Rare Disease Day - Combatting severe hunger and over-eating for children with Prader Willi Syndrome
Our Children’s Hospital currently looks after a cohort of 40 children with Prader-Willi Syndrome (PWS), a rare condition affecting one out of every 15,000 births – our clinician recently took part in an international multi-centre study to reduce the appetite in affected children to lower levels of obesity in children with PWS.
PWS is a complex genetic disorder affecting the metabolism and neuro-behaviour, meaning that children often experience food obsession, ‘aggressive food seeking’ and lack of satiation by the age of nine, later progressing to severe obesity if intake is not restricted. To date, there has been no effective treatment to support children with severe hunger as a result of PWS. The results have recently been published in the International Journal and the trial has now been completed and undertaken at the NIHR Wellcome Clinical Research Facility.
Professor Tim Barrett, our Honorary Consultant in Paediatric Endocrinology and Diabetes said:
“Prader Willi Syndrome makes it incredibly challenging for children to moderate their levels of hunger amongst many other symptoms that the condition presents. Being able to support our patients and monitor their body composition in response to the medication is important for us to be able to treat hyperphagia effectively. As a Rare Disease Centre for children, the DESTINY PWS study helps us to support children with severe hunger and over-eating which is one of the most difficult areas to manage for caregivers.”
Our Endocrinology team have been involved in the DESTINY PWS study, a randomised test to monitor the efficacy of the Diazoxide Choline Extended-Release Tablet (DCCR), designed to reduce hunger and over-eating, more commonly known in PWS patients as ‘Hyperphagia’. From a 2014 survey, caregivers of PWS patients noted that food-related behaviours were the most important unmet needs and required greater support in future therapeutic interventions. In response to this, the DESTINY PWS has tested the use of DCCR in children aged four and above to suppress hunger through a double-blind, randomised placebo-controlled trial to measure its ability to support our patients with PWS over a 13-week-period.
DCCR was found to be beneficial to our patients with severe baseline hyperphagia, allowing us a new treatment option for children most severely affected. We’re happy to be able to contribute to the trial to support our young patients, to improve quality of life and reduce rates of obesity into adulthood. This allows our severe patients the ability to moderate their food obsession and hunger to lead healthier lives.
Professor Barrett said: “It’s excellent to be able to take part in studies like DESTINY PWS to identify future therapeutic interventions to support our kids. If we can help in any way to make this life-long incurable disease easier to manage, we’re keen to play our part and continue looking towards further treatment to reduce hyperphagia in our patients.”