| 1. |
- Campbell, C., et al.
(author)
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Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy
- 2020
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In: Journal of Comparative Effectiveness Research. - : Becaris Publishing Limited. - 2042-6305 .- 2042-6313. ; 9:14
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Journal article (peer-reviewed)abstract
- Aim:Assess the totality of efficacy evidence for ataluren in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD).Materials & methods:Data from the two completed randomized controlled trials (ClinicalTrials.gov: NCT00592553; NCT01826487) of ataluren in nmDMD were combined to examine the intent-to-treat (ITT) populations and two patient subgroups (baseline 6-min walk distance [6MWD] >= 300-<400 or <400 m). Meta-analyses examined 6MWD change from baseline to week 48.Results:Statistically significant differences in 6MWD change with ataluren versus placebo were observed across all three meta-analyses. Least-squares mean difference (95% CI): ITT (n = 342), +17.2 (0.2-34.1) m, p = 0.0473; >= 300-<400 m (n = 143), +43.9 (18.2-69.6) m, p = 0.0008; <400 m (n = 216), +27.7 (6.4-49.0) m, p = 0.0109.Conclusion:These meta-analyses support previous evidence for ataluren in slowing disease progression versus placebo in patients with nmDMD over 48 weeks. Treatment benefit was most evident in patients with a baseline 6MWD >= 300-<400 m (the ambulatory transition phase), thereby informing future trial design.
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| 2. |
- McDonald, C. M., et al.
(author)
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Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients
- 2022
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In: Journal of Comparative Effectiveness Research. - : Becaris Publishing Limited. - 2042-6305 .- 2042-6313. ; 11:3
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Journal article (peer-reviewed)abstract
- Aim: We investigated the effect of ataluren plus standard of care (SoC) on age at loss of ambulation (LoA) and respiratory decline in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD) versus patients with DMD on SoC alone. Patients & methods: Study 019 was a long-term Phase III study of ataluren safety in nmDMD patients with a history of ataluren exposure. Propensity score matching identified Study 019 and CINRG DNHS patients similar in disease progression predictors. Results & conclusion: Ataluren plus SoC was associated with a 2.2-year delay in age at LoA (p = 0.0006), and a 3.0-year delay in decline of predicted forced vital capacity to <60% in nonambulatory patients (p = 0.0004), versus SoC. Ataluren plus SoC delays disease progression and benefits ambulatory and nonambulatory patients with nmDMD. ClinicalTrials.gov: .
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| 3. |
- Mercuri, E., et al.
(author)
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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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In: Journal of Comparative Effectiveness Research. - : Becaris Publishing Limited. - 2042-6305 .- 2042-6313. ; 9:5, s. 341-360
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Journal article (peer-reviewed)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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| 4. |
- Vroman, Heleen, et al.
(author)
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Continuous vital sign monitoring in patients after elective abdominal surgery: a retrospective study on clinical outcomes and costs
- 2023
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In: Journal of Comparative Effectiveness Research. - : Becaris Publishing. - 2042-6305 .- 2042-6313. ; 12:2
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Journal article (peer-reviewed)abstract
- Plain language summary What is this article about?Continuous vital sign monitoring assists in identifying deteriorating patients outside intensive care. This study analyses changes in clinical outcomes and costs before and after implementation of a clinical vital sign monitoring device in postsurgical patients on a general ward in a Dutch hospital. What were the results?Results show that after implementation, patients were less likely to be admitted to the intensive care unit, had shorter length of stay and had lower hospital stay costs. Aim: To assess changes in outcomes and costs upon implementation of continuous vital sign monitoring in postsurgical patients. Materials & methods: Retrospective analysis of clinical outcomes and in-hospital costs compared with a control period. Results: During the intervention period patients were less frequently admitted to the intensive care unit (ICU) (p = 0.004), had shorter length of stay (p < 0.001) and lower costs (p < 0.001). The intervention was associated with a lower odds of ICU admission (odds ratio: 0.422; p = 0.007) and ICU related costs (odds ratio: -662.4; p = 0.083). Conclusion: Continuous vital sign monitoring may have contributed to fewer ICU admissions and lower ICU costs in postsurgical patients. Tweetable abstractA retrospective study of clinical outcomes and costs compared with a control period indicated that implementation of continuous vital sign monitoring in postsurgical patients in a general hospital in The Netherlands may have contributed to fewer intensive care unit admissions and lower intensive care unit costs.
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