{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,7]],"date-time":"2026-10-07T19:24:41Z","timestamp":1791401081166,"version":"4.3.3"},"reference-count":26,"publisher":"Springer Science and Business Media LLC","issue":"8","license":[{"start":{"date-parts":[[2025,8,1]],"date-time":"2025-08-01T00:00:00Z","timestamp":1754006400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"},{"start":{"date-parts":[[2025,8,2]],"date-time":"2025-08-02T00:00:00Z","timestamp":1754092800000},"content-version":"vor","delay-in-days":1,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"}],"funder":[{"DOI":"10.13039\/501100008678","name":"Universit\u00e4t Leipzig","doi-asserted-by":"crossref","id":[{"id":"10.13039\/501100008678","id-type":"DOI","asserted-by":"crossref"}]}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Ann Hematol"],"published-print":{"date-parts":[[2025,8]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is an aggressive, poor-prognostic, and CD123-overexpressing orphan hematologic malignancy for which tagraxofusp, a CD123-targeted fusion protein, is the only approved drug. In this retrospective study, safety and efficacy of tagraxofusp were assessed through real-world clinical practice data, collected in patients with BPDCN who received tagraxofusp via a European Named Patient Program (2019\u20132024). Twenty-six adults with treatment-na\u00efve BPDCN received 12\u00a0\u00b5g\/kg tagraxofusp intravenously daily on days 1\u20135 (or by day 10) of a 21-day cycle. Primary endpoints were complete response (CR) rates and incidence\/severity of capillary leak syndrome (CLS). Secondary endpoints included hematopoietic stem cell transplantation (HSCT), overall survival (OS), and safety. At a median follow-up of 13.5 months, the overall response rate (ORR) was 90% (65% CR); the median duration of response (DOR) was 10.3 months. In 12 evaluable patients who were bridged to HSCT, the pre-transplant ORR was 92% (75% CR), with 14.7 months median DOR. Median OS was 20.2 months (95% CI 10.2\u2013not estimable) in the overall population and 37.0 months (95% CI 10.7\u2013not estimable) in patients bridged to HSCT. CLS events were diagnosed in 13 patients (50%); 68% occurred in cycle 1; 54% were grade 2, and 46% grade 3\/4. Treatment-related non-hematologic grade 3\/4 adverse events (AEs) or serious AEs were reported in 12 patients (46%); 9 patients (35%) had treatment-related grade 3\/4 hematologic AEs. These real-world findings showed no new safety signals and confirmed that tagraxofusp is the first-line treatment of choice for most patients with BPDCN.<\/jats:p>","DOI":"10.1007\/s00277-025-06493-w","type":"journal-article","created":{"date-parts":[[2025,8,2]],"date-time":"2025-08-02T05:22:52Z","timestamp":1754112172000},"page":"4121-4131","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":13,"title":["Durable outcomes with manageable safety leading to prolonged survival with tagraxofusp for treatment-na\u00efve patients with blastic plasmacytoid dendritic cell neoplasm: real world results from a European Named Patient Program"],"prefix":"10.1007","volume":"104","author":[{"given":"Marco","family":"Herling","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emanuele","family":"Angelucci","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tobias Matthieu","family":"Benoit","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Giulia","family":"Rivoli","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Antonio","family":"Curti","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Katharina S.","family":"G\u00f6tze","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stefan","family":"Wirths","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dimoula","family":"Erakli","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael","family":"Zuurman","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eric","family":"Deconinck","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2025,8,2]]},"reference":[{"key":"6493_CR1","doi-asserted-by":"publisher","first-page":"5007","DOI":"10.1182\/blood-2002-10-3297","volume":"101","author":"M Herling","year":"2003","unstructured":"Herling M, Teitell MA, Shen RR, Medeiros LJ, Jones D (2003) TCL1 expression in plasmacytoid dendritic cells (DC2s) and the related CD4\u2009+\u2009CD56\u2009+\u2009blastic tumors of skin. 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A central ethics committee approval was provided by the sponsor, the University of Leipzig, under #288\/22-ek.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics approval"}},{"value":"MH: research funding from Beigene and JanPix; consulting\/advisory role for and honoraria from BeiGene, Janssen, Stemline Menarini, Takeda; honoraria also from Roche; expert testimony for Stemline Menarini; travel\/accommodations and expenses from BeiGene, Janssen. EA: consulting\/advisory role for Regeneron, Novartis; honoraria from Menarini-Stemline, BMS; travel\/accommodations and expenses from AbbVie, Gilead; participation on a DSMB or advisory board for Vertex, BMS, Vifor. TMB: travel expenses for EBMT Congress 2024 from Neovii; honoraria from Vertex Pharmaceuticals. GR: research funding to institution from Syros (Site PI for SY-1425-301), Novartis (Site PI for CMBG 453B12203); travel accommodation for ASH 2022 from AbbVie. AC: advisory board for and honoraria from AbbVie, Pfizer, Novartis, Menarini-Stemline; honoraria also from BMS, Jazz Pharmaceuticals; DSMB for AbbVie, Novartis, Menarini-Stemline. KSG: advisory boards for and honoraria from AbbVie, BMS; honoraria from Jazz, BMS, Pfizer travel expenses for EHA 2023 from AbbVie. SW: consultancy for Stemline Therapeutics, AbbVie, AstraZeneca; honoraria from Lilly, Stemline Therapeutics; travel expenses from BeiGene, AbbVie. DE: no relevant conflicts of interest to disclose. MZ: employment with Menarini Group. ED: consultancy and honoraria for congress presentations from Stemline Menarini; travel accommodations\/expenses from Novartis, Pfizer, Gilead, Janssen, AbbVie.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}},{"value":"Based on national or local institutional regulations for secondary use of data, if so required, patients consented to collection of their anonymized electronic health record data via the centrally approved informed consent form for this study.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Patient consent statement"}}]}}