MYELODYPLASTIC SYNDROME

Myelodyplastic Syndrome – General Information

Myelodyplastic Syndrome – Symptoms

One in fifty MDS patients have no signs or symptoms and can discover that he has the syndrome, by chance, as a result of a routine blood test. Those patients who do have symptoms present with clinical features due to bone marrow failure. In about 80% of patients this is simple anemia, whilst about 20% present with infections or bleeding. Anemia tends to lead to fatigue and shortness of breath even on light exertion. Infections are usually either bacterial pneumonia or skin or throat abscesses. About 10% of patients will have an enlarged spleen. Those patients who do not have symptoms may require no treatment until symptoms develop. Virtually all patients will require supportive treatment including red cell transfusions at some stage during their illness. For patients who require treatment, the choice of therapy is based on their age.

The disease may also lead to a shortage of white blood cells resulting in an increased likelihood of infections. Another symptom of the syndrome is increased bleeding (blood in stool, nose bleeds, increased bruises or bleeding gums) which is due to low level of platelets. These symptoms can occur in any combination, depending on a given patient's specific subtype of MDS. Usually, the disease is diagnosed when you will perform a blood test, to count the number of each kind of blood cell. If the results of the blood test are not normal, the doctor will do a bone marrow biopsy.  

Myelodyplastic Syndrome – Treatment

The treatment of Myelodyplastic Syndrome (MDS) depends on patient’s age and whether he had other serious diseases. Patients with these diseases are treated by specialists, such as a hematologist or an oncologist. The treatment includes chemotherapy, growth factors, angiogenesis inhibitors, epigenetic agents, targeted therapy, supportive therapy, stem cell transplant, and a general approach to treatment of the disease.

Chemotherapy is the use of anticancer drugs, which can be injected into a vein or taken by mouth. For patients with Myelodyplastic Syndrome, a surgeon may place a small plastic tube (venous access device) into a large vein which is left there so that nurses can inject chemotherapy and other medications. Also, the chemotherapy slows blood production. This problem is usually made worse before it gets better. It can lead to lowered resistance to infection (due to low white blood cell counts), easy bruising and bleeding (due to low platelet counts), and fatigue (due to low red blood cell counts). Growth factors are hormone substances that stimulate bone marrow to produce blood cells. These substances grow naturally in the body, but scientists found a way to manufacture them in large quantities so the patient can get a large dose. Studies have tested several growth factors in patients with MDS, such as granulocyte colony stimulating factor (G-CSF or filgrastim) and granulocyte macrophage-colony stimulating factor (GM-CSF or sargramostim). These can benefit some Myelodyplastic Syndrome patients whose main problem is a shortage of granulocytes and are suffering from frequent infections. One of the angiogenesis inhibitors is thalidomide, and a few patients taking this drug have been able to avoid receiving blood transfusions because they regained the ability to make red blood cells. Others had increases in their granulocyte counts and platelet counts. Some people can have bad side effects and because of this a new drug, called lenalidomide, can also help treat MDS.

Epigenetic agents affect the way genes are controlled, and one such drug is azacytidne. Because the drug is really like a chemotherapy drug, it can also lower blood counts so it must be given very carefully. For about a year, 20% of patients are helped in that they are able to maintain higher blood counts and need fewer transfusions. With a stem cell transplant, the patient's bone marrow is intentionally destroyed by high-dose chemotherapy and total body irradiation. The abnormal bone marrow cells are then replaced with new, functioning blood-forming stem cells. The stem cells transplant is very risky, because side effects can appear. Because this disease most often occurs in older patients who may also have other medical problems, stem cell transplant may not be an option. Since no curative treatment is available for these individuals, the goal is to relieve symptoms of Myelodyplastic Syndrome and avoid complications and side effects of treatment. Treatments that can relieve or prevent symptoms of MDS (Myelodyplastic Syndrome) include transfusions, blood cell growth factors, and possibly hormones. Careful general medical care and measures to prevent and treat infections are very important. Patients should participate at more clinical activities.