Adnan I. Qureshi MD, Gillian Bartlett-Esquilant PhD, Alexandra Brown PhD, James McClay MD, MS, Mamatha Pasnoor MD, Richard J. Barohn MD
{"title":"神经学实用临床试验。","authors":"Adnan I. Qureshi MD, Gillian Bartlett-Esquilant PhD, Alexandra Brown PhD, James McClay MD, MS, Mamatha Pasnoor MD, Richard J. Barohn MD","doi":"10.1002/ana.27244","DOIUrl":null,"url":null,"abstract":"<p>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;97:1022–1037</p>","PeriodicalId":127,"journal":{"name":"Annals of Neurology","volume":"97 6","pages":"1022-1037"},"PeriodicalIF":8.1000,"publicationDate":"2025-04-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Pragmatic Clinical Trials in Neurology\",\"authors\":\"Adnan I. Qureshi MD, Gillian Bartlett-Esquilant PhD, Alexandra Brown PhD, James McClay MD, MS, Mamatha Pasnoor MD, Richard J. Barohn MD\",\"doi\":\"10.1002/ana.27244\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p>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;97:1022–1037</p>\",\"PeriodicalId\":127,\"journal\":{\"name\":\"Annals of Neurology\",\"volume\":\"97 6\",\"pages\":\"1022-1037\"},\"PeriodicalIF\":8.1000,\"publicationDate\":\"2025-04-22\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Annals of Neurology\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://onlinelibrary.wiley.com/doi/10.1002/ana.27244\",\"RegionNum\":1,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"CLINICAL NEUROLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Annals of Neurology","FirstCategoryId":"3","ListUrlMain":"https://onlinelibrary.wiley.com/doi/10.1002/ana.27244","RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"CLINICAL NEUROLOGY","Score":null,"Total":0}
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;97:1022–1037
期刊介绍：
Annals of Neurology publishes original articles with potential for high impact in understanding the pathogenesis, clinical and laboratory features, diagnosis, treatment, outcomes and science underlying diseases of the human nervous system. Articles should ideally be of broad interest to the academic neurological community rather than solely to subspecialists in a particular field. Studies involving experimental model system, including those in cell and organ cultures and animals, of direct translational relevance to the understanding of neurological disease are also encouraged.
求助内容：
应助结果提醒方式：
京公网安备 11010802042870号
