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- Mercuri, E., et al.
(författare)
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Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study
- 2020
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Ingår i: Journal of Comparative Effectiveness Research. - : Becaris Publishing Limited. - 2042-6305 .- 2042-6313. ; 9:5, s. 341-360
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Tidskriftsartikel (refereegranskat)abstract
- Aim: Strategic Targeting of Registries and International Database of Excellence (STRIDE) is an ongoing, multicenter registry providing real-world evidence regarding ataluren use in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). We examined the effectiveness of ataluren + standard of care (SoC) in the registry versus SoC alone in the Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study (DNHS), DMD genotype-phenotype/-ataluren benefit correlations and ataluren safety. Patients & methods: Propensity score matching was performed to identify STRIDE and CINRG DNHS patients who were comparable in established disease progression predictors (registry cut-off date, 9 July 2018). Results & conclusion: Kaplan-Meier analyses demonstrated that ataluren + SoC significantly delayed age at loss of ambulation and age at worsening performance in timed function tests versus SoC alone (p <= 0.05). There were no DMD genotype-phenotype/ataluren benefit correlations. Ataluren was well tolerated. These results indicate that ataluren + SoC delays functional milestones of DMD progression in patients with nmDMD in routine clinical practice. ClinicalTrials.gov identifier: NCT02369731. ClinicalTrials.gov identifier: NCT02369731.
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| 5. |
- Burisch, J., et al.
(författare)
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Environmental factors in a population-based inception cohort of inflammatory bowel disease patients in Europe : An ECCO-EpiCom study
- 2014
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Ingår i: Journal of Crohn's & Colitis. - : Oxford University Press. - 1873-9946 .- 1876-4479. ; 8:7, s. 607-616
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Tidskriftsartikel (refereegranskat)abstract
- Background and Aims: The incidence of inflammatory bowel disease (IBD) is increasing in Eastern Europe possibly due to changes in environmental factors towards a more "westernised" standard of Living. The aim of this study was to investigate differences in exposure to environmental factors prior to diagnosis in Eastern and Western European IBD patients.Methods: The EpiCom cohort is a population-based, prospective inception cohort of 1560 unselected IBD patients from 31 European countries covering a background population of 10.1 million. At the time of diagnosis patients were asked to complete an 87-item questionnaire concerning environmental factors.Results: A total of 1182 patients (76%) answered the questionnaire, 444 (38%) had Crohn's disease (CD), 627 (53%) ulcerative colitis (UC), and 111 (9%) IBD unclassified. No geographic differences regarding smoking status, caffeine intake, use of oral contraceptives, or number of first-degree relatives with IBD were found. Sugar intake was higher in CD and UC patients from Eastern Europe than in Western Europe while fibre intake was lower (p < 0.01). Daily consumption of fast food as well as appendectomy before the age of 20 was more frequent in Eastern European than in Western European UC patients (p < 0.01). Eastern European CD and UC patients had received more vaccinations and experienced fewer childhood infections than Western European patients (p < 0.01).Conclusions: In this European population-based inception cohort of unselected IBD patients, Eastern and Western European patients differed in environmental factors prior to diagnosis. Eastern European patients exhibited higher occurrences of suspected risk factors for IBD included in the Western lifestyle. (C) 2013 European Crohn's and Colitis Organisation. Published by Elsevier B.V. All rights reserved.
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- Marengo, D., et al.
(författare)
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Exploring the association between problem drinking and language use on Facebook in young adults
- 2019
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Ingår i: Heliyon. - : Elsevier Ltd. - 2405-8440. ; 5:10
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Tidskriftsartikel (refereegranskat)abstract
- Recent literature suggests that variations in both formal and content aspects of texts shared on social media tend to reflect user-level differences in demographic, psychosocial, and behavioral characteristics. In the present study, we examined associations between language use on Facebook and problematic alcohol use. We collected texts shared on Facebook by a sample of 296 adult social media users (66.9% females; mean age = 28.44 years (SD = 7.38)). Texts were mined using the closed-vocabulary approach based on the Linguistic Inquiry Word Count (LIWC) semantic dictionary, and an open-vocabulary approach performed via Latent Dirichlet Allocation (LDA). Then, we examined associations between emerging textual features and alcohol-drinking scores as assessed using the AUDIT-C questionnaire. As a final aim, we employed the Random Forest machine-learning algorithm to determine and compare the predictive accuracy of closed- and open-vocabulary features over users' AUDIT-C scores. We found use of words about family, school, and positive feelings and emotions to be negatively associated with alcohol use and problematic drinking, while words suggesting interest in sport events, politics and economics, nightlife, and use of coarse language were more frequent among problematic drinkers. Results coming from LIWC and LDA analyses were quite similar, but LDA added information that could not be retrieved only with LIWC analysis. Furthermore, open-vocabulary features outperformed closed-vocabulary features in terms of predictive power over participants’ AUDIT-C scores (r = .46 vs. r = .28, respectively). Emerging relationships between text features and offline behaviors may have important implications for alcohol screening purposes in the online environment.
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