What side effects are associated with this type of intervention?

Common treatments for Myelodysplastic Syndrome (MDS) that enhance erythroid maturation or stimulate red blood cell production include Luspatercept and Epoetin Alfa. Luspatercept, which enhances erythroid maturation, may cause side effects such as fatigue, diarrhea, dizziness, and increased risk of thromboembolic events. Epoetin Alfa, an erythropoiesis-stimulating agent, can lead to hypertension, headache, joint pain, and an increased risk of thromboembolic events. Both treatments aim to reduce the need for blood transfusions but require careful monitoring for these potential adverse effects.

What prior FDA approvals exist?

For the treatment of Myelodysplastic Syndrome (MDS), the FDA has approved several drugs that enhance erythroid maturation or stimulate red blood cell production. Epoetin Alfa, a synthetic form of erythropoietin, stimulates red blood cell production and has been used to treat anemia in MDS patients. Luspatercept, which enhances erythroid maturation, has been approved for patients with MDS who have ring sideroblasts and are refractory to erythropoiesis-stimulating agents (ESAs). Other related treatments include lenalidomide, hypomethylating agents like azacitidine and decitabine, and immunosuppressive therapies, which provide hematologic responses in selected subsets of MDS patients.

What other types of research exist?

Promising novel alternatives for MDS treatment in 2024 include imetelstat, an investigational antisense oligonucleotide directed against telomerase, which has shown partial and complete responses in advanced myelofibrosis and may have potential in MDS. Another alternative is venetoclax, a BCL2 inhibitor, which has shown efficacy in combination with low-dose cytarabine for AML and may be explored for MDS. These agents offer different mechanisms of action compared to Luspatercept and Epoetin Alfa.

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Cancer Treatment

Epoetin Alfa vs Luspatercept for Myelodysplastic Syndrome (ELEMENT-MDS Trial)

Phase 3

Recruiting

Research Sponsored by Bristol-Myers Squibb

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to week 96

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial is testing two treatments, Luspatercept and epoetin alfa, to see which is better for treating anemia in adults with certain types of myelodysplastic syndromes (MDS). The participants have not used similar treatments before and do not need regular blood transfusions. Luspatercept helps red blood cells mature, while epoetin alfa increases their production.

Who is the study for?

This trial is for adults with Myelodysplastic Syndrome (MDS) who have anemia but don't need blood transfusions. They should not have had previous treatments with erythropoiesis-stimulating agents, and their MDS should be classified as very low to intermediate-risk. Participants must also experience moderate symptoms of fatigue or other related issues due to anemia.

What is being tested?

The study compares Luspatercept against Epoetin Alfa in treating anemia caused by MDS in participants who haven't used similar drugs before. It aims to see which drug is more effective and safer for patients who aren't dependent on blood transfusions.

What are the potential side effects?

Possible side effects of Luspatercept may include tiredness, muscle aches, dizziness, high blood pressure, and bone pain. Epoetin Alfa could cause joint pain, injection site reactions, headache, or increased risk of blood clots.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to week 96

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to week 96

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to week 96 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Number of participants with lower-risk non-transfusion dependent myelodysplastic syndromes (NTD-MDS) who converted to Transfusion Dependence (TD) during any continuous 16-week interval within the 96-week treatment period

Secondary study objectives

Number of participants with a neutrophil response at Week 24, Week 48 and Week 96

Number of participants with a platelet response at Week 24, Week 48 and Week 96

Side effects data

From 2021 Phase 3 trial • 336 Patients • NCT02604433

42%

Upper respiratory tract infection

35%

Headache

32%

Back pain

23%

Arthralgia

22%

Bone pain

22%

Cough

21%

Pyrexia

18%

Oropharyngeal pain

18%

Diarrhoea

17%

Fatigue

16%

Pharyngitis

15%

Pain in extremity

13%

Abdominal pain

13%

Nausea

13%

Vomiting

13%

Myalgia

13%

Dizziness

12%

Asthenia

11%

Abdominal pain upper

11%

Influenza

10%

Hypertension

Dyspepsia

Influenza like illness

Musculoskeletal pain

Nasal congestion

Nasopharyngitis

Gastroenteritis

Urticaria

Toothache

Tonsillitis

Hyperuricaemia

Pain

Urinary tract infection

Viral upper respiratory tract infection

Neck pain

Osteoporosis

Constipation

Injection site pain

Transfusion reaction

Alanine aminotransferase increased

Spinal pain

Lethargy

Menstruation irregular

Liver iron concentration increased

Fall

Musculoskeletal chest pain

Anaemia

Septic shock

Extramedullary haemopoiesis

Deep vein thrombosis

Transient ischaemic attack

Cerebrovascular accident

Cholangitis

Cholecystitis acute

Cellulitis

Pneumonia

100%

80%

60%

40%

20%

Study treatment Arm

Luspatercept + BSC

Placebo + BSC

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Active Control

Group I: LuspaterceptExperimental Treatment1 Intervention

Group II: Epoetin AlfaActive Control1 Intervention

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Luspatercept

2018

Completed Phase 3

~1240

Do I qualify?Apply below to find out

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Luspatercept enhances erythroid maturation, aiding in the development and maturation of red blood cells, while Epoetin Alfa stimulates red blood cell production by mimicking erythropoietin. These treatments are vital for Myelodysplastic Syndrome patients as they address the hematologic deficiencies of the syndrome, improving symptoms, reducing transfusion needs, and enhancing quality of life.

Lenalidomide: Myelodysplastic syndromes with del(5q) and beyond.