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Review

Role of tagraxofusp in treating blastic plasmacytoid dendritic cell neoplasm (BPDCN)

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Pages 115-123 | Received 30 Sep 2019, Accepted 03 Dec 2019, Published online: 17 Dec 2019

References

  • Swerdlow SH, Campo E, Harris NL, et al. WHO classification of tumours of haematopoietic and lymphoid tissues. revised 4th ed. Lyon: International Agency for Research on Cancer (IARC); 2017. Available from: https://publications.iarc.fr/Book-And-Report-Series/Who-Iarc-Classification-Of-Tumours/Who-Classification-Of-Tumours-Of-Haematopoietic-And-Lymphoid-Tissues-2017
  • Sapienza MR, Abate F, Melle F, et al. Blastic plasmacytoid dendritic cell neoplasm: genomics mark epigenetic dysregulation as a primary therapeutic target. Haematologica. 2019 Apr;104(4):729–737.
  • Julia F, Petrella T, Beylot-Barry M, et al. Blastic plasmacytoid dendritic cell neoplasm: clinical features in 90 patients. Br J Dermatol. 2013 Sep;169(3):579–586.
  • Bekkenk MW, Jansen PM, Meijer CJ, et al. CD56+ hematological neoplasms presenting in the skin: a retrospective analysis of 23 new cases and 130 cases from the literature. Ann Oncol. 2004 Jul;15(7):1097–1108.
  • Khoury JD. Blastic plasmacytoid dendritic cell neoplasm. Curr Hematol Malig Rep. 2018;13(6):477–483.
  • Sukswai N, Aung PP, Yin CC, et al. Dual expression of TCF4 AND CD123 Is highly sensitive and specific for blastic plasmacytoid dendritic cell neoplasm. Am J Surg Pathol. 2019 Oct;43(10):1429–1437.
  • Leroux D, Mugneret F, Callanan M, et al. CD4(+), CD56(+) DC2 acute leukemia is characterized by recurrent clonal chromosomal changes affecting 6 major targets: a study of 21 cases by the Groupe Français de Cytogénétique Hématologique. Blood. 2002 Jun;99(11):4154–4159.
  • Menezes J, Acquadro F, Wiseman M, et al. Exome sequencing reveals novel and recurrent mutations with clinical impact in blastic plasmacytoid dendritic cell neoplasm. Leukemia. 2014 Apr;28(4):823–829.
  • Jardin F, Ruminy P, Parmentier F, et al. TET2 and TP53 mutations are frequently observed in blastic plasmacytoid dendritic cell neoplasm. Br J Haematol. 2011 May;153(3):413–416.
  • Feuillard J, Jacob MC, Valensi F, et al. Clinical and biologic features of CD4(+)CD56(+) malignancies. Blood. 2002 Mar;99(5):1556–1563.
  • Deotare U, Yee KW, Le LW, et al. Blastic plasmacytoid dendritic cell neoplasm with leukemic presentation: 10-Color flow cytometry diagnosis and HyperCVAD therapy. Am J Hematol. 2016 Mar;91(3):283–286.
  • Deotare U, Kim DD, Michelis FV, et al. Allogeneic hematopoietic stem cell transplantions in blastic plasmacytoid dendritic cell neoplasm in first complete remission: an effective therapy for a rare disease. Leuk Lymphoma. 2016;57(8):1942–1944.
  • Pagano L, Valentini CG, Pulsoni A, et al. Blastic plasmacytoid dendritic cell neoplasm with leukemic presentation: an Italian multicenter study. Haematologica. 2013 Feb;98(2):239–246.
  • Tsagarakis NJ, Kentrou NA, Papadimitriou KA, et al. Acute lymphoplasmacytoid dendritic cell (DC2) leukemia: results from the hellenic dendritic cell leukemia study group. Leuk Res. 2010 Apr;34(4):438–446.
  • Gruson B, Vaida I, Merlusca L, et al. L-asparaginase with methotrexate and dexamethasone is an effective treatment combination in blastic plasmacytoid dendritic cell neoplasm. Br J Haematol. 2013 Nov;163(4):543–545.
  • Martín-Martín L, López A, Vidriales B, et al. Classification and clinical behavior of blastic plasmacytoid dendritic cell neoplasms according to their maturation-associated immunophenotypic profile. Oncotarget. 2015 Aug;6(22):19204–19216.
