Need for consensus on primary end points and efficacy definitions in trials for adult acute lymphoblastic leukemia.

Abstract: The lack of consensus on acceptable primary end points and definitions of response and survival in phase 2/3 efficacy studies for adult acute lymphoblastic leukemia has led to widely different clinical trial designs. Inconsistency in primary end point selection and lack of consensus on response, survival end points, and adequate follow-up time lead to difficulty in interpreting completed studies and developing future trials. The lack of consensus also runs the risk of integrating ineffective or unacceptably toxic regimens into clinical practice and future trials. Increasingly, studies integrating highly active, targeted agents into chemotherapy use short-term end points of response, measurable residual disease-negative response, and early event-free survival without confidence that these end points will translate into improved late patient outcomes. This article highlights the current consequences and dilemmas caused by this lack of consensus. The hope is to stimulate discussion and ultimately consensus to improve the interpretation and application of clinical trial results.