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@ARTICLE{SparberSauer:177013,
      author       = {M. Sparber-Sauer and C. Vokuhl and G. Seitz and B. Sorg and
                      M. Tobias$^*$ and T. von Kalle and M. Münter and S. S.
                      Bielack and R. Ladenstein and G. Ljungman and F. Niggli and
                      M. Frühwald and S. Loff and T. Klingebiel and E.
                      Koscielniak},
      title        = {{I}nfantile myofibromatosis: {E}xcellent prognosis but also
                      rare fatal progressive disease. {T}reatment results of five
                      {C}ooperative {W}eichteilsarkom {S}tudiengruppe ({CWS})
                      trials and one registry.},
      journal      = {Pediatric blood $\&$ cancer},
      volume       = {69},
      number       = {3},
      issn         = {1545-5009},
      address      = {New York, NY},
      publisher    = {Wiley},
      reportid     = {DKFZ-2021-02243},
      pages        = {e29403},
      year         = {2022},
      note         = {Volume69, Issue3, March 2022, e29403},
      abstract     = {Infantile myofibromatosis (IM) is a rare benign soft tissue
                      tumor and often a self-limiting disease but rarely includes
                      life-threatening complications. Little is known about
                      optimal treatment of primary localized (LD) and multifocal
                      disease (MFD).Treatment and outcome of 95 children with IM
                      registered within five Cooperative Weichteilsarkom
                      Studiengruppe (CWS) trials and one registry (1981-2016) were
                      evaluated.LD was diagnosed in 71 patients at a median age of
                      0.4 years (range 0.0-17.7). MFD was present in 24 patients.
                      The mainstay of treatment was watch-and-wait strategy
                      $(w\&w)$ after initial biopsy or resection. Low-dose
                      chemotherapy (CHT) was administered to 16/71 $(23\%)$
                      patients with LD and eight of 24 $(33\%)$ patients with MFD,
                      imatinib was added in two. A delayed resection was possible
                      in eight of 71 $(11\%)$ and five of 24 $(21\%)$ patients
                      with LD and MFD, respectively. Overall, patients were alive
                      in complete remission (n = 77) and partial remission
                      (n = 10) at a median follow-up time of 3.4 years after
                      diagnosis (range 0.01-19.4); no data available (n = 5).
                      Three patients died of progressive disease (PD) despite CHT.
                      Gender, tumor size, and location correlated with a favorable
                      event-free survival (EFS) in patients with LD. The 5-year
                      EFS and overall survival of patients with LD were $73\%$
                      (±12, confidence interval [CI] $95\%)$ and $95\%$ (±6, CI
                      $95\%),$ respectively; for MFD $51\%$ (±22, CI $95\%)$ and
                      $95\%$ (±10, CI $95\%).Prognosis$ is excellent in patients
                      with LD and MFD. Targeted treatment needs to be evaluated
                      for rare fatal PD.},
      keywords     = {CWS Group (Other) / infantile myofibromatosis (Other) /
                      infants and children (Other) / localized and multifocal
                      disease (Other)},
      cin          = {C070},
      ddc          = {610},
      cid          = {I:(DE-He78)C070-20160331},
      pnm          = {313 - Krebsrisikofaktoren und Prävention (POF4-313)},
      pid          = {G:(DE-HGF)POF4-313},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:34636137},
      doi          = {10.1002/pbc.29403},
      url          = {https://inrepo02.dkfz.de/record/177013},
}