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. 2023 Nov 28;7(22):7007-7016.
doi: 10.1182/bloodadvances.2023011308.

Allogeneic hematopoietic cell transplantation for blastic plasmacytoid dendritic cell neoplasm: a CIBMTR analysis

Hemant S Murthy  1 , Mei-Jie Zhang  2   3 , Karen Chen  3 , Sairah Ahmed  4 , Uday Deotare  5 , Siddhartha Ganguly  6 , Ankit Kansagra  7 , Fotios V Michelis  8 , Taiga Nishihori  9   10 , Mrinal Patnaik  11 , Muhammad Bilal Abid  12 , Mahmoud Aljurf  13 , Yasuyuki Arai  14 , Ulrike Bacher  15 , Talha Badar  1 , Sherif M Badawy  16   17 , Karen Ballen  18 , Minoo Battiwalla  19 , Amer Beitinjaneh  20 , Nelli Bejanyan  9 , Vijaya Raj Bhatt  21 , Valerie I Brown  22 , Rodrigo Martino  23 , Jean-Yves Cahn  24 , Paul Castillo  25 , Jan Cerny  26 , Saurabh Chhabra  3   27 , Edward Copelan  28 , Andrew Daly  29 , Bhagirathbhai Dholaria  30 , Miguel Angel Diaz Perez  31 , César O Freytes  32 , Michael R Grunwald  28 , Shahrukh Hashmi  33   34 , Gerhard C Hildebrandt  35 , Omer Jamy  36 , Jacinth Joseph  37 , Christopher G Kanakry  38 , Nandita Khera  39 , Maxwell M Krem  40 , Yachiyo Kuwatsuka  41 , Hillard M Lazarus  42 , Lazaros J Lekakis  20 , Hongtao Liu  43 , Dipenkumar Modi  44 , Pashna N Munshi  45 , Alberto Mussetti  46   47 , Neil Palmisiano  48 , Sagar S Patel  49 , David A Rizzieri  50 , Sachiko Seo  51 , Mithun Vinod Shah  11 , Akshay Sharma  52 , Melhm Sohl  53 , Scott R Solomon  54 , Matthew Ulrickson  55 , Celalettin Ustun  56 , Marjolein van der Poel  57 , Leo F Verdonck  58 , John L Wagner  59 , Trent Wang  20 , Baldeep Wirk  60 , Amer Zeidan  61 , Mark Litzow  11 , Partow Kebriaei  62 , Christopher S Hourigan  63 , Daniel J Weisdorf  64 , Wael Saber  3 , Mohamed A Kharfan-Dabaja  1
Affiliations

Allogeneic hematopoietic cell transplantation for blastic plasmacytoid dendritic cell neoplasm: a CIBMTR analysis

Hemant S Murthy et al. Blood Adv. .

Abstract

Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare hematological malignancy with a poor prognosis and considered incurable with conventional chemotherapy. Small observational studies reported allogeneic hematopoietic cell transplantation (allo-HCT) offers durable remissions in patients with BPDCN. We report an analysis of patients with BPDCN who received an allo-HCT, using data reported to the Center for International Blood and Marrow Transplant Research (CIBMTR). We identified 164 patients with BPDCN from 78 centers who underwent allo-HCT between 2007 and 2018. The 5-year overall survival (OS), disease-free survival (DFS), relapse, and nonrelapse mortality (NRM) rates were 51.2% (95% confidence interval [CI], 42.5-59.8), 44.4% (95% CI, 36.2-52.8), 32.2% (95% CI, 24.7-40.3), and 23.3% (95% CI, 16.9-30.4), respectively. Disease relapse was the most common cause of death. On multivariate analyses, age of ≥60 years was predictive for inferior OS (hazard ratio [HR], 2.16; 95% CI, 1.35-3.46; P = .001), and higher NRM (HR, 2.19; 95% CI, 1.13-4.22; P = .02). Remission status at time of allo-HCT (CR2/primary induction failure/relapse vs CR1) was predictive of inferior OS (HR, 1.87; 95% CI, 1.14-3.06; P = .01) and DFS (HR, 1.75; 95% CI, 1.11-2.76; P = .02). Use of myeloablative conditioning with total body irradiation (MAC-TBI) was predictive of improved DFS and reduced relapse risk. Allo-HCT is effective in providing durable remissions and long-term survival in BPDCN. Younger age and allo-HCT in CR1 predicted for improved survival, whereas MAC-TBI predicted for less relapse and improved DFS. Novel strategies incorporating allo-HCT are needed to further improve outcomes.

