Impact of concurrent glomerulonephritis on outcomes of diffuse alveolar haemorrhage in antineutrophil cytoplasmic antibody-associated vasculitis.

OBJECTIVES: Antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) commonly presents with diffuse alveolar haemorrhage (DAH) and/or glomerulonephritis. Patients who present with DAH but without kidney involvement have been understudied. METHODS: Patients with DAH diagnosed by bronchoscopy and attributed to AAV over 8.5 years were retrospectively identified through electronic medical records and bronchoscopy reporting software. Patients with end-stage kidney disease (ESKD) or prior kidney transplant were excluded. Characteristics, treatments, and outcomes were abstracted. RESULTS: 30 patients were identified with DAH secondary to AAV. Five with ESKD or prior kidney transplant, and one with concomitant anti-glomerular basement membrane disease, were excluded, leaving 24 patients for analysis. At the time of qualifying bronchoscopy, six patients had no apparent kidney involvement by AAV, while eight of 18 with kidney involvement required dialysis. Of the eight patients dialysed during the initial hospitalisation, four were declared to have ESKD and three died in the subsequent year (one of whom did both). None of the 16 patients without initial dialysis requirement developed kidney involvement requiring dialysis in the subsequent year, though three of the six without initial evidence of kidney involvement by AAV ultimately developed it. No patient without initial kidney involvement died during follow-up. CONCLUSIONS: In our cohort, patients with DAH due to AAV without initial kidney involvement did not develop kidney involvement requiring dialysis or die during the follow-up period, though half of patients without initial evidence of kidney involvement subsequently developed it. Larger studies are warranted to better characterise this population and guide medical management.

Physicians' vs patients' global assessments of disease activity in rheumatology and musculoskeletal trials: A meta-research project with focus on reasons for discrepancies.

BACKGROUND: In most rheumatic and musculoskeletal diseases (RMDs), global assessments of disease activity by physicians and patients are 'anchor outcomes' in therapeutic trials evaluating whether a treatment is effective. OBJECTIVES: To compare physicians' vs patients' global assessments of disease activity in RMD trials and explore reasons for discrepancies between them. METHODS: Eligible trials were sampled from systematic reviews of treatments for RMDs by using the Cochrane database of systematic reviews (i.e., reviews from the Cochrane Musculoskeletal Group, [CMSG]). Randomized controlled trials (RCTs) of interventions for RMDs were eligible if they reported quantitative analyses of both physicians´ and patients´ global assessments at the same time point for the comparison of the same experimental intervention against the same comparator (i.e., placebo, no treatment, or other treatment). We accepted data from trial comparisons for each type of outcome, regardless of the type of intervention and type of RMD within the CMSG. Using mixed-effects meta-regression models, we assigned the dependent variable as the ratio of odds ratios (ROR) of global change with the experimental intervention, versus the control comparator. An ROR>1 would indicate that physicians rated the experimental intervention more favorable than their patients did. RESULTS: We were able to estimate the ROR (data from both physicians' and patients' global assessments) across 70 trials (116 randomized comparisons) in 7 diseases (ankylosing spondylitis, fibromyalgia, psoriatic arthritis, osteoarthritis, rheumatoid arthritis, systemic lupus erythematosus, and gout). The combined ROR across all effectiveness comparisons were rated significantly in favor of the intervention by physicians: ROR=1.15 CI 95% (1.07 to 1.23). This combined ROR was based on a substantial heterogeneity across comparisons (I2=89.1%). Across all the stratified analyses, the type of the RMD was an informative reason for discrepancies, with a statistically significant ROR in rheumatoid arthritis ROR=1.33, CI 95% (1.13 to 1.56), unlike the ROR in all other conditions (ROR=1.04, CI 95% (0.95-1.14). CONCLUSION: In comparative effectiveness research on rheumatology, physicians' global assessments of disease activity, surprisingly, are more in favor of the experimental interventions than are those of the patients.

ANCA-Associated Vasculitis: An Update.

Anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) represents a group of small vessel vasculitides characterized by granulomatous and neutrophilic tissue inflammation, often associated with the production of antibodies that target neutrophil antigens. The two major antigens targeted by ANCAs are leukocyte proteinase 3 (PR3) and myeloperoxidase (MPO). AAV can be classified into 3 categories based on patterns of clinical involvement: namely, granulomatosis with polyangiitis (GPA), microscopic polyangiitis (MPA) and eosinophilic GPA (EGPA). Clinically, AAV involves many organ systems including the lungs, kidneys, skin, and nervous system. The prognosis of AAV has improved dramatically due to advances in the understanding of its pathogenesis and treatment modalities. This review will highlight some of the recent updates in our understanding of the pathogenesis, clinical manifestations, and treatment options in patients with AAV focusing on kidney involvement.

Analysis of Patient Preferences in Lung Cancer - Estimating Acceptable Tradeoffs Between Treatment Benefit and Side Effects.

OBJECTIVE: Increased treatment options and longer survival for lung cancer have generated increased interest in patient preferences. Previous studies of patient preferences in lung cancer have not fully explored preference heterogeneity. We demonstrate a method to explore preference heterogeneity in the willingness of patients with lung cancer and caregivers to trade progression-free survival (PFS) with side effects. PATIENTS AND METHODS: Patients and caregivers attending a national lung cancer meeting completed a discrete-choice experiment (DCE) designed through a collaboration with patients. Participants answered 13 choice tasks described across PFS, short-term side effects, and four long-term side effects. Side effects were coded as a one-level change in severity (none-mild, mild-moderate, or moderate-severe). A mixed logit model in willingness-to-pay space estimated preference heterogeneity in acceptable tradeoffs (time equivalents) between PFS and side effects. The study was reported following quality indicators from the United States Food and Drug Administration's patient preference guidance. RESULTS: A total of 87 patients and 24 caregivers participated in the DCE. Participants would trade 3.7 month PFS (95% CI (CI): 3.3-4.1) for less severe functional long-term treatment side effects, 2.3 months for less severe physical long-term effects (CI: 1.9-2.8) and cognitive long-term effects (CI: 1.8-2.8), 0.9 months (CI: 0.4-1.4) for less severe emotional long-term effects, and 1.8 months (CI: 1.4-2.3) for less severe short-term side effects. Most participants (90%) would accept treatment with more severe functional long-term effects for 8.4 additional month PFS. CONCLUSION: Participants would trade PFS for changes in short-term side effects and long-term side effects, although preference heterogeneity existed. Lung cancer treatments that offer less PFS but also less severe side effects might be acceptable to some patients.