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Flexible Parametric Survival Analysis Using Stata:
Beyond the Cox Model
Patrick Royston & Paul C. Lambert


Table of contents


List of tables
List of figures
Preface
1 Introduction
  • 1.1 Goals
  • 1.2 A brief review of the Cox proportional hazards model
  • 1.3 Beyond the Cox model
    • 1.3.1 Estimating the baseline hazard
    • 1.3.2 The baseline hazard contains useful information
    • 1.3.3 Advantages of smooth survival functions
    • 1.3.4 Some requirements of a practical survival analysis
    • 1.3.5 When the proportional-hazards assumption is breached
  • 1.4 Why parametric models?
    • 1.4.1 Smooth baseline hazard and survival functions
    • 1.4.2 Time-dependent HR
    • 1.4.3 Modeling on different scales
    • 1.4.4 Relative survival
    • 1.4.5 Prediction out of sample
    • 1.4.6 Multiple time scales
  • 1.5 Why not standard parametric models?
  • 1.6 A brief introduction to stpm2
    • 1.6.1 Estimation (model fitting)
    • 1.6.2 Postestimation facilities (prediction)
  • 1.7 Basic relationships in survival analysis
  • 1.8 Comparing models
  • 1.9 The delta method
  • 1.10 Ado-file resources
  • 1.11 How our book is organized

2 Using stset and stsplit

  • 2.1 What is the stset command?
  • 2.2 Some key concepts
  • 2.3 Syntax of the stset command
  • 2.4 Variables created by the stset command
  • 2.5 Examples of using stset
    • 2.5.1 Standard survival data
    • 2.5.2 Using the scale( ) option
    • 2.5.3 Date of diagnosis and date of exit
    • 2.5.4 Date of diagnosis and date of exit with the scale( ) option
    • 2.5.5 Restricting the follow-up time
    • 2.5.6 Left-truncation
    • 2.5.7 Age as the time scale
  • 2.6 The stsplit command
    • 2.6.1 Time-dependent effects
    • 2.6.2 Time-varying covariates
  • 2.7 Conclusion

3 Graphical introduction to the principal datasets

  • 3.1 Introduction
  • 3.2 Rotterdam breast cancer data
  • 3.3 England and Wales breast cancer data
  • 3.4 Orchiectomy data
  • 3.5 Conclusion

4 Poisson models

  • 4.1 Introduction
  • 4.2 Modeling rates with the Poisson distribution
  • 4.3 Splitting the time scale
    • 4.3.1 The piecewise exponential model
    • 4.3.2 Time as just another covariate
  • 4.4 Collapsing the data to speed up computation
  • 4.5 Splitting at unique failure times
    • 4.5.1 Technical note: Why the Cox and Poisson approaches are equivalent
  • 4.6 Comparing a different number of intervals
  • 4.7 Fine splitting of the time scale
  • 4.8 Splines: Motivation and definition
    • 4.8.1 Calculating splines
    • 4.8.2 Restricted cubic splines
    • 4.8.3 Splines: Application to the Rotterdam data
    • 4.8.4 Varying the number of knots
    • 4.8.5 Varying the location of the knots
    • 4.8.6 Estimating the survival function
  • 4.9 FPs: Motivation and definition
    • 4.9.1 Application to Rotterdam data
    • 4.9.2 Higher order FP models
    • 4.9.3 FP function selection procedure
  • 4.10 Discussion

5 Royston–Parmar models

  • 5.1 Motivation and introduction
    • 5.1.1 The exponential distribution
    • 5.1.2 The Weibull distribution
    • 5.1.3 Generalizing the Weibull
    • 5.1.4 Estimating the hazard function
  • 5.2 Proportional hazards models
    • 5.2.1 Generalizing the Weibull
    • 5.2.2 Example
    • 5.2.3 Comparing parameters of PH(1) and Weibull models
  • 5.3 Selecting a spline function
    • 5.3.1 Knot positions
      Example
    • 5.3.2 How many knots?
  • 5.4 PO models
    • 5.4.1 Introduction
    • 5.4.2 The loglogistic model
    • 5.4.3 Generalizing the loglogistic model
    • 5.4.4 Comparing parameters of PO(1) and loglogistic models
      Example
  • 5.5 Probit models
    • 5.5.1 Motivation
    • 5.5.2 Generalizing the probit model
    • 5.5.3 Comparing parameters of probit(1) and lognormal models
    • 5.5.4 Comments on probit and POs models
  • 5.6 Royston–Parmar (RP) models
    • 5.6.1 Models with θ not equal to 0 or 1
    • 5.6.2 Example
    • 5.6.3 Likelihood function and parameter estimation
    • 5.6.4 Comparing regression coefficients
    • 5.6.5 Model selection
    • 5.6.6 Sensitivity to number of knots
    • 5.6.7 Sensitivity to location of knots
  • 5.7 Concluding remarks

