View clinical trials related to Anemia, Sickle Cell.
Filter by:In this protocol, the investigators propose to evaluate the biochemical effects of imatinib on sickle red blood cells (RBCs). Patients will be administered imatinib mesylate orally following the guidelines previously established for use of imatinib in other disorders. The biochemical effects of imatinib on sickle RBCs will be examined, including changes in their levels of band 3 tyrosine phosphorylation and the abundances of RBC-derived microparticles in their blood. In addition, the patients will be monitored for symptoms of sickle cell disease (SCD). The investigators expect band 3 tyrosine phosphorylation to decrease dramatically in patients treated with imatinib. The investigators also anticipate a reduction in the numbers of RBC-derived microparticles in circulation (quantitated by assaying the number of glycophorin A positive microparticles in peripheral blood samples by flow cytometry. Most importantly, the investigators expect to see a reduction in the frequency of vaso-occlusive crises, and possibly acute chest syndrome and utilization of opioids. The study duration is planned as 6 months in order to provide adequate time for potential change in the primary endpoints (e.g. percent irreversibly sickled cells).
TAK-755 (previously known as SHP655) is a medicine used to treat sickle cell disease (SCD). The main aim of the study is to measure the safety and tolerability of TAK-755 in SCD participants. Study participants will receive TAK-755 or placebo on Day 1. Their SCD will be treated by their doctor according to their doctor's usual clinical practice. During the study, participants will be asked to follow-up on 13 days following SHP655 or placebo administration for safety assessment. Maximum duration of participation is expected to be about 2 months.
The iPeer2Peer Sickle Cell Disease (SCD) study matches youth (12-18 years of age) with SCD to a mentor (trained young adult) who has learned to manage their SCD well, transitioned to adult care, and can support youth participants emotionally and socially. Participants will be randomly assigned one of two groups, either (1) The intervention group: Study group participants are matched with a mentor for 15 weeks, and are expected to have up to ten calls with one another; (2) The control group: This study group will be on a 15 week waitlist to receive a mentor. This study will first assess the feasibility of conducting this research with youth with SCD. Also, this study will assess the preliminary effectiveness of peer mentorship by comparing various health outcomes of the two study groups post-intervention.
Three methods are actually used in newborn screening for sickle cell disease (SCD) in France: isoelectric focusing, high performance liquid chromatography and capillary electrophoresis. New technologies are currently under development such as Matrix Assisted Laser Desorption Ionisation - Time of Flight (MALDI-TOF) and tandem mass spectrometry (MS/MS) using the SpOtOn Diagnostics Reagent Kit available in United Kingdom only. Zentech company (Liège, Belgium) is developing a package for SCD newborn screening using MS/MS technology. The main objective of the present study will be to compare this new technique with the technique actually used in the hospital center of Lille (sub-contractor for SCD newborn screening of Lyon) and the haemoglobin analysis to test its accuracy (sensitivity and specificity).
This study is designed to address the feasibility of a randomized, double masked, cross-over study of dronabinol as a palliative agent in the treatment of pain, inflammation, and other complications of sickle cell disease (SCD).
Sickle cell disease is the most common genetic disease in the world. It results in the synthesis of an abnormal hemoglobin (HbS) which, in its deoxygenated form, polymerizes and causes structural changes in red blood cells (RBCs). They become more rigid and less deformable. The fragility of sickle-cell RBCs leads to their massive destruction, leading to chronic anemia (i.e. low hemoglobin in the blood) and to low tissue oxygenation. More rigid and less deformable, sickle-shaped RBCs tend to obstruct microvessels, leading to particularly painful vaso-occlusive crisis (VOC), which can cause organ failure (spleen, kidneys, brain, lungs, heart, liver, bone ...) and patient's lifethreatning. A preliminary work on red blood cells of sickle cell patients showed alteration of a parameter measuring the overall deformability of RBCs by assessing the nature of their movement in a shear flow. This parameter is altered sickle cell patients at basal state compared to a population of healthy individuals. This alteration is increased when sickle cell patients are in crisis. The main objective of this project is to study the evolution of this parameter in sickle cell patients according to their health status (basal state vs vaso-occlusive crisis). The investigators hypothesize that the alteration of the RBC deformability parameter is significant before symptoms of vaso-occlusive crisis (several hours to several days). The main objective is a weekly analysis of the evolution of the parameter in 30 sickle cell patients (SS or SB°) in the basal state and daily in at least 6 patients at the beginning, during and just after a vaso-occlusive crisis. The comparison between the parameter measured in a subject in the basal state and in the same subject in crisis will be performed. The criteria for the presence of a vaso-occlusive crisis were: the appearance of a attacks of pain affecting at least two territories +/- fever> 38.3 +/- dyspnea and / or sputum. The investigators will differentiate the moderate VOC managed at home with low-level analgesics and VOC requiring hospitalization. The number of days of hospitalization, the occurrence of thrombotic complications, the degree of anemia and hemolysis will be noted. The measurement of the parameter will be performed on a capillary sampling of 40 microliters performed at the fingertip, weekly outside crises and daily when a crisis occurs.
