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New Studies on Bone Marrow Failure Diseases
The following studies on bone marrow failure diseases will be presented at the Bone Marrow Failure Scientific Symposium Oct. 18-19, L'Enfant Plaza Hotel in Washington, DC. Organized by the Aplastic Anemia & MDS International Foundation, Inc. (AA&MDSIF), the symposium is sponsored by the National Institutes of Health, including the Office of Rare Diseases, the National Heart, Lung and Blood Institute and the National Cancer Institute.
New Stem Cell Transplantation Technique May Match Donor For Every Patient
A new stem cell technology may greatly expand the donor pool for a variety of malignant and nonmalignant disorders, including bone marrow diseases. By using novel graft engineering technologies, a stem cell donor may be matched for almost every patient that needs one. There is currently only a 25 percent chance of finding a matched donor within the average nuclear family; many ethnic groups have less than a 10 percent chance. The technologies, developed by researchers at St. Jude Children's Research Hospital in Memphis and Children's University Hospital Tuebingen in Germany, allow HLA (genetic markers determining people's tissue types) mismatched and haploidentical stem cell donors into the donor pool and improve the outcome of stem cell transplantation by manipulating the graft according to the patient's underlying disease. Researchers performed either positive stem cell selection strategies or negative depletion of graft-versus-host inducing T-cells. The negative depletion strategy of mobilized peripheral haploidentical stem cells was performed for the first time by the research team.
The Role of Graft Engineering in Transplantation
Rupert Handgretinger, M.D., St. Jude Children's Research Hospital, Memphis, TN
Tuesday, Oct. 18, 8:45 AM
Rates of Aplastic Anemia in Thailand Far Exceed the West
The incidence of aplastic anemia in Thailand is two to three times higher than in the West, according to a new study by researchers at Siriraj Hospital, Mahidol University in Bangkok, Thailand. The new study supports the long-held belief that rates of aplastic anemia are far greater in Asia than in Western countries. An incidence and case-controlled study, partly supported by the National Heart, Lung, and Blood Institute, enrolled 541 patients and 2261 controls in Bangkok and Khonkaen. Some chemicals and drugs, especially exposure to benzene, sulfonamides, thiazides and mebendazole, were associated with aplastic anemia but their use could not account for the greater number of cases. In the countryside, the use of agricultural pesticides, including DDT, organophosphates and carbamates, was a risk factor; other unexpected risk factors were exposure to ducks and geese drinking other than bottled or distilled water—suggestive of an infectious cause for bone marrow failure.
Aplastic Anemia in the Orient
Surapol Issaragrisil, M.D., Siriraj Hospital, Mahidol University, Bangkok, Thailand Wednesday, Oct. 19, 10:30 AM
Genetic Risk Factors Identified For Aplastic Anemia
Researchers at NIH's National Heart, Lung and Blood Institute have identified genetic risk factors for acquired aplastic anemia that affect both the immune response and hematopoietic cell number and function. The class II histocompatability antigen HLA-DR2 is more prevalent in Asian and Western patients and may correlate with responsiveness to immunosuppressive therapies. Alterations in certain cytokine genes were found to be more prevalent among bone marrow failure patients. More recently, genetic studies have been performed of families of patients who had apparently acquired aplastic anemia as adults and lacked the physical abnormalities or a family history typical of inherited forms of bone marrow failure. Multiple pedigrees were defined in whom patients and relatives had mutations in genes called TERC and TERT. These genes are of critical importance in telomere repair: telomeres are the caps at the end of chromosomes that shorten with each cell division. Telomeres and telomere repair are fundamental to cell senescence, to aging in general, and to the development of cancer. These results provide a link between a basic biologic process and the failed bone marrow, and their identification in individual patients profoundly affects treatment and outcome.
Genetic Risk Factors for Bone Marrow Failure
Neal S. Young, M.D., National Heart, Lung and Blood Institute, Bethesda, MD.
Wednesday, Oct. 19, 10:30 AM
The Bone Marrow Failure Scientific Symposium is presenting the latest advances in aplastic anemia, myelodysplastic syndromes (MDS), paroxysmal nocturnal hemoglobinuria (PNH) and other bone marrow failure diseases. This is the first international meeting covering all bone marrow diseases—life-threatening conditions for which, in most cases, the cause is unknown. New research on stem cell transplantation, immunosuppressive treatments, drug therapies and alternative approaches will be presented by the world's leading physicians on bone marrow failure diseases.
More information on the symposium is at AA&MDSIF's web site: www.aamds.org
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Founded in 1983, the Aplastic Anemia & MDS International Foundation, Inc. is dedicated to serving as a resource for patient assistance, advocacy, and support. AA&MDSIF provides patients and their families with educational materials and medical information, and supporting research to find treatments and cures for aplasticanemia (AA), myelodysplastic syndromes (MDS), paroxysmal nocturnal hemogloburina (PNH) and other bone marrow failure diseases.
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