Collecting clinical data in primary ciliary dyskinesia- challenges and opportunities

Rationale: Primary ciliary dyskinesia (PCD) is under diagnosed and underestimated. Most clinical research has used some form of questionnaires to capture data but none has been critically evaluated particularly with respect to its end-user feasibility and utility. Objective: To critically appraise a clinical data collection questionnaire for PCD used in a large national PCD consortium in order to apply conclusions in future PCD research. Methods: We describe the development, validation and revision process of a clinical questionnaire for PCD and its evaluation during a national clinical PCD study with respect to data collection and analysis, initial completion rates and user feedback. Results: 14 centers participating in the consortium successfully completed the revised version of the questionnaire for 173 patients with various completion rates for various items. While content and internal consistency analysis demonstrated validity, there were methodological deficiencies impacting completion rates and end-user utility. These deficiencies were addressed resulting in a more valid questionnaire. Conclusions: Our experience may be useful for future clinical research in PCD. Based on the feedback collected on the questionnaire through analysis of completion rates, judgmental analysis of the content, and feedback from experts and end users, we suggest a practicable framework for development of similar tools for various future PCD research.

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Standardised clinical data from patients with primary ciliary dyskinesia: FOLLOW-PCD

ERJ Open Research ◽

10.1183/23120541.00237-2019 ◽

2020 ◽

Vol 6 (1) ◽

pp. 00237-2019 ◽

Cited By ~ 4

Author(s):

Myrofora Goutaki ◽

Jean-François Papon ◽

Mieke Boon ◽

Carmen Casaulta ◽

Ernst Eber ◽

...

Keyword(s):

Data Collection ◽

Real Time ◽

Clinical Data ◽

Primary Ciliary Dyskinesia ◽

Clinical Decision Making ◽

Clinical Decision ◽

Routine Care ◽

Standards Of Care ◽

Patient Questionnaire ◽

Ciliary Dyskinesia

Clinical data on primary ciliary dyskinesia (PCD) are limited, heterogeneous and mostly derived from retrospective chart reviews, leading to missing data and unreliable symptoms and results of physical examinations. We need standardised prospective data collection to study phenotypes, severity and prognosis and improve standards of care.A large, international and multidisciplinary group of PCD experts developed FOLLOW-PCD, a standardised clinical PCD form and patient questionnaire. We identified existing forms for clinical data collection via the Better Experimental Approaches to Treat PCD (BEAT-PCD) COST Action network and a literature review. We selected and revised the content items with the working group and patient representatives. We then revised several drafts in an adapted Delphi process, refining the content and structure.FOLLOW-PCD has a modular structure, to allow flexible use based on local practice and research focus. It includes patient-completed versions for the modules on symptoms and lifestyle. The form allows a comprehensive standardised clinical assessment at baseline and for annual reviews and a short documentation for routine follow-up. It can either be completed using printable paper forms or using an online REDCap database.Data collected in FOLLOW-PCD version 1.0 is available in real-time for national and international monitoring and research. The form will be adapted in the future after extensive piloting in different settings and we encourage the translation of the patient questionnaires to multiple languages. FOLLOW-PCD will facilitate quality research based on prospective standardised data from routine care, which can be pooled between centres, to provide first-line and real-time evidence for clinical decision-making.

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Clinical research 4: Qualitative data collection and analysis

International Emergency Nursing ◽

10.1016/j.aaen.2006.12.002 ◽

2008 ◽

Vol 16 (1) ◽

pp. 48-52 ◽

Cited By ~ 10

Author(s):

Ruth Endacott

Keyword(s):

Data Collection ◽

Clinical Research ◽

Qualitative Data ◽

Data Collection And Analysis ◽

Qualitative Data Collection

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Clinical research 4: qualitative data collection and analysis

Intensive and Critical Care Nursing ◽

10.1016/j.iccn.2004.10.001 ◽

2005 ◽

Vol 21 (2) ◽

pp. 123-127 ◽

Cited By ~ 27

Author(s):

Ruth Endacott

Keyword(s):

Data Collection ◽

Clinical Research ◽

Qualitative Data ◽

Data Collection And Analysis ◽

Qualitative Data Collection

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Clinical research 5: Quantitative data collection and analysis

International Emergency Nursing ◽

10.1016/j.aaen.2006.12.004 ◽

2008 ◽

Vol 16 (2) ◽

pp. 132-137 ◽

Cited By ~ 3

Author(s):

Mari Botti ◽

Ruth Endacott

Keyword(s):

Data Collection ◽

Clinical Research ◽

Quantitative Data ◽

Data Collection And Analysis

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Respiratory symptoms of Swiss people with Primary Ciliary Dyskinesia

10.1101/2021.11.29.21266978 ◽

2021 ◽

Author(s):

Myrofora Goutaki ◽

Leonie Huesler ◽

Yin Ting Lam ◽

Helena M Koppe ◽

Andreas Jung ◽

...

