View clinical trials related to Anemia, Sickle Cell.
Filter by:The main objective of this study is to evaluate at the national level (France) - the early healthcare practices for children with sickle cell disease screened at birth, - the adequacy of theses practices with the national recommendations, - their variability over time and according the characteristics of treatment centers. Will be studied in particular the diffusion of the latest preventive measures (practice of trans-cranial Doppler and pneumococcal conjugate vaccine) and their link with the residual risks of death, stroke and invasive pneumococcal infections. The study includes all patients born in France between 01/01/2006 and 31/12/2010. Events are recorded and analysed only during the first 5 years of life.
Sickle cell disease (SCD) is an autosomal recessive disorder resulting from a substitution in the β chain of hemoglobin (Hb) which causes hemoglobin S to polymerize when deoxygenated. SCD patients present immune abnormalities that have always been attributed to functional asplenia. It it is now being recognized that patients with SCD have a pro-inflammatory condition with altered immune system activation contributing to the pathology of SCD. Increased levels of neutrophils, monocytes or cytokines have been reported in SCD patients. SCD is associated with many acute and chronic complications requiring immediate support. Actual strongly recommended therapies include chronic blood transfusions (CT) and hydroxyurea (HU). In addition, episodic transfusions are recommended and commonly used to manage many acute SCD complications.There is strong evidence to support the use of HU in adults with 3 or more severe vaso-occlusive crises during any 12-month period, with SCD pain or chronic anemia, or with severe or recurrent episodes of acute chest syndrome. HU use is now also common in children with SCD. Some patients receive chronic monthly RBC transfusion with the objective to reduce the proportion of HbS to < 30 %. Long-term RBC transfusions prevent and treat complications of SCD decreasing the risk of stroke and the incidence of acute chest syndrome (ACS). Therapeutic complications, such as alloimmunization against RBC in 20-50% of patients or hematopoietic stem cell transplantation (HSCT) graft rejection, constitute an immune-based clinical issue in SCD. Poorly understood RBC alloimmunization is responsible for serious hemolytic transfusion reaction associated with severe mortality and morbidity underlying the need for a better understanding of the immunology of SCD to improve SCD transfusion support/outcome. Little evidence exists about HU effects on immune functions in SCD. HU treatment doesn't appear to have deleterious effects on immune function and appears to decrease the abnormally elevated number of total WBC and lymphocytes, while CT does not. Patients with SCD are at higher risk of infections and prophylactic vaccination is strongly recommended. Recent data suggest that vaccinal response to pneumococcal antigens in SCD patients is identical to healthy control while controversy concern the stability of the immune protection after vaccination of SCD patient. Antibody levels declined over the year and the need for more frequent vaccination in SCD patient should be investigated. Currently, there is no evidence whether HU may interfere with pneumococcal immune response. Purohit showed that immune response to inactivated influenza A (H1N1) virus vaccine was altered in patient with SCD receiving CT but little is known on immune response to vaccination in patients with SCD receiving HU. Recent data suggest that not only inflammatory status but also humoral immune response to antigens in SCD patients may differ according to treatment. Yazdanbakhsh reported an imbalance between regulatory T cell (Treg) and effector T cell (Teff) in alloimmunized SCD patients with as consequence an increase in antibody production. In a model proposed by the authors, the balance between Treg and Teff is dictated by the monocyte control of cytokines expression. Altered activity of monocyte heme oxidase-1 (HO-1) would be responsible of a decrease in IL-12 and an increase in IL-10 cytokines secretion impacting the Treg/Teff cells ratio and promoting antibody production by B cells. The objectives of the project are to assess whether different humoral immune responses to vaccines or to erythrocyte alloantigens are related to the type of treatment administered to patients with SCD. We also aim to study if these differences might be related to different expressions of HO-1 by monocytes.
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.
This study is being conducted to test an intervention for children and adolescents ages 8-17 years with sickle cell disease and their families. In the first phase of this study, key informant interviews are being conducted with health care providers and children ages 8-17 with sickle cell disease and their primary caregivers. Participants are asked to review the intervention and provide feedback that will inform revision to the intervention.
