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dc.contributor.authorSalek, Sam
dc.date.accessioned2020-02-13T01:26:12Z
dc.date.available2020-02-13T01:26:12Z
dc.date.issued2019-12-27
dc.identifier.citationSalek , S 2019 , ' Development of the Pyruvate Kinase Deficiency Diary (PKDD) and Pyruvate Kinase Deficiency Impact Assessment (PKDIA): Disease-Specific Assessments ' , European Journal of Haematology . https://doi.org/10.1111/ejh.13376
dc.identifier.issn1600-0609
dc.identifier.otherPURE: 19411315
dc.identifier.otherPURE UUID: 3881b88f-5943-4b87-b8e7-482449968161
dc.identifier.otherScopus: 85083904992
dc.identifier.urihttp://hdl.handle.net/2299/22210
dc.description© 2019 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd. This article has been accepted for publication and undergone full peer review but has not been through the copyediting, typesetting, pagination and proofreading process, which may lead to differences between this version and the Version of Record.
dc.description.abstractINTRODUCTION: Currently recommended patient-reported outcome (PRO) measures for patients with pyruvate kinase (PK) deficiency are non-disease-specific. The PK Deficiency Diary (PKDD) and PK Deficiency Impact Assessment (PKDIA) were developed to be more targeted measures for capturing the symptoms and impacts of interest to this patient population. METHODS: The instruments were developed based on concept elicitation interviews with 21 adults and modified based on 20 cognitive interviews. The domain structure and item concepts of the PKDD and PKDIA were compared to currently recommended measures, the EORTC QLQ-C30 and the SF-36v2®. RESULTS: The PKDD is a 7-item measure of the core signs and symptoms of PK deficiency. The PKDIA is a 14-item measure of the impacts of PK deficiency on patients’ health-related quality of life (HRQoL). Minimal similarities were found between the new measures and the EORTC QLQ-C30 (e.g. 43% of concepts were similar to the PKDD; 42% were similar to the PKDIA) and SF-36v2® (57% of concepts were similar to the PKDD; 17% were similar to the PKDIA). CONCLUSIONS: The PKDD and PKDIA fill a gap in the existing outcomes measurement strategy for PK deficiency. Future work includes psychometric evaluation of these newly developed measures.en
dc.format.extent20
dc.language.isoeng
dc.relation.ispartofEuropean Journal of Haematology
dc.subjectpyruvate kinase deficiency, anemia, hemolytic anemia, patient-reported outcome
dc.titleDevelopment of the Pyruvate Kinase Deficiency Diary (PKDD) and Pyruvate Kinase Deficiency Impact Assessment (PKDIA): Disease-Specific Assessmentsen
dc.contributor.institutionDepartment of Clinical and Pharmaceutical Sciences
dc.contributor.institutionCentre for Health Services and Clinical Research
dc.contributor.institutionPublic Health and Patient Safety Unit
dc.contributor.institutionSchool of Life and Medical Sciences
dc.description.statusPeer reviewed
rioxxterms.versionAM
rioxxterms.versionofrecordhttps://doi.org/10.1111/ejh.13376
rioxxterms.typeJournal Article/Review
herts.preservation.rarelyaccessedtrue


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