Keyword(s):

Clinical Data ◽

Primary Ciliary Dyskinesia ◽

Respiratory Symptoms ◽

Phenotypic Variability ◽

Shortness Of Breath ◽

Runny Nose ◽

Nasal Symptoms ◽

Patient Reported ◽

Almost All ◽

Ciliary Dyskinesia

Background: Mostly derived from chart reviews, where symptoms are recorded in a nonstandardised manner, clinical data about primary ciliary dyskinesia (PCD) are inconsistent, which leads to missing and unreliable information. We assessed the prevalence and frequency of respiratory symptoms and studied differences by age and sex among an unselected population of Swiss people with PCD. Methods: We sent a questionnaire that included items from the FOLLOW–PCD standardised questionnaire to all Swiss PCD registry participants. Results: We received questionnaires from 74 out of 86 (86%) invited persons or their caregivers (age range: 3–73 years; median age: 23), including 68% adults (≥18 years) and 51% females. Among participants, 70 (94%) reported chronic nasal symptoms, most frequently runny nose (65%), blocked nose (55%), or anosmia (38%). Ear pain and hearing problems were reported by 58% of the participants. Almost all (99%) reported cough and sputum production. The most common chronic cough complications were gastroesophageal reflux (n=11; 15%), vomiting (n=8; 11%), and urinary incontinence (n=6; 8%). Only 9 participants (12%) reported frequent wheeze, which occurred mainly during infection or exercise, while 49 persons (66%) reported shortness of breath; 9% even at rest or during daily activities. Older patients reported more frequent nasal symptoms and shortness of breath. We found no difference by sex or ultrastructural ciliary defect. Conclusion: This is the first study that describes patient–reported PCD symptoms. The consistent collection of standardised clinical data will allow us to better characterise the phenotypic variability of the disease and study disease course and prognosis.

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Clinical research data management in the United States: Where we've been and where we're going

Journal of the Society for Clinical Data Management ◽

10.47912/jscdm.61 ◽

2021 ◽

Author(s):

Mary Banach ◽

Kaye H Fendt ◽

Johann Proeve ◽

Dale Plummer ◽

Samina Qureshi ◽

...

Keyword(s):

Clinical Research ◽

Data Management ◽

Clinical Data ◽

Management Practices ◽

The United States ◽

Research Data ◽

Fda Guidance ◽

Research Data Management ◽

Challenges And Opportunities ◽

Clinical Research Data

With the focus of the COVID-19 pandemic, we wanted to reach all stakeholders representing communities concerned with good clinical data management practices. We wanted to represent not only data managers but bio-statisticians, clinical monitors, data scientists, informaticians, and all those who collect, organize, analyze, and report on clinical research data. In our paper we will discuss the history of clinical data management in the US and its evolution from the early days of FDA guidance. We will explore the role of biomedical research focusing on the similarities and differences in industry and academia clinical research data management and what we can learn from each other. We will talk about our goals for recruitment and training for the CDM community and what we propose for increasing the knowledge and understanding of good clinical data practice to all – particularly our front-line data collectors i.e., nurses, medical assistants, patients, other data collectors. Finally, we will explore the challenges and opportunities to see CDM as the hub for good clinical data research practices in all of our communities.We will also discuss our survey on how the COVID-19 pandemic has affected the work of CDM in clinical research.

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Report of the 2021 Primary ciliary dyskinesia foundation annual meeting

Translational Science of Rare Diseases ◽

10.3233/trd-210052 ◽

2021 ◽

pp. 1-4

Author(s):

Thomas G. Saba ◽

Steven L. Brody

Keyword(s):

Clinical Research ◽

Annual Meeting ◽

Primary Ciliary Dyskinesia ◽

Virtual Conference ◽

Ciliary Dyskinesia

In August 2021, the Primary Ciliary Dyskinesia (PCD) Foundation hosted a 2-day international virtual conference designed to share discoveries in PCD genetics, cilia biology, and clinical research and care. The conference was organized around the theme of building a community for clinical research. This article provides a report of the proceedings.

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