Active clinical trials for "Anemia, Sickle Cell"

In this study, patient blood samples from NMA transplants will be provided by Pittsburgh, and samples from myeloablative transplants will be provided by Atlanta (comparative controls). Samples would be obtained pre- and post-BMT from the recipient at a total of 7 timepoints, and from the donor at one timepoint.

This study will explore what may cause people with sickle cell anemia to have heart problems and an increased risk of sudden death. People 18 years of age and older with sickle cell anemia may be eligible for this study. Candidates are screened with a medical history and physical examination, electrocardiogram (EKG), echocardiogram (heart ultrasound), and blood tests. Participants undergo the following tests and procedures: Holter monitoring: The patient wears a small, battery-operated device to record heart rate and rhythm over 24 to 48 hours. QRST surface mapping: An EKG using 64 electrodes is done at rest and during exercise to provide a detailed look at the heart and its conduction system. Chest x-rays are taken to examine the lungs. Bicycle exercise echocardiography test: Blood pressure, pulse, heart rhythm and oxygen use are monitored while the patient exercises on a stationary bicycle. Ultrasound pictures are also obtained during the exercise. Echocardiogram: A heart ultrasound is done to check how well the heart is pumping blood. Pulmonary artery catheterization: A catheter (plastic tube) is inserted into a vein and advanced to the chambers of the heart, through the heart valve and into the lung artery. The pressures in the heart and lung blood vessels are measured while the patient is resting and during exercise, with the bed tilted up and down, and after giving 500 mls of fluid into a vein. Blood tests are done to measure a hormone called brain natriuretic peptide, which can increase with the development of heart failure, and nitrite, a substance that can affect blood vessel dilation. Some blood is stored to test for inflammatory markers and for possible future gene and protein analysis. Cardiac magnetic resonance imaging (cMRI): The patient lies in a donut-shaped magnet while pictures of the heart are obtained using a magnetic field and radio waves. Earplugs are worn to muffle the loud sounds that occur with electrical switching of the magnetic fields. A contrast agent called gadolinium may be injected to enhance the quality of the images. Invasive electrocardiographic (reveal) monitoring: This procedure permits study of the heart rhythms over a long time period. A small device is placed just under the skin on the left side of the chest. It can be left in for up to 14 months to monitor the heartbeat continuously during this time.

To establish a link among Chlamydia infection, sickle cell anemia, and stroke risk.

Patients with sickle cell disease often develop painful crisis without any obvious reasons. Some patients are more likely to develop this complication than others. It is now clear that painful crisis only occurs when sickled red blood cells stick to white blood cells that have been activated, usually by inflammation or infections. A recent study in mice with sickle cell disease showed that the use of long term antibiotics could reduce the number of activated white blood cells and reduce death of the mice during sickle cell crisis. The investigators believe that sickle cell patients who develop frequent painful crisis may have a different pattern of bacteria in their intestine when compared to those whose painful crisis occurs infrequently. In this study, the investigators propose to study sickle cell subject's blood to determine how many activated white blood cells he/she have. The investigators will also examine his/her stool to compare the bacteria in his/her stool to those other sickle cell patients. The investigators will then investigate whether or not the results from the blood and stool tests correlate with how frequently the patient develops painful crisis. The investigators will examine the patients' medical records to find out how many times they have been admitted to the hospital for sickle cell crisis in the last 12 months. The investigators will also obtain information on the following: their age, their sex, whether they are taking hydroxyurea or Penicillin, when they last had a transfusion or exchange transfusion therapy and painful crisis needing hospital admission, whether they have received any antibiotics (other than Penicillin) in the last 4 weeks, and whether they are experiencing a painful crisis at the time that they enter the study. The investigators will obtain, from their previous laboratory results, their levels of hemoglobin F and markers of inflammation. The investigators will check their hemoglobin F level if they have not already had this tested. The investigators expect to enroll 50 subjects into this study at Rhode Island Hospital/Hasbro Children's Hospital.

