Modernizing Clinical Trial Eligibility Criteria: Recommendations of the American Society of Clinical Oncology–Friends of Cancer Research Brain Metastases Working Group
- 20 November 2017
- journal article
- research article
- Published by American Society of Clinical Oncology (ASCO) in Journal of Clinical Oncology
- Vol. 35 (33), 3760-3773
- https://doi.org/10.1200/jco.2017.74.0761
Abstract
Broadening trial eligibility to improve accrual and access and to better reflect intended-to-treat populations has been recognized as a priority. Historically, patients with brain metastases have been understudied, because of restrictive eligibility across all phases of clinical trials. In 2016, after a literature search and series of teleconferences, a multistakeholder workshop was convened. Our working group focused on developing consensus recommendations regarding the inclusion of patients with brain metastases in clinical trials, as part of a broader effort that encompassed minimum age, HIV status, and organ dysfunction. The working group attempted to balance the needs of protecting patient safety, facilitating access to investigational therapies, and ensuring trial integrity. On the basis of input at the workshop, guidelines were further refined and finalized. The working group identified three key populations: those with treated/stable brain metastases, defined as patients who have received prior therapy for their brain metastases and whose CNS disease is radiographically stable at study entry; those with active brain metastases, defined as new and/or progressive brain metastases at the time of study entry; and those with leptomeningeal disease. In most circumstances, the working group encourages the inclusion of patients with treated/stable brain metastases in clinical trials. A framework of key considerations for patients with active brain metastases was developed. For patients with leptomeningeal disease, inclusion of a separate cohort in both early-phase and later-phase trials is recommended, if CNS activity is anticipated and when relevant to the specific disease type. Expanding eligibility to be more inclusive of patients with brain metastasis is justified in many cases and may speed the development of effective therapies in this area of high clinical need.This publication has 72 references indexed in Scilit:
- Clinicopathologic features, patterns of recurrence, and survival among women with triple‐negative breast cancer in the National Comprehensive Cancer NetworkCancer, 2012
- Phase I Clinical Trial Outcomes in 93 Patients with Brain Metastases: The MD Anderson Cancer Center ExperienceClinical Cancer Research, 2011
- Heterogeneous Blood–Tumor Barrier Permeability Determines Drug Efficacy in Experimental Brain Metastases of Breast CancerClinical Cancer Research, 2010
- Bevacizumab Safety in Patients with Central Nervous System MetastasesClinical Cancer Research, 2010
- Multicenter Phase II Study of Lapatinib in Patients with Brain Metastases from HER2-Positive Breast CancerClinical Cancer Research, 2009
- New response evaluation criteria in solid tumours: Revised RECIST guideline (version 1.1)European Journal Of Cancer, 2009
- Sites of distant recurrence and clinical outcomes in patients with metastatic triple‐negative breast cancerCancer, 2008
- Effect of Lapatinib on the Outgrowth of Metastatic Breast Cancer Cells to the BrainJNCI Journal of the National Cancer Institute, 2008
- New Guidelines to Evaluate the Response to Treatment in Solid TumorsJNCI Journal of the National Cancer Institute, 2000
- Recursive partitioning analysis (RPA) of prognostic factors in three radiation therapy oncology group (RTOG) brain metastases trialsInternational Journal of Radiation Oncology*Biology*Physics, 1997