A systematic review of pharmacotherapeutic clinical trial end-points for bronchiectasis in adults

Megan L. Crichton, Stefano Aliberti, James D. Chalmers (Lead / Corresponding author)

    Research output: Contribution to journalReview articlepeer-review

    20 Citations (Scopus)
    166 Downloads (Pure)


    Bronchiectasis is an increasing clinical problem, but multiple recent clinical trials have failed to reach their primary end-point. Difficulties in achieving "positive" bronchiectasis trials is reflected in a lack of agreement from trialists and regulators on what are the optimal end-points.To evaluate the use of end-points in bronchiectasis trials, we conducted a systematic review of published bronchiectasis trials from 2008 to 2018 and extracted end-points used, definitions, methods of analysis and responsiveness.Our analysis shows that quality of life and exacerbation end-points are most frequently used. Trials using exacerbation end-points have been characterised by varying definitions, multiple methods of analysis and durations of follow-up. There are multiple quality of life tools for bronchiectasis (Quality of Life - Bronchiectasis questionnaire, St George's Respiratory Questionnaire, etc.). The majority of studies measure lung function (e.g. forced expiratory volume in 1 s), but this is shown to be nonresponsive to the majority of interventions. Microbiology end-points frequently show statistically significant differences in phase 2 antibiotic studies but their correlation with clinical end-points is unknown.This systematic review demonstrates a need for guidance to standardise definitions and design features to improve reproducibility and increase the likelihood of demonstrating statistically significant benefits with new therapies.

    Original languageEnglish
    Article number180108
    Pages (from-to)1-14
    Number of pages14
    JournalEuropean Respiratory Review
    Issue number151
    Early online date14 Mar 2019
    Publication statusPublished - 31 Mar 2019


    • Bronchiectasis/diagnosis
    • Clinical Trials as Topic/methods
    • Disease Progression
    • Endpoint Determination/standards
    • Humans
    • Lung/drug effects
    • Quality of Life
    • Recovery of Function
    • Research Design/standards
    • Respiratory System Agents/adverse effects
    • Treatment Outcome


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