Focal Accumulation of Iron in Cerebral Regions in Early ALS (Amyotrophic Lateral Sclerosis) Patients...
ALS (Amyotrophic Lateral Sclerosis)Iron OverloadThe FAIR-ALS study is to investigate the safety and efficacy of a scavenger treatment of iron deferiprone, which would reduce the brain iron to limit the development of amyotrophic lateral sclerosis. It has been shown an excess of iron in the central nervous system carrying a sporadic ALS patients. Iron overload associated with a loss of motor neurons may explain the signs of the disease (atrophy). The investigators discuss the hypothesis that reducing excess iron, the investigators can reduce the loss of neurons and thus the progression of signs of the disease.
An Epidemiological Study to Assess Iron Overload Using MRI in Patients With Transfusional Siderosis...
ThalassemiaNon-transfusional-dependent Thalassemia (NTDT)2 moreIron, one of the most common elements in nature and the most abundant transition metal in the body, is readily capable of accepting and donating electrons. This capability makes iron a useful component of various, essential biochemical processes. Despite the essential role of iron, the excess of iron is toxic to the human body. It is critical for the human body to maintain iron balance, since humans have no physiologic mechanism for actively removing iron from the body. The development of iron overload occurs when iron intake exceeds the body's capacity to safely store the iron in the liver, which is the primary store for iron. Long-term transfusion therapy, a life-giving treatment for patients with intractable chronic anemia is currently the most frequent cause of secondary iron overload. The mounting evidence regarding the mortality and morbidity due to chronic iron overload in transfusion dependent anaemias has led to the establishment of guidelines that aim the improvement of patient outcomes. Further prospective studies are warranted in order to assess the impact of iron overload in patients with acquired anaemias. In this study, non-invasive R2- and T2*-MRI techniques were applied to the liver and the heart, respectively, to complement the primary variable (serum ferritin) assessed in patients with various transfusion-dependent anaemias. The main objective of this study was to assess the prevalence and severity of cardiac and liver siderosis in patients with transfusional siderosis. This study was also aim to establish possible correlations between cardiac and liver iron levels with clinical effects in patients with different transfusion-dependent anaemias. Patients were eligible for enrollment irrespective of receiving chelation therapy or not (and irrespective of the chelating agent used).
Long-term Safety and Efficacy of Ferriprox® in Iron Overloaded Patients With Sickle Cell Disease...
Iron OverloadSickle Cell Disease1 moreThis is a long-term follow-up to an earlier study, LA38-0411. Its purpose is to gather more information about the safety and efficacy of deferiprone in patients with sickle cell disease or other anemias who suffer from iron overload caused by regular blood transfusions.
Validation of a Point-of-care Device Measuring Ferritin With Capillary Blood
AnemiaIron Deficiency2 moreMeasurement of iron is important for identifying both low and high iron levels in the body. Measuring a protein in the blood called ferritin is currently the easiest way to determine a patient's iron status. However, the test requires a blood sample that is be sent to the laboratory for measurement, which can cause delays in obtaining the result. The investigators are testing whether a point of care device that measures ferritin levels using a capillary blood sample is accurate compared to current methods of testing. This could allow measurement of ferritin immediately in the clinic with only a drop of blood, and enable treatment decisions based on these results.
Study Of Efficacy,Safety of Combined Deferasirox and Deferiprone Versus Combined Deferiprone and...
Beta-thalassemia MajorSickle Cell Disease1 moreInterventional Allocation: Randomized Endpoint Classification: Safety/Efficacy Study of combined chelation therapy Masking: Open Label Primary Purpose: Treatment of transfusional iron overload Primary Outcome Measures: • The primary outcome measure is to assess efficacy in lowering serum ferritin level(the change in serum ferritin compared to baseline) with combining DFP and deferasirox compared to combined DFP and DFO in conditions with severe chronic iron overload; showing an up-trend of SF over previous 12 months on single chelator. Secondary Outcome Measures: • The secondary outcome measure is to determine the number of patients who will develop adverse events in order to assess safety upon administering the drugs in combination (DFP and DFX) compared to the combination of DFO and DFP.
