Genome-wide DNA methylation-analysis of blastic plasmacytoid dendritic cell neoplasm identifies distinct molecular features.


Journal

Leukemia
ISSN: 1476-5551
Titre abrégé: Leukemia
Pays: England
ID NLM: 8704895

Informations de publication

Date de publication:
10 Apr 2024
Historique:
received: 01 11 2023
accepted: 28 03 2024
revised: 27 03 2024
medline: 11 4 2024
pubmed: 11 4 2024
entrez: 10 4 2024
Statut: aheadofprint

Résumé

Blastic plasmacytoid dendritic cell neoplasm (BPDCN) constitutes a rare and aggressive malignancy originating from plasmacytoid dendritic cells (pDCs) with a primarily cutaneous tropism followed by dissemination to the bone marrow and other organs. We conducted a genome-wide analysis of the tumor methylome in an extended cohort of 45 BPDCN patients supplemented by WES and RNA-seq as well as ATAC-seq on selected cases. We determined the BPDCN DNA methylation profile and observed a dramatic loss of DNA methylation during malignant transformation from early and mature DCs towards BPDCN. DNA methylation profiles further differentiate between BPDCN, AML, CMML, and T-ALL exhibiting the most striking global demethylation, mitotic stress, and merely localized DNA hypermethylation in BPDCN resulting in pronounced inactivation of tumor suppressor genes by comparison. DNA methylation-based analysis of the tumor microenvironment by MethylCIBERSORT yielded two, prognostically relevant clusters (IC1 and IC2) with specific cellular composition and mutational spectra. Further, the transcriptional subgroups of BPDCN (C1 and C2) differ by DNA methylation signatures in interleukin/inflammatory signaling genes but also by higher transcription factor activity of JAK-STAT and NFkB signaling in C2 in contrast to an EZH2 dependence in C1-BPDCN. Our integrative characterization of BPDCN offers novel molecular insights and potential diagnostic applications.

Identifiants

pubmed: 38600314
doi: 10.1038/s41375-024-02240-8
pii: 10.1038/s41375-024-02240-8
doi:

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Informations de copyright

© 2024. The Author(s).

