Longitudinal magnetic resonance imaging in progressive supranuclear palsy: A new combined score for clinical trials.
Longitudinal magnetic resonance imaging in progressive supranuclear palsy: A new combined score for clinical trials.
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DOI：
10.1002/mds.26973
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发表时间：
2017-06
期刊：
影响因子：
--
通讯作者：
Movement Disorder Society-Endorsed PSP Study Group
中科院分区：
文献类型：
--
作者：
Höglinger GU;Schöpe J;Stamelou M;Kassubek J;Del Ser T;Boxer AL;Wagenpfeil S;Huppertz HJ;AL-108-231 Investigators;Tauros MRI Investigators;Movement Disorder Society-Endorsed PSP Study Group
Davunetide和Tideglusib的两项最近的两项随机安慰剂对照II/III期试验（ClinicalTrials.gov：NCT01110720，NCT01049399）和Tideglusib进行了渐进性上核瘫痪（PSP），产生了前瞻性的，1年的远程数据集生成高较高的远程数据集的高质量t1-weeight t1-weeight 3dd Mri。
为临床试验开发定量的MRI疾病进展测量。
我们采用基于地图集的体积进行了完全自动化的定量MRI分析，并根据在这些试验中分配给安慰剂的N = 99名PSP患者中收集的数据进行了样本量计算。基于基线时n = 44个脑部室和结构的单个体积和52周的随访，使用年度百分比变化的平均值和标准偏差用于估计标准化效应尺寸和每组所需的样本量，以使未来的两臂，两臂，安慰剂对照的治疗试验。
发现中脑，额叶和第三个心室的最高标准效应尺寸。使用这些结构的年度百分比变化，以检测1年进展的50％变化（80％的功率，显着性5％）需要较低的患者数量[第三脑室，n = 32;中脑，n = 37;额叶，n = 43]比最佳临床量表（PSP等级总分，n = 58）。这三个结构中体积变化的组合将所需患者的数量减少到n = 20，并且与临床尺度的进展最为相关。
我们提出，第三脑室，中脑和额叶的体积为1年变化，作为PSP中临床试验的组合成像读取，需要最少的患者来检测功效以减少脑萎缩。
Two recent randomized, placebo-controlled phase II/III trials (clinicaltrials.gov: NCT01110720, NCT01049399) of davunetide and tideglusib in progressive supranuclear palsy (PSP) generated prospective, 1-year longitudinal datasets of high-resolution T1-weighted 3D MRI. Develop a quantitative MRI disease progression measurement for clinical trials. We performed a fully automated quantitative MRI analysis employing atlas-based volumetry and provide sample size calculations based on data collected in N=99 PSP patients, assigned to placebo in these trials. Based on individual volumes of N=44 brain compartments and structures at baseline and 52 weeks follow-up, means and standard deviations of annualized percentage volume changes were used to estimate standardized effect sizes and the required sample sizes per group for future two-armed, placebo-controlled therapeutic trials. The highest standardized effect sizes were found for midbrain, frontal lobes, and third ventricle. Using the annualized percentage volume change of these structures to detect a 50% change in the 1-year progression (80% power, significance level 5%) required lower numbers of patients per group [third ventricle, N=32; midbrain, N=37; frontal lobe, N= 43] than the best clinical scale (PSP rating scale total score, N=58). A combination of volume changes in these three structures reduced the number of required patients to only N=20 and correlated best with the progression in the clinical scales. We propose the 1-year change in the volumes of third ventricle, midbrain, and frontal lobe as combined imaging read-out for clinical trials in PSP, requiring the least number of patients for detecting efficacy to reduce brain atrophy.
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影响因子：
48
作者：
Boxer, Adam L.;Lang, Anthony E.;Grossman, Murray;Knopman, David S.;Miller, Bruce L.;Schneider, Lon S.;Doody, Rachelle S.;Lees, Andrew;Golbe, Lawrence I.;Williams, David R.;Corvol, Jean-Cristophe;Ludolph, Albert;Burn, David;Lorenzl, Stefan;Litvan, Irene;Roberson, Erik D.;Hoeglinger, Guenter U.;Koestler, Mary;Jack, Clifford R., Jr.;Van Deerlin, Viviana;Randolph, Christopher;Lobach, Iryna V.;Heuer, Hilary W.;Gozes, Illana;Parker, Lesley;Whitaker, Steve;Hirman, Joe;Stewart, Alistair J.;Gold, Michael;Morimoto, Bruce H.
通讯作者：
Morimoto, Bruce H.
影响因子：
9.9
作者：
Dutt, Shubir;Binney, Richard J.;Boxer, Adam L.
通讯作者：
Boxer, Adam L.
影响因子：
17.7
作者：
Frazier, JA;Chiu, SF;Biederman, J
通讯作者：
Biederman, J
影响因子：
5.7
作者：
Fjell, Anders M.;Walhovd, Kristine B.;Dale, Anders M.
通讯作者：
Dale, Anders M.
DOI：
10.1002/mds.23912
发表时间：
2011-12
期刊：
Movement disorders : official journal of the Movement Disorder Society
影响因子：
--
作者：
Majid DS;Aron AR;Thompson W;Sheldon S;Hamza S;Stoffers D;Holland D;Goldstein J;Corey-Bloom J;Dale AM
通讯作者：
Dale AM