Journal of clinical epidemiology
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As psychopathology and social functioning can worsen with repeated psychotic episodes in schizophrenia, relapse prevention is critical. Because high nonadherence rates limit the efficacy of pharmacotherapy, the use of long-acting injectable (LAI) antipsychotics is considered an important treatment option. To date, many studies comparing LAIs and oral antipsychotics have been conducted; however, the results are mixed, and careful interpretation of the data is required. ⋯ Divergent results from studies using different methodologies create a dilemma for comparative effectiveness research, and LAI studies may serve as an example of a situation in which a conventional RCT is not the gold standard. Traditional RCTs generally increase adherence compared with clinical practice and, therefore, might not be well suited to detect differences between LAIs and oral medications, because any increase in adherence affects patients on oral medications more than those on LAIs and thus leads to an underestimation of any potential difference in effectiveness. A possible solution would be the implementation of a true effectiveness trial in which post-randomization involvement would be kept to a minimum to better reflect routine practice.
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Examining covariate balance is the prescribed method for determining the degree to which propensity score methods should be successful at reducing bias. This study assessed the performance of various balance measures, including a proposed balance measure based on the prognostic score (similar to a disease risk score), to determine which balance measures best correlate with bias in the treatment effect estimate. ⋯ Researchers should consider using prognostic score-based balance measures for assessing the performance of propensity score methods for reducing bias in nonexperimental studies.
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We evaluated the inter-rater reliability (IRR) of assessing the quality of evidence (QoE) using the Grading of Recommendations, Assessment, Development, and Evaluation (GRADE) approach. ⋯ Our findings suggest that trained individuals using the GRADE approach improves reliability in comparison to intuitive judgments about the QoE and that two individual raters can reliably assess the QoE using the GRADE system.
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Comparative Study
Stepped wedge designs could reduce the required sample size in cluster randomized trials.
The stepped wedge design is increasingly being used in cluster randomized trials (CRTs). However, there is not much information available about the design and analysis strategies for these kinds of trials. Approaches to sample size and power calculations have been provided, but a simple sample size formula is lacking. Therefore, our aim is to provide a sample size formula for cluster randomized stepped wedge designs. ⋯ For CRTs, the stepped wedge design is far more efficient than the parallel group and ANCOVA design in terms of sample size.
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Competing risks endpoints are frequently encountered in hematopoietic stem cell transplantation where patients are exposed to relapse and treatment-related mortality. Both cause-specific hazards and direct models for the cumulative incidence functions have been used for analyzing such competing risks endpoints. For both approaches, the popular models are of a proportional hazards type. ⋯ We argue that a complete understanding of the event dynamics requires that both hazards and cumulative incidence be analyzed side by side, and that this is generally the most rigorous scientific approach to analyzing competing risks data. That is, understanding the effects of covariates on cause-specific hazards and cumulative incidence functions go hand in hand. A case study illustrates our proposal.