Luspatercept

Pronunciation: REB-low-zil
Generic name: luspatercept
Brand name: Reblozyl
Dosage form: subcutaneous injection (25 mg/vial, 75 mg/vial)
Drug class: Miscellaneous erythropoiesis agents

Medically reviewed by Carmen Pope, BPharm. Last updated on Nov 7, 2024.

What is luspatercept?

Luspatercept (brand name Reblozyl) is used to treat anemia (low red blood cells) in certain patients with beta-thalassemia or myelodysplastic syndromes (MDS).

• Luspatercept should not be used instead of blood transfusions in patients with severe anemia who need immediate treatment.
• The full biological name of luspatercept is luspatercept-aamt.

Luspatercept's mechanism of action involves specifically targeting and blocking molecules that prevent red blood cells (RBCs) from maturing, allowing them to mature properly. Luspatercept helps overcome ineffective erythropoiesis (the development of RBCs) seen in conditions such as beta-thalassemia and myelodysplastic syndromes, increasing the number of RBCs in the plasma and reducing the need for blood transfusions. Luspatercept belongs to the drug class called erythroid maturation agents.

Luspatercept gained FDA approval on November 8, 2019 under the brand name Reblozyl. There is no luspatercept generic or biosimilar.

Luspatercept Uses

Luspatercept is used to treat anemia in adults with:

• beta-thalassemia who need regular red blood cell (RBC) transfusions
• myelodysplastic syndromes who may need regular blood transfusion but have never received an erythropoiesis-stimulating agent (ESA)
• myelodysplastic syndromes with ring sideroblasts (MDS-RS) or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T), who need 2 or more RBC units over 8 weeks and have not responded well to an ESA.

Luspatercept is not used as a substitute for RBC transfusions in people needing immediate anemia treatment.

It is not known if luspatercept is safe and effective in children. 

Luspatercept side effects

The most common side effects of luspatercept are:

• stomach pain, diarrhea; nausea;
• headache, dizziness;
• feeling tired;
• cough; trouble breathing;
• muscle pain, bone pain, joint pain: or
• allergic reactions.

Serious side effects and warnings

Luspatercept may cause the following serious side effects.

Blood clots. Blood clots in the arteries, veins, brain, and lungs have happened in people with beta-thalassemia during treatment with luspatercept. The risk of blood clots may be higher in people who have had their spleen removed or who take hormone replacement therapy or birth control (oral contraceptives). Call your healthcare provider or get medical help right away if you get any of these symptoms:

• chest pain
• trouble breathing or shortness of breath
• pain in your leg, with or without swelling
• a cold or pale arm or leg
• sudden numbness or weakness that are both short-term or continue to happen over a long period,
  especially on one side of the body
• severe headache or confusion
• sudden problems with vision, speech, or balance (such as trouble speaking, difficulty walking, or dizziness).

High blood pressure.   Luspatercept may cause an increase in your blood pressure. Your healthcare provider will
check your blood pressure before you receive your dose. Your healthcare provider may prescribe you
medicine to treat high blood pressure or increase the dose of medicine you already take to treat high blood pressure if you develop high blood pressure during treatment with luspatercept.

Extramedullary Hematopoietic (EMH) Masses. EMH masses have happened in people with beta-thalassemia during treatment with luspatercept. You may have a higher risk of developing EMH masses if you have a history of EMH masses, have had your spleen removed, have or have had an enlarged spleen or liver, or have low hemoglobin levels. Your healthcare provider will monitor you before you start and during treatment with luspatercept. Call your healthcare provider or get medical help right away if you get any of these symptoms:

• severe pain in the back
• numbness, weakness, or loss of voluntary movement in feet, legs, hands, or arms
• loss of bowel and bladder control.

Get emergency medical help if you have signs of an allergic reaction to luspatercept, such as hives, difficulty breathing, or swelling of your face, lips, tongue, or throat.

Luspatercept may harm an unborn baby or cause fertility problems in females which may affect your ability to become pregnant. Talk to your healthcare provider if this is a concern for you. See below for more information about pregnancy and breastfeeding.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.

Related/similar drugs

Before receiving

Before receiving luspatercept, tell your healthcare provider about all of your medical conditions, including if you:

• have or have had blood clots
• take hormone replacement therapy or birth control pills (oral contraceptives)
• have had your spleen removed (splenectomy)
• smoke
• have or have had high blood pressure (hypertension)
• have a history of extramedullary hematopoietic (EMH) masses
• have or have had enlarged spleen or liver
• are pregnant or plan to become pregnant
• are breastfeeding or plan to breastfeed.

Pregnancy

Luspatercept may harm your unborn baby. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with luspatercept. Females who can become pregnant:

• Your healthcare provider should do a pregnancy test before you start treatment with luspatercept.
• You should use effective birth control (contraception) during treatment with luspatercept and for at least 3 months after the last dose.

Luspatercept may cause fertility problems in females. This could affect your ability to become pregnant. Talk to your healthcare provider if this is a concern for you.

