Treatment Trials in Young Patients with Chronic Obstructive Pulmonary Disease and Pre–Chronic Obstructive Pulmonary Disease Patients

Fernando J. Martinez, Alvar Agusti, Bartolome R. Celli, Mei Lan K. Han, James P. Allinson, Surya P. Bhatt, Peter Calverley, Sanjay H. Chotirmall, Badrul Chowdhury, Patrick Darken, Carla A. da Silva, Gavin Donaldson, Paul Dorinsky, Mark Dransfield, Rosa Faner, David M. Halpin, Paul Jones, Jerry A. Krishnan, Nicholas Locantore, Fernando D. MartinezHana Mullerova, David Price, Klaus F. Rabe, Colin Reisner, Dave Singh, Jørgen Vestbo, Claus F. Vogelmeier, Robert A. Wise, Ruth Tal-Singer, Jadwiga A. Wedzicha

Research output: Contribution to journalReview articlepeer-review

2 Scopus citations

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.

Original languageEnglish (US)
Pages (from-to)275-287
Number of pages13
JournalAmerican journal of respiratory and critical care medicine
Volume205
Issue number3
DOIs
StatePublished - Feb 1 2022

Keywords

  • Clinical trials
  • COPD
  • Early
  • Pre-COPD
  • Young age

ASJC Scopus subject areas

  • Pulmonary and Respiratory Medicine
  • Critical Care and Intensive Care Medicine

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