Active clinical trials for "Anemia, Sickle Cell"

The purpose of this study is to examine how abnormal blood flow in the small vessels (microvessels) of the heart, muscle and kidney in paroxysmal nocturnal hemoglobinuria (PNH) or sickle cell disease leads to poor functioning of the heart and kidney. To test this question, the investigators will perform imaging tests (contrast ultrasound perfusion imaging) to look at the flow and function of these microvessels and compare this information to heart and kidney function. To further look at this question, patients who have PNH will be studied before and after starting a new drug (Soliris) that decreases damage to blood cells. In patients with sickle cell disease, patients will be studied at baseline (not during a pain crisis) and also during a pain crisis if one develops.

Since 1987, a screening for β Thalassemia in pregnant women is carried on in northern Israel, and from 1999 all the samples were tested also for Hgb S, Hgb C, Hgb D, Hgb O Arab and others. In this study, the investigators intend to summarize the results of this preventive program aiming to detect couples at risk for having offspring with Thalassemia or SCA, the compliance regard to genetic counseling and prenatal diagnosis and the incidence of new affected babies born.

This study will summarized the clinical and laboratory data and the outcome of all the patients suffering from Sickle Cell Anemia (Including Sickle cell thalassemia) admitted to the pediatric ward.

In sickle cell disease (SCD), polymerisation of haemoglobin S and the resulting shape change of the red blood cells (RBC) lead to vascular occlusion and severe painful crises. Permanent inflammatory state and abnormal RBC adhesion to the endothelium trigger these phenomenon. Hydroxyurea (HU) is the only drug that has been shown to reduce clinical severity of SCD, and this was initially attributed to the stimulation of foetal haemoglobin (HbF). However, the clinical response does not correlate consistently with the degree and time of HbF increment, suggesting that HU clinical benefits may involve other mechanisms such as the induction of natural anti-inflammatory response via the hypothalami-pituitary-adrenal axis.

The investigators' primary objective is to study prevalences of myocardial iron overload, defined as a cardiac T2*< 20 ms, in 3 populations of multiply transfused patients, affected with thalassemia, sickle cell disease, and myelodysplasia.

The BABY HUG Treatment Study was designed to see if treatment with the drug hydroxyurea (also called HU) in children with sickle cell disease could prevent organ damage, especially in the spleen and kidneys. There was also a chance that treatment could prevent painful crises, lung disease, stroke, and blood infection.

Background: - Sickle cell disease changes the shape of red cells. This makes them more likely to break down as they get stuck in small blood vessels. This leads to low red cell count and also damage to small blood vessels that supply many organs. One of the affected organs is the liver. Sickle cell disease and its treatment through blood transfusion can lead to significant liver damage. This disease also can cause the liver to regrow abnormally after damage. This can cause high blood pressure in the liver. Researchers want to know if curing sickle cell disease with a stem cell transplant improves liver damage. Objectives: - To explore specific factors that improve or worsen sickle cell liver disease after a stem cell transplant. Eligibility: - Adults ages 18 and older with sickle cell liver disease. Design: Participation will take approximately 7 days over 2 years. Visit 1: participants will be screened with medical history and review of current treatment regimen. Visit 2: participants will return to the clinic for explanation of the study and physical exam. They will also have blood and urine tests, and scans of the liver. All participants will have a 2-night stay at the clinic. They will have a liver biopsy and a test of liver pressure. They will be sedated and a tube will be inserted in a vein in their neck. Participants who have a stem cell transplant will have a second biopsy about 24 months later. Over the 2-year study period, participants will have blood drawn 2-4 times and stool samples collected 2 times.

Sickle Cell Disease is a serious disease that is life-threatening for patients being homozygous for the SS form or heterozygous for the SC or bthal forms. The CHU Brugmann hospital currently regularly treats about 70 homozygous adult patients and this number is in constant augmentation. The age average of the patients is below 30. The hospital developed a close collaboration with the Queen Fabiola Kids University Hospital to optimize the transition of young sickle cell patients from the pediatric to the adult network. The emergency care of sickle cell patients remains a source of worry. Even with a correct treatment (Hydroxy-urea or exsanguineous transfusions), patients suffer from frequent sickle cell disease crisis when stress or infection cause hemolysis. The pain level is intolerable and causes emergency hospital admission (2 to 3 crisis per patient per year on average). The crisis are more frequent with poor compliance to the treatments. There are several obstacles to the rapid and optimal management of these patients: fear of causing addiction to heavy pain releaf products (high dosis of morphine) lack of biological parameters for the determination of the crisis severity. The prognostic value of the lactate dehydrogenase (LDH) level in a vaso-occlusive crisis was recently stressed while activation of the coagulation, translated by the elevation of various parameters including the rate of DD dimers, seemed associated with clinical complications. The deleterious role of increased oxidative stress has also been recently demonstrated in patients with sickle cell disease, opening new therapeutic avenues. This study aims to prospectively evaluate the management of sickle cell patients being admitted in the emergency department for a vaso-occlusive crisis. The level of satisfaction of the patients will be measured. The investigators will also evaluate the predictive value of several routine biological parameters regarding the severity of the crisis, including the values of nitrous albumin (PNA) as marker of oxydative stress. This last dosage will be made in collaboration with the team of Dr Wayenberg and Pr Bottari in Grenoble.

Youth diagnosed with sickle cell disease (SCD) may have difficulty taking medication as prescribed (adherence). Hydroxyurea (HU) is one medication that youth may take to help manage SCD. Electronic adherence monitoring is widely considered the gold standard in objective adherence measurement. These monitors provide continuous, real-time records of medication adherence and reveal problematic behavior patterns, including underdosing, overdosing, delayed dosing, "drug holidays" (i.e. where individuals do not take medications for a specified interval of time), and "white coat" adherence (i.e., a pattern of drug adherence as a function of time where individuals display good adherence immediately before and after clinic attendance with worsening adherence in the period between). Overall, electronic adherence measures are considered valid, reliable, and accurate, with clear advantages over pharmacy refill records, physician estimates and self-report measures. Currently, only one electronic measure capable of monitoring medications in both pill and liquid form is being manufactured: WisePill and WiseBag. While data are limited regarding its validity and reliability, preliminary data support the use of Wise technology to measure adherence to medication. The current study will determine the Wise device's ability to feasibly measure adherence to liquid and solid form HU medication in a pediatric SCD population.

The purpose of this study is to use comprehensive exercise testing to examine causes of exercise limitation in children and young adults with sickle cell anemia.