Background
Home mechanical ventilation (HMV) represents one of the most advanced, highly specialized, and complex therapies offered to patients outside of a hospital setting. Users are dependent on medical technology for the vital life function of breathing. Neuromuscular diseases are the most common indication for HMV in children. Although the HMV population is relatively small, the incidence of HMV initiation and associated healthcare utilization and costs are increasing, with high mortality rates that are not changing over time.
Despite the medical complexity of these patients and their intense healthcare service utilization and associated costs, there are few randomized controlled trials (RCTs) in children requiring HMV. Barriers to conducting high quality clinical trials in the HMV population include inadequate sample sizes within single centers, lack of comparability among data due to heterogeneity in clinical practice, absence of existing national infrastructure to support HMV RCTs, and lack of a core outcome set resulting in heterogeneity of outcomes and measures reported in trials. Given the growing prevalence yet high levels of morbidity and mortality in this patient population, a core outcome set is crucial to informing future research. Furthermore, we have chosen to produce a core outcome set for children as we anticipate outcomes of importance will be different to those considered important by an adult patient population.
Aim
To establish a core outcome set for studies of interventions for children requiring home mechanical ventilator support (either invasive or non-invasive).
Objectives
1. To characterise selection and definition of outcomes and measures used in existing studies of interventions for children requiring home mechanical ventilator support (either invasive or non-invasive); and
2. To obtain consensus on a core outcome set for future studies of interventions for children requiring home mechanical ventilator support.
Methods
Item generation
1 Systematic Review: we will extract data on primary and secondary outcomes reported, measures used and measurement time-points from published studies recruiting an paeditaric population.
2. We will conduct semi-structured interviews with parents and caregivers, children (where able) and other relevant stakeholders to establish outcomes they consider important to measure in future studies of interventions for adults requiring home mechanical ventilator support.
Consensus Building
1. We will conduct a modified Delphi study to establish consensus on outcomes for inclusion in a core outcome set. We will recruit an international expert panel representing key stakeholders and end users including parents, family members, and children requiring home ventilation.
2. If consensus on a core outcome set is not achieved via the Delphi process, we will host a virtual consensus meeting using nominal group technique and voting to establish the core outcomes.
Louise Rose (PI)
King's College London
Reshma Amin (co-PI)
The Hospital for Sick Children, University of Toronto
Andrea Gershon
Sunnybrook Health Sciences Centre, University of Toronto, ICES, The Hospital for Sick Children Research Institute
Francine Buchanan
The Hospital for Sick Children
Marta Kaminska
McGill University
Tetyana Kendzerska
University of Ottawa, Ottawa Hospital
Lena Xiao
University of British Columbia, BC Children's Hospital
Colleen O'Connell
University of New Brunswick, Dalhousie University
Jean-Paul Praud
University of Sherbrooke
David Zielinski
McGill University, McGill University Health Centre
Tim Ramsay
Ottawa Methods Centre, Ottawa Hospital Research Institute, University of Ottawa
Cheryl Lartta
University of Alberta
Irvin Mayers
University of Alberta
Homira Osman
MD Canada
Andrea Loewen
University of Calgary, Peter Lougheed Center
Anu Tandon
University of Toronto, Sunnybrook Health Sciences Centre
Maria Castro Codesal
University of Alberta
Indra Narng
University of Toronto, The Hospital for Sick Children
Tom Kovesi
University of Ottawa, Children's Hospital of Eastern Ontario
Susi Vander Wyk
Cure SMA Canada
David Berlowitz
University of Melbourne, Victorian Respiratory Support Service
Lisa Wolfe
Northwestern University
Marie Wright
BC Children’s Hospital, University of British Columbia
Aditi Shah
Vancouver General Hospital, University of British Columbia
Joanna MacLean
Alberta Children’s Hospital, University of Alberta
Kevin John Solverson
University of Calgary, Peter Lougheed Center
Jenny Shi
University of Calgary, Alberta Children’s Hospital
Eleni Giannouli
University of Manitoba
Tareq Alabdoulsalam
University of Manitoba
David Leasa
London Health Sciences
Ron Butler
London Health Sciences, Western University
Aaron St-Laurent
London Health Sciences, Western University
Doug McKim
Ottawa Hospital, University of Ottawa
Kevan Mehta
McMaster Children’s Hospital, McMaster University
Audrey Lim
McMaster Children’s Hospital, McMaster University
Jalal Moolji
Alberta Health Services
Jackie Chiang
The Hospital for Sick Children, University of Toronto
Nisha Cithiravel
The Hospital for Sick Children
Tuyen Tran
The Hospital for Sick Children
Mika Nonoyama
The Hospital for Sick Children, Ontario Tech University
Faiza Syed
The Hospital for Sick Children
Refika Ersu
Children’s Hospital of Eastern Ontario, University of Ottawa
Marielena Dibartolo
Children’s Hospital of Eastern Ontario
Robert Varadi
WestPark Rehabilitation Hospital
Craig Dale
Sunnybrook Health Sciences, University of Toronto
The Thanh-diem Bguyen
CHU Sainte-Justine, University of Montreal
Mathew Cherian
McGill University
Veroniqie Adam
National Program for Home Ventilatory Assistance
Dimas Mateos
IWK Hospital, Dalhousie University
Regina Pizzuti
Ontario Ventilator Equipment Pool, Kingston General Hospital
Disease Category: Lungs & airways
Disease Name:
Age Range: 0 - 19
Sex: Either
Nature of Intervention: Mechanical ventilation
- Charities
- Clinical experts
- Consumers (caregivers)
- Consumers (patients)
- Families
- Researchers
- Service providers
- Service users
- COS for clinical trials or clinical research
- Consensus meeting
- Delphi process
- Interview
- Systematic review
Methods
Item generation
1 Systematic Review: we will extract data on primary and secondary outcomes reported, measures used and measurement time-points from published studies recruiting an adult population.
2. We will conduct semi-structured interviews with adults using home ventilation, family members and other relevant stakeholders to establish outcomes they consider important to measure in future studies of interventions for adults requiring home mechanical ventilator support.
Consensus Building
1. We will conduct a modified Delphi study to establish consensus on outcomes for inclusion in a core outcome set. We will recruit an international expert panel representing key stakeholders and end users including adults requiring home mechanical ventilator support and family members.
2. If consensus on a core outcome set is not achieved via the Delphi process, we will host a virtual consensus meeting using nominal group technique and voting to establish the core outcomes.