Session: 732. Allogeneic Transplantation: Disease Response and Comparative Treatment Studies: Poster III
Hematology Disease Topics & Pathways:
Biological therapies, Therapies, Transplantation
Methods: In this retrospective single-center analysis, we evaluated outcomes of 31 BPDCN patients treated with allo-HCT between September 2000 and June 2023 at our institution.
Results: Median age of our cohort was 52 years [range 16-76 y]. Patient characteristics are summarized in the attached Table. Only 6 patients (19%) had their disease confined to skin and 4 (13%) had CNS/CSF involvement at diagnosis. Four patients (13%) had prior hematologic malignancies (HM), and 9 patients (29%) had a cytogenetic abnormality at baseline. Thirteen (42%) patients, who were evaluated by a next-generation sequencing Leukemia Mutation Panel, had TET2 mutation. Median time from diagnosis to HCT was 6.2 (3.2-42.4) months. Eight (26%) patients received tagraxofusp, matched unrelated donor was the most common donor type (39%), and fludarabine + busulfan-based conditioning was used in 17 (55%) patients. Post-transplant cyclophosphamide (PTCy) was used for GVHD prophylaxis in 23 (74%) patients. Median follow up was 23.9 (0.9-119.9) months. One-hundred day and 1-year non-relapse mortality was 9.7% and 23.1%, respectively. Grade 2 acute GVHD was seen in 9 patients (29%), and no grade 3-4 GVHD has been seen so far. Limited or extensive chronic GVHD was seen in 6 (21%) of 28 evaluable patients. Only 6 of the 31 (19%) allo-HCT patients progressed after HCT. Two-year PFS and OS was 56% and 67%, respectively. Patients receiving allo-HCT in first remission had significantly better median PFS (not reached vs. 11.3 months; p=0.034) and OS (not reached vs. 12.6 months; p=0.045).
Conclusions: These results demonstrate the safety and efficacy of allo-HCT in BPDCN. Patients undergoing allo-HCT in first remission had significantly better outcomes. Prospective studies are needed to better define the role of allo-HCT in BPDCN.
Disclosures: Qazilbash: Amgen: Research Funding; Angiocrine: Research Funding; Janssen: Research Funding; Bioline: Other: Advisory board; NexImmune: Research Funding. Kebriaei: Jazz: Consultancy, Honoraria; Pfizer: Consultancy, Honoraria. Khouri: Pfizer: Research Funding. Rezvani: Takeda: Patents & Royalties; Affimed: Other: License agreement. Nieto: Secura Bio: Research Funding; Astra Zeneca: Research Funding; Affimed: Research Funding. Srour: Orca Bio: Research Funding. Saini: GSK: Research Funding; Panbela Theraputics: Research Funding. Konopleva: AbbVie, Ablynx, Allogene, AstraZeneca, Cellectis, Daiichi, FortySeven, Genentech, Gilead, Immunogen, MEI Pharma, Precision Biosciences, Rafael Pharmaceutical, Sanofi Aventis, Stemline-Menarini: Research Funding; AbbVie, AstraZeneca, Genentech, Gilead, Janssen, MEI Pharma, Sanofi Aventis, Stemline-Menarini, Vincerx: Consultancy; Reata Pharmaceuticals.: Current holder of stock options in a privately-held company, Patents & Royalties. Champlin: Cell Source: Research Funding; Kadmon: Consultancy; Arog: Consultancy; Takeda Corporation: Patents & Royalties; Johnson & Johnson/Janssen: Consultancy; Omeros: Consultancy; Actinium Pharmaceuticals: Consultancy; Orca Bio: Consultancy. Shpall: Syena: Other: License agreement; Takeda: Other: License agreement; Axio: Membership on an entity's Board of Directors or advisory committees; Fibrobiologics: Membership on an entity's Board of Directors or advisory committees; Celaid Therapeutics: Membership on an entity's Board of Directors or advisory committees; Navan: Membership on an entity's Board of Directors or advisory committees; Adaptimmune: Membership on an entity's Board of Directors or advisory committees; NY Blood Center: Membership on an entity's Board of Directors or advisory committees; Affimed: Other: License agreement. Bashir: Pfizer: Research Funding; GSK: Research Funding; Acrotech: Research Funding; Stemline: Research Funding. Pemmaraju: PharmaEssentia: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Incyte: Consultancy, Membership on an entity's Board of Directors or advisory committees; EUSA Pharma: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; ImmunoGen: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Patient Power: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Menarini Group: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Karger Publishers: Other: Licenses; ASCO Cancer.Net Editorial Board: Other: Leadership; Novartis Pharmaceuticals: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; United States Department of Defense (DOD): Research Funding; Dan's House of Hope: Membership on an entity's Board of Directors or advisory committees; ClearView Healthcare Partners: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Medscape: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; CTI BioPharma: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Physician Education Resource (PER): Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; OncLive: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; ASH Committee on Communications: Other: Leadership; Curio Science: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Stemline: Consultancy, Membership on an entity's Board of Directors or advisory committees; Protagonist Therapeutics, Inc.: Consultancy, Membership on an entity's Board of Directors or advisory committees; National Institute of Health/National Cancer Institute (NIH/NCI): Research Funding; PeerView Institute for Medical Education: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Neopharm: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Pacylex: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Aplastic Anemia & MDS International Foundation: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; CancerNet: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; CareDx: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Celgene: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Dava Oncology: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Harborside Press: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Imedex: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Intellisphere: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Magdalen Medical Publishing: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Cimeio Therapeutics AG: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; BMS: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Astellas: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Blueprint: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Aptitude Health: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; AbbVie: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; HemOnc Times/Oncology Times: Other: Uncompensated; Bristol Myers Squibb Pharmaceuticals: Consultancy, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau.