The BAPN is committed to both high quality basic science and translational research, and multicentre clinical trials across all areas of quantitative and qualitative research. The structure of BAPN research is now geared to achieve the latter aspect with maximum efficiency in the current NHS funding environment. The responsibility of the Research Secretary (currently Drs Richard Coward and Sally Hulton) is to co-ordinate the UK paediatric nephrology centres in moving clinical trials from ideas to protocol development to funding and implementation,involving as many centres as possible. The structure has recently become embedded within the UK Clinical Research Networks (UKCRN), the research arm of the NHS. One of the six research priority topics for UKCRN is the Medicines for Children Research Network (MCRN). This is a funded organisation, with dedicated research infrastructure across England, including research nurses, statisticians, pharmacists etc. MCRN has established Clinical Study Groups (CSG) in paediatric subspecialties, and nephrology now has a CSG, with a dedicated Chair (position jointly held by Richard Coward and Sally Hulton), and members from almost all UK nephrology centres. This is an exciting time for clinical research, and means that nephrology is now in a strong position to take forward identified clinical trials,with support for funding applications from the CSG.
A £12-million Charity Open Access Fund project has been set up by the Wellcome Trust, Cancer Research UK, the Breast Cancer Campaign, Arthritis Research UK, Leukaemia and Lymphoma Research, and the British Heart Foundation. Researchers with funding from the charities will be eligible to apply to the fund to cover costs incurred when publishing papers with immediate, unrestricted open access. Further information is available online.
Charity to invest record £5.1m into renal research
In 2014/15, Kidney Research UK will invest a record £5.1m into renal research. The charity announced the news during their recent AGM in Peterborough.
The extra investment is due to a successful year of fundraising in 2013/14, made possible by the generosity of the public and their donations. This success meant that Trustees of the charity were able to release extra funds into the research budget.
Kidney Research UK is the largest funder dedicated to kidney research and kidney problems in the UK, with the aim of finding better treatments and ultimately cures for kidney diseases. The charity receives very little government funding, and relies almost wholly on donations from the public.
To cement Kidney Research UK’s commitment to helping develop scientists for the future, the charity has funded 14 Intercalated Degree Awards in 2014/15 at a total cost of £70,000 – an increase on the previous three years. Intercalated Degree Awards are for medical students, and aim to encourage and engage undergraduates with an interest in renal medicine at this early stage in their career development.
Elaine Davies, Director of Research at Kidney Research UK, said:
“The cost of funding research into kidney disease can vary from £5,000 to £250,000. Any of these research investments could deliver a breakthrough and will increase our understanding of kidney disease and its causes, allowing us to work towards developing treatments for the disease.
“This is a brilliant result and enables us to continue building the UK’s renal research capacity. We want to thank our supporters for their help in increasing our investment, but more support is vital to enable us to continue our work.”
To find out more about the different types of funding awarded by Kidney Research UK, click here.
A full copy of our annual report is available here.
Big congratulations to Emma Cope who has been awarded the Just Giving Young Fundraiser of the Year Award for 2014. She has been fund raising for the NEST charity which supports research into nephrotic syndrome. The young fundraiser award starts at 11 min 30s or you can watch on the Livestream website.
New studies seeking recruiting centres
RaDaR: UK Registry for Rare Kidney Diseases
The purpose of the registry is to assemble cohorts of patients whose kidney disease is thoroughly investigated and characterised. Patients in the registry can then be approached to participate in “translational” research. Translational research aims to connect the basic scientific investigation of a disease to tangible benefit for the patient. Benefits might include better diagnosis, patient information, treatment trials, and ultimately better care.
Patient involvement in the registry is strongly encouraged.
RADAR is an initiative of the Renal Association and the British Association for Paediatric Nephrology. It operates within the UK Renal Registry, and its governance lies with the Renal Association. The initiative is supported by the Medical Research Council and Kidney Research UK.