6 Prognostic models

  • 6.1 Introduction
  • 6.2 Developing and reporting a prognostic model
  • 6.3 What does the baseline hazard function mean?
    • 6.3.1 Example
  • 6.4 Model selection
    • 6.4.1 Choice of scale and baseline complexity
      Example
    • 6.4.2 Selection of variables and functional forms
      Example
  • 6.5 Quantitative outputs from the model
    • 6.5.1 Survival probabilities for individuals
    • 6.5.2 Survival probabilities across the risk spectrum
    • 6.5.3 Survival probabilities at given covariate values
    • 6.5.4 Survival probabilities in groups
    • 6.5.5 Plotting adjusted survival curves
    • 6.5.6 Plotting differences between survival curves
    • 6.5.7 Centiles of the survival distribution
  • 6.6 Goodness of fit
    • 6.6.1 Example
  • 6.7 Discrimination and explained variation
    • 6.7.1 Example
    • 6.7.2 Harrell’s C index of concordance
  • 6.8 Out-of-sample prediction: Concept and applications
    • 6.8.1 Extrapolation of survival functions: Basic technique
    • 6.8.2 Extrapolation of survival functions: Further investigations
    • 6.8.3 Validation of prognostic models: Basics
    • 6.8.4 Validation of prognostic models: Further comments
  • 6.9 Visualization of survival times
    • 6.9.1 Example
  • 6.10 Discussion

7 Time-dependent effects

  • 7.1 Introduction
  • 7.2 Definitions
  • 7.3 What do we mean by a TD effect?
  • 7.4 Proportional on which scale?
  • 7.5 Poisson models with TD effects
    • 7.5.1 Piecewise models
    • 7.5.2 Using restricted cubic splines
  • 7.6 RP models with TD effects
    • 7.6.1 Piecewise HRs
    • 7.6.2 Continuous TD effects
    • 7.6.3 More than one TD effect
    • 7.6.4 Stratification is the same as including TD effects
  • 7.7 TD effects for continuous variables
  • 7.8 Attained age as the time scale
    • 7.8.1 The orchiectomy data
    • 7.8.2 Proportional hazards model
    • 7.8.3 TD model
  • 7.9 Multiple time scales
  • 7.10 Prognostic models with TD effects
    • 7.10.1 Example
  • 7.11 Discussion

8 Relative survival

  • 8.1 Introduction
  • 8.2 What is relative survival?
  • 8.3 Excess mortality and relative survival
    • 8.3.1 Excess mortality
    • 8.3.2 Relative survival is a ratio
  • 8.4 Motivating example
  • 8.5 Life-table estimation of relative survival
    • 8.5.1 Using strs
  • 8.6 Poisson models for relative survival
    • 8.6.1 Piecewise models
    • 8.6.2 Restricted cubic splines
  • 8.7 RP models for relative survival
    • 8.7.1 Likelihood for relative survival models
    • 8.7.2 Proportional cumulative excess hazards
    • 8.7.3 RP models on other scales
    • 8.7.4 Application to England and Wales breast cancer data
    • 8.7.5 Relative survival models on other scales
    • 8.7.6 Time-dependent effects
  • 8.8 Some comments on model selection
  • 8.9 Age as a continuous variable
  • 8.10 Concluding remarks

9 Further topics

  • 9.1 Introduction
  • 9.2 Number needed to treat
    • 9.2.1 Example
  • 9.3 Average and adjusted survival curves
    • 9.3.1 Renal data
  • 9.4 Modeling distributions with RP models
    • 9.4.1 Example 1: Rotterdam breast cancer data
    • 9.4.2 Example 2: CD4 lymphocyte data
    • 9.4.3 Example 3: Prostate cancer data
  • 9.5 Multiple events
    • 9.5.1 Introduction
    • 9.5.2 The AG model
    • 9.5.3 The WLW model
    • 9.5.4 The PWP model
    • 9.5.5 Multiple events in RP models
    • 9.5.6 Summary
  • 9.6 Bayesian RP models
    • 9.6.1 Introduction
    • 9.6.2 The “zeros trick” in WinBUGS
    • 9.6.3 Fitting a RP model
    • 9.6.4 Summary
  • 9.7 Competing risks
    • 9.7.1 Summary
  • 9.8 Period analysis
    • 9.8.1 Introduction
    • 9.8.2 What is period analysis?
    • 9.8.3 Application to England and Wales breast cancer data
  • 9.9 Crude probability of death from relative survival models
    • 9.9.1 Introduction
    • 9.9.2 Application to England and Wales breast cancer data
    • 9.9.3 Conclusion
  • 9.10 Final remarks
References
Author index
Subject index