SONG-PKD
Research type
Research Study
Full title
Standardised Outcomes in Nephrology – Polycystic Kidney Disease (SONG-PKD): study protocol for establishing a core outcome set in polycystic kidney disease
IRAS ID
251410
Contact name
Albert Ong
Contact email
Sponsor organisation
The University of Sydney
Duration of Study in the UK
0 years, 11 months, 31 days
Research summary
Research Summary:
Autosomal dominant polycystic kidney disease (ADPKD) is the most common inherited kidney disorder and is responsible for 5-10 percent of end-stage kidney disease (ESKD) requiring renal replacement therapy. ADPKD is life-threatening, irreversible, and affects an estimated 12.5 million people worldwide occurring equally in men and women, without ethnic disparities. Gene defects which lead to development of ADPKD cause disruption in renal tubular epithelial differentiation leading to the formation of multiple cysts, and their expansion due to ongoing cellular proliferation and fluid secretion. The cysts progressively grow, enlarging kidneys up to 20 times bigger than normal size, displacing and destroying normal kidney tissue culminating in fibrosis and ultimately kidney failure. Up to 70% of patients with ADPKD progress to ESKD by age of 65 years, and annual cost of RRT provision for ADPKD patients in Europe alone approximates to 1.5 billion Euros. Patients with ADPKD are also at high-risk of complications including debilitating kidney pain, hepatic cysts, infection, intracranial aneurysms and cardiovascular disease that may severely impair quality of life and well-being.The increasing recognition of inefficiency and waste in medical research, attributed to problems with outcome selection, has given rise to numerous initiatives across medical specialties worldwide to develop core outcome sets – defined as an agreed minimum set of standardized outcomes that should be measured and reported in all trials on a specific clinical topic. This does not preclude trialists adding other outcomes specific to the trial population and intervention.
In Nephrology, the Standardized Outcomes in Nephrology (SONG, www.songinitiative.org) initiative was formed in 2015 to establish core outcomes across the full spectrum of CKD. The SONG-Polycystic Kidney Disease (SONG-PKD) project aims to establish a core outcome set for trials and other types of clinical research involving patients with ADPKD.
Summary of Results:
Kidney function, mortality, cardiovascular disease, and pain were established as the core outcomes for ADPKD.REC name
HSC REC A
REC reference
18/NI/0149
Date of REC Opinion
23 Aug 2018
REC opinion
Further Information Favourable Opinion