Thesis BFJ Goudsmit
Preface
1
General introduction
Research in context
A short history of liver transplantation
The start of liver allocation
Survival prediction
Measuring model prediction performance
The MELD score
Justifying why research is needed: central argument
Thesis layout: research questions and addressed problems
Considering sodium
Updating coefficients
Past and current disease
Acute-on-chronic liver disease
Benefit of transplantation: life years gained
References
Part I: Forms of MELD
2
Validation of the Model for End‐stage Liver Disease sodium (MELD‐Na) score in the Eurotransplant region
Introduction
Methods
Study design and population
Statistical analysis
Results
Study population
MELD-Na performance
Impact on the waiting list
Discussion
Conclusion
References
3
Refitting the Model for End-stage Liver Disease for the Eurotransplant region
Introduction
Methods
Patient data
Statistical methods
Results
Model development
Model performance
Impact on the waiting list
Discussion
Lower bounds
Upper bounds
Sodium addition
Impact on the WL
Limitations
Conclusion
References
Part II: Disease over time
4
Joint modelling of liver transplant candidates outperforms the model for end-stage liver disease: the effect of disease development over time on patient outcome
Introduction
Methods
Study population
Statistical analysis
Longitudinal analysis
Combining longitudinal and survival analysis
Prediction performance
Impact on the waiting list
Online LT-JM prediction tool
Results
Population characteristics
JM properties
JM performance
JM impact on the waiting list
Online prediction tools
Discussion
Longitudinal analysis
Prediction performance
Impact on the waiting list
Limitations
Conclusion
References
5
Development and validation of a dynamic survival prediction model for patients with acute-on-chronic liver failure
Introduction
Methods
Study population
Identification of ACLF
Development of the ACLF-JM
Validation of the ACLF-JM
ACLF-JM impact on the transplantation waiting list
Clinical application of the ACLF-JM
Results
Study population
Model properties
Model validation
ACLF-JM impact on the transplantation waiting list
Clinical application of the ACLF-JM
Discussion
Disease development over time
ACLF-JM validation
Clinical application of the ACLF-JM
Limitations
Conclusion
References
Part III: Survival with and without transplantation
6
Survival benefit from liver transplantation for patients with and without hepatocellular carcinoma
Introduction
Methods
Patient population
Benefit definition
Statistical analysis
Online benefit score calculator
Results
Patient characteristics at transplantation
Waiting list survival model
Post-transplantation survival model
Survival without and with LT
Survival benefit: life-years gained per 5 years
Liver transplant benefit scores
Discussion
Benefit definition
Estimation of benefit
Non-HCC and HCC benefit
Using benefit scores
Limitations
Conclusion
References
Part IV: Summary, general discussion, and future perspectives
7
Summary
8
General discussion
Part I: Forms of MELD
MELD 3.0
Part II: Disease over time
Joint modeling disease and survival over time
Joint modeling acute-on-chronic liver failure
Part III: Survival with and without transplantation
Benefit from liver transplantation
9
Future perspectives
Simulation
New model implementation
From urgency to benefit
Conclusion
References
10
Appendix
10.1
Supplement Chapter: The Model for End-stage Liver Disease 3.0: an update without proven accuracy
Letter
References
10.2
Supplement Chapter: The role of the model for end-stage liver disease-sodium score and joint models for 90-day mortality prediction in patients with acute-on-chronic liver failure.
Letter
References
11
Nederlandse samenvatting (Summary in Dutch)
Verbetering van voorspellingsmodellen voor levertransplantatiekandidaten
Deel I
Deel II
Deel III
Dankwoord
Curriculum vitae
List of publications
Published with bookdown
Improving survival prediction models for liver transplantation candidates
Part I: Forms of MELD
“All models are wrong, but some are useful.”
— George Box