered

Luspatercept is given as a subcutaneous injection (an injection under the skin). It comes as a sterile, preservative-free powder in single-dose vials, which is reconstituted with sterile water before use. The injection is typically administered by a healthcare professional every 3 weeks. The dose is based on your body weight and may be adjusted depending on your response and side effects. Common injection sites include the upper arm, thigh, or abdomen.

How it works

Luspatercept is an erythroid maturation agent, which means it helps red blood cells mature and develop properly. It is a recombinant fusion protein that binds to certain proteins in the body (TGF-β superfamily ligands), reducing abnormal signaling that can interfere with red blood cell production. By diminishing this signaling, luspatercept allows for improved maturation of red blood cell precursors in the bone marrow, leading to increased production of healthy red blood cells.

This mechanism is particularly helpful in conditions where the bone marrow does not produce enough mature red blood cells, such as myelodysplastic syndromes (MDS) and beta thalassemia. For patients with these blood disorders, luspatercept can reduce the need for blood transfusions and help manage anemia.

Who Should take it

Luspatercept is indicated for adults with:

• Anemia associated with beta thalassemia who require regular red blood cell transfusions.
• Anemia in adults with very low- to intermediate-risk myelodysplastic syndromes (MDS) who may require regular red blood cell transfusions and have not previously used erythropoiesis-stimulating agents (ESA-naïve).
• Anemia in adults with very low- to intermediate-risk MDS with ring sideroblasts or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis, who have failed an erythropoiesis-stimulating agent and require 2 or more red blood cell units over 8 weeks.

It is not indicated for use as a substitute for red blood cell transfusions in patients who need immediate correction of anemia.

Who should not take it

There are no absolute contraindications listed for luspatercept. However, it should not be used in patients who are pregnant, as it may cause harm to an unborn baby. Women of childbearing potential should use effective contraception during treatment and for at least 3 months after the last dose.

Luspatercept is not recommended for use in pediatric patients, as safety and effectiveness have not been established in this group. It should also be avoided in patients who require immediate correction of anemia, as it is not a substitute for urgent red blood cell transfusions.

Commonly used with

Luspatercept is often used alongside supportive care measures such as red blood cell transfusions, iron-chelating agents (to manage iron overload from transfusions), and other medications to manage symptoms or complications of blood disorders.

It is not typically combined with other disease-modifying agents for MDS or beta thalassemia, but your healthcare provider will determine the best treatment plan for your individual needs.

Commonly tested with

Luspatercept has been tested in clinical trials against placebo and active comparators such as epoetin alfa (an erythropoiesis-stimulating agent) in patients with myelodysplastic syndromes. It has also been studied in combination with best supportive care, which may include transfusions and iron chelation therapy.

Clinical trials have focused on its use in patients with MDS, beta thalassemia, and related blood disorders, often comparing it to standard care or supportive treatments.