Highly symptomatic patients have an increased risk of exacerbations and benefit from long-term macrolide treatment in Bronchiectasis

<bold>Introduction:</bold> Prior exacerbations are the main risk factor for exacerbations in bronchiectasis. Guidelines recommend macrolide only in frequent exacerbators. Other studies suggest that symptoms are related to increased exacerbation risk. We aimed to investigate if symptoms could independently predict future exacerbations and identify responders to long-term macrolide therapy. <bold>Methods:</bold> Prospective observational study including 8328 patients from EMBARC registry with symptoms (Quality-of-life questionnaire Bronchiectasis (QoL-B) at baseline) and follow-up for at least one year, and posthoc analysis of 3 randomized clinical trials (RCTs) of macrolides in 341 bronchiectasis patients. <bold>Results:</bold> Prior exacerbations (RR for every exacerbation=1.10, 95%CI 1.09-1.11, p<0.001) and symptoms (RR for every 10 points lower QoL-B=1.10, 95%CI 1.08-1.11, p<0.001) were identified as independent risk factors for future exacerbations in the EMBARC registry. The expected number of exacerbations in 1-year was similar between the group with 3 prior exacerbations and average symptom scores (1.49, 95% CI 1.38-1.60) and the group with no prior exacerbations but high symptom scores (1.47, 95% CI 1.34-1.60). The same pattern was observed in the post-hoc analysis of RCTs, both in macrolide and placebo arms. The number needed to treat to prevent exacerbations with macrolides was similar among patients selected based on frequent exacerbations and in those with no prior/few exacerbations but high symptom scores. <bold>Conclusion:</bold> Symptoms are an independent risk factor for exacerbations in bronchiectasis, and highly symptomatic patients may benefit from long-term macrolides.

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Published
2024-09-14
DOI
https://doi.org/10.1183/13993003.congress-2024.pa2375
Primary Topic
Cystic Fibrosis Research Advances
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article
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article

Highly symptomatic patients have an increased risk of exacerbations and benefit from long-term macrolide treatment in Bronchiectasis

Francesco Blasi, Wim Boersma, Arietta Spinou, Lucy Burr et al.
Cystic Fibrosis Research Advances
article

Highly symptomatic patients have an increased risk of exacerbations and benefit from long-term macrolide treatment in Bronchiectasis

Francesco Blasi, Wim Boersma, Arietta Spinou, Lucy Burr, Michael R. Loebinger, Josje Altenburg, Jeremy Brown, Michal Shteinberg, Lídia Perea, Menno M. van der Eerden, Oriol Sibila, John Robert Hurst, Charles Haworth, J.S. Elborn, Felix C. Ringshausen, Raja Dhar, Eva Polverino, Katerina Dimakou, Noel Karalus, Jamie Stobo, Rosario Menéndez, Paula Kauppi, Pieter Goeminne, Apostolos Bossios, Lata Jayaram, Natalie Lorent, Sanjay H. Chotirmall, Tobias Welte, Melissa J. McDonnell, Montserrat Vendrell, Steven Taylor, Anthony De Soyza, Lotte Terpstra, Yong‐hua Gao, Hayoung Choi, Conroy Wong, Pierre‐Régis Burgel, Stefano Aliberti, Antoni Torres, Megan Crichton, James D. Chalmers, Emma D Johnson, Megan Martin
article en

Abstract

<bold>Introduction:</bold> Prior exacerbations are the main risk factor for exacerbations in bronchiectasis. Guidelines recommend macrolide only in frequent exacerbators. Other studies suggest that symptoms are related to increased exacerbation risk. We aimed to investigate if symptoms could independently predict future exacerbations and identify responders to long-term macrolide therapy. <bold>Methods:</bold> Prospective observational study including 8328 patients from EMBARC registry with symptoms (Quality-of-life questionnaire Bronchiectasis (QoL-B) at baseline) and follow-up for at least one year, and posthoc analysis of 3 randomized clinical trials (RCTs) of macrolides in 341 bronchiectasis patients. <bold>Results:</bold> Prior exacerbations (RR for every exacerbation=1.10, 95%CI 1.09-1.11, p<0.001) and symptoms (RR for every 10 points lower QoL-B=1.10, 95%CI 1.08-1.11, p<0.001) were identified as independent risk factors for future exacerbations in the EMBARC registry. The expected number of exacerbations in 1-year was similar between the group with 3 prior exacerbations and average symptom scores (1.49, 95% CI 1.38-1.60) and the group with no prior exacerbations but high symptom scores (1.47, 95% CI 1.34-1.60). The same pattern was observed in the post-hoc analysis of RCTs, both in macrolide and placebo arms. The number needed to treat to prevent exacerbations with macrolides was similar among patients selected based on frequent exacerbations and in those with no prior/few exacerbations but high symptom scores. <bold>Conclusion:</bold> Symptoms are an independent risk factor for exacerbations in bronchiectasis, and highly symptomatic patients may benefit from long-term macrolides.

Délégation Paris 5 (FR), Queen's University Belfast (GB), Mater Health Services (AU), Humanitas University (IT), The University of Melbourne (AU), University of Dundee (GB), King's College London (GB), University of Milan (IT), Université Paris Cité (FR), Royal Papworth Hospital (GB), Helsinki University Hospital (FI), Middlemore Hospital (NZ), Sacred Heart Hospital (IE), Waikato Hospital (NZ), Victorian Curriculum and Assessment Authority (AU), University Hospital Galway (IE), Amsterdam UMC Location University of Amsterdam (NL), Medizinische Hochschule Hannover (DE), Hallym University Kangnam Sacred Heart Hospital (KR), Hospital Clínico Universitario de Valencia (ES), Royal Brompton Hospital (GB), Assistance Publique – Hôpitaux de Paris (FR), Vall d'Hebron Institut de Recerca (ES), Svenska Örtmedicinska Institute (SE), Carmel Medical Center (IL), Hospital Clínic de Barcelona (ES), Newcastle upon Tyne Hospital (GB), Vlaamse Vereniging voor Obstetrie en Gynaecolo (BE), Hospital General Universitario De Valencia (ES), South Australian Health and Medical Research Institute (AU), Fortis Hospital (IN), The London College (GB), Hospital Universitari de Girona Doctor Josep Trueta (ES), First Affiliated Hospital of Zhengzhou University (CN), Medisch Centrum Alkmaar (NL), Consorci Institut D'Investigacions Biomediques August Pi I Sunyer (ES), Sotiria General Hospital (GR), University College London (GB), Singapore Management University (SG), Rotterdam University of Applied Sciences (NL), KU Leuven (BE)
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Cystic Fibrosis Research Advances
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