Conditions


Myelodysplastic syndrome (MDS)

Overview

The Myelodysplastic syndrome (MDS) are a group of diseases causing progressive failure of the bone marrow to produce blood. Blood is composed of three main cell types: red, white and platelets. Red cells carry oxygen to the muscles and provide the body with energy. Platelets are the clotting cells that prevent bleeding. White blood cells are the cells of the immune system. Failure to produce blood can over time place patients at risk for bleeding, infections and direct organ damage (principally heart damage). As the bone marrow deteriorates, approximately 40percent of patients will progress to acute leukemia, a rapidly advancing form of blood cancer (discussed above). MDS has also been called pre-leukemia (although most patients never develop leukemia).

MDS is common in older individuals. Although the true incidence is unknown, and is suspected to be in the range of 20-30,000 cases, recent research performed at Hackensack University Medical Center and presented by Dr Stuart Goldberg at the 2007 International MDS Meetings has suggested that the disease occurs in approximately 180 persons per 100,000 in the US (far more common than previously suspected).

Hackensack University Medical Center has been designated as a "Center of Excellence&quot: by the MDS Foundation for its clinical and research work in MDS. At The Cancer Center, our chief of the Division of Leukemia, Stuart L. Goldberg, M.D., is recognized as an authority on MDS and all types of leukemia. Because many patients with MDS go on to develop acute myeloid leukemia, it is beneficial for them to be under the care of a hematologist/oncologist like Dr. Goldberg who is highly skilled in the diagnosis, treatment, and management of both MDS and acute myeloid leukemia. He also serves as the medical advisor to the Northern NJ Chapter of the Leukemia and Lymphoma Society. Dr. Goldberg leads an active research team that is conducting about 30 clinical trials for new treatments for leukemia and MDS. Many with MDS at The Cancer Center are enrolled in a clinical trial. National Cancer Institute research groups, pharmaceutical companies, and independent research agencies frequently turn to Dr. Goldberg to collaborate on clinical research studies because of his successful accruals (enrollments) and his ability to attract new patients to The Cancer Center.

Risk Factors

The MDS are primarily diseases of the elderly. Myelodysplastic syndrome can result from prior cancer treatments, working with pesticides, or from an autoimmune response that causes the patient's immune system to attack its own bone marrow.

Symptoms

Symptoms of MDS may appear suddenly or may slowly progress. Some other common symptoms:

  • fever, chills, and other flu-like symptoms
  • frequent infections
  • weakness and fatigue (due to anemia)
  • loss of appetite and/or weight loss
  • swollen or tender lymph nodes, liver, or spleen
  • easy bruising or bleeding
  • tiny red spots (called petechiae) under the skin
  • swollen or bleeding gums
  • sweating, especially at night
  • bone or joint pain

Diagnostic Services

A diagnosis of MDS is made after several tests have been completed. Samples of cells are taken from a patient's blood and bone marrow. A crucial component to the accurate diagnosis of MDS is The Cancer Center's Special Diagnostic Immunology Laboratory, one of only several sites in New Jersey where comprehensive tests are available to detect cancer at the molecular level and to stage and classify it.

One of the major advances in the field of MDS has been the development of an accurate prognostic scoring system known as the IPSS. Patients with lower risk disease as determined by the IPSS may be followed without treatment, whereas patients with higher risk disease are frequently encouraged to receive treatments or enroll on one of the many clinical trials.

Treatment Services

As a Center of Excellence for MDS, the Cancer Center conducts many clinical trials in MDS. Your hematologist/oncologist will decide which treatment or combination of treatments is best for you. The decision to receive treatments can often be quite difficult but may be based on the patient's subtype of MDS, general health, age, and overall desires. Shortly after diagnosis a comprehensive review of the type of MDS is performed to determine the aggressiveness of the cancer and to provide prognostic information that may be helpful in chosing a treatment course.

Supportive Treatment

Whole blood transfusions or transfusions of platelets, white blood cells, and red blood cells may be given as supportive treatment. Transfusions form the core treatment for most patients. To maximize quality of life issues for our patients, the Cancer Center has a dedicated transfusion clinic to allow same day outpatient transfusions, thus minimizing the need for hospitalizations for most of our patients. Growth factors, such as erythropoietin (Procrit) or filgrastim (Neupogen) may stimulate the failing bone marrow and improve blood counts. Research at Hackensack University Medical Center with an iron chelation agent (Exjade) is striving to reduce the complications of iron overload seen in many patients receiving chronic transfusion therapy.

Low Intensity Chemotherapy

Within the past 5 years, several new low intensity chemotherapy agents have been developed to stimulate the failing bone marrow and to reduce the risk of transformation to acute leukemia. These therapies are typically well tolerated, even in older individuals. Hypomethylating agents such as 5-azacitidine and decitabine are administered in the outpatient Cancer Center monthly. Both agents have been found to be beneficial for patients with intermediate risk IPSS disease in improving blood counts. Ongoing studies at Hackensack University Medical Center are seeking to determine how best to give these medications. Clinical research studies are also exploring combining these medications with other agents to develop a more effective "cocktail&quot:. A second class of medications, known as the immodulators (Thalidomide and Revlimid) have also only recently become widely available for the treatment of this disease. These pill medications improve blood counts but require careful monitoring in the Cancer Center.

The Cancer Center has been active in new drug development in MDS. Recent studies have included trials with SCIO-469 and FG-2213. Additional studies with new agents are ongoing.

High-Intensity Treatments (Stem Cell Transplantation)

Stem cell transplantation may be used to restore blood-producing stem cells that have been destroyed by disease or cancer treatment. Stem cell transplantation is a highly advanced type of treatment that is available at only several hospitals in New Jersey. Our Adult Blood and Marrow Stem Cell Transplantation Program is one of the nation's 10 largest. Each year more than 200 persons undergo stem cell transplantation here at The Cancer Center. The program is the only one in New Jersey where stem cell transplants using cells from unrelated donors are performed. Our chief of the Division of Leukemia, Stuart L. Goldberg, M.D., and other experts in stem cell transplantation at The Cancer Center are responsible for some of the major advances in the techniques used in stem cell transplantation, including the use of reduced-intensity "mini&quot: transplants to treat older patients and those who are too frail to undergo the rigors of a standard transplant.

Stem cell transplantation represents the only curative therapy for MDS. Younger individuals, especially those with higher risk IPSS disease, are introduced to members of the transplantation team shortly after diagnosis and early identification of appropriate stem cell donors is begun. Careful discussions regarding the appropriate timing of the transplant are key to maximizing success. A multi-divisional approach to MDS is facilitated at our center by weekly joint meetings between members of the Leukemia and Transplantation Divisions.

High Intensity Treatments (Immunosuppressive Therapy)

Approximately 10-20 percent of MDS have an underlying autoimmune component to their disease. Many of these patients with have an unusual HLA (human leukocyte antigen) tissue type HLA-DR15. Patients with this type of MDS may respond to immunosuppressive therapies including anti-thymocyte globulin (commonly known as horse serum) or cyclosporine.

Prevention

Last updated: 2010-07-30