Patient-reported outcome assessment in vasculitis may provide important data and a unique perspective.

OBJECTIVE: To assess patients' self-estimates of the burden of disease in vasculitis and to compare data across patient populations from the US, Germany, and the UK. Outcome assessment in vasculitis primarily focuses on physician evaluations of disease activity and damage. Little is known about the patients' perspectives regarding the burden of disease. METHODS: Patients ranked (scale 0-5) a list of vasculitis-related items to estimate a combination of pain, interference with daily function, discomfort and/or annoyance, anxiety/psychological impact, and medical importance. Patients were also asked to list the 5 most troublesome aspects of the disease. RESULTS: Data from 264 patients with vasculitis from 3 countries were collected. Wegener's granulomatosis was the predominant disease, comprising 63% of the cohort. Diagnoses were confirmed by physicians in 98% of cases. The most common item from the free-text sections was fatigue (75%), followed by pain (31%), musculoskeletal symptoms (24%), difficulty breathing (19%), financial aspects (13%), nasal discharge/crusting (14%), and weight gain (10%). Rankings of individual items varied substantially: fatigue (3.5), loss of energy (3.4), weight gain (3.1), joint pain (3.0), and sinusitis (3.0) were the highest-ranked symptoms among those manifestations experienced by at least 50% of patients. Several severe manifestations (e.g., dialysis, seizures, oxygen dependency) were ranked lower (<3.0). CONCLUSION: Fatigue and reduced energy level are considered the most important disease burdens by patients with vasculitis; manifestations associated with organ damage were rated lower. The patients' perspectives of the impact of vasculitis differ from the physicians' ratings. Future outcome assessment in vasculitis should include the patients' perspectives.

The future of damage assessment in vasculitis.

Damage denotes the aspects of chronic disease that do not reverse with therapy. This concept is particularly important for the primary systemic vasculitides, since the careful differentiation between activity and damage may help avoid unnecessary exposure to cytotoxic medications. Damage significantly influences both longterm prognosis and quality of life. Because the primary systemic vasculitides have diverse manifestations, the use of a damage assessment instrument is crucial to ensure reproducibility. The Vasculitis Damage Index (VDI) is the only validated measure for damage assessment in vasculitis. Use of the VDI in recent clinical trials has shown that it may not adequately determine the full spectrum of damage experienced by patients with vasculitis of small- and medium-size vessels. We propose reexamining the way in which damage is assessed, focusing on vasculitides of small- and medium-size vessels, and outline an initiative to create a substantially revised and improved damage assessment instrument using data-driven approaches. This initiative is part of a larger international effort to create a unified approach to disease assessment for the primary systemic vasculitides.

Effect of Wegener's granulomatosis on work disability, need for medical care, and quality of life in patients younger than 40 years at diagnosis.

OBJECTIVES: To evaluate the effects of Wegener's granulomatosis (WG) on employment status, work disability, and need for medical care of 60 consecutive WG patients aged < or = 40 years at diagnosis. METHODS: Sixty WG patients (26 male, 34 female) with a median age of 36 years (range 17-48 years) and a median duration of disease of 39 months (range 0-228 months) completed self-administered questionnaires on hospitalization, medical care, and employment status plus the Medical Outcomes Study-Short Form-36 (SF-36) estimating their health-related quality of life. RESULTS: Thirty-two of the 60 patients reported full- or part-time employment more than 3 years after diagnosis. Only 14 of the 51 patients employed at diagnosis (27%) were currently receiving a permanent work disability pension due to WG. Two additional patients had lost work because of WG. Women who were employed at diagnosis had a nearly 3-fold higher risk of losing their jobs compared with men (P = 0.0006). There were no differences with regard to age at diagnosis, disease duration, disease severity, or education level between employed and unemployed patients. Employed patients had missed a median of 14 workdays (range 0-18 days) due to WG within the past 12 months. More than half of all patients (33 of 60) had been hospitalized during the previous 12 months because of WG. Ninety-three percent of all patients had visited their physician once or more per month, more than half of them at least once per week, regardless of employment status, severity of disease, or type of current medication. Unemployed WG patients experienced significant reductions in social and physical function and in their perceived degree of general health as assessed by the SF-36. CONCLUSIONS: Twenty-seven percent of WG patients younger than age 40 who were employed at diagnosis received permanent work disability within a disease duration of 39 months. Unemployment is followed by a considerable reduction in disease-related quality of life compared with employed patients, independent of severity and extent of disease. Furthermore, because patients were followed closely by an interdisciplinary team, a high rate of hospitalization and frequent visits to physicians resulted.