View clinical trials related to Anemia, Sickle Cell.
Filter by:Our goal is to improve the self-management of pain and stress in adult outpatients with sickle cell disease (SCD) by determining the feasibility of a self-managed guided relaxation (GR) stress reduction intervention using a tablet-based mobile device. Currently, opioid analgesics are primarily used to treat SCD pain while self-managed behavioral modalities such as GR, are rarely used. Little is known about the effects or mechanisms of GR on pain and stress, in adults with SCD. Emerging evidence from the hypothalamic pituitary adrenal (HPA) axis theory offer insights for understanding the mechanisms. Adding GR as a supplement to analgesic therapies will address the paucity of self-management strategies for controlling pain in SCD. GR is a simple and cost-effective non-drug intervention that could reduce pain and stress in outpatients with SCD. GR is an intervention where outpatients with SCD are directed to listen to and view audio-visual recordings while they visualize themselves being immersed in that scene.
The goal of this research study is to improve the self-management of pain, stress, and cognitive/affective symptoms that may result in adult inpatients with sickle cell disease (SCD) by determining the feasibility of a self-management guided relaxation (GR) stress reduction intervention using a tablet-based mobile device. Currently, opioid analgesics are primarily used to treat SCD pain while self-managed behavioral modalities such as GR, are rarely used, particularly, in inpatient settings. Little is known about the effects or mechanisms of GR on pain, stress, and cognitive/affective symptoms in adults with SCD hospitalized with pain. Emerging evidence from the hypothalamic pituitary adrenal (HPA) axis theory offer insights for understanding the mechanisms. Adding GR as a supplement to analgesic therapies will address the dearth of self-management strategies for controlling pain in SCD. GR is a simple and cost-effective non-drug intervention that could reduce pain and stress in inpatients with SCD. GR is an intervention where inpatients with SCD are directed to listen to and view audio-visual recordings while they visualize themselves being immersed in that scenario.
The purpose of this study is to evaluate the relationship between plasma DNA levels and micro- and macro-circulatory vascular remodelling in patients with sickle cell disease
The purpose of this study is to collect and store samples and health information for current and future research to learn more about the causes and treatment of blood diseases. This is not a therapeutic or diagnostic protocol for clinical purposes. Blood, bone marrow, hair follicles, nail clippings, urine, saliva and buccal swabs, left over tissue, as well as health information will be used to study and learn about blood diseases by using genetic and/or genomic research. In general, genetic research studies specific genes of an individual; genomic research studies the complete genetic makeup of an individual. It is not known why many people have blood diseases, because not all genes causing these diseases have been found. It is also not known why some people with the same disease are sicker than others, but this may be related to their genes. By studying the genomes in individuals with blood diseases and their family members, the investigators hope to learn more about how diseases develop and respond to treatment which may provide new and better ways to diagnose and treat blood diseases. Primary Objective: - Establish a repository of DNA and cryopreserved blood cells with linked clinical information from individuals with non-malignant blood diseases and biologically-related family members, in conjunction with the existing St. Jude biorepository, to conduct genomic and functional studies to facilitate secondary objectives. Secondary Objectives: - Utilize next generation genomic sequencing technologies to Identify novel genetic alternations that associate with disease status in individuals with unexplained non-malignant blood diseases. - Use genomic approaches to identify modifier genes in individuals with defined monogenic non-malignant blood diseases. - Use genomic approaches to identify genetic variants associated with treatment outcomes and toxicities for individuals with non-malignant blood disease. - Use single cell genomics, transcriptomics, proteomics and metabolomics to investigate biomarkers for disease progression, sickle cell disease (SCD) pain events and the long-term cellular and molecular effects of hydroxyurea therapy. - Using longitudinal assessment of clinical and genetic, study the long-term outcomes and evolving genetic changes in non-malignant blood diseases. Exploratory Objectives - Determine whether analysis of select patient-derived bone marrow hematopoietic progenitor/stem (HSPC) cells or induced pluripotent stem (iPS) cells can recapitulate genotype-phenotype relationships and provide insight into disease mechanisms. - Determine whether analysis of circulating mature blood cells and their progenitors from selected patients with suspected or proven genetic hematological disorders can recapitulate genotype-phenotype relationships and provide insight into disease mechanisms.
The primary goal of the study is to determine the safety and tolerability of ambrisentan. It is also expected that ambrisentan will improve blood flow in the lungs, decrease inflammation, and reduce pain in sickle cell patients. An additional goal is to evaluate the use of select biomarkers in evaluating sickle nephropathy.
This is a retrospective cohort study of Sickle Cell Disease (SCD) patients attending 32 treatment centers across Italy. The aim of this study will be to report the Italian experience with the use of hydroxyurea in a large cohort of SCD patients and to evaluate the benefits and safety of this intervention for the prevention and management of a wide range of clinical morbidities
Sickle cell disease (SCD) is the most common inherited blood disorder affecting 80,000 to 90,000 individuals in the United States.[10] There are 13,000 hospital admissions for a sickle cell crises, costing $448 million dollars annually.[10] In our hospital, the sickle cell population is known to have some of the longest length of stays. Between October 2014 and September 2015, there were 89 admissions for a vaso-occlusive crisis with an average length of stay of 6 days and 12 admissions greater than 10 days and 5 admissions greater than 20 days. We propose to evaluate the feasibility of the new CPP in a pilot randomized control trial to determine if pain and length of stay can be reduced in patients with sickle cell disease. We also propose to evaluate a sleep regimen to determine if this can reduce the hospital stay and help with pain. We hypothesize increased physical activity and proper sleep, as implemented in the CPP, are correlated with decreased hospital length of stay and decreased pain. Additionally, we believe that creating a standardized nighttime environment at the hospital will help the children stay in their circadian rhythm thus promoting improved sleep and a more effective inpatient disease management.
Sickle cell disease is a genetic red blood cell disorder characterized by vaso-occlusion from sickling of red blood cells, that can lead to pain or organ complications such as acute chest syndrome. Sickle cell disease is associated with low amounts of nitric oxide, a compound important for dilating the blood vessel wall. Citrulline is a substance that is known to increase nitric oxide. The goal of this Phase I study are to find the highest safe dose of continuous IV citrulline that can be given to individuals with sickle cell disease experiencing a sickle cell pain crisis or acute chest syndrome without causing severe side effects.
The aim of this study is to evaluate the overall safety and feasibility of using haploidentical or one antigen mismatch unrelated hematopoietic stem cell transplant (HSCT) for adult patients with severe sickle cell disease (SCD) who undergo a non-myeloablative preparative regimen consisting of total body irradiation (TBI), cyclophosphamide and alemtuzumab (and fludarabine for haplo-SCT only) and graft vs. host disease (GvHD) prophylaxis consisting of post-transplant cyclophosphamide (PT-Cy), mycophenolate mofetil (MMF), and sirolimus. The investigators anticipate that this approach will expand the donor pool and offer a safe and less toxic curative intervention.
This is a follow-up trial to NYMC 526 (NCT01461837) to assess the safety, efficacy and toxicity of administering Defibrotide prophylaxis for high-risk sickle cell or beta thalassemia patients undergoing a familial haploidentical allogeneic stem cell transplantation with CD34 enrichment and T-cell addback. This patient population historically has a risk of developing sinusoidal obstructive syndrome (SOS) and Defibrotide has demonstrated efficacy in treatment of SOS. The Funding Source is FDA OOPD.