MDS treatment essentially has two goals, depending on whether it is a low-risk IPSS form or a high-risk IPSS form. In the first case, it tries to get an increase in values, hemoglobin, platelets and neutrophils, and thus improve the quality of life, with no blood transfusions. On the other hand, in the most serious cases where there is possible an acute leukemia evolution, the therapy is aimed to distance this evolution and to prolonge life, improving the values ​​of blood count and eliminating marrow blasts.There is no definitive cure for myelodysplasia now, with the exception of bone marrow / stem cell transplant, which is unfortunately only applicable to a small minority of patients, as it can be done in people without other major pathologies up to 65/70 years of age.Many factors  influence the therapeutic choices :

  • Sometimes patients with only moderately decreased values ​​have significant symptoms. For example, the anemia in cardiopathic patients causes difficulty in breathing and considerable fatigue.
  • The age
  • The kind of myelodysplasia
  • IPSS risk score
  • Comorbidity (ie other health problems of the patient)
  • The presence of a stem cell donor available (preferably a family member)

Here are the general approaches to treating myelodysplasia  that depend on the kind of MDS and its severity:

  • Watch and wait: in patients with low IPSS risk and without symptoms
  • Transfusion Support Therapy to help the Patient to manage the symptoms due to myelodysplasia
  • Therapy with growth factors for red blood cell in symptomatic anemia
  • Pharmacological therapy to stop the growth of abnormal cells and improve the blood count values
  • Immunosuppressive therapy that can lower the immune response of the body
  • Chemotherapy
  • Stem cell transplant to replace damaged bone marrow cells with healthy cells
  • Experimental therapies when standard and approved therapies have proved to be ineffective

The doctor may decide to observe the trend of myelodysplasia when the patient does not accuse very serious symptoms and blood values ​​are not particularly alarming. Blood count and bone marrow tests are performed at regular intervals to check whether the situation is stable or changes occur. If the values ​​get worse or the number of blasts in the marrow increases, the doctor will decide to start the most appropriate therapy.

Blood transfusions

When hemoglobin exhibits particularly low values, typically below 8g / dL, the physician may decide to proceed with red blood cell transfusions, but sometimes platelet transfusions are also required. This will increase the blood values, with an improvement of the symptoms. In the case of red blood cell transfusions prolonged for months, it will also be necessary to remove excess iron with therapies called iron chelators.


Iron Chelator therapy

This therapy is used when the patient has “iron overload”, so it is aimed to eliminate the excess  of iron that accumulates as a result of red blood cell transfusions and which can cause problems  to vital organs (heart, liver, endocrine glands ).

Growth factors – Red blood cells stimulating agents

It consist in the administration of the artificial version of hormones already present in the blood, stimulating it to produce more red blood cells and white blood cells.

Some growth factors, such as erythropoietin (EPO) or Darbopoetin alpha, may affect the production of red blood cells and decrease or eliminate the need for blood transfusion. In some anemic patients, in fact, their EPO level is not sufficient  to stimulate  the sick bone marrow and in these cases the administration (subcutaneously) of synthetic erythropoietin (produced in laboratory) can be anf help. This therapy is effective in the majority of patients and it is chronic.

Other growth factors, on the other hand, are given to stimulate white blood cells, helping to treat the recurrent infections due to neutropenia, but they are not administered  continuously.



It is an oral drug specifically used in myelodysplasia with deletion of chromosome 5q and requiring blood transfusions. Lenalidomide interferes with the immune system activity by blocking cell growth with chromosomal abnormality. In these cases it has a very high efficacy, sometimes reaching to normalization of hemoglobin levels.


Immunosuppressive Therapy

It is especially indicated in low-risk myelodysplastic syndromes, in patients not too elderly and in good general conditions, which have a non-populated bone marrow and some clinical and biological characteristics. This therapy decreases the immune response with the aim of preventing the destruction of bone marrow’s hematopoietic cells and allowing the stem cells to grow, resulting in improved blood values.


Hypomethylating drugs

In patients with intermediate-high IPSS risk not eligible for transplant or high-dose chemotherapy, may be used  this type of  therapy (5 Azacitidine and Decitabine), which, by lowering DNA  methylation levels, reprogram the myelodysplastic  cells and renovate  their function and production of healthy cells. Azacitidine is given as outpatient treatment and is the only one of the two hypomethylating agents approved in Europe for the treatment of myelodysplasia, while Decitabine is approved only for elderly acute myeloid leukemia.



In cases of high or intermediate 2 IPSS  risk myelodysplasia, there is a greater probability of having an acute myeloid leukemia evolution. Sometimes the physician can opt for intensive chemotherapy with the aim of killing abnormal bone marrow cells. This type of therapy is normally used in young patients and / or who can be transplanted (and who have no other serious associated illness). It is a therapy that requires hospitalization in isolation for a long time and should always be followed whenever possible by transplant.

Stem cell transplant

It is also sometimes called bone marrow transplant. The procedure first involves the administration of a preparatory chemotherapy that destroys all bone marrow cells and then the reinfusion in the patient of healthy donor stem cells, which have the task of repopulating the blood and the marrow itself.

However, this procedure is not always applicable due to the lack of a suitable donor, or the presence of comorbidities or other risk factors (such as older age that makes more fragile) that make it difficult or completely exclude the choice of this therapeutic approach.

The Scientific research, trying  to improve the condition of myelodysplasic  patients, never stops and aims to look for new ways to prevent, identify and treat the disease. The physician, considering the single case, may propose to the patient the participation in an experimental clinical protocol, , if he thinks that  it could be a better therapeutic choice. This is particularly recommended  if classical and standard therapies have lost effectiveness or have showed to be inactive.

It therefore concerns to dealing with myelodysplasia with drugs that have not yet entered the current clinical practice for the treatment of the disease and which can only be accessed by participating in highly controlled trials in selected hospitals.