As you age, feeling tired or short of breath may be dismissed as a part of getting older, but it’s important to talk to a healthcare provider if you are experiencing these symptoms. The risk of certain conditions, myelodysplastic syndromes (my-el-odys-plastic sin-dromes or MDS), increases with age and can also present as fatigue or other signs that may be mistaken for natural aging.
What is MDS?
Myelodysplastic syndromes (MDS) is a group of closely related blood disorders in which the bone marrow does not produce enough healthy red blood cells, white blood cells and platelets.1 This can happen over time as you age, but also in response to certain mutation-causing exposures like chemicals or radiation.2 MDS worsens over time and can result in symptoms like anemia, caused by low red blood cell counts, vulnerability to infection due to low white blood cell counts, or risk of heavy bleeding or bruising due to low platelet counts.
Approximately 33 to 55 Americans are diagnosed with MDS every day, and since the average age of diagnosis is 77 years old, most people living with MDS attribute their symptoms to natural aging.1,3 “While that may be true for some patients, significant fatigue could be correlated to anemia or the evolution of MDS itself,” says Dr. Ashish Shah, a physician at Advanced Care Oncology and Hematology Associates (ACOHA) in New Jersey with over 15 years of experience treating hematological malignancies and MDS, specifically. As MDS progresses, there is also a risk that it may transform into acute myeloid leukemia (AML), an aggressive form of blood cancer that can cause a rapid decline in health.4 Thus, it is even more imperative to emphasize early diagnosis and close monitoring of these high-risk patients.
Because there is no MDS-specific diagnostic test, the path to a clear diagnosis may take a few weeks or even in some cases, months. Lack of universal testing and standardized screening may prevent timely diagnosis and delays in treatment.5
“It’s not a one-size-fits-all type of diagnosis,” says Dr. Shah. “And that’s a really important factor for patients to understand. Since there are peaks and valleys when dealing with this disease, patients and their doctors partner to find the treatment that works for them.”
The first step in diagnosis includes a number of tests, such as a complete blood count that looks at the number of red blood cells, white blood cells and platelets; a blood smear that looks at the makeup of blood under a microscope; genetic analysis and a bone marrow biopsy.5
Current Treatments
The only known cure for MDS is a type of bone marrow transplant called hematopoietic stem cell transplantation (HSCT).6 This procedure can be risky in older adults, and since MDS typically occurs in individuals over 50 years of age, most people living with this condition must rely on other therapies. With long-term survival rates between 30-40%, the process can also require many months of treatment and hospitalization.6
“For the majority of patients, there is a small period of time where we watch and wait, which is
effectively assessing for disease progression, monitoring for worsening side effects and then
considering treatment when those factors become more clinically relevant,” Dr. Shah explains.
Over time, a patient is monitored and if the disease progresses, red blood cell transfusions may be necessary to keep a healthy amount of red blood cells in their system. People who have anemia caused by MDS and require regular blood transfusions may experience side effects.7 For example, frequent blood transfusions can cause an overload of iron in the body, and that can lead to problems like arthritis and long-term damage to important organs like the liver and heart.7 “Transfusion dependence can cause complications that lead to hospitalization or even complications that can occur from the transfusion itself, such as transfusion reactions and infections,” says Dr. Shah.
Patients who require transfusions frequently spend approximately two hours in the clinic as often as every other week, for years to receive transfusions.4 These patients have to plan their vacations around transfusions — their social interactions, and their weekly routines — for the rest of their lives. And their health depends on how long the effect of those blood transfusions last. For this reason, an important goal of managing anemia due to MDS is to reduce the need for transfusions.
There are certain types of drug therapies called ESAs (erythropoiesis stimulating agents) that can increase the body’s natural production and release of blood cells, helping to reduce the frequency at which transfusions are needed.5 However, these therapies do not eliminate the need for transfusions completely, with many patients continuing to rely on them to combat their anemia.8
Why Reblozyl® (luspatercept-aamt)?
