View clinical trials related to Anemia, Sickle Cell.
Filter by:To determine the pharmacokinetics of methadone in children and adults with SCD who are experiencing a painful episode.
This purpose of this study is to understand the differences between people who have a good response to deferasirox (exjade) compared to people who have a poor response to this medication when used for transfusion-dependent iron overload. The hypothesis is that patients with poor responses have physiologic barriers to deferasirox that may include absorption, pharmacokinetics of drug metabolism, hepatic clearance and/or genetic factors.
Acute chest syndrome (ACS) is a frequent and potentially life-threatening pulmonary illness. It is a complication of sickle cell disease and is the leading cause of death from this disease in adults. Several pathologic processes are recognized causes of ACS, including infectious diseases, hypoventilation secondary to chest pain, in situ thrombosis and pulmonary fat embolism. Inhaled nitric oxide (iNO) has been shown to be a pulmonary vasodilatator with minimal systemic effects and has also been shown to improve gas exchange in both animal and human acute lung injury (ALI). The combined effects of iNO gas of improving pulmonary ventilation to perfusion matching, reducing alveolar and systemic inflammation, modulate the course of acute chest syndrome, which combine the physiopathology of vaso-occlusive crisis and acute lung injury. We hypothesise inhaled NO will improve oxygenation and clinical outcome of sickle cell disease patients with acute chest syndrome.
Sickle cell disease (SCD), also known as sickle cell anemia, is an inherited blood disease that can cause organ damage, stroke, and intense pain episodes. A blood stem cell transplant is a treatment option for someone with a severe form of the disease. Prior to undergoing a transplant, people typically receive a conditioning regimen of high doses of chemotherapy and other medications to prepare the body to accept the transplant. A conditioning regimen that uses lower doses of chemotherapy and medications may be safer for transplant recipients. This study will evaluate the safety and effectiveness of blood stem cell transplants, using bone marrow from unrelated donors, in children with severe SCD who receive a reduced intensity conditioning regimen prior to the transplant.
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.
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.
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 is a clinical trial of bone marrow transplantation for patients with the diagnosis of a genetic disease of blood cells that do not have an HLA-matched sibling donor. Genetic diseases of blood cell include: Red blood cell defects e.g. hemoglobinopathies (sickle cell disease and thalassemia), Blackfan-Diamond anemia and congenital or chronic hemolytic anemias; White blood cells defects/immune deficiencies e.g. chronic granulomatous disease, Wiskott-Aldrich syndrome,Osteopetrosis, Kostmann's syndrome (congenital neutropenia), Hereditary Lymphohistiocytosis (HLH); Platelets defects e.g.Congenital amegakaryocytic thrombocytopenia; Metabolic/storage disorders e.g. leukodystrophies,mucopolysaccharidoses as Hurler disease;Stem cell defects e.g.reticular agenesis, among many other rare similar conditions. The study treatment plan uses a new transplant treatment regimen that aims to try to decrease the acute toxicities and complications associated with the standard treatment plans and to improve outcome The blood stem cells will be derived from either unrelated donor or unrelated umbilical cord blood.
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.
Medication non-adherence is a true public health problem. Despite advancements in the molecular understanding of disease and improvements in therapy, patient health outcomes will not improve unless patients take prescribed medications regularly. Decreasing the gap between efficacious and effective therapy for patients with SCD is an essential research agenda. Hydroxyurea has been shown to be safe and efficacious in children and infants. However, the effectiveness of the prophylaxis depends on adherence to the recommended regimen. Medication adherence in SCD has previously been found to be sub-optimal in patients taking penicillin, desferoxamine, and pain medication. Adherence to HU has been studied to some extent in children with SCD. Based on estimates of adherence in other chronic illness we expect approximately 50% of patients to be >80% adherent with their HU administration. There is no gold standard for improving adherence to treatment. There have been a few attempts in the SCD population to improve adherence. These include a day camp to promote education about desferoxamine and peer support, a combination of a slide-show about SCD and it complications, weekly phone calls by the clinic social worker and a calendar, and a seven-phase educational program. Given the striking improvements in the peripheral blood smear findings of patients with SCD on HU therapy, with reduction in the numbers of sickled cells, we hypothesize that viewing the peripheral blood smear of patients with poor adherence to HU compared to a blood smear of someone on HU can be used to improve adherence in non-adherent patients. We will conduct a randomized trial between the intervention of regularly showing children and their parents the peripheral blood smear and standard care, including reminders of the importance of compliance and review of complete blood count parameters, including WBC, MCV, and Hgb concentration. The outcome measures will be increase in hemoglobin concentration and %HbF and increase in perceived QOL. QOL will be measured with age-appropriate and parent/proxy PedsQL™. Medication adherence will also be monitored throughout the study with pharmacy prescription refills, physician assessment, and self-report via a visual analogue scale. Adherence estimates, hemoglobin concentration, %HbF and QOL will be measured at baseline, 3 months and 6 months.