From Eureka Alert, Release from WESTMEAD INSTITUTE FOR MEDICAL RESEARCH, AUSTRALIA
DR GOPI RANGAN AND THE TEAM FROM THE WESTMEAD INSTITUTE FOR MEDICAL RESEARCH AND THE WESTMEAD HOSPITAL
A cheap, safe and effective treatment to polycystic kidney disease may soon be available, thanks to a new national clinical out of Westmead, Australia, which is trialing water to treat the disease.
The trial, known as PREVENT-ADPKD, will investigate whether drinking the right amount of can prevent adult polycystic kidney disease (ADPKD) progressing to kidney failure.
ADPKD is an inherited disease in which the kidneys deteriorate because of cysts that grow and destroy healthy tissue.
The study team - led by Dr Annette Wong, Carly Mannix, Professor David Harris and Dr Gopi Rangan - at the Westmead Hospital and the Westmead Institute for Medical Research are optimistic that drinking water is an effective treatment to reduce kidney cyst growth in ADPKD patients.
"Water stops the hormone that makes these cysts grow, so ensuring you aren't thirsty reduces the chance of cysts growing,"Dr Rangan said."This is important, because there is no cure for polycystic kidney disease. More than 50 per cent of patients eventually develop kidney failure, requiring dialysis or kidney transplantation.
"If successful, our approach could have major benefits for the health of people with ADPKD. If we can slow the disease at an early stage, we could potentially prevent kidney failure occurring altogether.
"A positive study result will show that water is a cheap, safe and effective treatment," he explained.
The team's previous research showed that increased water intake was highly effective at reducing cyst growth in animals with polycystic kidney disease.
This new study protocol, published in BMJ Open, will be the first of its kind to provide evidence on whether prescribed water intake is an effective treatment for ADPKD in humans. The three-year randomised controlled clinical trial will use MRI to assess the rate of cyst growth in the kidney throughout the study.
Patients with ADPKD develop hundreds of cysts in the kidney, which form in early childhood and grow by five to ten per cent each year. It is the most common genetic kidney disease in adults, affecting one in every 2500 individuals. More than 2000 Australians with ADPKD currently receive dialysis or need a kidney transplant.
More than 240 people have already enrolled in the trial, and full results will be available in 2020.
Recruitment for the study closes on 28 February 2018. Participants interested in participating in the trial should contact the study team by email: preventadpkd@sydney.edu.au
The study protocol is available online at BMJ Open: http://bmjopen.bmj.com/content/8/1/e018794.full?ijkey=mxfWKbi5X6my8li&keytype=ref
From International Journal of Nephrology and Renovascular Disease
1Department of Nephrology, Hospital del Mar, Institut Mar for Medical Research, Barcelona, Spain; 2Faculty of Medicine, Université Paris-Descartes, Assistance Publique-Hôpitaux de Paris, Service de Néphrologie, Hôpital Necker-Enfants Malades, Paris, France
Abstract: Standard of care therapies for autosomal dominant polycystic kidney disease (ADPKD) may limit morbidity and mortality due to disease-related complications, but they do not delay disease progression. Tolvaptan, a selective vasopressin V2 receptor antagonist, delays the increase in kidney volume (a surrogate marker for disease progression), slows the decline in renal function, and reduces pain in ADPKD patients with relatively preserved renal function. The most common adverse events of tolvaptan are linked to its aquaretic effect, and rare cases of idiosyncratic hepatitis were observed. Additional ongoing studies will determine whether the benefits are sustained over time, whether they can be observed in patients with advanced kidney disease, and whether they can be translated in terms of quality of life and cost/effectiveness parameters. Tolvaptan is currently approved in Europe and several countries throughout the world. In real-life conditions, selection of patients that would be good theoretical candidates to tolvaptan is a key but complex question. Eligibility criteria slightly differ from one country to another, and several models (based on conventional data, genetics, renal volume) were recently proposed to identify patients with evidence or risk of rapid disease progression. Eligible patients will ultimately make the decision to start tolvaptan, after complete information, consideration, and balancing of benefits, adverse events, and risks.
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