% IMPORTANT: The following is UTF-8 encoded. This means that in the presence % of non-ASCII characters, it will not work with BibTeX 0.99 or older. % Instead, you should use an up-to-date BibTeX implementation like “bibtex8” or % “biber”. @ARTICLE{Neyazi:267352, author = {Neyazi, Sina and Yamazawa, Erika and Hack, Karoline and Tanaka, Shota and Nagae, Genta and Kresbach, Catena and Umeda, Takayoshi and Eckhardt, Alicia and Tatsuno, Kenji and Pohl, Lara and Hana, Taijun and Bockmayr, Michael and Kim, Phyo and Dorostkar, Mario M and Takami, Toshihiro and Obrecht, Denise and Takai, Keisuke and Suwala, Abigail K and Komori, Takashi and Godbole, Shweta and Wefers, Annika K and Otani, Ryohei and Neumann, Julia E and Higuchi, Fumi and Schweizer, Leonille and Nakanishi, Yuta and Monoranu, Camelia-Maria and Takami, Hirokazu and Engertsberger, Lara and Yamada, Keisuke and Ruf, Viktoria and Nomura, Masashi and Mohme, Theresa and Mukasa, Akitake and Herms, Jochen and Takayanagi, Shunsaku and Mynarek, Martin and Matsuura, Reiko and Lamszus, Katrin and Ishii, Kazuhiko and Kluwe, Lan and Imai, Hideaki and von Deimling, Andreas and Koike, Tsukasa and Benesch, Martin and Kushihara, Yoshihiro and Snuderl, Matija and Nambu, Shohei and Frank, Stephan and Omura, Takaki and Hagel, Christian and Kugasawa, Kazuha and Mautner, Viktor F and Ichimura, Koichi and Rutkowski, Stefan and Aburatani, Hiroyuki and Saito, Nobuhito and Schüller, Ulrich}, title = {{T}ranscriptomic and epigenetic dissection of spinal ependymoma ({SP}-{EPN}) identifies clinically relevant subtypes enriched for tumors with and without {NF}2 mutation.}, journal = {Acta neuropathologica}, volume = {147}, number = {1}, issn = {0001-6322}, address = {Heidelberg}, publisher = {Springer}, reportid = {DZNE-2024-00116}, pages = {22}, year = {2024}, abstract = {Ependymomas encompass multiple clinically relevant tumor types based on localization and molecular profiles. Tumors of the methylation class 'spinal ependymoma' (SP-EPN) represent the most common intramedullary neoplasms in children and adults. However, their developmental origin is ill-defined, molecular data are scarce, and the potential heterogeneity within SP-EPN remains unexplored. The only known recurrent genetic events in SP-EPN are loss of chromosome 22q and NF2 mutations, but neither types and frequency of these alterations nor their clinical relevance have been described in a large, epigenetically defined series. Transcriptomic (n = 72), epigenetic (n = 225), genetic (n = 134), and clinical data (n = 112) were integrated for a detailed molecular overview on SP-EPN. Additionally, we mapped SP-EPN transcriptomes to developmental atlases of the developing and adult spinal cord to uncover potential developmental origins of these tumors. The integration of transcriptomic ependymoma data with single-cell atlases of the spinal cord revealed that SP-EPN display the highest similarities to mature adult ependymal cells. Unsupervised hierarchical clustering of transcriptomic data together with integrated analysis of methylation profiles identified two molecular SP-EPN subtypes. Subtype A tumors primarily carried previously known germline or sporadic NF2 mutations together with 22q loss (bi-allelic NF2 loss), resulting in decreased NF2 expression. Furthermore, they more often presented as multilocular disease and demonstrated a significantly reduced progression-free survival as compared to SP-EP subtype B. In contrast, subtype B predominantly contained samples without NF2 mutation detected in sequencing together with 22q loss (monoallelic NF2 loss). These tumors showed regular NF2 expression but more extensive global copy number alterations. Based on integrated molecular profiling of a large multi-center cohort, we identified two distinct SP-EPN subtypes with important implications for genetic counseling, patient surveillance, and drug development priorities.}, keywords = {Adult / Child / Humans / Transcriptome / Gene Expression Profiling / Ependymoma / Spinal Cord Neoplasms / Mutation / Epigenesis, Genetic / Classification (Other) / DNA methylation (Other) / Ependymoma (Other) / NF2-related schwannomatosis (Other) / Transcriptomics (Other)}, cin = {AG Herms}, ddc = {610}, cid = {I:(DE-2719)1110001}, pnm = {352 - Disease Mechanisms (POF4-352)}, pid = {G:(DE-HGF)POF4-352}, typ = {PUB:(DE-HGF)16}, pubmed = {pmid:38265489}, pmc = {pmc:PMC10808175}, doi = {10.1007/s00401-023-02668-9}, url = {https://pub.dzne.de/record/267352}, }