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Article: A standard set of outcome measures for the comprehensive assessment of osteogenesis imperfecta

TitleA standard set of outcome measures for the comprehensive assessment of osteogenesis imperfecta
Authors
KeywordsOsteogenesis imperfecta
Brittle bone disease
Continuous quality improvement
Learning health care
Outcomes
Patient-reported outcomes measures
Value-based health care
Clinical outcome measures
Issue Date2021
PublisherBioMed Central Ltd. The Journal's web site is located at http://www.ojrd.com
Citation
Orphanet Journal of Rare Diseases, 2021, v. 16, article no. 140 How to Cite?
AbstractBackground: 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.
Persistent Identifierhttp://hdl.handle.net/10722/307911
ISSN
2023 Impact Factor: 3.4
2023 SCImago Journal Rankings: 1.182
PubMed Central ID
ISI Accession Number ID

 

DC FieldValueLanguage
dc.contributor.authorNijhuis, W-
dc.contributor.authorFranken, A-
dc.contributor.authorAyers, K-
dc.contributor.authorDamas, C-
dc.contributor.authorFolkestad, L-
dc.contributor.authorForlino, A-
dc.contributor.authorFraschini, P-
dc.contributor.authorHill, C-
dc.contributor.authorJanus, G-
dc.contributor.authorKruse, R-
dc.contributor.authorWekre, LL-
dc.contributor.authorMichiels, L-
dc.contributor.authorMontpetit, K-
dc.contributor.authorPanzeri, L-
dc.contributor.authorPorquet‑Bordes, V-
dc.contributor.authorRauch, F-
dc.contributor.authorSakkers, R-
dc.contributor.authorSalles, J-
dc.contributor.authorSemler, O-
dc.contributor.authorSun, J-
dc.contributor.authorTo, M-
dc.contributor.authorTosi, L-
dc.contributor.authorYao, Y-
dc.contributor.authorYeung, EHK-
dc.contributor.authorZhytnik, L-
dc.contributor.authorZillikens, MC-
dc.contributor.authorVerhoef, M-
dc.date.accessioned2021-11-12T13:39:42Z-
dc.date.available2021-11-12T13:39:42Z-
dc.date.issued2021-
dc.identifier.citationOrphanet Journal of Rare Diseases, 2021, v. 16, article no. 140-
dc.identifier.issn1750-1172-
dc.identifier.urihttp://hdl.handle.net/10722/307911-
dc.description.abstractBackground: 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.-
dc.languageeng-
dc.publisherBioMed Central Ltd. The Journal's web site is located at http://www.ojrd.com-
dc.relation.ispartofOrphanet Journal of Rare Diseases-
dc.rightsThis work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License.-
dc.subjectOsteogenesis imperfecta-
dc.subjectBrittle bone disease-
dc.subjectContinuous quality improvement-
dc.subjectLearning health care-
dc.subjectOutcomes-
dc.subjectPatient-reported outcomes measures-
dc.subjectValue-based health care-
dc.subjectClinical outcome measures-
dc.titleA standard set of outcome measures for the comprehensive assessment of osteogenesis imperfecta-
dc.typeArticle-
dc.identifier.emailTo, M: mikektto@hku.hk-
dc.identifier.authorityTo, M=rp00302-
dc.description.naturepublished_or_final_version-
dc.identifier.doi10.1186/s13023-021-01682-y-
dc.identifier.pmid33743784-
dc.identifier.pmcidPMC7980586-
dc.identifier.scopuseid_2-s2.0-85103174185-
dc.identifier.hkuros329776-
dc.identifier.volume16-
dc.identifier.spagearticle no. 140-
dc.identifier.epagearticle no. 140-
dc.identifier.isiWOS:000631539300003-
dc.publisher.placeUnited Kingdom-

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