RESUMEN
In traditional survival analyses, hazard ratio (HR) is commonly used to evaluate treatment effects. However, HR may not be easy to interpret. Restricted mean survival time is a viable alternative to HR, particularly when the proportional hazards assumption is not satisfied. We developed a conditional restricted mean survival time (CRMST) estimator for a time interval of interest using counting process. The variance of CRMST was estimated using a perturbation re-sampling method for asymptotic normality. The utility of our CRMST seems promising based on comprehensive simulation studies and a real data case study.
Asunto(s)
Enfermedades Cardiovasculares/tratamiento farmacológico , Enfermedades Cardiovasculares/mortalidad , Ensayos Clínicos Controlados Aleatorios como Asunto/estadística & datos numéricos , Método Doble Ciego , Estudios de Seguimiento , Humanos , Inhibidores de Agregación Plaquetaria/uso terapéutico , Modelos de Riesgos Proporcionales , Ensayos Clínicos Controlados Aleatorios como Asunto/métodos , Procesos Estocásticos , Tasa de Supervivencia/tendenciasRESUMEN
In pulmonary hypertension, as in many other diseases, there is a need for a smarter approach to evaluating new treatments. The traditional randomized controlled trial has served medical science well, but constrains the development of treatments for rare diseases. A workshop was established to consider alternative clinical trial designs in pulmonary hypertension and here discusses their merits, limitations and challenges to implementation of novel approaches.