{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,30]],"date-time":"2026-10-30T03:27:25Z","timestamp":1793330845869,"version":"4.5.0"},"reference-count":8,"publisher":"Springer Science and Business Media LLC","issue":"3","license":[{"start":{"date-parts":[[2025,2,20]],"date-time":"2025-02-20T00:00:00Z","timestamp":1740009600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"},{"start":{"date-parts":[[2025,2,20]],"date-time":"2025-02-20T00:00:00Z","timestamp":1740009600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Ann Hematol"],"published-print":{"date-parts":[[2025,3]]},"abstract":"<jats:title>Abstract<\/jats:title>\n          <jats:p>Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a highly aggressive and rare hematologic malignancy characterized by poor response to multiagent chemotherapy and dismal survival outcomes of 8\u201316 months. Tagraxofusp, the first-in-class CD123-directed antineoplastic agent, has emerged as a highly effective therapy and is the only FDA approved drug for BPDCN. Nonetheless, significant treatment-related toxicities with tagraxofusp such as hepatotoxicity and capillary leak syndrome are unfortunately not uncommon and can be prohibitive for older or unfit patients. The success of venetoclax (VEN) with a hypomethylating agent (HMA) has recently been described in the literature however, clinical outcomes are limited to case reports and small case series. To confirm these findings, we performed a multicenter, retrospective cohort study utilizing the TriNetX Networks database to compare survival outcomes between BPDCN patients (\u226560 years-of-age) who received VEN\u2009+\u2009HMA versus tagraxofusp. In total, 32 and 39 patients received VEN\u2009+\u2009HMA and tagraxofusp, respectively, between February 1, 2019 and September 1, 2024. Median follow-up time was 7.4 and 9.3 months in the VEN\u2009+\u2009HMA and tagraxofusp cohorts, respectively. Overall survival (OS) between VEN\u2009+\u2009HMA and tagraxofusp-treated patients was comparable at 12-months (41.2% vs. 53%; HR 1.15; 95% CI, 0.53\u20132.48; <jats:italic>P<\/jats:italic>\u2009=\u20090.73). In a subgroup analysis of older adult patients (\u226575 years-of-age), OS at 12-months (38.1% vs. 56.5%; HR 1.20; 95% CI, 0.47\u20133.04; <jats:italic>P<\/jats:italic>\u2009=\u20090.71) was not significantly different. In conclusion, this large-scale, retrospective database analysis suggests that VEN\u2009+\u2009HMA is an effective therapeutic alternative to tagraxofusp in older patients for the management of BPDCN. Future studies are needed to prospectively validate these findings.<\/jats:p>","DOI":"10.1007\/s00277-025-06221-4","type":"journal-article","created":{"date-parts":[[2025,2,20]],"date-time":"2025-02-20T03:09:28Z","timestamp":1740020968000},"page":"2069-2071","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":5,"title":["Venetoclax and hypomethylating agents versus tagraxofusp in older patients with blastic plasmacytoid dendritic cell neoplasm"],"prefix":"10.1007","volume":"104","author":[{"given":"Benjamin J.","family":"Lee","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2025,2,20]]},"reference":[{"key":"6221_CR1","doi-asserted-by":"publisher","first-page":"4838","DOI":"10.1182\/bloodadvances.2020002474","volume":"4","author":"K Laribi","year":"2020","unstructured":"Laribi K, Baugier de Materre A, Sobh M et al (2020) Blastic plasmacytoid dendritic cell neoplasms: results of an international survey on 398 adult patients. Blood Adv 4:4838\u20134848","journal-title":"Blood Adv"},{"issue":"7","key":"6221_CR2","doi-asserted-by":"publisher","first-page":"1006","DOI":"10.1016\/j.bbmt.2013.01.027","volume":"19","author":"MA Kharfan-Dabaja","year":"2013","unstructured":"Kharfan-Dabaja MA, Lazarus HM, Nishihori T, Mahfouz RA, Hamadani M (2013) Diagnostic and therapeutic advances in blastic plasmacytoid dendritic cell neoplasm: a focus on hematopoietic cell transplantation. Biol Blood Marrow Transpl 19(7):1006\u20131012","journal-title":"Biol Blood Marrow Transpl"},{"issue":"2","key":"6221_CR3","doi-asserted-by":"publisher","first-page":"239","DOI":"10.3324\/haematol.2012.072645","volume":"98","author":"L Pagano","year":"2013","unstructured":"Pagano L, Valentini CG, Pulsoni A et al (2013) Blastic plasmacytoid dendritic cell neoplasm with leukemic presentation: an Italian multicenter study. Haematologica 98(2):239\u2013246","journal-title":"Haematologica"},{"key":"6221_CR4","doi-asserted-by":"publisher","first-page":"156","DOI":"10.1158\/2159-8290.CD-16-0999","volume":"7","author":"J Montero","year":"2017","unstructured":"4, Montero J, Stephansky J, Cai T et al (2017) Blastic plasmacytoid dendritic cell neoplasm is dependent on BCL2 and sensitive to venetoclax. Cancer Discov 7:156\u2013164","journal-title":"Cancer Discov"},{"issue":"2","key":"6221_CR5","doi-asserted-by":"publisher","first-page":"E62","DOI":"10.1002\/ajh.26417","volume":"97","author":"N Gangat","year":"2022","unstructured":"Gangat N, Konopleva M, Patnaik MM et al (2022) Venetoclax and hypomethylating agents in older\/unfit patients with blastic plasmacytoid dendritic cell neoplasm. Am J Hematol 97(2):E62\u2013E67","journal-title":"Am J Hematol"},{"issue":"15","key":"6221_CR6","doi-asserted-by":"publisher","first-page":"1479","DOI":"10.1056\/NEJMc1808354","volume":"379","author":"ME Agha","year":"2018","unstructured":"Agha ME, Monaghan SA, Swerdlow SH (2018) Venetoclax in a patient with a blastic plasmacytoid dendritic-cell neoplasm. N Engl J Med 379(15):1479\u20131481","journal-title":"N Engl J Med"},{"key":"6221_CR7","unstructured":"TriNetX (2023) Publication Guidelines. Available at: https:\/\/trinetx.com\/trinetx-publicationguidelines\/. Accessed October 23"},{"issue":"17","key":"6221_CR8","doi-asserted-by":"publisher","first-page":"1628","DOI":"10.1056\/NEJMoa1815105","volume":"380","author":"N Pemmaraju","year":"2019","unstructured":"Pemmaraju N, Lane AA, Sweet KL et al (2019) Tagraxofusp in Blastic Plasmacytoid dendritic-cell neoplasm. N Engl J Med 380(17):1628\u20131637","journal-title":"N Engl J Med"}],"container-title":["Annals of Hematology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s00277-025-06221-4.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1007\/s00277-025-06221-4\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s00277-025-06221-4.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,4,25]],"date-time":"2025-04-25T10:10:54Z","timestamp":1745575854000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/s00277-025-06221-4"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,2,20]]},"references-count":8,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2025,3]]}},"alternative-id":["6221"],"URL":"https:\/\/doi.org\/10.1007\/s00277-025-06221-4","relation":{},"ISSN":["0939-5555","1432-0584"],"issn-type":[{"value":"0939-5555","type":"print"},{"value":"1432-0584","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,2,20]]},"assertion":[{"value":"19 November 2024","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"17 January 2025","order":2,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"20 February 2025","order":3,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"The study was performed using the TriNetX database that uses deidentified aggregate patient data for analysis. No protected health information or personal data are made available to the users of the platform. As a result, institutional review board approval was not required for this study.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethical approval"}},{"value":"The authors declare no competing interests.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}}]}}