Multistate models on pleural effusion after allogeneic hematopoietic stem cell transplantation

Abstract:

A multistate model is more complicated than competing risk models and is composed of a finite number of states and transitions between states. Unlike competing risk models, this model has the ability to assess the effect of occurrence order of time-to-event data. Pleural effusion (PE) is a severe complication that often occurs after allogeneic hematopoietic stem cell transplantation (HSCT). Many patients develop PE during the first 100 days after allogeneic HSCT, and graft-versus-host disease (GVHD) occurs either before or after the development of PE, implying that the occurrence order of PE and GVHD (i.e., PE after GVHD vs. GVHD after PE) would influence on the incidence, risk factors, and mortality of PE. One can use either Cox proportional models or competing risk models to evaluate these values, but neither method is able to incorporate the occurrence order of incidence into the model. To resolve this difficulty, we developed a multistate model describing several possible events and event-related dependencies and applied to a retrospective study of 606 patients, including eight covariates.

Keywords::

• Cox proportional model
• hematopoietic stem cell transplantation
• multistate model
• pleural effusion
• survival analysis

Acknowledgments

This work has been partially supported by National Science Foundation grant DMS-1312603. The Biostatistics Core is supported in part by National Institute of Health Cancer Center Support Grant P30 CA022453 to the Karmanos Cancer Institute at Wayne State University.

Part of this work has been presented at the 2016 Joint Statistical Meetings (JSM) in Chicago, IL, USA, on August 1, 2016, as a poster presentation.

Disclosure

The authors report no conflicts of interest in this work.

Supplementary materials

Table S1 Predicted transition probabilities at time (a) 0 year, (b) 0.5 year, (c) 1 year, and (d) 1.5 years after the transplantation

Table S2 Predicted transition probabilities at time (a) 0 year, (b) 0.5 years, (c) 1 year, and (d) 1.5 years after the transplantation (starting state is PE)

Table S3 Predicted transition probabilities at time (a) 0 year, (b) 0.5 year, (c) 1 year, and (d) 1.5 years after the transplantation (starting state is GVHD)