Fortunately, treatments are emerging to treat anemia due to MDS, including Reblozyl, which was approved in August 2023 by the FDA as a first-line treatment — meaning certain MDS patients with anemia can be prescribed this treatment as a first step, rather than ESAs.9 As a result of this new approval, people with anemia due to MDS now have a longer-lasting and more convenient first-line treatment option available to them.10
“With Reblozyl, people with MDS potentially have a chance to stop the need for frequent transfusions earlier in their disease journey,” says Dr. Shah.
Reblozyl is indicated for the treatment of anemia without previous ESA use (ESA-naïve) in adult patients with very low- to intermediate-risk MDS who may require regular red blood cell (RBC) transfusions. Reblozyl is not indicated for use as a substitute for transfusions in patients who require immediate correction of their anemia. The most common side effects for patients receiving Reblozyl for first-line, lower-risk MDS are diarrhea, fatigue, high blood pressure, swelling of the hands, legs and feet, nausea and trouble breathing. Please see below for additional Important Safety Information, prescribing information, and the medication guide.
Reblozyl was initially approved as an alternative second-line treatment for anemia in MDS with ring sideroblasts (MDS-RS) after patients tried an ESA. Now, with this new approval, certain people with anemia due to MDS can try Reblozyl, an under-the-skin injection, as a first step in their treatment journey.9
“Reblozyl is a treatment modality that we use regularly at my own practice. Until recently, we prescribed it to patients who didn’t respond to prior treatment, but it’s great that we can now consider this as a first-line option sooner for certain patients. The goal remains to help patients go from transfusion dependent to transfusion independent,” says Dr. Shah.
Reblozyl was approved after a phase 3, open-label, randomized clinical trial showed more than half of people receiving this treatment at diagnosis maintained red blood cell transfusion independence for 12 weeks and had a hemoglobin increase (>1.5g/dL) within the first 24 weeks. This was significantly better than the results for people in the study who received an ESA, with nearly twice as many people responding with Reblozyl compared to an ESA. The most common (>10%) adverse reactions were diarrhea, fatigue, hypertension, peripheral edema, nausea, dyspnea, and COVID-19.10
“The greatest satisfaction,” Dr. Shah says, “is when patients are no longer dependent on transfusions, when they’re not being chased by their blood counts. This has the potential to make a difference in people’s lives, which is the goal of treatment.”
Progress in Treating Anemia Due to MDS
Early diagnosis, treatment for MDS and monitoring one’s health are important. Now with treatment options like Reblozyl available earlier, people living with MDS may have a better chance to reduce the frequency of transfusion and improve their anemia.
For more information, visit www.reblozyl.com.
What is REBLOZYL® (luspatercept-aamt)?
REBLOZYL is a prescription medicine used to treat anemia (low red blood cells) in adults with:
- myelodysplastic syndromes who may need regular RBC transfusions and have never received an erythropoiesis-stimulating agent (ESA).
- myelodysplastic syndromes with ring sideroblasts (MDS-RS) or myelodysplastic/myeloproliferative neoplasms 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 erythropoiesis-stimulating agent (ESA).
REBLOZYL is not for use as a substitute for RBC transfusions in people who need immediate treatment for anemia. It is not known if REBLOZYL is safe or effective in children.
Before receiving REBLOZYL, 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)
- are pregnant or plan to become pregnant. REBLOZYL may harm your unborn baby. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with REBLOZYL.
For females who are able to become pregnant:
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- Your healthcare provider should do a pregnancy test before you start treatment with REBLOZYL.
- You should use effective birth control (contraception) during treatment with REBLOZYL and for at least 3 months after the last dose.
- are breastfeeding or plan to breastfeed. It is not known if REBLOZYL passes into your breast milk.
- Do not breastfeed during treatment with REBLOZYL and for 3 months after the last dose. Talk to your healthcare provider about the best way to feed your baby during this time.
