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Every patient’s situation should be evaluated individually by a hematologist-oncologist who specializes in treating MDS and who will discuss the disease subtype, prognostic factors, and treatment options with the patient. It is also important to seek treatment at a center that has experience in treating MDS.
Finding the best treatment approach for MDS
The treatment your doctor recommends is based on several factors, including:
- Whether you are in the low-risk or high-risk category
- Your MDS subtype
- Your overall health
- Other health conditions you may have, called comorbidities, such as heart disease, kidney disease, lung disease, or diabetes
Because lower-risk MDS is more likely to progress slowly, low-intensity treatments are used first.
The goals for low-risk MDS patients are to:
- Improve blood counts
- Lessen the need for blood transfusions
- Lower the risk of infection
- Improve quality of life
High-risk MDS tends to grow quickly and progress to acute myeloid leukemia (AML) within a shorter time. For this reason, more intensive treatments are generally used. The goals for high-risk MDS patients are to slow or stop MDS progression to AML and to lengthen survival.
You may find it helpful to bring a loved one with you to your doctor's visits to support you, take notes, and ask follow-up questions. It is a good idea to prepare questions you would like to ask before you visit your doctor. You can also record your conversations with your doctor and listen more closely when you get home.
Learn more about communicating with your blood cancer specialist or find a list of suggested questions to ask your healthcare providers.
Types of treatment for MDS
Doctors use several types of approaches and treatment combinations for MDS:
Supportive care
The goal of supportive care in MDS treatment is to improve quality of life by reducing symptoms caused by low blood cell counts.
Blood transfusions
Many people with MDS receive blood transfusions to help relieve symptoms of low red blood cell counts and/or low platelet counts. A transfusion can help relieve symptoms for a short time. Open each section below to learn more.
Patients with symptoms of low red blood cell counts such as severe tiredness, dizziness and shortness of breath may receive red blood cell transfusions to increase their red blood cell counts. A red blood cell transfusion is a slow injection of donated red blood cells into a patient's body through a vein.
Red blood cells contain iron. When a person receives many red blood cell transfusions, too much iron can build up in their liver, heart and other organs, affecting how these organs work. This condition is called iron overload.
Iron overload requires special treatment to remove the extra iron from the body. The treatment is called iron chelation therapy. The most common drugs used in iron chelation therapy include:
- Deferasirox (Exjade® or Jadenu®)—This medication is a pill taken daily. Exjade and Jadenu are the same medication, but Jadenu may be easier for some patients to digest.
- Deferoxamine mesylate (Desferal®)—This drug is administered as a slow subcutaneous (under-the-skin) or intramuscular (into a muscle) infusion.
Some MDS patients may have low platelet counts that can cause easy bruising or uncontrolled bleeding. These patients may receive treatment to increase their platelet counts. A platelet transfusion is a slow injection of donated platelets into a vein.
For patients with severe low platelet counts or who have uncontrolled bleeding that does not respond to platelet transfusions, the doctor may recommend the drugs aminocaproic acid and tranexamic acid.
MDS and MDS treatments often cause low white blood cell counts. This can increase the risk of infection. Your doctor will pay close attention to any infection or fever. If a bacterial infection is found, you will be treated with antibiotics.
White blood cell transfusions are generally not used for patients with MDS, so doctors sometimes use medications called growth factors to help increase a patient's white blood cell count. Filgrastim, pegfilgrastim, and sargramostim (Leukine®) are growth factors that can help patients increase their white blood cell counts.
Learn more about infections and how to manage them.
For information about the drugs listed on this page, visit our cancer drug listing.
Treatment of lower-risk MDS
For patients with lower-risk disease based on their prognostic score, MDS is typically slow growing. The primary goals of treatment are to improve blood cell counts, reduce
the need for blood transfusions, lower the risk of infection and improve quality of life. Open each section below to learn more.
Lower-risk patients without symptoms may not need to start treatment right away. A watch-and-wait approach allows the doctor to monitor the MDS but not start treatment. Once there are signs and symptoms of the disease, the doctor will start treatment.
Learn more about watch and wait.
