The OMERACT Core Set of Outcome Measures for Use in Clinical Trials of ANCA-associated Vasculitis

Abstract

There has been a marked increase in the past 15 years in the number and quality of clinical trials in the idiopathic inflammatory vasculitides, especially the small-vessel vasculitides known as antineutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis [AAV; granulomatosis, with polyangiitis (Wegener's)]. These trials have been conducted by multicenter, international groups in Europe and the United States with financial support provided by government agencies and biopharmaceutical companies. This increased clinical trial activity in vasculitis has been accompanied by the development and validation of new outcome measures — a challenging process for these complex, multiorgan system diseases. The international OMERACT Vasculitis Working Group has developed and implemented an iterative research agenda that has utilized accumulated experience and datasets from several multicenter clinical trials and large cohort studies. This work has led to the development, evaluation, validation, and endorsement, through the OMERACT consensus and validation processes, of a “core set” of outcome measurements for use in clinical trials of AAV. The core set includes domains of disease activity, damage assessment, patient-reported outcomes, and mortality; there is at least one validated outcome measurement instrument available for each domain. This report reviews the domains of illness in AAV included in the OMERACT core set, describes the instruments validated to measure these domains, and presents the approved core set.

Damage in the anca-associated vasculitides: long-term data from the European Vasculitis Study group (EUVAS) therapeutic trials

“…13 In conclusion, this study highlights the extent of damage seen over an average of 7 years in the ANCA-associated vasculitides, specifically in terms of potentially treatment-related damage, such as cardiovascular outcomes, diabetes, osteoporosis and malignancy. Damage should, therefore, continue to be an important outcome in AAV clinical trials and long-term cohort studies, 8 with use of the VDI as a primary outcome measure. Such focus may potentially aid refinement of therapeutic regimens and limit long-term damage, 29 with possible benefits in terms of associated mortality.…”

“…4 Relapses are common, occurring in 40%, 5 and chronic comorbidities, secondary to vasculitis or its treatment, such as cardiovascular disease, are also increased. 6 7 The concept of damage infers irreversible aspects of disease which will not respond to escalation of immunosuppressive treatment, 8 in contrast with potentially reversible disease activity. The Vasculitis Damage Index (VDI) 9 is a validated checklist of 64 items, recording all damage from the onset of vasculitis, irrespective of whether it is caused by disease or treatment.…”

“…10 The number of relapses is associated with an increase in VDI score 13 14 and early disease activity scores correlate moderately well with later VDI scores. 14 15 The VDI has been approved by the Outcome Measures in Rheumatology (OMERACT) 8 group and the European League Against Rheumatism (EULAR), 16 as the key outcome measure to record damage in AAV clinical trials.…”

Effectiveness of Rituximab for the Otolaryngologic Manifestations of Granulomatosis With Polyangiitis (Wegener's)

“…Although the BVAS/WG includes 5 ENT items in the measurement of disease activity, direct otolaryngologic visualization and audiometric assessment are not required to assess manifestations such as subglottic disease or hearing loss (22). Therefore, the assessment of ENT disease activity with otolaryngologic examination at each visit in this study was more stringent and objective than the BVAS/ WG, which is widely accepted as the gold standard of disease activity measurement in GPA (23). The importance of objective assessment of ENT disease activity in GPA has recently been espoused by Del Pero et al, who created an ENT disease activity score based on endoscopic and audiometric examination (24).…”

The Value of the Russian National Registry of Patients With Anca-Associated Systemic Vasculitis as an Innovative Tool of Personalized Induction and Maintenance Therapy

“…Совокупность применяемых в НРАВ методов клинической оценки (BVAS, VDI, SF-36) рекомендована международными экспертами как основа рационального подхода к стандартизации мониторинга больных СВ, вы-бора оптимального протокола лечения и оценки прогрес-сирования патологического процесса [14]. Эта концеп-ция была поддержана в рекомендациях EULAR и ERA-EDTA, Британского общества ревматологов [3,15,16], но еще не получила широкого распространения в клини-ческой практике.…”