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@ARTICLE{Dorman:302789,
      author       = {K. Dorman$^*$ and C. J. Auernhammer and C. Spitzweg and R.
                      Schmidmaier and S. Nölting and M. Kroiss and M. Reincke and
                      C. Schulz and M. Angele and J. Werner and C. Schmid-Tannwald
                      and J. Rauch and M. Zacherl and T. Knösel$^*$ and J.
                      Kumbrink$^*$ and A. Jung$^*$ and F. Klauschen$^*$ and A.
                      Tufman and D. Zhang$^*$ and L. Weiss and S. Böck$^*$ and M.
                      von Bergwelt-Baildon$^*$ and V. Heinemann$^*$ and C. B.
                      Westphalen$^*$ and K. Heinrich$^*$},
      title        = {{P}recision {O}ncology in {R}are {E}ndocrine and
                      {N}euroendocrine {N}eoplasms: {E}xperiences and {C}hallenges
                      of the {CCCM}unich{LMU} {M}olecular {T}umor {B}oard.},
      journal      = {Targeted oncology},
      volume       = {20},
      number       = {4},
      issn         = {1776-2596},
      address      = {Paris},
      publisher    = {Springer Verlag France S.A.R.L.},
      reportid     = {DKFZ-2025-01329},
      pages        = {715-724},
      year         = {2025},
      note         = {2025 Jul;20(4):715-724},
      abstract     = {Comprehensive genomic profiling (CGP) has become more
                      generally accessible to patients with rare cancer, but data
                      on the results and benefits are limited.Our objective was to
                      gain a real-world understanding of the molecular landscape
                      and targeted treatment options in neuroendocrine tumors,
                      neuroendocrine carcinomas, adrenocortical carcinomas,
                      pheochromocytomas, and carcinoids.In this retrospective
                      cohort study, we analyzed CGP results and clinical data from
                      patients with neuroendocrine tumors, neuroendocrine
                      carcinomas, adrenocortical carcinomas, pheochromocytomas,
                      and carcinoids who were discussed in the CCCMunichLMU
                      Molecular Tumor Board (MTB) between May 2017 and April
                      2023.In total, 104 patients with endocrine and
                      neuroendocrine neoplasms were discussed in the MTB. CGP was
                      technically successful in 99 patients. The most commonly
                      mutated genes were TP53 $(29.3\%),$ RB1 $(11.1\%),$ and KRAS
                      $(10.1\%).$ The highest overall prevalence of pathogenic
                      alterations was detected in neuroendocrine carcinomas
                      $(76.9\%)$ and carcinoids $(83.3\%),$ and the lowest
                      prevalence of pathogenic alterations was seen in
                      adrenocortical carcinoma $(37.5\%).$ Of the 99 patients with
                      successful CGP, 35 received a treatment recommendation from
                      the MTB based on the CGP results. Of these, ten patients
                      ultimately received the recommended treatment. Of the ten
                      treated patients, four experienced a longer progression-free
                      survival under the targeted treatment than under their
                      previous treatment.One-third of patients with rare endocrine
                      and neuroendocrine neoplasms who underwent CGP had a
                      druggable alteration and received a treatment recommendation
                      from the MTB. However, only $28.6\%$ of these patients were
                      treated accordingly. Our experience highlights the unmet
                      medical need for targeted treatment options in patients with
                      rare cancers.},
      cin          = {MU01},
      ddc          = {610},
      cid          = {I:(DE-He78)MU01-20160331},
      pnm          = {899 - ohne Topic (POF4-899)},
      pid          = {G:(DE-HGF)POF4-899},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:40587033},
      doi          = {10.1007/s11523-025-01152-6},
      url          = {https://inrepo02.dkfz.de/record/302789},
}