U.S. flag

An official website of the United States government

NCBI Bookshelf. A service of the National Library of Medicine, National Institutes of Health.

Clinical Review Report: Cysteamine delayed-release capsules (Procysbi): Horizon Pharma Ireland Ltd. Indication: For the treatment of nephropathic cystinosis [Internet] Ottawa (ON): Canadian Agency for Drugs and Technologies in Health; 2018 Feb.

Cover of Clinical Review Report: Cysteamine delayed-release capsules (Procysbi)

Clinical Review Report: Cysteamine delayed-release capsules (Procysbi): Horizon Pharma Ireland Ltd. Indication: For the treatment of nephropathic cystinosis [Internet]

Show details

Abbreviations

AE

adverse event

BMI

body mass index

BSA

body surface area

CDR

CADTH Common Drug Review

CI

confidence interval

CSR

Clinical Study Report

CTNS

cystinosin, lysosomal cystine transporter

eGFR

estimated glomerular filtration rate

ESRD

end-stage renal disease

HRQoL

health-related quality of life

ITT

intent-to-treat

LSM

least squares mean

MCID

minimal clinically important difference

PedsQL 4.0

Pediatric Quality of Life Inventory version 4.0

PPI

proton pump inhibitor

RCT

randomized controlled trial

SAE

serious adverse event

SD

standard deviation

SE

standard error

SF-36

Short Form 36

ULN

upper limit of normal

VAS

visual analogue scale

WBC

white blood cell

WDAE

withdrawal due to adverse event

Copyright © 2018 Canadian Agency for Drugs and Technologies in Health.

The copyright and other intellectual property rights in this document are owned by CADTH and its licensors. These rights are protected by the Canadian Copyright Act and other national and international laws and agreements. Users are permitted to make copies of this document for non-commercial purposes only, provided it is not modified when reproduced and appropriate credit is given to CADTH and its licensors.

Except where otherwise noted, this work is distributed under the terms of a Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International licence (CC BY-NC-ND), a copy of which is available at http://creativecommons.org/licenses/by-nc-nd/4.0/

Bookshelf ID: NBK534004

Views

  • PubReader
  • Print View
  • Cite this Page
  • PDF version of this title (1.6M)

Other titles in this collection

Recent Activity

Your browsing activity is empty.

Activity recording is turned off.

Turn recording back on

See more...