Translation of the quality-of-life measure for adults with primary ciliary dyskinesia and its application in patients in Brazil

Queiroz, Ana Paula; Athanazio, Rodrigo Abensur; Olm, Mary Anne Kowal; Rubbo, Bruna; Casal, Yuri Reis; Lucas, Jane S.; Behan, Laura

doi:10.1590/1806-3713/e20170358

Cited by 5 publications

(4 citation statements)

References 9 publications

(12 reference statements)

Supporting

Mentioning

Contrasting

Order By: Relevance

“…The limitations of the present study include the small sample size; the fact that some patients had undergone surgery before the publication of a PCD-specific quality of life questionnaire (QOL-PCD), 21 which precluded the use of this instrument; and the absence of a control group.…”

Section: Discussionmentioning

confidence: 99%

Outcomes of Endoscopic Sinus Surgery for Chronic Rhinosinusitis in Adults with Primary Ciliary Dyskinesia

Plantier

Pinna

Olm

et al. 2023

Int Arch Otorhinolaryngol

Self Cite

View full text Add to dashboard Cite

Introduction Primary ciliary dyskinesia (PCD) is a rare inherited disease associated with impairment of mucociliary transport and, consequently, with a high incidence of chronic rhinosinusitis. For patients with chronic rhinosinusitis who remain symptomatic despite medical treatment, endoscopic sinus surgery is a safe and effective therapeutic option. However, to date, no studies have been found evaluating the effect of surgery on the quality of life associated with the effect on olfaction and nasal endoscopy findings of patients with primary ciliary dyskinesia and chronic rhinosinusitis. Objective To describe the effect of endoscopic sinus surgery on the quality of life, on olfaction, and on nasal endoscopy findings of adults with PCD and chronic rhinosinusitis. Methods Four patients who underwent endoscopic sinus surgery were included. The Sinonasal Outcome Test-22 (SNOT-22) score, the Nasal Obstruction Symptom Evaluation (NOSE) questionnaire, and the Lund-Kennedy score were collected preoperatively and at 3 and 6 months postoperatively. The olfaction as assessed with the University of Pennsylvania Smell Identification Test (UPSIT), which was administered preoperatively and 3 months postoperatively. Results A total of 4 patients with a mean age of 39.3 years old (3 men and 1 woman) completed the study. All patients showed clinically significant improvement in the SNOT-22, NOSE, and Lund-Kennedy scores at 3 months postoperatively, and this improvement was sustained throughout the follow-up period. However, olfaction did not improve after surgery. Conclusion The endoscopic sinus surgery treatment of chronic rhinosinusitis in adults with PCD was associated with improvement in quality of life and endoscopic findings. However, no improvement in olfaction was demonstrated. Studies with a larger number of patients and control groups should help confirm these findings.

show abstract

Section: Discussionmentioning

confidence: 99%

Outcomes of Endoscopic Sinus Surgery for Chronic Rhinosinusitis in Adults with Primary Ciliary Dyskinesia

Plantier

Pinna

Olm

et al. 2023

Int Arch Otorhinolaryngol

Self Cite

View full text Add to dashboard Cite

show abstract

“…A systematic review on the patient's experience of PCD reported worsening of respiratory symptoms with age, which was also associated with decline in the physical and mental domains [ 148 ]. QOL-PCD, a HRQoL instrument, is the only validated disease- and age-specific cross-cultural clinical outcome measure in PCD [ 71 , 128 , 129 , 149 , 150 ]. QOL-PCD correlated well with the Sino-Nasal Outcome Test (SNOT-20) for upper airway symptoms, the COPD-specific SGRQ for lower airways symptoms and SF-36 for physical functioning, role functioning and mental health [ 71 ].…”

Section: Discussionmentioning

confidence: 99%

Lower airway clinical outcome measures for use in primary ciliary dyskinesia research: a scoping review

et al. 2021

Self Cite

View full text Add to dashboard Cite

ObjectivesDisease-specific, well-defined, and validated clinical outcome measures are essential in designing research studies. Poorly defined outcome measures hamper pooling of data and comparisons between studies. We aimed to identify and describe pulmonary outcome measures that could be used for follow-up of patients with primary ciliary dyskinesia (PCD).MethodsWe conducted a scoping review by systematically searching Medline, Embase and Cochrane Systematic Review online databases for studies published from 1996 to 2020 that included ≥10 PCD adult and/or paediatric patients.ResultsWe included 102 studies (7289 patients). Eighty-three studies reported on spirometry, 11 on body plethysmography, 15 on multiple breath washout, 36 on high-resolution computed tomography (HRCT), 57 on microbiology, and 17 on health-related quality of life. Measurement and reporting of outcomes varied considerably between studies (e.g. different scoring systems for chest HRCT scans). Additionally, definitions of outcome measures varied (e.g. definition of chronic colonisation by respiratory pathogen), impeding direct comparisons of results.ConclusionsThis review highlights the need for standardisation of measurements and reporting of outcome measures to enable comparisons between studies. Defining a core set of clinical outcome measures is necessary to ensure reproducibility of results and for use in future trials and prospective cohorts.

show abstract

“…Ensuring QOL-PCD is both valid and robust required International collaboration; rigorous literature review [41]; expert and patient interviews with psychometric analysis to ensure the clarity and importance of each question. BESTCILIA and BEAT-PCD facilitated opportunities to undertake translation of QOL-PCD into more than 8 languages [42,43]. Siobhan Carr (UK) and Jane Lucas (UK) led the pulmonary exacerbation consensus group.…”

Section: Work Group Activities and Training Schoolthe Highlights Of Bmentioning

confidence: 99%

Proceedings of the 4th BEAT-PCD Conference and 5th PCD Training School

et al. 2020

Self Cite

View full text Add to dashboard Cite

Primary ciliary dyskinesia (PCD) is an inherited ciliopathy leading to chronic suppurative lung disease, chronic rhinosinusitis, middle ear disease, sub-fertility and situs abnormalities. As PCD is rare, it is important that scientists and clinicians foster international collaborations to share expertise in order to provide the best possible diagnostic and management strategies. 'Better Experimental Approaches to Treat Primary Ciliary Dyskinesia' (BEAT-PCD) is a multidisciplinary network funded by EU COST Action (BM1407) to coordinate innovative basic science and clinical research from across the world to drive advances in the field. The fourth and final BEAT-PCD Conference and fifth PCD Training School were held jointly in March 2019 in Poznan, Poland. The varied program of plenaries, workshops, break-out sessions, oral and poster presentations were aimed to enhance the knowledge and skills of delegates, whilst also providing a collaborative platform to exchange ideas. In this final BEAT-PCD conference we were able to build upon programmes developed throughout the lifetime of the COST Action. These proceedings report on the conference, highlighting some of the successes of the BEAT-PCD programme.

show abstract

Translation of the quality-of-life measure for adults with primary ciliary dyskinesia and its application in patients in Brazil

Cited by 5 publications

References 9 publications

Outcomes of Endoscopic Sinus Surgery for Chronic Rhinosinusitis in Adults with Primary Ciliary Dyskinesia

Outcomes of Endoscopic Sinus Surgery for Chronic Rhinosinusitis in Adults with Primary Ciliary Dyskinesia

Lower airway clinical outcome measures for use in primary ciliary dyskinesia research: a scoping review

Proceedings of the 4th BEAT-PCD Conference and 5th PCD Training School

Contact Info

Product

Resources

About