A fixed one-sided significance level of 5% is commonly used to interpret the statistical significance of randomized clinical trial (RCT) outcomes. While it is necessary to reduce the false positive rate, the threshold used could be chosen quantitatively and transparently to specifically reflect patient preferences regarding benefit-risk tradeoffs as well as other considerations. How can patient preferences be explicitly incorporated into RCTs in Parkinson's disease (PD), and what is the impact on statistical thresholds for device approval? In this analysis, we apply Bayesian decision analysis (BDA) to PD patient preference scores elicited from survey data. BDA allows us to choose a sample size () and significance level () that maximizes the overall expected value to patients of a balanced two-arm fixed-sample RCT, where the expected value is computed under both null and alternative hypotheses. For PD patients who had previously received deep brain stimulation (DBS) treatment, the BDA-optimal significance levels fell between 4.0% and 10.0%, similar to or greater than the traditional value of 5%. Conversely, for patients who had never received DBS, the optimal significance level ranged from 0.2% to 4.4%. In both of these populations, the optimal significance level increased with the severity of the patients' cognitive and motor function symptoms. By explicitly incorporating patient preferences into clinical trial designs and the regulatory decision-making process, BDA provides a quantitative and transparent approach to combine clinical and statistical significance. For PD patients who have never received DBS treatment, a 5% significance threshold may not be conservative enough to reflect their risk-aversion level. However, this study shows that patients who previously received DBS treatment present a higher tolerance to accept therapeutic risks in exchange for improved efficacy which is reflected in a higher statistical threshold.
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http://dx.doi.org/10.1080/10543406.2023.2170400 | DOI Listing |
Implement Sci Commun
January 2025
Center for School and Community Partnerships, Boise State University, Boise, ID, USA.
Background: Rural School Support Strategies (RS3) is a bundle of implementation supports (including training, technical assistance, and a virtual learning collaborative) designed for the scale-up of universal prevention initiatives. This study addresses mechanisms of action, exploring whether positive effects of RS3 on implementation fidelity are attributable to improvements in functioning of school implementation teams, and increases in organizational readiness.
Methods: Data are from a cluster-randomized hybrid Type 3 implementation-effectiveness trial of RS3 among rural Idaho schools implementing Positive Behavioral Interventions and Supports (PBIS).
Acta Neuropathol Commun
January 2025
Hopp Children's Cancer Center Heidelberg (KiTZ), Heidelberg, Germany.
Recent genomic studies have allowed the subdivision of intracranial ependymomas into molecularly distinct groups with highly specific clinical features and outcomes. The majority of supratentorial ependymomas (ST-EPN) harbor ZFTA-RELA fusions which were designated, in general, as an intermediate risk tumor variant. However, molecular prognosticators within ST-EPN ZFTA-RELA have not been determined yet.
View Article and Find Full Text PDFPerioper Med (Lond)
January 2025
Department of Anesthesia, Characteristic Medical Center of Chinese People's Armed Police Force (PAP), Tianjin, China.
Background: We investigated the consistency and accuracy of the Index of Consciousness (IoC) and the Bispectral Index (BIS) in monitoring the sedative effect of ciprofol during the induction of general anesthesia. There is extensive literature that reports good consistency and correlations between the IoC1 and the BIS in reflecting the sedation levels induced by propofol and sevoflurane but not by ciprofol.
Objective: The aim was to compare the consistency and accuracy of the IoC and BIS in monitoring the sedative effect of ciprofol during the induction of general anesthesia.
Diabetol Metab Syndr
January 2025
The International Peace Maternity and Child Health Hospital, School of Medicine, Shanghai Jiao Tong University, No. 910, Hengshan Rd., Shanghai, 200030, China.
Background: Triglyceride-glucose (TyG) index was suggested as a possible surrogate for insulin resistance and a predictor for cardiovascular diseases and diabetes in the non-pregnant population. However, the relationship between TyG index in early pregnancy and adverse pregnancy outcomes (APOs), and the contribution of pre-pregnancy body mass index (BMI) was still illusive.
Methods: A large retrospective cohort study involving 67,936 pregnant Chinese women between 2017 and 2022 was conducted.
J Transl Med
January 2025
Department of Clinical Laboratory, The First Hospital of Jilin University, Changchun, 130000, China.
Background: Recent studies suggest a connection between immunoglobulin light chains (IgLCs) and coronary heart disease (CHD). However, current diagnostic methods using peripheral blood IgLCs levels or subtype ratios show limited accuracy for CHD, lacking comprehensive assessment and posing challenges in early detection and precise disease severity evaluation. We aim to develop and validate a Coronary Health Index (CHI) incorporating total IgLCs levels and their distribution.
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