Improving patient outcomes in systemic vasculitis

Abstract

The systemic vasculitides are a group of rare heterogeneous conditions where the primary problem is inflammation of blood vessels. The underlying aetiology for most forms of vasculitis is unknown. The resulting blockage of blood flow causes tissue ischaemia which can result in organ damage or death. Making an early diagnosis is difficult. Treatment is usually toxic, and morbidity and mortality from vasculitis is high. Improving the long-term outcomes for patients with these conditions needs a multifaceted approach that encompasses early diagnosis, a better knowledge of the underlying aetiology, understanding the disease course and associated morbidity, and finding safer long-term treatment strategies. This thesis has a very clinical focus and brings together work that addresses many of these themes. The publications that form the core of this thesis cover current classification criteria for systemic vasculitis, how to make a diagnosis of vasculitis, the need for diagnostic criteria, morbidity and mortality in ANCA-associated vasculitis with a focus on cardiovascular disease and neuropathy, and validation of outcome measures which are now endorsed for use in trials of ANCA-associated vasculitis. The overarching goal of all the work presented is to improve outcomes for patients diagnosed with systemic vasculitis, whether it be by improving speed and accuracy of diagnosis, understanding the morbidity and mortality of vasculitis better in order to optimise therapy or by providing the tools to assess and monitor disease which in turn will enable physicians, funders or regulatory agencies to provide access to appropriate therapy.