scholarly journals Pulmonary exacerbations in patients with primary ciliary dyskinesia: an expert consensus definition for use in clinical trials

2019 ◽  
Vol 5 (1) ◽  
pp. 00147-2018 ◽  
Author(s):  
Jane S. Lucas ◽  
Florian Gahleitner ◽  
Adelina Amorim ◽  
Mieke Boon ◽  
Philippa Brown ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Primary Ciliary Dyskinesia ◽  
Consensus Definition ◽  
Face To Face ◽  
Modified Delphi ◽  
Chronic Lung Diseases ◽  
Sputum Volume ◽  
Pulmonary Exacerbations ◽  
Definition Of ◽  
Ciliary Dyskinesia

Pulmonary exacerbations are a cause of significant morbidity in patients with primary ciliary dyskinesia (PCD) and are frequently used as an outcome measure in clinical research into chronic lung diseases. So far, there has been no consensus on the definition of pulmonary exacerbations in PCD.30 multidisciplinary experts and patients developed a consensus definition for children and adults with PCD. Following a systematic review, the panel used a modified Delphi process with a combination of face-to-face meetings and e-surveys to develop a definition that can be used in research settings for children and adults with PCD.A pulmonary exacerbation was defined by the presence of three or more of the following seven items: 1) increased cough, 2) change in sputum volume and/or colour, 3) increased shortness of breath perceived by the patient or parent, 4) decision to start or change antibiotic treatment because of perceived pulmonary symptoms, 5) malaise, tiredness, fatigue or lethargy, 6) new or increased haemoptysis, and 7) temperature >38°C.The consensus panel proposed that the definition should be used for future clinical trials. The definition should be validated and the usability assessed during these studies.

scholarly journals Pulmonary exacerbation in adults with bronchiectasis: a consensus definition for clinical research

2017 ◽  
Vol 49 (6) ◽  
pp. 1700051 ◽  
Author(s):  
Adam T. Hill ◽  
Charles S. Haworth ◽  
Stefano Aliberti ◽  
Alan Barker ◽  
Francesco Blasi ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Clinical Research ◽  
Working Group ◽  
Exercise Tolerance ◽  
Research Collaboration ◽  
Round Table ◽  
Consensus Definition ◽  
The Us ◽  
Sputum Volume ◽  
Definition Of

There is a need for a clear definition of exacerbations used in clinical trials in patients with bronchiectasis. An expert conference was convened to develop a consensus definition of an exacerbation for use in clinical research.A systematic review of exacerbation definitions used in clinical trials from January 2000 until December 2015 and involving adults with bronchiectasis was conducted. A Delphi process followed by a round-table meeting involving bronchiectasis experts was organised to reach a consensus definition. These experts came from Europe (representing the European Multicentre Bronchiectasis Research Collaboration), North America (representing the US Bronchiectasis Research Registry/COPD Foundation), Australasia and South Africa.The definition was unanimously approved by the working group as: a person with bronchiectasis with a deterioration in three or more of the following key symptoms for at least 48 h: cough; sputum volume and/or consistency; sputum purulence; breathlessness and/or exercise tolerance; fatigue and/or malaise; haemoptysis AND a clinician determines that a change in bronchiectasis treatment is required.The working group proposes the use of this consensus-based definition for bronchiectasis exacerbation in future clinical research involving adults with bronchiectasis.

scholarly journals Defining predatory journals and responding to the threat they pose: a modified Delphi consensus process

BMJ Open ◽  
2020 ◽  
Vol 10 (2) ◽  
pp. e035561 ◽  
Author(s):  
Samantha Cukier ◽  
Manoj Lalu ◽  
Gregory L Bryson ◽  
Kelly D Cobey ◽  
Agnes Grudniewicz ◽  
...  
Keyword(s):  
Delphi Survey ◽  
Educational Outreach ◽  
Consensus Definition ◽  
Ongoing Research ◽  
Predatory Journals ◽  
Policy Initiatives ◽  
International Group ◽  
Modified Delphi ◽  
Definition Of ◽  
Predatory Journal

