Surgical IIH Treatment Trial

IIH 手术治疗试验

• 批准号: 9518911
• 负责人: Mark J Kupersmith
• 金额: $ 450.57万
• 依托单位: ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI
• 依托单位国家: 美国
• 财政年份: 2017
• 资助国家: 美国
• 起止时间: 2017-07-01 至 2020-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9518911
• 关键词: Acetazolamide; Address; Affect; American; Basement membrane; Biomechanics; Biometry; Blindness; Cerebrospinal Fluid Pressure; Cerebrospinal fluid shunts procedure; Clinical Trials Design; Complication; Data; Development; Diet; Disease; Dose; Enrollment; Epidemic; Epithelial; Evidence based treatment; Eye; Female of child bearing age; Ganglion Cell Layer; Goals; Healthcare; Image; Incidence; Individual; Institution; Intention; Intervention; Intracranial Hypertension; Intracranial Pressure; Investigation; Leadership; Masks; Measures; Medical; Methods; Nerve Fibers; Nerve Sheaths; Newly Diagnosed; Obesity; Operative Surgical Procedures; Ophthalmology; Optic Disk; Optic Nerve; Optical Coherence Tomography; Outcome; Overweight; Papilledema; Patients; Perimetry; Placebos; Predictive Value; Probability; Pseudotumor Cerebri; Qualifying; Quality of life; Randomized; Reading; Regimen; Research; Research Design; Research Infrastructure; Research Personnel; Resources; Retina; Retinal; Retinal Ganglion Cells; Retinal Pigments; Site; Testing; Thick; Thinness; Time; Treatment Failure; Treatment outcome; Vision; Visit; Visual; Visual Fields; Weight maintenance regimen; Woman; comparative efficacy; evidence base; experience; fiber cell; high risk; improved; placebo controlled study; pressure; prevent; primary outcome; programs; prospective; secondary outcome; success; treatment group; treatment strategy; treatment trial; young woman

PROJECT SUMMARY / ABSTRACT Idiopathic intracranial hypertension (IIH) is a disorder of obese young women characterized by elevated intracranial pressure (ICP) of unknown cause. It affects at least 100,000 Americans and its incidence is increasing with the obesity epidemic. Because of pressure on the optic nerve (papilledema) about 10% develop blinding visual loss. We recently completed the Idiopathic Intracranial Hypertension Treatment Trial, a multicenter randomized, double-masked, placebo-controlled study of 165 subjects with IIH and mild visual loss. We showed that acetazolamide-plus diet was significantly superior to placebo-plus-diet for improving 1) perimetric mean deviation (PMD, a global measure of visual field loss), 2) papilledema grade, 3) quality of life (QoL) measures and ICP. Further, high dose acetazolamide (up to 4gm/day) was well tolerated. However, in subjects with moderate to severe visual loss, no intervention, neither medical nor the various surgical approaches, has been verified as efficacious by well-designed clinical trials. We will enroll 180 newly diagnosed IIH subjects with a PMD between -8.5 and -30 dB in at least one eye, as determined by automated perimetry. We will compare the efficacy of 1) maximal medical therapy (MMT), 2) surgical intervention with optic nerve sheath fenestration + MMT, and 3) surgical intervention with intracranial shunting + MMT using an intention-to-treat analysis. The primary outcome will be the change in PMD from baseline to 6 months in the eye with the best qualifying PMD. Secondary outcomes include: OCT measures; changes in papilledema grade, ICP; and QoL scores. We will investigate how interventions that purportedly lower ICP determine a) biomechanical changes of the optic nerve head and b) if retina ganglion cell layer thinning at baseline is associated with a poor visual outcome. We will follow subjects for at least two years to determine the long-term outcomes of these treatment strategies. The Neuro-ophthalmology Research Investigator Consortium (NORDIC) will provide the infrastructure and experienced leadership to conduct the proposed study, consisting of 41 sites, the Data Coordination and Biostatistics Center, three Reading Centers (under a Resource Center), and an Enrollment Center.

项目总结/摘要 特发性颅内高压（IIH）是一种肥胖年轻女性的疾病，其特征是颅内压升高， 颅内压（ICP）不明原因。它影响了至少10万美国人，其发病率是 随着肥胖症的流行而增加。由于视神经受压（视乳头水肿）约占10% 会导致失明 我们最近完成了特发性颅内高压治疗试验，一项多中心随机， 双盲、安慰剂对照研究，165例IIH和轻度视力丧失受试者。我们发现 乙酰唑胺+饮食显著上级安慰剂+饮食改善1）周长平均值 偏差（PMD，视野损失的整体测量），2）视乳头水肿等级，3）生活质量（QoL）测量 和ICP。此外，高剂量乙酰唑胺（高达4gm/天）耐受性良好。然而，在具有 中度至重度视力丧失，没有干预，无论是医疗还是各种手术方法， 经过精心设计的临床试验验证有效。 我们将入组180名新诊断的IIH受试者，至少一只眼睛的PMD在-8.5和-30 dB之间， 通过自动视野检查确定。我们将比较1）最大药物治疗（MMT），2） 视神经鞘开窗+ MMT的手术干预，和3）颅内 分流+ MMT使用意向治疗分析。主要结果将是PMD从 基线至6个月，具有最佳合格PMD。次要结局包括：OCT测量; 视神经乳头水肿等级、ICP和QoL评分的变化。我们将调查据称是 较低的ICP确定a）视神经乳头的生物力学变化和B）视网膜神经节细胞层 基线变薄与较差的视觉结果相关。我们将跟踪研究对象至少两年， 确定这些治疗策略的长期结果。 神经眼科研究者联盟（NORDIC）将提供基础设施， 由41个研究中心、数据协调和 生物统计中心，三个阅读中心（在资源中心下），和一个注册中心。