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Karl Johnson Katherine W. Saylor Isabella Guynn Karen Hicklin Jonathan S. Berg Kristen Hassmiller Lich 《Genetics in medicine》2022,24(2):262-288
PurposeUnderstanding the value of genetic screening and testing for monogenic disorders requires high-quality, methodologically robust economic evaluations. This systematic review sought to assess the methodological quality among such studies and examined opportunities for improvement.MethodsWe searched PubMed, Cochrane, Embase, and Web of Science for economic evaluations of genetic screening/testing (2013-2019). Methodological rigor and adherence to best practices were systematically assessed using the British Medical Journal checklist.ResultsAcross the 47 identified studies, there were substantial variations in modeling approaches, reporting detail, and sophistication. Models ranged from simple decision trees to individual-level microsimulations that compared between 2 and >20 alternative interventions. Many studies failed to report sufficient detail to enable replication or did not justify modeling assumptions, especially for costing methods and utility values. Meta-analyses, systematic reviews, or calibration were rarely used to derive parameter estimates. Nearly all studies conducted some sensitivity analysis, and more sophisticated studies implemented probabilistic sensitivity/uncertainty analysis, threshold analysis, and value of information analysis.ConclusionWe describe a heterogeneous body of work and present recommendations and exemplar studies across the methodological domains of (1) perspective, scope, and parameter selection; (2) use of uncertainty/sensitivity analyses; and (3) reporting transparency for improvement in the economic evaluation of genetic screening/testing. 相似文献
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Hajrunisa Cubro Karl A. Nath Sonja Suvakov Oscar Garcia-Valencia Santosh Parashuram Wendy M. White Tracey L. Weissgerber Meryl C. Nath Natasa M. Milic Fernando Sontag Livius V. d’Uscio Yi Zhu James L. Kirkland Tamar Tchkonia Mariam P. Alexander Reade A. Quinton Zvonimir S. Katusic Joseph P. Grande Vesna D. Garovic 《Kidney international》2021,99(3):646-656
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Sergey Motov Stefan Mann Jefferson Drapkin Mahlaqa Butt Antonios Likourezos Elizabeth Yetter Jason Brady Nechama Rothberger Ankit Gohel Peter Flom Mo Mai Christian Fromm John Marshall 《The American journal of emergency medicine》2019,37(2):220-227
Study objective
We compare the analgesic efficacy and safety of subdissociative intravenous-dose ketamine (SDK) versus morphine in geriatric Emergency Department (ED) patients.Methods
This was a prospective, randomized, double-blind trial evaluating ED patients aged 65 and older experiencing moderate to severe acute abdominal, flank, musculoskeletal, or malignant pain. Patients were randomized to receive SDK at 0.3?mg/kg or morphine at 0.1?mg/kg by short intravenous infusion over 15?min. Evaluations occurred at 15, 30, 60, 90, and 120?min. Primary outcome was reduction in pain at 30?min. Secondary outcomes included overall rates of adverse effects and incidence of rescue analgesia.Results
Thirty patients per group were enrolled in the study. The primary change in mean pain scores was not significantly different in the ketamine and morphine groups: 9.0 versus 8.4 at baseline (mean difference 0.6; 95% CI ?0.30 to 1.43) and 4.2 versus 4.4 at 30?min (mean difference ?0.2; 95% CI ?1.93 to1.46). Patients in the SDK group reported higher rates of psychoperceptual adverse effects at 15, 30, and 60?min post drug administration. Two patients in the ketamine group and one in the morphine group experienced brief desaturation episodes. There were no statistically significant differences with respect to changes in vital signs and need for rescue medication.Conclusion
SDK administered at 0.3?mg/kg over 15?min provides analgesic efficacy comparable to morphine for short-term treatment of acute pain in the geriatric ED patients but results in higher rates of psychoperceptual adverse effects.ClinicalTrials.gov Registration #: NCT02673372. 相似文献7.
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Robert Justo Cameron Ward Anthony Slater Julie McEniery Phil Sargent Alan Isles Tom R Karl Nelson Alphonso 《Journal of paediatrics and child health》2020,56(6):833-837
We describe a complex change process for the paediatric cardiac service in Queensland that involved transitioning the service out of an essentially adult hospital into one of two children's hospitals in Brisbane. This initial step was complex as the governance was changed from Queensland Health to Mater Health, an independent faith‐based organisation who became the new employer. Six years later, the service was again transitioned; this time to the newly constructed Queensland Children's Hospital, with a Hospital and Health Services Board as the employer under the aegis of Queensland Health. This was a complex journey. As with all change processes there was resistance to change on the part of some individuals. Five years on from the second major change, the service is settled, has an excellent workplace culture, has excellent clinical outcomes and has become research intensive. 相似文献