ObjectiveTo conduct a Delphi survey informing a consensus definition of predatory journals and publishers.DesignThis is a modified three-round Delphi survey delivered online for the first two rounds and in-person for the third round. Questions encompassed three themes: (1) predatory journal definition; (2) educational outreach and policy initiatives on predatory publishing; and (3) developing technological solutions to stop submissions to predatory journals and other low-quality journals.ParticipantsThrough snowball and purposive sampling of targeted experts, we identified 45 noted experts in predatory journals and journalology. The international group included funders, academics and representatives of academic institutions, librarians and information scientists, policy makers, journal editors, publishers, researchers involved in studying predatory journals and legitimate journals, and patient partners. In addition, 198 authors of articles discussing predatory journals were invited to participate in round 1.ResultsA total of 115 individuals (107 in round 1 and 45 in rounds 2 and 3) completed the survey on predatory journals and publishers. We reached consensus on 18 items out of a total of 33 to be included in a consensus definition of predatory journals and publishers. We came to consensus on educational outreach and policy initiatives on which to focus, including the development of a single checklist to detect predatory journals and publishers, and public funding to support research in this general area. We identified technological solutions to address the problem: a ‘one-stop-shop’ website to consolidate information on the topic and a ‘predatory journal research observatory’ to identify ongoing research and analysis about predatory journals/publishers.ConclusionsIn bringing together an international group of diverse stakeholders, we were able to use a modified Delphi process to inform the development of a definition of predatory journals and publishers. This definition will help institutions, funders and other stakeholders generate practical guidance on avoiding predatory journals and publishers.

Definition Of Clinical Criteria For Diagnosis Of Primary Ciliary Dyskinesia

2012 ◽  
Author(s):  
Margaret W. Leigh ◽  
Adam J. Shapiro ◽  
Jessica E. Pittman ◽  
Stephanie D. Davis ◽  
Hyeseung Lee ◽  
...  
Keyword(s):  
Primary Ciliary Dyskinesia ◽  
Clinical Criteria ◽  
Criteria For Diagnosis ◽  
Definition Of ◽  
Ciliary Dyskinesia

scholarly journals A Consensus Definition of Misophonia: Using a Delphi Process to Reach Expert Agreement

2021 ◽  
Author(s):  
Susan Swedo ◽  
David M. Baguley ◽  
Damiaan Denys ◽  
Laura J. Dixon ◽  
Mercede Erfanian ◽  
...  
Keyword(s):  
Delphi Method ◽  
Clinical Evidence ◽  
Consensus Building ◽  
Expert Committee ◽  
Consensus Definition ◽  
Modified Delphi ◽  
Building Process ◽  
Definition Of ◽  
Common Understanding ◽  
Iterative Voting

AbstractMisophonia is a disorder of decreased tolerance to specific sounds or their associated stimuli that has been characterized using different language and methodologies. The absence of a common understanding or foundational definition of misophonia hinders progress in research to understand the disorder and develop effective treatments for individuals suffering from misophonia. From June 2020 through January 2021, a project was conducted to determine whether a committee of experts with diverse expertise related to misophonia could develop a consensus definition of misophonia. An expert committee used a modified Delphi method to evaluate candidate definitional statements that were identified through a systematic review of the published literature. Over four rounds of iterative voting, revision, and exclusion, the committee made decisions to include, exclude, or revise these statements in the definition based on the currently available scientific and clinical evidence. A definitional statement was included in the final definition only after reaching consensus at 80% or more of the committee agreeing with its premise and phrasing. The results of this rigorous consensus-building process were compiled into a final definition of misophonia that is presented here. This definition will serve as an important step to bring cohesion to the growing field of researchers and clinicians who seek to better understand and support individuals experiencing misophonia.

Lung clearance index predicts pulmonary exacerbations in individuals with primary ciliary dyskinesia: a multicentre cohort study

Thorax ◽  
2021 ◽  
pp. thoraxjnl-2020-215504
Author(s):  
Florian Singer ◽  
Anne Schlegtendal ◽  
Sylvia Nyilas ◽  
François Vermeulen ◽  
Mieke Boon ◽  
...  
Keyword(s):  
Cohort Study ◽  
Lung Function ◽  
Primary Ciliary Dyskinesia ◽  
Response To Treatment ◽  
Pulmonary Exacerbation ◽  
Lung Clearance ◽  
Lung Clearance Index ◽  
Pulmonary Exacerbations ◽  
The Impact ◽  
Ciliary Dyskinesia

