评估药物开发中的患者风险,益处和结果:对regorafenib临床试验的观察性研究
Brody Dennis1, Chance Bratten1, Griffin K Hughes1
1Oklahoma State University Center for Health Sciences, Tulsa, Oklahoma, USA.
BMJ oncology
|January 31, 2025
概括
雷戈拉费尼布的试验显示,随着时间的推移,患者的风险增加和客观反应率较低,特别是在结直肠癌中. 需要更高质量的研究来澄清其真正的风险/收益概况.
科学领域:
- 在瘤学瘤学.
- 临床药理学 临床药理学
- 药物重用 药物重用
背景情况:
- 药物开发是昂贵的,促使制药公司通过临床试验将现有药物重新用于新的用途.
- 综合综合分析对于评估药物的效果与风险益处概况的评估至关重要.
研究的目的:
- 对所有涉及regorafenib的瘤临床试验进行全面分析.
- 为了建立一个完整的风险-益处概况regorafenib在癌症治疗.
主要方法:
- 对PubMed,Embase,Cochrane (CENTRAL) 和ClinicalTrials.gov进行系统查,以对固体癌症进行regorafenib试验.
- 提取关键数据,包括无进展生存率,总生存率,不良事件率和客观反应率 (ORR).
- 根据预先规定的终点和耐受性,将研究分类为积极,消极或不确定的.
主要成果:
- 分析包括56项试验,包括4960名参与者,涉及13种适应症;78.75%是非盲目的,73.21%是非随机的.
- 结肠直肠癌试验最初显示出积极的结果,但随着时间的推移趋向于负面,患者的风险增加,ORR始终低.
- 随着时间的推移,患者的累积风险增加,而客观反应率在所有研究中始终保持在低水平.
结论:
- 自从Regorafenib最初获得FDA对其主要指示的批准以来,其风险概况有所增加.
- 发现了大量非随机单臂试验,突出显示了需要更高质量的研究.
- 雷戈拉费尼布的疗效和安全性可能在癌症中比目前批准的适应症更为有利.
更多相关视频
08:12Detection of a Circulating MicroRNA Custom Panel in Patients with Metastatic Colorectal Cancer
Published on: March 14, 2019
5.4K
09:29Development and Maintenance of a Preclinical Patient Derived Tumor Xenograft Model for the Investigation of Novel Anti-Cancer Therapies
Published on: September 30, 2016
13.6K
相关概念视频
Types of Biopharmaceutical Studies: Controlled and Non-Controlled Approaches
114
Biopharmaceutical studies constitute a vital field aiming to enhance drug delivery methods and refine therapeutic approaches, drawing upon diverse interdisciplinary knowledge. In research methodologies, the choice between controlled and non-controlled studies significantly influences the study's reliability and accuracy.
Non-controlled studies, commonly employed for initial exploration, lack a control group, rendering them susceptible to biases and external influences. In contrast,...
Non-controlled studies, commonly employed for initial exploration, lack a control group, rendering them susceptible to biases and external influences. In contrast,...
114
Clinical Trials
6.6K
Clinical trials are prospective experimental studies conducted on humans to determine the safety and efficacy of treatments, drugs, diet methods, and medical devices. Using statistics in clinical trials enables researchers to derive reasonable and accurate conclusions from the collected data, allowing them to make wise decisions in uncertain situations. In medical research, statistical methods are crucial for preventing errors and bias.
There are four phases in a clinical trial. A phase one...
There are four phases in a clinical trial. A phase one...
6.6K
Cancer Survival Analysis
321
Cancer survival analysis focuses on quantifying and interpreting the time from a key starting point, such as diagnosis or the initiation of treatment, to a specific endpoint, such as remission or death. This analysis provides critical insights into treatment effectiveness and factors that influence patient outcomes, helping to shape clinical decisions and guide prognostic evaluations. A cornerstone of oncology research, survival analysis tackles the challenges of skewed, non-normally...
321
Hazard Ratio
78
The hazard ratio (HR) is a widely used measure in clinical trials to compare the risk of events, such as death or disease recurrence, between two groups over time. It reflects the ratio of hazard rates—the instantaneous risk of the event occurring—between a treatment group and a control group. This measure provides valuable insights into the relative effectiveness of a treatment by assessing how the risk of an event differs between the two groups.
For example, in a clinical trial...
For example, in a clinical trial...
78
Comparing the Survival Analysis of Two or More Groups
131
Survival analysis is a cornerstone of medical research, used to evaluate the time until an event of interest occurs, such as death, disease recurrence, or recovery. Unlike standard statistical methods, survival analysis is particularly adept at handling censored data—instances where the event has not occurred for some participants by the end of the study or remains unobserved. To address these unique challenges, specialized techniques like the Kaplan-Meier estimator, log-rank test, and...
131
