Inhaled Mometasone to Promote Reduction in Vaso-occlusive Events (IMPROVE)

吸入莫米松可促进减少血管闭塞事件（改进）

基本信息

批准号：
9304272
负责人：
Jeffrey Avins Glassberg
金额：
$ 19.21万
依托单位：
ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI
依托单位国家：
美国
项目类别：
财政年份：
2013
资助国家：
美国
起止时间：
2013-08-16 至 2018-07-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/9304272
关键词：
Accident and Emergency department Acute Adherence Admission activity Adolescent Adrenal Cortex Hormones Adult Affect American Asthma Attenuated Award Bayesian Analysis Biological Biological Markers Blood Vessels Breathing Caring Cessation of life Characteristics Child Clinic Clinical Clinical Trials Clinical Trials Design Coughing Data Diagnosis Dose Double-Blind Method Emergency Department Physician Emergency department visit Enrollment Event Exhalation Expenditure FDA approved Feasibility Studies Future Goals Hemoglobin Hemolysis Hypoxia Inborn Genetic Diseases Individual Inflammation Injury Institution Intervention Studies Learning Literature Logistics Lung Measures Mentors Methods Modeling Morbidity - disease rate Multicenter Trials Nitric Oxide Observational Study Outcome Outcome Measure Pain Pain-Free Participant Pathogenesis Patient Self-Report Patients Pharmaceutical Preparations Phase Physicians Placebo Control Placebos Play Preparation Principal Investigator Property Publishing Pulmonary Inflammation Randomized Randomized Controlled Trials Rare Diseases Recruitment Activity Recurrence Reporting Research Research Personnel Respiratory Signs and Symptoms Risk Risk Factors Sample Size Sampling Sickle Cell Sickle Cell Anemia Spirometry Structure Subgroup Symptoms Techniques Testing Therapeutic Therapeutic Trials Time Training United States Vascular Cell Adhesion Molecule-1 Visit Wheezing acute chest syndrome age group aged body system career clinical investigation cost design diaries health related quality of life hydroxyurea improved innovation insight meetings mortality patient oriented prevent programs research study simulation skills standard of care therapy development treatment response trend trial design vaso-occlusive pain

项目摘要

DESCRIPTION (provided by applicant): The goal of the proposed studies and structured educational plan is to develop the PI into an independent investigator with the career goal of preventing acute complications and improving emergent management of sickle cell disease (SCD), with focus on learning innovative approaches to trial design to meet challenges specific to SCD research. The Principal Investigator will receive advanced individualized and classroom training that will build upon the candidate's strengths as an emergency physician and prior training in clinical investigation to learn specific quantitative skills necessary to transition ito the next phase of his career, including adaptive trial design methods, Bayesian analysis and longitudinal modeling techniques. SCD is an inherited disorder of hemoglobin with manifestations in all organ systems. One of the greatest risk factors for vaso-occlusion and death in adolescents and adults with SCD is the presence of respiratory symptoms such as cough or wheeze. Preliminary data indicate that recurrent cough or wheeze occurs in nearly half of individuals with SCD who do not carry a physician diagnosis of asthma. The proposed research is designed to test the global hypothesis that inhaled corticosteroids (ICS), a therapy developed to treat asthma, will prevent vaso-occlusive painful episodes in adults with SCD who wheeze, but do not meet criteria for a diagnosis of asthma. The specific aims of this proposal are 1) Conduct a feasibility study - a randomized controlled trial of ICS for adults with SCD who do not meet criteria for a diagnosis of asthma but report recurrent cough or wheezing, 2) Measure the effects of ICS on biological correlates of pulmonary inflammation (as determined by exhaled nitric oxide) and vascular injury (as determined by sVCAM) in SCD, and 3) Compare properties of traditional and Bayesian adaptive clinical trial design for therapeutic trials in SCD in preparation for designing a definitive trial of ICS. These aims have the potentia to 1) change the standard of care for individuals with SCD and recurrent cough or wheeze, 2) provide insight into the pathogenesis of non-asthmatic wheezing in SCD and its response to treatment, 3) explore the suitability of innovative clinical trial designs to overcome the challenges that have hindered therapeutic innovation for SCD.

描述（由申请人提供）：拟议的研究和结构化的教育计划的目的是将PI开发为独立研究者，其职业生涯是防止急性并发症并改善对镰状细胞疾病（SCD）的新兴管理（SCD），重点侧重于学习创新的试验设计方法，以应对SCD研究的挑战。首席研究人员将接受高级的个性化和课堂培训，这些培训将基于候选人作为急诊医师的优势以及临床调查的先前培训，以学习过渡到他职业生涯的下一阶段所必需的特定定量技能，包括自适应试验设计方法，贝叶斯分析和纵向建模技术。 SCD是一种在所有器官系统中表现出来的血红蛋白遗传疾病。 SCD的青少年和成年人中血管封闭和死亡的最大危险因素之一是存在呼吸道症状，例如咳嗽或喘息。初步数据表明，在近一半的SCD患者中，反复咳嗽或喘息发生，这些患者不接受医生诊断哮喘。拟议的研究旨在检验全球假设，即吸入的皮质类固醇（ICS）是一种用于治疗哮喘的疗法，将防止患有喘息的SCD的成年人的血管熟悉的疼痛发作，但不符合诊断性哮喘的标准。该提案的具体目的是1）进行可行性研究 - IC的随机对照试验对患有SCD的成年人的随机对照试验，这些试验不符合诊断哮喘的标准但报告复发性咳嗽或喘息的情况，2）测量IC对肺部炎症的影响（如呼出的氧化物（确定）（如呼出的氧化）（确定）（如呼出的氧化）（确定）（如呼出的氧化）（确定）（如呼出的氧化）（确定））（如SESD）（所确定的）（如呼出）（确定的）（如SED）（确定）（如呼出的氧化）。 SCD治疗试验的传统和贝叶斯自适应临床试验设计的特性，以准备设计最终的ICS试验。这些目的具有预期的1）更改SCD和经常性咳嗽或喘息的人的护理标准，2）提供有关SCD中非质量喘息的发病机理及其对治疗反应的发病机理，3）探索创新的临床试验设计的适用性，以克服越来越多的挑战，以阻碍具有Innovitic in Innovication scd的挑战。

项目成果

期刊论文数量（2）

专著数量（0）

科研奖励数量（0）

会议论文数量（0）

专利数量（0）

Inhaled steroids reduce pain and sVCAM levels in individuals with sickle cell disease: A triple-blind, randomized trial.

DOI：
10.1002/ajh.24742
发表时间：
2017-07
期刊：
American journal of hematology
影响因子：
12.8
作者：
Glassberg J;Minnitti C;Cromwell C;Cytryn L;Kraus T;Skloot GS;Connor JT;Rahman AH;Meurer WJ
通讯作者：
Meurer WJ

Bayesian analyses demonstrate tissue blood volume is not decreased during acute sickle cell pain episodes: A preliminary study.