{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,29]],"date-time":"2026-08-29T04:58:30Z","timestamp":1787979510785,"version":"build-2784847793"},"reference-count":17,"publisher":"Wiley","issue":"8","license":[{"start":{"date-parts":[[2021,4,15]],"date-time":"2021-04-15T00:00:00Z","timestamp":1618444800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Pediatric Blood &amp; Cancer"],"published-print":{"date-parts":[[2021,8]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Immunosuppressive therapy with horse antithymocyte globulin and cyclosporine currently remains the standard therapy for children with severe aplastic anemia (SAA) who lack human leukocyte antigen (HLA)\u2010identical sibling. The thrombopoietin receptor agonist eltrombopag has been recently approved for SAA patients 2\u00a0years and older. However, there are limited data on its safety and efficacy in pediatric cohorts.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>We conducted a retrospective study of patients \u226418\u00a0years old consecutively diagnosed with SAA between 2000 and 2018. Patients received either standard immunosuppressive therapy (IST\u2010Std) or IST with eltrombopag (IST\u2010Epag). The primary outcome was the objective response (OR), including partial and complete response (CR), at 6 and 12\u00a0months after starting therapy.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>We identified 16 patients receiving IST\u2010Std and nine IST\u2010Epag treatment (seven of nine as upfront therapy and two of seven after previously failed IST). The OR at 6 and 12\u00a0months in IST\u2010Std arm was 71% and 100%, with CR in 29% and 58%, respectively. Seven patients receiving upfront IST\u2010Epag had OR at 6 and 12\u00a0months, with two of seven (29%) achieving CR at 6 and 12\u00a0months. Two patients who previously failed standard IST did not respond to eltrombopag. No significant differences were observed in both cohorts with regard to infections. One IST\u2010Epag\u2010treated patient developed transient grade 3 transaminitis. Finally, no changes in paroxysmal nocturnal hemoglobinuria (PNH) clone size and cytogenetic abnormalities were seen in either cohort.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>The addition of eltrombopag to standard IST was well tolerated and resulted in satisfactory hematological response at 6 and 12\u00a0months in this single\u2010institution experience. A larger cohort with longer follow\u2010up is required to assess response durability.<\/jats:p><\/jats:sec>","DOI":"10.1002\/pbc.29066","type":"journal-article","created":{"date-parts":[[2021,4,15]],"date-time":"2021-04-15T22:22:39Z","timestamp":1618525359000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":27,"title":["Eltrombopag in children with severe aplastic anemia"],"prefix":"10.1002","volume":"68","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-5496-3839","authenticated-orcid":false,"given":"Harry","family":"Lesmana","sequence":"first","affiliation":[{"name":"Department of Hematology St. Jude Children's Research Hospital  Memphis Tennessee USA"},{"name":"Department of Pediatric Hematology, Oncology and Bone Marrow Transplantation, Cleveland Clinic  Cleveland Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Timothy","family":"Jacobs","sequence":"additional","affiliation":[{"name":"Department 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USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6774-9698","authenticated-orcid":false,"given":"Winfred","family":"Wang","sequence":"additional","affiliation":[{"name":"Department of Hematology St. Jude Children's Research Hospital  Memphis Tennessee USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marcin","family":"Wlodarski","sequence":"additional","affiliation":[{"name":"Department of Hematology St. Jude Children's Research Hospital  Memphis Tennessee 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