Use of the patient-reported outcomes measurement information system (PROMIS®) to assess late-onset Pompe disease severity.

ABSTRACT:

Background

Patient-Reported Outcomes provide an opportunity for patients to establish dialogue with pharmaceutical or biotechnology companies about their health conditions without interpretation by a clinician or anyone else. However, Patient-Reported Outcomes that can be widely applicable for use in patient-focused drug development or clinical trial designs are not yet validated for all diseases. The aim of this study report was to provide supportive evidence of the construct and content validity of selected Patient-Reported Outcomes Measurement Information System (PROMIS®) questionnaires compared with other disease-relevant clinical outcome measures, including the 6-Minute Walk Distance, forced vital capacity, and Manual Muscle Test, in late-onset Pompe disease and to provide sup

SUBMITTER: Harfouche M 

PROVIDER: S-EPMC7547055 | biostudies-literature | 2020 Oct

REPOSITORIES: biostudies-literature