aHUSUK participated in the Second Meeting of the Rare Disease Group for aHUS. RDG aHUS is part of the Rare Renal Project set up by the Renal Association (see website news item 30 September 2013 )
The Group consists of haematologists, nephrologists, renal surgeons, clinical biologists and complementologists who undertake research in Universities in England, Scotland and Wales.
Their individual research interests relate specifically to aHUS causes, diagnosis and treatment through greater understanding of the component parts and the related mechanisms of the complement system and complement related antibodies. Some study the bio chemistry activities taking place within and between components, what can go wrong with components as well as what part the endolethial cells play. Others are looking at diagnostics including such matters as how complement activity can be measured and several are seeking new therapies and leading in clinical trials of these new therapies of which eculizumab is well known to aHUS patients.
The meeting focused on issues which are prerequisite to sustainable future aHUS research including:
- The accurate application of the newly created aHUS codes in aHUS patient records;
- Establishing patient registries for those who wish to participate in research;
- The logistics for collecting and storing patient blood samples in a biobank;
- Transferring other clinical data about patients into the registry;
- Providing aHUS information for clinicians and patients ( click here for a Patient Leaflet) on the Rare Renal website.
At present few adult patients have been included in the aHUS Registry and there are clearly some organisational and motivational barriers to be overcome before registration numbers become sufficient. aHUSUK would like to see the UK match the numbers of USA patients registered in the Alexion Registry in the UK Registry, which means a tenfold increase from current levels to over 100 aHUS patients.
This is just another example of why we need an aHUS Expert Centre so that constraints to good practice can be removed for the benefit of aHUS patients.