A non-interventional observational study to identify and validate clinical outcome assessments for adults with phenylketonuria for use in clinical trials

Burton, Barbara; Skalicky, Anne; Baerwald, Christoph; Bilder, Deborah; Harding, Cary; Ilan, Aaron; Jurecki, Elaina; Longo, Nicola; Madden, David; SİVRİ, SERAP; Wilcox, Gisela; Thomas, Janet; Delaney, Kathleen

doi:10.1016/j.ymgmr.2021.100810

A non-interventional observational study to identify and validate clinical outcome assessments for adults with phenylketonuria for use in clinical trials

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Burton B. K., Skalicky A., Baerwald C., Bilder D. A., Harding C. O., Ilan A. B., ...More

MOLECULAR GENETICS AND METABOLISM REPORTS, vol.29, 2021 (SCI-Expanded)

Publication Type: Article / Article
Volume: 29
Publication Date: 2021
Doi Number: 10.1016/j.ymgmr.2021.100810
Journal Name: MOLECULAR GENETICS AND METABOLISM REPORTS
Journal Indexes: Science Citation Index Expanded (SCI-EXPANDED), Scopus, EMBASE, Directory of Open Access Journals
Keywords: Phenylketonuria, PKU, Clinical outcome assessments, Adults, Patient-reported outcomes, Observer-reported outcomes, Clinician-reported outcomes, QUALITY-OF-LIFE, PRO INSTRUMENTS, PKU, DISORDERS, ADHERENCE, SYMPTOMS, ISSUES
Hacettepe University Affiliated: Yes

Abstract

Introduction: Current clinical outcome assessments (COAs) are not effectively capturing the complex array of symptoms of adults with phenylketonuria (PKU). This study aimed to identify concepts of interest relevant to adults with PKU. Based on these concepts, COAs for patient-reported outcomes (PROs), observer-reported out-comes (ObsROs), and clinician-reported outcomes (ClinROs) were selected or developed and content validity was assessed.