特发性颅内高压治疗试验：设计考量与方法
The idiopathic intracranial hypertension treatment trial: design considerations and methods.
作者信息
Friedman Deborah I, McDermott Michael P, Kieburtz Karl, Kupersmith Mark, Stoutenburg Ann, Keltner John L, Feldon Steven E, Schron Eleanor, Corbett James J, Wall Michael
机构信息
Departments of Neurology & Neurotherapeutics and Ophthalmology (DIF), University of Texas Southwestern Medical Center, Dallas, Texas; Departments of Ophthalmology, Neurology, Neurosurgery and Visual Science (SEF), Center for Human Experimental Therapeutics (KK, AS), Department of Biostatistics and Computational Biology and Department of Neurology (MM), University of Rochester School of Medicine and Dentistry, Rochester, New York; Neurology (MW), University of Iowa College of Medicine and Iowa City Veterans Affairs Health Care System, Iowa City, Iowa; National Eye Institute (ES), Bethesda, Maryland; Department of Ophthalmology and Vision Science (JK), University of California Davis Medical Center, Sacramento, California; Departments of Neurology and Ophthalmology (MK), Mount Sinai School of Medicine, New York, New York.
出版信息
J Neuroophthalmol. 2014 Jun;34(2):107-17. doi: 10.1097/WNO.0000000000000114.
BACKGROUND
The objectives of this study were to present the rationale for the main aspects of the study design and describe the trial methodology for the Idiopathic Intracranial Hypertension Treatment Trial (IIHTT).
METHODS
Eligible candidates with mild visual field loss (automated perimetric mean deviation [PMD] -2 to -7 dB) were randomized to receive either acetazolamide or matching placebo tablets. Randomized participants were offered participation in a supervised dietary program. The primary outcome variable, PMD, was measured at 6 months. Additionally, cerebrospinal fluid from subjects and serum from study participants and matched controls were collected for genetic analysis and vitamin A studies. An ancillary optical coherence substudy was added to investigate the changes of papilledema in the optic nerve head and retina that correlate with Frisén grading, visual field deficits, and low-contrast visual acuity.
RESULTS
The randomized trial entered 165 participants from March 17, 2010, through November 27, 2012, from the United States and Canada. The primary outcome (month 6) visits were successfully completed by June 15, 2013. Blood specimens were obtained from 165 controls without IIH to investigate vitamin A metabolism and genetic markers of potential risk factors for IIH.
CONCLUSIONS
The IIHTT is the first randomized, double-masked placebo-controlled trial to study the effectiveness of medical treatment for patients with IIH.
背景
本研究的目的是阐述研究设计主要方面的基本原理，并描述特发性颅内高压治疗试验（IIHTT）的试验方法。
方法
符合条件的轻度视野缺损（自动视野计平均偏差[PMD]为-2至-7 dB）的受试者被随机分配接受乙酰唑胺或匹配的安慰剂片。随机分组的参与者可参加一个有监督的饮食计划。主要结局变量PMD在6个月时进行测量。此外，收集受试者的脑脊液以及研究参与者和匹配对照的血清用于基因分析和维生素A研究。增加了一项辅助光学相干子研究，以调查视神经乳头和视网膜视乳头水肿的变化，这些变化与弗里森分级、视野缺损和低对比度视力相关。
结果
从2010年3月17日至2012年11月，来自美国和加拿大的165名参与者进入了该随机试验。主要结局（第6个月）访视于2013年6月15日成功完成。从165名无IIH的对照中获取血样，以研究IIH潜在危险因素的维生素A代谢和基因标记。
结论
IIHTT是首个研究IIH患者药物治疗有效性的随机、双盲、安慰剂对照试验。
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