Definitions, methodological, and statistical issues for phase III clinical trials in chronic myeloid leukemia: a proposal by the European LeukemiaNet
Cet article résume les recommandations d'un groupe d'experts européens en matière d'essais cliniques de phase III pour le traitement d'une leucémie myéloïde chronique
The treatment policy of chronic myeloid leukemia (CML), particularly with tyrosine kinase inhibitors (TKIs), has been influenced by several recent studies that were well designed and rapidly performed, but their interpretation is of some concern because different endpoints and methodologies were used. In order to understand and compare the results of the previous and future studies and to translate their conclusion into clinical practice, there is a need for common definitions and methods for analyses of CML studies. A panel of experts was appointed by the European LeukemiaNet with the aim of developing a set of definitions and recommendations to be used in design, analyses and reporting of phase III clinical trials in this disease. This paper summarizes the consensus of the panel on events and major endpoints of interest in CML. It also focuses on specific issues concerning the intention to treat principle, and longitudinal data analyses in the context of long term follow-up. The panel proposes that future clinical trials follow these recommendations.
Blood , résumé, 2012