Diseases & Research

Myelodysplastic Syndrome

Hutchinson Center research in stem cell transplantation has led to some of the highest cure rates for myelodysplastic syndrome (MDS) patients. Our researchers are working on new ways to detect and diagnose MDS, and are also trying to better understand how MDS develops and progresses to acute myeloid leukemia (AML). Several clinical trials now underway may lead to new MDS treatments or offer new options to patients who are not candidates for existing therapies.

In 2010 the Hutchinson Center and the Aplastic Anemia & MDS International Foundation hosted a one-day conference on MDS, myeloproliferative disorders (MPD), paroxysmal nocturnal hemoglobinuria (PNH) and aplastic anemia. The event offered insight from leading experts about diagnosis and treatment options for the disease, as well as how patients can optimize emotional and physical health during all phases of the illness.

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Fast Facts

  • Myelodysplastic syndrome, or myelodysplasia, is an umbrella term for several diseases in which the bone marrow does not function normally. Bone marrow is the spongy tissue inside the bones where blood cells are produced.

  • In MDS patients, the production of blood cells is ineffective, in part because blood cells die prematurely in the bone marrow.

  • MDS may progress slowly, over years, or rapidly, over a few months. Patients develop anemia, bruising, bleeding or infections.

  • About one-third of MDS patients will develop acute myeloid leukemia. MDS is more common than AML, with about 9,700 new cases per year in the United States.

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Prevention & Causes

Aging and MDS – Dr. Derek Stirewalt and colleagues are studying the changes that normally occur in blood cells with age as science has observed that some changes associated with aging may also be involved with the development of MDS. Hutchinson Center researchers are also examining how environmental exposures may, over time, promote damage to blood stem cells that can increase the risk of developing MDS.

Cell death – Research led by Drs. Joachim Deeg, A. Mario Marcondes and colleagues focuses on interactions among cells that control leukemic cell death responses in the bone-marrow microenvironment. The team is working to confirm a cause-and-effect relationship between the formation of blood cells and changes in the microenvironment that compromise cell maturation and cell death. By better understanding these processes scientists may develop a new therapeutic approach to treating MDS. Learn more »

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Detection & Diagnosis

Calculating more-accurate MDS incidence rates – Drs. Joachim Deeg, Anneclaire De Roos, and Scott Davis are working to determine the actual incidence rate of MDS through a population-based study that draws from a health maintenance organization's records. Accurate estimates can help manufacturers produce adequate supplies of drugs.The first-of-its-kind study also aims to identify patient characteristics, such as blood cell counts, that help to predict who develops MDS. Those findings will lead to better ways of accurately identifying MDS cases in health care databases, which may lead to further research on risk factors for the disease. Learn more »

Improving MDS diagnosis – The variable nature of MDS has made it difficult to develop a reliable system to classify the disease, which poses a challenge for doctors aiming to predict its course and advise their patients about appropriate treatments. A new approach led by our researchers offers promise for refining MDS classification, which could ultimately improve patient care and outcomes. Researchers have carried out genetic analyses to identify genes that are abnormally expressed in patients with MDS and may be related to the disease's course for a given patient with MDS. They have also carried out studies using flow cytometric analysis—in essence, a computer method that identifies abnormalities in cell structure and the expression of antigens on the cell surface. They recently showed that some patterns are associated with a higher probability and others with a lower probability of being cured of MDS through transplantation. Learn more »

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Treatment & Prognosis

Transplant – Transplant physicians at the Hutchinson Center and its partner, the Seattle Cancer Care Alliance, have reported some of the highest cure rates for patients with MDS. The Center’s pioneering research in bone marrow and stem cell transplantation, which has saved hundreds of thousands of lives, was recognized in 1990 when Dr. E. Donnall Thomas received the Nobel Prize in Physiology or Medicine. Learn more »

Mini-transplant – By minimizing the radiation therapy that patients receive before their bone marrow transplant, Hutchinson Center researchers have helped make this life-saving procedure less toxic and available to more MDS patients. Pioneered by Dr. Rainer Storb and colleagues more than a decade ago, this “mini” transplant has been shown in studies to yield similar results as traditional bone marrow transplants. Learn more »

Radiotherapy – Dr. John Pagel and colleagues are treating patients with targeted radiotherapy that uses molecules called antibodies to carry radiation directly to cells involved with MDS. This promising therapy reduces damage to healthy tissue and is being studied by researchers to learn more about its effectiveness. Our researchers were the first to use a novel combination of radiotherapy, low-intensity chemotherapy and stem cell transplantation to successfully treat high-risk MDS patients.

Drugs – Dr. Joachim Deeg and colleagues are investigating a new drug called treosulfan, a chemotherapy agent that appears less toxic than an existing therapy known as busulfan and may improve survival.

Immunotherapy – Research from Drs. Deeg, Bart Scott and colleagues suggest that abnormal cells in MDS patients release proteins called cytokines that suppress or even kill normal blood cells. This discovery has lead to treatment strategies that block these proteins and modify the patient’s immune system, which improves blood cell counts and helps prevent anemia, infections and bruising.

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Treatment Options

MDS treatment at Seattle Cancer Care Alliance

SCCA combines the expertise and experience of the Hutchinson Center, UW Medicine and Seattle Children's.

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Information about current studies involving new treatments for Myelodysplastic Syndrome.

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