Aplastic Anemia Clinical Trials

The most successful treatment for aplastic anemia is bone marrow transplantation. However, few patients are eligible for this procedure. For others, treatment usually consists of immunosuppressive agents, such as antithymocyte globulin (ATG) and cyclosporine. Unfortunately, even with immunosuppressive therapy, relapse is common. New combinations of medications may offer alternative and ...

Before treatment begins, patients will be evaluated to confirm that they meet the requirements of this study. Participants will need to have a central line. This is a thin plastic catheter or tube that is placed during surgery into one of the large veins in the chest or neck. Central ...

This is a two-part study, interventional Phase II, single-arm, multicenter, open-label study to investigate the efficacy and safety of a combination regimen of eltrombopag and cyclosporine in patients with severe aplastic anemia (SAA) as first line therapy and an extension with up to 60-months follow-up. All patients will receive eltrombopag ...

This is a phase II, open label, multi-center, intra-patient dose escalation study to characterize the pharmacokinetics after oral administration of eltrombopag in combination with immunosuppressive therapy in pediatric patients with previously untreated or relapsed/refractory severe aplastic anemia or recurrent aplastic anemia. All patients will be treated with eltrombopag for the ...

PRIMARY OBJECTIVES: I. To determine the cumulative incidence of extensive chronic graft versus host disease (GVHD) at 1 year after transplantation utilizing the novel conditioning/GVHD prophylactic regimen for patients undergoing allogeneic hematopoietic cell transplantation, in patients who do not progress before day 100. SECONDARY OBJECTIVES: I. To evaluate clinical response, ...

PRIMARY OBJECTIVES: I. To evaluate the rate of relapse, defined as recurrence of underlying disease or progression of underlying disease, at 1 year in patients who receive haploidentical peripheral blood stem cells (PBSCs) after reduced intensity conditioning and post-transplant cyclophosphamide. SECONDARY OBJECTIVES: I. To evaluate safety including development of acute ...

Severe aplastic anemia (SAA) and myelodysplastic syndrome (MDS) are life-threatening bone marrow disorders. For SAA patients, long term survival can be achieved with immunosuppressive treatment. However, of those patients treated with immunosuppressive therapy, one quarter to one third will not respond, and about 50% of responders will relapse. Combined haplo-cord ...

Fludarabine-based preparative regimen followed by an allogeneic hematopoietic stem cell transplant using related or unrelated donor in persons 0-70 years of age diagnosed with dyskeratosis congenita or severe aplastic anemia who have bone marrow failure characterized by a requirement for red blood cell and platelet transfusions. Three different preparative regimens ...

Background A prospective cohort of Inherited Bone Marrow Failure Syndrome (IBMFS) will provide new information regarding cancer rates and types in these disorders. Mutations in IBMFS genes are relevant to carcinogenesis in sporadic cancers. Patients with IBMFS who develop cancer differ in their genetic and/or environmental features from patients with ...

OBJECTIVES Primary To determine the probability of engraftment in patients with Fanconi anemia treated with cyclophosphamide, fludarabine phosphate, and antithymocyte globulin followed by HLA-genotypically identical sibling donor hematopoietic stem cell transplantation that is T-cell depleted. Secondary To evaluate the incidence of acute graft-versus-host disease (GVHD) and chronic GVHD in patients ...