A familial study of childhood renal artery stenosis
We are conducting a familial study of childhood renal artery stenosis at GOSH and would be interested if BAPN research centres could contact Steve Marks at email@example.com if they have patients with renal artery stenosis that they would consider for inclusion into the study. We have started up a DNA bank from over 10 patients seen at GOSH as well as their family members (parents and siblings) and wish to extend the study to all UK centres where RAS is treated. Our aims are to collect DNA from those families and in the future, carry out a genome search to test candidate genes affecting vascular growth in a large population. Please find below an abstract outlining information on the study: Background: Childhood renal artery stenosis (RAS) leading to renovascular hypertension (RVH) is usually associated with fibromuscular dysplasia (FMD). RAS/FMD is most likely a primary disorder of arterial maturation, and two previous studies reported that it can be inherited as a dominant, non-syndromic trait. Aims: To determine the incidence of hypertension in first-degree relatives of a cohort of non-syndromic index cases with RVH as a first step to answering whether RAS/FMD commonly runs in families. Methods: Recruitment of individuals diagnosed with childhood RVH and their first-degree relatives. Exclusion criteria of index cases with defined syndromes (neurofibromatosis, Williams and Alagille syndrome). Family histories were obtained and visits arranged for blood pressure measurement (BPM) and, when indicated, 24-hour ambulatory blood pressure monitoring (ABPM). Results: Ten unrelated index cases (60% male) aged 7-32 (median 14) years were recruited, with 27 first-degree relatives comprising 16 (63% mothers) parents aged 28-58 (median 44) years and 11 (64% sisters) siblings aged 5-30 (median 19) years, including one consanguineous family. Hypertension was evident in 25% of parents from different families (three were on anti-hypertensive medications, with one mother discovered to be hypertensive by BPM and ABPM. All six adult siblings were normotensive. Of the five siblings aged <18 years, one female teenager was pre-hypertensive (90-95th centile BP for age, sex and height). The remaining siblings <18 years were normotensive (two with BP<50th and two with BP 50th-90th centiles). Conclusion: Given that the incidence of hypertension in the general adult population is around 25%, the incidence of hypertension in parents of our index cases was not increased. Although none of the hypertensive parents have had RAS formally excluded by renal angiography, this finding, together with the lack of overt hypertension in siblings, can be interpreted as evidence that familial RAS/FMD leading to hypertension is a rare occurrence.
Proposed research projects:
- Does addition of an ARB maintain GFR in children with CKD on ACEi? link to protocol
Familial Hypomagnesemia with hypercalciuria and nephrocalcinosis (FHHNC)
Dear BAPN members, In our laboratory we started an international scientific collaboration aimed at identifying the spectrum of mutations in paracellin-1 gene and other candidate genes that may be involved in the pathogenesis of familial hypomagnesemia with hypercalciuria and nephrocalcinosis (FHHNC). The study would imply the investigation of clinically well-characterized FHHNC isolated cases and pedigrees from different populations and countries: this will allow the inference of the genetic epidemiology of the disorder and the study of the genotype-phenotype relationship. We are trying to expand the number of FHHNC cases enrolled in this study and seek help from the national communities of pediatric nephrologists. All what we need is a blood sample from which DNA could be isolated. Physicians providing cases, blood (or isolated DNA), and basic clinical details will appear as co-authors in all the publications arising from the outcome of the study. All the costs of laboratory investigations and manuscript(s)preparation and submission will be covered by our University. Blood collection shipping costs will be covered by the participating physicians. Many thanks for your kind attention to this proposal of collaboration. Please, let me know if you need further details on the project and the collaboration. Best regards. Roberto Colombo ========================== Roberto Colombo, PhD Professor of Clinical Genetics Head Laboratory of Human Molecular Biology and Genetics Catholic University of the Sacred Heart P.za Buonarroti 30 20145 Milan (Italy) Phone: +39 02 460055 Fax: + 39 02 468629 E-mail: firstname.lastname@example.org
PREDnisolone in NephrOtic Syndrome (PREDNOS)
Link to PREDNOS Website.
PREDNOS is a national multicentre randomised double blind trial of long-term tapering versus standard prednisolone (steroid) therapy for the treatment of the initial episode of childhood nephrotic syndrome.
Aim of the study
To compare an extended (sixteen week) tapering prednisolone regimen with the standard eight week regimen as originally proposed by the International Study of Kidney Disease in Children (ISKDC).
PREDNOS Sample Size
A total of 224 children will be recruited into the study (112 in each arm).
PREDNOS Study Duration
The study duration is 4.5 years in total. The accrual period is for 2 years and all patients will be followed up for at least 24 months and for a variable time period beyond 24 months until the end of the trial.
Link to PREDNOS 2 Website.
PREDNOS 2 is a national multicentre double blind randomised controlled trial of short course daily prednisolone therapy at the time of upper respiratory tract infection in children with relapsing steroid sensitive nephrotic syndrome (SSNS).
Full Title: Short course daily prednisolone therapy at the time of upper respiratory tract infection in children with relapsing steroid sensitive nephrotic syndrome.
Short Title: PREDNOS 2 study
Aim of the study: To evaluate the effectiveness of a six day course of daily prednisoline therapy at the time of URTI in reducing the development of subsequent nephrotic syndrome relapse in children with relapsing SSNS.
Study design: Double blind randomised controlled trial (RCT).
Sample size: 300 patients will be recruited into the study (150 in each arm) over a 2 year period.
Study Duration: The accrual period is for 2 years and all patients will be followed up for 12 months. The end of trial will be 6 months after the last data capture. The last data capture will be 12 months following recruitment of the last patient. The total study duration is 4 years.
Timeframes: NIHR HTA grant start date: 1st October 2012. Trial set up will take place in 6 months, recruitment will take 24 months, all patients will be followed up for 12 months and 6 months has been allocated for data analysis and report writing. The first patient was randomised on 19th March 2013.