Statistical methods for the analysis and presentation of the results of bone marrow transplants. Part I: Unadjusted analysis

Abstract

In this paper, we describe modern statistical methods for presentation of the results of studies of bone marrow transplantation. We focus here on 'univariate' or unadjusted techniques to describe the outcomes of such studies. In another paper we will discuss multivariate methods. We discuss the type of data one may have available to make inference about outcomes. We explain the differences between the Kaplan-Meier estimator of the survival function and the cumulative incidence curve, how these curves should be interpreted and when each is the appropriate summary statistic. We discuss the weighted log rank statistic and show how different weights can be used to put emphasis on detecting differences between groups in different time periods. We also present a simple estimate of current leukemia-free survival which is useful in summarizing post-transplant events.