Références

Chaperot L, Bendriss N, Manches O, Gressin R, Maynadie M, Trimoreau F, et al. Identification of a leukemic counterpart of the plasmacytoid dendritic cells. Blood. 2001;97:3210–7.
pubmed: 11342451 doi: 10.1182/blood.V97.10.3210
Arber DA, Orazi A, Hasserjian RP, Borowitz MJ, Calvo KR, Kvasnicka HM, et al. International consensus classification of myeloid neoplasms and acute leukemias: integrating morphologic, clinical, and genomic data. Blood. 2022;140:1200–28.
pubmed: 35767897 pmcid: 9479031 doi: 10.1182/blood.2022015850
Khoury JD, Solary E, Abla O, Akkari Y, Alaggio R, Apperley JF, et al. The 5th edition of the World Health Organization Classification of haematolymphoid tumours: myeloid and histiocytic/dendritic neoplasms. Leukemia. 2022;36:1703–19.
pubmed: 35732831 pmcid: 9252913 doi: 10.1038/s41375-022-01613-1
Renosi F, Roggy A, Giguelay A, Soret L, Viailly PJ, Cheok M, et al. Transcriptomic and genomic heterogeneity in blastic plasmacytoid dendritic cell neoplasms: from ontogeny to oncogenesis. Blood Adv. 2021;5:1540–51.
pubmed: 33687433 pmcid: 7948279 doi: 10.1182/bloodadvances.2020003359
Carmona-Saez P, Varela N, Luque MJ, Toro-Dominguez D, Martorell-Marugan J, Alarcon-Riquelme ME, et al. Metagene projection characterizes GEN2.2 and CAL-1 as relevant human plasmacytoid dendritic cell models. Bioinformatics. 2017;33:3691–5.
pubmed: 28961902 doi: 10.1093/bioinformatics/btx502
Künstner A, Schwarting J, Witte HM, Bernard V, Stölting S, Kusch K, et al. Integrative molecular profiling identifies two molecularly and clinically distinct subtypes of blastic plasmacytoid dendritic cell neoplasm. Blood Cancer J. 2022;12:101.
pubmed: 35788129 pmcid: 9253119 doi: 10.1038/s41408-022-00699-1
Laribi K, Baugier de Materre A, Sobh M, Cerroni L, Valentini CG, Aoki T, et al. Blastic plasmacytoid dendritic cell neoplasms: results of an international survey on 398 adult patients. Blood Adv. 2020;4:4838–48.
pubmed: 33027528 pmcid: 7556130 doi: 10.1182/bloodadvances.2020002474
Togami K, Chung SS, Madan V, Booth CAG, Kenyon CM, Cabal-Hierro L, et al. Sex-biased ZRSR2 mutations in myeloid malignancies impair plasmacytoid dendritic cell activation and apoptosis. Cancer Discov. 2022;12:522–41.
Xiao W, Chan A, Waarts MR, Mishra T, Liu Y, Cai SF, et al. Plasmacytoid dendritic cell expansion defines a distinct subset of RUNX1-mutated acute myeloid leukemia. Blood. 2021;137:1377–91.
pubmed: 32871587 pmcid: 7955409 doi: 10.1182/blood.2020007897
Zalmai L, Viailly PJ, Biichle S, Cheok M, Soret L, Angelot-Delettre F, et al. Plasmacytoid dendritic cells proliferation associated with acute myeloid leukemia: phenotype profile and mutation landscape. Haematologica. 2021;106:3056–66.
Sangle NA, Schmidt RL, Patel JL, Medeiros LJ, Agarwal AM, Perkins SL, et al. Optimized immunohistochemical panel to differentiate myeloid sarcoma from blastic plasmacytoid dendritic cell neoplasm. Mod Pathol. 2014;27:1137–43.
pubmed: 24390220 doi: 10.1038/modpathol.2013.238
Garnache-Ottou F, Vidal C, Biichle S, Renosi F, Poret E, Pagadoy M, et al. How should we diagnose and treat blastic plasmacytoid dendritic cell neoplasm patients? Blood Adv. 2019;3:4238–51.
pubmed: 31869411 pmcid: 6929390 doi: 10.1182/bloodadvances.2019000647
