Imaging and clinical end points in brain metastases trials

摘要

When designing a clinical trial on brain metastases the choice of end points is critical. The Response Assessment in Neuro-Oncology (RANO) International Group has recently critically reviewed the different end points used in the clinical trials on brain metastases [1,2].Some factors heavily influence the choice of end points. First, patients with solid tumors may differ in prognosis and competing risk of extracranial progression. Second, the trial setting (Phase II vs Phase III trials) and type of intervention (CNS-directed vs systemic therapies) also require different end points.The knowledge of prognostic factors, in other words, of those factors influencing the outcome regardless of treatments, is critical in order to identify subgroups of patients with different outcomes (e.g., Recursive Partitioning Analysis [RPA] and Graded Prognostic Assessment [GPA] classes) . In clinical trials, these subgroups are essential either as a stratification factor or inclusion criteria or even for post hoc analyses.