Background: Risk-based analyses are increasingly popular for understanding heterogeneous treatment effects (HTE) in clinical trials. For time-to-event analyses, the assumption that high-risk patients benefit most on the clinically important absolute scale when hazard ratios (HRs) are constant across risk strata might not hold. Absolute treatment effects can be measured as either the risk difference (RD) at a given time point or the difference in restricted mean survival time (ΔRMST) which aligns more closely with utilitarian medical decision-making frameworks.
View Article and Find Full Text PDFBackground: Healthcare use by High-Need High-Cost (HNHC) patients is believed to be modifiable through better coordination of care. To identify patients for care management, a hybrid approach is recommended that combines clinical assessment of need with model-based prediction of cost. Models that predict high healthcare costs persisting over time are relevant but scarce.
View Article and Find Full Text PDFFirst-line treatment for advanced-stage diffuse large B-cell lymphoma (DLBCL) typically involves 6x R-CHOP21 or 6x R-CHOP21 with two additional rituximab administrations (6x R-CHOP21 + 2 R). In contemporary practice, this treatment choice might be guided by interim PET scan results. This nationwide, population-based study investigates the comparative effectiveness of these treatment regimens in an era where interim PET-guided treatment decisions were not standard practice.
View Article and Find Full Text PDFBackground: Prediction models are often externally validated with data from a single study or cohort. However, the interpretation of performance estimates obtained with single-study external validation is not as straightforward as assumed. We aimed to illustrate this by conducting a large number of external validations of a prediction model for functional outcome in subarachnoid hemorrhage (SAH) patients.
View Article and Find Full Text PDFBackground: Factor (F)XII triggers contact activation by binding to foreign surfaces, with the epidermal growth factor-like 1 (EGF-1) domain being the primary binding site. Blocking FXII surface-binding might hold therapeutic value to prevent medical device-induced thrombosis.
Objectives: To unravel and prevent EGF-1-mediated FXII surface-binding with a variable domain of heavy chain-only antibody (VH).