Roos-Weil D, Dietrich S, Boumendil A, Polge E, Bron D, Carreras E, et al. Stem cell transplantation can provide durable disease control in blastic plasmacytoid dendritic cell neoplasm: a retrospective study from the European Group for Blood and Marrow Transplantation. Blood. 2013;121:440–6.
pubmed: 23203822 doi: 10.1182/blood-2012-08-448613
Aoki T, Suzuki R, Kuwatsuka Y, Kako S, Fujimoto K, Taguchi J, et al. Long-term survival following autologous and allogeneic stem cell transplantation for blastic plasmacytoid dendritic cell neoplasm. Blood. 2015;125:3559–62.
pubmed: 25918345 doi: 10.1182/blood-2015-01-621268
Pemmaraju N, Lane AA, Sweet KL, Stein AS, Vasu S, Blum W, et al. Tagraxofusp in blastic plasmacytoid dendritic-cell neoplasm. N. Engl J Med. 2019;380:1628–37.
pubmed: 31018069 doi: 10.1056/NEJMoa1815105
Capper D, Jones DTW, Sill M, Hovestadt V, Schrimpf D, Sturm D, et al. DNA methylation-based classification of central nervous system tumours. Nature. 2018;555:469–74.
pubmed: 29539639 pmcid: 6093218 doi: 10.1038/nature26000
Koelsche C, Schrimpf D, Stichel D, Sill M, Sahm F, Reuss DE, et al. Sarcoma classification by DNA methylation profiling. Nat Commun. 2021;12:498.
pubmed: 33479225 pmcid: 7819999 doi: 10.1038/s41467-020-20603-4
Arber DA, Orazi A, Hasserjian R, Thiele J, Borowitz MJ, Le Beau MM, et al. The 2016 revision to the World Health Organization classification of myeloid neoplasms and acute leukemia. Blood. 2016;127:2391–405.
pubmed: 27069254 doi: 10.1182/blood-2016-03-643544
Pemmaraju N, Kantarjian HM, Khoury JD, Loghavi S, O’Brien S, Cortes JE. et al. Blastic plasmacytoid dendritic cell neoplasm (BPDCN) commonly presents in the setting of prior or concomitant hematologic malignancies (PCHM): patient characteristics and outcomes in the rapidly evolving modern targeted therapy era. Blood. 2019;134:2723.
doi: 10.1182/blood-2019-132185
Batta K, Bossenbroek HM, Pemmaraju N, Wilks DP, Chasty R, Dennis M, et al. Divergent clonal evolution of blastic plasmacytoid dendritic cell neoplasm and chronic myelomonocytic leukemia from a shared TET2-mutated origin. Leukemia. 2021;35:3299–303.
pubmed: 33833384 pmcid: 8550946 doi: 10.1038/s41375-021-01228-y
Brunetti L, Di Battista V, Venanzi A, Schiavoni G, Martelli MP, Ascani S, et al. Blastic plasmacytoid dendritic cell neoplasm and chronic myelomonocytic leukemia: a shared clonal origin. Leukemia. 2017;31:1238–40.
pubmed: 28111467 doi: 10.1038/leu.2017.38
Chakravarthy A, Furness A, Joshi K, Ghorani E, Ford K, Ward MJ, et al. Pan-cancer deconvolution of tumour composition using DNA methylation. Nat Commun. 2018;9:3220.
pubmed: 30104673 pmcid: 6089972 doi: 10.1038/s41467-018-05570-1
Guidry K, Vasudevaraja V, Labbe K, Mohamed H, Serrano J, Guidry BW, et al. DNA methylation profiling identifies subgroups of lung adenocarcinoma with distinct immune cell composition, DNA methylation age, and clinical outcome. Clin Cancer Res. 2022;28:3824–35.
pubmed: 35802677 doi: 10.1158/1078-0432.CCR-22-0391
Pidsley R, Zotenko E, Peters TJ, Lawrence MG, Risbridger GP, Molloy P, et al. Critical evaluation of the Illumina MethylationEPIC BeadChip microarray for whole-genome DNA methylation profiling. Genome Biol. 2016;17:208.
pubmed: 27717381 pmcid: 5055731 doi: 10.1186/s13059-016-1066-1
