Mariah
Osteosarcoma Survivor
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Josh
Brain Tumor Survivor
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Sydney
Leukemia Survivor
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Alijah
Leukemia Survivor
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Rosie
Wilms Tumor Survivor
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Ryan
Leukemia Survivor
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Sydney
Retinoblastoma Survivor
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Myeloma Myelodysplastic Syndromes (MDS)

A disease that may lead to leukemia

Myelodysplastic syndrome (MDS) is a label for several similar cancers of the blood and bone marrow. MDS is caused by problems in the immature blood cells known as myeloid cells. In a healthy body, any blood stem cells that mature into myeloid cells will continue their development by becoming either white blood cells, red bloods cells, or platelets. But the myeloid cells in a patient with MDS are abnormal, and can’t develop correctly. These useless cells, or blasts, take up so much room that the levels of normal blood cells drop dangerously.

Quick facts

  • The average MDS patient is around 75 years old at diagnosis, although it can occur at any age.
  • In about one-third of patients, MDS progresses into acute myelogenous leukemia (AML).
  • Blood or bone marrow transplant (BMT, also known as a stem cell transplant) is the only known cure for MDS.

Symptoms

MDS may cause symptoms, but in many cases it does not, and is discovered following a routine blood test. When MDS causes symptoms, they include:

  • Shortness of breath.
  • Weakness or feeling tired.
  • Having skin that is paler than usual.
  • Easy bruising or bleeding.
  • Petechiae (flat, pinpoint spots under the skin caused by bleeding).
  • Fever or frequent infections.

Diagnosis

If there is reason to suspect your child has MDS, the doctor will order tests to gather more information. The tests will include:

  • Blood test: This consists of withdrawing blood from a child’s arm by needle — the kind of test most children have had at least once by the time they are five years old in a routine physical exam. The blood sample is then magnified so the cells can be identified and counted.
  • Bone marrow aspiration and biopsy: For this procedure, the child is sedated and given a numbing shot in the hip area. Thin, hollow needles are inserted into a bone in the hip and a small amount of marrow, blood, and bone is removed. The sample is examined under the microscope to determine the kinds of cells present, their developmental stage, and their population strength.

There are several different types of MDS. They are categorized based on certain changes in the blood cells and bone marrow. The diseases include:

  • Refractory anemia: There are too few red blood cells in the blood and the patient has anemia. The number of white blood cells and platelets is normal.
  • Refractory anemia with ringed sideroblasts: There are too few red blood cells in the blood and the patient has anemia. The red blood cells have too much iron. The number of white blood cells and platelets is normal.
  • Refractory anemia with excess blasts: There are too few red blood cells in the blood and the patient has anemia. Five percent to 19% of the cells in the bone marrow are blasts and there are a normal number of blasts found in the blood. There also may be changes to the white blood cells and platelets. Refractory anemia with excess blasts may progress to acute myeloid leukemia (AML).
  • Refractory anemia with excess blasts in transformation: There are too few red blood cells, white blood cells, and platelets in the blood and the patient has anemia. Twenty percent to 30% of the cells in the bone marrow are blasts and more than 5% of the cells in the blood are blasts. Refractory anemia with excess blasts in transformation is sometimes called acute myeloid leukemia (AML).
  • Refractory cytopenia with multilineage dysplasia: There are too few of at least two types of blood cells. Less than 5% of the cells in the bone marrow are blasts and less than 1% of the cells in the blood are blasts. If red blood cells are affected, they may have extra iron. Refractory cytopenia may progress to acute leukemia.
  • Myelodysplastic syndrome associated with an isolated del(5q) chromosome abnormality: There are too few red blood cells in the blood and the patient has anemia. Less than 5% of the cells in the bone marrow and blood are blasts. There is a specific change in the chromosome.
  • Unclassifiable myelodysplastic syndrome: There are too few of one type of blood cell in the blood. The number of blasts in the bone marrow and blood is normal, and the disease is not one of the other myelodysplastic syndromes.

