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@ARTICLE{Heinz:284617,
      author       = {A. T. Heinz and A. Schönstein and M. Ebinger and J. Fuchs
                      and B. Timmermann$^*$ and G. Seitz and C. Vokuhl and M.
                      Münter and K. Pajtler$^*$ and S. Stegmaier and T. von Kalle
                      and C. P. Kratz and G. Ljungman and H. Juntti and T.
                      Klingebiel and E. Koscielniak and M. Sparber-Sauer},
      collaboration = {C. W. Studiengruppe},
      title        = {{S}ignificance of fusion status, {O}berlin risk factors,
                      local and maintenance treatment in pediatric and adolescent
                      patients with metastatic rhabdomyosarcoma: {D}ata of the
                      {E}uropean {S}oft {T}issue {S}arcoma {R}egistry
                      {S}o{T}i{S}a{R}.},
      journal      = {Pediatric blood $\&$ cancer},
      volume       = {71},
      number       = {1},
      issn         = {1545-5009},
      address      = {New York, NY},
      publisher    = {Wiley},
      reportid     = {DKFZ-2023-02036},
      pages        = {e30707},
      year         = {2024},
      note         = {2024 Jan;71(1):e30707},
      abstract     = {Outcome of primary metastatic rhabdomyosarcoma (RMS) is
                      poor. Certain risk factors as fusion status, Oberlin score,
                      and local treatment of primary tumor are known to influence
                      prognosis.Patients with metastatic RMS were treated
                      according to Cooperative Weichteilsarkom Studiengruppe (CWS)
                      guidance with chemotherapy (CHT), radiotherapy (RT)
                      excluding total lung irradiation (TLI), complete resection
                      of the primary tumor, and metastasectomy if possible.
                      Kaplan-Meier estimators and Cox proportional hazard models
                      were used to examine event-free survival (EFS) and overall
                      survival (OS) involving also landmark analyses.In the
                      European Soft Tissue Sarcoma Registry SoTiSaR (2009-2018),
                      211 patients were analyzed. Many patients had
                      fusion-positive alveolar RMS (n = 83; $39\%).$ Median age
                      was 9.4 years [0.1-19.7 years]. Treatment primarily
                      consisted of CHT with CEVAIE (carboplatin, epirubicine,
                      vincristine, actinomycin-D, ifosfamide, etoposide: $86\%,$
                      other regimens: $14\%),$ RT $(71\%),$ resection of primary
                      tumor $(37\%),$ metastasectomy $(19\%),$ and lymph node
                      sampling/dissection $(21\%).$ Maintenance treatment (MT)
                      (oral trofosfamide, idarubicin, etoposide) was added in
                      $74\%$ of patients. Oberlin factors, fusion status, and MT
                      were predictive for EFS and OS. MT with O-TIE was not
                      improving outcome when adjusting for the immortal time bias.
                      Local treatment of the primary tumor and radical irradiation
                      (except TLI) improved EFS, not OS, when adjusting for the
                      Oberlin score. Patients with fusion-negative alveolar RMS (n
                      = 9) had an excellent outcome with a 5-year EFS and OS of
                      $100\%,$ compared to patients with embryonal RMS
                      $(49\%/62\%),$ PAX7- $(22\%/47\%)$ and PAX3/FOXO1-positive
                      ARMS $(10/13\%),$ respectively (p < .001).Prognosis of
                      metastatic RMS primarily depends on fusion status and
                      Oberlin score. Fusion status needs to be considered in
                      future trials to optimize treatment outcome. The role of
                      radical irradiation needs further investigation.},
      keywords     = {Oberlin score (Other) / fusion status (Other) / local
                      treatment (Other) / metastatic disease (Other) /
                      pediatric/adolescent oncology (Other) / rhabdomyosarcoma
                      (Other)},
      cin          = {ED01 / B062 / HD01},
      ddc          = {610},
      cid          = {I:(DE-He78)ED01-20160331 / I:(DE-He78)B062-20160331 /
                      I:(DE-He78)HD01-20160331},
      pnm          = {312 - Funktionelle und strukturelle Genomforschung
                      (POF4-312)},
      pid          = {G:(DE-HGF)POF4-312},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:37814424},
      doi          = {10.1002/pbc.30707},
      url          = {https://inrepo02.dkfz.de/record/284617},
}