Sickle cell disease (SCD) is characterized by recurrent vaso-occlusive pain crisis (VOC), which may evolve to acute chest syndrome (ACS), the most common cause of death among adult patients with SCD. Currently, there is no safe and effective treatment to abort VOC or prevent secondary ACS. Management of VOC mostly involve a symptomatic approach including hydration, analgesics, transfusion, and incentive spirometry, which was investigated in a very limited number of patients (<30). The polymerisation of HbS is one major feature in the pathogenesis of vaso-occlusion. Among factors determining the rate and extent of HbS polymer formation, the hypoxic stimulus is one of the most potent and readily alterable. Current guidelines recommend oxygen therapy in patients with VOC in order to maintain a target oxygen saturation of 95%. Low-flow nasal oxygen (LFNO) is routinely used to achieve this normoxia approach, particularly in patients at risk of secondary ACS because they may experience acute desaturation. In contrast, various case series suggest a potential beneficial role of intensified oxygen therapy targeting hyperoxia for the management of VOC, particularly with the use of hyperbaric oxygen, but the latter is difficult to implement in routine clinical practice. A recent high-flow nasal oxygen (HFNO) technology allows the delivery of humidified gas at high fraction of inspired oxygen (FiO2) through nasal cannula. The FiO2 can be adjusted up to 100% (allowing hyperoxia that may reverse sickling) and the flow can be increased up to 60 L/min (which generates positive airway pressure and dead space flushing, that may prevent evolution of VOC towards ACS by alleviating atelectasis and opioid-induced hypercapnia). In patients with acute respiratory failure, HFNO has been shown to improve patient's comfort, oxygenation, and survival as compared to standard oxygen or non-invasive ventilation. The aim of the present study is to test the efficacy and safety of HFNO for the management of VOC and prevention of secondary ACS. The investigators will use a multi-arm multi-stage (MAMS) design to achieve these goals. HFNO will be delivered through AIRVO 2 (Fisher and Paykel Healthcare, New Zealand), a device that incorporates a turbine allowing its use in hospital wards.
Sickle Cell Disease (SCD) is a serious inherited blood disorder affecting red blood cells. When oxygen levels drop the red cells become abnormally shaped and unable to move through the blood vessels easily. Blood and oxygen do not reach body organs, resulting in episodes of severe pain and other complications. Pregnant women with SCD have an increased risk of both sickle and pregnancy complications, including raised blood pressure. Their babies may grow more slowly in the womb, are more likely to be born early and need special care, and have a higher risk of dying. The only treatments currently available for women with SCD are Hydroxycarbamide (which cannot be used during pregnancy) and blood transfusion. Currently, blood transfusion is only used during pregnancy to treat emergency complications. It has been suggested that giving blood transfusions throughout pregnancy could improve outcomes for both mother and babies. In Serial Prophylactic Exchange Blood Transfusion (SPEBT), sickle blood is mechanically removed and simultaneously replaced with donor red cells. A trial is needed to assess SPEBT given every 6-10 weeks, starting before 18 weeks of pregnancy, compared to standard care. This trial will evaluate outcomes for women (e.g. hospital admission, frequency of crisis) and their infants (e.g. early delivery, birthweight). However, the feasibility of such a study needs to be assessed before embarking on a large multicentre trial. This study is therefore a feasibility study in which we will randomly allocate participants to have either SPEBT or standard care. The study will be carried out in multiple maternity units in England and last two years. The willingness of eligible women to join the study will be assessed, along with how many participants remain part of the study until the end and if participants find the intervention acceptable.
Sickle cell disease (SCD) necessitates a paediatric treatment plan that considers the influence of psychological, family and intercultural factors. At the Louis-Mourier Hospital (APHP) in Colombes, France, a paediatric-psychological partnership where a clinical psychologist accompanies the paediatrician at programmed consultations was introduced. The psychological repercussions of SCD were assessed among children and their parents treated in Colombes and in two other paediatric units without a paediatric-psychological partnership.
ATS (acute thoracic syndrome) refers to acute pulmonary involvement in a sickle cell patient. The diagnosis is based on the association of clinical signs (fever or respiratory symptoms) with a recent pulmonary infiltrate on the chest x-ray. The main objective of the study is to evaluate the place of the pulmonary ultrasound for the diagnosis of ATS, in comparison with frontal chest x-ray.