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18-08-2020

Longitudinal analysis of health-related quality of life in cancer clinical trials: methods and interpretation of results

Auteurs: Caroline Bascoul-Mollevi, Antoine Barbieri, Céline Bourgier, Thierry Conroy, Bruno Chauffert, Mohamed Hebbar, William Jacot, Beata Juzyna, Hélène De Forges, Sophie Gourgou, Franck Bonnetain, Célia Touraine, Amélie Anota

Gepubliceerd in: Quality of Life Research | Uitgave 1/2021

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Abstract

Purpose

Health-related quality of life (HRQoL) is assessed by self-administered questionnaires throughout the care process. Classically, two longitudinal statistical approaches were mainly used to study HRQoL: linear mixed models (LMM) or time-to-event models for time to deterioration/time until definitive deterioration (TTD/TUDD). Recently, an alternative strategy based on generalized linear mixed models for categorical data has also been proposed: the longitudinal partial credit model (LPCM). The objective of this article is to evaluate these methods and to propose recommendations to standardize longitudinal analysis of HRQoL data in cancer clinical trials.

Methods

The three methods are first described and compared through statistical, methodological, and practical arguments, then applied on real HRQoL data from clinical cancer trials or published prospective databases. In total, seven French studies from a collaborating group were selected with longitudinal collection of QLQ-C30. Longitudinal analyses were performed with the three approaches using SAS, Stata and R software.

Results

We observed concordant results between LMM and LPCM. However, discordant results were observed when we considered the TTD/TUDD approach compared to the two previous methods. According to methodological and practical arguments discussed, the approaches seem to provide additional information and complementary interpretations. LMM and LPCM are the most powerful methods on simulated data, while the TTD/TUDD approach gives more clinically understandable results. Finally, for single-item scales, LPCM is more appropriate.

Conclusion

These results pledge for the recommendation to use of both the LMM and TTD/TUDD longitudinal methods, except for single-item scales, establishing them as the consensual methods for publications reporting HRQoL.
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Metagegevens
Titel
Longitudinal analysis of health-related quality of life in cancer clinical trials: methods and interpretation of results
Auteurs
Caroline Bascoul-Mollevi
Antoine Barbieri
Céline Bourgier
Thierry Conroy
Bruno Chauffert
Mohamed Hebbar
William Jacot
Beata Juzyna
Hélène De Forges
Sophie Gourgou
Franck Bonnetain
Célia Touraine
Amélie Anota
Publicatiedatum
18-08-2020
Uitgeverij
Springer International Publishing
Gepubliceerd in
Quality of Life Research / Uitgave 1/2021
Print ISSN: 0962-9343
Elektronisch ISSN: 1573-2649
DOI
https://doi.org/10.1007/s11136-020-02605-3

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