Page 267 - The Vasculitides Volumes 2

In: The Vasculitides, Volume 2 ISBN: 978-1-63463-112-9
Editors: David S. Younger © 2015 Nova Science Publishers, Inc.

Chapter 33

Future Directions of Therapeutic Trials

Rona M. Smith, M.D.? and David R. W. Jayne, M.D.

Addenbrooke?s Hospital, Cambridge University Hospitals, University of
Cambridge School of Clinical Medicine, and the National Health Service

Foundation Trust, Cambridge, UK

Abstract

The vasculitides are heterogeneous disorders characterized by inflammation of blood
vessels. The introduction of immunosuppressive therapy with corticosteroids and
cyclophosphamide transformed systemic vasculitis from a fatal disease to a largely
treatable condition. Over the past 40 years, considerable progress has also been achieved
in refining immunosuppressive regimens with a focus on minimizing toxicity. A
proportion of patients are refractory to current therapies with up to 50% of patients
experiencing clinical relapse within five years, and excess morbidity and disability as
well as mortality, in part related to treatment toxicity. As knowledge of the pathogenesis
of vasculitis has grown, mirrored by the availability of biological agents, there has been a
revolution in vasculitis treatment. Lymphocyte and cytokine targeted agents are being
evaluated and entering the routine therapeutic arena with enhanced potential to improve
treatment outcomes. As rare diseases, treatment advances in the vasculitides depend on
international collaborative research networks to establish an evidence-base for newer
agents, develop recommendations for patient management and implement randomized
clinical trials to establish efficacy.

Introduction

The systemic vasculitides include a diverse range of conditions characterized by
leukocyte infiltration of blood vessel walls, fibrinoid necrosis and vascular damage with

? Corresponding author: Rona M. Smith, M.D. Vasculitis and Lupus Clinic, Addenbrooke?s Hospital Cambridge,
Hills Road, Cambridge CB2 UK. E-mail: [email protected].

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