Clinical trial design for local therapies for brain metastases: a guideline by the Response Assessment in Neuro-Oncology Brain Metastases working group

被引：46

作者：

Alexander, Brian M. ^{[1
,17
]}

Brown, Paul D. ^{[2
]}

Ahluwalia, Manmeet S. ^{[3
]}

Aoyama, Hidefumi ^{[5
]}

Baumert, Brigitta G. ^{[6
,7
]}

Chang, Susan M. ^{[8
]}

Gaspar, Laurie E. ^{[9
]}

Kalkanis, Steven N. ^{[10
]}

Macdonald, David R. ^{[11
]}

Mehta, Minesh P. ^{[12
]}

Soffietti, Riccardo ^{[13
,14
]}

Suh, John H. ^{[4
]}

van den Bent, Martin J. ^{[15
]}

Vogelbaum, Michael A. ^{[3
]}

Wefel, Jeffrey S. ^{[16
]}

Lee, Eudocia Q. ^{[18
]}

Wen, Patrick Y. ^{[18
]}

机构：

[1] Harvard Med Sch, Harvard Program Therapeut Sci, Boston, MA 02115 USA

[2] Mayo Clin, Dept Radiat Oncol, Rochester, MN USA

[3] Cleveland Clin, Rose Ella Burkhardt Brain Tumor & Neuro Oncol Ctr, Cleveland, OH 44106 USA

[4] Cleveland Clin, Dept Radiat Oncol, Cleveland, OH 44106 USA

[5] Niigata Univ, Dept Radiol & Radiat Oncol, Grad Sch Med & Dent Sci, Niigata, Japan

[6] Univ Munster, Dept Radiat Oncol, Paracelsus Clin Osnabruck, Munster, Germany

[7] Maastricht Univ, Dept Radiat Oncol, MAASTRO Clin, Med Ctr, Maastricht, Netherlands

[8] Univ Calif San Francisco, Dept Neurol Surg, San Francisco, CA USA

[9] Univ Colorado, Dept Radiat Oncol, Sch Med, Aurora, CO USA

[10] Wayne State Univ, Henry Ford Hlth Syst, Dept Neurosurg, Detroit, MI USA

[11] London Reg Canc Program, London, ON, Canada

[12] Miami Canc Inst, Dept Radiat Oncol, Miami, FL USA

[13] Univ Turin, Dept Neurooncol, Turin, Italy

[14] City Hlth & Sci Hosp, Turin, Italy

[15] Erasmus MC, Brain Tumor Ctr, Rotterdam, Netherlands

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

[17] Brigham & Womens Hosp, Dana Farber Canc Inst, Dept Radiat Oncol, 75 Francis St, Boston, MA 02115 USA

[18] Brigham & Womens Hosp, Dana Farber Canc Inst, Ctr Neurooncol, 75 Francis St, Boston, MA 02115 USA

来源：

LANCET ONCOLOGY | 2018年 / 19卷 / 01期

关键词：

QUALITY-OF-LIFE; STEREOTACTIC RADIOSURGERY; RADIATION-THERAPY; RANDOMIZED-TRIAL; RANO GROUP; NEUROCOGNITIVE FUNCTION; SURGICAL RESECTION; SINGLE METASTASES; RADIOTHERAPY; SURVIVAL;

D O I：

10.1016/S1470-2045(17)30692-7

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 [肿瘤学];

摘要：

The goals of therapeutic and biomarker development form the foundation of clinical trial design, and change considerably from early-phase to late-phase trials. From these goals, decisions on specific clinical trial design elements, such as endpoint selection and statistical approaches, are formed. Whereas early-phase trials might focus on finding a therapeutic signal to make decisions on further development, late-phase trials focus on the confirmation of therapeutic impact by considering clinically meaningful endpoints. In this guideline from the Response Assessment in Neuro-Oncology Brain Metastases (RANO-BM) working group, we highlight issues related to, and provide recommendations for, the design of clinical trials on local therapies for CNS metastases from solid tumours. We discuss endpoint selection criteria, the analysis appropriate for early-phase and late-phase trials, the association between tumour-specific and clinically meaningful endpoints, and possible issues related to the estimation of local control in the context of competing risks. In light of these discussions, we make specific recommendations on the clinical trial design of local therapies for brain metastases.

引用

页码：E33 / E42

页数：10

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