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Myelodysplastic Syndrome

Myelodysplastic Syndrome Symptoms and Causes

The Hematologic Malignancies Program at the North Shore-LIJ Cancer Institute offers state-of -the-art diagnostic and therapeutic approaches for myelodysplastic syndromes and other cancers of the blood and lymphatic systems. Our cancer experts are world renowned leaders in both the study and frontline treatment of hematologic cancers. They work closely with an interdisciplinary team of specialists to provide compassionate and individualized care using the latest protocols of chemotherapy, immunotherapy and targeted drug therapy.

Our Institute is one of the largest acute leukemia treatment centers in the nation. We have been designated a “Myelodysplastic Syndrome Center of Excellence” by the Myelodysplastic Syndrome Foundation. Our basic science research program for chronic lymphocytic leukemia led by Dr. Nicholas Chiorazzi and clinical therapeutic trials program under the leadership of Dr. Kanti Rai are models in their field and enjoy an international reputation for excellence.

Regional Leaders in Leukemia and Stem Cell Transplants

Our dedicated leukemia and stem cell transplant centers are among the largest in the New York area. Our Adult and Pediatric Bone Marrow and Stem Cell Transplant Programs are the only transplant programs in the Long Island, Queens, and Brooklyn area accredited by the prestigious Foundation for Accreditation in Cellular Therapy (FACT) for exceptional patient care and medical practices.

World Leaders in Chronic Lymphocytic Leukemia Treatment

Our Chronic Lymphocytic Leukemia Research and Treatment Program has become a world-renowned landmark for patients with chronic lymphocytic leukemia (CLL), one of the most common forms of leukemia in adults.

Myelodysplastic Syndromes: An Overview

Myelodysplastic syndromes are diseases of the blood and bone marrow. Normally, the bone marrow makes blood stem cells (immature cells) that develop into mature blood cells over time. A blood stem cell may become a myeloid stem cell or a lymphoid stem cell. The lymphoid stem cell develops into a white blood cell. The myeloid stem cell develops into one of three types of mature blood cells:

  • Red blood cells that carry oxygen and other materials to all tissues of the body
  • White blood cells that fight infection and disease
  • Platelets that help prevent bleeding by causing blood clots to form

In myelodysplastic syndromes, the blood stem cells do not mature into healthy red blood cells, white blood cell or platelets. The immature blood cells, called blasts, do not function normally and either die in the bone marrow or soon after they enter the blood. This leaves less room for healthy white blood cells, red blood cells and platelets to develop in the bone marrow. When there are fewer blood cells, infection, anemia or easy bleeding may occur.There are several types of myelodysplastic syndromes.

Myelodysplastic syndromes have too few of one or more types of healthy blood cells in the bone marrow or blood. Myelodysplastic syndromes include the following diseases:

  • Refractory anemia
  • Refractory anemia with ringed sideroblasts
  • Refractory anemia with excess blasts
  • Refractory anemia with excess blasts in transformation
  • Refractory cytopenia with multilineage dysplasia
  • Myelodysplastic syndrome associated with an isolated del(5q) chromosome abnormality
  • Unclassifiable myelodysplastic syndrome

Different types of treatments are available for patients with myelodysplastic syndromes. 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 for patients with a myelodysplastic syndrome. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Patients may want to think about taking part in a clinical trial. Some clinical trials are open only to patients who have not started treatment.

Treatment for myelodysplastic syndromes aims to relieve symptoms, slow progression and improve quality of life.

Treatment options for patients with myelodysplastic syndromes range from supportive care that helps relieve symptoms to aggressive treatment that may slow or prevent progression of the disease.

Problems caused by low blood cell counts, such as fatigue and infections, may be treated with transfusions of blood products or the use of growth factors.

Chemotherapy may be used to delay progression of the disease. Other drug therapy may be used to lessen the need for transfusions. Certain patients may benefit from aggressive treatment with chemotherapy followed by stem cell transplant using stem cells from a donor.

Three types of standard treatment are used:

  • Chemotherapy – In myelodysplastic syndromes, chemotherapy is a treatment that uses drugs to stop the growth of immature blood cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cancer cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly into the cerebrospinal fluid, an organ or a body cavity such as the abdomen, the drugs mainly affect cancer cells in those areas (regional chemotherapy). The way the chemotherapy is given depends on the disease being treated.
  • 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 – Transfusion therapy (blood transfusion) is a method of giving red blood cells, white blood cells, or platelets to replace blood cells destroyed by disease or treatment. Patients who receive frequent red blood cell transfusions may have their tissues and organs damaged from the buildup of extra iron. Iron chelation therapy is a treatment that uses drugs that attach to the extra iron. The drug and the iron are removed from the body in the urine. Platelet transfusions are usually given when the patient is bleeding or is having a procedure that may cause bleeding.
    • 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 enhance the effects of the treatment.
    • Drug therapy
      • Deferoxamine may be used to treat the build-up of too much iron in the blood of patients receiving blood transfusions. It is sometimes given with vitamin C.
      • Lenalidomide may be used to lessen the need for transfusions in patients who have myelodysplastic syndrome caused by a specific chromosome change.
      • Antithymocyte globulin (ATG) may also be used to lessen the need for transfusions in patients with a certain form of myelodysplastic syndrome.
      • Antibiotics may be given to fight infections.
  • Chemotherapy with stem cell transplant – Stem cell transplant is a method of giving chemotherapy and replacing blood-forming cells destroyed by the treatment. Stem cells (immature blood cells) are removed from the blood or bone marrow of a donor and are frozen for storage. After the chemotherapy is completed, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells.

(Source: American Cancer Society)

Myelodysplastic Syndromes Clinical Trials

The North Shore-LIJ Cancer Institute offers a full array of clinical trials. The result of this research not only impacts survival, but also enhances the quality of life. For more information about clinical trials for Myelodysplastic Syndrome, visit Cancer Clinical Trials.

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