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dc.contributor.authorSalek, Mir-Saeed Shayegan
dc.contributor.authorIonova, Tatyana
dc.contributor.authorJohns, Jeffrey
dc.contributor.authorOliva, Esther Natalie
dc.date.accessioned2019-01-08T16:00:01Z
dc.date.available2019-01-08T16:00:01Z
dc.date.issued2018-11-19
dc.identifier.citationSalek , M-S S , Ionova , T , Johns , J & Oliva , E N 2018 , ' Appraisal of patient-reported outcome measures in analogous diseases and recommendations for use in phase II and III clinical trials of pyruvate kinase deficiency ' , Quality of Life Research . https://doi.org/10.1007/s11136-018-2025-y
dc.identifier.issn0962-9343
dc.identifier.otherPURE: 15436160
dc.identifier.otherPURE UUID: 022d8e97-009d-47cf-a3a4-eab719472fe8
dc.identifier.otherScopus: 85056908708
dc.identifier.urihttp://hdl.handle.net/2299/20928
dc.description.abstractPurpose: Pyruvate kinase deficiency (PKD) is a rare disease and understanding of its epidemiology and associated burden remains limited. With no current curative therapy, clinical manifestations can be life threatening, clinically managed by maintaining adequate hemoglobin levels through transfusion and subsequent support, but with frequent complications. Treatment goals are to maintain/improve the patient’s quality of life. With new therapies, reliable, valid, and relevant patient-reported outcome (PRO) tools are required for use in clinical trials. Methods: Systematic literature search identified no current PRO tools for capturing/measuring the impact of PKD and treatments in clinical trials. Therefore, the search strategy was revised to consider conditions analogous to PKD in terms of symptoms and impacts that might serve as parallels to the experience in PKD; this included sickle cell anemia, thalassemia, and hemolytic anemia. Psychometric properties, strengths, and weakness of selected appropriate PRO instruments were compared, and recommendations made for choice of PRO tools. Results: In adult populations, EORTC QLQ C30 and SF-36v2 are recommended, the former being a basic minimum, covering generic HRQoL, and core symptoms such as fatigue. In pediatric populations, PedsQL Generic Core Scale to measure HRQoL and PedsQL MFS scale to measure fatigue are recommended. Conclusions: Some symptoms/life impacts may be unique to PKD and not observable in analogous conditions. A ‘Physico-Psychosocial Model’ derived from the ‘Medical Model’ is proposed to form the basis for a hypothesized conceptual framework to address the development of PKD-specific PRO instruments.en
dc.language.isoeng
dc.relation.ispartofQuality of Life Research
dc.rights/dk/atira/pure/core/openaccesspermission/open
dc.subjectPRO
dc.subjectPatient-reported outcome
dc.subjectPyruvate kinase deficiency
dc.subjectQuality of life
dc.subjectPublic Health, Environmental and Occupational Health
dc.titleAppraisal of patient-reported outcome measures in analogous diseases and recommendations for use in phase II and III clinical trials of pyruvate kinase deficiencyen
dc.contributor.institutionDepartment of Pharmacy, Pharmacology and Postgraduate Medicine
dc.contributor.institutionPublic Health and Patient Safety Unit
dc.contributor.institutionCentre for Health Services and Clinical Research
dc.contributor.institutionSchool of Life and Medical Sciences
dc.contributor.institutionDepartment of Clinical and Pharmaceutical Sciences
dc.description.statusPeer reviewed
dc.identifier.urlhttp://www.scopus.com/inward/record.url?scp=85056908708&partnerID=8YFLogxK
dc.relation.schoolSchool of Life and Medical Sciences
dc.description.versiontypeFinal Published version
dcterms.dateAccepted2018-10-09
rioxxterms.versionVoR
rioxxterms.versionofrecordhttps://doi.org/10.1007/s11136-018-2025-y
rioxxterms.licenseref.urihttp://creativecommons.org/licenses/by/4.0/
rioxxterms.typeJournal Article/Review
herts.preservation.rarelyaccessedtrue
herts.rights.accesstypeopenAccess


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