  • Gilis L, Lebras L, Bouafia-Sauvy F, et al. Sequential combination of high dose methotrexate and L-asparaginase followed by allogeneic transplant: a first-line strategy for CD4+/CD56+ hematodermic neoplasm. Leuk Lymphoma. 2012 Aug;53(8):1633–1637.
  • Dalle S, Beylot-Barry M, Bagot M, et al. Blastic plasmacytoid dendritic cell neoplasm: is transplantation the treatment of choice? Br J Dermatol. 2010 Jan;162(1):74–79.
  • Taylor J, Haddadin M, Upadhyay VA, et al. Multicenter analysis of outcomes in blastic plasmacytoid dendritic cell neoplasm offers a pre-targeted therapy benchmark. Blood. 2019 Aug 22;134(8):678–687 .
  • Kharfan-Dabaja MA, Al Malki MM, Deotare U, et al. Haematopoietic cell transplantation for blastic plasmacytoid dendritic cell neoplasm: a North American multicentre collaborative study. Br J Haematol. 2017 12;179(5):781–789.
  • Kharfan-Dabaja MA, Lazarus HM, Nishihori T, et al. Diagnostic and therapeutic advances in blastic plasmacytoid dendritic cell neoplasm: a focus on hematopoietic cell transplantation. Biol Blood Marrow Transplant. 2013 Jul;19(7):1006–1012.
  • Aoki T, Suzuki R, Kuwatsuka Y, et al. Long-term survival following autologous and allogeneic stem cell transplantation for blastic plasmacytoid dendritic cell neoplasm. Blood. 2015 Jun;125(23):3559–3562.
  • CT J, Upchurch D, SJ S, et al. The interleukin-3 receptor alpha chain is a unique marker for human acute myelogenous leukemia stem cells. Leukemia. 2000 Oct;14(10):1777–1784.
  • Testa U, Pelosi E, Frankel A. CD 123 is a membrane biomarker and a therapeutic target in hematologic malignancies. Biomark Res. 2014 Feb;2(1):4.
  • Frankel A, Liu JS, Rizzieri D, et al. Phase I clinical study of diphtheria toxin-interleukin 3 fusion protein in patients with acute myeloid leukemia and myelodysplasia. Leuk Lymphoma. 2008 Mar;49(3):543–553.
  • Angelot-Delettre F, Roggy A, Frankel AE, et al. In vivo and in vitro sensitivity of blastic plasmacytoid dendritic cell neoplasm to SL-401, an interleukin-3 receptor targeted biologic agent. Haematologica. 2015 Feb;100(2):223–230.
  • Frankel AE, Seilles E, Biichle S, et al. Preclinical studies of SL-401, a targeted therapy directed to the interleukin-3 receptor (IL3-R), in blastic plasmacytoid dendritic cell neoplasm (BPDCN): potent activity in bpdcn cell lines, primary tumor, and in an in vivo model. Blood. 2013;122(21):3942.
  • Frankel AE, Woo JH, Ahn C, et al. Activity of SL-401, a targeted therapy directed to interleukin-3 receptor, in blastic plasmacytoid dendritic cell neoplasm patients. Blood. 2014 Jul;124(3):385–392.
  • Pemmaraju N, Lane AA, Sweet KL, et al. Tagraxofusp in blastic plasmacytoid dendritic-cell neoplasm. N Engl J Med. 2019 04;380(17):1628–1637.
  • Hall PD, Willingham MC, Kreitman RJ, et al. DT388-GM-CSF, a novel fusion toxin consisting of a truncated diphtheria toxin fused to human granulocyte-macrophage colony-stimulating factor, prolongs host survival in a SCID mouse model of acute myeloid leukemia. Leukemia. 1999 Apr;13(4):629–633.
  • Testa U, Riccioni R, Diverio D, et al. Interleukin-3 receptor in acute leukemia. Leukemia. 2004 Feb;18(2):219–226.
  • Zahran AM, Aly SS, Rayan A, et al. Survival outcomes of CD34. Oncotarget. 2018 Sep;9(75):34056–34065.
  • Schmidt JR, Rücker-Braun E, Heidrich K, et al. Pilot study on mass spectrometry-based analysis of the proteome of CD34⁺CD123⁺ progenitor cells for the identification of potential targets for immunotherapy in acute myeloid leukemia. Proteomes. 2018 Feb;6(1).
  • Lucas N, Duchmann M, Rameau P, et al. Biology and prognostic impact of clonal plasmacytoid dendritic cells in chronic myelomonocytic leukemia. Leukemia. 2019 Oct;33(10):2466–2480.
