4.5 Article

Comparison between splines and fractional polynomials for multivariable model building with continuous covariates: a simulation study with continuous response

Journal

STATISTICS IN MEDICINE
Volume 32, Issue 13, Pages 2262-2277

Publisher

WILEY
DOI: 10.1002/sim.5639

Keywords

continuous covariates; fractional polynomials; model selection; nonlinear effects; simulation; splines

Funding

  1. Deutsche Forschungsgemeinschaft [SA 580/4-2]
  2. UK Medical Research Council [MC_US_A737_0002]
  3. Medical Research Council [MC_EX_G0800814] Funding Source: researchfish
  4. MRC [MC_EX_G0800814] Funding Source: UKRI

Ask authors/readers for more resources

In observational studies, many continuous or categorical covariates may be related to an outcome. Various spline-based procedures or the multivariable fractional polynomial (MFP) procedure can be used to identify important variables and functional forms for continuous covariates. This is the main aim of an explanatory model, as opposed to a model only for prediction. The type of analysis often guides the complexity of the final model. Spline-based procedures and MFP have tuning parameters for choosing the required complexity. To compare model selection approaches, we perform a simulation study in the linear regression context based on a data structure intended to reflect realistic biomedical data. We vary the sample size, variance explained and complexity parameters for model selection. We consider 15 variables. A sample size of 200 (1000) and R2=0.2 (0.8) is the scenario with the smallest (largest) amount of information. For assessing performance, we consider prediction error, correct and incorrect inclusion of covariates, qualitative measures for judging selected functional forms and further novel criteria. From limited information, a suitable explanatory model cannot be obtained. Prediction performance from all types of models is similar. With a medium amount of information, MFP performs better than splines on several criteria. MFP better recovers simpler functions, whereas splines better recover more complex functions. For a large amount of information and no local structure, MFP and the spline procedures often select similar explanatory models. Copyright (c) 2012 John Wiley & Sons, Ltd.

Authors

I am an author on this paper
Click your name to claim this paper and add it to your profile.

Reviews

Primary Rating

4.5
Not enough ratings

Secondary Ratings

Novelty
-
Significance
-
Scientific rigor
-
Rate this paper

Recommended

Article Statistics & Probability

Is there a role for statistics in artificial intelligence?

Sarah Friedrich, Gerd Antes, Sigrid Behr, Harald Binder, Werner Brannath, Florian Dumpert, Katja Ickstadt, Hans A. Kestler, Johannes Lederer, Heinz Leitgob, Markus Pauly, Ansgar Steland, Adalbert Wilhelm, Tim Friede

Summary: Statistics plays a significant role in both the theoretical and practical understanding of artificial intelligence (AI) and in its future development. It contributes to methodological development, planning and design of studies, assessment of data quality and data collection, differentiation of causality and associations, as well as evaluation of uncertainty in results. Integrating statistical aspects into AI teaching is crucial for schools and universities.

ADVANCES IN DATA ANALYSIS AND CLASSIFICATION (2022)

Review Biochemical Research Methods

Stratified neural networks in a time-to-event setting

Fabrizio Kuruc, Harald Binder, Moritz Hess

Summary: This study explores the use of different data sets and loss functions to improve parameter learning of deep neural networks. The results show that employing stratified loss functions leads to better predictive power and lower prediction error, with known prognostic genes receiving higher importance.

BRIEFINGS IN BIOINFORMATICS (2022)

Review Multidisciplinary Sciences

Review of guidance papers on regression modeling in statistical series of medical journals

Christine Wallisch, Paul Bach, Lorena Hafermann, Nadja Klein, Willi Sauerbrei, Ewout W. Steyerberg, Georg Heinze, Geraldine Rauch

Summary: Although regression models are central in medical research, misconceptions still exist and recent statistical advancements are not adequately reflected in publications. Developing statistical guidance for addressing nonlinear effects, model specification, and variable selection is essential to better support medical researchers in performing or interpreting regression analyses.

