Comprhensive Sickle Cell Center Program Project

综合镰状细胞中心计划项目

基本信息

批准号：
7355388
负责人：
MARIE J. STUART
金额：
$ 9.77万
依托单位：
THOMAS JEFFERSON UNIVERSITY
依托单位国家：
美国
项目类别：
财政年份：
2003
资助国家：
美国
起止时间：
2003-07-01 至 2008-03-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/7355388
关键词：
3&apos Untranslated Regions 5 year old Acceleration Accident and Emergency department Acute Adherence Adhesions Adolescent Adult African American Age Air Alleles American American Society of Hematology Amino Acids Analgesics Anemia Angiotensinogen Anti-Inflammatory Agents Anti-inflammatory Area Arts Asthma Basic Science Binding Biological Assay Biological Markers Blood Blood Gas Analysis Blood Platelets Blood Vessels Blood specimen Breathing Budgets CD36 gene COS Cells Candidate Disease Gene Carboxyhemoglobin Caring Case Study Categories Cell Adhesion Cell Adhesion Molecules Cell Count Cell membrane Cells Characteristics Chest wall structure Child Child Care Childhood Chronic Cities Clinical Clinical Practice Guideline Clinical Research Clinical Trials Clinical Trials Network Clinical Trials, Other Coagulation Process Collaborations Communication Communities Community Health Education Community Outreach Complex Conflict (Psychology)Cooley&apos s anemia Count Cues Daily Data Data Set Development Diagnostic Discipline of Nursing Disease Disease regression Dissociation Doctor of Philosophy Education Educational Materials Educational process of instructing Elements Elevation Employee Strikes End Point Endothelial Cells Endothelium Enrollment Erythrocytes Evaluation Event Exhalation Exhibits Exposure to Family Fetal Hemoglobin Fluorescence Frequencies Functional disorder Funding Future General Population Generations Genes Genetic Polymorphism Genotype Goals Grant Grouping Guidelines Health Personnel Hematocrit procedure Hemoglobin Hemoglobin SC Disease Hemoglobin SS Hemoglobin concentration result Hemostatic Agents Hemostatic function High Prevalence Hip region structure Hispanics Home environment Hospitals Human Hypertrophy Hypoxemia Hypoxia In Vitro Indium Individual Infant Inflammation Inflammatory Institution Integrins Intervention Iron Overload Judgment Jurkat Cells Kentucky L-Selectin Laboratories Laboratory Study Leukocytes Life Link Liquid substance Longitudinal Studies Lung Master of Public Health Measures Mediating Mediator of activation protein Medical Medicine Methemoglobin Methodology Microcirculation Modality Molecular Biology Monitor Names Network-based New York City Nitric Oxide Nitric Oxide Donors Numbers Nurses Nursing Staff Observational Study Organ Oxygen Oxygen saturation measurement Oxyhemoglobin Pain Pain Assessment Tool Pain Measurement Pain management Palmar-plantar erythrodysesthesia syndrome Paper Parents Partial Pressure Participant Parvovirus Pathogenesis Pathology Patient Care Patient Self-Report Patients Pediatric Hospitals Pennsylvania Personal Satisfaction Persons Pharmaceutical Preparations Phase Phase III Clinical Trials Philadelphia Phosphatidylserines Phospholipids Physicians Physiologic pulse Physiological Plasma Play Point Mutation Polysomnography Population Prevalence Proteins Protocols documentation Psychologist Publications Publishing Pulse Oximetry Pulse taking Purpose Randomized Range Rate Reader Recruitment Activity Report (document)Reporting Research Research Personnel Research Project Grants Respiratory physiology Reticulocytes Risk Role Running Sample Size San Francisco School-Age Population Schools Selectins Series Seroprevalences Services Severities Severity of illness Siblings Sickle Cell Sickle Cell Anemia Sickle Hemoglobin Signal Transduction Site Sleep Sleep Apnea Syndromes Societies Spirometry Staging Steroids Stress Students Supplementation Surface Survival Analysis Symptoms Syndrome System TFRC gene Techniques Testing Therapeutic Therapeutic Intervention Thrombin Thrombophilia Thrombosis Time Tonsil Transactivation Transfusion Translating Transplantation United States National Institutes of Health Universities University Hospitals Up-Regulation Update Variant Vascular Cell Adhesion Molecule-1 Venous blood sampling Work Zinc abstracting acute chest syndrome age group airway hyperresponsiveness airway obstruction artist base cell type cerebrovascular chronic pain clinical research site cohort college concept coping day diaries dyshemoglobins editorial experience falls grandparent hydroxyurea in vivo inhaled nitric oxide innovation interest macrovascular disease medical schools member microchip new technology novel pediatric department phosphatidylserine receptor polymerization prevent programs prophylactic prospective protective effect pulmonary function sickling skills success surfactant symposium tool trial comparing virtual

项目摘要

I. > The fundamental challenge of sickle cell disease (SCD)is how a point mutation, which changes a single amino acid in a single protein, in a single circulating cell, causes a disease with protean manifestations, and complex and unpredictable clinical symptoms. The Marian Anderson Comprehensive Sickle Cell Center has been using the first few years of life, a physiologically crucial period when infants with SCD manifest high levels of HbF, to longitudinally evaluate whether potentially important relationships exist between HbF and other biologic parameters related to SCD pathophysiology, and has demonstrated important relationships between HbF, fluid phase coagulation and adhesion markers. The proposed continuation of this prospective, longitudinal study of infants and children (3 months to 4 yrs) will provide critical new information on the importance of specific biologic markers as they relate to the pathophysiology of the microvessel occlusive phenomenon. In addition, these longitudinal studies will create a "biologic footprint as the infant grows, providing a unique look at the temporal sequence of changes in adherence, endothelial, platelet, white cell and hemostatic activation in the infant and young child as the protective effects of HbF decline, and the subject begins to experience the unfolding systemic effects of HbS polymerization including anemia and pain. An additional clinical project not only provides the fundamental information on the pain experienced by these infants and young children needed to make the biologic and physiologic correlates, it also proposes using innovative new technologies to facilitate clinical and research communication "_L-V"V_I ..... I =Igl.ll.lll._.g;, _.-,.o;II.IV.._.g_I_Ue_tV;Id,_, cit,,_r¿_ _UllIrUl_ *_1-.1I1;1_,_;1P¿=l ¿U__Utl_l, iFF T¿it=,Iv Iutbt,i,,l_ity _v,f t-,h_v=v=v 4:_= t..=....._.....h....r....t..r...t...lngil=_will hmhwthP.r demonstrated in the development and evaluation of a parent-mediated pain management protocol for young children with SCD. An examination of endothelial cell receptors for PS-positive sickle erythrocytes will serve as the Center's basic science study and combines new information obtained by current Center investigators with innovative molecular biology studies assisted by investigators new to the Center. This unique blend of meticulous clinical care and research is highlighted in the collaborative network protocol which proposes a clinical trial comparing hydroxyurea to hydroxyurea and phlebotomy in SC disease using statistical techniques and preliminary data developed from the Center's previous pain studies, and detailed laboratory studies to help understand the pathophysiology of this poorly understood sickle syndrome. Supporting these studies is a clinical core of dedicated staff that also supports the Center' growing patient population, which now includes adult and pediatric patients inPhiladelphia, and a virtual Center at the University of Louisville in Kentucky. Rounding out the Center is a patient services core with a focus on translating our state-of-the-art research and patient care into practice through education and community outreach in Pennsylvania and Kentucky.