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Conflict of interest statement

Conflict-of-interest disclosure: H.S.M. reports advisory board participation from CRISPR Therapeutics, Senti Biosciences, Jazz Pharmaceuticals, and Incyte Corporation. S.A. reports compensation (research funding) from Seattle Genetics, Merck, Xencor, and Tessa Therapeutics, and has a membership on Tessa Therapeutic’s advisory committee. S.G. reports advisory board participation for Bristol Myers Squibb, Sanofi, Astellas, Daiichi Sankyo, Kite Pharma, Janssen, and AstraZeneca, and speaker’s bureau participation for Seattle Genetics. T.N. reports clinical trial support (institution) from Novartis and clinical trial support (drug only supply to the institution) from Karyopharm. M.B. reports research support to institution from Novartis. M.R.G. reports receiving consulting fees from AbbVie, Amgen, Astellas, Blueprint Medicines, Bristol Myers Squibb, Cardinal Health, CTI BioPharma, Daiichi Sankyo, Gamida Cell, Genentech, Gilead, GlaxoSmithKline/Sierra Oncology, Incyte, Invitae, Jazz, Karius, Novartis, Ono Pharmaceutical, Pfizer, Pharmacosmos, Premier, Servier/Agios, and Stemline Therapeutics; research support from Incyte and Janssen; and stock ownership for Medtronic. N.K. reports honorarium from Optum for educational event. H.L. reports advisory board meeting participation for Agios, Pfizer, Nkarta, CTI Biopharm, and BeiGene; research support from Bristol Myers Squibb, Karyopharm, and Miltenyi Biotec; being a consultant for NGM BioPharma; and being a speaker/lecturer for Society of Immunotherapy of Cancer and Chinese American Hematologist and Oncologist Network. M.U. reports advisory board participation for Stemline and Gilead. N.B. reports advisory board participation or consultancy services for Magenta Therapeutics, Medexus Pharma, CTI BioPharma, CareDx and Sanofi. V.R.B. reports safety monitoring committee participation for Protagonist; consulting fees from Genentech, Rigel, Agios, Incyte, Servier Pharmaceuticals LLC, Omeros, Takeda, Partnership for Health Analytic Research, LLC (which in turn, receives funds from Jazz Pharmaceuticals), and AbbVie; research funding (institutional) from AbbVie, Pfizer, Incyte, Jazz, Tolero Pharmaceuticals, Inc, and National Marrow Donor Program; and drug support (institutional) from Oncoceutics for a trial. S.C. reports research funding (institutional) from Bristol Myers Squibb, Amgen, Janssen, Novartis, Syndax, Ionis, Sanofi, and GlaxoSmithKline, and honorarium from GlaxoSmithKline, Sanofi (advisory board), and Omeros (speaker’s bureau). E.C. reports consultative council participation for Amgen. B.D. reports research funding (institutional) from Janssen, Angiocrine, Pfizer, Poseida, MEI, and Orcabio, and consultancy/adviser for Jazz, Gamida Cell, MJH BioScience, Arivan Research, and BEAM Therapeutics. J.J. reports advisory board meeting participation for Elzonris in January 2022. M.M.K. reports being a consultant for Secura Bio (unrelated to current study). N.P. reports consultancy for AbbVie, Genentech, Takeda, and Foundation One, and research support from AbbVie, Genentech, and Incyte. D.M. reports advisory board participation for MorphoSys and Seagen; research funding from Genentech, MorphoSys, and ADC Therapeutics; and honorarium from AstraZeneca. P.N.M. reports being a consultant for Kite and speaker’s bureau participation for Incyte. A.M. reports an investigator grant from Gilead. D.A.R. reports advisory board and speaker’s bureau participation for Stemline Therapeutics. A.S. reports being a consultant for Spotlight Therapeutics in 2020, Medexus Inc in 2021, and Vertex Pharmaceuticals in 2021 and 2022; research funding from CRISPR Therapeutics in 2021 and 2022; and research collaboration with Magenta Therapeutics since 2021. C.U. reports honoraria for advisory board or speaker’s bureau participation from Blueprint and Takeda (unrelated to current study). P.K. reports compensation (advisory board participation) for Jazz, Kite, and Pfizer, and clinical trial support from Amgen and Ziopharm. C.S.H. reports a government conflict of interest. The remaining authors declare no competing financial interests.

Figures

None
Graphical abstract
Figure 1.
Figure 1.
OS by age and remission status. Rel, relapse; PIF, primary induction failure.
Figure 2.
Figure 2.
DFS by age and remission status. Rel, relapse; PIF, primary induction failure.
See this image and copyright information in PMC

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