Pragmatic Clinical Trials in Neurology

被引:0
作者
Qureshi, Adnan I. [1 ]
Bartlett-Esquilant, Gillian [2 ]
Brown, Alexandra [3 ]
McClay, James [2 ]
Pasnoor, Mamatha [4 ]
Barohn, Richard J. [5 ]
机构
[1] Univ Missouri, Dept Neurol, Columbia, MO 65212 USA
[2] Texas A&M, Dept Primary Care & Rural Med, Coll Med, College Stn, TX USA
[3] Univ Kansas, Med Ctr, Dept Biostat & Data Sci, Kansas City, KS USA
[4] Univ Kansas, Sch Med, Dept Neurol, Kansas City, KS USA
[5] Univ Missouri, Off Execut Vice Chancellor Hlth Affairs, Columbia, MO USA
关键词
REMITTING MULTIPLE-SCLEROSIS; CLUSTER-RANDOMIZED-TRIALS; TRANSIENT ISCHEMIC ATTACK; GUILLAIN-BARRE-SYNDROME; ACUTE STROKE; OPEN-LABEL; SWALLOWING DYSFUNCTION; CAROTID-ENDARTERECTOMY; GLATIRAMER ACETATE; INFORMED-CONSENT;
D O I
10.1002/ana.27244
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
The need for pragmatic clinical trials evaluating therapeutic interventions in patients with neurological disease is continually increasing due to availability of multiple therapeutic interventions (comparative effectiveness), multifaceted approaches (multiple concurrent synergistic therapeutic interventions), and gaps in trial-specific and real-world population outcomes. Several designs for pragmatic trials are available, including individual randomized trials with pragmatic characteristics, cluster-randomized and non-randomized trials, and observational prospective cohort studies. Cluster trials may have parallel cluster and crossover (unidirectional, bidirectional, and alternating crossover) designs. There are unique aspects of consenting and data collection leveraging existing registries, electronic health records (EHRs), and claims data that make pragmatic trials most suited to study the effectiveness of therapeutic interventions in patients with neurological diseases in real-world settings. ANN NEUROL 2025
引用
收藏
页码:1022 / 1037
页数:16
相关论文
共 141 条
[11]  
Bhandari M, 2004, CAN MED ASSOC J, V170, P477
[12]   A Randomized Trial of a Multifactorial Strategy to Prevent Serious Fall Injuries [J].
Bhasin, Shalender ;
Gill, Thomas M. ;
Reuben, David B. ;
Latham, Nancy K. ;
Ganz, David A. ;
Greene, Erich J. ;
Dziura, James ;
Basaria, Shehzad ;
Gurwitz, Jerry H. ;
Dykes, Patricia C. ;
McMahon, Siobhan ;
Storer, Thomas W. ;
Gazarian, Priscilla ;
Miller, Michael E. ;
Travison, Thomas G. ;
Esserman, Denise ;
Carnie, Martha B. ;
Goehring, Lori ;
Fagan, Maureen ;
Greenspan, Susan L. ;
Alexander, Neil ;
Wiggins, Jocelyn ;
Ko, Fred ;
Siu, Albert L. ;
Volpi, Elena ;
Wu, Albert W. ;
Rich, Jeremy ;
Waring, Stephen C. ;
Wallace, Robert B. ;
Casteel, Carri ;
Resnick, Neil M. ;
Magaziner, Jay ;
Charpentier, Peter ;
Lu, Charles ;
Araujo, Katy ;
Rajeevan, Haseena ;
Meng, Can ;
Allore, Heather ;
Brawley, Brooke F. ;
Eder, Rich ;
McGloin, Joanne M. ;
Skokos, Eleni A. ;
Duncan, Pamela W. ;
Baker, Dorothy ;
Boult, Chad ;
Correa-de-Araujo, Rosaly ;
Peduzzi, Peter .
