Improving Pain in Sickle Cell Patients With Targeted Antithrombotic Therapy

通过靶向抗血栓治疗改善镰状细胞病患者的疼痛

• 批准号: 8821374
• 负责人: Nirmish R Shah
• 金额: $ 13.34万
• 依托单位: DUKE UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2014
• 资助国家: 美国
• 起止时间: 2014-12-01 至 2017-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8821374
• 关键词: Address; Admission activity; Adult; Age; Anticoagulants; Anticoagulation; Antithrombin III; Award; Childhood; Chronic; Clinical; Clinical Research; Clinical Trials; Coagulation Process; Data; Disease; Dose; Double-Blind Method; Employee Strikes; Extramural Activities; FDA approved; Fatigue; Fibrin fragment D; Fibrinolytic Agents; Frequencies; Funding; Generations; Goals; Health Care Costs; Hematology; Hemostatic function; Hospitalization; Impact evaluation; Inflammation; Information Systems; Interleukin-6; Intervention; Investigation; Laboratories; Lead; Leadership; Low-Molecular-Weight Heparin; Measurement; Measures; Medicine; Mentors; Mentorship; Oral; Organ; Outcome; Outpatients; Pain; Patient Outcomes Assessments; Patient-Controlled Analgesia; Patients; Pharmaceutical Preparations; Phase; Physical Function; Pilot Projects; Placebo Control; Placebos; Positioning Attribute; Principal Investigator; Prophylactic treatment; Quality of life; Questionnaires; Reagent; Recording of previous events; Recruitment Activity; Recurrence; Research; Research Design; Research Methodology; Research Personnel; Resources; SECTM1 gene; Sickle Cell; Sickle Cell Anemia; Solid; Symptoms; Technology; Testing; Therapeutic; Therapeutic Agents; Therapeutic Intervention; Thrombin; Thromboembolism; Thrombophilia; Thrombosis; Time; Training; Translational Research; Treatment Effectiveness; Venous; Visual; analog; arm; career; computerized; double-blind placebo controlled trial; experience; health related quality of life; hydroxyurea; improved; inflammatory marker; innovation; innovative technologies; insight; medical attention; medication compliance; meetings; mobile application; mortality; novel; novel therapeutics; prevent; prophylactic; public health relevance; skills; subcutaneous; translational study

 DESCRIPTION (provided by applicant): Sickle cell disease (SCD) is a chronic hemolytic disorder characterized by recurrent vaso-occlusive episodes (VOC) and progressive organ damage. It strikes at a young age, with a clinical course punctuated by painful disabling crises, yet treatment options are few. Once patients develop VOC, intervention is limited to pain alleviation; no therapy is currently available to alter the course of VOC. Therefore, efforts are needed to evaluate potential therapeutic interventions and determine the potential impact. My research efforts have focused on pediatric and adult patients with SCD, with a specific goal of measuring coagulation by innovative technologies and determining potential therapeutic options. I recently completed a small pilot study evaluating prophylactic dose anticoagulation for patients with SCD admitted for VOC. I found a greater absolute decrease in pain scores and shorter time on patient controlled analgesia, both arguing for further investigations. I now have positioned myself to examine the effect of an oral factor Xa inhibitor administered daily to decrease chronic daily pain and hypercoagulability. Outcomes will include daily pain scores, patient reported outcomes and hypercoagulable markers. To provide a comprehensive evaluation of the impact of pain in patients with SCD, I will use Patient Reported Outcome Measurement Information System (PROMIS), a computerized adaptive questionnaire, to evaluate fatigue, physical function and pain impact. Pain scores will be recorded through the Sickle cell Mobile Application to Record symptoms via Technology (SMART) app. The SMART app was developed by my research group to track symptoms and improve medication adherence. To allow successful completion of the study, I have established solid mentorship with the leaders in coagulation and SCD, in addition to assembling a scientific advisory board to promote further expertise and insight. I will also pursue completion of coursework required to obtain a Masters in Clinical Research, to strengthen my background in clinical research methodology. Support from this award will provide the opportunity for me to gain the necessary experience, didactic training, and mentorship to become a successful and independent researcher in hematology. Importantly, it will also build a framework for my long-term career in academic medicine; performing innovative research evaluating novel therapeutic agents in patients with SCD.

 描述（由申请人提供）：镰状细胞病（SCD）是一种慢性溶血性疾病，其特征为反复发生血管闭塞发作（VOC）和进行性器官损伤。它在年轻时发作，临床过程被痛苦的致残危机打断，但治疗选择很少。一旦患者发生VOC，干预仅限于缓解疼痛;目前没有治疗方法可以改变VOC的病程。因此，需要努力评估潜在的治疗干预措施，并确定潜在的影响。我的研究工作集中在儿童和成人SCD患者，具体目标是通过创新技术测量凝血并确定潜在的治疗方案。我最近完成了一项小型试点研究，评估了因VOC入院的SCD患者的预防性抗凝剂量。我发现疼痛评分的绝对下降幅度更大，患者自控镇痛的时间更短，这两个结果都值得进一步研究。我现在已经定位自己来检查口服Xa因子抑制剂每天给药的效果，以减少慢性日常疼痛和高凝状态。结局将包括每日疼痛评分、患者报告的结局和高凝标志物。为了全面评价疼痛对SCD患者的影响，我将使用患者报告结局测量信息系统（PROMIS），一种计算机化自适应问卷，来评价疲劳、身体功能和疼痛影响。疼痛评分将通过镰状细胞移动的应用程序记录症状，通过技术（SMART）应用程序。SMART应用程序是由我的研究小组开发的，用于跟踪症状和改善药物依从性。为了成功完成这项研究，我与凝血和SCD领域的领导者建立了坚实的指导关系，此外还组建了一个科学咨询委员会，以促进进一步的专业知识和见解。我也将追求完成课程要求获得临床研究硕士学位，以加强我在临床研究方法的背景。从这个奖项的支持将提供机会，让我获得必要的经验，教学培训，并指导成为一个成功的和独立的血液学研究人员。重要的是，它还将为我在学术医学方面的长期职业生涯建立一个框架;进行创新研究，评估SCD患者的新型治疗药物。