Characteristics of alpha-1 antitrypsin deficiency related lung disease exacerbations using a daily symptom diary and urinary biomarkers

Paul Ellis*, Gita Parekh, Annelyse Duvoix, Lynne Watson, Alex Sharp, Farah Mobeen, Anita Pye, Robert Stockley, Alice Turner

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

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Abstract

Background: Pulmonary exacerbations in alpha-1 antitrypsin deficiency (AATD) related lung disease are a significant contributor to disease burden, as with usual COPD. Separating the early stages of an exacerbation from the day-to-day variation in stable COPD is central to the concerns of both clinicians and patients and has been identified as a research priority by NIHR.

Clinical tools that distinguish baseline symptoms from those of an exacerbation could allow early and appropriate treatment of AECOPD to reduce the impact and potentially may slow disease progression thereby improving survival and quality of life. Candidate tools include symptom diaries and biomarkers of infection and acute inflammation. Urinary biomarkers of AECOPD have yet to be explored in AATD related COPD.

Methods: 55 patients with AATD related lung disease with a history of 2 or more AECOPD in the preceding year were prospectively followed for 18 months. Each patient recorded symptom scores daily via an electronic symptom diary (eDiary) based on Bronkotest. Urinary biomarkers for AAT, NE, CRP, TIMP1 and desmosine were measured weekly using a home urinary lateral flow device. During self-reported AECOPD patients were asked to perform urine analysis on the first 7 consecutive days.

Results: Type I Anthonisen exacerbations and episodes occurring in autumn/winter lasted longer than Type II/III exacerbations and spring/summer episodes respectively. Median urinary CRP concentration across all study participants increased during Type I AECOPD. eDiary adherence was 68% over a median of 17.8 months (IQR 15.7 to 18.5).

Conclusions: Use of an eDiary and urinary biomarkers to detect and characterise AECOPD remotely in AATD related lung disease is feasible over a prolonged period and paves the way for precision detection of exacerbations.
Original languageEnglish
Article numbere0297125
Number of pages12
JournalPLOS One
Volume19
Issue number2
DOIs
Publication statusPublished - 2 Feb 2024

Bibliographical note

Funding:
The alpha-1 foundation funded the grant for this work but had no input on the final analysis or manuscript. The funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.

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