Tagraxofusp maintenance post-hematopoietic stem cell transplantation provides long-term survival and manageable safety for a patient with blastic plasmacytoid dendritic cell neoplasm
- PMID: 40612021
- PMCID: PMC12213941
- DOI: 10.1016/j.lrr.2025.100518
Tagraxofusp maintenance post-hematopoietic stem cell transplantation provides long-term survival and manageable safety for a patient with blastic plasmacytoid dendritic cell neoplasm
Abstract
Presented here is the case of a 68-year-old woman with blastic plasmacytoid dendritic cell neoplasm (BPDCN) treated with tagraxofusp (TAG) maintenance therapy post-allogeneic hematopoietic stem cell transplantation (allo-HCT). Prior to allo-HCT, the patient was treated with hydroxyurea and mini-CVD (cyclophosphamide, vincristine, and dexamethasone alternating with methotrexate (Methotrexate) and cytarabine) + venetoclax + TAG for 5 cycles, which induced morphologic complete remission with minimal residual disease. After allo-HCT, the patient had persistent cytogenic abnormalities 45,XX,der(7)add(7)(p13)del(7)(q11.2q22)add(7)(q32),add(12)(p13),-15,del(16)(q23),-17,+22,+2mar[1]/46,XX[19], and was then treated with TAG maintenance therapy at 9 mg/kg on a 28-day cycle for 16 cycles. At mid-treatment (cycle 6 of 16 cycles of TAG) and prior to TAG discontinuation (due to travel burden), the patient was negative for minimal residual disease and was in complete remission. There was no high-grade toxicity or capillary leak syndrome. The patient remains in complete remission as of present day (>17 months CR). This is the first case study illustrating the feasibility of long-term TAG maintenance post-allo-HCT to control BPDCN.
Keywords: Allogeneic stem cell transplant; BPDCN; Blastic plasmacytoid dendritic cell neoplasm; CD123; Tagraxofusp.
© 2025 The Authors.
Conflict of interest statement
Declaration of Funding: The study from which this case was extracted was funded by Menarini Group. Declaration of Financial/Other Relationships: QB: honoraria from Binaytara Foundation; research funding from Acrotech, GSK, Pfizer, and Stemline. MK: Leadership with Immune Oncology (Board of Directors); institutional research funding from Abbvie, Allogene, AstraZeneca, Cellectis, Genentech, Gilead, ImmunoGen, Janssen, MEI Pharma, Pfizer, Precision Biosciences, Rafael Pharmaceuticals, Sanofi Aventis, Stemline Therapeutics; consulting/advisory role with AbbVie, AstraZeneca, Boehringer, F. Hoffman-La Roche, Genentech, Gilead, Janssen, Legend Biotech, MEI Pharma, Redona, Sanofi Aventis, Sellas, Stemline Therapeutics, Vincerx; intellectual property with Reata Pharmaceuticals; participation on a DSMB or advisory board for AbbVie, Auxenion GmbH, Bakx Therapeutics, Dark Blue Therapeutics, F. Hoffman-La Roche, Genentech, Gilead, Stemline Therapeutics, Vincerx. GA, JR, SAS, AA, URP, YN, REC, and GA have nothing to disclose. CH: grants or contracts from BioLine Rx and Incyte. EJS: royalties or licenses from Affimed, Takeda, and RegeNexus; consulting fees from New York Blood Center and Celaid Therapeutics; honoraria from Montefiore Medical Center, AcCELLerate Forum, ASTCT Corporate Council Meeting, Stanford BMT-CT Symposium, and CART Summit; support for attending meetings/travel from Dava Oncology, NMDP, Stanford BMT-CT Symposium, and IDRC; leadership role with NMDP; scientific advisor for Adaptimmune Unlimited, Navan Technologies, Axio Research, Zelluna Immunotherapy, and FibroBiologics. MQ: payments from pharmaceutical companies including Amgen, Angiocrine Bioscience, BioLineRx, Janssen, and NexImmune. NP: Institutional research grant from US Dept of Defense, NIH/NCI; Consultant/scientific advisory board/speaking for AbbVie, Aplastic Anemia & MDS International Foundation, Aptitude Health, Astellas Pharma US, Blueprint Medicines, Bristol-Myers Squibb Pharmaceuticals, CancerNet, CareDx, Celgene, Cimeio Therapeutics AG, ClearView Healthcare Partners, CTI BioPharma, Curio Science, Dava Oncology, EUSA Pharma, Harborside Press, Imedex, Immunogen, Intellisphere, Karyopharm, Magdalen Medical Publishing, Medscape, Menarini Group, Morphosys, Neopharm, Novartis Pharmaceuticals, OncLive, Pacylex, Patient Power, PeerView Institute for Medical Education, Pharma Essentia, Physician Education Resource (PER); Board of directors/management for Dan’s House of Hope; Leadership with ASH Committee on Communications, ASCO Cancer.Net Editorial Board; Licenses with Karger Publishers; Editorial board (unpaid) for HemOnc Times/Oncology Times.