{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,10]],"date-time":"2026-09-10T04:28:05Z","timestamp":1789014485822,"version":"build-2803163510"},"reference-count":39,"publisher":"Wiley","issue":"10","license":[{"start":{"date-parts":[[2022,6,21]],"date-time":"2022-06-21T00:00:00Z","timestamp":1655769600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/100016615","name":"Curing Kids Cancer","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100016615","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":[[2022,10]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Current therapies for relapsed\/refractory (R\/R) pediatric myeloid neoplasms are inadequately effective. Real\u2010world data (RWD) can improve care by augmenting traditional studies and include individuals not eligible for clinical trials. The Therapeutic Advances in Childhood Leukemia and Lymphoma (TACL) consortium recently completed T2016\u2010003, a phase 1 study of decitabine, vorinostat, fludarabine, cytarabine, and granulocyte colony\u2010stimulating factor (G\u2010CSF) in R\/R acute myeloid leukemia (AML), which added epigenetic drugs to a cytotoxic backbone. We report results of RWD from six centers that treated 28 pediatric patients (26 with AML, two with other myeloid neoplasms) identically to the TACL study but who were not enrolled. This allowed unique analyses and the ability to compare data with the 35 TACL study patients. The overall response rate (ORR) (complete response [CR] plus CR with incomplete count recovery) among 26 RWD evaluable patients was 65%. The ORR of 13 patients with relapsed AML with epigenetic alterations was 69% (T2016\u2010003 + RWD: 68%, <jats:italic>n<\/jats:italic>\u00a0=\u00a025), of eight patients with refractory AML was 38% (T2016\u2010003 + RWD: 41%, <jats:italic>n<\/jats:italic>\u00a0=\u00a017) and of five patients with therapy\u2010related AML (t\u2010AML) was 80% (T2016\u2010003 + RWD: 75%, <jats:italic>n<\/jats:italic>\u00a0=\u00a08). The mean number of Grade 3\/4 toxicities experienced by the T2016\u2010003\u2010eligible RWD population (<jats:italic>n<\/jats:italic>\u00a0=\u00a022) (one per patient\u2010cycle) was not meaningfully different than those (<jats:italic>n<\/jats:italic>\u00a0=\u00a06) who would have been TACL study\u2010ineligible secondary to comorbidities (two per patient\u2010cycle). Overall, this therapy was well tolerated and effective in pediatric patients with R\/R myeloid neoplasms, particularly those with epigenetic alterations, t\u2010AML, and refractory disease.<\/jats:p>","DOI":"10.1002\/pbc.29812","type":"journal-article","created":{"date-parts":[[2022,6,21]],"date-time":"2022-06-21T11:53:42Z","timestamp":1655812422000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":14,"title":["Real\u2010world experience in treating pediatric relapsed\/refractory or therapy\u2010related myeloid malignancies with decitabine, vorinostat, and FLAG therapy based on a phase 1 study run by the TACL consortium"],"prefix":"10.1002","volume":"69","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-7063-512X","authenticated-orcid":false,"given":"Eric S.","family":"Schafer","sequence":"first","affiliation":[{"name":"Baylor College of Medicine  Houston Texas USA"},{"name":"Texas Children's Cancer Center  Houston Texas 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