Unmet needs in ANCA-associated vasculitis: Physicians’ and patients’ perspectives

Abstract

In recent years, clinical research has increased significantly and therapies for antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis have improved. However, there are still unanswered questions and unmet needs about AAV patients. The purpose of this review is to examine the frontiers of research related to emerging biomarkers eventually predicting relapse, and new therapeutic approaches, not to mention new quality of life assessment tools. Identifying predictors of relapse may help optimize therapeutic strategies, minimize disease recurrence, and reduce treatment-related side effects. In addition, it is important to recognize that patients may suffer long-term consequences of the disease and its treatment, which, although life-saving, is often associated with significant side effects. Our goal, therefore, is to highlight what has been achieved, the pitfalls, and what still needs to be done, comparing the views of physicians and patients.

Validation of the Italian version of the ANCA-associated vasculitis patient-reported outcome (AAV-PRO) questionnaire

“…The VDI and BVASv3 did not appear to be related to the AAV-PRO_ita scores ( r < 0.3, i.e. weak correlation), suggesting that the perspective of physicians and patients might be very different [ 7 ]. This lack of correlation was also confirmed recently by another study [ 13 ], encouraging the role of AAV-PRO as an additional and complementary endpoint to traditional clinical instruments (i.e.…”

“…In particular, nearly one-third of patients classified fatigue as moderate or severe, which indicates that it has a significant role in the psychosocial impact of this illness. Currently, there is no specific treatment for fatigue, and fatigue is not addressed in the current management guidelines [ 17 ], and this represents a case of unmet needs in AAV [ 7 ]. With regard to the PF (i.e.…”

“…It is a 29-item disease-specific PRO measure in the English language, proving to be valid, reliable, feasible and able to discriminate among disease states. AAV-PRO could become an extremely useful tool to support physicians in their choice of treatment and to investigate the point of view of patients on disease activity [ 7 ].…”

Systemic vasculitis: one year in review 2024

“…Despite the great amount of consolidated evidence, there are still numerous unmet needs in AAV management, which have been the focus of research efforts in 2023 (116). The AAV patientsreported outcomes (PRO) questionnaire plays a complementary role alongside BVAS and Vasculitis Damage Index (VDI), capturing aspects of the patient experience that may not be captured by traditional measures (117).…”

Anti-LAMP-2 Antibody Seropositivity in Children with Primary Systemic Vasculitis Affecting Medium- and Large-Sized Vessels

“…Seropositivity and specificity of ANCA for PR3 or MPO has some proven utility in the diagnosis and differentiation of "ANCA-associated vasculitis (AAV)" subtypes and more recently were demonstrated to have power in predicting diseaseassociated risks in adult-onset disease [4]. Similar diagnostic and prognostic tools do not exist for "ANCA-negative" vasculitis [7], which in children includes ~10-30% of cases of small-vessel vasculitis and the majority, if not all, forms of vasculitis affecting medium to large blood vessels [8].…”