The National Study of Nephrotic Syndrome, NephroS Study
Nephrotic syndrome may be caused by primary (idiopathic) renal disease or by a variety of secondary causes. Idiopathic Nephrotic Syndrome is normally treated with steroids. In rare cases the disease is resistant to this treatment and called Steroid Resistant Nephrotic Syndrome (SRNS). There are currently around 270 children in the UK with SRNS and 10% of these are on the end stage renal failure programme. Approximately 12 children and an unknown number of adults will undergo a transplant each year in the UK. As many as half of these will have a recurrence post-transplant and up to 100% will have a recurrence after their second or third transplant. This is a heavy burden on patients and health and social care services. It has been difficult to further understanding of this rare disease due to the small number of patients.
Funded by National Institute of Health Research, Kidney Research UK and pharmaceutical companies this national observational study aims to learn more about SRNS, develop tests that can predict a recurrence of the disease post-transplant and identify common symptoms that can be taken forward to a new study to test new pharmaceutical drugs. You can read the full research protocol here http://rarerenal.org/wp-content/uploads/2015/03/NephroS-Protocol-v7-23-11-2016.pdf
The development of the Rare Kidney Disease Registry (RaDaR) is essential to this study as it will allow for identification of a large cohort of children and adults with the disease for the first time.
The study is open and currently recruiting. Progress and information is available via the website here http://rarerenal.org/clinician-information/nephrotic-syndrome-clinician-information/nephros-study/ and newsletters with updates are published regularly http://rarerenal.org/wp-content/uploads/2017/06/NephroS-Newsletter-June-2017.pdf . The study will continue to recruit until they have at least 800 participants, and as this is a rare disease study there is no time limit set for recruitment. We need data on all cases fitting the inclusion/exclusion criteria, and a key result will be the number of patients we can learn from in one data source.
This is the first UK database of Steroid Resistant Nephrotic Syndrome (SRNS) and it will be continually updated. The database will improve understanding of the disease and thus improve patient outcomes.
People living with rare disease conditions can often feel isolated. This research has brought people and families living with the disease together and connected with charity and support organisations. An example is the National Nephrotic Syndrome Day which brings clinicians and people affected by the condition together to share. People affected by the condition feel less isolated and develop wider support structures and networks. Clinical teams and policy makers can also listen to patient experiences and improve their understanding of the needs of people affected by this rare disease. Service provision will be more focussed and directed to key needs, the impact of this is reduced NHS and third sector costs https://nstrust.co.uk/news/2018/03/07/national-nephrotic-syndrome-day-2018