Active clinical trials for "Anemia, Sickle Cell"

This study will determine eligibility for participation in research studies on blood disorders conducted by the National Heart, Lung and Blood Institute and the National Institute of Diabetes, Digestive, and Kidney Diseases. Healthy volunteers, patients with blood disorders under study by NHLBI and NIDDK and potential stem cell donors for patients with blood disorders who are 8 years of age and older may be eligible for this screening protocol. (Healthy volunteers who qualify for research protocols would serve as control subjects.) Participants undergo the following tests and procedures: Healthy Volunteers Medical history, physical examination, blood tests and urine sample collection. Buccal mucosa sample collection. (Cells are collected from the inside of the cheek by gentle scraping with a bristly brush.) Bone marrow aspiration (only for volunteers 18 years of age and older). Potential Stem Cell Donor -Same as for healthy volunteers plus evaluations that may include electrocardiogram, echocardiogram, imaging studies (X-rays, CT scans, MRI scans and others), heart evaluation, and lung function tests. Patient with Blood Disorder Same as for stem cell donors plus additional evaluations and treatments that may include radiation oncology evaluation, catheter placement, blood transfusions, kidney and liver biopsies. Short courses of drug treatment for induction of fetal hemoglobin in sickle cell patients, and/or iron chelation in patients receiving chronic red cell transfusions may be included as well.

Patients diagnosed as having hemoglobinopathies are exposed to serious bacterial infections, principally those patients that underwent splenectomy. Since the introduction of anti pneumococcal vaccine the incidence decreased significantly but other bacteria besides encapsulated bacteria takes place as principal cause of invasive infections. The purpose of this study is to analyse in a retrospective study the incidence of those infections in a group of patients suffering from thalassemia and sickle cell anemia treated in our clinic.

Hydroxyurea was found to be a good treatment in adult patients with sickle cell anemia with significant decrease in the frequency of vaso-occlusive crises and other crises related to SCA. Several studies were published with relative short term follow up in pediatric and young adult age. The purpose of this study is to assess the long term follow up in a group of patients that initiated Hydroxyurea treatment in childhood.

DISPLACE is a three part, multi-center U.S. based study to evaluate the barriers to stroke screening and prevention in children with sickle cell anemia (SCA). In the United States, TCD (Transcranial Doppler ultrasound) is a proven method of screening children with SCA for stroke. However, many children are not getting the screening they need. This study will examine the issues that hinder and help children get the screening at 28 different hospitals and sickle cell centers to improve care for all children with sickle cell anemia. The investigators will then plan a study (part 3) aimed to improve stroke screening and prevention in sickle cell anemia.

Sickle cell disease is the most common inherited genetic disorder, accounting for 300,000 births worldwide per year. It is caused by an autosomal recessive mutation of the β-globin gene, responsible for an abnormal hemoglobin, the main protein in red blood cells, responsible for transporting oxygen from the lungs to the tissues. The abnormal hemoglobin, known as "Sickle" or S, deforms the red blood cell, causing chronic hemolytic anemia, organ damage (heart, spleen, etc.) and vaso-occlusive crises. Therapeutic progress and specialised patient follow-up have considerably improved the vital and functional prognosis of children and adolescents with sickle cell disease. Physical fitness, measured during a cardiorespiratory exercise test (CPET), is used to determine maximal oxygen uptake (VO2max). Patients with sickle cell disease have a multifactorial limitation of exercise tolerance, which may affect their physical fitness. Authors have shown that VO2max is impaired in children and adolescents with sickle cell disease, independently of their baseline hemoglobin level. Yet VO2max is a key determinant of health-related quality of life (HRQoL) in patients being monitored for a chronic disease. In the past, our team has contributed to the assessment of HRQoL in several groups of pediatric patients suffering from chronic disease (congenital heart disease, PAH). To date, the link between impaired physical fitness and HRQoL has not been demonstrated in sickle cell children. The pathophysiological determinants of reduced physical capacity and exercise tolerance in sickle cell patients have also not been fully elucidated. Studying these factors will enable us to propose appropriate treatment in the future, with the aim of improving physical fitness and HRQoL in children and adolescents with sickle cell disease.

This project will promote the development of transdisciplinary analyses. Neuropsychological disorders will be explored with the usual appropriate tests done by psychologists and neuropsychologists regularly involved in the management of sickle cell disease affected children. For the social sciences' component, various methods will be used: Measure of the Life habits (MHAVIE), Measure of Environmental Quality (MQE) and semi-guided interviews will complete the collection of qualitative data. The expected results concern the identification of the barriers or facilitators the sickle cell patients might face in their social participation, whether they are affected or not by neurological disorders.

The objective of this study is to to determine the rate of nasopharyngeal carriage of Streptococcus pneumoniae (Sp) in children with sickle cell disease over 6 months and under 15 years of age over a 9-month period in Ile-De-France.

The objective of this study is to evaluate the incidence of ACS within the DREPADOM system and compare it to expected incidences of ACS (historic cohorts of PRESEV1 and PRESEV2)

Patients with sickle cell disease may be at risk for acute kidney injury (AKI)during sickle cell crisis (pain or acute chest syndrome). This study will evaluate the role of hemolysis during SCD crisis on the development of AKI and the role for monitoring urine biomarkers during an admission for crisis and during well clinic follow-up.

The spleen in Sickle Cell Anemia and Sickle Cell Thalassemia is usually enlarged in the first years of life but the immune protection provided is considered insufficient. In homozygous Sickle cell patients the spleen usually developed recurrent infarcts and after the first decade of age become fibrotic. Acute splenic sequestration is also frequent in those patients and this is considered as an indication for splenectomy. In comparison in Sickle cell thalassemia patients, hypersplenism is more frequent. The purpose of this study is to compare the clinical and laboratory issues related to the spleen in two groups of Sickle cell patients.