Cargando…
EPEN-54. ACNS0831, PHASE III RANDOMIZED TRIAL OF POST-RADIATION CHEMOTHERAPY IN PATIENTS WITH NEWLY DIAGNOSED EPENDYMOMA AGES 1 TO 21 YEARS
PURPOSE: The primary objective of this study is to determine the EFS and OS of children with gross total and near totally resected ependymoma (EPN) treated with post-operative focal radiation therapy (RT) followed by randomization to either RT alone or RT + 4 cycles of maintenance chemotherapy with...
Autores principales: | , , , , , , , , , |
---|---|
Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Oxford University Press
2020
|
Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7715961/ http://dx.doi.org/10.1093/neuonc/noaa222.187 |
_version_ | 1783619078531842048 |
---|---|
author | Smith, Amy Onar-Thomas, Arzu Ellison, David Owens-Pickle, Emily Wu, Shengjie Leary, Sarah E S Fouladi, Maryam Merchant, Thomas Gajjar, Amar Foreman, Nicholas |
author_facet | Smith, Amy Onar-Thomas, Arzu Ellison, David Owens-Pickle, Emily Wu, Shengjie Leary, Sarah E S Fouladi, Maryam Merchant, Thomas Gajjar, Amar Foreman, Nicholas |
author_sort | Smith, Amy |
collection | PubMed |
description | PURPOSE: The primary objective of this study is to determine the EFS and OS of children with gross total and near totally resected ependymoma (EPN) treated with post-operative focal radiation therapy (RT) followed by randomization to either RT alone or RT + 4 cycles of maintenance chemotherapy with vincristine, cisplatin, cyclophosphamide and etoposide. Secondary objectives include estimating the EFS and OS of children not randomized, evaluation of neurobehavioral and quality of life (QoL) endpoints, and EPN biomarkers. RESULTS: 479 patients enrolled, 451 were eligible. Of 325 eligible randomized patients, 161 were randomized to RT alone and 164 to RT + maintenance chemotherapy. Age range (1–21 years, median 4.9 years). The planned primary analysis was based on intent-to-treat, irrespective of actual treatment received. Based on the data available as of 12/31/2019, estimated 3-year EFS in the RT + maintenance chemotherapy vs. RT arms were 78% vs. 72%, respectively, which did not meet statistical criteria to establish the benefit of maintenance chemotherapy post RT (1-sided log-rank p-value=0.074). Due to significant noncompliance (30.5% in the RT + maintenance vs 4.3% in the RT arm), a planned secondary “as treated” analysis was performed. With median follow-up of 42.6 months among patients without events, the 3 year EFS estimates for patients who received any chemotherapy (n=114) vs. those who received RT only (n=196) were 80% vs. 71%, respectively (1-sided p-value = 0.0121). CONCLUSION: Early results in this randomized trial suggest that there may be some EPN patients who benefit from maintenance chemotherapy. Genomic analyses are ongoing. |
format | Online Article Text |
id | pubmed-7715961 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2020 |
publisher | Oxford University Press |
record_format | MEDLINE/PubMed |
spelling | pubmed-77159612020-12-09 EPEN-54. ACNS0831, PHASE III RANDOMIZED TRIAL OF POST-RADIATION CHEMOTHERAPY IN PATIENTS WITH NEWLY DIAGNOSED EPENDYMOMA AGES 1 TO 21 YEARS Smith, Amy Onar-Thomas, Arzu Ellison, David Owens-Pickle, Emily Wu, Shengjie Leary, Sarah E S Fouladi, Maryam Merchant, Thomas Gajjar, Amar Foreman, Nicholas Neuro Oncol Ependymoma PURPOSE: The primary objective of this study is to determine the EFS and OS of children with gross total and near totally resected ependymoma (EPN) treated with post-operative focal radiation therapy (RT) followed by randomization to either RT alone or RT + 4 cycles of maintenance chemotherapy with vincristine, cisplatin, cyclophosphamide and etoposide. Secondary objectives include estimating the EFS and OS of children not randomized, evaluation of neurobehavioral and quality of life (QoL) endpoints, and EPN biomarkers. RESULTS: 479 patients enrolled, 451 were eligible. Of 325 eligible randomized patients, 161 were randomized to RT alone and 164 to RT + maintenance chemotherapy. Age range (1–21 years, median 4.9 years). The planned primary analysis was based on intent-to-treat, irrespective of actual treatment received. Based on the