Skip to main content
Top
Gepubliceerd in: Quality of Life Research 3/2023

17-09-2022

Flexible modeling of longitudinal health-related quality of life data accounting for informative dropout in a cancer clinical trial

Auteurs: Audrey Winter, Benjamin Cuer, Thierry Conroy, Beata Juzyna, Sophie Gourgou, Caroline Mollevi, Célia Touraine

Gepubliceerd in: Quality of Life Research | Uitgave 3/2023

Log in om toegang te krijgen
share
DELEN

Deel dit onderdeel of sectie (kopieer de link)

  • Optie A:
    Klik op de rechtermuisknop op de link en selecteer de optie “linkadres kopiëren”
  • Optie B:
    Deel de link per e-mail

Abstract

Purpose

A joint modeling approach is recommended for analysis of longitudinal health-related quality of life (HRQoL) data in the presence of potentially informative dropouts. However, the linear mixed model modeling the longitudinal HRQoL outcome in a joint model often assumes a linear trajectory over time, an oversimplification that can lead to incorrect results. Our aim was to demonstrate that a more flexible model gives more reliable and complete results without complicating their interpretation.

Methods

Five dimensions of HRQoL in patients with esophageal cancer from the randomized clinical trial PRODIGE 5/ACCORD 17 were analyzed. Joint models assuming linear or spline-based HRQoL trajectories were applied and compared in terms of interpretation of results, graphical representation, and goodness of fit.

Results

Spline-based models allowed arm-by-time interaction effects to be highlighted and led to a more precise and consistent representation of the HRQoL over time; this was supported by the martingale residuals and the Akaike information criterion.

Conclusion

Linear relationships between continuous outcomes (such as HRQoL scores) and time are usually the default choice. However, the functional form turns out to be important by affecting both the validity of the model and the statistical significance.

