anemia

How To Diagnose and Treat Myelodysplastic Syndromes

How Is MDS Diagnosed?

Table of Contents

If your doctor suspects that you have MDS, a number of tests may be done in order to confirm the diagnosis. These tests include: Blood tests. These are usually the first tests done if MDS is suspected.Abnormally low blood counts may be the first sign of MDS. Results of a complete blood count (CBC) may show low red blood cell counts (anemia), low numbers of infection-fighting white blood cells, or low platelet counts. The CBC may also show unusually large or small red blood cells, or immature white blood cells called blasts. Other blood tests may include a reticulocyte count and peripheral smear examination, which can give information about how well your bone marrow is making new blood cells; a test for fetal hemoglobin (HbF); and a liver and kidney function test. Bone marrow aspiration and biopsy. This procedure involves removing a sample of bone marrow fluid (aspiration) and tissue (biopsy) from your hipbone or breastbone with a needle. This sample can be examined in the laboratory to look for abnormalities in blood cell production and structure that suggest MDS. In some cases, the sample can also be tested for chromosome abnormalities that are often seen in MDS.

How Is MDS Treated?

Your treatment options depend on the type of MDS you have and your symptoms. Also, consider what your goals are for treatment. Do you want to stop the disease from getting worse? Or do you want to have a stem cell transplant?

Your doctor will look at your overall health and your specific type of MDS when recommending treatments. You may need more than one treatment. These are common ones:

Blood transfusions. This is the most common treatment for people with Low Blood Counts (Anemia) caused by MDS.

Growth factors. These drugs help the bone marrow make healthy blood cells. They’re given as shots under the skin or through a vein (IV).

Chemotherapy. These drugs can help stop abnormal blood cells from growing, but they can also kill healthy ones. You may need to take them for a short time during treatment or long-term if the MDS keeps coming back.

Cytotoxic agents. Doctors use these drugs to destroy abnormal cells in people with certain types of MDS that haven’t responded well to other treatments, including chemotherapy and growth factors.

Immunosuppressive drugs. These medications slow down or stop your immune system from attacking healthy blood cells in people with MDS.

Treatment Options for MDS

The treatment of MDS depends on the type of MDS, its stage, and the patient’s age and overall health. Treatment may include blood transfusions, medications to stimulate red blood cell production, and/or chemotherapy.

Blood Transfusions

Blood transfusions are often used in MDS at diagnosis to raise the hemoglobin level and reduce the need for transfusions. Patients with a low platelet count should be monitored closely for bleeding problems so that they can be treated promptly.

Medications to Stimulate Red Blood Cell Production

Certain drugs, including erythropoietin (EPO) and darbepoetin (Aranesp), can increase red blood cell production. These drugs are effective in about 40%–50% of patients with lower-risk MDS. They are not effective in higher-risk forms of MDS.

Chemotherapy

Treatment options include:

Single Drug Therapy: Lower-risk patients who require therapy are often treated with azacitidine (Vidaza) or decitabine (Dacogen). Both drugs have been shown to prolong survival, but neither is considered curative.

Stem Cell Transplant for MDS

Stem cell transplant is a procedure in which healthy blood stem cells are used to replace the unhealthy ones. People who have MDS usually need a stem cell transplant as soon as possible after they are diagnosed, but only if they are healthy enough to survive the procedure. In certain cases, people with MDS may be able to wait and get the transplant later, when they are sicker. This can reduce the risk of relapse due to “clonal evolution” during the waiting period and also allows time for new drugs that may increase survival after transplant. This strategy is called “watch and wait” or “delayed transplant.”

If you need a stem cell transplant, your doctor will discuss with you whether to use your own stem cells (autologous) or stem cells from a donor (allogeneic). Autologous transplants can be performed only if you have few or no symptoms of MDS and your disease has not transformed into AML. Allogeneic transplants can be done after autologous transplants fail or if your MDS is too advanced for an autologous transplant.

Clinical Trials for MDS

There are many different types of clinical trials that may be appropriate for patients with myelodysplastic syndromes (MDS). The goal of a clinical trial is to help scientists find new and better ways to detect, treat, or prevent the disease. Some of these studies look at new treatments or compare existing treatments. Others look at ways to improve comfort or quality of life for people living with MDS. Still others focus on prevention or screening.

Patients who participate in a clinical trial may receive the standard treatment, a new treatment, or no treatment at all. The choice depends on what is being studied and what researchers already know about the disease they are studying.

Research studies often involve more than one type of healthcare professional. Doctors, nurses, social workers and other healthcare professionals work together during a study to care for the needs and concerns of patients with MDS who volunteer to participate in a clinical trial.

Clinic for Myeloid Malignancies Precursor Conditions

The Clinic for Myeloid Malignancies and Precursor Conditions, one of the first clinics of its kind in the world, provides comprehensive care for patients with myeloproliferative neoplasms (MPNs) and myelodysplastic syndrome (MDS). At this clinic, patients meet with a team of specialists who work together to determine the best treatment options.

Our services include:

  • Consultation with a hematologist
  • Lab tests and imaging
  • Bone marrow biopsy, if needed
  • Clinical trials

Improving Care for MDS through Research

If you have MDS, participating in a clinical trial or research study may give you access to treatment options not available outside the clinical trial setting. These options could be brand-new therapies or treatments that have already been approved by the FDA, but that aren’t yet widely used in treating MDS.

Even if there is no chance of your benefiting directly, your participation may provide information that will help others with MDS in the future.