Surgical IIH Treatment Trial

IIH 手术治疗试验

• 批准号: 9209029
• 负责人: Mark J Kupersmith
• 金额: $ 342.63万
• 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 2022-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9209029
• 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; Eligibility Determination; 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.

项目摘要 /摘要 特发性颅内高血压（IIH）是一种肥胖的年轻女性，其特征是升高 未知原因的颅内压（ICP）。它影响至少100,000美国人，其发病率是 随着肥胖流行的增加。由于视神经的压力（乳头毛体）约10％ 发展视觉损失。 我们最近完成了特发性颅内高血压治疗试验，这是一项多中心随机， 对165名IIH和轻度视觉丧失的受试者进行了双掩盖的安慰剂对照研究。我们表明了这一点 乙酰唑胺加饮食明显优于安慰剂 - 加上饮食，以改善1）均值平均值 偏差（PMD，全球视野损失量度），2）乳头毛瘤等级，3）生活质量（QOL）措施 和ICP。此外，高剂量乙酰唑胺（每天最高4gm）的耐受性良好。但是，在有主题 中度至重度视觉丧失，没有干预，医疗和各种手术方法都没有 通过精心设计的临床试验被证实是有效的。 我们将在至少一只眼睛中注册180名新诊断的IIH受试者，PMD在-8.5和-30 dB之间 由自动围场确定。我们将比较1）最大药物治疗（MMT）的功效，2） 视神经鞘膜 + MMT的手术干预，3）颅内手术干预 使用意向治疗分析分流 + MMT。主要结果将是PMD的变化 凭借最佳资格PMD的基线至6个月。次要结果包括：OCT措施； 乳头乳头级的变化，ICP；和QOL分数。我们将调查据称的干预措施 较低的ICP确定a）视神经头的生物力学变化，b）如果视网膜神经节细胞层 基线时的变薄与视觉结果差有关。我们将关注主题至少两年 确定这些治疗策略的长期结果。 神经跨性研究研究者联盟（北欧）将提供基础设施和 经验丰富的领导层进行拟议的研究，包括41个站点，数据协调和 生物统计学中心，三个阅读中心（资源中心下）和一个入学中心。