A Promising Option for Treating Anemia in Low-Risk MDS
- Luspatercept (brand name Reblozyl) is now FDA approved as the first treatment for adults with Lower-Risk Myelodysplastic Syndromes (MDS) who may require transfusions
- MDS occurs when there is a dysfunction in the bone marrow, which leads to the production of defective blood cells.
- Many patients who have MDS will have to deal with some degree of anemia, which is when there isn’t enough healthy red blood cells.
- A drug called luspatercept (brand name: Reblozyl) showed promise in reducing how often patients with low-risk MDS needed blood transfusions.
- Luspatercept should be considered a paradigm shift in the treatment of anemia for patients with low-risk MDS, says lead author Dr. Guillermo Garcia-Manero.
- Luspatercept is currently approved in the United States for MDS patients with ring sideroblastic anemia with low-risk features after failure of standard erythropoiesis-stimulating agent treatment, or who were ineligible to receive ESAs.
The drug luspatercept (sold under the brand name Reblozyl) would, ideally, be given as an initial treatment to manage anemia in patients with low-risk MDS and now is approved by the FDA for this purpose.
Read MoreThe data is being presented at the 2023 ASCO Annual Meeting in Chicago.
Dr. Jun Choi, a Hematology & Oncology Specialist ad Clinical Assistant Professor at the Department of Medicine at NYU Grossman School of Medicine, told SurvivorNet that this data is “great news” because there has been a “lack of effective therapy in most low-risk MDS patients other than ESA.”
Because luspatercept had only been approved “so far for a limited number of MDS patients,” like those with ring sideroblasts (immature red blood cells) or the SF3B1 mutation, only a small number of MDS patients benefited from the drug. The new approval could help open the door for a larger number of patients to benefit.
Expert MDS Resources
- What is Myelodysplastic Syndrome (MDS)?
- Managing the Emotional Toll of Living With Myelodysplastic Syndrome (MDS)
- Living With Myelodysplastic Syndrome (MDS): Coping With Anemia
- Making Treatment Decisions for Myelodysplastic Syndrome (MDS)
- How Reblozyl Is Used to Treat A Specific Type Of MDS
- How Does A Bone Marrow Transplant Treat MDS?
What Is MDS?
MDS, short for myelodysplastic syndrome, is a condition characterized by an abnormality in the bone marrow, leading to the production of faulty blood cells and a decrease in overall blood cell production. This disorder affects all types of blood cells and is ultimately classified as a form of bone marrow failure.
As a result of the impaired ability of the bone marrow to generate an adequate number of healthy and functional blood cells, individuals with MDS may experience the following symptoms:
- Frequent infections
- Fatigue
- Shortness of breath
- Easy bleeding or bruising
If a physician suspects MDS based on low blood cell counts, they will likely recommend a bone marrow biopsy to confirm the diagnosis and gather additional information about the disease.
Anemia and MDS
Anemia is a major burden for patients with MDS. Chronic anemia and transfusion dependency are significant clinical challenges in low-risk MDS and can increase the risk of complications and death.
Many patients who have myelodysplastic syndrome will have to deal with some degree of anemia. Anemia occurs when a person does not have enough healthy red blood cells.
WATCH: What Is MDS?
How much a patient's life is affected by anemia can vary a lot from patient to patient. Some patients are anemic, but they're not so anemic that they need to be checked more than, say, once every three months because their anemia is relatively stable.
However, there are other patients who are so anemic that they need blood transfusions once a week, and that can be very disruptive to a person's life.
Anemia can cause symptoms like:
- Fatigue/Tiredness
- Dizziness
- Shortness of breath.
- Weakness
Blood Transfusion as a Treatment Option for Anemia in MDS
To treat anemia, patients with MDS are sometimes given blood transfusions. During a blood transfusion, healthy donor blood is infused into the MDS patient via an IV. The process takes about two to three hours and the vast majority of patients feel much better after a blood transfusion.
However, too many blood transfusions can cause unwanted side effects. If you get many transfusions over a long period of time, the iron in those blood transfusions can be deposited in your organs, potentially causing injury to some organs such as the liver, heart, and pancreas.
That is why researchers are looking into other options for treating MDS that will reduce the number of transfusions they might need.
What Is Luspatercept?
Luspatercept (brand name Reblozyl) is a medication prescribed for the treatment of anemia in adults with specific types of MDS or myelodysplastic/myeloproliferative neoplasms.
These individuals require regular transfusions of red blood cells and have either not responded to or cannot be treated with an erythropoiesis-stimulating agent. Additionally, luspatercept is utilized for managing anemia in adults with beta thalassemia, an inherited blood disorder, who also rely on regular red blood cell transfusions.
Luspatercept is not chemotherapy. It is a biotherapy. This medication aids in increasing the production of red blood cells in the bone marrow and belongs to the class of antianemics and recombinant fusion proteins.
Patients will require fewer trips to the doctor for injections because luspatercept is given once every 3 weeks. ESAs are given weekly.
Side effects may include:
- Allergic reaction
- Nausea
- Diarrhea
- Fatigue
Which MDS patients will benefit the most?
“Certainly luspatercept still has the highest efficacy in MDS patients with ringed sideroblasts (an immature red blood cell) or SF3B1 mutation. But with these new data, patients who are refractory to ESA or has EPO level greater than 500 will benefit the most since they do not have any other good option to treat their severe anemia once they become transfusion dependent,” Dr. Choi said.
A New Treatment Option?
The results of this study revealed a notable increase in the likelihood of transfusion independence among individuals treated with luspatercept compared to those treated with epoetin alfa (the current standard of care treatment).
This finding holds significant importance since erythropoiesis-stimulating agents (ESA – such as epoetin alfa) have traditionally served as the primary treatment option for patients with lower-risk MDS for many years now.
The new published study demonstrated a nearly twofold increase in the number of individuals achieving transfusion independence (meaning they did not need blood transfusions) for a duration of 12 weeks or longer, compared to the current standard of care treatment.
“Luspatercept is the first and only therapy at this point to demonstrate superiority on a head-to-head study against an ESA in transfusion-dependent low-risk MDS, and it should be considered a paradigm shift in the treatment of this group of patients with anemia,“ adds Dr. Garcia-Manero.
The introduction of luspatercept has the potential to reshape this treatment landscape, allowing patients to receive luspatercept as an initial therapy instead of erythropoiesis-stimulating agents.
As a result, patients would experience reduced clinic visits and fewer blood transfusions, ultimately leading to an enhanced quality of life and improved outcomes.
Questions to Ask Your Doctor
If you have MDS and anemia, here are some questions you can consider asking your doctor to help understand your options:
- What risk level is my MDS? Is it low-risk?
- Are there tests available to assess my risk level?
- What treatment options can help reduce my need for blood transfusions?
- Am I eligible for Rebolzyl?
- How does that medication differ from what I am currently receiving?
- Are their side effects associated with Reblozyl?
- Are there ways to manage those side effects?
WATCH: Determining How and When to Treat MDS
About the Study
The global phase III COMMANDS clinical trial was an open-label, randomized phase III trial which included 354 people from more than 20 countries who had lower-risk MDS and required red blood cell transfusions to treat anemia.
The participants in this study had not previously received treatment with an ESA. The participants were divided into two groups:
- 178 received luspatercept by injection once every 3 weeks for at least 24 weeks
- 176 received epoetin alfa (an ESA) by injection once a week for at least 24 weeks
Participants in this study were adults aged 18 years and older. The median age of the participants was 74 years, and most were white (80%) and men (56%).
When compared with epoetin alfa, luspatercept nearly doubled the number of patients who no longer needed blood transfusions.
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