Breastfeeding

It is not known if luspatercept passes into your breast milk. Do not breastfeed during treatment with luspatercept and for 3 months after the last dose. Talk to your healthcare provider about the best way to feed your baby during this time.

How is luspatercept administered?

Luspatercept is given by a healthcare provider as an injection under the skin (subcutaneously [SC]).

• Luspatercept injection may be administered into the upper arm, thigh, or stomach (abdomen).
• Luspatercept injection is usually given 1 time every 3 weeks.

Before each injection, you will need a blood test to check your hemoglobin levels to monitor if your anemia is getting better before each injection and during your treatment with luspatercept.

• Be sure to tell your healthcare provider when you last had a blood transfusion.

Your blood pressure will need to be checked often.

• You may be given other medications to help control your blood pressure. Keep using this medicine for as long as your doctor has prescribed.

Luspatercept dosing

Luspatercept doses are based on weight. Your healthcare provider will prescribe luspatercept at a dose that is right for you. Your dose needs may change if you gain or lose weight, and also depends on how you respond to treatment.

Usual adult luspatercept dose for anemia in beta-thalassemia:

Initial starting dose: luspatercept 1 mg/kg SC once every 3 weeks

• If there is no reduction in RBC transfusion burden after 2 doses (6 weeks), increase to 1.25 mg/kg SC once every 3 weeks.
• If there is no reduction in RBC transfusion burden after 3 consecutive doses (9 weeks) at 1.25 mg/kg SC once every 3 weeks, then treatment should be discontinued.
• Maximum dose: 1.25 mg/kg.

See the luspatercept Prescribing information for more detailed information about luspatercept dosing, including dose modification according to hemoglobin levels.

Usual adult luspatercept dosage for myelodysplastic syndromes associated anemia

Initial starting dose: luspatercept 1 mg/kg SC once every 3 weeks

• If there is no reduction in RBC transfusion burden after 2 doses (6 weeks), increase to 1.33 mg/kg SC once every 3 weeks.
• If there is no reduction in RBC transfusion burden after 2 consecutive doses (6 weeks) at 1.33 mg/kg SC once every 3 weeks, then increase the dose to 1.75 mg/kg every 3 weeks.
• If there is no reduction in RBC transfusion burden including no increase from baseline hemoglobin after at least 3 consecutive doses (9 weeks) at 1.75 mg/kg then treatment should be discontinued.
• Maximum dose: 1.75 mg/kg.

See the Reblozyl (luspatercept) Prescribing information for more detailed information about luspatercept dosing, including dose modification according to hemoglobin levels.

What happens if I miss a dose?

If you miss your luspatercept injection, call your healthcare provider right away to reschedule your appointment. You should receive an injection as soon as you can, and then go back to your regular schedule. At least 3 weeks should pass between each injection.

What happens if I overdose?

Since luspatercept is given by a healthcare professional in a medical setting, an overdose is unlikely to occur.

Luspatercept Package Insert

Review the Reblozyl (luspatercept) Package Insert for more detailed information about this medicine. Discuss any medical questions you have with your doctor or other health care provider. This is not all the information you need to know about this medicine for safe and effective use, and it does not take the place of talking to your doctor about your treatment.

What other drugs will affect luspatercept?

Other drugs may affect luspatercept, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell your doctor about all your current medicines and any medicine you start or stop using.

Not all possible drug interactions are listed here. To check for interactions, click on the link below.

Luspatercept ingredients

Luspatercept is only available under the brand name Reblozyl.

Active ingredient: luspatercept-aamt

Inactive ingredients: citric acid monohydrate, polysorbate 80, sucrose, and tri-sodium citrate dihydrate.

Luspatercept is available as:

• luspatercept 25 mg lyophilized powder in a single-dose vial for reconstitution
• luspatercept  75 mg lyophilized powder in a single-dose vial for reconstitution

Luspatercept J Code is J0896

Storage

Your healthcare provider will store luspatercept vials refrigerated at 2°C to 8°C (36°F to 46°F) in the original cartons to protect them from light. Do not freeze.

Storage of reconstituted solution is not used immediately:

• Store at room temperature at 20°C to 25°C (68°F to 77°F) in the original vial for up to 8 hours. Discard if not used within 8 hours of reconstitution.
• Alternatively, store refrigerated at 2°C to 8°C (36°F to 46°F) for up to 24 hours in the original vial. Remove from refrigerated condition 15-30 minutes prior to injection to allow solution to reach room temperature for a more comfortable injection. Discard if not used within 24 hours of reconstitution.
• Do not freeze the reconstituted solution.

Discard any unused portion. Do not pool unused portions from the vials. Do not administer more than 1 dose from a vial. Do not mix with other medications.

Who makes luspatercept?

Celgene Corporation, a Bristol-Myers Squibb Company, manufactures luspatercept under the brand name Reblozyl.

Luspatercept Biosimilars

Biosimilar and interchangeable products are biological products that are highly similar to and have no clinically meaningful differences from the reference product.

Reference products

These are biological products that have already been approved by the FDA, against which biosimilar products are compared. There is 1 for luspatercept.

Further information

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.

Medical Disclaimer