Core Outcome Measures in Effectiveness Trials

Developing a Core Outcome Set for Childhood Obstructive Sleep-Disordered Breathing

General Information

Summary:
Childhood obstructive Sleep Disordered-Breathing (oSDB) encompasses breathing problems whilst asleep due to obstruction of the upper airways. Severity ranges from primary snoring without respiratory compromise, to obstructive sleep apnoea (OSA) syndrome with significant desaturations overnight and potential adverse neurocognitive and cardiovascular outcomes. Despite growing interest in oSDB, clinicians and parents face many uncertainties about how best to diagnose and manage this condition. Inconsistency in outcome measures and the outcome measurement instruments across studies of oSDB play a key role in this uncertainty. There is therefore an urgent need for research that identifies which outcome measures and measurement instruments best capture the impact of obstructive sleep-disordered breathing in children. We aim to develop a Core Outcome Set (COS) for childhood oSDB that involves patients, healthcare professionals and other stakeholders which will maximise the value of future research and inform clinical guidance.


Contributors:
Dr Laurie Rigueros Springford, Gold Coast University Hospital, 1 Hospital Boulevard, Southport, QLD, Australia 4215

Dr Roderick Venekamp, Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands

Dr Helen Blackshaw, evidENT, Ear Institute, University College London, Royal National Throat Nose and Ear Hospital, 330 Gray’s Inn Road, London, U.K, WC1X 8DA

Dr Francois Abel, Paediatric Consultant in Respiratory and Sleep Medicine, Great Ormond Street Hospital for Children London, WC1N 3JH, UK

Dr Hazel Evans, University Hospital Southampton NHS Foundation Trust, Tremona Road, Southampton, Hampshire, SO16 6YD, UK

Mr Bilal Alkhaffaf, Department of Oesophago-Gastric Surgery, Manchester Royal Infirmary, Manchester University NHS Foundation Trust, Manchester Academic Health Science Centre, Manchester, M13 9WL, UK

Professor Iain Bruce, Paediatric ENT Department, Royal Manchester Children’s Hospital, Manchester University NHS Foundation Trust, Manchester Academic Health Science Centre, Manchester, M13 9WL, UK

Professor Anne Schilder, evidENT, Ear Institute, University College London, Royal National Throat Nose and Ear Hospital, 330 Gray’s Inn Road, London, U.K, WC1X 8DA

Further Study Information

Current Stage:
Planning
Date:
Not stated
Funding source(s):
To be confirmed

Health Area

Disease Category
Ear, nose, & throat

Disease Name
Obstructive sleep disordered breathing

Target Population

Age Range
2 - 18

Sex
Either


Nature / type of Intervention
Any

Method(s)

Consensus meeting
Delphi process
Focus group(s)
Interview
Systematic review

The methodology has been adapted from the publications:

Williamson PR, Altman DG, Bagley H, Barnes KL, Blazeby JM, Brookes ST, Clarke M, Gargon E, Gorst S, Harman N, Kirkham JJ, McNair A, Prinsen CAC, Schmitt J, Terwee CB, Young B. The Comet Handbook: Version 1.0. 2017. Trials. 18(Suppl 3):280

Harman NL, Bruce IA, Kirkham JJ, Tierney S, Callery P, O'Brien K, Bennett AM, Chorbachi R, Hall PN, Harding-Bell A, Parfect VH, Rumsey N, Sell D, Sharma R, Williamson PR. The Importance of Integration of Stakeholder Views in Core Outcome Set Development: Otitis Media with Effusion in Children with Cleft Palate. 2015. Plos One. 10(6).

Alkhaffaf B, Glenny AM, Blazeby JM, Williamson P, Bruce IA. Standardising the Reporting of Outcomes in Gastric Cancer Surgery Trials: Protocol for the Development of a Core Outcome Set and Accompanying Outcome Measurement Instrument Set (the GASTROS Study). 2017. Trials. 18(1).


