Clinical trial design for systemic agents in patients with brain metastases from solid tumours: a guideline by the Response Assessment in Neuro-Oncology Brain Metastases working group

被引：82

作者：

Camidge, D. Ross ^{[1
]}

Lee, Eudocia Q. ^{[2
]}

Lin, Nancy U. ^{[3
]}

Margolin, Kim ^{[5
]}

Ahluwalia, Manmeet S. ^{[6
]}

Bendszus, Martin ^{[7
]}

Chang, Susan M. ^{[8
]}

Dancey, Janet ^{[9
]}

de Vries, Elisabeth G. E. ^{[10
]}

Harris, Gordon J. ^{[11
]}

Hodi, F. Stephen ^{[3
,4
]}

Lassman, Andrew B. ^{[12
,13
]}

Macdonald, David R. ^{[14
,15
]}

Peereboom, David M. ^{[6
]}

Schiff, David ^{[16
]}

Soffietti, Ricardo ^{[17
]}

van den Bent, Martin J. ^{[18
]}

Wefel, Jeffrey S. ^{[19
]}

Wen, Patrick Y. ^{[2
]}

机构：

[1] Univ Colorado, Anschutz Med Campus, Aurora, CO 80045 USA

[2] Harvard Med Sch, Dana Farber Canc Inst, Ctr Neurooncol, Boston, MA USA

[3] Harvard Med Sch, Dana Farber Canc Inst, Dept Med Oncol, Boston, MA USA

[4] Harvard Med Sch, Dana Farber Canc Inst, Dept Med, Boston, MA USA

[5] City Hope Natl Med Ctr, Dept Med Oncol, 1500 E Duarte Rd, Duarte, CA 91010 USA

[6] Cleveland Clin, Rose Ella Burkhardt Brain Tumor & Neurooncol Ctr, Solid Tumor Oncol, Cleveland, OH 44106 USA

[7] Heidelberg Univ, Dept Neuroradiol, Heidelberg, Germany

[8] Univ Calif San Francisco, Dept Neurosurg, San Francisco, CA USA

[9] Queens Univ, Dept Oncol, Kingston, ON, Canada

[10] Univ Groningen, Univ Med Ctr Groningen, Dept Med Oncol, Groningen, Netherlands

[11] Harvard Med Sch, Massachusetts Gen Hosp, Dept Radiol, Imaging Lab 3D, Boston, MA USA

[12] Columbia Univ, Dept Neurol, Med Ctr, New York, NY 10027 USA

[13] Columbia Univ, Herbert Irving Comprehens Canc Ctr, Med Ctr, New York, NY 10027 USA

[14] Western Univ, Dept Oncol, Schulich Sch Med & Dent, London, ON, Canada

[15] Western Univ, Dept Clin Neurol Sci, Schulich Sch Med & Dent, London, ON, Canada

[16] Univ Virginia, Div Neurooncol, Charlottesville, VA USA

[17] Univ Turin, Dept Neurol Neurooncol, Turin, Italy

[18] Erasmus Univ, Brain Tumor Inst, Med Ctr, Rotterdam, Netherlands

[19] Univ Texas MD Anderson Canc Ctr, Dept Neurooncol, Houston, TX 77030 USA

来源：

LANCET ONCOLOGY | 2018年 / 19卷 / 01期

关键词：

CELL LUNG-CANCER; HER2-POSITIVE BREAST-CANCER; LAPATINIB PLUS CAPECITABINE; TRASTUZUMAB EMTANSINE T-DM1; OLIGOPROGRESSIVE DISEASE; RADIATION-THERAPY; OPEN-LABEL; PHASE-II; MELANOMA; CRITERIA;

D O I：

10.1016/S1470-2045(17)30693-9

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 [肿瘤学];

摘要：

Patients with active CNS disease are often excluded from clinical trials, and data regarding the CNS efficacy of systemic agents are usually obtained late in the drug development process or not at all. In this guideline from the Response Assessment in Neuro-Oncology Brain Metastases (RANO-BM) working group, we provide detailed recommendations on when patients with brain metastases from solid tumours should be included or excluded in clinical trials of systemic agents. We also discuss the limitations of retrospective studies in determining the CNS efficacy of systemic drugs. Inclusion of patients with brain metastases early on in the clinical development of a drug or a regimen is needed to generate appropriate CNS efficacy or non-efficacy signals. We consider how to optimally incorporate or exclude such patients in systemic therapy trials depending on the likelihood of CNS activity of the agent by considering three scenarios: drugs that are considered very unlikely to have CNS antitumour activity or efficacy; drugs that are considered very likely to have CNS activity or efficacy; and drugs with minimal baseline information on CNS activity or efficacy. We also address trial design issues unique to patients with brain metastases, including the selection of appropriate CNS endpoints in systemic therapy trials.

引用

页码：E20 / E32

页数：13

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