학술논문
Treatment Trials in Young Patients with Chronic Obstructive Pulmonary Disease and Pre-Chronic Obstructive Pulmonary Disease Patients: Time to Move Forward.
Document Type
Academic Journal
Author
Martinez FJ; Weill Cornell Medicine, NewYork-Presbyterian Hospital, New York, New York.; Agusti A; Catedra Salut Respiratoria and.; Institut Respiratorio, Hospital Clinic, Barcelona, Spain.; Institut d'investigacions biomediques August Pi i Sunyer (IDIBAPS), Barcelona, Spain.; Centro de Investigacion Biomedica en Red de Enfermedades Respiratorias (CIBERES), Madrid, Spain.; Celli BR; Brigham and Women's Hospital, Harvard Medical School, Boston, Massachusetts.; Han MK; University of Michigan Health System, Ann Arbor, Michigan.; Allinson JP; National Heart and Lung Institute, Imperial College, London, United Kingdom.; Bhatt SP; Heersink School of Medicine, University of Alabama at Birmingham, Birmingham, Alabama.; Calverley P; Institute of Ageing and Chronic Disease, University of Liverpool, Liverpool, United Kingdom.; Chotirmall SH; Lee Kong Chian School of Medicine, Nanyang Technological University, Singapore.; Chowdhury B; Savara Pharmaceuticals, Inc., Austin, Texas.; Darken P; AstraZeneca, Gaithersburg, Maryland.; Da Silva CA; Clinical Development, Research and Early Development, Respiratory and Immunology, BioPharmaceuticals R&D, AstraZeneca, Gothenburg, Sweden.; Donaldson G; National Heart and Lung Institute, Imperial College, London, United Kingdom.; Dorinsky P; AstraZeneca, Gaithersburg, Maryland.; Dransfield M; Heersink School of Medicine, University of Alabama at Birmingham, Birmingham, Alabama.; Faner R; Department of Biomedical Sciences, University of Barcelona, Barcelona, Spain.; Halpin DM; Royal Devon and Exeter Hospital, Exeter, United Kingdom.; Jones P; St. George's University of London, London, United Kingdom.; Krishnan JA; University of Illinois, Chicago, Illinois.; Locantore N; Nimirga LLC, Durham, North Carolina.; Martinez FD; University of Arizona, Tucson, Arizona.; Mullerova H; AstraZeneca, Cambridge, United Kingdom.; Price D; Observational and Pragmatic Research Institute, Singapore.; Centre of Academic Primary Care, Division of Applied Health Sciences, University of Aberdeen, Aberdeen, United Kingdom.; Rabe KF; LungenClinic Grosshansdorf, Member of the German Center for Lung Research, Grosshansdorf, Germany.; Department of Medicine, Christian Albrechts University Kiel, Member of the German Center for Lung Research Kiel, Germany.; Reisner C; DevPro Biopharma, Basking Ridge, New Jersey.; Singh D; Medicines Evaluation Unit and.; Vestbo J; Manchester University NHS Trust, Manchester, United Kingdom.; Vogelmeier CF; Department of Medicine, Pulmonary and Critical Care Medicine, University of Marburg, Member of the German Center for Lung Research, Marburg, Germany.; Wise RA; Johns Hopkins Medicine, Baltimore, Maryland; and.; Tal-Singer R; COPD Foundation, Washington, DC.; Wedzicha JA; National Heart and Lung Institute, Imperial College, London, United Kingdom.
Source
Publisher: American Thoracic Society Country of Publication: United States NLM ID: 9421642 Publication Model: Print Cited Medium: Internet ISSN: 1535-4970 (Electronic) Linking ISSN: 1073449X NLM ISO Abbreviation: Am J Respir Crit Care Med Subsets: MEDLINE
Subject
Language
English
Abstract
Chronic obstructive pulmonary disease (COPD) is the end result of a series of dynamic and cumulative gene-environment interactions over a lifetime. The evolving understanding of COPD biology provides novel opportunities for prevention, early diagnosis, and intervention. To advance these concepts, we propose therapeutic trials in two major groups of subjects: "young" individuals with COPD and those with pre-COPD. Given that lungs grow to about 20 years of age and begin to age at approximately 50 years, we consider "young" patients with COPD those patients in the age range of 20-50 years. Pre-COPD relates to individuals of any age who have respiratory symptoms with or without structural and/or functional abnormalities, in the absence of airflow limitation, and who may develop persistent airflow limitation over time. We exclude from the current discussion infants and adolescents because of their unique physiological context and COPD in older adults given their representation in prior randomized controlled trials (RCTs). We highlight the need of RCTs focused on COPD in young patients or pre-COPD to reduce disease progression, providing innovative approaches to identifying and engaging potential study subjects. We detail approaches to RCT design, including potential outcomes such as lung function, patient-reported outcomes, exacerbations, lung imaging, mortality, and composite endpoints. We critically review study design components such as statistical powering and analysis, duration of study treatment, and formats to trial structure, including platform, basket, and umbrella trials. We provide a call to action for treatment RCTs in 1 ) young adults with COPD and 2 ) those with pre-COPD at any age.