{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,22]],"date-time":"2026-09-22T01:25:44Z","timestamp":1790040344815,"version":"4.0.1"},"reference-count":34,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2024,2,18]],"date-time":"2024-02-18T00:00:00Z","timestamp":1708214400000},"content-version":"vor","delay-in-days":17,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["eJHaem"],"published-print":{"date-parts":[[2024,2]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Blastic plasmacytoid dendritic cell neoplasm (BPDCN) predominantly occurs in adults \u226560 years old; 10\u201320% of cases are pediatric or adolescent\/young adult (AYA) patients. Tagraxofusp (TAG, Elzonris<jats:sup>\u00ae<\/jats:sup>) is the only approved treatment for BPDCN; in the United States it is approved for patients aged \u22652 years. Data on treating pediatric and AYA BPDCN patients are limited. We present a case series of pediatric and AYA patients with BPDCN treated with TAG. Eight patients (five newly diagnosed; three relapsed\/refractory [R\/R]), aged 2\u201321 years, received 12 mcg\/kg TAG. Seven patients were female; most had skin (<jats:italic>n<\/jats:italic> = 6) and\/or bone marrow (<jats:italic>n<\/jats:italic> = 4) involvement. No new safety signals were identified. Grade 3 adverse events were headache (<jats:italic>n<\/jats:italic> = 1) and transaminitis (<jats:italic>n<\/jats:italic> = 2). Three patients with newly diagnosed BPDCN achieved complete response, one achieved partial response, and one had stable disease (SD). One patient with R\/R BPDCN achieved a minor response; one had SD. Seven patients (88%) were bridged to stem cell transplant: 80% of newly diagnosed patients and 100% of R\/R patients. Five patients remained alive at last follow\u2010up. These cases highlight the efficacy and safety of TAG in pediatric and AYA patients for whom there is no other approved BPDCN therapy.<\/jats:p>","DOI":"10.1002\/jha2.856","type":"journal-article","created":{"date-parts":[[2024,2,18]],"date-time":"2024-02-18T17:09:30Z","timestamp":1708276170000},"page":"61-69","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":12,"title":["Efficacy and manageable safety of tagraxofusp in blastic plasmacytoid dendritic cell neoplasm: a case series of pediatric and adolescent\/young adult patients"],"prefix":"10.1002","volume":"5","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-1670-6513","authenticated-orcid":false,"given":"Naveen","family":"Pemmaraju","sequence":"first","affiliation":[{"name":"Department of Leukemia The University of Texas MD Anderson Cancer Center  Houston Texas 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