Danazol for Genetic Bone Marrow and Lung Disorders Background: - Some people have bone marrow and lung disorders that are caused by genetic problems. These problems often involve damage to the ends of the chromosomes that pass down genes....

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A Pilot Study of Fludarabine Plus Cyclophosphamide in Refractory Severe Aplastic Anemia Background: - Severe aplastic anemia (SAA) can lead to problems with bone marrow health and result in low blood cell counts, which require frequent transfusions. Standard initial ...

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Alemtuzumab to Treat Severe Aplastic Anemia This study will evaluate the safety and usefulness of a new immunosuppressive drug, alemtuzumab (Campath ), in patients with severe aplastic anemia (SAA). SAA is a rare and serious blood d...

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Horse ATG/CsA in Aplastic Anemia Patients Unresponsive to or With a Suboptimal Response to Rabbit ATG/CsA Treatment Background: - Severe plastic anemia can lead to problems with bone marrow platelet production and result in low blood platelet counts, which require frequent platelet transfusions...

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Sirolimus (Rapamune ) for Relapse Prevention in People With Severe Aplastic Anemia Responsive to Immunosuppressive Therapy Background: People with severe aplastic anemia (SAA) do not make enough red and white blood cells, and/or platelets. Their body s immune system stops the bone marrow from making these cel...

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Rabbit Antithymocyte Globulin Versus Campath-1H for Treating Severe Aplastic Anemia Severe aplastic anemia, characterized by pancytopenia and a hypocellular bone marrow, is effectively treated by immunosuppressive therapy, usually a combination of antithymocyte globulin (...

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Cyclophosphamide Plus Cyclosporine in Treatment-Naive Severe Aplastic Anemia Background: - Severe aplastic anemia (SAA) can lead to problems with bone marrow health and result in low blood cell counts, which require frequent transfusions. Standard treatmen...

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Eltrombopag With Standard Immunosuppression for Severe Aplastic Anemia Background: - Severe aplastic anemia is a rare and serious blood disorder. It happens when the immune system starts to attack the bone marrow cells. This causes the bone marrow to...

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A Pilot Study of a Thrombopoietin-Receptor Agonist, Eltrombopag, in Patients With Low to Int-2 Risk Myelodysplastic Syndrome (MDS) Background: - Myelodysplastic syndromes (MDS) are bone marrow disorders characterized by anemia, neutropenia, and thrombocytopenia (low red blood cell, white blood cell, and plate...

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Three Immunosuppressive Treatment Regimens for Severe Aplastic Anemia Severe aplastic anemia (SAA) is a life-threatening bone marrow failure disorder characterized by pancytopenia and a hypocellular bone marrow. Allogeneic bone marrow transplantation offers ...

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