Explained variation and degrees of necessity and of sufficiency for competing risks survival data.

Fine and Gray model competing risks explained variation necessary condition sufficient condition

Journal

Biometrical journal. Biometrische Zeitschrift
ISSN: 1521-4036
Titre abrégé: Biom J
Pays: Germany
ID NLM: 7708048

Informations de publication

Date de publication:
Mar 2024
Historique:
revised: 15 11 2023
received: 24 05 2023
accepted: 08 12 2023
medline: 27 2 2024
pubmed: 27 2 2024
entrez: 27 2 2024
Statut: ppublish

Résumé

In this contribution, the Schemper-Henderson measure of explained variation for survival outcomes is extended to accommodate competing events (CEs) in addition to events of interest. The extension is achieved by moving from the unconditional and conditional survival functions of the original measure to unconditional and conditional cumulative incidence functions, the latter obtained, for example, from Fine and Gray models. In the absence of CEs, the original measure is obtained as a special case. We define explained variation on the population level and provide two different types of estimates. Recently, the authors have achieved a multiplicative decomposition of explained variation into degrees of necessity and degrees of sufficiency. These measures are also extended to the case of competing risks survival data. A SAS macro and an R function are provided to facilitate application. Interesting empirical properties of the measures are explored on the population level and by an extensive simulation study. Advantages of the approach are exemplified by an Austrian study of breast cancer with a high proportion of CEs.

Identifiants

pubmed: 38409618
doi: 10.1002/bimj.202300140
doi:

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

e2300140

Informations de copyright

© 2024 The Authors. Biometrical Journal published by Wiley-VCH GmbH.

