The European Scleroderma Trials and Research group (EUSTAR) task force for the development of revised activity criteria for systemic sclerosis: derivation and validation of a preliminarily revised EUSTAR activity index

BACKGROUND: Validity of European Scleroderma Study Group (EScSG) activity indexes currently used to assess disease activity in systemic sclerosis (SSc) has been criticised. METHODS: Three investigators assigned an activity score on a 0-10 scale for 97 clinical charts. The median score served as gold standard. Two other investigators labelled the disease as inactive/moderately active or active/very active. Univariate-multivariate linear regression analyses were used to define variables predicting the 'gold standard', their weight and derive an activity index. The cut-off point of the index best separating active/very active from inactive/moderately active disease was identified by a receiver-operating curve analysis. The index was validated on a second set of 60 charts assessed by three different investigators on a 0-10 scale and defined as inactive/moderately active or active/very active by other two investigators. One hundred and twenty-three were investigated for changes over time in the index and their relationships with those in the summed Medsger severity score (MSS). RESULTS: A weighted 10-point activity index was identified and validated: Δ-skin=1.5 (Δ=patient assessed worsening during the previous month), modified Rodnan skin score (mRss) >18=1.5, digital ulcers=1.5, tendon friction rubs=2.25, C-reactive protein >1 mg/dL=2.25 and diffusing capacity of the lung for CO (DLCO) % predicted <70%=1.0. A cut-off ≥2.5 was found to identify patients with active disease. Changes in the index paralleled those of MSS (p=0.0001). CONCLUSIONS: A preliminarily revised SSc activity index has been developed and validated, providing a valuable tool for clinical practice and observational studies

Effectiveness of TNF-inhibitors, abatacept, IL6-inhibitors and JAK-inhibitors in 31 846 patients with rheumatoid arthritis in 19 registers from the 'JAK-pot' collaboration

Background: JAK-inhibitors (JAKi), recently approved in rheumatoid arthritis (RA), have changed the landscape of treatment choices. We aimed to compare the effectiveness of four current second-line therapies of RA with different modes of action, since JAKi approval, in an international collaboration of 19 registers. Methods: In this observational cohort study, patients initiating tumour necrosis factor inhibitors (TNFi), interleukin-6 inhibitors (IL-6i), abatacept (ABA) or JAKi were included. We compared the effectiveness of these treatments in terms of drug discontinuation and Clinical Disease Activity Index (CDAI) response rates at 1 year. Analyses were adjusted for patient, disease and treatment characteristics, including lines of therapy and accounted for competing risk. Results: We included 31 846 treatment courses: 17 522 TNFi, 2775 ABA, 3863 IL-6i and 7686 JAKi. Adjusted analyses of overall discontinuation were similar across all treatments. The main single reason of stopping treatment was ineffectiveness. Compared with TNFi, JAKi were less often discontinued for ineffectiveness (adjusted HR (aHR) 0.75, 95% CI 0.67 to 0.83), as was IL-6i (aHR 0.76, 95% CI 0.67 to 0.85) and more often for adverse events (aHR 1.16, 95% CI 1.03 to 1.33). Adjusted CDAI response rates at 1 year were similar between TNFi, JAKi and IL-6i and slightly lower for ABA. Conclusion: The adjusted overall drug discontinuation and 1 year response rates of JAKi and IL-6i were similar to those observed with TNFi. Compared with TNFi, JAKi were more often discontinued for adverse events and less for ineffectiveness, as were IL-6i.</p

Using respondent-driven sampling (RDS) to recruit illegal poly-substance users in Cape Town, South Africa: implications and future directions

Background: South Africa continues to witness an increase in illicit poly-substance use, although a precise measurement continues to be compounded by difficulties in accessing users. In a pilot attempt to use respondent-driven sampling (RDS)—a chain referral sampling method used to access populations of individuals who are ‘hard-to-reach’—this article documents the feasibility of the method as recorded in a simultaneously run, multisite, poly-substance study in Cape Town. Here we aim to a) document the piloting of RDS among poly-substance users in the three socio-economic disparate communities targeted; b) briefly document the results; and c) review the utility of RDS as a research tool. Methods: Three cross-sectional surveys using standard RDS procedures were used to recruit active poly-substance users and were concurrently deployed in three sites. Formative research was initially conducted to assess the feasibility of the survey. To determine whether RDS could be used to successfully recruit poly-substance users, social network characteristics, such as network size was determined. Results: A 42.5 % coupon return rate was recorded in total from 12 initial seeds. There were vast differences in the recruitment chains of individual seeds—two generated more than 90 recruits, and 2 of the 10 recruitment chains showing a length of more than 10 waves. Findings include evidence of the use of 3 or more substances in all three sites, high levels of unemployment among users, with more than a third of participants in two sites reporting arrest for drug use in the past 12 months. Conclusions: Our results indicate that RDS was a feasible and acceptable sampling method for recruiting participants who may not otherwise be accessible. Future studies can use RDS to recruit such cohorts, and the method could form part of broader efforts to document vulnerable populations