The Iron Content of Ferritin in Serum and Urine of Children With High Serum Ferritin Levels
Iron OverloadA serum ferritin level can reflect the total body iron content, thus a very low serum ferritin is commonly used as an indicator of iron deficiency and a very high serum ferritin is commonly used as a marker of iron overload. Ferritin is a shell of protein in which iron is stored. Ferritin is an acute phase reactant, and serum ferritin levels can increase during inflammatory conditions. Consequently, an elevated ferritin level might mean there is an excess of storage iron, or might simply mean that inflammation has resulted in high levels of the ferritin shell, containing little iron. The research team is able to quantify the amount of iron in ferritin using inductively conducted plasma mass spectrometry, in the Heme and Iron Core Laboratory at the University of Utah. Thus, it can be determined whether in a child with a very high serum ferritin level, that ferritin is loaded with iron or is actually very low in iron. Neonates and young children with certain liver disorders characteristically have a very high serum ferritin level. These conditions are gestational alloimmune liver disease (GALD) and hemophagocytic lymphohistiocytosis (HLH). It is not clear what the iron content of the ferritin is in these neonates. Knowing this will be a step toward understanding whether the pathogenesis of these conditions involves iron overload. Additionally, if urine ferritin and iron levels correlate with serum ferritin and iron levels, urine may be used as a non-invasive way to monitor iron status. In this study, serum and urine samples will be collected from children with high serum ferritin levels and confirmed iron toxicity. Both ferritin and iron content within ferritin will be measured in the serum and urine samples and compared for correlation.
Therapeutic Effects of Silymarin in Patients With B-thalassemia Major
Beta-thalassemia MajorIron OverloadSilymarin, a flavonolignan complex isolated from Silybum marianum, has a strong antioxidant, hepatoprotective and iron chelating activities. The present study has been designed to investigate the therapeutic activity of orally administered silymarin in patients with thalassemia major under conventional iron chelation therapy. A 6-month randomized, double-blind, clinical trial was conducted in 140 beta-thalassemia major patients in two well-matched groups. Patients are randomized to receive a silymarin tablet (140 mg) three times a day plus conventional desferrioxamine therapy or the same therapy but a placebo tablet instead of silymarin. Clinical laboratory tests of iron status and liver function are assessed at the beginning and the end of the trial.
Amlodipine as Adjuvant Treatment to Iron Chelation for Prevention of Cardiac Iron Overload in Thalassemia...
Beta-ThalassemiaIron OverloadThis is a randomized, open label, two arms superiority trial of a representative population of patients with a primary diagnosis of transfusion dependent thalassemia with evidence of moderate cardiac iron overload, defined as an average T2* MRI parameter at the mid inter-ventricular septum between 10 and 20ms.
Massive Iron Deposit Assessment
Iron OverloadExcessive Body Iron BurdenIron overload is a severe complication of multiple blood transfusions. As the body has no physiologic mechanism for clearing iron, repeated transfusions cause iron accumulation in organs and lead to iron toxicity. Accurate assessment of iron overload is paramount to quantify excessive iron accumulation and to monitor response to iron chelation therapy. Magnetic resonance imaging (MRI) methods have been used to noninvasively measure hepatic iron concentration (HIC). Although MRI-based measurements of transverse relaxation rates (R2 and R2*) accurately predict biopsy-proven HICs below 15 mg Fe/g, previous studies have shown that their precision is limited for HICs above 15 mg Fe/g and inaccurate above 25 mg Fe/g. Current R2* gradient-echo (GRE) MR techniques fail occasionally for very high iron overloads (HIC ~ 15-25 mg Fe/g) and always for massive iron overloads (HIC > 25 mg Fe/g) because R2* is so high that the MR signal decays before it can be measured accurately. Overall accrual: 200 patients Purpose: To determine if a new MRI (UTE) can measure the amount of iron in the liver of people with large amounts of iron and compare the results with the same patient's liver bx. Estimated patient accrual is 150. It is estimated that 41 of these patients will have clinical indication for liver biopsy.
Quantification of Liver Iron Overload and Steatosis Using Magnetic Resonance Imaging
Iron OverloadMetabolic Syndrome X3 moreIron excess is increasingly regarded as an important cofactor in the morbidity attributed to many disorders. Assessment of body iron stores by measurement of serum ferritin concentrations has poor specificity and the most reliable method is histological or biochemical assessment from a liver biopsy. Because liver biopsy is an invasive procedure, imaging methods have been developed to detect and quantify hepatic iron content. The aim of the study is to use a simplified magnetic resonance imaging (MRI) technique to quantify simultaneously iron and fat contents in the liver and to compare the results to the quantification obtained biochemically.