Références

Amabile, S., Roccuzzo, G., Pala, V., Tonella, L., Rubatto, M., Merli, M., Fava, P., Ribero, S., Fierro, M. T., Queirolo, P., & Quaglino, P. (2021). Clinical significance of distant metastasis-free survival (DMFS) in melanoma: A narrative review from adjuvant clinical trials. Journal of Clinical Medicine, 10, 5475. https://doi.org/10.3390/jcm10235475
Austin, P. C., & Fine, J. P. (2017). Practical recommendations for reporting Fine-Gray model analyses for competing risk data. Statistics in Medicine, 36, 4391-4400. https://doi.org/10.1002/sim.7501
Beyersmann, J., Allignol, A., & Schumacher, M. (2012). Competing risks and multistate models with R. Springer.
Cox, D. R. (1972). Regression models and life-tables. Journal of theRoyal Statistical Society, Series B Methodology, 34, 187-220.
Crowder, M. (2001). Classical competing risks. Chapman & Hall/CRC.
Fine, J. P., & Gray, R. J. (1999). A proportional hazards model for the subdistribution of a competing risk. Journal of the American Statistical Association, 94, 496-509. https://doi.org/10.1080/01621459.1999.10474144
Geskus, R. B. (2016). Data analysis with competing risks and intermediate states. CRC Press.
Gleiss, A., Henderson, R., & Schemper, M. (2021). Degrees of necessity and of sufficiency: Further results and extensions, with an application to Covid-19 mortality in Austria. Statistics in Medicine, 40, 3352-3366. https://doi.org/10.1002/sim.8961
Gleiss, A., & Schemper, M. (2019). Quantifying degrees of necessity and of sufficiency in cause-effect relationships with dichotomous and survival outcomes. Statistics in Medicine, 38, 4733-4748. https://doi.org/10.1002/sim.8331
Gleiss, A., Zeillinger, R., Braicu, E. I., Trillsch, F., Vergote, I., & Schemper, M. (2016). Statistical controversies in clinical research: the importance of importance. Annals of Oncology, 27, 1185-1189. https://doi.org/10.1093/annonc/mdw159
Harrell, F. E. (2015). Regression modeling strategies (2nd ed.). Springer.
Healy, M. J. R. (1990). Measuring importance. Statistics in Medicine, 9, 633-637. https://doi.org/10.1002/sim.4780090609
Heinze, G., & Schemper, M. (2003). Comparing the importance of prognostic factors in Cox and logistic regression using SAS. Computer Methods and Programs in Biomedicine, 71(2), 155-163. https://doi.org/10.1016/S0169-2607(02)00077-9
Hielscher, T., Zucknick, M., Werft, W., & Benner, A. (2010). On the prognostic value of survival models with application to gene expression signatures. Statistics in Medicine, 29, 818-829. https://doi.org/10.1002/sim.3768
Kattan, M. W. (2004). Evaluating a new marker's predictive contribution. Clinical Cancer Research, 10, 822-824. https://doi.org/10.1158/1078-0432.CCR-03-0061
Lambertini, M., Agbor-Tarh, D., Metzger-Filho, O., Ponde, N. F., Poggio, F., Hilbers, F. S., Korde, L. A., Chumsri, S., Werner, O., Del Mastro, L., Caparica, R., Moebus, V., Moreno-Aspitia, A., Piccart, M. J., & de Azambuja, E. (2020). Prognostic role of distant disease-free interval from completion of adjuvant trastuzumab in HER2-positive early breast cancer: analysis from the ALTTO (BIG 2-06) trial. ESMO Open, 5(6), e000979. https://doi.org/10.1136/esmoopen-2020-000979
Morris, T. P., White, I. R., & Crowther, M. J. (2019). Using simulation studies to evaluate statistical methods. Statistics in Medicine, 38, 2074-2102. https://doi.org/10.1002/sim.8086
Putter, H., Fiocco, M., & Geskus, R. B. (2007). Tutorial in biostatistics: Competing risks and multi-state models. Statistics in Medicine, 26, 2389-2430. https://doi.org/10.1002/sim.2712
Schemper, M. (2003). Predictive accuracy and explained variation. Statistics in Medicine, 22, 2299-2308. https://doi.org/10.1002/sim.1486
Schemper, M., & Henderson, R. (2000). Predictive accuracy and explained variation in Cox regression. Biometrics, 56, 249-255. https://doi.org/10.1111/j.0006-341X.2000.00249.x
Schemper, M., & Smith, T. L. (1996). A note on quantifying follow-up in studies of failure time. Controlled Clinical Trials, 17, 343-346. https://doi.org/10.1016/0197-2456(96)00075-X
Schmid, M., Jakesz, R., Samonigg, H., Kubista, E., Gnant, M., Menzel, C., Seifert, M., Seifert, M., Taucher, S., Taucher, S., Steindorfer, P., Kwasny, W., Stierer, M., Tausch, C., Fridrik, M., Wette, V., Steger, G., & Hausmaninger, H. (2003). Randomized trial of tamoxifen versus tamoxifen plus aminoglutethimide as adjuvant treatment in postmenopausal breast cancer patients with hormone receptor-positive disease: Austrian breast and colorectal cancer study group trial 6. Journal of Clinical Oncology, 21, 984-990. https://doi.org/10.1200/JCO.2003.01.138
Schoop, R., Beyersmann, J., Schumacher, M., & Binder, H. (2011). Quantifying the predictive accuracy of time-to-event models in the presence of competing risks. Biometrical Journal, 53, 88-112. https://doi.org/10.1002/bimj.201000073
Wolbers, M., Koller, M. T., Stel, V. S., Schaer, B., Jager, K. J., Leffondré, K., & Heinze, G. (2014). Competing risks analyses: Objectives and approaches. European Heart Journal, 35, 2936-2941. https://doi.org/10.1093/eurheartj/ehu131
Wu, C., & Li, L. (2018). Quantifying and estimating the predictive accuracy for censored time-to-event data with competing risks. Statistics in Medicine, 37, 3106-3124. https://doi.org/10.1002/sim.7806

Auteurs

Andreas Gleiss (A)

Center for Medical Data Science, Institute of Clinical Biometrics, Medical University of Vienna, Vienna, Austria.

Michael Gnant (M)

Comprehensive Cancer Center, Medical University of Vienna, Vienna, Austria.

Michael Schemper (M)

Center for Medical Data Science, Institute of Clinical Biometrics, Medical University of Vienna, Vienna, Austria.

Classifications MeSH