How it is administered

Luspatercept is given as a subcutaneous injection (an injection under the skin). It comes as a sterile, preservative-free, lyophilized (freeze-dried) powder that is reconstituted with sterile water before use. The dose is based on your body weight and is usually given once every 3 weeks. The injection can be administered in the upper arm, thigh, or abdomen by a healthcare professional.

How it works

Luspatercept is an erythroid maturation agent. It works by binding to certain proteins in the body (TGF-β superfamily ligands), which decreases abnormal signaling that can interfere with the production and maturation of red blood cells. By reducing this abnormal signaling, luspatercept helps promote the development and maturation of red blood cells in the bone marrow.

For patients with myelodysplastic syndromes (MDS), luspatercept helps improve anemia by increasing the number of mature red blood cells, which can reduce the need for blood transfusions. It is especially useful in patients who have not responded well to other treatments, such as erythropoiesis-stimulating agents (ESAs).

Who Should take it

Luspatercept is indicated for adults with certain types of anemia associated with blood disorders, including:

• 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).
• Adults with very low- to intermediate-risk MDS with ring sideroblasts (MDS-RS) or myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T) who have failed an ESA and require 2 or more red blood cell units over 8 weeks.

Luspatercept is also used in adults with beta thalassemia who require regular red blood cell transfusions.

It is not meant to replace urgent 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 reproductive potential should use effective contraception during treatment and for at least 3 months after the last dose.

Luspatercept is not recommended for use in children, as safety and effectiveness have not been established in pediatric patients. 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 as part of supportive care in patients with MDS or beta thalassemia. Patients may also receive red blood cell transfusions, iron-chelating agents (to manage iron overload from transfusions), and other supportive treatments as needed.

It is not typically combined with other disease-modifying agents for MDS, but may be used alongside medications to manage symptoms or complications of the underlying disease.

Commonly tested with

Luspatercept has been tested in clinical trials alongside best supportive care, which may include red blood cell transfusions and iron-chelating agents. In studies for MDS, it has also been compared to erythropoiesis-stimulating agents (such as epoetin alfa) to evaluate its effectiveness in reducing transfusion needs and improving hemoglobin levels.