Background: Sickle Cell Disease (SCD) is a blood disorder that occurs mainly in people of African descent. Researchers want to learn more about the painful attacks and complications associated with SCD. They want to look for a relationship between SCD and specific changes in the blood. They want to study the role of genetics, inflammation, and blood clotting factors in SCD. They will do this with blood samples collected during an acute painful attack and in between attacks. Objective: To learn more about the painful attacks and complications associated with SCD. Eligibility: People ages 18-80 with SCD or who are healthy Africans or African Americans without SCD Design: - Participants will be screened with medical history and physical exam. - Healthy participants will have one visit. - Participants with SCD will have their first visit when they are not having a pain attack. They will have their next visit during a pain attack. About 3-4 months after this attack, they will have a final visit. - Visits will include a physical exam, and blood and urine tests. - Participants may have their blood samples used for genetic testing for research.
This is an open label, single arm study which enrolled 5 subjects with SCD who previously participated in the GBT440-001 study (NCT02285088).
The overall goal of this proposed project is to 1) increase co-management between sickle cell specialists and primary care providers (PCP's); 2) increase the use of hydroxyurea (HU) which prevents Vaso-Occlusive Episode (VOE), EDs and subsequent hospitalizations, and death; 3) identify and link patients not receiving primary or SCD specialty care to care, and 4) shift healthcare use from EDs and hospitalizations to primary and specialty co-management. Many persons with SCD experience a poor quality of life, serious medical complications and frequent painful events that require treatment from SCD specialty care, primary care and emergency department (ED) providers. There are two dominating models of care in the United States; neither are ideal. Many people with SCD have all of their healthcare needs addressed by sickle cell specialists who do not typically provide primary care and are often geographically distant from the patients' home. Other sickle cell patients receive all of their care in EDs. Both models are inadequate and result in an alarmingly high number of ED visits for many patients. Current care models are neither cost efficient nor promoting optimal patient outcomes. To improve outcomes, the investigators will implement a new model of care for SCD using nurse care managers, web based-interactive algorithms, and test if additional patient provided coaching can improve outcomes.
A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study of Voxelotor Administered Orally to Patients With Sickle Cell Disease
The primary aim of this study is to test the feasibility and acceptability of implementing a multimedia computerized cognitive behavioral therapy (cCBT) program for reducing SCD pain symptoms in a single-arm pilot pragmatic clinical trial. The investigators will recruit 40 SCD patients with chronic pain and/or on chronic opioid pain treatment and randomize them 3:1 to two groups (cCBT and e-Education respectively), randomizing unevenly in order to best gather feasibility data for the cCBT. Both groups will use a mobile app to track daily pain/mood. The cCBT group will receive sessions of the CALM-SCD program to complete via mobile device and will have weekly follow-up with a care coach. The Education group will receive online education modules to complete via mobile device and will also have weekly follow-up with a care coach. The primary outcomes of the trial include feasibility (recruitment, retention, provider and patient feedback) and acceptability (sessions completed) of the CALM-SCD program.
This is a pilot study, single-blind, randomized, multicenter, therapeutic clinical trial designed to evaluate the feasibility of enrolling infants and toddlers (9 months to 36 months) with sickle cell anemia (SCA; HbSS or HbSβ^0thalassemia), regardless of disease severity, to a therapeutic trial. A prior clinical trial at St. Jude Children's Research Hospital (SJCRH) (BABYHUG, NCT01783990) demonstrated that a fixed dose (20 mg/kg/day) of hydroxyurea was safe and effective in decreasing SCA-related complications in very young children (9-18 months), and largely due to these findings, hydroxyurea is recommended to be offered to all children (≥9 months old) with SCA, independent of disease severity. Nevertheless, children in the treatment arm of BABYHUG continued to experience vaso-occlusive symptoms and to incur organ damage. In clinical trials of older children with SCA, intensification of hydroxyurea to a maximum tolerated dosage (MTD), defined by mild to moderate myelosuppression, may be associated with improved laboratory parameters compared to fixed lower-dosing, but the clinical benefits gained from dose intensification have not been described. Therefore, in this trial, children in the standard treatment arm will receive a fixed dose of hydroxyurea (20 mg/kg/day), and participants in the experimental arm will receive hydroxyurea intensified to MTD, defined by a goal absolute neutrophil count (ANC) of 1500-3000 cells/µL. This trial aims to establish a multicenter infrastructure that will identify, enroll and randomize very young children (9-36 months) to receive fixed dose versus intensified-dose hydroxyurea in a single blinded manner, and to obtain prospective pilot data comparing the clinical and laboratory outcomes between the treatment arms to facilitate design of a definitive phase III trial.