BackgroundLung clearance index (LCI) is a promising lung function outcome in individuals with primary ciliary dyskinesia (PCD). The impact of events clinically important for individuals with PCD, such as pulmonary exacerbations, on LCI is unknown.MethodsWe conducted an international, multicentre, observational cohort study to assess the association of LCI and risk of pulmonary exacerbation, specific changes in LCI during pulmonary exacerbation and global variability of LCI across four visits every 4 months. Ninety individuals with PCD, aged 3–41 years, underwent nitrogen multiple-breath washout (MBW) and spirometry measurements. The association of LCI and pulmonary exacerbations was assessed by Cox proportional hazards and random-effects regression models.ResultsWe obtained 430 MBW and 427 spirometry measurements. In total, 379 person-years at risk contributed to the analysis. Per one unit increase (deterioration) in LCI, the risk of future pulmonary exacerbation increased by 13%: HR (95% CI), 1.13 (1.04 to 1.23). If LCI changed from a range of values considered normal to abnormal, the risk of future pulmonary exacerbations increased by 87%: 1.87 (1.08 to 3.23). During pulmonary exacerbations, LCI increased by 1.22 units (14.5%). After pulmonary exacerbations, LCI tended to decline. Estimates of variability in LCI suggested lower variation within individuals compared with variation between individuals. Findings were comparable for forced expiratory volume in 1 s.ConclusionOn a visit-to-visit basis, LCI measurement may add to the prediction of pulmonary exacerbations, the assessment of lung function decline and the potential lung function response to treatment of pulmonary exacerbations.

scholarly journals Changes in airway inflammation during pulmonary exacerbations in patients with cystic fibrosis and primary ciliary dyskinesia

2015 ◽  
Vol 47 (3) ◽  
pp. 829-836 ◽  
Author(s):  
Felix Ratjen ◽  
Valerie Waters ◽  
Michelle Klingel ◽  
Nancy McDonald ◽  
Sharon Dell ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Inflammatory Response ◽  
Neutrophil Elastase ◽  
Primary Ciliary Dyskinesia ◽  
Bacterial Pathogens ◽  
Pulmonary Exacerbation ◽  
Elastase Activity ◽  
Cell Counts ◽  
Pulmonary Exacerbations ◽  
Ciliary Dyskinesia

Lung disease in patients with both primary ciliary dyskinesia (PCD) or cystic fibrosis (CF) is associated with impaired mucociliary clearance; however, clinical outcomes are typically worse in CF patients. We assessed whether CF and PCD patients differ in inflammatory response in the airways during pulmonary exacerbation.We first studied clinically stable PCD patients with a spectrum of bacterial pathogens to assess inflammatory response to different pathogens. Subsequently, PCD and CF patients with similar bacterial pathogens were studied at the time of a pulmonary exacerbation and after 21 days of antibiotics treatment. Qualitative and quantitative microbiology, cell counts, interleukin-8 concentrations, and neutrophil elastase activity were assessed in sputum samples obtained before and after treatment.In stable PCD patients, no significant differences were found in sputum inflammatory markers between individuals colonised with different bacterial pathogens. Pulmonary exacerbation severity assessed by a pulmonary exacerbation score and lung function decline from their previous baseline did not differ between CF and PCD patients. Bacterial density for Staphylococcus aureus and Haemophilus influenzae was higher in CF versus PCD (p<0.05), but absolute neutrophil counts were higher in PCD patients (p=0.02). While sputum elastase activity was similar in PCD and CF at the time of exacerbation, it decreased with antibiotic therapy in PCD (p<0.05) but not CF patients.PCD patients differ from those with CF in their responses to treatment of pulmonary exacerbations, with higher neutrophil elastase activity persisting in the CF airways at the end of treatment.

scholarly journals Agitation in cognitive disorders: International Psychogeriatric Association provisional consensus clinical and research definition

2014 ◽  
Vol 27 (1) ◽  
pp. 7-17 ◽  
Author(s):  
Jeffrey Cummings ◽  
Jacobo Mintzer ◽  
Henry Brodaty ◽  
Mary Sano ◽  
Sube Banerjee ◽  
...  
Keyword(s):  
Cognitive Impairment ◽  
Work Group ◽  
Cognitive Disorders ◽  
Development Programs ◽  
Consensus Definition ◽  
Face To Face ◽  
Dementia Syndrome ◽  
Widespread Agreement ◽  
Definition Of ◽  
Strongly Agree

ABSTRACTBackground:Agitation is common across neuropsychiatric disorders and contributes to disability, institutionalization, and diminished quality of life for patients and their caregivers. There is no consensus definition of agitation and no widespread agreement on what elements should be included in the syndrome. The International Psychogeriatric Association formed an Agitation Definition Work Group (ADWG) to develop a provisional consensus definition of agitation in patients with cognitive disorders that can be applied in epidemiologic, non-interventional clinical, pharmacologic, non-pharmacologic interventional, and neurobiological studies. A consensus definition will facilitate communication and cross-study comparison and may have regulatory applications in drug development programs.Methods:The ADWG developed a transparent process using a combination of electronic, face-to-face, and survey-based strategies to develop a consensus based on agreement of a majority of participants. Nine-hundred twenty-eight respondents participated in the different phases of the process.Results:Agitation was defined broadly as: (1) occurring in patients with a cognitive impairment or dementia syndrome; (2) exhibiting behavior consistent with emotional distress; (3) manifesting excessive motor activity, verbal aggression, or physical aggression; and (4) evidencing behaviors that cause excess disability and are not solely attributable to another disorder (psychiatric, medical, or substance-related). A majority of the respondents rated all surveyed elements of the definition as “strongly agree” or “somewhat agree” (68–88% across elements). A majority of the respondents agreed that the definition is appropriate for clinical and research applications.Conclusions:A provisional consensus definition of agitation has been developed. This definition can be used to advance interventional and non-interventional research of agitation in patients with cognitive impairment.