NEW ENGLAND JOURNAL OF MEDICINE, 2020, 383 (02) :129-140
[13]   Strategies to Reduce Injuries and Develop Confidence in Elders (STRIDE): A Cluster-Randomized Pragmatic Trial of a Multifactorial Fall Injury Prevention Strategy: Design and Methods [J].
Bhasin, Shalender ;
Gill, Thomas M. ;
Reuben, David B. ;
Latham, Nancy K. ;
Gurwitz, Jerry H. ;
Dykes, Patricia ;
McMahon, Siobhan ;
Storer, Thomas W. ;
Duncan, Pamela W. ;
Ganz, David A. ;
Basaria, Shehzad ;
Miller, Michael E. ;
Travison, Thomas G. ;
Greene, Erich J. ;
Dziura, James ;
Esserman, Denise ;
Allore, Heather ;
Carnie, Martha B. ;
Fagan, Maureen ;
Hanson, Catherine ;
Baker, Dorothy ;
Greenspan, Susan L. ;
Alexander, Neil ;
Ko, Fred ;
Siu, Albert L. ;
Volpi, Elena ;
Wu, Albert W. ;
Rich, Jeremy ;
Waring, Stephen C. ;
Wallace, Robert ;
Casteel, Carri ;
Magaziner, Jay ;
Charpentier, Peter ;
Lu, Charles ;
Araujo, Katy ;
Rajeevan, Haseena ;
Margolis, Scott ;
Eder, Richard ;
McGloin, Joanne M. ;
Skokos, Eleni ;
Wiggins, Jocelyn ;
Garber, Lawrence ;
Clauser, Steven B. ;
Correa-De-Araujo, Rosaly ;
Peduzzi, Peter .
JOURNALS OF GERONTOLOGY SERIES A-BIOLOGICAL SCIENCES AND MEDICAL SCIENCES, 2018, 73 (08) :1053-1061
[14]   Benefits of Stroke Treatment Using a Mobile Stroke Unit Compared With Standard Management The BEST-MSU Study Run-In Phase [J].
Bowry, Ritvij ;
Parker, Stephanie ;
Rajan, Suja S. ;
Yamal, Jose-Miguel ;
Wu, Tzu-Ching ;
Richardson, Laura ;
Noser, Elizabeth ;
Persse, David ;
Jackson, Kamilah ;
Grotta, James C. .
STROKE, 2015, 46 (12) :3370-3374
[15]  
Boy S., 2012, PERS MED, V1, P77, DOI [10.1016/j.permed.2012.02.002, DOI 10.1016/J.PERMED.2012.02.002]
[16]   Thrombolysis for acute stroke in routine clinical practice [J].
Bravata, DM ;
Kim, N ;
Concato, J ;
Krumholz, HM ;
Brass, LM .
ARCHIVES OF INTERNAL MEDICINE, 2002, 162 (17) :1994-2001
[17]  
Campbell M., 2014, How to Design, Analyse and Report Cluster Randomized Trials in Medicine and Health Related Research
[18]   Balance algorithm for cluster randomized trials [J].
Carter, Ben R. ;
Hood, Kerenza .
BMC MEDICAL RESEARCH METHODOLOGY, 2008, 8 (1)
[19]   Higher satisfaction and adherence with glatiramer acetate 40 mg/mL TIW vs 20 mg/mL QD in RRMS [J].
Cutter, Gary ;
Veneziano, Antonella ;
Grinspan, Augusto ;
Al-Banna, Mahir ;
Boyko, Alexey ;
Zakharova, Maria ;
Maida, Eva ;
Pasic, Marija Bosnjak ;
Gandhi, Sanjay K. ;
Everts, Robin ;
Cordioli, Cinzia ;
Rossi, Silvia .
MULTIPLE SCLEROSIS AND RELATED DISORDERS, 2019, 33 :13-21
[20]   Real-world evidence: How pragmatic are randomized controlled trials labeled as pragmatic? [J].
Dal-Re, Rafael ;
Janiaud, Perrine ;
Ioannidis, John P. A. .
BMC MEDICINE, 2018, 16