Pancreatic enzyme prescription following ivacaftor licensing: A retrospective analysis of the US and UK cystic fibrosis registries.

BACKGROUND: Relieving gastrointestinal symptoms is a research priority in cystic fibrosis. Emerging evidence highlights effects of cystic fibrosis transmembrane conductance regulator (CFTR) modulators on gastrointestinal function, including pancreatic sufficiency. This study explores ivacaftor licensing and treatment on recorded pancreatic enzyme replacement therapy (PERT) prescription in the US and UK CF registries. METHODS: Retrospective longitudinal registry study of recorded pancreatic PERT use between 2008 and 2017. Interrupted time series analysis in propensity-matched cohorts estimated annual change and step change according to ivacaftor eligibility before and after licensing year, 2012. Generalised estimating equations assessed adjusted risk of PERT use in individuals treated with ivacaftor after 2012 compared to untreated individuals. RESULTS: In the US CF registry, the difference in annual change in prevalence of PERT use post-2012 between eligible cases and ineligible controls was -5.0 per 1000 people/year (95 %CI -7.6; -2.3, p = 0.001). The step change and annual change in prevalence of PERT use in eligible cases was not significantly different to controls in the UK CF registry. Relative to the relationship in 2013, ivacaftor treatment in the US CF registry was associated with a lower adjusted risk ratio of PERT use compared to untreated individuals by 2016 (0.97, 95 %CI 0.96; 0.99), which was not observed in the UK CF registry. CONCLUSIONS: Licensing of ivacaftor was followed by a lower prevalence of PERT use in the eligible US population compared to pre-licensing period, as well as lower risk of PERT use in those who received treatment. Inconsistencies in US and UK CF registries were observed.

Prospective randomized pilot study of a novel patient-centered pathology report for colorectal polyps.

Objective: US patients have increased access to their medical records, yet the information is not always understandable. To improve patient understanding, we tested a patient-centered pathology report (PCPR) containing results for recent colon cancer screening or surveillance colonoscopy. Methods: A pilot randomized trial assessed the impact of addition of the PCPR to a standard pathology report on knowledge accuracy, decisional self-efficacy and control, and therapeutic alliance. Results: 55 participants were enrolled; 20 participants in the intervention group and 24 controls completed follow-up. There was no significant difference in polyp knowledge between groups at baseline or 30-days, with similar confidence in understanding their diagnoses, decisional self-efficacy, and therapeutic alliance. Most participants receiving a PCPR felt that it helped them understand their diagnosis better and should always be provided with the standard pathology report. Conclusion: Although patient attitudes toward the PCPR were positive, receiving it did not significantly improve knowledge accuracy or measures of self-efficacy. Further iterations should be explored to communicate key knowledge about colorectal polyp results. Innovation: A stakeholder-driven approach to PCPR development facilitated construction of a personalized document that has potential to increase patient's understanding for their results and needed follow-up.

Can computerized brain training games be used to identify early cognitive impairment in cirrhosis?

OBJECTIVES: We evaluated whether commercially available, computerized "brain-training" games can be used to identify subtle cognitive impairments in patients with cirrhosis. METHODS: We compared patients with cirrhosis who did not have overt encephalopathy (n=31), patients with pre-cirrhotic chronic liver disease (n=28), and normal controls without liver disease (n=16) with respect to their scores on the number connection test-A (NCT-A), the Inhibitory Control Test (ICT), and five, short (∼2.5 min), brain-training games that were administered on an Apple iPad and tested different cognitive domains. RESULTS: Patients with cirrhosis had similar scores to patients with pre-cirrhotic liver disease and slightly worse scores than normal controls in the NCT-A and the ICT, although these differences were not statistically significant. In contrast, patients with cirrhosis had significantly worse scores than patients with pre-cirrhotic liver disease and even more so than normal controls in all five of the brain-training games. After adjustment for age and educational attainment, these differences remained significant for two of the tests, "Color Match" which is a version of the Stroop test and measures selective attention, and "Memory Matrix", which measures visuospatial memory. The area under the receiver operating characteristic curve discriminating cirrhosis from pre-cirrhotic liver disease was 0.56 (95% confidence interval (CI) 0.41-0.72) for the ICT and 0.58 (95% CI 0.43-0.73) for the NCT-A, indicating no discrimination, while it was 0.75 (95% CI 0.63-0.87) for "Color Match" and 0.77 (95% CI 0.64-0.90) for "Memory Matrix", indicating good discrimination. CONCLUSIONS: Short, brain-training games administered on an iPad can be used as psychometric tests to detect subtle cognitive impairments in patients with cirrhosis without overt encephalopathy that could not be detected by the NCT-A or the ICT.