This type of drug stimulates the bone marrow to make more red blood cells. It is used to treat people with low red blood cell counts. Treatment with ESAs may decrease the need for red blood cell transfusions. Examples of ESAs include darbepoetin alfa (Aranesp®) and epoetin alfa.
This type of drug is used to treat anemia in adults who need to have regular blood transfusions. It is used when ESAs are not effective in increasing red blood cell counts. One erythroid maturation agent used to treat MDS is luspatercept-ammt (Reblozyl®).
These are drugs that modify different parts of the immune system. People with MDS who need frequent red blood cell transfusions and have MDS cells that are missing a part of chromosome 5 may be treated with lenalidomide (Revlimid®).
MDS patients with low platelet counts can bruise easily and have uncontrolled bleeding. Platelet growth factors are drugs used to help the body produce platelets. Romiplostim (Nplate®) and eltrombopag (Promacta®) are being investigated for treatment of MDS patients who have low platelet counts.
Drugs that suppress certain parts of the immune system can help some patients with lower-risk MDS. Antithymocyte globulin (ATG, Atgam®, Thymoglobulin®), cyclosporine (Neoral®, Sandimmune®) and tacrolimus (Prograf®) suppress certain parts of the immune system and help the body make more healthy blood cells.
If other treatments do not increase low blood cell counts, hypomethylating agents may be a treatment option. They are a type of chemotherapy that may help improve blood cell counts, which may lead to fewer blood transfusions and improve quality of life. Azacitidine (Vidaza®), decitabine (Dacogen®) and decitabine and cedazuridine (Inqovi®) are three hypomethylating agents used to treat MDS.
Certain patients may be cured with an allogeneic stem cell transplantation. Some lower-risk patients, particularly younger patients, may benefit from allogeneic stem cell transplantation. Patients may be considered for this treatment if they have had many therapies and have not responded to treatment. At this time, stem cell transplantation remains the only potential cure for MDS.
An allogeneic transplant is a treatment that uses stem cells from a donor. The stem cells in the donor’s blood must be a “match” to the patient. The donor may be a brother or sister (siblings are most often the best match). The donor may also be an unrelated person with stem cells that match the patient’s cells.
Allogeneic transplants are done in the hospital. After the patient achieves a remission, the process of allogeneic transplant is as follows:
- Stem cells are collected from a donor.
- The patient is given high-dose chemotherapy, with or without radiation therapy.
- The donor stem cells are given to the patient through an intravenous (IV) line or central line.
- The donor stem cells move from the patient’s blood to the bone marrow and begin to start a new supply of red blood cells, white blood cells, and platelets
Allogeneic stem cell transplantation is a type of treatment that destroys cells in the bone marrow and then replaces them with new, healthy stem cells from another person. This treatment is not for all patients, and has a high risk of serious, potentially life-threatening complications.
One serious risk of allogeneic stem cell transplantation is graft-versus host disease (GVHD), which develops if the donor's immune cells attack your normal tissue. GVHD's effects can range from minor to life threatening. Talk with your doctor to see if a stem cell transplant is a treatment option for you or your loved one.
This procedure uses lower doses of chemotherapy than a standard allogeneic transplant. This type of stem cell transplant is for patients who may not be able to tolerate the high doses of chemotherapy that are given to patients during a standard stem cell transplant. Some older or sicker patients may be helped by this type of treatment.
Learn more about allogeneic stem cell transplantation.
For information about the drugs listed on this page, visit our cancer drug listing.
Treatment of higher-risk MDS
Higher-risk MDS tends to grow quickly and is more likely to progress to acute myeloid leukemia (AML). Treatment for higher-risk patients depends on whether they can receive a stem cell transplantation; most cannot. This may be due to many factors including advanced age, other major health problems, or no available stem cell donor.
People with MDS who are not eligible for stem cell transplantation have multiple treatment options. Open each section below to learn more.
These drugs are a type of chemotherapy that may help improve blood cell counts, which may lead to fewer blood transfusions and improve quality of life. Azacitidine (Vidaza®), decitabine (Dacogen®) and decitabine and cedazuridine (Inqovi®) are three hypomethylating agents used to treat MDS.