  • Mani R, Goswami S, Gopalakrishnan B, et al. The interleukin-3 receptor CD123 targeted SL-401 mediates potent cytotoxic activity against CD34. Haematologica. 2018;103(8):1288–1297.
  • Riccioni R, Diverio D, Riti V, et al. Interleukin (IL)-3/granulocyte macrophage-colony stimulating factor/IL-5 receptor alpha and beta chains are preferentially expressed in acute myeloid leukaemias with mutated FMS-related tyrosine kinase 3 receptor. Br J Haematol. 2009 Feb;144(3):376–387.
  • Broughton SE, Hercus TR, Nero TL, et al. A dual role for the N-terminal domain of the IL-3 receptor in cell signalling. Nat Commun. 2018;9(1):386.
  • Urieto JO, Liu T, Black JH, et al. Expression and purification of the recombinant diphtheria fusion toxin DT388IL3 for phase I clinical trials. Protein Expr Purif. 2004 Jan;33(1):123–133.
  • Black JH, McCubrey JA, Willingham MC, et al. Diphtheria toxin-interleukin-3 fusion protein (DT(388)IL3) prolongs disease-free survival of leukemic immunocompromised mice. Leukemia. 2003 Jan;17(1):155–159.
  • Ray A, Das DS, Song Y, et al. A novel agent SL-401 induces anti-myeloma activity by targeting plasmacytoid dendritic cells, osteoclastogenesis and cancer stem-like cells. Leukemia. 2017 12;31(12):2652–2660.
  • Frolova O, Benito J, Brooks C, et al. SL-401 and SL-501, targeted therapeutics directed at the interleukin-3 receptor, inhibit the growth of leukaemic cells and stem cells in advanced phase chronic myeloid leukaemia. Br J Haematol. 2014 Sep;166(6):862–874.
  • Frankel AE, Weir MA, Hall PD, et al. Diptheria toxin-interleukin 3 fusion protein therapy of patients with elderly or relapsed/refractory acute myeloid leukemia (AML). J Clin Oncol. 2006;24(18_suppl):6569.
  • Sweet KL, Pemmaraju N, Lane AA, et al. Lead-in stage results of a pivotal trial of SL-401, an interleukin-3 receptor (IL-3R) targeting biologic, in patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN) or acute myeloid leukemia (AML). Blood. 2015;126(23):3795.
  • Pemmaraju N, Ali H, Gupta V, et al. Results from ongoing phase 1/2 clinical trial of tagraxofusp (SL-401) in patients with intermediate or high risk relapsed/refractory myelofibrosis. J Clin Oncol. 2019;37(15_suppl):7058.
  • Patnaik MM, Ali H, Gupta V, et al. Results from ongoing phase 1/2 clinical trial of tagraxofusp (SL-401) in patients with relapsed/refractory chronic myelomonocytic leukemia (CMML). Blood. 2018;132(Suppl 1):1821.
  • Lane AA, Sweet KL, Wang ES, et al. Results from ongoing phase 1/2 trial of SL-401 as consolidation therapy in patients with acute myeloid leukemia (AML) in remission with minimal residual disease (MRD). Blood. 2017;130(Suppl 1):2583.
  • Martín-Martín L, Almeida J, Pomares H, et al. Blastic plasmacytoid dendritic cell neoplasm frequently shows occult central nervous system involvement at diagnosis and benefits from intrathecal therapy. Oncotarget. 2016 Mar;7(9):10174–10181.
  • black-box warning: capillary leak syndrome – FDA. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2018/761116s000lbl.pdf
  • Dhillon S. moxetumomab pasudotox: first global approval. Drugs. 2018 Nov;78(16):1763–1767.
  • Duvic M, Geskin L, Prince HM. Duration of response in cutaneous T-cell lymphoma patients treated with denileukin diftitox: results from 3 phase III studies. Clin Lymphoma Myeloma Leuk. 2013 Aug;13(4):377–384.
  • Jeong GH, Lee KH, Lee IR, et al. Incidence of capillary leak syndrome as an adverse effect of drugs in cancer patients: a systematic review and meta-analysis. J Clin Med. 2019;8:2.
  • Togami K, Pastika T, Stephansky J, et al. DNA methyltransferase inhibition overcomes diphthamide pathway deficiencies underlying CD123-targeted treatment resistance. J Clin Invest. 2019 Aug;129:5005–5019.
