2019 RaDaR meeting

On Monday 2nd September 2019 a joint patient and clinician event was held at in London to discuss current and future research around the RaDaR rare disease registry, as well as ways to boost patient engagement in research. 

The agenda, a report from the day and pdfs of the presentations can be found below.

RaDaR Meeting Agenda

RaDaR Meeting Report

Presentations

Current status of RaDaR – Melanie Dillon, RaDaR Operations Officer

Ethics and re-consent – Dr Kate Bramham, Rare Diseases Committee Deputy

ADPKD – Dr Regada El-Damanawi, Research Fellow 

NephroS/NURTuRE – Prof Moin Saleem, Paediatric Nephrologist, Bristol

PREDICT – Dr Kate Bramham Rare Diseases Committee Deputy Chair

ERKNet – Tess Harris, PKD Charity

NIHR BioResource – Dr Kathy Stirrups, Head of Sample Management

NCARDRS Mary Bythell, Head of Rare Disease Registration

NHS Digital – Jackie Shears, Associate Director, Data Acquisitions

Patient Council and information resources – Fiona Loud, Kidney Care Policy Director

Kidney Research UK support – Pete Storey, KRUK Rare Disease Programme Lead

Care coordination research opportunity

COordiNated Care Of Rare Diseases (CONCORD) is a research study which aims to investigate how services are currently coordinated for rare conditions and how they should be coordinated in the future.

The study is funded by the National Institute for Health Research and is being led by researchers at University College London, in collaboration with Genetic Alliance UK and others. They are currently looking for patients, carers and healthcare professionals to share their priorities and views about the coordination of care for rare conditions, via an online survey. The survey will take around 30 minutes to complete. The findings will be used to make recommendations with the aim of influencing policy.

The survey can be accessed at: http://bit.ly/concord-survey

Further information about the study can be found at:

www.geneticalliance.org.uk/our-work/healthcare-and-delivery/coordinated-care-of-rare-diseases-concord/

Further research opportunities

As well as taking part in the RaDaR Rare Disease Registry, we recommend that patients who are interested in research visit the NIHR’s new Be Part of Research website which allows you to search for studies by condition and/or location.

Studies that are being led or endorsed by Clinicians involved in RaDaR are advertised on the Patient Information pages for each condition.

New RaDaR condition – BK Nephropathy

We are pleased to announce that RaDaR is now open to a new condition – BK Nephropathy.

No new approvals are needed to start recruiting these patients.

Detailed instructions on the inclusion criteria and data entry are available to download from https://tinyurl.com/BK-Neph-data or on request from Melanie.Dillon@renalregistry.nhs.uk

Hyperoxaluria research trials

There are trials for adult patients with primary hyperoxaluria types 1, 2 & 3 and enteric hyperoxaluria, which are all recruiting or about to start. Here are the key inclusion criteria:

•Adult patient with PH1: eGFR>45 and 24h U Oxalate >700 umol/24h

•Adult patient with PH1 or 2: eGFR>30 and 24h U Oxalate >600 umol/24h

•Adult patient with PH1,2 or 3: eGFR between 15 and 90 and relatively high urine oxalate (likely to give plasma oxalate>10 umol/l but it is not necessary to measure this)

•Adult with enteric hyperoxaluria: eGFR>30 and 24h U Oxalate >550 umol/24h

These are with investigational products from Alnylam, Dicerna, Oxthera and Allena respectively.

Enquiries will be directed to the units conducting these trials. We are also happy to see patients as a one-off or on-going or just discuss details of these trials with patients or clinicians. If you are a patient or relative of someone with one of these conditions, please ask your doctor about these trials, or contact Melanie.dillon@renalregistry.nhs.uk

World Kidney Day 2019

Help us bring together millions of people across the world and unite with one powerful voice for kidney health awareness.

World Kidney Day in the UK is led by the Kidney Charities Together group (Kidney Care UK, Kidney Research UK, NKF (Kidney Patients UK) and the PKD Charity). Help us bring together millions of people across the world and unite with one powerful voice for kidney health awareness.

Over 180 support packs have already been sent out to renal and transplant units as well as individuals and patient groups supporting the day by organising an awareness activity or event.

What are you planning, following the 2019 global theme of Kidney Health for Everyone Everywhere?

Ideas for activities and downloadable resources are available from www.worldkidneyday.co.uk and you can apply for a support pack www.worldkidneyday.co.uk/order-support-pack/ but don’t delay – stocks are running low

Follow us on Facebook, Twitter or Instagram and share our daily  Kidney Facts (from 7-14  March). You can also add a Facebook frameorTwibbon to your profile picture to show your support.

NURTuRE Study for Nephrotic Syndrome patients

NURTuRE (the National Unified Renal Translational Research Enterprise) is a unique kidney biobank for chronic kidney disease (CKD) and idiopathic nephrotic syndrome (INS), covering England, Scotland and Wales, which has the potential to unlock answers to some of the biggest questions about CKD and INS.

Biological samples (plasma, serum, urine, DNA and tissue) from 3,000 patients with CKD and over 800 patients with INS are being collected and stored under strict Standard Operating Procedures for both academic and industry research worldwide. This will lead to a greater ability to identify patients who will benefit from better, earlier diagnosis and person-specific new treatments, leading to better health outcomes.

We need patient volunteers for NURTuRE – no extra hospital visits… just your consent to be involved in this exciting, pioneering study.

If you are a patient with NS at one of the participating renal units, you may be offered the opportunity to take part in the NURTuRE biobank study. If you haven’t yet been approached, and you are interested in participating, please ask the doctor or nurse at your renal unit.

 NURTuRE is a research collaboration involving a charity, academia and industry, managed by Kidney Research UK and jointly led by the University of Bristol (NURTuRE-INS (NephroS) study) and the University of Nottingham (NURTuRE-CKD study).

Full details at www.nurturebiobank.org/patients