Sickle cell disease (SCD) is an inherited disorder of the red blood cell. It is now the commonest genetic disorder in the UK and of childhood stroke, with up to 40% of children having a stroke (clinical or picked up on a scan) by school age. Patients are prone to develop acute crises necessitating hospital admission and resulting in long-term complications. Such events result in considerable morbidity, disability and mortality with its consequent burden on patients, families, the health service and society as a whole. Doctors have very little ability to predict who will get ill and when and so it is very difficult to known when and how to administer treatments. Furthermore there is very little in the way of treatments available and the mainstay of prevention is a chronic blood transfusion programme which is expensive, requires time off work and school and can be fraught with complications. This, in a population who is frequently educationally and socially disadvantaged at the outset. Recent evidence in sickle cell disease and other diseases that have similar underlying processes, points towards the importance of microparticles (circulating broken pieces of cells) and the coagulation system as being important. By comparing levels of these particles and molecules in patients with those found in healthy volunteers and with other measures known to be important, this study hopes to identify their role so as to improve the management of these patients and potentially to lead the way for new therapies. Participants will be required to donate a small amount of blood (1 teaspoon in the very young, two in older children and adults). The investigators aim to take this sample where possible when people are having a blood test in any case.

Sickle cell disease is a life-threatening genetic disorder that can be effectively treated following early diagnosis via newborn screening. However, sickle cell disease is most prevalent in low-resource regions of the world, where newborn screening is rare due to the cost and logistical burden of laboratory-based methods. In many such regions, >80% of affected children die, undiagnosed, before the age of five years. A convenient and inexpensive point-of-care test for sickle cell disease is thus crucially needed. In this study we will conduct a blinded, multicenter, prospective diagnostic accuracy study of HemoTypeSC(TM), an inexpensive 15-minute point-of-care immunoassay for detecting sickle cell disease, hemoglobin C disease, and trait phenotypes in newborns, children, and adults.

Children with sickle cell disease systematically receive a transfusion 2 to 5 days before scheduled surgery (with the exception of minor surgeries) in order to avoid post-operative complications of which the vaso-occlusive crisis and acute thoracic syndrome are the most frequent. This standardized preoperative protocol was established on the basis of the results of large-scale randomized studies, most of which date back over ten years, and which have demonstrated the beneficial effects of transfusion (or transfusion exchange) preoperatively. To date, several other more recent studies (but not controlled) have questioned this type of systematic management. The purpose of this study is to review retrospectively data of sickle cell children who have undergone elective surgery at the Huderf in the last ten years and to identify the eventual complications encountered. The most common procedures in these patients are: tonsillectomy with or without associated adenoids, splenectomy and cholecystectomy. General data on sickle cell disease (history, genotype, G6PD deficiency, biology and previous complications), pre-surgical preparation, surgery and post surgical management and complications will be collected and analyzed. This retrospective analysis will allow an objective assessment of the current quality of care and will provide useful data to improve patient management.

Most patients with sickle-cell disease have periodic intensely painful episodes. To manage this pain, we are proposing the drinking of at least 500 mL of water followed by far infrared radiation.

Recommendations for exercise prescription currently do not exist for individuals with sickle cell anemia (SCA) despite the known impact that SCA-related complications has on physical functioning and fitness. A major barrier to increasing physical activity in SCA is the concern that the well-described inflammatory effects of exercise could precipitate or exacerbate complications such as vaso-occlusive pain or airway bronchoconstriction (i.e. exercise-induced asthma). Although the investigator's preliminary data suggest that increasing physical activity may be beneficial rather than harmful in children with SCA, the pro-inflammatory effects associated with repeated bouts of moderate to vigorous exercise remain poorly understood in this population. The long term goal is to address the safety and health impact of regular exercise in children with SCA. This proposal would help establish the safety of moderate to vigorous intensity exercise in children with SCA and importantly, will inform the design of future clinical trials focused on exercise training as a transformative strategy to improve fitness and overall well-being in this population.

BIOCADRE is a CADRE substudy and aims to characterize more precisely the sickle cell patients with extreme phenotype.