View clinical trials related to Severe Aplastic Anemia (SAA).
Filter by:Background: - This research is being done to describe the types of bacteria found in the mouths of patients who have severe aplastic anemia (SAA) and are treated with drugs that suppress the immune system or with stem cell transplant. People with SAA who receive these treatments are more likely to get infections. Studies show that there might be a link between the bacteria in your mouth and those bacteria that can cause infections. The bacteria found in the mouths of patients with SAA will be described. Objectives: - To understand the changes in mouth bacteria that are related to treatment and to describe the oral bacterial environment. Eligibility: - Adults at least 18 years of age who are going to be treated for SAA. - Healthy volunteers at least 18 years of age. Design: - Participants will answer questions about their medical history and dental care. Their mouths will be examined. - Participants with SAA will be tested during treatment for their disease, over the course of 1 year. All participants with SAA will be tested at 3 scheduled appointments. Any participants who require a breathing tube will receive additional tests. - Healthy volunteers will be tested during 1 visit. - Participants will give two samples each time. A saliva sample will be taken with a disposable padded tool. Skin cells will be collected from the tongue with a small plastic brush.
Background: - Eltrombopag is a drug being tested for treating severe aplastic anemia. It can help improve blood counts in these patients. However, researchers do not know how long the drug can and should be taken for this type of anemia. Objectives: - To look at whether 6 months of treatment with eltrombopag can improve patient s blood counts. Eligibility: - Individuals at least 2 years of age who are taking eltrombopag for severe aplastic anemia. Design: - Participants will take eltrombopag by mouth once a day for 6 months. - Blood samples will be collected every 2 weeks for the first 6 months. Bone marrow samples will be collected at 3 and 6 months. These samples will look at the effects of the study drug on the marrow. - Participants will continue to take the study drug for as long as it is effective and if the side effects are not severe.
This study will evaluate the safety and effectiveness of treating patients with severe aplastic anemia (SAA) or myelodysplastic syndrome (MDS) with both peripheral blood stem cells from a family member and umbilical cord blood stem cells from an unrelated donor. Patients with SAA or MDS for whom other treatments have failed or are not available may be eligible for this study. Candidates may not have a tissue-matched sibling or matched unrelated donor and must have a family member who is a partial tissue type match. Participants undergo the following tests and procedures: - Insertion of a central intravenous (IV) line (plastic tube) into a large vein. The tube is used for giving the donated stem cells and antibiotics and other medicines, for transfusions of red blood cells and platelets, and for collecting blood samples. - Preparatory chemotherapy (fludarabine, cyclophosphamide and anti-thymocyte globulin) and total body irradiation to suppress immunity and prevent rejection of the donated cells. - Infusion of the donated stem cells and umbilical cord cells. - Immune suppression with the drugs tacrolimus, mycophenolate mofetil and prednisone to prevent rejection of the donated cells and to prevent graft-versus-host disease (GVHD), a complication of stem cell transplants in which the donors immune cells destroy the patients healthy tissues. The average hospital stay after stem cell transplantation is 3 to 4 weeks. Patients return for frequent follow-up visits for the first 2 to 4 months after transplantation. Once the patient returns home, his or her referring physician is asked to send results of any laboratory testing to the NIH researchers at least every 3 months for the first 3 years and annually thereafter. Patient follow-up visits are scheduled at NIH at 1, 2, 3, 4 and 5 years after transplantation to monitor for signs of disease or post-transplantation complications, such as infection or GVHD. After 5 years, participants are offered the opportunity to enroll in NHLBIs long-term evaluation and follow-up care protocol.
Severe Aplastic Anemia (SAA) is a rare and very serious blood disorder in which the bone marrow stops producing the cells which make up blood; red blood cells, white blood cells, and platelets. Researchers believe this is caused by an autoimmune reaction, a condition in which the natural defense system of the body begins attacking itself. In SAA the immune system begins attacking the bone marrow. Red blood cells are responsible for carrying oxygen to all of the organ systems in the body, and low numbers (anemia) can cause difficulty breathing and fatigue. Platelets are responsible for normal blood clotting and low numbers can result in easy bruising and bleeding which can be deadly. White blood cells are responsible for fighting infections, and low numbers of these can lead to frequent infections, the most common cause of death in patients with aplastic anemia. SAA can be treated by bone marrow transplant (BMT) or by drugs designed to slow down the immune system (immunosuppressants). BMT can be successful, but it requires a donor with matched bone marrow, making this therapy available only to a few patients. BMT with unmatched bone marrow can fail and cause dangerous side effects. Presently, the two drugs used to treat SAA by slowing down the immune system (immunosuppression) are antithymocyte globulin (ATG) and cyclosporin A (CSA). When used in combination these two drugs can improve most patients condition. However, one third of the patients who respond to this therapy experience a relapse of SAA. In addition, some patients treated with ATG/CSA can later develop other disorders of the blood. Recently, researchers have found that another immunosuppressive drug called cyclophosphamide, has been successful at treating patients with SAA. In addition, patients treated with cyclophosphamide do not experience relapses or develop other disorders of the blood. In this study researchers would like to compare the combinations of antithymocyte globulin (ATG) and cyclosporin A (CSA) to cyclophosphamide and cyclosporin A (CSA) for the treatment of SAA.