Statistical Methods in HCT and Cellular Therapies

The analysis of data describing the outcomes of patients who have received an HCT is not only fundamental to assessing the effectiveness of the treatment but can provide invaluable information on the prognostic role of disease and patient factors. Thus, the appropriate analysis and understanding of such data are of paramount importance. This document provides an overview of the main and well-established statistical methods, as well as a brief introduction of more novel techniques. More insight is provided in the EBMT Statistical Guidelines (Iacobelli 2013). Additionally, the paper by de Wreede et al. (2022) explains the most important concepts and related pitfalls in the analysis of HCT studies.