HTA34 The Role of the Mainz Severity Score Index (MSSI) on Disease Related-Clinical Events in Patients with Fabry Disease

Abstract

Fabry disease (FD) is a rare, chronic, slowly progressing multisystem disorder. Prevention of renal, cardiovascular, and cerebrovascular events (ie, Fabry clinical events; FCEs) is the main treatment goal. Because such events are challenging to track given the limited timeframes of clinical studies, a holistic measurement tool covering all three domains of interest and providing long-term outcome prediction would be valuable. The Mainz Severity Score Index (MSSI) constitutes a promising endpoint in FD because it considers general, neurological, cardiovascular, and renal signs and symptoms and score changes are seen shortly after starting treatment. Currently, its predictive value for FCEs in FD is not well established. The present study was aimed at investigating MSSI-to-FCE relationship in FD.