Patients who are eligible for intensive therapy but who do not have a stem cell donor may receive chemotherapy regimens used to treat acute myeloid leukemia (AML). Because these agents tend to cause more severe side effects, they are generally used in higher-risk MDS that is likely to progress to AML. The drugs used may include:
- Cytarabine (cytosine arabinoside, ara-C; Cytosar-U®)
- Idarubicin (Idamycin®)
- Daunorubicin (Cerubidine®)
- Mitoxantrone (Novantrone®)
Chemotherapy regimens may consist of a single drug or a combination of two or three different drugs (combination chemotherapy).
This type of treatment uses drugs or other substances to identify and attack specific types of cancer cells with less harm to normal cells. Not all cancers have the same targets. Each type of targeted therapy works a little bit differently, but they all interfere with the growth and survival of cancer cells. Some targeted therapies for treating MDS include:
- IDH Inhibitors. In some people with MDS, the cancer cells have a mutation of the IDH1 or IDH2 gene. These patients may receive:
- Ivosidenib (Tibsovo®), for adult patients with an IDH1 mutation with relapsed or refractory MDS
- Enasidenib (Idhifa®), an off-label treatment for MDS with an IDH2 mutation. Off-label describes the legal use of a prescription drug to treat a disease for which the drug has not been approved by the FDA.
- BCL2 Inhibitors. Some studies have shown that the BCL2 inhibitor venetoclax (Venclexta®) in combination with hypomethylating agents may reduce the number of cancer cells in the bone marrow for patients with high-risk MDS.
- FLT3 Inhibitors. Some MDS patients have a mutation in the FLT3 gene that can increase the growth and division of cancer cells. FLT3 inhibitors are drugs that target these gene mutations. For patients with FLT3 mutations, midostaurin (Rydapt®), gilteritinib (Xospata®), quizartinib (Vanflyta®) or sorafenib (Nexavar®) may be prescribed. These drugs are not FDA-approved to treat MDS, but they are being studied in clinical trials and are also available as off-label treatments.
Taking part in a clinical trial may be the best treatment choice for some MDS patients. Clinical trials are underway for all MDS-risk types. Because today's standard treatments for cancer are based on earlier clinical trials, LLS continues to invest funds in MDS research.
Read more about clinical trials.
Get free clinical trial support! Visit our Clinical Trial Support Center (CTSC).
Connect with registered nurses with expertise in blood cancers who can personally assist you or your caregiver through each step of the clinical trial process.
Follow-up care
Those who have been treated for MDS are encouraged to:
Maintain regular follow-up appointments with their hematologist-oncologist. Your doctor will monitor you for signs of relapse and detect any side effects from treatment. A follow-up visit may also discover the onset of any other medical problems.
Keep a record of your cancer diagnosis, treatments, and follow-up care needs. This is often called a “survivorship care plan.” Ask your doctor for a written survivorship care plan. Share this information with any new healthcare providers you see. The plan should include the following information:
- List of all healthcare providers
- Diagnosis summary with specifics such as subtype and/or genetic markers
- Treatment summary with specifics such as:
- Names, dates, and dosages of chemotherapy or other drugs
- Site of radiation treatment, surgery and/or transplantation information
- Response to treatment, and side effects
- Maintenance treatment information, if applicable
- List of possible late effects
- Schedule for ongoing monitoring with recommended tests, frequency, and coordinating provider
- Health and wellness recommendations such as nutrition, exercise, or other disease screenings
- Seek medical and psychosocial support for fatigue, depression, and other long-term effects, if needed
- Consider cancer risk-reduction strategies, such as smoking cessation, skin protection against prolonged sun exposure, healthy eating, and exercising.
Find more information about follow-up care, including what to expect, long-term and late effects of treatment, survivorship clinics, and other resources, such as The National Comprehensive Cancer Network (NCCN) treatment guidelines.
Use the Survivorship Workbook to collect all of the important information you need throughout diagnosis, treatment, follow-up care, and long-term management of a blood cancer.
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