Challenges relating to solid tumour brain metastases in clinical trials, part 1: patient population, response, and progression. A report from the RANO group

Nancy U. Lin*, Eudocia Q. Lee, Hidefumi Aoyama, Igor J. Barani, Brigitta G. Baumert, Paul D. Brown, D. Ross Camidge, Susan M. Chang, Janet Dancey, Laurie E. Gaspar, Gordon J. Harris, F. Stephen Hodi, Steven N. Kalkanis, Kathleen R. Lambom, Mark E. Linskey, David R. Macdonald, Kim Margolin, Minesh P. Mehta, David Schiff, Riccardo SoffiettiJohn H. Suh, Martin J. van den Bent, Michael A. Vogelbaum, Jeffrey S. Wefel, Patrick Y. Wen

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review


Therapeutic outcomes for patients with brain metastases need to improve. A critical review of trials specifically addressing brain metastases shows key issues that could prevent acceptance of results by regulatory agencies, including enrolment of heterogeneous groups of patients and varying definitions of clinical endpoints. Considerations specific to disease, modality, and treatment are not consistently addressed. Additionally, the schedule of CNS imaging and consequences of detection of new or progressive brain metastases in trials mainly exploring the extra-CNS activity of systemic drugs are highly variable. The Response Assessment in Neuro-Oncology (RANO) working group is an independent, international, collaborative effort to improve the design of trials in patients with brain tumours. In this two-part series, we review the state of clinical trials of brain metastases and suggest a consensus recommendation for the development of criteria for future clinical trials.
Original languageEnglish
Pages (from-to)E396-E406
JournalLancet oncology
Issue number10
Publication statusPublished - Sept 2013

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