Treatments | Aplastic Anemia & MDS International Foundation Return to top.


Introductory image: CDMRP - Group photo
Kevin McQueen, Former President, Board of Directors, Fanconi Anemia Research Foundation
Neil Horikoshi, CEO & Executive Director, AAMDSIF
Honorable Doris Matsui
Debby Cook, Board Member, AAMDSIF
Andre Hessels, President, Board of Directors, Fanconi Anemia Research Foundation

Your doctor considers these factors to determine the best treatment plan for you:

  • Your symptoms
  • Your age
  • Which subtype of MDS you have 
  • Your IPSS (International Prognostic Scoring System) disease risk score
  • Other serious conditions or diseases you have
  • Whether someone is willing and able to donate matching bone marrow to you (preferably a family member)

Treatments for Myelodysplastic Syndromes have a variety of goals. They may:

  • Help healthy cells mature
  • Increase the number of healthy cells in your blood
  • Kill abnormal bone marrow cells 
  • Reduce the number of blasts (young white blood cells) in your bone marrow that have an abnormal shape, size, or appearance.

There are 6 general approaches to the treatment of MDS depending on the type you have and the severity of your case:

  1. Wait and watch, which might be suggested if your blood counts are not too low and your symptoms are not too bad
  2. Supportive care to help you manage the symptoms of your MDS
  3. Drug Therapy with medicines specifically approved to treat MDS and stop abnormal cells from growing
  4. Immunosuppressive therapy, which can lower your body's immune response
  5. Chemotherapy, using drugs that kill abnormal cells
  6. Bone marrow transplantation to replace damaged bone marrow stem cells with healthy ones

Wait and Watch

Your doctor might suggest the wait and watch approach, also called watchful waiting, if your blood counts are not too low and your symptoms are not too bad. In this approach, your doctor will monitor your blood counts and bone marrow to see how they change over time. As blood counts go lower and bone marrow blast counts rise, your doctor will discuss other treatment options.


Blood Transfusions

A blood transfusion is a safe and common procedure. Most people who have a bone marrow failure disease like aplastic anemia, MDS or PNH will receive at least one blood transfusion. When you receive a blood transfusion, parts of blood from a donor are put into your bloodstream. This can help some patients with low blood counts.


If you have MDS and have a high-risk or intermediate-2 risk IPSS score, you have a higher risk of developing Acute Myelogenous Leukemia (AML). To prevent this from happening, your doctor may prescribe intensive chemotherapy in order to kill bone marrow cells that have an abnormal size, shape, or look.

Growth Factors

Growth factors are naturally occurring hormones in your body that signal your bone marrow to make more of certain types of blood cells. Man-made growth factors may be given to some people with bone marrow failure diseases to help increase red blood cell, white blood cell or platelet counts.

Immunosuppressive Therapy

Immunosuppressive drug therapy lowers your body's immune response. This prevents your immune system from attacking your bone marrow, allowing bone marrow stem cells to grow, which raises blood counts.

Iron Chelation

Iron chelation therapy is the main treatment used when you have a condition called iron overload. Iron overload means you have too much iron in your body. This can be a problem for people who get lots of red blood cell transfusions.

MDS Drug Therapy

There are four medicines approved in the U.S. and European Union to treat MDS. Azacitidine (Vidaza®) and decitabine (Dacogen®) are approved to treat both low- and high-risk patients with all sub-types of MDS. Lenalidomide (Revlimid®) is approved for transfusion-dependent MDS patients with isolated del(5q) and with a low or intermediate-1 risk IPSS score. Luspatercept-aamt (REBLOZYL®) is approved to treat adult MDS patients with ring sideroblasts (MDS-RS) or myelodysplastic/myeloproliferative neoplasms with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T), who have been failed an erythropoiesis stimulating agent (ESA) or are unlikely to respond to an ESA, and are requiring transfusions of 2 or more red blood cell (RBC) units over 8 weeks. INQOVI® (previously known as ASTX727) is a prescribed oral tablet combination of decitabine and cedazuridine approved by the US Food and Drug Administration for adult patients with myelodysplastic syndromes (MDS) and patients with chronic myelomonocytic leukemia (CMML) including the following: • previously treated and untreated, de novo and secondary MDS with the following French-American-British subtypes (refractory anemia, refractory anemia with ringed sideroblasts, refractory anemia with excess blasts, and CMML) and• intermediate-1, intermediate-2, and high-risk International Prognostic Scoring System groups.

Bone Marrow Transplant

A bone marrow transplant (BMT) is also called a stem cell transplant (SCT) or hematopoietic stem cell transplant (HSCT).The procedure replaces unhealthy blood-forming stem cells with healthy ones and offers some patients the possibility of a cure. But for many patients, a BMT is not an option due to the risks and potential long-term side effects as an "imperfect cure".

Clinical Trials

Clinical research is at the heart of all medical advances, identifying new ways to prevent, detect or treat disease. If you have a bone marrow failure disease, you may want to consider taking part in a clinical trial, also called a research study.

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