Zhang H, Polavarapu VK, Xing P, Zhao M, Mathot L, Zhao L, et al. Profiling chromatin accessibility in formalin-fixed paraffin-embedded samples. Genome Res. 2022;32:150–61.
pubmed: 34261731 pmcid: 8744681 doi: 10.1101/gr.275269.121
Roy R, Ramamoorthy S, Shapiro BD, Kaileh M, Hernandez D, Sarantopoulou D, et al. DNA methylation signatures reveal that distinct combinations of transcription factors specify human immune cell epigenetic identity. Immunity. 2021;54:2465–80.e2465.
pubmed: 34706222 pmcid: 9190145 doi: 10.1016/j.immuni.2021.10.001
Zhang X, Ulm A, Somineni HK, Oh S, Weirauch MT, Zhang HX, et al. DNA methylation dynamics during ex vivo differentiation and maturation of human dendritic cells. Epigenetics Chromatin. 2014;7:21.
pubmed: 25161698 pmcid: 4144987 doi: 10.1186/1756-8935-7-21
Tyner JW, Tognon CE, Bottomly D, Wilmot B, Kurtz SE, Savage SL, et al. Functional genomic landscape of acute myeloid leukaemia. Nature. 2018;562:526–31.
pubmed: 30333627 pmcid: 6280667 doi: 10.1038/s41586-018-0623-z
Palomo L, Malinverni R, Cabezon M, Xicoy B, Arnan M, Coll R, et al. DNA methylation profile in chronic myelomonocytic leukemia associates with distinct clinical, biological and genetic features. Epigenetics. 2018;13:8–18.
pubmed: 29160764 pmcid: 5837079 doi: 10.1080/15592294.2017.1405199
Liu Y, Easton J, Shao Y, Maciaszek J, Wang Z, Wilkinson MR, et al. The genomic landscape of pediatric and young adult T-lineage acute lymphoblastic leukemia. Nat Genet. 2017;49:1211–8.
pubmed: 28671688 pmcid: 5535770 doi: 10.1038/ng.3909
Griffin GK, Booth CAG, Togami K, Chung SS, Ssozi D, Verga JA, et al. Ultraviolet radiation shapes dendritic cell leukaemia transformation in the skin. Nature. 2023;618:834–41.
pubmed: 37286599 pmcid: 10284703 doi: 10.1038/s41586-023-06156-8
Duran-Ferrer M, Clot G, Nadeu F, Beekman R, Baumann T, Nordlund J, et al. The proliferative history shapes the DNA methylome of B-cell tumors and predicts clinical outcome. Nat Cancer. 2020;1:1066–81.
pubmed: 34079956 pmcid: 8168619 doi: 10.1038/s43018-020-00131-2
Yu M, Hazelton WD, Luebeck GE, Grady WM. Epigenetic aging: more than just a clock when it comes to cancer. Cancer Res. 2020;80:367–74.
pubmed: 31694907 doi: 10.1158/0008-5472.CAN-19-0924
Bell CG, Lowe R, Adams PD, Baccarelli AA, Beck S, Bell JT, et al. DNA methylation aging clocks: challenges and recommendations. Genome Biol. 2019;20:249.
pubmed: 31767039 pmcid: 6876109 doi: 10.1186/s13059-019-1824-y
Udtha M, Lee SJ, Alam R, Coombes K, Huff V. Upregulation of c-MYC in WT1-mutant tumors: assessment of WT1 putative transcriptional targets using cDNA microarray expression profiling of genetically defined Wilms’ tumors. Oncogene. 2003;22:3821–6.
pubmed: 12802290 doi: 10.1038/sj.onc.1206597
Yin CC, Pemmaraju N, You MJ, Li S, Xu J, Wang W, et al. Integrated clinical genotype-phenotype characteristics of blastic plasmacytoid dendritic cell neoplasm. Cancers. 2021;13:5888.
pubmed: 34884997 pmcid: 8656770 doi: 10.3390/cancers13235888
Bastidas Torres AN, Cats D, Mei H, Fanoni D, Gliozzo J, Corti L, et al. Whole-genome analysis uncovers recurrent IKZF1 inactivation and aberrant cell adhesion in blastic plasmacytoid dendritic cell neoplasm. Genes Chromosom Cancer. 2020;59:295–308.
pubmed: 31846142 doi: 10.1002/gcc.22831
