Drug updated on 4/18/2024
| Dosage Form | Injection (subcutaneous; 25 mg/vial, 75 mg/vial) |
| Drug Class | Erythroid maturation agents |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for the treatment of anemia in adult patients with beta thalassemia who require regular red blood cell (RBC) transfusions.
- Indicated for the treatment of anemia without previous erythropoiesis stimulating agent use (ESA-naïve) in adult patients with very low- to intermediate-risk myelodysplastic syndromes (MDS) who may require regular red blood cell (RBC) transfusions.
- Indicated for the treatment of anemia failing an erythropoiesis stimulating agent and requiring 2 or more RBC units over 8 weeks in adult patients with very low- to intermediate-risk myelodysplastic syndromes with ring sideroblasts (MDS-RS) or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T).
Summary
- Luspatercept-aamt (Reblozyl) is indicated for the treatment of anemia in various adult patient groups, including those with beta thalassemia requiring regular red blood cell transfusions and patients with very low to intermediate-risk myelodysplastic syndromes. It functions as an erythroid maturation agent that inhibits the Smad2/3 cascade, downregulating the transforming growth factor-beta pathway, which plays a crucial role in beta-thalassemia.
- A single systematic review/meta-analysis covered literature from 2015 to 2022 sourced from PubMed, Google Scholar, and the Cochrane database, resulting in five selected studies involving a total of 1161 participants across phase 2 and phase 3 trials.
- Regarding the population distribution within these trials, there was almost equal representation between transfusion-dependent beta-thalassemia (TD) and non-transfusion-dependent beta-thalassemia (NTD) patients during Phase II. During Phase III trials, two-thirds received Reblozyl while one-third were given a placebo, providing substantial data on its effectiveness and safety.
- Compared to placebos used in clinical trials, luspatercept-aamt showed superior results by improving hemoglobin levels and significantly reducing the frequency of required blood transfusions among beta-thalassemia sufferers, thereby potentially enhancing their quality of life due to lessening the burden of frequent treatments needed to manage the disease symptoms effectively.
- Despite reported side effects such as hyperuricemia, arthralgia, dizziness, influenza, hypertension, and bone pain, these were deemed manageable within the context of trial settings, suggesting a positive benefit-risk balance, making it a viable treatment option for this specific patient group.
- Luspatercept provides a targeted approach compared to other traditional anemia treatments, like erythropoiesis-stimulating agents, offering a therapeutic advantage for beta-thalassemia patients by rectifying underlying dysregulated erythropoiesis. This reflects its innovative mechanism of action and potential in improving specific clinical outcomes within this subgroup.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Reblozyl (luspatercept–aamt) Prescribing Information | 2023 | Celgene Corporation., Summit, NJ |
Systematic Reviews / Meta-Analyses
| Document Title | Year | Source |
|---|---|---|
| Efficacy and safety of luspatercept in the treatment of β-thalassemia: a systematic review. | 2022 | Cureus |