PLOS ONE (2022)

Article Health Care Sciences & Services

Investigating treatment-effect modification by a continuous covariate in IPD meta-analysis: an approach using fractional polynomials

Willi Sauerbrei, Patrick Royston

Summary: In clinical trials, it is important to investigate whether a treatment effect is consistent in all patients or if certain variables indicate a different response to treatment. Categorizing continuous predictors into groups using cutpoints has several weaknesses. A better approach is to keep the variables continuous and analyze them using methods like Subpopulation Treatment Effect Pattern Plot (STEPP) and multivariable fractional polynomial interaction (MFPI). Meta-analysis techniques can be used to study treatment heterogeneity if individual patient data (IPD) from multiple studies are available. The recently proposed Meta-STEPP method was used to investigate the interaction of estrogen receptors with chemotherapy in eight randomized controlled trials (RCTs) of primary breast cancer patients.

BMC MEDICAL RESEARCH METHODOLOGY (2022)

Article Medicine, General & Internal

Structured reporting to improve transparency of analyses in prognostic marker studies

Willi Sauerbrei, Tim Haeussler, James Balmford, Marianne Huebner

Summary: Most prognostic factor studies are poorly reported and analyzed, which has severe consequences for related systematic reviews and meta-analyses.

BMC MEDICINE (2022)

Article Cardiac & Cardiovascular Systems

Renin, aldosterone, the aldosterone-to-renin ratio, and incident hypertension among normotensive subjects from the general population

Natalie Arnold, Iris M. Hermanns, Andreas Schulz, Omar Hahad, Volker H. Schmitt, Marina Panova-Noeva, Juergen H. Prochaska, Harald Binder, Norbert Pfeiffer, Manfred Beutel, Karl J. Lackner, Thomas Muenzel, Philipp S. Wild

Summary: This study investigates the predictive ability of direct plasma renin and aldosterone concentrations as well as their ratio (aldosterone-to-renin, ARR) for incident hypertension in the general population. The study finds that ARR has a stronger predictive value for incident hypertension than renin or aldosterone alone, especially in obese subjects.

CARDIOVASCULAR RESEARCH (2023)

Article Multidisciplinary Sciences

Comparison of variable selection procedures and investigation of the role of shrinkage in linear regression-protocol of a simulation study in low-dimensional data

Edwin Kipruto, Willi Sauerbrei

Summary: This paper aims to compare variable selection methods and investigate the impact of shrinkage of regression estimates in a simulation study within the framework of a classical linear regression model for low-dimensional data. It emphasizes the importance of conducting neutral comparison studies to address bias and improve the design and reporting of simulation studies in statistical methodology research.

PLOS ONE (2022)

Article Oncology

REMARK guidelines for tumour biomarker study reporting: a remarkable history

Daniel F. Hayes, Willi Sauerbrei, Lisa M. McShane

Summary: In 2005, experts in tumor biomarker research published the REporting recommendations for Tumor MARKer prognostic studies (REMARK) criteria, which, combined with the subsequent Biospecimen Reporting for Improved Study Quality (BRISQ) criteria, provide a framework for transparent reporting of study conduct and analyses.

BRITISH JOURNAL OF CANCER (2023)

Article Computer Science, Information Systems

The role of data sharing in survey dropout: a study among scientists as respondents

Urs Alexander Fichtner, Lukas Maximilian Horstmeier, Boris Alexander Bruhmann, Manuel Watter, Harald Binder, Jochen Knaus

Summary: This study aims to investigate the effect of data sharing on researchers' response patterns from the perspective of survey participants. The results show that data sharing does not have an impact on response bias or dropout for the researchers, and provide insights into the experiences and behaviors of the participants regarding data sharing.

