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

Atıf İçin Kopyala

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

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

Yayın Türü: Makale / Tam Makale
Cilt numarası: 29
Basım Tarihi: 2021
Doi Numarası: 10.1016/j.ymgmr.2021.100810
Dergi Adı: MOLECULAR GENETICS AND METABOLISM REPORTS
Derginin Tarandığı İndeksler: Science Citation Index Expanded (SCI-EXPANDED), Scopus, EMBASE, Directory of Open Access Journals
Anahtar Kelimeler: 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 Üniversitesi Adresli: Evet

Özet

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.