In clinical trials, investigators are often interested in the effect of a given study treatment on a subgroup of patients with certain clinical or biological attributes in addition to its effect on the overall study population. Such a subgroup analysis would become even more important to the study sponsor if an efficacy claim can be made for the subgroup when the test for the overall study population fails at a prespecified alpha level. In practice, such a claim is often dependent on prespecification of the subgroup and certain implicit or explicit requirements placed on the study results due to ethical or regulatory concerns. By carefully considering these requirements, we propose a general statistical methodology for testing both the overall and subgroup hypotheses, which has optimal power and strongly controls the familywise Type I error rate.
Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.1002/sim.2825 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Abuse potential and analgesic efficacy of intravenous hydromorphone bolus administration among hospitalized patients with cancer pain: A double-blind, double dummy, randomized crossover trial.
Cancer
February 2025
Department of Palliative, Rehabilitation and Integrative Medicine, The University of Texas MD Anderson Cancer, Houston, Texas, USA.
Background: There is much concern that opioids administered as intravenous (iv) bolus for pain relief may inadvertently increase their risk for abuse. However, there is insufficient data to support this. The authors compared the abuse liability potential, analgesic efficacy, and adverse effect profile of fast (iv push) versus slow (iv piggyback) administration of iv hydromorphone among hospitalized patients requiring iv opioids for pain.
View Article and Find Full Text PDFImproving the Effectiveness of Sample Size Re-Estimation: An Operating Characteristic Focused, Hybrid Frequentist-Bayesian Approach.
Stat Med
February 2025
Biostatistics, Innovatio Statistics Inc., Bridgewater, New Jersey, USA.
Sample size re-estimation (SSR) is perhaps the most used adaptive procedure in both frequentist and Bayesian adaptive designs for clinical trials. The primary focus of all current frequentist and Bayesian SSR procedures is type I error control. We propose a hybrid frequentist-Bayesian SSR approach that focuses on optimizing operating characteristics (OC), which uses simulations to investigate the associated OC and adjusts accordingly.
View Article and Find Full Text PDFCardio-renal effect of dapagliflozin and dapagliflozin- saxagliptin combination on CD34 + ve hematopoietic stem cells (HSCs) and podocyte specific markers in type 2 diabetes (T2DM) subjects: a randomized trial.
Stem Cell Res Ther
January 2025
Department of Medicine, Veterans Affairs Medical Center, Washington, DC, USA.
Introduction: Effects of Dapagliflozin (Dapa) and Dapagliflozin-Saxagliptin combination (Combo) was examined on peripheral blood derived CD34 + Hematopoetic Stem Cells (HSCs) as a cellular CVD biomarker. Both Dapa (a sodium-glucose co-transporter 2 or SGLT2, receptor inhibitor) and Saxagliptin (a Di-peptydl-peptidase-4 or DPP4 enzyme inhibitor) are commonly used type 2 diabetes mellitus or T2DM medications, however the benefit of using the combination has not been evaluated for cardio-renal risk assessment, in a real-life practice setting, compared to a placebo.
Hypothesis: We hypothesized that Dapa will improve the outcomes when compared to placebo and the Combo maybe even more beneficial.
Persistent effect of salt reduction in schoolchildren and their families: 1-year follow-up after an application-based cluster randomized controlled trial.
BMC Med
January 2025
Yueyang Centre for Disease Control and Prevention, Yueyang, Hunan Province, China.
Background: A 12-month cluster randomized controlled trial (RCT) demonstrated the effectiveness of an application-based education program in reducing the salt intake and systolic blood pressure (SBP) of schoolchildren's adult family members. This study aimed to assess whether the effect at 12 months persisted at 24 months.
Methods: Fifty-four schools were randomly assigned to either the intervention or control group.
Second-generation anti-amyloid monoclonal antibodies for Alzheimer's disease: current landscape and future perspectives.
Transl Neurodegener
January 2025
Department of Biochemistry, College of Medicine, Konyang University, 158, Gwanjeodong-Ro Seo-Gu, Daejeon, 35365, Republic of Korea.
Alzheimer's disease (AD) is the most common type of dementia. Monoclonal antibodies (MABs) serve as a promising therapeutic approach for AD by selectively targeting key pathogenic factors, such as amyloid-β (Aβ) peptide, tau protein, and neuroinflammation. Specifically, based on their efficacy in removing Aβ plaques from the brains of patients with AD, the U.
View Article and Find Full Text PDFWant AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!