{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,13]],"date-time":"2026-03-13T16:33:28Z","timestamp":1773419608927,"version":"3.50.1"},"reference-count":35,"publisher":"Wiley","issue":"7","license":[{"start":{"date-parts":[[2024,4,16]],"date-time":"2024-04-16T00:00:00Z","timestamp":1713225600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"},{"start":{"date-parts":[[2024,4,16]],"date-time":"2024-04-16T00:00:00Z","timestamp":1713225600000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"funder":[{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Pediatric Blood &amp; Cancer"],"published-print":{"date-parts":[[2024,7]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>Novel therapies are needed for relapsed and refractory rhabdomyosarcoma (RRMS). Phase II clinical trials in RRMS have typically utilized radiologic response as the primary activity endpoint, an approach that poses several limitations in RRMS. In this analysis, we aimed to estimate an event\u2010free survival (EFS) endpoint for RRMS that could be used as a benchmark for future studies.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Procedure<\/jats:title>\n                    <jats:p>We performed a retrospective study of patients with RRMS enrolling on 13 single\u2010agent phase II Children's Oncology Group and legacy group trials from 1997 to 2016. All included trials used radiographic response as their primary activity endpoint. Six\u2010month EFS was estimated from time of trial enrollment with 95% confidence intervals. Clinical characteristics, including trial of enrollment, sex, age, race, histology, number of prior chemotherapies, and radiographic response were evaluated for their impact on 6\u2010month EFS.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>We identified 175 patients across 13 trials. The 6\u2010month EFS was 16.8% (11.6%\u201322.8%). No differences were seen in 6\u2010month EFS based on age, sex, race, or histology. There were nonsignificant trends toward improved 6\u2010month EFS for patients with less than or equal to two prior lines of therapy versus higher than two, for patients enrolled on trials that achieved their primary radiographic response endpoint versus trials that did not, and for patients who achieved complete or partial response compared to those achieving stable disease.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>The prognosis of RRMS enrolled on single\u2010agent phase II trials is poor. This pooled 6\u2010month EFS of RRMS on single\u2010agent trials may be used as a RRMS\u2010specific benchmark for future single\u2010agent phase II trials.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/pbc.31009","type":"journal-article","created":{"date-parts":[[2024,4,16]],"date-time":"2024-04-16T23:53:18Z","timestamp":1713311598000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":8,"title":["Event\u2010free survival in relapsed and refractory rhabdomyosarcoma treated on cooperative group phase II trials: A report from the Children's Oncology Group"],"prefix":"10.1002","volume":"71","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-1068-7857","authenticated-orcid":false,"given":"Jonathan","family":"Metts","sequence":"first","affiliation":[{"name":"Sarcoma Department Moffitt Cancer Center  Tampa Florida USA"},{"name":"Cancer and Blood Disorders Institute Johns Hopkins All Children's Hospital  St Petersburg Florida USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Wei","family":"Xue","sequence":"additional","affiliation":[{"name":"Department of Biostatistics University of Florida  Gainesville Florida USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Zhengya","family":"Gao","sequence":"additional","affiliation":[{"name":"Department of Biostatistics University of Florida  Gainesville Florida USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6071-0042","authenticated-orcid":false,"given":"Sapna","family":"Oberoi","sequence":"additional","affiliation":[{"name":"Department of Pediatric Hematology\/Oncology CancerCare Manitoba  Winnipeg Manitoba Canada"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8352-3963","authenticated-orcid":false,"given":"Aaron R.","family":"Weiss","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Maine Medical Center  Portland Maine USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Rajkumar","family":"Venkatramani","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Baylor College of Medicine  Houston Texas USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6776-4852","authenticated-orcid":false,"given":"Douglas J.","family":"Harrison","sequence":"additional","affiliation":[{"name":"Department of Pediatrics University of Texas MD Anderson Cancer Center  Houston Texas 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