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Receiving a Myelodysplastic Syndromes (MDS) diagnosis can feel overwhelming. It is important to remember you’ve got help and support. Consult with your doctor to look into potential treatment options and their side effects to determine how you would like to move forward in treating your MDS. 

Treatment options for MDS vary based on several factors: MDS type, prognostic score, age, health, and the patient's preferences. A doctor can help you determine which treatment option(s) may be best for you based on the above factors.

Currently, treatment options for MDS include:

  • Watch and Wait
  • Stem Cell Transplant
  • Supportive Care
  • Chemotherapy

Watch and Wait

Patients whose blood count levels aren’t too low and are not exhibiting severe symptoms may choose not to be treated right away and will enter into a “watch and wait” period. In this period, their blood levels are monitored by their doctor and they do not receive any treatment until their counts drop to a point where some intervention is necessary.

Stem Cell Transplant

A stem cell transplant (SCT) is currently considered to be the only way to “cure” MDS. A younger, relatively healthy patient may choose to receive a SCT. Patients who are older or in poor general health, which unfortunately makes up a majority of MDS patients, may not be good candidates for an SCT because of the toll the procedure can take on the body.

As a part of this treatment, patients are given high-dose chemotherapy to kill the cells in their bone marrow. They are then given new stem cells. Typically, an allogeneic SCT is used for MDS patients. This type of SCT requires that the stem cells come from a donor. Patients can be matched to a donor, not related to them, who’s HLA type is similar to the patients cell type, but results are best when the donor is closely related to the patient (like a brother or sister) and the donor's HLA type is a close match to the patient's cell type. 

Side effects of an allogeneic SCT can be pretty severe, even life-threatening. Patients experience typical chemotherapy side effects (mouth sores, vomiting, hair loss, low blood counts, and loss of appetite) but are also at risk of graft-versus-host disease (GVHD). GVHD occurs when the patient's new immune system, from the donor, begins to recognize the patient's cells and tissue as foreign and begins attacking them. This can affect any part of the body, and is watched for closely by doctors following the procedure. 

Supportive Care

Supportive care treatments do not treat MDS directly. Their purpose is instead to help patients improve their quality of life and be more comfortable. To do this, these treatments treat the symptoms of MDS. As one of the main symptoms of MDS is low blood counts, supportive care treatments seek to improve patients blood counts.

Treating Anemia (low red blood cell count)

There are two main ways doctors will go about treating anemia in MDS patients. The first is usually injections of a manmade version of the growth factor erythropoietin which can aid the bone marrow in growing and producing more red blood cells. 

If a patient doesn’t find success with a growth factor, they may need to receive red blood cell transfusions. Patients may be given a cell maturation agent along with the transfusion which can help them go longer between transfusions. 

Treating Neutropenia (low white blood cell count) 

Patients with low white cell counts are at an increased risk of infection, and of those infections becoming serious. Patients are encouraged to take measures to try to prevent catching infections. If a patient does notice signs of an infection, they should alert their doctor. Patients with a bacterial infection will be treated with an antibiotic. 

If the infection does turn serious, patients may be given a white blood cell growth factor to help increase their white blood cell count to help them better fight off the infection.

Treating Thrombocytopenia (low platelet count)

Patients with low platelet counts may be given platelet transfusions or growth factors. If these treatments do not help patients with their symptoms (easy bruising and excess bleeding) they may be given an antifibrinolytic agent, a type of drug that helps to reduce bleeding by blocking the breaking down of blood clots. 


The purpose of chemotherapy in treating MDS is to target the abnormal stem cells in the bone marrow. Depending on the goal decided upon by you and your doctor, different types of chemotherapy drugs may be used. 

Standard Chemotherapy Drugs

Standard chemotherapy drugs are used specifically to attack and destroy MDS cells. Standard chemotherapy drugs are most often used in younger, otherwise healthy, High Risk-MDS patients as they are needed at high doses to be effective, and can be especially hard on the body. Patients who receive high dose standard chemotherapy are the most likely to go into remission, but the side effects can be serious and potentially life threatening. 

Hypomethylating agents 

Hypomethylating agents help to slow the growth of MDS cells by blocking DNA that helps cancer cells to grow. They also help promote healthy cell maturation and formation. 

Immunosuppression Agents

Immunosuppressive agents help to suppress a patient's immune system. Suppressing the immune system allows stem cells the chance to grow and make new blood cells, but does come with risks associated with a lowered immune system.  

Immunomodulating Drugs

Immunomodulating drugs help doctors alter a patient's immune system. This allows doctors to target and depress only specific areas of the immune system, keeping the patient at lower risk of complications as their full immune system is not affected. 

With each type of chemotherapy, side effects exist. Common symptoms are fever, nausea, fatigue, weakness, diarrhea/constipation, hair loss, mouth sores, and loss of appetite. These symptoms are typically most severe with the use of standard chemotherapy drugs. 

Patients with MDS already have lowered blood counts, and the use of chemotherapy drugs will initially lower blood counts further before they begin to take effect and ultimately help to increase the patients blood counts. Because of this, some doctors may choose to administer these drugs in the hospital so the patient can be monitored more closely.


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