Optimizing Treatment Decisions for Acute Demyelinating Optic Neuritis

优化急性脱髓鞘性视神经炎的治疗决策

• 批准号: 10237984
• 负责人: Lindsey Blake De Lott
• 金额: $ 20.76万
• 依托单位: UNIVERSITY OF MICHIGAN AT ANN ARBOR
• 依托单位国家: 美国
• 财政年份: 2018
• 资助国家: 美国
• 起止时间: 2018-09-30 至 2023-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10237984
• 关键词: Acute; Address; Adherence; Adopted; Adrenal Cortex Hormones; Adverse event; Behavior; Belief; Benefits and Risks; Big Data; Blindness; Caring; Clinical; Clinical Trials Design; Complex; Country; Coupled; Data; Databases; Decision Aid; Decision Making; Decision Support Systems; Development; Disease; Evidence based practice; Evidence based treatment; Eye; Eye diseases; Foundations; Funding; Goals; Health Policy; Health Services Research; Hospital Costs; Hospitalization; Individual; Infusion procedures; Injury; Institutes; Intervention; Intravenous; Mentorship; Methodology; Methods; Michigan; Mission; Modeling; Modernization; Neurologic; Neurology; Ophthalmology; Optic Neuritis; Optical Coherence Tomography; Outcome; Outpatients; Patient Care; Patient Rights; Patients; Perception; Pharmaceutical Preparations; Physician' s Role; Physicians; Prognosis; Prospective Studies; Provider; Psychologist; Randomized Clinical Trials; Recovery; Registries; Research; Research Personnel; Right to Treatments; Shapes; Structure; Subgroup; Techniques; Testing; Thick; Training; Translational Research; Treatment Efficacy; United States National Institutes of Health; Universities; Variant; Vision; Visual; Work; acceptability and feasibility; base; care providers; career; career development; clinical practice; design; disability; evidence base; functional outcomes; individual patient; innovation; multidisciplinary; personalized medicine; programs; response; retinal nerve fiber layer; risk stratification; side effect; simulation; social norm; theories; therapy design; tool; treatment effect; treatment optimization; treatment risk; treatment trial; usability; young adult

ABSTRACT: A wide gap exists between the evidence for acute demyelinating optic neuritis (ON) treatment and physicians’ clinical practice. The proposed research seeks to narrow this gap by designing an intervention to promote evidence-based, personalized treatment decisions. ON can cause permanent vision loss, but varia- tion exists in the visual prognosis. The Optic Neuritis Treatment Trial (ONTT) showed that, for the average pa- tient, intravenous corticosteroids did not improve visual outcomes, but may hasten recovery. This modest aver- age corticosteroids benefit comes with potential harms: medication side effects, and the inconvenience and cost of hospital admissions or outpatient infusions. Furthermore, the ONTT lacks modern neuro-axonal struc- tural outcomes and adequately powered prospective studies are lacking. Despite the variability in prognosis, modest benefits/risks of treatment, and limited understanding of corticosteroids effects on structural outcomes, providers treat nearly all patients with intensive corticosteroid therapy--adopting a “one-size-fits-all” approach to treatment. Personalizing ONTT data using multivariable risk stratified analyses and simulation analyses of neuroaxonal structural outcomes has great potential to inform individual treatment decisions. This strategy cir- cumvents the weaknesses of subgroup analyses, by examining the complex ways that multiple clinical factors shape an individual’s overall risks/benefits from treatment. However, information alone is not enough to change decision-making behavior. Perceptions about the treatment are important determinants of decision making be- havior. The objective of this proposal is to design a decision support intervention with personalized risk/benefit information that is informed by physician’s treatment perceptions to promote more nuanced, evidence-based ON treatment decisions. This objective will be addressed through 3 specific aims. (1) Establish the key treat- ment perceptions underlying physician’s ON decision making using a theory-based, mixed methods approach. (2) Develop models of the risks/benefits of corticosteroids at the individual level. (3) Use the results from Aims 1 and 2 to develop and pilot the usability, feasibility and acceptability of a decision support intervention. Dr. De Lott has a unique background in neuro-ophthalmology and health services research. She will develop addi- tional expertise in multivariable risk stratified analyses, simulation analyses, mixed methods, clinical trials, and design of decision support systems to become an independently-funded investigator committed to developing strategies to optimize evidence-based care. This proposal capitalizes on the unique strengths of the University of Michigan: Neurology and Ophthalmology Health Services Research Programs and the Institute for Health Care Policy and Innovation-among the largest academic health services research centers in the country. Dr. De Lott will be supported by an outstanding multi-disciplinary mentorship team: Dr. Kevin Kerber, a leader in neurological health services research, Dr. Paul Lee, an expert in T3 translational research for eye care, and Dr. Brian Zikmund-Fisher, a decisional psychologist and leader in decision aid development.

摘要：急性脱髓鞘性视神经炎 (ON) 治疗的证据之间存在很大差距 以及医生的临床实践。拟议的研究旨在通过设计干预措施来缩小这一差距 促进基于证据的个性化治疗决策。 ON 可导致永久性视力丧失，但变化 化存在于视觉预后中。视神经炎治疗试验 (ONTT) 表明，对于平均 静脉注射皮质类固醇并不能改善视力结果，但可能会加速康复。这个谦虚的平均 年龄皮质类固醇的好处伴随着潜在的危害：药物副作用以及不便和 住院或门诊输液的费用。此外，ONTT 缺乏现代神经轴突结构 缺乏实际结果和充分有力的前瞻性研究。尽管预后存在差异， 治疗的益处/风险不大，并且对皮质类固醇对结构结果影响的了解有限， 提供者对几乎所有患者进行强化皮质类固醇治疗——采用“一刀切”的方法 来治疗。使用多变量风险分层分析和模拟分析对 ONTT 数据进行个性化 神经轴突结构结果具有为个体治疗决策提供信息的巨大潜力。这一策略围绕 通过检查多个临床因素的复杂方式，弥补了亚组分析的弱点 塑造个人治疗的总体风险/收益。然而，仅靠信息不足以改变 决策行为。对治疗的看法是决策的重要决定因素 行为。该提案的目标是设计具有个性化风险/收益的决策支持干预措施 根据医生的治疗观念提供的信息，以促进更细致、基于证据的治疗 ON 治疗决定。这一目标将通过 3 个具体目标来实现。 （一）制定重点治理措施 医生使用基于理论的混合方法做出 ON 决策的认知。 (2) 建立个体水平上皮质类固醇的风险/益处模型。 (3) 使用 Aims 的结果 1 和 2 开发和试点决策支持干预的可用性、可行性和可接受性。德博士 洛特在神经眼科和健康服务研究方面拥有独特的背景。她将开发附加功能 多变量风险分层分析、模拟分析、混合方法、临床试验和 设计决策支持系统，成为一名独立资助的研究者，致力于开发 优化循证护理的策略。该提案充分利用了大学的独特优势 密歇根州：神经学和眼科健康服务研究项目和健康研究所 护理政策和创新——全国最大的学术健康服务研究中心之一。博士。 德洛特将得到一支杰出的多学科导师团队的支持：Kevin Kerber 博士，他是 神经健康服务研究，眼睛护理 T3 转化研究专家 Paul Lee 博士和 Brian Zikmund-Fisher，决策心理学家和决策辅助开发领域的领导者。