scholarly journals Is Quarantine for COVID-19 Pandemic Associated with Psychological Burden in Primary Ciliary Dyskinesia?

2020 ◽  
Vol 17 (21) ◽  
pp. 8099
Author(s):  
Maria Pia Riccio ◽  
Melissa Borrelli ◽  
Maria Teresa Fioretti ◽  
Margherita Del Bene ◽  
Carmela Bravaccio ◽  
...  
Keyword(s):  
Parental Stress ◽  
Primary Ciliary Dyskinesia ◽  
Well Being ◽  
Chest Physiotherapy ◽  
Exacerbation Rate ◽  
Psychological Well Being ◽  
Psychological Burden ◽  
Significant Difference ◽  
Pulmonary Exacerbations ◽  
Ciliary Dyskinesia

Background: Information on psychological impact of COVID-19 quarantine in primary ciliary dyskinesia (PCD), a chronic disorder with recurrent pulmonary exacerbations, is lacking. Psychological well-being was prospectively assessed during COVID-19 lockdown in Italy in a PCD population. Methods: we recruited 27 PCD patients and 27 healthy controls. To assess psychological well-being, psychological general well-being index and parenting stress index-short questionnaires were administered to participants ≥15 years-old and to mothers of participants <15 years-old, respectively. The PCD exacerbations since outbreak onset and frequency of quarantine weekly chest physiotherapy were compared to the same period of 2019. Outcomes: 70% of PCD mothers and 90% of PCD patients did not show parental stress levels or distress levels, respectively, and these groups showed no significant difference in stress compared to controls. The PCD pulmonary exacerbations occurred less frequently and weekly chest physiotherapy sessions significantly increased compared to the same period during 2019 (p < 0.05). Interpretation: During COVID-19 quarantine, a PCD population showed psychological well-being. Low exacerbation rate, explained by lower infectious exposure or improved compliance to chest physiotherapy, likely contributed to psychological well-being. Evaluating psychological burden and parental stress is a valuable tool for measuring the emotional impact of PCD and improving PCD medical care.

scholarly journals International consensus guideline for reporting transmission electron microscopy results in the diagnosis of primary ciliary dyskinesia (BEAT PCD TEM Criteria)

2020 ◽  
Vol 55 (4) ◽  
pp. 1900725 ◽  
Author(s):  
Amelia Shoemark ◽  
Mieke Boon ◽  
Christoph Brochhausen ◽  
Zuzanna Bukowy-Bieryllo ◽  
Maria M. De Santi ◽  
...  
Keyword(s):  
Electron Microscopy ◽  
Transmission Electron Microscopy ◽  
Primary Ciliary Dyskinesia ◽  
Supporting Evidence ◽  
Consensus Guideline ◽  
Delphi Consensus ◽  
International Consensus ◽  
Transmission Electron ◽  
Definition Of ◽  
Ciliary Dyskinesia

Primary ciliary dyskinesia (PCD) is a heterogeneous genetic condition. European and North American diagnostic guidelines recommend transmission electron microscopy (TEM) as one of a combination of tests to confirm a diagnosis. However, there is no definition of what constitutes a defect or consensus on reporting terminology. The aim of this project was to provide an internationally agreed ultrastructural classification for PCD diagnosis by TEM.A consensus guideline was developed by PCD electron microscopy experts representing 18 centres in 14 countries. An initial meeting and discussion were followed by a Delphi consensus process. The agreed guideline was then tested, modified and retested through exchange of samples and electron micrographs between the 18 diagnostic centres.The final guideline a) provides agreed terminology and a definition of Class 1 defects which are diagnostic for PCD; b) identifies Class 2 defects which can indicate a diagnosis of PCD in combination with other supporting evidence; c) describes features which should be included in a ciliary ultrastructure report to assist multidisciplinary diagnosis of PCD; and d) defines adequacy of a diagnostic sample.This tested and externally validated statement provides a clear guideline for the diagnosis of PCD by TEM which can be used to standardise diagnosis internationally.

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