Dataset Information

Statistical fundamentals on cancer research for clinicians: Working with your statisticians.

ABSTRACT:

Purpose

To facilitate understanding statistical principles and methods for clinicians involved in cancer research.

Methods

An overview of study design is provided on cancer research for both observational and clinical trials addressing study objectives and endpoints, superiority tests, non-inferiority and equivalence design, and sample size calculation. The principles of statistical models and tests including contemporary standard methods of analysis and evaluation are discussed. Finally, some statistical pitfalls frequently evident in clinical and translational studies in cancer are discussed.

Results

We emphasize the practical aspects of study design (superiority vs non-inferiority vs equivalence study) and assumptions underpinning power calculations and sample size estimation. The differences between relative risk, odds ratio, and hazard ratio, understanding outcome endpoints, purposes of interim analysis, and statistical modeling to minimize confounding effects and bias are also discussed.

Conclusion

Proper design and correctly constructed statistical models are critical for the success of cancer research studies. Most statistical inaccuracies can be minimized by following essential statistical principles and guidelines to improve quality in research studies.

SUBMITTER: Xu W

PROVIDER: S-EPMC7829109 | biostudies-literature | 2021 Mar

REPOSITORIES: biostudies-literature

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Publications

Statistical fundamentals on cancer research for clinicians: Working with your statisticians.

Xu Wei W Huang Shao Hui SH Su Jie J Gudi Shivakumar S O'Sullivan Brian B

Clinical and translational radiation oncology 20210116

<h4>Purpose</h4>To facilitate understanding statistical principles and methods for clinicians involved in cancer research.<h4>Methods</h4>An overview of study design is provided on cancer research for both observational and clinical trials addressing study objectives and endpoints, superiority tests, non-inferiority and equivalence design, and sample size calculation. The principles of statistical models and tests including contemporary standard methods of analysis and evaluation are discussed. ...[more]

PMID: 33532634

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Project description:The highly regulated pH of cells and the less-regulated pH of the surrounding extracellular matrix (ECM) is the result of a delicate balance between metabolic processes and proton production, proton transportation, chemical buffering, and vascular removal of waste products. Malignant cells show a pronounced increase in metabolic processes where the 10- to 15-fold rise in glucose consumption is only the tip of the iceberg. Aerobic glycolysis (Warburg effect) is one of the hallmarks of cancer metabolism that implies excessive production of protons, which if stayed inside the cells would result in fatal intracellular acidosis (maintaining a strict acid-base balance is essential for the survival of eukaryotic cells). Malignant cells solve this problem by increasing mechanisms of proton transportation which expel the excess acidity. This allows cancer cells to keep a normal intracellular pH, or even overshooting this mechanism permits a slightly alkaline intracellular tendency. The proton excess expelled from malignant cells accumulates in the ECM, where chronic hypoxia and relative lack of enough blood vessels impede adequate proton clearance, thus creating an acidic microenvironment. This microenvironment is quite heterogeneous due to the tumor's metabolic heterogeneity and variable degrees of hypoxia inside the tumor mass. The acidic environment (plus other necessary cellular modifications) stimulates migration and invasion and finally intravasation of malignant cells which eventually may result in metastasis. Targeting tumor pH may go in two directions: 1) increasing extracellular pH which should result in less migration, invasion, and metastasis; and 2) decreasing intracellular pH which may result in acidic stress and apoptosis. Both objectives seem achievable at the present state of the art with repurposed drugs. This hypothesis analyzes the altered pH of tumors and its implications for progression and metastasis and also possible repurposed drug combinations targeting this vulnerable side of cancer development. It also analyzes the double-edged approach, which consists in pharmacologically increasing intracellular proton production and simultaneously decreasing proton extrusion creating intracellular acidity, acid stress, and eventual apoptosis.

Dataset Information

Statistical fundamentals on cancer research for clinicians: Working with your statisticians.

Purpose

Methods

Results

Conclusion

Publications

Statistical fundamentals on cancer research for clinicians: Working with your statisticians.

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