Corticosteroid use endpoints in neuro-oncology: Response Assessment in Neuro-Oncology Working Group

Nils D. Arvold, Terri S. Armstrong, Katherine E. Warren, Susan M. Chang, Lisa M. Deangelis, Jaishri Blakeley, Marc C. Chamberlain, Erin Dunbar, Herbert H. Loong, David R. Macdonald, David A. Reardon, Michael A. Vogelbaum, Ying Yuan, Michael Weller, Martin Van Den Bent, Patrick Y. Wen

Research output: Contribution to journalArticle

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.

Original languageEnglish (US)
Pages (from-to)897-906
Number of pages10
JournalNeuro-Oncology
Volume20
Issue number7
DOIs
StatePublished - Jun 18 2018

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Adrenal Cortex Hormones
Clinical Trials
Dexamethasone
Karnofsky Performance Status
Brain Neoplasms
Nervous System
Edema
Outcome Assessment (Health Care)
Therapeutics

Keywords

  • corticosteroids
  • endpoints
  • peritumor edema
  • RANO

ASJC Scopus subject areas

  • Oncology
  • Clinical Neurology
  • Cancer Research

Cite this

Arvold, N. D., Armstrong, T. S., Warren, K. E., Chang, S. M., Deangelis, L. M., Blakeley, J., ... Wen, P. Y. (2018). Corticosteroid use endpoints in neuro-oncology: Response Assessment in Neuro-Oncology Working Group. Neuro-Oncology, 20(7), 897-906. https://doi.org/10.1093/neuonc/noy056

Corticosteroid use endpoints in neuro-oncology : Response Assessment in Neuro-Oncology Working Group. / Arvold, Nils D.; Armstrong, Terri S.; Warren, Katherine E.; Chang, Susan M.; Deangelis, Lisa M.; Blakeley, Jaishri; Chamberlain, Marc C.; Dunbar, Erin; Loong, Herbert H.; Macdonald, David R.; Reardon, David A.; Vogelbaum, Michael A.; Yuan, Ying; Weller, Michael; Van Den Bent, Martin; Wen, Patrick Y.

In: Neuro-Oncology, Vol. 20, No. 7, 18.06.2018, p. 897-906.

Research output: Contribution to journalArticle

Arvold, ND, Armstrong, TS, Warren, KE, Chang, SM, Deangelis, LM, Blakeley, J, Chamberlain, MC, Dunbar, E, Loong, HH, Macdonald, DR, Reardon, DA, Vogelbaum, MA, Yuan, Y, Weller, M, Van Den Bent, M & Wen, PY 2018, 'Corticosteroid use endpoints in neuro-oncology: Response Assessment in Neuro-Oncology Working Group', Neuro-Oncology, vol. 20, no. 7, pp. 897-906. https://doi.org/10.1093/neuonc/noy056
Arvold, Nils D. ; Armstrong, Terri S. ; Warren, Katherine E. ; Chang, Susan M. ; Deangelis, Lisa M. ; Blakeley, Jaishri ; Chamberlain, Marc C. ; Dunbar, Erin ; Loong, Herbert H. ; Macdonald, David R. ; Reardon, David A. ; Vogelbaum, Michael A. ; Yuan, Ying ; Weller, Michael ; Van Den Bent, Martin ; Wen, Patrick Y. / Corticosteroid use endpoints in neuro-oncology : Response Assessment in Neuro-Oncology Working Group. In: Neuro-Oncology. 2018 ; Vol. 20, No. 7. pp. 897-906.
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T2 - Response Assessment in Neuro-Oncology Working Group

AU - Arvold, Nils D.

AU - Armstrong, Terri S.

AU - Warren, Katherine E.

AU - Chang, Susan M.

AU - Deangelis, Lisa M.

AU - Blakeley, Jaishri

AU - Chamberlain, Marc C.

AU - Dunbar, Erin

AU - Loong, Herbert H.

AU - Macdonald, David R.

AU - Reardon, David A.

AU - Vogelbaum, Michael A.

AU - Yuan, Ying

AU - Weller, Michael

AU - Van Den Bent, Martin

AU - Wen, Patrick Y.

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N2 - 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.

AB - 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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