Wouter Nijhuis1, Anton Franken2, Kara Ayers3, Chantal Damas4, Lars Folkestad5, Antonella Forlino6, Paolo Fraschini7, Claire Hill8, Guus Janus2, Richard Kruse9, Lena Lande Wekre10, Lieve Michiels11, Kathleen Montpetit12, Leonardo Panzeri13, Valerie Porquet-Bordes14, Frank Rauch4, Ralph Sakkers15, Jean-Pierre Salles14, Oliver Semler16, Jony Sun17, Michael To18, Laura Tosi19, Yangyang Yao20, Eric Hiu Kwong Yeung18, Lidiia Zhytnik21, Maria Carola Zillikens22, Marjolein Verhoef15. 1. University Medical Center Utrecht, Utrecht, The Netherlands. W.H.Nijhuis-2@umcutrecht.nl. 2. Isala Zwolle, Utrecht, The Netherlands. 3. Cincinnati Children's Hospital Medical Center, Cincinnati, USA. 4. Shriners Hospitals for Children, Montreal, Canada. 5. University of Southern Denmark, Odense, Denmark. 6. University of Pavia, Pavia, Italy. 7. Rizzoli Institute Bologna, Milano, Italy. 8. Sheffields Children's NHS Trust Foundation, Sheffield, UK. 9. Nemours/Alfred Dupont Hospital for Children, Delaware, USA. 10. TRS National Resource Center for Rare Disorders, Sunnaas Rehabilitation Hospital, Oslo, Norway. 11. ZOI (Flemish OI Association), Brussels, Belgium. 12. Advisor Care4BrittleBones Foundation, Montreal, Canada. 13. AS.IT.O.I. (Italian OI Association), Rome, Italy. 14. CHU de Toulouse, Toulouse, France. 15. University Medical Center Utrecht, Utrecht, The Netherlands. 16. Department of Paediatrics, University of Cologne, Cologne, Germany. 17. China-Dolls Center for Rare Disorders (CCRD), Bejing, China. 18. The University of Hong Kong - Shenzhen Hospital, Hong Kong SAR, Shenzhen, China. 19. Children's National Hospital, Washington, USA. 20. Shandong Provincial Hospital, Jihan, China. 21. Tartu University Hospital, Tartu, Estonia. 22. University Medical Center Erasmus Rotterdam, Rotterdam, The Netherlands.
Abstract
BACKGROUND: Osteogenesis Imperfecta (OI) is a genetic disorder also known as 'brittle bone disease'. The clinical manifestation of OI shows a wide variation. Therefore, care for patients with OI requires an interdisciplinary approach. The effectiveness of particular interventions and treatment protocols of interdisciplinary teams is not clear due to a non-standardized and wide variation of patient outcomes thus making the comparison of outcome measures available in the literature difficult. It is only by agreeing on a common, standard set of outcome measures for the comprehensive appraisal of OI that comparisons across interdisciplinary treatment centers for OI will be possible in the future. METHODS: The Key4OI international interdisciplinary working group of 27 members used a consensus-driven modified Delphi approach to develop a set of global outcome measures for patients with OI. The International Classification of Functioning, Disability and Health (ICF), was used to define domains and organize the outcomes from the literature search. After reviewing the outcomes extracted from the literature, trials and registries, the working group agreed on a final selection of domains and their definition (ICF definition as well as a lay description). These domains were then presented to the focus groups who prioritized the outcome domains by taking into account the items important to the OI community. All content was collected and analyzed and final domains were determined. A consensus of appropriate measuring instruments for each domain was reached with Delphi rounds. The entire approach was in line with the International Consortium for Health Outcomes Measurement ICHOM methodology. RESULTS: More than 400 different outcome measures were identified in our literature search. After three Delphi rounds, 24 domains were selected. After the focus group sessions, the number of domains were reduced to 15. A consensus was reached on the measuring instruments to cover these domains for both children and adults. CONCLUSION: The Key4OI project resulted in standard set of outcome measures focused on the needs and wishes of individuals with OI and their families. This outcome set will enable healthcare teams and systems to compare and to improve their care pathways and quality of care worldwide. Further studies are needed to evaluate the implementation of this standardized outcome set.
BACKGROUND:Osteogenesis Imperfecta (OI) is a genetic disorder also known as 'brittle bone disease'. The clinical manifestation of OI shows a wide variation. Therefore, care for patients with OI requires an interdisciplinary approach. The effectiveness of particular interventions and treatment protocols of interdisciplinary teams is not clear due to a non-standardized and wide variation of patient outcomes thus making the comparison of outcome measures available in the literature difficult. It is only by agreeing on a common, standard set of outcome measures for the comprehensive appraisal of OI that comparisons across interdisciplinary treatment centers for OI will be possible in the future. METHODS: The Key4OI international interdisciplinary working group of 27 members used a consensus-driven modified Delphi approach to develop a set of global outcome measures for patients with OI. The International Classification of Functioning, Disability and Health (ICF), was used to define domains and organize the outcomes from the literature search. After reviewing the outcomes extracted from the literature, trials and registries, the working group agreed on a final selection of domains and their definition (ICF definition as well as a lay description). These domains were then presented to the focus groups who prioritized the outcome domains by taking into account the items important to the OI community. All content was collected and analyzed and final domains were determined. A consensus of appropriate measuring instruments for each domain was reached with Delphi rounds. The entire approach was in line with the International Consortium for Health Outcomes Measurement ICHOM methodology. RESULTS: More than 400 different outcome measures were identified in our literature search. After three Delphi rounds, 24 domains were selected. After the focus group sessions, the number of domains were reduced to 15. A consensus was reached on the measuring instruments to cover these domains for both children and adults. CONCLUSION: The Key4OI project resulted in standard set of outcome measures focused on the needs and wishes of individuals with OI and their families. This outcome set will enable healthcare teams and systems to compare and to improve their care pathways and quality of care worldwide. Further studies are needed to evaluate the implementation of this standardized outcome set.
Entities:
Keywords:
Brittle bone disease; Clinical outcome measures; Continuous quality improvement; Learning health care; Osteogenesis imperfecta; Outcomes; Patient-reported outcomes measures; Value-based health care
Authors: Maarten Boers; John R Kirwan; George Wells; Dorcas Beaton; Laure Gossec; Maria-Antonietta d'Agostino; Philip G Conaghan; Clifton O Bingham; Peter Brooks; Robert Landewé; Lyn March; Lee S Simon; Jasvinder A Singh; Vibeke Strand; Peter Tugwell Journal: J Clin Epidemiol Date: 2014-02-28 Impact factor: 6.437