Sapienza MR, Abate F, Melle F, Orecchioni S, Fuligni F, Etebari M, et al. Blastic plasmacytoid dendritic cell neoplasm: genomics mark epigenetic dysregulation as a primary therapeutic target. Haematologica. 2019;104:729–37.
pubmed: 30381297 pmcid: 6442957 doi: 10.3324/haematol.2018.202093
Sakamoto K, Katayama R, Asaka R, Sakata S, Baba S, Nakasone H, et al. Recurrent 8q24 rearrangement in blastic plasmacytoid dendritic cell neoplasm: association with immunoblastoid cytomorphology, MYC expression, and drug response. Leukemia. 2018;32:2590–603.
pubmed: 29795241 doi: 10.1038/s41375-018-0154-5
Wang L, Yang M, Zhang X, Yang C, Huang X, Wang Z, et al. ARID1A mutation in blastic plasmacytoid dendritic cell neoplasm. Haematologica. 2017;102:e470–2.
pubmed: 28751556 pmcid: 5664412 doi: 10.3324/haematol.2017.172742
Suzuki K, Suzuki Y, Hama A, Muramatsu H, Nakatochi M, Gunji M, et al. Recurrent MYB rearrangement in blastic plasmacytoid dendritic cell neoplasm. Leukemia. 2017;31:1629–33.
pubmed: 28344318 doi: 10.1038/leu.2017.101
Sapienza MR, Fuligni F, Agostinelli C, Tripodo C, Righi S, Laginestra MA, 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;28:1606–16.
pubmed: 24504027 pmcid: 4294271 doi: 10.1038/leu.2014.64
Bormann F, Rodriguez-Paredes M, Lasitschka F, Edelmann D, Musch T, Benner A, et al. Cell-of-Origin DNA methylation signatures are maintained during colorectal carcinogenesis. Cell Rep. 2018;23:3407–18.
pubmed: 29898408 doi: 10.1016/j.celrep.2018.05.045
Khanlari M, Yin CC, Takahashi K, Lachowiez C, Tang G, Loghavi S, et al. Bone marrow clonal hematopoiesis is highly prevalent in blastic plasmacytoid dendritic cell neoplasm and frequently sharing a clonal origin in elderly patients. Leukemia. 2022;36:1343–50.
Papaemmanuil E, Gerstung M, Malcovati L, Tauro S, Gundem G, Van Loo P, et al. Clinical and biological implications of driver mutations in myelodysplastic syndromes. Blood. 2013;122:3616–27.
pubmed: 24030381 pmcid: 3837510 doi: 10.1182/blood-2013-08-518886
Cai X, Gaudet JJ, Mangan JK, Chen MJ, De Obaldia ME, Oo Z, et al. Runx1 loss minimally impacts long-term hematopoietic stem cells. PLoS One. 2011;6:e28430.
pubmed: 22145044 pmcid: 3228772 doi: 10.1371/journal.pone.0028430
Wu JN, Roberts CW. ARID1A mutations in cancer: another epigenetic tumor suppressor? Cancer Discov. 2013;3:35–43.
pubmed: 23208470 doi: 10.1158/2159-8290.CD-12-0361
Hetzel S, Mattei AL, Kretzmer H, Qu C, Chen X, Fan Y, et al. Acute lymphoblastic leukemia displays a distinct highly methylated genome. Nat Cancer. 2022;3:768–82.
pubmed: 35590059 pmcid: 9236905 doi: 10.1038/s43018-022-00370-5
McCormick SM, Heller NM. Commentary: IL-4 and IL-13 receptors and signaling. Cytokine. 2015;75:38–50.
pubmed: 26187331 pmcid: 4546937 doi: 10.1016/j.cyto.2015.05.023
Straining R, Eighmy W. Tazemetostat: EZH2 Inhibitor. J Adv Pr Oncol. 2022;13:158–63.
Aung PP, Sukswai N, Nejati R, Loghavi S, Chen W, Torres-Cabala CA, et al. PD1/PD-L1 expression in blastic plasmacytoid dendritic cell neoplasm. Cancers. 2019;11:695.
pubmed: 31109153 pmcid: 6562447 doi: 10.3390/cancers11050695
Skvortsova K, Zotenko E, Luu PL, Gould CM, Nair SS, Clark SJ, et al. Comprehensive evaluation of genome-wide 5-hydroxymethylcytosine profiling approaches in human DNA. Epigenetics Chromatin. 2017;10:16.
pubmed: 28428825 pmcid: 5397694 doi: 10.1186/s13072-017-0123-7

