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Clinical Trials: Endpoints and Outcome Assessment

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Central Nervous System Metastases

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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.

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Authors and Affiliations

  1. Division of Breast Oncology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA, USA

    Nancy U. Lin

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  1. Nancy U. Lin
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Correspondence to Nancy U. Lin .

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Editors and Affiliations

  1. Brain Tumor and Neuro-Oncology Center, Cleveland Clinic, Cleveland, OH, USA

    Manmeet Ahluwalia

  2. Department of Neurosurgery and Neuro-Oncology, Clairval Hospital Center, Ramsay-Générale de Santé, Marseille, France, Neurophysiopathology Institute, Aix-Marseille Université, Marseille, France

    Philippe Metellus

  3. Department of Neuro-Oncology, University and City of Health and Science Hospital, Turin, Italy

    Riccardo Soffietti

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Lin, N.U. (2020). Clinical Trials: Endpoints and Outcome Assessment. In: Ahluwalia, M., Metellus, P., Soffietti, R. (eds) Central Nervous System Metastases. Springer, Cham. https://doi.org/10.1007/978-3-030-23417-1_32

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  • DOI: https://doi.org/10.1007/978-3-030-23417-1_32

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