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Clinical Trials: Endpoints and Outcome Assessment

  • Nancy U. LinEmail author
Chapter

Abstract

Central nervous system (CNS) metastases are the most common cause of malignant brain tumors in adults. Historically, clinical trials in oncology have only rarely included patients with active CNS metastases, thus leaving gaps in the development of standard methodologies by which to assess trial endpoints. Not only have trial endpoints been variably assessed and defined, but the most clinically relevant outcomes may differ by type of trial. This chapter (1) provides recommendations for more active inclusion of patients with brain metastases into prospective clinical trials, (2) summarizes measures that could be considered as primary or secondary trial endpoints, (3) reviews efforts to develop more standard definitions of trial endpoints, and (4) discusses the selection of trial endpoints most appropriate for the given target population and proposed intervention.

Keywords

Brain metastases Leptomeningeal metastases Clinical trials Endpoints Outcome assessment 

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Copyright information

© Springer Nature Switzerland AG 2020

Authors and Affiliations

  1. 1.Division of Breast Oncology, Dana-Farber Cancer InstituteHarvard Medical SchoolBostonUSA

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