View clinical trials related to Anemia, Sickle Cell.
Filter by:Fludarabine and clofarabine are chemotherapy drugs used extensively in bone marrow transplantation. The goal of this study is to determine what causes some children to have different drug concentrations of clofarabine and fludarabine in their bodies and if drug levels are related to whether or not a child experiences severe side-effects during their bone marrow transplant. The hypothesis is that clinical and individual factors cause changes in clofarabine and fludarabine drug levels in pediatric bone marrow transplant patients and that high levels may cause severe side-effects.
Stroke is a frequent complication of sickle cell disease (SCD), with varying levels of central nervous system (CNS) involvement. The summation of several ischemic events, even when silent, can lead to devastating consequences, from reduced academic performance to physical dependence. Despite knowledge that brain flow velocities evaluated by Doppler ultrasound identify pediatric SCD patients at a greater stroke risk (Adams et al, NEJM 1998; 339:5-11), this method is not able to predict the occurrence of strokes in adults. There is also no consensus on the management of adult patients in relation to primary and secondary prevention. The aim of this study is to evaluate the effects of the administration of Simvastatin on CNS structural and functional vascular changes in 30 adult patients with SCD (SS and Sβ), above 35 years of age, observed through Magnetic Resonance Imaging (MRI). The data on the effect of simvastatin on disease manifestations is quite scarce, however this drug reportedly significantly reduces plasma concentrations of adhesion molecules and inflammatory markers, such as E-selectin, VEGF, CRP and IL-6 (Hoppe et al, BJH 2011; 153:655-663; Hoppe et al, BJH 2017;177:620-629). Thus, in addition to the search for early diagnostic markers and risk stratification for primary or recurrent stroke, we will also compare CNS images before and 12 months after the administration of Simvastatin. The drug alter stroke recurrence rates in the general adult population, but their effects on vascular changes in patients with SCD have not yet been adequately elucidated. This is particularly important because these are low cost drugs which present good tolerability, and could be part of the therapeutic arsenal of SCD, even in low income settings. Concomitantly with the CNS evaluation, this study also intends to investigate molecular pathways that may be affected by the drugs. We will evaluate microvesicle release patterns, as well as the content of microRNAs possibly involved in the occurrence of stroke, in addition to metabolomic studies and plasma cytokine profile.
This multi-center study will compare the effectiveness of adding virtual peer mentoring (PM) to a structured education-based (STE) transition program for emerging adults with sickle cell disease to determine its effect on decreasing the number of acute care visits per year, improving patient-reported outcomes, and reducing healthcare utilization among emerging adults with sickle cell disease (EA-SCD)
This multisite prospective study seeks to determine if HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus (Sickle transplant Using a Nonmyeloablative approach, "SUN") can decrease the toxicity of transplant while achieving a high cure rate for children with sickle cell disease (SCD).
The purpose of this proposal is to integrate family-centered self-management strategies with mobile health (mHealth) technology to improve reach, self-management behaviors, and child and caregiver physical and psychosocial symptoms and quality of life. Specifically, the investigators propose to conduct feasibility testing of SMYLS, which has been adapted based on user feedback in the first phase of this study. First the investigators will work with the Medical University of South Carolina (MUSC) Pediatric Sickle Cell Clinic to identify and recruit families with children with sickle cell disease (SCD) in the community, statewide. Next, the investigators will test the feasibility of the intervention with 30 dyads of children ages 8 - 17 with sickle cell disease and their parent or primary caregiver, (N=60)
The trial is a prospective, randomized study to determine the efficacy of training for use of a web based Decision Aid for Hydroxyurea(HU) usage among subjects with Sickle Cell Disease(SCD). Subjects are randomized primarily either to using a web based decision aid with training versus without training. Subjects in each group will be further randomized to 1) subjects receiving pretest surveys; and 2) subjects that do not receive pretest surveys.
Open Label Extension Study of Voxelotor Clinical Trial Participants with Sickle Cell Disease Who Participated in Voxelotor Clinical Trials
Background: Sickle cell disease (SCD) is a common inherited blood disorder. Many people with SCD are at risk to get pulmonary hypertension (PH). PH means that the blood pressure in the blood vessels to the lungs is high, and is a serious disease and. Very few studies have looked at the success of treatments for PH in people with SCD. Researchers want to learn more about treating PH with a type of drug called phosphodiesterase type 5 inhibitors (PDE5-I). They will look at the records of people who have already joined other studies. Objective: To identify people who already joined NIH SCD protocols whose medical records should be reviewed. The review will look at the description of SCD patients with PH who have already taken PDE5-I and the outcomes for these people. Eligibility: Adults ages 18 and older with SCD and PH. They must have joined certain NIH studies and taken PDE5-I therapy for at least 16 weeks. Design: This study is a review of medical records. Researchers will collect data from databases of existing studies. They will identify people in those studies who have SCD and PH and took the study drug for at least 16 weeks. Researchers will review the full medical records of those people. From that review, researchers will find participants who meet the inclusion criteria. They will extract data from those records. Researchers will analyze the data. This includes results from heart and lung tests, imaging, and walking tests. It will also include results of a procedure called right heart catheterization. Demographic data and lab data will also be collected. Researchers will remove identifying information from the data, then share it in a database.
The purpose of this pilot study is to investigate the effects of a 6-session music therapy protocol on the pain, mood, quality of life, coping skills, and self-efficacy of adult patients with sickle cell disease (SCD) as compared to adult patients with SCD who receive standard care alone. The investigators will also determine the feasibility (delivery, acceptability, and usefulness) of the music therapy intervention for pain management and quality of life.
The purpose of this study is to determine whether IV acetaminophen can decrease the need for subsequent opioid administration in the acute management of sickle cell crisis pain in the pediatric emergency room.