Pulmonary exacerbations in patients with primary ciliary dyskinesia – A prospective multicenter study
<bold>Background:</bold> Pulmonary exacerbations cause significant morbidity in patients with primary ciliary dyskinesia (PCD) and their frequency can be used as a primary outcome in prospective clinical trials. Two definitions (Def) of pulmonary exacerbations in PCD have been published recently: <italic>Def-1</italic>, based on a consensus agreement<sup>1</sup>; <italic>Def-2</italic>, based on antibiotics use<sup>2,3</sup>. To date little is known on the burden of pulmonary exacerbations in PCD patients. <bold>Aim:</bold> We aimed to record the exacerbation rate within one year based on the above definitions, as well as based on self-reported assessments. Furthermore, we aimed to assess the agreement between the two definitions, and their correlation with the self-reported exacerbation rate. <bold>Methods:</bold> Data were prospectively collected in monthly telephone interviews via a structured questionnaire. <bold>Results:</bold> In total 358 patients (45.8% females) from 11 centers, median age 17.5 years (range 0-72), with confirmed diagnosis of PCD participated in the study. Within 12 months, 80.2% of patients had at least one exacerbation per year, as defined by <italic>Def-1</italic> or <italic>Def-2</italic> (Table 1). Self-reported exacerbations rate is significantly higher compared to <italic>Def-1</italic> and <italic>Def-2</italic> (Wilcoxon test, p<0.0001). Between <italic>Def-1</italic> and <italic>Def-2</italic> there is a moderate agreement (Cohen’s kappa=0.59). <bold>Conclusion:</bold> This is the first study that provides prospective evidence on the burden of pulmonary exacerbations in patients with PCD. Table 1: Exacerbations rate within one year <table-wrap><object-id>erj;64/suppl_68/PA2382/TB1</object-id><object-id>T1</object-id><object-id>TB1</object-id><table><colgroup><col></col><col></col><col></col></colgroup><tbody><tr><td>Exacerbations/year</td><td>Mean</td><td>Median (IQR)</td></tr><tr><td>Self-reported*</td><td>3.003</td><td>2 (1-12)</td></tr><tr><td><italic>Def-1</italic>*</td><td>2.259</td><td>1 (0-4)</td></tr><tr><td><italic>Def-2</italic>*</td><td>1.927</td><td>1 (0-3)</td></tr></tbody></table></table-wrap> *Friedman test, p<0.0001 <sup>1</sup>Lucas, et al. ERJ Open Res 2019 <sup>2</sup>Kobbernagel, et al. BMC Pulm Med 2016 <sup>3</sup>Ratjen, et al. Eur Respir J 2016
Authors
- Pinelopi Anagnostopoulou (ORCID: https://orcid.org/0000-0003-2597-8016)
- June K. Marthin (ORCID: https://orcid.org/0000-0002-4420-3091)
- Heymut Omran (ORCID: https://orcid.org/0000-0003-0282-6765)
- Debora Maj
- Melissa Borrelli (ORCID: https://orcid.org/0000-0002-7282-6018)
- Panayiotis Κouis (ORCID: https://orcid.org/0000-0003-0511-5352)
- Nicos Middleton (ORCID: https://orcid.org/0000-0001-6358-8591)
- Dilber Ademhan Tural (ORCID: https://orcid.org/0000-0002-0334-6336)
- Nicola Ullmann (ORCID: https://orcid.org/0000-0003-1111-5690)
- Panayiotis Yiallouros
- Sandra Rovira‐Amigo (ORCID: https://orcid.org/0000-0001-9896-0621)
- Elpis Hatziagorou (ORCID: https://orcid.org/0000-0001-9505-2054)
- Renato Cutrera (ORCID: https://orcid.org/0000-0001-7711-5672)
- Antonio Moreno (ORCID: https://orcid.org/0000-0002-2496-9786)
- Kim G. Nielsen (ORCID: https://orcid.org/0000-0001-5906-9449)
- Yasemi̇n Gökdemi̇r (ORCID: https://orcid.org/0000-0002-0853-7932)
- Marcus Alexander Mall (ORCID: https://orcid.org/0000-0002-4057-2199)
- Ela Erdem Eralp (ORCID: https://orcid.org/0000-0001-8829-3431)
- Bulent Taner Karadag (ORCID: https://orcid.org/0000-0003-0605-8871)
- Simona Basilicata
- Simone Helms
- Sarah Altaraihi
- Johanna Raidt (ORCID: https://orcid.org/0000-0002-1571-2647)
- Massimo Pifferi (ORCID: https://orcid.org/0000-0002-8266-1923)
- Uğur Özçelik (ORCID: https://orcid.org/0000-0003-1587-5216)
- Jobst Röhmel
- Serena Grazzi
- Niklas Zieghan
- Hatice Nur Coemert
- Nagehan Emiralioglou
Institutions
- University of Pisa (IT)
- Cyprus University of Technology (CY)
- Aristotle University of Thessaloniki (GR)
- Copenhagen University Hospital (DK)
- Rigshospitalet (DK)
- University of Cyprus (CY)
- Humboldt-Universität zu Berlin (DE)
- Augenstern (DE)
- University Hospital Münster (DE)
- Ippokrateio General Hospital of Thessaloniki (GR)
- Federico II University Hospital (IT)
- Bambino Gesù Children's Hospital (IT)
- Vall d'Hebron Hospital Universitari (ES)
- Azienda Ospedaliera Universitaria Pisana (IT)
- Hippocration General Hospital (GR)
- Hacettepe University (TR)
- Marmara University (TR)
Publication Details
- Published
- 2024-09-14
- DOI
- https://doi.org/10.1183/13993003.congress-2024.pa2382
- Citations
- 1
- Primary Topic
- Cystic Fibrosis Research Advances
- Type
- article
- Field-Weighted Citation Impact
- 0.42