Myelodysplastic syndromes (MDS) affect the way your body makes new red blood cells, white blood cells, and platelets. MDS treatments slow the disease, help relieve symptoms like tiredness and shortness of breath, and help you feel better.
Not everyone with MDS needs treatment right away. If you don't have symptoms that bother you and your blood cell counts aren't low, you may be able to wait.
Once you do need treatment, your doctor will help you choose the right one or more therapies based on your:
- MDS type
- Age
- Health
- International Prognostic Scoring System (IPSS) disease severity score. This is used to determine the outcomes and help develop a treatment plan. It’s based on your blood cell count, changes in genes and chromosomes of MDS cells, and number of abnormal blood cells
Three types of treatment are available for MDS:
- Supportive care to manage symptoms
- Medicine to slow the disease
- Stem cell transplant to possibly cure MDS
Supportive Treatments for MDS
These treatments relieve MDS symptoms and prevent complications from having too few healthy blood cells. Some of them work by boosting your blood cell count. Others treat infections or stop you from bleeding too much.
You can get supportive therapies by themselves or together with other treatments.
Transfusions
MDS prevents young blood cells from growing into healthy mature cells in your bone marrow – the spongy tissue inside your bones. You need red blood cells to carry oxygen to your body, white blood cells to fight infections, and platelets to stop you from bleeding.
A transfusion gives you the healthy blood cells your body needs. The new blood cells go into your bloodstream through a needle.
After you've been getting transfusions for many years, too much iron can build up in your blood. Chelating agents are medicines that remove the extra iron. They come as a pill or as an IV you get under your skin.
Growth factors
Growth factors are man-made versions of hormones your body makes to increase your blood cell counts. Darbepoetin (Aranesp) and erythropoietin (Procrit) stimulate your bone marrow to make new red blood cells. Filgrastim (Neupogen), pegfilgrastim (Neulasta), and sargramostim (Leukine) help your body make new white blood cells.
Blood cell maturation drugs
Luspatercept (Reblozyl) is a medicine that helps your red blood cells grow faster. You may not need to have as many blood transfusions if you take it.
Anti-clotting medicines
MDS can leave you low in platelets – the cells that help your blood form clots to stop you from bleeding too much if you cut yourself. Medicines called antifibrinolytic drugs help your blood clot when your body doesn't make enough platelets.
Antibiotics
White blood cells protect your body from germs like bacteria and viruses. Having too few of these cells puts you at higher risk for infections. If you do get a bacterial infection, your doctor may prescribe antibiotics to treat it.
Medicines to Slow MDS
These treatments work in different ways to reduce your body's production of abnormal blood cells and help you make more healthy blood cells.
Lenalidomide
About 15% of people with MDS have a type of the disease called deletion 5q, where part of their fifth chromosome is missing. This chromosome helps your body make new red blood cells.
The cancer drug lenalidomide (Revlimid) treats deletion 5q MDS by increasing red blood cells. It can also help some people who don't have deletion 5q. One side effect of Revlimid is that it can lower white blood cell and platelet counts.
Chemotherapy
The chemotherapy drugs azacitidine (Vidaza), decitabine (Dacogen), and decitabine-cedazuridine (Inqovi) slow the genes that help abnormal cells grow in MDS. These medicines also kill quickly dividing abnormal cells.
They help your bone marrow work better. They reduce the number of unhealthy cells and increase healthy blood cells in your bone marrow. This relieves symptoms like tiredness and may help you need fewer blood transfusions.
You might get other types of chemotherapy drugs if you have a lot of abnormal blood cells in your bone marrow, or if your MDS is turning into the cancer acute myeloid leukemia (AML).
You'll get chemo treatment once every 2 to 3 weeks. Side effects of these medicines include:
- Hair loss
- Tiredness
- Appetite loss
- Nausea or vomiting
- Diarrhea
Your doctor can give you other medicines to lessen chemotherapy side effects during your treatment.
Immunosuppressants
In some people with MDS, immune system cells disrupt the body's blood cell production. You may need to take medicines that suppress the immune system and boost blood cell counts, such as:
- Antithymocyte globulin (ATG, Atgam, Thymoglobulin)
- Cyclosporine (Neoral, Sandimmune)
- Eltrombopag (Promacta)
- Tacrolimus (Prograf)
Stem Cell Transplant for MDS
Stem cells are the early cells that grow into new blood cells. A stem cell transplant replaces abnormal cells in your bone marrow with healthy ones. Another name for this procedure is a bone marrow transplant.
To get this treatment, you need to be healthy and younger than 75. You also need a donor who is a good match for you. If you're a good candidate, a stem cell transplant might cure MDS.
You’ll first get high doses of chemotherapy to kill off your damaged stem cells. Then you get an IV of new stem cells taken either from a donor or from your own bone marrow before you have chemo.
Making MDS Treatment Decisions With Your Doctor
You and your doctor will talk over your MDS treatment options. Make sure you understand the pros and cons of each treatment, and whether it is a good fit for you.
Another option is to join a clinical trial. These studies test out new MDS drugs and combinations of treatments. Enrolling in a clinical trial could give you access to a new treatment before it is available to everyone else.
Before you join a clinical trial, make sure you understand:
- The purpose of the study
- How the new treatment might help you
- Whether you might get the standard treatment or an inactive treatment inside of the new drug
- What side effects the study drug might cause