Trial registration

This study is registered with ClinicalTrials.gov, number NCT00861094.
Bijlagen
Alleen toegankelijk voor geautoriseerde gebruikers
Literatuur
2.
go back to reference Ediebah, D. E., Galindo-Garre, F., Uitdehaag, B. M. J., Ringash, J., Reijneveld, J. C., Dirven, L., Zikos, E., Coens, C., van den Bent, M. J., Bottomley, A., & Taphoorn, M. J. B. (2015). Joint modeling of longitudinal health-related quality of life data and survival. Quality of Life Research, 24, 795–804. https://doi.org/10.1007/s11136-014-0821-6CrossRefPubMed Ediebah, D. E., Galindo-Garre, F., Uitdehaag, B. M. J., Ringash, J., Reijneveld, J. C., Dirven, L., Zikos, E., Coens, C., van den Bent, M. J., Bottomley, A., & Taphoorn, M. J. B. (2015). Joint modeling of longitudinal health-related quality of life data and survival. Quality of Life Research, 24, 795–804. https://​doi.​org/​10.​1007/​s11136-014-0821-6CrossRefPubMed
4.
go back to reference Rizopoulos, D. (2012). Joint models for longitudinal and time-to-event data. Chapman and Hall/CRC.CrossRef Rizopoulos, D. (2012). Joint models for longitudinal and time-to-event data. Chapman and Hall/CRC.CrossRef
5.
go back to reference Touraine, C., Cuer, B., Conroy, T., Beata, J., Gourgou, S., & Mollevi C. (2021). When a joint model should be preferred over a linear mixed model for analysis of longitudinal health-related quality of life data in cancer clinical trials. BMC Medical Research Methodology (under review). Touraine, C., Cuer, B., Conroy, T., Beata, J., Gourgou, S., & Mollevi C. (2021). When a joint model should be preferred over a linear mixed model for analysis of longitudinal health-related quality of life data in cancer clinical trials. BMC Medical Research Methodology (under review).
6.
go back to reference Conroy, T., Galais, M.-P., Raoul, J.-L., Bouché, O., Gourgou-Bourgade, S., Douillard, J.-Y., Etienne, P.-L., Boige, V., Martel-Lafay, I., Michel, P., Llacer-Moscardo, C., François, E., Créhange, G., Abdelghani, M. B., Juzyna, B., Bedenne, L., & Adenis, A. (2014). Definitive chemoradiotherapy with FOLFOX versus fluorouracil and cisplatin in patients with oesophageal cancer (PRODIGE5/ACCORD17): Final results of a randomised, phase 2/3 trial. The Lancet Oncology, 15, 305–314. https://doi.org/10.1016/S1470-2045(14)70028-2CrossRefPubMed Conroy, T., Galais, M.-P., Raoul, J.-L., Bouché, O., Gourgou-Bourgade, S., Douillard, J.-Y., Etienne, P.-L., Boige, V., Martel-Lafay, I., Michel, P., Llacer-Moscardo, C., François, E., Créhange, G., Abdelghani, M. B., Juzyna, B., Bedenne, L., & Adenis, A. (2014). Definitive chemoradiotherapy with FOLFOX versus fluorouracil and cisplatin in patients with oesophageal cancer (PRODIGE5/ACCORD17): Final results of a randomised, phase 2/3 trial. The Lancet Oncology, 15, 305–314. https://​doi.​org/​10.​1016/​S1470-2045(14)70028-2CrossRefPubMed
8.
go back to reference Aaronson, N. K., Ahmedzai, S., Bergman, B., Bullinger, M., Cull, A., Duez, N. J., Filiberti, A., Flechtner, H., Fleishman, S. B., Haes, J. C. J. M. D., Kaasa, S., Klee, M., Osoba, D., Razavi, D., Rofe, P. B., Schraub, S., Sneeuw, K., Sullivan, M., & Takeda, F. (1993). The European organization for research and treatment of cancer QLQ-C30: A quality-of-life instrument for use in international clinical trials in oncology. JNCI Journal of the National Cancer Institute, 85, 365–376. https://doi.org/10.1093/jnci/85.5.365CrossRefPubMed Aaronson, N. K., Ahmedzai, S., Bergman, B., Bullinger, M., Cull, A., Duez, N. J., Filiberti, A., Flechtner, H., Fleishman, S. B., Haes, J. C. J. M. D., Kaasa, S., Klee, M., Osoba, D., Razavi, D., Rofe, P. B., Schraub, S., Sneeuw, K., Sullivan, M., & Takeda, F. (1993). The European organization for research and treatment of cancer QLQ-C30: A quality-of-life instrument for use in international clinical trials in oncology. JNCI Journal of the National Cancer Institute, 85, 365–376. https://​doi.​org/​10.​1093/​jnci/​85.​5.​365CrossRefPubMed
9.
go back to reference Blazeby, J. M., Conroy, T., Hammerlid, E., Fayers, P., Sezer, O., Koller, M., Arraras, J., Bottomley, A., Vickery, C. W., Etienne, P. L., & Alderson, D. (2003). Clinical and psychometric validation of an EORTC questionnaire module, the EORTC QLQ-OES18, to assess quality of life in patients with oesophageal cancer. European Journal of Cancer, 39, 1384–1394. https://doi.org/10.1016/S0959-8049(03)00270-3CrossRefPubMed Blazeby, J. M., Conroy, T., Hammerlid, E., Fayers, P., Sezer, O., Koller, M., Arraras, J., Bottomley, A., Vickery, C. W., Etienne, P. L., & Alderson, D. (2003). Clinical and psychometric validation of an EORTC questionnaire module, the EORTC QLQ-OES18, to assess quality of life in patients with oesophageal cancer. European Journal of Cancer, 39, 1384–1394. https://​doi.​org/​10.​1016/​S0959-8049(03)00270-3CrossRefPubMed
10.
go back to reference James, G., Witten, D. M., Hastie, T., & Tibshirani, R. (2021). An introduction to statistical learning. Springer.CrossRef James, G., Witten, D. M., Hastie, T., & Tibshirani, R. (2021). An introduction to statistical learning. Springer.CrossRef
11.
go back to reference Klein, J. P., & Moeschberger, M. L. (2005). Survival analysis: Techniques for censored and truncated data, Second edition, corrected third printing. Springer. Klein, J. P., & Moeschberger, M. L. (2005). Survival analysis: Techniques for censored and truncated data, Second edition, corrected third printing. Springer.
13.
go back to reference Verbeke, G., & Molenberghs, G. (2001). Linear mixed models for longitudinal data Springer Series in Statistics. Springer. Verbeke, G., & Molenberghs, G. (2001). Linear mixed models for longitudinal data Springer Series in Statistics. Springer.
15.
go back to reference Fitzmaurice, G., Laird, N., & Ware, J. (2011). Applied longitudinal analysis. Wiley.CrossRef Fitzmaurice, G., Laird, N., & Ware, J. (2011). Applied longitudinal analysis. Wiley.CrossRef
17.
go back to reference Rizopoulos, D. (2010). JM: An R package for the joint modelling of longitudinal and time-to-event data. Journal of Statistical Software, 35, 1–33.CrossRef Rizopoulos, D. (2010). JM: An R package for the joint modelling of longitudinal and time-to-event data. Journal of Statistical Software, 35, 1–33.CrossRef
18.
go back to reference Harrell, F. E. (2015). Regression modeling strategies: With applications to linear models, logistic and ordinal regression, and survival analysis Springer Series in Statistics. Springer International Publishing.CrossRef Harrell, F. E. (2015). Regression modeling strategies: With applications to linear models, logistic and ordinal regression, and survival analysis Springer Series in Statistics. Springer International Publishing.CrossRef
19.
21.
Metagegevens
Titel
Flexible modeling of longitudinal health-related quality of life data accounting for informative dropout in a cancer clinical trial
Auteurs
Audrey Winter
Benjamin Cuer
Thierry Conroy
Beata Juzyna
Sophie Gourgou
Caroline Mollevi
Célia Touraine
Publicatiedatum
17-09-2022
Uitgeverij
Springer International Publishing
Gepubliceerd in
Quality of Life Research / Uitgave 3/2023
Print ISSN: 0962-9343
Elektronisch ISSN: 1573-2649
DOI
https://doi.org/10.1007/s11136-022-03252-6

Andere artikelen Uitgave 3/2023

Quality of Life Research 3/2023 Naar de uitgave