  • Stephansky J, Togami K, Ghandi M, et al. Resistance to SL-401 in AML and BPDCN is associated with loss of the diphthamide synthesis pathway enzyme dph1 and is reversible by azacitidine. Blood. 2017;130(Suppl 1):797.
  • Hu X, Wei H, Xiang L, et al. Methylation of the DPH1 promoter causes immunotoxin resistance in acute lymphoblastic leukemia cell line KOPN-8. Leuk Res. 2013 Nov;37(11):1551–1556.
  • Sun W, Liu H, Kim Y, et al. First pediatric experience of SL-401, a CD123-targeted therapy, in patients with blastic plasmacytoid dendritic cell neoplasm: report of three cases. J Hematol Oncol. 2018 May;11(1):61.
  • Pemmaraju N, Utengen A, Gupta V, et al. Blastic plasmacytoid dendritic cell neoplasm (BPDCN) on social media: #BPDCN-increasing exposure over two years since inception of a disease-specific twitter community. Curr Hematol Malig Rep. 2018;13(6):581–587.
  • Montero J, Stephansky J, Cai T, et al. Blastic plasmacytoid dendritic cell neoplasm is dependent on BCL2 and sensitive to venetoclax. Cancer Discov. 2017 02;7(2):156–164.
  • DiNardo CD, Rausch CR, Benton C, et al. Clinical experience with the BCL2-inhibitor venetoclax in combination therapy for relapsed and refractory acute myeloid leukemia and related myeloid malignancies. Am J Hematol. 2018;93(3):401–407.
  • Grushchak S, Joy C, Gray A, et al. Novel treatment of blastic plasmacytoid dendritic cell neoplasm: a case report. Medicine (Baltimore). 2017 Dec;96(51):e9452.
  • Agha ME, Monaghan SA, Swerdlow SH. Venetoclax in a patient with a blastic plasmacytoid dendritic-cell neoplasm. N Engl J Med. 2018 Oct;379(15):1479–1481.
  • Pemmaraju N, Konopleva M, Lane AA. More on blastic plasmacytoid dendritic-cell neoplasms. N Engl J Med. 2019 02;380(7):695–696.
  • Micol JB, Pilorge S, Ghez D. More on blastic plasmacytoid dendritic-cell neoplasms. N Engl J Med. 2019;380(7):696.
  • Zhang Q, Cai T, Han L, et al. Pre-clinical efficacy of CD123-targeting antibody-drug conjugate IMGN632 in blastic plasmacytoid dendritic cell neoplasm (BPDCN) models. Blood. 2018;132(Suppl 1):3956.
  • Daver NG, Erba HP, Papadantonakis N, et al. A phase I, first-in-human study evaluating the safety and preliminary antileukemia activity of IMGN632, a novel CD123-targeting antibody-drug conjugate, in patients with relapsed/refractory acute myeloid leukemia and other CD123-positive hematologic malignancies. Blood. 2018;132(Suppl 1):27.
  • Sapienza MR, Fuligni F, Agostinelli C, et al. Molecular profiling of blastic plasmacytoid dendritic cell neoplasm reveals a unique pattern and suggests selective sensitivity to NF-kB pathway inhibition. Leukemia. 2014 Aug;28(8):1606–1616.
  • Aung PP, Sukswai N, Nejati R, et al. PD1/PD-L1 expression in blastic plasmacytoid dendritic cell neoplasm. Cancers (Basel). 2019 May;11(5):1-12.
  • Sakamoto K, Katayama R, Asaka R, et al. Recurrent 8q24 rearrangement in blastic plasmacytoid dendritic cell neoplasm: association with immunoblastoid cytomorphology, MYC expression, and drug response. Leukemia. 2018 12;32(12):2590–2603.
  • Pemmaraju NNP. Potential therapeutic strategies for blastic plasmacytoid dendritic cell neoplasm (BPDCN): CD123 and beyond. Curr Hematol Malig Rep. 2017 12;12(6):510–512.
  • Emadali A, Hoghoughi N, Duley S, et al. Haploinsufficiency for NR3C1, the gene encoding the glucocorticoid receptor, in blastic plasmacytoid dendritic cell neoplasms. Blood. 2016;127(24):3040–3053.
  • Ceribelli M, Hou ZE, Kelly PN, et al. A druggable tcf4- and brd4-dependent transcriptional network sustains malignancy in blastic plasmacytoid dendritic cell neoplasm. Cancer Cell. 2016 Nov;30(5):764–778.

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