View clinical trials related to Anemia, Sickle Cell.
Filter by:Sickle cell anemia (SCA) is an inherited blood disorder that can cause organ damage. The BABY HUG study is evaluating the use of the medication hydroxyurea at preventing organ damage in children with SCA. The purpose of this follow-up study is to evaluate the long-term effects of hydroxyurea in children who have participated in the BABY HUG study.
Quality of life of adult patients with sickle cell disease is deeply impaired by severe adverse medical events that inadvertently occur throughout their time life. Indeed, patients not presenting a life threatening condition often present to the emergency department with sickle cell disease crisis related pain. Currently, the effectiveness of specific analgesic strategies for treating sickle cell disease crisis related pain are mostly based on acetaminophen and opioid derivates combination along with oxygen delivery. Those strategies are effective but may last up to half an hour to obtain pain relief. This delay mostly depends on the availability of venous access and on individual patient response to treatment. Nitrous oxide is a volatile efficient analgesic therapy that has been repeatedly shown to allow rapid analgesia in the emergency department setting. The investigators hypothesise that a new analgesic strategy (rapid optimized analgesic strategy) including nitrous oxide and nefopam would be as safe and more rapidly effective than current analgesic strategy.
Children with sickle cell disease (SCD) are at risk for central nervous system (CNS) complications, which may affect academic achievement. This study will evaluate an educational support program for parents that aims to improve academic achievement in children with SCD.
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 who are treated for anemia with monthly blood transfusions for 6 months versus adults with SCD who receive usual care for 6 months.
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.
The purpose of this study is to assess the safety and tolerability of HQK-1001 administered for a total of 12 weeks (with one dosing break) in subjects with sickle cell disease.
The purpose of this study is to validate a brief survey tool, the SCD Pain Burden Interview (SCPBI), which can be used in the clinical and/or research settings to assess the impact of pain on children with sickle cell disease.
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.
Sickle Cell Disease (SCD) is the most prevalent genetic disease of haemoglobin.The underlying abnormality in the red blood cell (RBC) of SCD is the presence of abnormal sickle cell hemoglobin (HbS), which, when deoxygenated, becomes relatively insoluble, forms aggregates with other hemoglobin molecules within the RBC and causes rigid deformation of the cell. Acute pain vaso-occlusive crisis, strokes and acute chest syndrome are the main acute complications, sometimes life-threatening, often leading to organic and functional squeal. Although the common SS form of SCD is a unique gene disorder, the range of the clinical severity is remarkably wide and striking, suggesting that clinical polymorphism is due to modifier genes and environmental factors.Most of the research efforts have been focused on the biology of haemoglobin and of the red cells. Meanwhile, the complex pathophysiology of SCD is undoubtedly influenced by the many physiologic functions of the vascular wall. In line with this hypothesis are several reports of increased circulating levels of endothelium-derived surface molecules in SCD patients suggesting marked endothelial stress in SCD. Similarly, other processes that involve the endothelium, such as leukocyte adhesion and activation, may play a role in vascular occlusion. This accumulation of data raises the unanswered question of the mechanisms of endothelium maintenance and regeneration in SCD. Through these mechanisms, it is likely that function or dysfunction of the vascular endothelium contributes to the overall vascular pathobiology of this disease, which includes recurrent vaso-occlusions, stroke, leg ulcers, chronic organ ischemic damages, and neovascularizing retinopathy that affect nearly one-half (48%) of the surviving patients by the fifth decade.Thus, our groups have combined their respective clinical and biological expertises to test the hypothesis that SCD is a condition of specific endothelial stress and dysfunction upon chronic and Paracystic abnormal interactions with circulating cells and abnormal oxygen delivery to tissues. Specifically, we hypothesize that chronic endothelial stress with detachment of activated endothelial cells require increases mobilisation of the Endothelial Progenitor Cells (EPCs) that maintain endothelial homeostasis to avoid major thromboembolic events and vasospasm. Inappropriate mobilisation or maturation of the EPCs in SCD may participate to the severity of the disease.
The goal of this research study is to establish chimerism and avoid graft-versus-host disease in patients with Hemoglobinopathies to halt disease progression.