-Author name in bold denotes the presenting author
-Asterisk * with author name denotes a Non-ASH member
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4988 Patient Characteristics and Outcomes of Allogeneic Hematopoietic Stem Cell Transplantation for Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)

Program: Oral and Poster Abstracts
Session: 732. Allogeneic Transplantation: Disease Response and Comparative Treatment Studies: Poster III
Hematology Disease Topics & Pathways:
Biological therapies, Therapies, Transplantation
Monday, December 11, 2023, 6:00 PM-8:00 PM

Muzaffar H. Qazilbash, MD1, Hina N. Khan, MD1,2, Denái R. Milton, MS3*, Oren Pasvolsky, MD1,4,5*, Mark R. Tanner, PhD1*, Uday R. Popat, MD1, Partow Kebriaei, MD1, Chitra Hosing, MD1, Issa F. Khouri, MD1, Katy Rezvani, MD6, Yago Nieto, MD, PhD1, Betul Oran, MD, MS1*, Samer A. Srour, MD1, Neeraj Y. Saini, MD1, Amanda L. Olson, MD1, Sairah Ahmed, MD1,7, Gheath Alatrash, PhD, DO1, Gabriela Rondon, MD1, Marina Y. Konopleva8, Richard E. Champlin, MD1, Elizabeth J. Shpall, MD1, Qaiser Bashir, MD1 and Naveen Pemmaraju, MD8

1Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, TX
2Department of Hematology and Oncology, The University of Texas Health Science Center McGovern Medical School, Houston, TX
3Department of Biostatistics, The University of Texas MD Anderson Cancer Center, Houston, TX
4Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel
5Institute of Hematology, Davidoff Cancer Center, Rabin Medical Center, Petah-Tikva, Israel
6Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas M D Anderson Cancer Center, Houston, TX
7Department of Lymphoma and Myeloma, The University of Texas MD Anderson Cancer Center, Houston, TX
8Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX

Background: Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare, aggressive hematologic malignancy arising from plasmacytoid dendritic cells. It commonly presents as skin lesions with or without bone marrow, lymph node, central nervous system (CNS), or systemic involvement. Limited retrospective data have shown durable remissions after allogeneic (allo) hematopoietic cell transplantation (HCT).

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.

*signifies non-member of ASH