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.
What are the possible side effects of REBLOZYL?
REBLOZYL may cause serious side effects, including:
- Blood clots. Blood clots in the arteries, veins, brain, and lungs have happened in people with beta thalassemia during treatment with REBLOZYL. 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 have 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 of time, 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. REBLOZYL may cause an increase in your blood pressure. Your healthcare provider will check your blood pressure before you receive your REBLOZYL 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 REBLOZYL
The most common side effects of REBLOZYL include:
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REBLOZYL 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.
These are not all of the possible side effects of REBLOZYL. Call your doctor for medical advice about side effects. You are encouraged to report side effects to the FDA at 1-800-FDA-1088 or http://www.fda.gov/medwatch.
Please see accompanying US Full Prescribing Information and Patient Information, for REBLOZYL.
References
- Zeidan AM, Shallis RM, Wang R, Davidoff A, Ma X. Epidemiology of myelodysplastic syndromes: Why characterizing the beast is a prerequisite to taming it. Blood Reviews. 2019;34:1-15. doi:10.1016/j.blre.2018.09.001.
- What Causes Myelodysplastic Syndromes? (n.d.). American Cancer Society. https://www.cancer.org/cancer/types/myelodysplastic-syndrome/causes-risks-prevention/what-causes.html.
- What is MDS? | MDS Foundation. (2018). MDS-Foundation.org. https://www.mds-foundation.org/what-is-mds/.
- Myelodysplastic Syndrome. (n.d.). Johns Hopkins Medicine. https://www.hopkinsmedicine.org/kimmel-cancer-center/cancers-we-treat/leukemia-program/myelodysplastic-syndrome.
- Platzbecker U, Kubasch AS, Homer-Bouthiette C, Prebet T. Current challenges and unmet medical needs in myelodysplastic syndromes. Leukemia. 2021;35(8):2182–2198. doi:10.1038/s41375-021-01265-7.
- Zeidan AM, Stahl M, Hu X, Wang R, Huntington SF, Podoltsev NA, Gore SD, Ma X, Davidoff AJ. Long-term survival of older patients with MDS treated with HMA therapy without subsequent stem cell transplantation. Blood. 2018;131(7):818–821. doi:10.1182/blood-2017-10-811729.
- Rasel M, Mahboobi SK. Transfusion Iron Overload. PubMed; StatPearls Publishing. http://www.ncbi.nlm.nih.gov/books/NBK562146. Published 2023. Accessed November 2023.
- Meunier M, Park S. Lower‐risk myelodysplastic syndromes: Current treatment options for anemia. EJHaem. 2022. https://doi.org/10.1002/jha2.523.
- Reblozyl U.S. Prescribing Information. Available at: https://packageinserts.bms.com/pi/pi_reblozyl.pdf. Accessed November 2023.
- Platzbecker U, Della G, Santini V, Zeidan AM, Komrokji RS, Shortt J, Valcárcel D, Jonasova A, Dimicoli-Salazar S, Ing Soo Tiong, Lin C-C, Li J, Zhang J, Ana Carolina Giuseppi, Kreitz S, Pozharskaya V, K. Keeperman, Rose S, Shetty JK, Sheida Hayati. Efficacy and safety of luspatercept versus epoetin alfa in erythropoiesis-stimulating agent-naive, transfusion-dependent, lower-risk myelodysplastic syndromes (COMMANDS): interim analysis of a phase 3, open-label, randomised controlled trial. The Lancet. 2023;402(10399):373–385. doi:10.1016/s0140-6736(23)00874-7.
REBLOZYL® is a trademark of Celgene Corporation, a Bristol Myers Squibb company.
REBLOZYL® is licensed from Merck & Co. Inc., Rahway, NJ, USA and its affiliates.
© 2023 Bristol-Myers Squibb Company
2007-US-2300484 11/23