JOURNAL OF DOCUMENTATION (2023)

Article Mathematical & Computational Biology

Deep learning and differential equations for modeling changes in individual-level latent dynamics between observation periods

Goeran Koeber, Raffael Kalisch, Lara M. C. Puhlmann, Andrea Chmitorz, Anita Schick, Harald Binder

Summary: When modeling longitudinal biomedical data, both dimensionality reduction and dynamic modeling are needed. This study proposes an extension that allows different sets of differential equation parameters for observation subperiods, improving the model's fit with a small dataset. The approach successfully identifies individual-level parameters and promising predictors for predicting resilience and informing future study design.

BIOMETRICAL JOURNAL (2023)

Article Medicine, General & Internal

Statistical analysis of high-dimensional biomedical data: a gentle introduction to analytical goals, common approaches and challenges

Joerg Rahnenfuehrer, Riccardo De Bin, Axel Benner, Federico Ambrogi, Lara Lusa, Anne-Laure Boulesteix, Eugenia Migliavacca, Harald Binder, Stefan Michiels, Willi Sauerbrei, Lisa McShane

Summary: This review provides an overview of key aspects and methods for analyzing high-dimensional data in biomedical research. It addresses the challenges and opportunities of analyzing high-dimensional data, and offers solutions and references for researchers.

BMC MEDICINE (2023)

Article Health Care Sciences & Services

How do (false) positively screened patients experience a screening programme for liver cirrhosis or fibrosis in Germany? A qualitative study

Urs A. Fichtner, Anita Arslanow, Harald Binder, Peter R. Galle, Christian Labenz, Frank Lammert, Julia Ortner, Dominikus Stelzer, Louis Velthuis, Erik Farin-Glattacker

Summary: This study aimed to explore the psychosocial consequences of positive liver screening results and identify influencing factors for perceived strain within a multistage screening program for liver cirrhosis and fibrosis in Germany. The results showed that the screening had emotional and behavioral impacts on the patients. Negative emotional consequences were primarily driven by suboptimal patient-provider communication and could be worsened by a lack of transparent information transfer. Patients sought information and support from their social environment. All patients had positive attitudes towards liver screening. To minimize the potential psychosocial consequences, transparent information and regular health communication should be emphasized during the screening process.

HEALTH EXPECTATIONS (2023)

Article Multidisciplinary Sciences

Needs assessment towards research data management at the Medical Faculty of the University of Freiburg-Data of the BE-KONFORM study

Urs A. Fichtner, Lukas M. Horstmeier, Boris A. Bruehmann, Harald Binder, Jochen Knaus

Summary: The BE-KONFORM study was conducted to investigate employees' needs of the Medical Faculty of the University of Freiburg regarding research data management. The study involved qualitative video interviews with four researchers and a standardized online survey. The final dataset included 236 complete cases, with 90% in German and 10% in English. The data offers the potential for connection with data collected in other contexts.

DATA IN BRIEF (2023)

Article Health Care Sciences & Services

App-Controlled Treatment Monitoring and Support for Patients With Head and Neck Cancer Undergoing Radiotherapy: Results From a Prospective Randomized Controlled Trial

Tanja Sprave, Michelle Pfaffenlehner, Raluca Stoian, Eleni Christofi, Alexander Ruehle, Daniela Zoeller, Alexander Fabian, Harald Fahrner, Harald Binder, Henning Schaefer, Eleni Gkika, Anca-Ligia Grosu, Felix Heinemann, Nils Henrik Nicolay

Summary: This study aimed to investigate the feasibility of integrating electronic patient-reported outcomes (ePROs) in the treatment surveillance pathway of patients with head and neck cancer (HNC) and assess the influence of app-based ePRO monitoring on patient satisfaction and quality of life. Results showed that ePRO monitoring was feasible and led to an increased reporting of HNC-specific symptom burden, while significantly improving certain domains of patient satisfaction.

JOURNAL OF MEDICAL INTERNET RESEARCH (2023)

No Data Available