{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,29]],"date-time":"2026-09-29T20:26:56Z","timestamp":1790713616660,"version":"4.1.0"},"reference-count":156,"publisher":"Frontiers Media SA","license":[{"start":{"date-parts":[[2023,6,1]],"date-time":"2023-06-01T00:00:00Z","timestamp":1685577600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["frontiersin.org"],"crossmark-restriction":true},"short-container-title":["Front. Cell Dev. Biol."],"abstract":"<jats:p>Acute megakaryoblastic leukemia (AMKL) is a rare subtype of acute myeloid leukemia (AML) in which leukemic blasts have megakaryocytic features. AMKL makes up 4%\u201315% of newly diagnosed pediatric AML, typically affecting young children (less than 2\u00a0years old). AMKL associated with Down syndrome (DS) shows <jats:italic>GATA1<\/jats:italic> mutations and has a favorable prognosis. In contrast, AMKL in children without DS is often associated with recurrent and mutually exclusive chimeric fusion genes and has an unfavorable prognosis. This review mainly summarizes the unique features of pediatric non-DS AMKL and highlights the development of novel therapies for high-risk patients. Due to the rarity of pediatric AMKL, large-scale multi-center studies are needed to progress molecular characterization of this disease. Better disease models are also required to test leukemogenic mechanisms and emerging therapies.<\/jats:p>","DOI":"10.3389\/fcell.2023.1170622","type":"journal-article","created":{"date-parts":[[2023,6,1]],"date-time":"2023-06-01T04:51:50Z","timestamp":1685595110000},"update-policy":"https:\/\/doi.org\/10.3389\/crossmark-policy","source":"Crossref","is-referenced-by-count":14,"title":["Advances in molecular characterization of pediatric acute megakaryoblastic leukemia not associated with Down syndrome; impact on therapy development"],"prefix":"10.3389","volume":"11","author":[{"given":"Jixia","family":"Li","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maggie L.","family":"Kalev\u2010Zylinska","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1965","published-online":{"date-parts":[[2023,6,1]]},"reference":[{"key":"B1","doi-asserted-by":"publisher","first-page":"571","DOI":"10.1038\/s41375-022-01800-0","article-title":"High caspase 3 and vulnerability to dual BCL2 family inhibition define ETO2::GLIS2 pediatric leukemia","volume":"37","author":"Aid","year":"2023","journal-title":"Leukemia"},{"key":"B2","doi-asserted-by":"publisher","first-page":"4252","DOI":"10.1182\/bloodadvances.2019032029","article-title":"The stem cell-specific long noncoding RNA HOXA10-AS in the pathogenesis of KMT2A-rearranged leukemia","volume":"3","author":"Al-Kershi","year":"2019","journal-title":"Blood Adv."},{"key":"B3","doi-asserted-by":"publisher","first-page":"e28055","DOI":"10.1002\/pbc.28055","article-title":"CBFA2T3-GLIS2-positive acute megakaryoblastic leukemia in a patient with Down syndrome","volume":"67","author":"Amano","year":"2020","journal-title":"Pediatr. 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