Identification of outcomes measures

(1) Systematic review, protocol design, ethics approval

The first stage will aim to capture the outcomes and outcome measurement instruments (OMIs) used in the published literature concerning childhood oSDB. To achieve this, we will systematically review observational and interventional studies and extract the outcomes (whether primary or secondary outcome), OMIs (instruments/tools/indicators) and time point(s) for measurements. The named collaborators will act as the Study Management Group (SMG). In conjunction with recommendations made by COMET, a protocol demonstrating development of the core outcome set will be published. Ethical approval will be sought.

(2) Interviews and focus groups

The views of parents and children (including a representative sample of ages and genders) with oSDB will be sought by the use of semi structured interviews. Interviews will be recorded with audio-visual equipment, transcribed and interpreted by the SMG. Outcomes gathered from the interviews will be used to supplement the list extracted from systematic review of the literature.

In order to finalise the items which will be presented in the eDelphi survey, we will employ the help of a focus group of selected key stakeholders (including parents) (acting as the Study Advisory Group (SAG)). The name and definition of each outcome will be openly discussed, critically analysed, refined and admitted to the eDelphi survey once agreement has been reached by the SAG. Members of the SAG will be given the chance to add outcomes which they consider important to the outcomes long-list.

(3) Delphi process followed by consensus meeting to finalise COS.

The information assembled from the previous steps will be used in a multi-round eDelphi survey using Delphi-Manager software. This process will involve the participation of key stakeholder groups in an online consensus exercise. Each outcome will be included in the survey. They will be described with lay terminology and a respective scientific description. Participants will be asked to rank the importance of each outcome. After each round, outcomes will either be retained or discarded. In the early stages participants will be given the chance to add new outcomes.

(4) Consensus meeting

Finally, a face to face meeting, where key stakeholders participate in discussion to agree and ratify on the final core outcome set for oSDB identified from the eDelphi exercise.


(5) Dissemination

The work conducted will be reported using the COS-STAR guidance. Communication of information relevant to the core outcome set and measurement instruments in oSDB will be disseminated through publications in peer review medical journals, international oral and poster presentations, patient events and involvement with associations/societies. Development of multimedia methods of dissemination will be sought including the creation of a website, Twitter and Facebook profile. Importantly, the information will be distributed to the wider audience involved with children with oSDB, both nationally and internationally.
?Identification of outcome measurement instruments

To select appropriate measurement instruments for the respective outcome, a practical guideline developed by COSMIN (COnsensus-based Standards for the selection of health Measurement INstruments) will be followed. This involves a four-stage process:

(1) Conceptual considerations of the outcomes measured

(2) Identification of the outcome measurement instruments by literature review

(3) Quality assessment of the measurement instruments

(4) Practical recommendations, including a further consensus meeting to finalise the outcome measurement instruments

Importantly, this will identify current outcomes which do not have associated validated measurement instruments. Recommendations will also be made regarding the need for the development of further validated instruments for future observational and interventional studies.

Details of the COSMIN practical guide which will be used can be found:

Prinsen CAC, Vohra S, Rose MR, Boers M, Tugwell P, Clarke M, Williamson PR, Terwee CB. How to Select Outcome Measurement Instruments for Outcomes Included in a "core Outcome Set" - a Practical Guideline. 2016. Trials. 17(1).

An international and comprehensive collaboration of stakeholders will be considered, including:

- Allied healthcare professionals
- Clinicians and clinical experts
- Device manufacturers and industry representatives
- Educational experts and teachers
- Members of relevant societies and associations, including: The British Paediatric Sleep Association, European Respiratory Society and The International Pediatric Otolaryngology Group (IPOG)
- Patients
- Parents and caregivers
- Researchers and research scientists.
- Policy makers and regulatory agency representatives
- Support groups and charities


Stakeholders Involved

Carer organisation/ support group representatives
Charities
Clinical experts
Consumers (caregivers)
Consumers (patients)
Device manufacturers
Other
Patient/ support group representatives
Pharmaceutical industry representatives
Policy makers
Regulatory agency representatives
Researchers

Study Type

COS for clinical trials or clinical research

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