Auteurs

Axel Künstner (A)

Medical Systems Biology Group, University of Lübeck, Ratzeburger Allee 160, 23538, Lübeck, Germany.
University Cancer Center Schleswig-Holstein, University Hospital of Schleswig-Holstein, Campus Lübeck, 23538, Lübeck, Germany.

Julian Schwarting (J)

University Cancer Center Schleswig-Holstein, University Hospital of Schleswig-Holstein, Campus Lübeck, 23538, Lübeck, Germany.
Department of Hematology and Oncology, University Hospital of Schleswig-Holstein, Campus Lübeck, Ratzeburger Allee 160, 23538, Lübeck, Germany.
Hämatopathologie Lübeck, Consultation Centre for Lymph Node Pathology and Hematopathology, 23562, Lübeck, Germany.

Hanno M Witte (HM)

University Cancer Center Schleswig-Holstein, University Hospital of Schleswig-Holstein, Campus Lübeck, 23538, Lübeck, Germany.
Department of Hematology and Oncology, University Hospital of Schleswig-Holstein, Campus Lübeck, Ratzeburger Allee 160, 23538, Lübeck, Germany.
Department of Hematology and Oncology, Federal Armed Forces Hospital Ulm, Oberer Eselsberg 40, 89081, Ulm, Germany.

Pengwei Xing (P)

Department of Immunology, Genetics and Pathology, Uppsala University, 751 85, Uppsala, Sweden.

Veronica Bernard (V)

Hämatopathologie Lübeck, Consultation Centre for Lymph Node Pathology and Hematopathology, 23562, Lübeck, Germany.

Stephanie Stölting (S)

Hämatopathologie Lübeck, Consultation Centre for Lymph Node Pathology and Hematopathology, 23562, Lübeck, Germany.

Philipp Lohneis (P)

Hämatopathologie Lübeck, Consultation Centre for Lymph Node Pathology and Hematopathology, 23562, Lübeck, Germany.

Florian Janke (F)

Division of Cancer Genome Research, German Cancer Research Center (DKFZ), 69120, Heidelberg, Germany.
German Cancer Consortium (DKTK), 69120, Heidelberg, Germany.

Maede Salehi (M)

Department of Immunology, Genetics and Pathology, Uppsala University, 751 85, Uppsala, Sweden.

Xingqi Chen (X)

Department of Immunology, Genetics and Pathology, Uppsala University, 751 85, Uppsala, Sweden.

Kathrin Kusch (K)

Hämatopathologie Lübeck, Consultation Centre for Lymph Node Pathology and Hematopathology, 23562, Lübeck, Germany.

Holger Sültmann (H)

Division of Cancer Genome Research, German Cancer Research Center (DKFZ), 69120, Heidelberg, Germany.
German Cancer Consortium (DKTK), 69120, Heidelberg, Germany.

Emil Chteinberg (E)

Institute of Human Genetics Ulm University and Ulm University Medical Center, 89081, Ulm, Germany.

Anja Fischer (A)

Institute of Human Genetics Ulm University and Ulm University Medical Center, 89081, Ulm, Germany.

Reiner Siebert (R)

Institute of Human Genetics Ulm University and Ulm University Medical Center, 89081, Ulm, Germany.

Nikolas von Bubnoff (N)

University Cancer Center Schleswig-Holstein, University Hospital of Schleswig-Holstein, Campus Lübeck, 23538, Lübeck, Germany.
Department of Hematology and Oncology, University Hospital of Schleswig-Holstein, Campus Lübeck, Ratzeburger Allee 160, 23538, Lübeck, Germany.

Hartmut Merz (H)

Hämatopathologie Lübeck, Consultation Centre for Lymph Node Pathology and Hematopathology, 23562, Lübeck, Germany.

Hauke Busch (H)

Medical Systems Biology Group, University of Lübeck, Ratzeburger Allee 160, 23538, Lübeck, Germany.
University Cancer Center Schleswig-Holstein, University Hospital of Schleswig-Holstein, Campus Lübeck, 23538, Lübeck, Germany.

Alfred C Feller (AC)

Hämatopathologie Lübeck, Consultation Centre for Lymph Node Pathology and Hematopathology, 23562, Lübeck, Germany.

Niklas Gebauer (N)

University Cancer Center Schleswig-Holstein, University Hospital of Schleswig-Holstein, Campus Lübeck, 23538, Lübeck, Germany. Niklas.Gebauer@uksh.de.
Department of Hematology and Oncology, University Hospital of Schleswig-Holstein, Campus Lübeck, Ratzeburger Allee 160, 23538, Lübeck, Germany. Niklas.Gebauer@uksh.de.

Classifications MeSH