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The generalizability of bronchiectasis randomized controlled trials: A multicentre cohort study

Lookup NU author(s): Dr John Davidson, Professor Anthony De SoyzaORCiD


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Introduction: Randomized controlled trials (RCTs) for bronchiectasis have experienced difficulties with recruitment and in reaching their efficacy end-points. To estimate the generalizability of such studies we applied the eligibility criteria for major RCTs in bronchiectasis to 6 representative observational European Bronchiectasis cohorts.Methods: Inclusion and exclusion criteria from 10 major RCTs were applied in each cohort. Demographics and outcomes were compared between patients eligible and ineligible for RCTs.Results: 1672 patients were included. On average 33.0% were eligible for macrolide trials, 15.0% were eligible for inhaled antibiotic trials, 15.9% for the DNAse study and 47.7% were eligible for a study of dry powder mannitol. Within these groups, some trials were highly selective with only 1-9% of patients eligible. Eligible patients were generally more severe with higher mortality during follow-up (mean 17.2 vs 9.0% for macrolide studies, 19.2%% vs 10.7% for inhaled antibiotic studies), and a higher frequency of exacerbations than ineligible patients. As up to 93% of patients were ineligible for studies, however, numerically more deaths and exacerbations occurred in ineligible patient across studies (mean 56% of deaths occurred in ineligible patients across all studies).Conclusion: Our data suggest that patients enrolled in RCT's in bronchiectasis are only partially representative of patients in clinical practice. The majority of mortality and morbidity in bronchiectasis occurs in patients ineligible for many current trials. (C) 2016 Elsevier Ltd. All rights reserved.

Publication metadata

Author(s): Chalmers JD, McDonnell MJ, Rutherford R, Davidson J, Finch S, Crichton M, Dupont L, Hill AT, Fardon TC, De Soyza A, Aliberti S, Goeminne P

Publication type: Article

Publication status: Published

Journal: Respiratory Medicine

Year: 2016

Volume: 112

Pages: 51-58

Print publication date: 01/03/2016

Online publication date: 29/01/2016

Acceptance date: 22/01/2016

ISSN (print): 0954-6111

ISSN (electronic): 1532-3064

Publisher: W.B. Saunders Co.


DOI: 10.1016/j.rmed.2016.01.016


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Funder referenceFunder name
Bayer HealthCare in the set-up and development of the European Bronchiectasis Registry
European bronchiectasis registry
European Respiratory Society
MR/L011263/1Medical Research Council (UK)