data available as of 12/31/2019, estimated 3-year EFS in the RT + maintenance chemotherapy vs. RT arms were 78% vs. 72%, respectively, which did not meet statistical criteria to establish the benefit of maintenance chemotherapy post RT (1-sided log-rank p-value=0.074). Due to significant noncompliance (30.5% in the RT + maintenance vs 4.3% in the RT arm), a planned secondary “as treated” analysis was performed. With median follow-up of 42.6 months among patients without events, the 3 year EFS estimates for patients who received any chemotherapy (n=114) vs. those who received RT only (n=196) were 80% vs. 71%, respectively (1-sided p-value = 0.0121). CONCLUSION: Early results in this randomized trial suggest that there may be some EPN patients who benefit from maintenance chemotherapy. Genomic analyses are ongoing. Oxford University Press 2020-12-04 /pmc/articles/PMC7715961/ http://dx.doi.org/10.1093/neuonc/noaa222.187 Text en © The Author(s) 2020. Published by Oxford University Press on behalf of the Society for Neuro-Oncology. http://creativecommons.org/licenses/by-nc/4.0/ This is an Open Access article distributed under the terms of the Creative Commons Attribution Non-Commercial License (http://creativecommons.org/licenses/by-nc/4.0/), which permits non-commercial re-use, distribution, and reproduction in any medium, provided the original work is properly cited. For commercial re-use, please contact journals.permissions@oup.com |
spellingShingle | Ependymoma Smith, Amy Onar-Thomas, Arzu Ellison, David Owens-Pickle, Emily Wu, Shengjie Leary, Sarah E S Fouladi, Maryam Merchant, Thomas Gajjar, Amar Foreman, Nicholas EPEN-54. ACNS0831, PHASE III RANDOMIZED TRIAL OF POST-RADIATION CHEMOTHERAPY IN PATIENTS WITH NEWLY DIAGNOSED EPENDYMOMA AGES 1 TO 21 YEARS |
title | EPEN-54. ACNS0831, PHASE III RANDOMIZED TRIAL OF POST-RADIATION CHEMOTHERAPY IN PATIENTS WITH NEWLY DIAGNOSED EPENDYMOMA AGES 1 TO 21 YEARS |
title_full | EPEN-54. ACNS0831, PHASE III RANDOMIZED TRIAL OF POST-RADIATION CHEMOTHERAPY IN PATIENTS WITH NEWLY DIAGNOSED EPENDYMOMA AGES 1 TO 21 YEARS |
title_fullStr | EPEN-54. ACNS0831, PHASE III RANDOMIZED TRIAL OF POST-RADIATION CHEMOTHERAPY IN PATIENTS WITH NEWLY DIAGNOSED EPENDYMOMA AGES 1 TO 21 YEARS |
title_full_unstemmed | EPEN-54. ACNS0831, PHASE III RANDOMIZED TRIAL OF POST-RADIATION CHEMOTHERAPY IN PATIENTS WITH NEWLY DIAGNOSED EPENDYMOMA AGES 1 TO 21 YEARS |
title_short | EPEN-54. ACNS0831, PHASE III RANDOMIZED TRIAL OF POST-RADIATION CHEMOTHERAPY IN PATIENTS WITH NEWLY DIAGNOSED EPENDYMOMA AGES 1 TO 21 YEARS |
title_sort | epen-54. acns0831, phase iii randomized trial of post-radiation chemotherapy in patients with newly diagnosed ependymoma ages 1 to 21 years |
topic | Ependymoma |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7715961/ http://dx.doi.org/10.1093/neuonc/noaa222.187 |
work_keys_str_mv | AT smithamy epen54acns0831phaseiiirandomizedtrialofpostradiationchemotherapyinpatientswithnewlydiagnosedependymomaages1to21years AT onarthomasarzu epen54acns0831phaseiiirandomizedtrialofpostradiationchemotherapyinpatientswithnewlydiagnosedependymomaages1to21years AT ellisondavid epen54acns0831phaseiiirandomizedtrialofpostradiationchemotherapyinpatientswithnewlydiagnosedependymomaages1to21years AT owenspickleemily epen54acns0831phaseiiirandomizedtrialofpostradiationchemotherapyinpatientswithnewlydiagnosedependymomaages1to21years AT wushengjie epen54acns0831phaseiiirandomizedtrialofpostradiationchemotherapyinpatientswithnewlydiagnosedependymomaages1to21years AT learysarahes epen54acns0831phaseiiirandomizedtrialofpostradiationchemotherapyinpatientswithnewlydiagnosedependymomaages1to21years AT fouladimaryam epen54acns0831phaseiiirandomizedtrialofpostradiationchemotherapyinpatientswithnewlydiagnosedependymomaages1to21years AT merchantthomas epen54acns0831phaseiiirandomizedtrialofpostradiationchemotherapyinpatientswithnewlydiagnosedependymomaages1to21years AT gajjaramar epen54acns0831phaseiiirandomizedtrialofpostradiationchemotherapyinpatientswithnewlydiagnosedependymomaages1to21years AT foremannicholas epen54acns0831phaseiiirandomizedtrialofpostradiationchemotherapyinpatientswithnewlydiagnosedependymomaages1to21years |