View clinical trials related to Anemia, Aplastic.
Filter by:Severe aplastic anemia (SAA) is a life-threatening blood disease which can be effectively treated with immunosuppressive drug regimens or allogeneic stem cell transplantation. However, 20-40% of patients without transplant options do not respond to immunosuppressive therapies, and have persistent severe cytopenias, requiring regular platelet transfusions, which are expensive and inconvenient, and are a risk for further serious bleeding complications. Thrombopoietin (TPO) is the principal endogenous regulator of platelet production and also stimulates hematopoietic stem and progenitor cells. A small molecule oral TPO-agonist, eltrombopag has been shown to increase platelets in healthy subjects and in patients with immune thrombocytopenic purpura (ITP), and received FDA approval in 2008 for the treatment of thrombocytopenia in ITP. This Phase 2, non-randomized pilot study of eltrombopag in aplastic anemia patients with immunosuppressive therapy refractory thrombocytopenia will test the safety and potential efficacy of eltrombopag treatment patients with refractory thrombocytopenia following immunosuppression for aplastic anemia. Subjects will initiate study medication at an oral dose of 50 mg/day, which will be increased up to 150 mg/day as clinically indicated to the lowest dose that maintains a stable platelet count 20,000/(micro)L above baseline while maximizing tolerability. Response will be assessed at 3-4 months. Platelet response is defined as platelet count increases to 20,000/L above baseline at three months. or stable platelet counts with transfusion independence for a minimum of 8 weeks. Erythroid response for subjects with a pretreatment hemoglobin of less than 9 g/dL will be defined as an increase in hemoglobin by greater than or equal to 1.5g/dL without packed red blood cell (PRBC) transfusion support, or a reduction in the units of transfusions by an absolute number of at least 4 PRBC transfusions for eight consecutive weeks compared with the pretreatment transfusion number in the previous 8 weeks. Neutrophil response will be defined in those with a pretreatment absolute neutrophil count (ANC) of less than 0.5 times 10(9)/L as at least a 100 percent increase or an absolute increase greater than 0.5 times 10(9)/L. Subjects with response at 3-4 months may continue study medication (extended access) until they meet an off study criteria. The primary objective is to assess the safety and efficacy of the oral thrombopoietin receptor agonist (TPO-R agonist) eltrombopag in aplastic anemia patients with immunosuppressive-therapy refractory thrombocytopenia. Secondary objectives include the analysis of the incidence and severity of bleeding episodes, and the impact on quality of life.
To determine the time to and rate of hematologic engraftment following unrelated umbilical cord blood transplantation in adults with one or two cord blood units using total body irradiation and fludarabine as the transplant conditioning regimen and cyclosporine/MMF as graft-versus-host disease prophylaxis.
This clinical trial is studying how well giving fludarabine phosphate and melphalan together with total-body irradiation followed by donor stem cell transplant works in treating patients with hematologic cancer or bone marrow failure disorders. Giving low doses of chemotherapy and total-body irradiation before a donor peripheral blood stem cell transplant helps stop the growth of cancer cells or abnormal cells. It may also stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune cells and help destroy any remaining cancer or abnormal cells (graft-versus-tumor effect)
The goal of this clinical research study is to learn if combining the drugs thymoglobulin, methylprednisolone, cyclosporine, and G-CSF (NeupogenTM or NeulastaTM ) can help to control severe aplastic anemia (AA) or hypoplastic myelodysplastic syndrome (MDS). The safety of this combination therapy will also be studied.
This study will use neuropsychological tests to look at nervous system side effects of Cyclosporine (CsA) in patients with aplastic anemia. CsA is used as part of an immunosuppressive regimen in treating severe aplastic anemia. The drug can produce nervous system side effects, such as tremor and, less commonly, insomnia, anxiety, headache, confusion or seizures. This study will look at effects of CsA on intellectual ability, depression, anxiety, attention, concentration, memory, perception, coordination, and thought processing in patients Patients 15 years of age or older who have severe aplastic anemia may be eligible for this study if they: - are co-enrolled in a Clinical Center protocol in which they will receive CsA - have not taken CsA for 6 months before enrolling in this study Participants undergo neuropsychological testing. In addition, they provide blood samples and their clinical data are reviewed for things that may influence the interpretation of findings from the testing, such as results of blood tests, types of medications taken, number of transfusions required, etc. The procedures are as follows: Before first dose of cyclosporine: - Patients are asked about prior problems with their nervous system, prior treatment for their aplastic anemia (including transfusions), prior infections, and current medications. They then complete the following sets of tests: - Battery 1: A set of three tests that measure intellectual ability, level of depression (if any) and level of anxiety (if any). - Battery 2: A set of seven tests that measure changes in the central nervous system and how these changes affect attention, concentration, memory, perception, coordination, and thought processing. - Patients provide a half teaspoon of blood for this study at the same time blood is collected for their primary treatment protocol. 6 months and 12 months after starting cyclosporine - Patients are asked about treatment for their aplastic anemia (including transfusions), infections, and changes in medications that have occurred since they started taking cyclosporine. They then repeat the set of tests in Battery 2. - Patients provide a half teaspoon of blood for this study at the same time blood is collected for their primary treatment protocol.
The main purpose of this investigational (not approved by the FDA) Phase I research is to test whether transplantation of umbilical cord blood cells can be safely supplemented with a transfusion of a portion of these cells that have been sorted (collected from a special machine called a cell sorter) and then either infused a few hours after the standard transplant or for some patients grown in a special system in the laboratory prior to the transplant, designed to increase the number of stem cells transplanted. This system is currently in the early phases of testing.
Objectives: 1. To evaluate disease free survival after Campath 1H-based in vivo T-cell depletion and non-myelo-ablative ablative stem cell transplantation in patients with hematologic malignancies. 2. To evaluate the incidence and severity of acute and chronic GVHD after Campath 1H-based in vivo T-cell depletion, in patients with hematologic malignancies undergoing non-myelo-ablative stem cell transplantation. 3. To evaluate engraftment and chimerism after Campath 1H-based in vivo T-cell depletion and non-myelo-ablative ablative stem cell transplantation in patients with hematologic malignancies.
This study is designed to determine whether Umbilical Cord Transplantation (UCB) can be substituted for adult bone marrow cells in the standard stem cell transplant regimens used at this hospital for subjects who do not have stem cell donors.
Subjects will be diagnosed with a hematological malignancy (cancer of the blood), which is unlikely to be cured with conventional non-transplant therapy. The best results of bone marrow transplant are obtained with the donor is a relative that has identical tissue type (HLA-type). These subjects will not have such a donor available but they will have a appropriately matching unrelated umbilical cord blood unit (UCB). However, the cord blood unit does not contain a high enough number of cells and may take longer to engraft (or grow). The purpose of this study is to determine whether the addition of stem cells from a family member to supplement a standard unrelated cord blood transplant is safe and will increase the success of the cord blood transplantation procedure. Subjects enrolled in this study will receive an unrelated cord blood transplant plus a haplo-identical (half-matched), T-cell depleted stem transplant from a related donor. The goal of this study is to determine whether the addition of the related stem cells accelerates bone marrow recovery and improves long-term disease free survival.
The purpose of this study is to provide allogeneic stem cell transplantation to patients who have not traditionally undergone this procedure because of it high incidence of treatment related side effects. We hope to decrease these side effects by decreasing the chemotherapy dose prior to transplant (non-myeloablative, smaller dose of chemotherapy given so bone marrow is not completely eliminated) and by using donated stem cells to treat cancer of the blood.