Optimizing Treatment Decisions for Acute Demyelinating Optic Neuritis

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 和医生的临床实践。拟议中的研究试图通过设计一种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，但各种... 视力预后存在一定的差异。视神经炎治疗试验（ONT）显示，对于平均年龄， 然而，静脉注射皮质类固醇不能改善视力，但可能加速恢复。这个谦虚的断言- 年龄皮质类固醇的好处伴随着潜在的危害：药物副作用，和不便， 住院或门诊输液的费用。此外，ONT缺乏现代神经轴突结构， 缺乏合理的结果和足够有力的前瞻性研究。尽管预后存在差异， 治疗获益/风险适中，对皮质类固醇对结构结局的影响了解有限， 提供者采用“一刀切”的方法对几乎所有患者进行强化皮质类固醇治疗 接受治疗使用多变量风险分层分析和模拟分析个性化ONT数据 神经轴突结构的结果有很大的潜力，为个人治疗决策提供信息。这一战略将- 通过检查多种临床因素的复杂方式， 塑造个人从治疗中获得的总体风险/收益。然而，单靠信息还不足以改变现状 决策行为。对治疗的看法是决策的重要决定因素， 你好本提案的目的是设计具有个性化风险/获益的决策支持干预措施 信息是由医生的治疗观念，以促进更细致入微，循证 关于治疗的决定。这一目标将通过三个具体目标来实现。(1)建立关键治疗- 使用以理论为基础的混合方法方法来评估医生的ON决策。 (2)在个体水平上建立皮质类固醇的风险/获益模型。(3)使用Aims的结果 1和2，以开发和试验决策支持干预措施的可用性、可行性和可接受性。德博士 洛特在神经眼科学和卫生服务研究方面有着独特的背景。她会发展出更多的- 多变量风险分层分析、模拟分析、混合方法、临床试验和 决策支持系统的设计，成为一个独立资助的调查致力于发展 优化循证护理的战略。这项建议充分利用了大学的独特优势 密歇根州：神经病学和眼科健康服务研究计划和健康研究所 护理政策和创新-全国最大的学术卫生服务研究中心之一。博士 De Lott将得到一个杰出的多学科导师团队的支持：Kevin Kerber博士， 神经健康服务研究，保罗李博士，在T3转化研究的眼睛护理专家，和博士。 Brian Zikmund-Fisher是一位决策心理学家，也是决策辅助开发的领导者。

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.