SAT0578 TOWARDS EUROPEAN HARMONISATION OF HEALT CARE FOR PATIENTS WITH RARE IMMUNE DISORDERS: ERN RITA RESULT FROM THE REGISTRIES SURVEY

Abstract

Background Rare Immunodeficiency, AutoInflammatory and AutoImmune Disease (RITA) network is a European Research Network that brings together the leading centres for rare immune disorders (RID). Objectives To know the state of the art about diseases registries and research networks in Europe regarding patients with RID. Methods On April 2018 an on-line survey was sent to all 126 RITA members, of whom 45 are healthcare providers and 8 patients/family organizations. All data were collected and analysed using Excel program. Results Ninety RITA members (71%) replied. Collectively, 27 members are coordinating 25 registries, 53 members are participating in 38 registries, and 27 members knew the existence of 16 registries without participating. Only two members are not involved in any registries. Data about 53 different registries were collected across 14 European countries. Almost 50% of registries collect data on autoimmune disorders, while others are dedicated to primary immunodeficiencies and autoinflammatory diseases (respectively 15 registries, 28%, and 12 registries, 23%). Fifteen registries (28%) enrolled patients with a single specific disorder: in particular, three registries are devoted to systemic lupus erythematosus, two registries to Kawasaki disease or Behcet disease, and single registry to juvenile dermatomyositis, juvenile systemic sclerosis, juvenile idiopathic arthritis (JIA)-related uveitis, systemic JIA, Blau syndrome, sarcoidosis, Guillain-Barre syndrome, and myasthenia gravis. More than 55000 patients with RID are enrolled. The majority of registries (36; 68%) enrol only patients from national territories. Among the internationals, six collect data on autoimmune disorders (Pharmachild, BrainWorks, EuroMyositis, EULAR web library, UKIVAS registry and JIR cohort), five on primary immunodeficiencies (ESID, EBMT, SCETIDE, PCID and HLH registry), and three are devoted to autoinflammatory diseases (Eurofever, Infevers, and ImmunAID), despite also ESID registry and JIR cohort collect data on autoinflammatory diseases. Data usually collected in these registries are demography, diagnosis, clinical manifestations, laboratory tests and treatment, while genetic and imaging data are less frequently reported (respectively in 38% and 9% of registries). A treatment safety profile is reported in 29 registries (55%). Collectively, fifteen biobanks are counted. Conclusion The survey highlighted the pivotal role of national and international organizations in Europe to collect and organize clinical data on immune diseases, allowing the rapidly growing knowledge on these rare disorders, creating research networks and providing significant numbers of data to support new discoveries in the field. RITA network could improve the coordination of these numerous entities, supporting initiatives of collaboration. As a first attempt, the present survey revealed that the collection of key parameters about patient safety, as well as outcome data and quality of life measures should be improved among the registries of RITA network.