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@ARTICLE{Fangusaro:300575,
      author       = {J. Fangusaro and A. Onar-Thomas and T. Young Poussaint and
                      S. Lensing and A. H. Ligon and N. Lindeman and A. Banerjee
                      and L. B. Kilburn and A. Lenzen and N. Pillay-Smiley and I.
                      F. Pollack and N. J. Robison and S. Partap and I. Qaddoumi
                      and D. Landi and D. Jones$^*$ and C. F. Stewart and M.
                      Fouladi and I. J. Dunkel},
      title        = {{A} {P}hase 2 {PBTC} {S}tudy of {S}elumetinib for
                      {R}ecurrent/{P}rogressive {P}ediatric {L}ow-{G}rade
                      {G}lioma: {S}trata 2, 5, and 6 with {L}ong-term {O}utcomes
                      on {S}trata 1, 3, and 4.},
      journal      = {Neuro-Oncology},
      volume       = {nn},
      issn         = {1522-8517},
      address      = {Oxford},
      publisher    = {Oxford Univ. Press},
      reportid     = {DKFZ-2025-00808},
      pages        = {nn},
      year         = {2025},
      note         = {epub},
      abstract     = {PBTC-029B was a phase 2 trial evaluating efficacy of
                      selumetinib in children with recurrent/progressive low-grade
                      glioma. We report results of strata 2, 5, and 6 with updated
                      survivals for strata 1, 3, and 4.Stratum 2 included
                      recurrent/progressive pilocytic astrocytoma (PA) not
                      associated with neurofibromatosis type-1 (NF1) that screened
                      negative for the BRAF-KIAA1549 fusion and BRAFV600E
                      mutation. Stratum 5 enrolled non-PA that screened positive
                      for one of the BRAF aberrations. Stratum 6 enrolled children
                      who consented to tissue screening, but there was an assay
                      failure. For long-term survivals, stratum 1 included non-NF1
                      PA positive for one of the BRAF aberrations; stratum 3
                      included NF1-associated pLGG; and stratum 4 included non-NF1
                      optic pathway/hypothalamic tumors.Stratum 2: among 14
                      evaluable patients, there was 1 partial response (PR), 7
                      stable disease (SD) and 6 progressive disease (PD); overall
                      response rate (ORR) was $7.1\%.$ Two-year progression-free
                      survival (PFS)/overall survival (OS) were $57.1\%/100\%,$
                      respectively. Stratum 5: among 23 evaluable patients, there
                      was 1 complete response (CR), 4 PR, 12 SD, and 6 PD; ORR was
                      $21.7\%.$ Two-year PFS/OS were $74.8\%/100\%,$ respectively.
                      Stratum 6: among 26 evaluable patients, there were 7 PR, 14
                      SD, and 5 PD; ORR was $26.9\%.$ Two-year PFS/OS were
                      $72.0\%/100\%,$ respectively. The median follow-up for
                      patients on strata 1, 3, and 4 without events are 60.4,
                      60.4, and 58.1 months, and 5-year PFS/OS were
                      $30.8\%/88.9\%,$ $54.2\%/100\%,$ and $51.0\%/100\%,$
                      respectively.Selumetinib provided stability and responses
                      across many pLGG subgroups, and some patients achieved
                      prolonged disease control without additional therapy.},
      keywords     = {Recurrent (Other) / and MEK inhibitor (Other) / pediatric
                      low-grade glioma (pLGG) (Other) / selumetinib (Other)},
      cin          = {B360},
      ddc          = {610},
      cid          = {I:(DE-He78)B360-20160331},
      pnm          = {312 - Funktionelle und strukturelle Genomforschung
                      (POF4-312)},
      pid          = {G:(DE-HGF)POF4-312},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:40241281},
      doi          = {10.1093/neuonc/noaf065},
      url          = {https://inrepo02.dkfz.de/record/300575},
}