Statistics Seminar - 04/09/24

Abstract: Allogeneic hematopoietic cell transplantation (alloHCT) is frequently considered a curative therapy for high-risk hematologic malignancies. However, chronic Graft-versus-Host Disease (cGVHD) is a serious complication following alloHCT, and traditional endpoints in oncology such as overall survival and disease-free survival do not typically account for cGVHD. Using a recently completed pediatric study of alloHCT as a motivating example, this project studies how to evaluate success while accounting for cGVHD status. We find that a popular method, CGRFS (Current Graft-versus-Host Disease-Free Relapse-Free Survival), can have challenging interpretations in the small sample sizes consistent with pediatric studies of alloHCT. CGRFS arises from a tradition of methods based on multi-state modeling. By proposing an alternative multi-state model with differing biological assumptions, we introduce an alternative method, AERFS (Adverse Event-Free Relapse-Free Survival), that we believe will address limitations of CGRFS.