Semi-parametric modelling of the distribution of the baseline risk in meta-analysis

Stat Med. 2007 Dec 30;26(30):5434-44. doi: 10.1002/sim.3066.

Abstract

In meta-analysis of clinical trials, often meta-regression analyses are performed to explain the heterogeneity in treatment effects that usually exist between trials. A popular explanatory variable is the risk observed in the control group, the baseline risk. The relationship between the treatment effect and the baseline risk is investigated by fitting a linear model that allows randomness on the true baseline risk by assuming a normal distribution with unknown mean and variance. However, the normality assumption could be too strong to adequately describe the underlying distribution. Therefore, we developed a new semi-parametric method that relaxes the normality assumption to a more flexible and general distribution. We applied a penalized Gaussian mixture distribution to represent the baseline risk distribution. Furthermore, a bivariate hierarchical model is formulated in order to take into account the correlation between the baseline and treatment effect. To fit the proposed model, a penalized likelihood function is maximized by an Expectation Maximization (EM) algorithm. We illustrate our method on a number of simulated data sets and on a published meta-analysis data set.

MeSH terms

  • Anticholesteremic Agents / therapeutic use
  • Clinical Trials as Topic / statistics & numerical data
  • Coronary Disease / drug therapy
  • Coronary Disease / mortality
  • Effect Modifier, Epidemiologic
  • Humans
  • Likelihood Functions
  • Linear Models
  • Longitudinal Studies
  • Meta-Analysis as Topic*
  • Reference Values
  • Risk*
  • Statistical Distributions*
  • Statistics, Nonparametric
  • Stochastic Processes
  • Survival Analysis
  • Treatment Outcome*

Substances

  • Anticholesteremic Agents