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Papers of the Week


2022 Sep 06


Adv Ther

Validation of the PRUCISION Instruments in Pediatric Patients with Progressive Familial Intrahepatic Cholestasis.

Authors

Gwaltney C, Ivanescu C, Karlsson L, Warholic N, Kjems L, Horn P
Adv Ther. 2022 Sep 06.
PMID: 36066745.

Abstract

Patients with cholestatic liver disease, including progressive familial intrahepatic cholestasis (PFIC) and Alagille syndrome, may have debilitating pruritus, and reducing pruritus is a key therapeutic goal. However, few instruments are available that adequately measure pruritus in pediatric patients with cholestatic liver disease. The objectives of the current study were to establish the measurement properties of the novel PRUCISION patient-reported outcome (PRO) and observer-reported outcome (ObsRO) instruments and to estimate a threshold for clinically meaningful change in pruritus score.