Pronostico anca vasculitis

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O u t c o m e M e a s u re s in ANCA-associated Vas c u l i t i s
Ravi Suppiah, BHB, MBChB, PGDipSM, Joanna Robson, Raashid Luqmani, DM, FRCP, FRCP(E)*
KEYWORDS      Vasculitis  Outcome measures Birmingham Vasculitis Activity Score Vasculitis Damage Index Combined Damage Assessment Index Patient-reported outcome measures

The antineutrophil cytoplasm antibody(ANCA)–associated group of vasculitides encompass Wegener’s granulomatosis (WG), microscopic polyangiitis (MPA), and Churg-Strauss syndrome (CSS) but also includes some patients with syndromes that do not neatly fit into these disease categories. The ANCA-associated vasculitides are multisystem diseases that predominantly affect small vessels (including the renal glomeruli and lung capillaries) and, if leftuntreated, have a uniformly high mortality.1–4 Modern therapy has improved outcome,5 but early death and chronic morbidity (eg, dialysis dependence) remains a feature of these diseases.6–12 With advances in acute therapy for vasculitis, other long-term consequences such as cardiovascular events, thromboembolic events, and malignancy become increasingly important. End-stage organ failure such askidney failure or respiratory failure can make life difficult for survivors. This morbidity coupled with the effects of drug toxicity can significantly reduce quality of life, which is a crucial end point for patients.13,14 In addition, relapse, and low-grade persistent disease manifestations are common and require ongoing immunosuppression that may result in poor long-term outcomes.15–18 Clinicaltrials in the past decade have attempted to determine the optimal therapeutic strategy for induction and maintenance therapy.10–12,19–21 Outcomes can be judged in terms of life, death, and defined organ failure, but for chronic low-grade morbidity, it is useful to quantify this in a systematic way for clinical trials and for practical use when evaluating the accrual of morbidity in daily practice.To accurately quantify disease morbidity, make appropriate treatment decisions (ie, only use immunosuppression

RS is funded by the Rose Hellaby Medical Scholarship, New Zealand. Department of Rheumatology, Nuffield Orthopaedic Centre, Windmill Road, Oxford, OX3 7LD, UK * Corresponding author. E-mail address: Rheum Dis Clin N Am 36 (2010) 587–607doi:10.1016/j.rdc.2010.04.001 0889-857X/10/$ – see front matter ª 2010 Elsevier Inc. All rights reserved.


Suppiah et al

for active disease rather than chronic scarring), and to serve as appropriate outcome measures in clinical trials, several clinical tools have been developed to specifically measure disease activity and disease damage. In addition to reporting the incidence ofdeath and renal failure, these tools can be used to define the main outcome being measured in trials of ANCA-associated vasculitis. This article provides an overview of the outcomes measured in clinical trials of ANCA-associated vasculitis and describes the main clinical tools that are used to quantify these concepts. Table 1 provides a summary of definitions and outcome measures, some of which aremore comprehensively validated than others. The current focus is on disease activity and damage, but future attention will be on patient-reported outcome measures and functional status (eg, ability to work or participate in normal activities).

Death is an important and valid outcome measure when assessing the balance of efficacy and safety of treatment regimens. Most early deaths inANCA vasculitis are attributable to infection or active vasculitis,6,22 whereas late deaths may relate to increased cardiovascular disease and malignancy.23–25 Severity and distribution of organ involvement are currently the best predictors of short- and medium-term survival.16,26,27 Predictors of mortality in ANCA-associated vasculitis are shown in Table 1.6,27–29 As a result of routine...
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