Project 12

National Rare Disease Working Group for Membranous Nephropathy

Establishment of a National Rare Disease Working Group for membranous Nephropathy

Prof Paul Brenchley and Dr Michael Venning at MRI have led the formation of a National Rare Disease working group for patients with Membranous nephropathy which has attracted financial support of £12,000 from the KRUK/Renal Association scheme. The working group will be established in the Autumn and will develop over the next 3 years these three main proposals;

Proposal for developing evidenced based clinical care pathways

The availability of a test for anti-PLA2R antibodies which are present in  75% of cases of primary MN will provide an evidence base for clinical decisions on whom to treat when to treat and how long to treat. For instance, data already gathered shows that patients with high anti-PLA2R levels are unlikely to experience spontaneous remission and therefore earlier intervention with immunosuppression may be justified for preserving renal function.

We need to collect data on both the existing immunosuppressive drugs and new drugs in terms of their effects on anti-PLA2R levels and efficiency of inducing partial and complete remission. The long term benefit of achieving partial remission needs stronger validation as this clinical outcome may have use as a primary endpoint for clinical trials.

We will develop ideas around different care pathways for mild versus severe disease, relapsing disease, maintenance  therapy, and the treatment of persistent refractory disease. A coordinated approach to performing observational studies on large cohorts is only possible through a structure such as RDWG.

Proposal for empowering and informing patients and families

Currently there is no large patient support group focused on MN and this is an ideal opportunity to establish a UK patient group. Advice from local renal patient associations confirms that patients would value such a support group and our patient representative has firsthand experience of local support groups. The MN RDWG website will through Renal RADAR and Renal Patient View encourage patients to view and upload clinical data online. Information suitable for patients about MN either written by members of the RDWG or available through web links to other sites will be available on the website.

The breakthough in understanding the autoimmune basis for MN will encourage more clinical trials in MN and so it will be important to make information available to patients on all new clinical trials in MN through the website. Similarly, outcomes of globally important clinical and lab research studies in MN will be summarised on the website. During the first three years of the RDWG in MN, we will look for opportunities to link with existing renal patient support groups to focus attention on MN.

Proposal for auditing clinical outcomes

Development of a UK National Registry for MN patients will allow robust audit of outcomes. We have two goals

To establish a registry of biopsy proven prevalent cases and new incident cases of both primary and secondary MN starting from Jan 2012. We anticipate that there could be 1200 cases of prevalent MN at CKD 2-4 in addition to 600 cases on renal replacement therapy and expect approx 100 incident cases per year. In total we seek to capture data on 2000 patients during the 3 year duration of this grant with a minimum of one complete data return per patient per year.

To capture and analyse clinical outcome data on patients entered into large national studies on MN since 2000 where patients consented for clinical outcome data to be collected and analysed (MRC-KRUK GN Biobank and MRC Trial of immunosuppression). Our current MRC study involves identifying all prevalent cases of MN that have progressed to CKD5 and information from Renal Registry identifies approx 600 cases with half on dialysis and half transplanted. We plan to audit the clinical outcomes of those who get recurrent disease compared to those who don’t with reference to primary/secondary disease and anti-PLA2R status.

The MN registry will enable clinical audit of immunosuppression usage, relapse rates, adverse events (infection/cardiovascular events) participation in clinical trials and quality of care indicators. Audit reports will be made available to clinicians and patients via the website and presented to relevant clinical and patient meetings.