High-Tc Susceptometer to Monitor Transfusional Iron Overload
Transfusional Iron OverloadThalassemia Major3 moreThe proposed research project will continue the application and development of a new method (biomagnetic susceptometry) that measures magnetic fields to determine how much iron is in the liver. The amount of iron in the liver is the best indicator of the amount of iron in the whole body. Measuring the amount of iron in the body is important because either too much (iron overload) or too little iron (iron deficiency) can be harmful. At present, the most reliable way to measure the amount of iron in the liver is to remove a sample of the liver by biopsy, either by surgery or by using a needle which pierces the skin and liver. Iron stored in the liver can be magnetized to a small degree when placed in a magnetic field. In patients with iron overload, the investigators previous studies have shown that magnetic measurements of liver iron in patients with iron overload are quantitatively equivalent to biochemical determinations on tissue obtained by biopsy. In the past the investigators have developed a device to measure the amount of magnetization, which was called a SQUID (Superconducting QUantum Interference Device) susceptometer. This device was validated and in use for over 20 years. The safety, ease, rapidity and comfort of magnetic measurements make frequent, serial studies technically feasible and practically acceptable to patients. The investigators have now developed a new susceptometer, which uses very similar technology to the SQUID, but the investigators believe is more accurate and precise. This study aims to validate this new instrument. The investigators will do prospective, serial studies of the diagnosis and management of patients with iron overload, including thalassemia major (Cooley's anemia), sickle cell disease, aplastic anemia, myelodysplasia, hereditary hemochromatosis, and other disorders. Funding Source - FDA OOPD.
Effectiveness and Safety of MMSCs for Enhancing Hematopoietic Recovery and Prophylaxis of Neutropenic...
Neutropenic EnterocolitisMyeloablative Chemotherapy Induced Bone Marrow AplasiaSubjects will undergo peripheral blood stem cell mobilisation and collection with subsequent high-dose chemotherapy. After finalization of high-dose chemotherapy subjects will receive bone marrow derived allogeneic multipotent mesenchymal stromal cells intravenous infusion two hours prior to autologous peripheral blood cells infusion. This is a single arm study with no control. All patients receive cell therapy.
Extended Platelet Parameters as a Means to Differentiate Immune Thrombocytopenia From Hypo-proliferative...
Immune ThrombocytopeniaChemotherapy Induced Thrombocytopenia2 moreTo utilise extended platelet parameters in order to individuate Immune Thrombocytopenia (ITP) from hypo-proliferative causes of thrombocytopenia. To develop the clinical potential of the extended platelet parameters as they pertain to distinguishing different causes of thrombocytopenia from one another. To test the hypothesis that mean platelet component (MPC) and mean platelet mass (MPM) might distinguish between thrombocytopenia related to bone marrow dysfunction and immune mediated destruction of platelets.
Electronic Patient-reported Outcome Monitoring in Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria...
Aplastic AnemiaParoxysmal Nocturnal HemoglobinuriaAplastic anemia (AA) and paroxysmal nocturnal hemoglobinuria (PNH) are interrelated and very rare diseases. Therefore, little data about clinical characteristics, especially the variety of symptoms in the course of the respective disease are available. As a consequence, patients may be left on their own between infrequent follow-ups at a specialist center. A web-based symptom-monitoring application can support selfmanagement and patient empowerment and promotes a patient- centered interdisciplinary team approach in the context of a "disease management program". This pilot study is to investigate usability and feasibility of the electronic Patient-Reported Outcome (ePRO) application in AA/PNH by assessing recruitment, app utilization, data collection, functionality, acceptability after using and working with the ePRO application.
Drug Etiology of Aplastic Anemia and Related Dyscrasias
Blood DiseaseAnemia4 moreTo determine the role of drugs in the etiology of aplastic anemia, agranulocytosis, and thrombocytopenic purpura. Drugs used in chemotherapy and immunotherapy were excluded.
Multi Center Case Control Study on Multiple Risk Factors of Aplastic Anemia
Aplastic AnemiaTo ascertain the risk factors of AA in ZheJiang Province of China at the moment, especially the comprehensive multiple factors risks. The author conducted a case-control research included 338 AA cases and 1464 controls. Subjects were surveyed using same standard questionnaire including personal data, past histories of diseases, family history and histories of occupational and daily exposure to harmful substances. Single and multiple Logistic regression analyses were made using SPSS17.0 on the dates to study potential factors in the development of AA.
Investigation of the Cylex® ImmuKnow® Assay
LeukemiaNon-Hodgkin's Lymphoma7 moreCurrently, there is no accurate way of predicting the occurrence of Graft vs Host Disease (GvHD) or infection. The purpose of this study is to analyze blood with the ImmuKnow® Assay to see if doctors can detect which patients are at risk for GvHD and for getting an infection before they occur.
Non-Myeloablative Allogeneic Stem Cell Transplantation With Matched Unrelated Donors for Treatment...
Acute Myeloid LeukemiaMyelodysplasia9 moreThe primary objective of this study is to examine transplant related mortality (TRM) at 100 days <30%. A TRM of >50% is considered unacceptable. This study also seeks a TRM at 12 months that is <50%, engraftment >90% (defined as donor cells >80% at 6 months), and 1 year overall survival >50%.
Protection Against Benzene Toxicity
LeukemiaAplastic AnemiaWe are investigating the role of an enzyme (NQO1) in protection against the bone marrow toxicity of the occupational and environmental toxicant benzene. All of the proposed studies involve use of human bone marrow cells in-vitro to define mechanisms of NQO1-mediated protection. Cells are obtained from healthy volunteers and protocols have undergone IRB review and approval.
A Description of Bacteria in the Mouths of Patients With Severe Aplastic Anemia
Severe Aplastic Anemia (SAA)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.