Active clinical trials for "Anemia, Sickle Cell"

The purpose of this study is to initiate pilot studies to demonstrate that a sufficient number of iron-overloaded thalassemia, SCD and DBA populations with similar duration of chronic transfusion, and age at start of transfusions would be available for a confirmatory study and to validate that proposed multicenter MRI and biochemical studies can be completed. The study will examine the hypothesis that a chronic inflammatory state in Sickle Cell Disease (SCD) leads to hepcidin- and cytokine-mediated iron withholding within the RES (reticuloendothelial system), lower plasma NTBI (non transferrin bound iron) levels, less distribution of iron to the heart in SCD.

Pulmonary hypertension (PH) at rest is a risk factor for death in patients with sickle-cell anemia (SCA). Exercise echocardiography (EE) can detect latent PH. We sought to investigate the occurrence of exercise-induced PH in patients with SCA and normal pulmonary pressure (PP) at rest, and its relationship with clinical and echocardiographic variables.Forty-four patients with SCA and normal PP at rest were studied and divided into two groups: exhibiting normal PP after treadmill EE (TRV≤2.7m/s) (G1), and exhibiting exercise-induced PH (TRV>2.7m/s) (G2). TRV cutoff points at rest and during exercise were based on data from healthy control subjects, matched for age, sex, and body surface area. Data obtained from EE were correlated with clinical, echocardiographic and ergometric variables.Exercise-induced PH occurred in 57% of the sample (G2), significantly higher than those of G1. Exercise-induced PH was related to higher levels of creatinine (p<0.05), increased left atrial volume (p<0.05) and right ventricular diastolic area (p<0.05), larger E/Em waves ratio derived from spectral and tissue Doppler (p<0.05), and higher TRV at rest (p<0.005).We concluded that patients with SCA and normal PP at rest may exhibit exercise-induced PH, which was related to renal function, increased cardiac chambers, abnormal indices of diastolic function and baseline TRV levels.

To determine the natural history of sickle cell disease from birth to death in order to identify those factors contributing to the morbidity and mortality of the disease.

To conduct a prospective study aimed at the early detection and treatment of cerebral vascular disease prior to irreversible brain injury in young children with sickle cell anemia (SCA).

Stroke is a frequent complication of sickle cell disease (SCD), with varying levels of central nervous system (CNS) involvement. The summation of several ischemic events, even when silent, can lead to devastating consequences, from reduced academic performance to physical dependence. Despite knowledge that brain flow velocities evaluated by Doppler ultrasound identify pediatric SCD patients at a greater stroke risk (Adams et al, NEJM 1998; 339:5-11), this method is not able to predict the occurrence of strokes in adults. There is also no consensus on the management of adult patients in relation to primary and secondary prevention. The aim of this study is to evaluate the effects of the administration of Simvastatin on CNS structural and functional vascular changes in 30 adult patients with SCD (SS and Sβ), above 35 years of age, observed through Magnetic Resonance Imaging (MRI). The data on the effect of simvastatin on disease manifestations is quite scarce, however this drug reportedly significantly reduces plasma concentrations of adhesion molecules and inflammatory markers, such as E-selectin, VEGF, CRP and IL-6 (Hoppe et al, BJH 2011; 153:655-663; Hoppe et al, BJH 2017;177:620-629). Thus, in addition to the search for early diagnostic markers and risk stratification for primary or recurrent stroke, we will also compare CNS images before and 12 months after the administration of Simvastatin. The drug alter stroke recurrence rates in the general adult population, but their effects on vascular changes in patients with SCD have not yet been adequately elucidated. This is particularly important because these are low cost drugs which present good tolerability, and could be part of the therapeutic arsenal of SCD, even in low income settings. Concomitantly with the CNS evaluation, this study also intends to investigate molecular pathways that may be affected by the drugs. We will evaluate microvesicle release patterns, as well as the content of microRNAs possibly involved in the occurrence of stroke, in addition to metabolomic studies and plasma cytokine profile.

