Modelling the distribution of health-related quality of life of advanced melanoma patients in a longitudinal multi-centre clinical trial using M-quantile random effects regression

Stat Methods Med Res. 2018 Feb;27(2):549-563. doi: 10.1177/0962280216636651. Epub 2016 Mar 17.

Abstract

Health-related quality of life assessment is important in the clinical evaluation of patients with metastatic disease that may offer useful information in understanding the clinical effectiveness of a treatment. To assess if a set of explicative variables impacts on the health-related quality of life, regression models are routinely adopted. However, the interest of researchers may be focussed on modelling other parts (e.g. quantiles) of this conditional distribution. In this paper, we present an approach based on quantile and M-quantile regression to achieve this goal. We applied the methodologies to a prospective, randomized, multi-centre clinical trial. In order to take into account the hierarchical nature of the data we extended the M-quantile regression model to a three-level random effects specification and estimated it by maximum likelihood.

Keywords: Hierarchical data; Phase III study; Rotterdam Symptom Checklist; quantile regression; robust estimation.

Publication types

  • Clinical Trial, Phase III
  • Multicenter Study
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use
  • Biostatistics
  • Humans
  • Longitudinal Studies
  • Melanoma / drug therapy*
  • Models, Statistical
  • Prospective Studies
  • Quality of Life*
  • Regression Analysis
  • Self Report