Stages

To describe the scope of most cancers, doctors use a “staging system,” assigning each case of cancer to a particular “stage” based on how far it has spread. There are specific treatment plans to follow for each different stage. But for MDS, there is no staging system. Cases of MDS are still sorted into groups, but the groups are based on how the patient developed the disease.  MDS is grouped for treatment as follows:

  • De novo myelodysplastic syndrome develops without any known cause.
  • Secondary myelodysplastic syndrome develops after the patient was treated with chemotherapy or radiation therapy for other diseases, or after being exposed to radiation or certain chemicals that are linked to the development of MDS. Secondary MDS may be harder to treat than de novo MDS.
  • Previously treated myelodysplastic syndrome has been treated before but has not gotten better.

Treatment

Different types of treatments are available for patients with MDS. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments.

Three types of standard treatment are used:

  • Chemotherapy: For this treatment, the patient is given strong drugs that fight cancer by either killing the cells or stopping their growth. The chemo may be taken by mouth, or it may be given in an injection.

     

  • Supportive care: Supportive care is given to lessen the problems caused by the disease or its treatment. Supportive care may include the following:

    • Transfusion therapy: Red blood cells, white blood cells, or platelets destroyed by disease or treatment are replaced in this therapy with a blood transfusion. Patients who experience a buildup of extra iron from frequent transfusions may be given iron chelation therapy.
    • Growth factor therapy: Erythropoietin may be given to increase the number of red blood cells and lessen the effects of anemia. Sometimes granulocyte colony-stimulating factor (G-CSF) is given with erythropoietin to help the treatment work better.
    • Drug therapy: To lesson the need for transfusions and treat iron build-up, patients may be given drugs including deferoxamine, lenalidomide, antithymocyte globulin (ATG), or antibiotics.

     

  • Chemotherapy with blood or bone marrow transplant (BMT, also known as stem cell transplant) For this treatment, stem cells are removed from the blood or bone marrow of a donor and are frozen for storage. The patient gets chemo to destroy cancerous cells. After the chemotherapy is completed, in order to replenish healthy blood cells, the stored stem cells are thawed and given back to the patient through an infusion.

Treatment by type of MDS

Treatment of de novo and secondary myelodysplastic syndromes may include the following:

  • Supportive care with transfusion therapy.
  • High-dose chemo with BMT using stem cells from a donor.
  • Supportive care with growth factor therapy.
  • Chemotherapy with azacitidine, decitabine, or other anticancer drugs.
  • Supportive care with drug therapy.
  • A clinical trial of a new anticancer drug.
  • A clinical trial of low-dose chemo with BMT using stem cells from a donor.
  • A clinical trial of a combination of treatments.
  • A clinical trial of growth factor therapy.

Treatment of previously treated myelodysplastic syndromes may include the following:

  • High-dose chemo with BMT using stem cells from a donor.
  • Chemo with azacitidine or decitabine.
  • Supportive care with transfusion therapy, growth factor therapy, and/or drug therapy.
  • A clinical trial of low-dose chemo with BMT using stem cells from a donor.
  • A clinical trial of new drug therapy.
  • A clinical trial of a combination of treatments.
  • A clinical trial of growth factor therapy.

If the MDS progresses to acute myeloid leukemia (AML), treatment will be the same as treatment for the newly diagnosed patient with AML.

Research leads the way

The Masonic Cancer Center at the University of Minnesota has a strong national reputation for its members’ research on the causes and prevention of cancer. In the Center’s Prevention and Etiology Research Program, scientists work collaboratively to identify risk factors, evaluate strategies for early detection, develop and test approaches for prevention, and improve life for survivors.

Children’s Cancer Research Fund’s relationship with the University of Minnesota supports this work. The National Cancer Institute has awarded a $2.5 million grant to Masonic Cancer Center scientist and Children’s Cancer Research Fund’s Chief Medical Advisor Julie Ross, Ph.D. Dr. Ross is leading the AIMMS study, a state-wide epidemiology study aiming to identify environmental, lifestyle, and genetic factors that may predispose a person to MDS. The doctors and researchers hope to answer crucial questions about this rare disease, making an important contribution to the study of MDS.

For more information about how our researchers are leading the way to a cure, visit our Leukemia Research page.

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