Myelodysplastic syndromes (MDS) refer to a group of disorders in which the bone marrow produces too few mature and/or functioning red blood cells, white blood cells or platelets. There are different types of MDS, depending on the types of blood cells that are affected.

Treatment Options

Physicians at the Johns Hopkins Kimmel Cancer Center take an aggressive, comprehensive approach to myelodysplastic syndromes treatment, from state-of-the-art testing to involving patients in ongoing clinical trials. Patients are seen in a multidisciplinary clinic at the Center for Bone Marrow Failures, where patients with difficult cases are discussed by a group of experts who work together to develop short- and long-term goals for the overall best treatment plan.

Blood Transfusion

Blood transfusions can increase the number of normal red blood cells in circulation, helping to relieve anemia and treat symptoms like fatigue and shortness of breath. Platelets, another type of small blood cell, can also be transfused to prevent or stop bleeding if a patient’s platelet counts are very low.

Growth Factors

Some patients with MDS may receive doses of hormone-like drugs called growth factors that stimulate the patient’s bone marrow to produce blood cells. Growth factors that increase red blood cell production include erythropoietin (Epo or Procrit) or long-acting darbepoetin alfa (Aranesp). Growth factors like G-CSF, neupogen, or filgrastin and GM-CSF or sargramostim can improve white blood cell production among MDS patients with frequent infections. Pegfilgrastim (Neulasta) is a long-acting form of G-CSF.

Chemotherapy

Because MDS can progress to acute leukemia, patients are often treated with chemotherapy used for the disease. Your physician will recommend one or more chemotherapy drugs for your treatment, which are given as either pills or as fluids administered through a needle or tube inserted into a vein. Chemotherapy is typically given every two to three weeks for several cycles. Side effects can include fatigue, hair loss, poor appetite, nausea or vomiting, diarrhea, or infertility.

One class of chemotherapy drugs used in the treatment of MDS is called DNA methyltransferase inhibitors or hypomethylating agents. These drugs affect the way genes are controlled. They slow down genes that promote cell growth and kill cells that are dividing rapidly. Examples of this type of drug include azacytidine (Vidaza) and decitabine (Dacogen). In some MDS patients, these drugs improve blood counts, lower the chance of getting leukemia, and in the case of azacytidine prolong life. Red blood cell counts may improve enough to stop transfusions. These drugs have mild side effects including fatigue and temporary lowering of the blood counts.

Other Medications

Another drug used for MDS is lenalidomide (Revlimid). This drug, taken as a pill, may work well in low-grade MDS, eliminating the need for transfusions in about a quarter of the patients treated. The drug works much better in patients whose MDS cells are missing a part of chromosome number 5.

Medications that lower the immune system (immunosuppressants) improve blood counts in some patients with MDS. These drugs include anti-thymocyte globulin (ATG) and cyclosporine A.

A bone marrow transplant or stem cell transplant gives a patient healthy stem cells – immature cells that grow into different types of blood cells. These stem cells come from marrow, the soft, fatty tissue inside the bones, replacing marrow that is either not working properly or has been destroyed by chemotherapy or radiation. Stem cells may be taken from the patient’s marrow before getting chemotherapy or radiation. They also may come from a relative or other donor, or from umbilical cord blood.

Most patients get high doses of chemotherapy, radiation or both before the bone marrow transplant to kill any cancer cells that might remain and make room for new stem cells to grow. Doctors then deliver the stem cells through a tube injected into the bloodstream. While receiving stem cells, patients may experience pain, chills, fever, hives, chest pain or other symptoms. (Learn more about the Johns Hopkins Bone Marrow Transplant Program.)

Clinical Trials

Clinical trials test new drugs and new ways of treating patients with myelodysplastic syndromes. Our experts conduct these studies to find better treatments. Many clinical trials become the next standard of care. To determine if a trial is right for you, make an appointment for a consultation at 410.955.8964.

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