Medication for Myelodysplastic Syndromes

NYU Langone doctors use medications to manage myelodysplastic syndromes. The types of medications prescribed depend on whether the condition has a low or intermediate risk of progressing to acute myeloid leukemia, or AML.

Although people with high-risk syndromes can also receive medications, doctors typically recommend stem cell transplantation to those people if they are eligible.


Doctors may prescribe lenalidomide to people with a low-risk form of the condition, called myelodysplastic syndrome associated with isolated deletion 5q. Lenalidomide is an immunomodulating medication, meaning it affects how the immune system functions. It is taken by mouth each day.

This medication can help to improve red blood cell levels, so people can avoid blood transfusions to manage their anemia.

Side effects of lenalidomide may include diarrhea, constipation, skin rash, and nausea. White blood cell and platelet levels may drop. Some people may have an increased risk of developing blood clots.

Doctors can adjust the dose of lenalidomide or prescribe other medications to help manage these side effects.

Growth Factors

People with an intermediate risk of myelodysplastic syndrome progressing may benefit from growth factors. These medications stimulate the bone marrow to produce more blood cells.


For people with anemia, doctors can prescribe erythropoietin. This medication contains synthetic protein that helps the bone marrow produce red blood cells. It is given by injection every other week for a length of time determined by your doctor.

Side effects may include joint, muscle, or bone pain, as well as fever, cough, and rash. People taking this medication may have an increased risk of developing blood clots. Doctors can prescribe other medications or adjust the dose of erythropoietin to manage side effects.

Granulocyte Colonyā€“Stimulating Factor

Granulocyte colonyā€“stimulating factor is a protein that can boost white blood cell levels in the bone marrow. This helps manage the risk of infection in people who have a myelodysplastic syndrome with low levels of white blood cells.

This medication is given by injection every day as needed.

Sometimes giving granulocyte colonyā€“stimulating factor with erythropoietin can also help increase red blood cell levels.

Side effects of granulocyte colonyā€“stimulating factor may include bone and muscle pain. Doctors can prescribe or recommend medication to relieve discomfort.

Hypomethylating Agents

Doctors may prescribe hypomethylating agents to people with intermediate-risk myelodysplastic syndrome, or to people with high-risk myelodysplastic syndrome who are not healthy enough for stem cell transplantation.

They may also prescribe these agents to reduce the percentage of blasts in the bone marrow. This can make people healthy enough for stem cell transplantation.

Hypomethylating agents help to destroy rapidly dividing, abnormal cells in the bone marrow. They also interfere with genes that promote cell growth. This helps normalize blood cell levels.

Examples of hypomethylating agents used to manage myelodysplastic syndrome include decitabine and azacitidine.

Decitabine is given via intravenous (IV) infusion into a vein in the arm. Treatment is daily, for a period of 5 to 10 consecutive days, followed by several weeks of rest, so you can recover. This treatment schedule is called a cycle. Doctors give decitabine for four or more cycles.

Azacitidine is injected under the skin or given through a vein with IV infusion once a day for a week, followed by several weeks of rest. This cycle may be repeated four or more times.

Side effects of hypomethylating agents can include nausea and vomiting, diarrhea or constipation, and weakness and fatigue. These effects are usually mild but can be managed by reducing the dose. Sometimes, blood cell levels may temporarily drop, making blood transfusions necessary.

Clinical Trials

Thrombopoietin mimetics are medications that stimulate the production of platelets in the bone marrow. Doctors at NYU Langone are studying whether these medications help restore healthy platelet levels in people with myelodysplastic syndrome who have a reduced amount of platelets. This may help people who have problems with bleeding or bruising.

Your doctor can discuss whether a clinical trial is right for you.