title: Challenges relating to solid tumour brain metastases in clinical trials, part 1: patient population, response, and progression. A report from the RANO group.
abstract: 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.
authors.affiliation: Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA, USA. Electronic address: nlin@partners.org.
authors.forename: Nancy U
authors.initials: NU
authors.lastname: Lin
authors.forename: Eudocia Q
authors.initials: EQ
authors.lastname: Lee
authors.forename: Hidefumi
authors.initials: H
authors.lastname: Aoyama
authors.forename: Igor J
authors.initials: IJ
authors.lastname: Barani
authors.forename: Brigitta G
authors.initials: BG
authors.lastname: Baumert
authors.forename: Paul D
authors.initials: PD
authors.lastname: Brown
authors.forename: D Ross
authors.initials: DR
authors.lastname: Camidge
authors.forename: Susan M
authors.initials: SM
authors.lastname: Chang
authors.forename: Janet
authors.initials: J
authors.lastname: Dancey
authors.forename: Laurie E
authors.initials: LE
authors.lastname: Gaspar
authors.forename: Gordon J
authors.initials: GJ
authors.lastname: Harris
authors.forename: F Stephen
authors.initials: FS
authors.lastname: Hodi
authors.forename: Steven N
authors.initials: SN
authors.lastname: Kalkanis
authors.forename: Kathleen R
authors.initials: KR
authors.lastname: Lamborn
authors.forename: Mark E
authors.initials: ME
authors.lastname: Linskey
authors.forename: David R
authors.initials: DR
authors.lastname: Macdonald
authors.forename: Kim
authors.initials: K
authors.lastname: Margolin
authors.forename: Minesh P
authors.initials: MP
authors.lastname: Mehta
authors.forename: David
authors.initials: D
authors.lastname: Schiff
authors.forename: Riccardo
authors.initials: R
authors.lastname: Soffietti
authors.forename: John H
authors.initials: JH
authors.lastname: Suh
authors.forename: Martin J
authors.initials: MJ
authors.lastname: van den Bent
authors.forename: Michael A
authors.initials: MA
authors.lastname: Vogelbaum
authors.forename: Jeffrey S
authors.initials: JS
authors.lastname: Wefel
authors.forename: Patrick Y
authors.initials: PY
authors.lastname: Wen
mesh_terms: D001932:Brain Neoplasms / Q000556:secondary* / Q000628:therapy; D002986:Clinical Trials as Topic; D018572:Disease-Free Survival; D006801:Humans; D008279:Magnetic Resonance Imaging