Nils D Arvold1, Terri S Armstrong2, Katherine E Warren3, Susan M Chang4, Lisa M DeAngelis5, Jaishri Blakeley6, Marc C Chamberlain7, Erin Dunbar8, Herbert H Loong9, David R Macdonald10, David A Reardon11, Michael A Vogelbaum12, Ying Yuan13, Michael Weller14, Martin van den Bent15, Patrick Y Wen11. 1. St Luke's Radiation Oncology Associates, St Luke's Cancer Center, University of Minnesota, Duluth, Minnesota, USA. 2. Neuro-Oncology Branch, National Cancer Institute, Bethesda, Maryland. 3. Pediatric Oncology Branch, National Cancer Institute, Bethesda, Maryland, USA. 4. Department of Neurosurgery, University of California San Francisco, San Francisco, California, USA. 5. Department of Neuro-Oncology, Memorial Sloan Kettering Cancer Center, New York, New York, USA. 6. Department of Neurology, Johns Hopkins Hospital, Baltimore, Maryland, USA. 7. Cascadian Therapeutics, Seattle, Washington, USA. 8. Piedmont Brain Tumor Center, Atlanta, Georgia, USA. 9. Department of Clinical Oncology, The Chinese University of Hong Kong, Prince of Wales Hospital, Hong Kong, China. 10. Schulich School of Medicine and Dentistry, Western University, London, Ontario, Canada. 11. Center for Neuro-Oncology, Dana-Farber Cancer Institute, Boston, Massachusetts, USA. 12. Rose Ella Burkhardt Brain Tumor and Neuro-Oncology Center, Cleveland Clinic, Cleveland, Ohio, USA. 13. Department of Biostatistics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA. 14. Department of Neurology, University Hospital and University of Zurich, Zurich, Switzerland. 15. Brain Tumor Institute at Erasmus University Medical Center, Rotterdam, Netherlands.
Abstract
Background: Corticosteroids are the mainstay of treatment for peritumor edema but are often associated with significant side effects. Therapies that can reduce corticosteroid use would potentially be of significant benefit to patients. However, currently there are no standardized endpoints evaluating corticosteroid use in neuro-oncology clinical trials. Methods: The Response Assessment in Neuro-Oncology (RANO) Working Group has developed consensus recommendations for endpoints evaluating corticosteroid use in clinical trials in both adults and children with brain tumors. Results: Responders are defined as patients with a 50% reduction in total daily corticosteroid dose compared with baseline or reduction of the total daily dose to ≤2 mg of dexamethasone (or equivalent dose of other corticosteroid); baseline dose must be at least 4 mg of dexamethasone daily (or equivalent dose of other corticosteroids) for at least one week. Patients must have stable or improved Neurologic Assessment in Neuro-Oncology (NANO) score or Karnofsky performance status score or Eastern Cooperative Oncology Group (ECOG) (Lansky score for children age <16 y), and an improved score on a relevant clinical outcome assessment tool. These criteria must be sustained for at least 4 weeks after baseline assessment to be considered a response, and are confirmed 4 weeks after that (ie, 8 wk after baseline assessment) to be considered a sustained response. Conclusions: This RANO proposal for corticosteroid use endpoints in neuro-oncology clinical trials may need to be refined and will require prospective validation in clinical studies.
Background: Corticosteroids are the mainstay of treatment for peritumor edema but are often associated with significant side effects. Therapies that can reduce corticosteroid use would potentially be of significant benefit to patients. However, currently there are no standardized endpoints evaluating corticosteroid use in neuro-oncology clinical trials. Methods: The Response Assessment in Neuro-Oncology (RANO) Working Group has developed consensus recommendations for endpoints evaluating corticosteroid use in clinical trials in both adults and children with brain tumors. Results: Responders are defined as patients with a 50% reduction in total daily corticosteroid dose compared with baseline or reduction of the total daily dose to ≤2 mg of dexamethasone (or equivalent dose of other corticosteroid); baseline dose must be at least 4 mg of dexamethasone daily (or equivalent dose of other corticosteroids) for at least one week. Patients must have stable or improved Neurologic Assessment in Neuro-Oncology (NANO) score or Karnofsky performance status score or Eastern Cooperative Oncology Group (ECOG) (Lansky score for children age <16 y), and an improved score on a relevant clinical outcome assessment tool. These criteria must be sustained for at least 4 weeks after baseline assessment to be considered a response, and are confirmed 4 weeks after that (ie, 8 wk after baseline assessment) to be considered a sustained response. Conclusions: This RANO proposal for corticosteroid use endpoints in neuro-oncology clinical trials may need to be refined and will require prospective validation in clinical studies.
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