Treatment Trials in Young Patients with Chronic Obstructive Pulmonary Disease and Pre-Chronic Obstructive Pulmonary Disease Patients: Time to Move Forward.: Influence Statistics

Expert Impact

Concepts for which they have has direct influence: Chronic obstructive , Pulmonary disease , Obstructive pulmonary , Disease copd , Disease chronic , Respiratory symptoms , Environment interactions .

Key People For Chronic Obstructive

Top KOLs in the world
#1
Peter M A Calverley
chronic obstructive pulmonary disease patients copd
#2
Peter John Barnes
nitric oxide chronic obstructive oxidative stress
#3
Jørgen Vestbo
chronic obstructive pulmonary disease lung function
#4
Bartolome R Celli†
copd patients chronic obstructive pulmonary disease
#5
Alvar G N Agustı́
chronic obstructive pulmonary disease patients copd
#6
Jadwiga Anna Wedzicha
chronic obstructive pulmonary disease copd exacerbations

Treatment Trials in Young Patients with Chronic Obstructive Pulmonary Disease and Pre-Chronic Obstructive Pulmonary Disease Patients: Time to Move Forward.

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.