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


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) identifier identifier identifier

  • 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.