Fludarabine and clofarabine are chemotherapy drugs used extensively in bone marrow transplantation. The goal of this study is to determine what causes some children to have different drug concentrations of clofarabine and fludarabine in their bodies and if drug levels are related to whether or not a child experiences severe side-effects during their bone marrow transplant. The hypothesis is that clinical and individual factors cause changes in clofarabine and fludarabine drug levels in pediatric bone marrow transplant patients and that high levels may cause severe side-effects.

Sickle cell disease is the most common genetic disease in the world. It results in the synthesis of an abnormal hemoglobin (HbS) which, in its deoxygenated form, polymerizes and causes structural changes in red blood cells (RBCs). They become more rigid and less deformable. The fragility of sickle-cell RBCs leads to their massive destruction, leading to chronic anemia (i.e. low hemoglobin in the blood) and to low tissue oxygenation. More rigid and less deformable, sickle-shaped RBCs tend to obstruct microvessels, leading to particularly painful vaso-occlusive crisis (VOC), which can cause organ failure (spleen, kidneys, brain, lungs, heart, liver, bone ...) and patient's lifethreatning. A preliminary work on red blood cells of sickle cell patients showed alteration of a parameter measuring the overall deformability of RBCs by assessing the nature of their movement in a shear flow. This parameter is altered sickle cell patients at basal state compared to a population of healthy individuals. This alteration is increased when sickle cell patients are in crisis. The main objective of this project is to study the evolution of this parameter in sickle cell patients according to their health status (basal state vs vaso-occlusive crisis). The investigators hypothesize that the alteration of the RBC deformability parameter is significant before symptoms of vaso-occlusive crisis (several hours to several days). The main objective is a weekly analysis of the evolution of the parameter in 30 sickle cell patients (SS or SB°) in the basal state and daily in at least 6 patients at the beginning, during and just after a vaso-occlusive crisis. The comparison between the parameter measured in a subject in the basal state and in the same subject in crisis will be performed. The criteria for the presence of a vaso-occlusive crisis were: the appearance of a attacks of pain affecting at least two territories +/- fever> 38.3 +/- dyspnea and / or sputum. The investigators will differentiate the moderate VOC managed at home with low-level analgesics and VOC requiring hospitalization. The number of days of hospitalization, the occurrence of thrombotic complications, the degree of anemia and hemolysis will be noted. The measurement of the parameter will be performed on a capillary sampling of 40 microliters performed at the fingertip, weekly outside crises and daily when a crisis occurs.

The purpose of this research is to study the causes of Sickle Cell kidney disease, as well as to collect and store samples and information about people with Sickle Cell Disease.

This project will improve the efficiency and quality of healthcare for persons with sickle cell disease, an under-served and at risk population by implementing a co-management model of care. Many patients with sickle cell disease (SCD) receive care primarily from specialty physicians and emergency departments (ED), thus resulting in a lack of primary care and a high number of ED visits and hospitalizations. The goal is to improve PCP and SCD specialist co-management. The overall purpose of this dissemination project is to evaluate utilization data, as well as patient and provider reported outcomes associated with the dissemination of a toolbox of decision support tools to PCP's and ED providers across NC and SC.

The objective of this project is to determine the prevalence of hypertension, hyperlipidemia and hyperglycemia in the pediatric population with sickle cell disease who are obese in Mississippi compared to those pediatric patients with sickle cell disease who are not overweight/obese. The pediatric hematology department at the University of Mississippi Medical Center (UMMC) has a relatively large population of patients with sickle cell disease who are overweight and obese. This is a paradoxical trend since high-energy expenditure of the body to produce new red blood cells usually results in underweight to normal weight patients. From our previous chart review, the investigators found our pediatric patients with sickle cell disease to have similar rates of overweight and obesity to that of state and national levels. The metrics our team will measure include: blood pressure, blood cholesterol levels and blood glucose levels. The investigators expect to find higher rates of hypertension, high cholesterol and high glucose levels in the overweight and obese patients with SCD compared to that of underweight and normal weight. Our ultimate goal for follow up projects will be to determine the baseline risk of hypertension, hyperlipidemia and hyperglycemia in this population so we can then determine effective, sustainable interventions for weight and the co-morbidities that come with increasing weight status. Our goal would also be to educate the patient and families on these interventions and provide them with resources, which could lead to an overall improvement in health and patients quality of life.