Active clinical trials for "Anemia, Sickle Cell"

Evaluation of knowledge about contraception in sickle cell adolescents.

The intent of this open-label, multicenter Expanded Access Program (EAP) is to provide early access to treatment with voxelotor prior to market authorization for pediatric patients age 6 months to 11 years with sickle cell disease (SCD) who have no alternative treatment options and are ineligible to participate in clinical trials of voxelotor. Cohort A, ages 4 to 11 years is now closed to enrollment.

Vitamin D deficiency may be under-diagnosed in sickle cell disease French children. Therefore, the investigator need an epidemiologic study about the prevalence of vitamin D deficiency in this specific population. There are not specific guidelines neither testing nor treatment. The investigator propose to test vitamin D status in all children with sickle cell disease who are consulting their referring haematologist doctor or in the emergency ward.

In this prospective observational trial, participants with chronic hemolysis will be assessed with echocardiogram for elevated tricuspid jet velocity and other evidence of pulmonary hypertension. Participants will have laboratory studies evaluating: severity of hemolysis, splenic function, inflammation, endothelial dysfunction, and hypercoagulability. There will be 3 main categories of participants enrolled in this study: (1) pediatric participants with severe sickle cell disease (SCD) (HbSS, HbS/β° thalassemia ) who are not receiving treatment (e.g., hydroxyurea or chronic transfusions); (2) pediatric participants with other forms of SCD or severe SCD (HbSS, HbS/β° thalassemia) patients being treated with hydroxyurea or chronic transfusions; and (3) pediatric and adult participants with other non-sickling hematological disorders.

Sickle cell disease (SCD), also known as sickle cell anemia, is an inherited blood disease that can cause intense pain episodes and may lead to organ failure. Preliminary studies have shown that adults with SCD may have brain abnormalities that contribute to problems with cognitive functioning, including attention and memory difficulties. This study will use brain magnetic resonance imaging (MRI) and neuropsychological testing to examine the differences in cognitive functioning in adults with SCD and adults without SCD. 212 subjects participated in this cross-sectional study consisting of screening questionnaires, a neuropsychological testing battery, and MRI testing. Enrollment into this study ended in May 2008.

This study intends to analyze the clinical characteristics of teenagers admitted to pediatric wards due to organic illness and non organic diagnosis, including patients suffering from chronic diseases and comorbidity.

The study will look at the risk factors for pulmonary hypertension (high blood pressure in the lungs) in children and adolescents with sickle cell anemia (SCA) and examine the role of hypoxia (oxygen shortage) in the disease. In patients with SCA, red blood cells become sickle-shaped and tend to form clumps that get stuck in blood vessels, blocking blood flow to the limbs and organs. Blocked blood vessels can cause pain, serious infections, and organ damage. Many patients with SCA also develop pulmonary hypertension. Children and adolescents with SCA or Chuvash polycythemia (another blood disorder that carries an increased risk for pulmonary hypertension) may be eligible for this study. Participants undergo the following procedures at the beginning (baseline) and end of the study: History, physical examination and blood tests . Echocardiography (ultrasound study of heart function). Transcranial doppler (brain ultrasound study to measure brain blood flow). Lung function tests. 6-minute walk (measure of the distance covered in 6 minutes of walking). In addition, patients are followed by telephone or by clinic visits every 6 months for a review of their medical history and medications. A physical examination is also done at 12 months.

This study is being done to see what impact having a Lifeport device has on quality of life for children with sickle cell who are getting chronic transfusions, from the child's perspective.

To continue to follow the newborn cohort and the over-35 years of age cohort from the Cooperative Study of Sickle Cell Disease (CSSCD), a study of the natural history of sickle cell disease.

To develop a clinical severity index that could prospectively identify sickle cell disease patients who were at high risk for a turbulent clinical course and a poor prognosis.