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@ARTICLE{Schnung:303248,
      author       = {M. Schönung$^*$ and S. Rathmann and S. Ramamoorthy and D.
                      Lebrecht and T. Klingebiel$^*$ and F. Locatelli and K. Nysom
                      and C. Rossig and J. Starý and M. Zecca and M. Patturajan
                      and M. Erlacher and B. Strahm and C. M. Niemeyer and D.
                      Lipka$^*$ and C. Flotho$^*$},
      title        = {{A}ssessment of peripheral blood {DNA} methylation
                      signatures as pharmacodynamic and predictive biomarkers
                      during azacitidine therapy in juvenile myelomonocytic
                      leukaemia: {R}esults of the {EWOG}-{MESRAT} study.},
      journal      = {British journal of haematology},
      volume       = {nn},
      issn         = {0007-1048},
      address      = {Oxford [u.a.]},
      publisher    = {Wiley-Blackwell},
      reportid     = {DKFZ-2025-01593},
      pages        = {nn},
      year         = {2025},
      note         = {#EA:B340# / epub},
      abstract     = {EWOG-MESRAT (European Working Group-Methylation Signatures
                      and Response to Azacitidine Therapy; DRKS00007185) is an
                      investigator-initiated trial that studied EPIC array-based
                      DNA methylation patterns and next generation sequencing
                      (NGS)-based variant allele frequencies (VAFs) of driver
                      mutations in peripheral blood (PB) and bone marrow (BM) of
                      11 patients with newly diagnosed juvenile myelomonocytic
                      leukaemia (JMML) during therapy with azacitidine. We
                      demonstrate that the pharmacodynamic activity of azacitidine
                      can efficiently be monitored in PB and BM. DNA methylation
                      subgroup classification was linked to clinical response
                      after three cycles of azacitidine and found to be conserved
                      between PB and BM in all patients. In contrast, neither
                      changes in VAFs nor changes in DNA methylation patterns
                      during the course of therapy correlated with therapy outcome
                      among the 11 study patients. This work thus supports the
                      value of DNA methylation subgroup classification from PB
                      samples for response prediction of single-agent azacitidine
                      in patients with JMML.},
      keywords     = {DNA methylation (Other) / JMML (Other) / azacitidine
                      (Other) / epigenetics (Other)},
      cin          = {B340 / FM01 / HD01 / FR01},
      ddc          = {610},
      cid          = {I:(DE-He78)B340-20160331 / I:(DE-He78)FM01-20160331 /
                      I:(DE-He78)HD01-20160331 / I:(DE-He78)FR01-20160331},
      pnm          = {312 - Funktionelle und strukturelle Genomforschung
                      (POF4-312)},
      pid          = {G:(DE-HGF)POF4-312},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:40740143},
      doi          = {10.1111/bjh.70046},
      url          = {https://inrepo02.dkfz.de/record/303248},
}