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@ARTICLE{Desai:299823,
      author       = {A. V. Desai and A. Bagchi and A. E. Armstrong and C. M. van
                      Tilburg$^*$ and E. M. Basu and G. W. Robinson and H. Wang
                      and M. Casanova and N. André and Q. Campbell-Hewson and Y.
                      Wu and A. Cardenas and B. Ci and C. Ryklansky and C. E.
                      Devlin and G. Meneses-Lorente and J. Wulff and K. E.
                      Hutchinson and A. Gajjar and E. Fox},
      title        = {{E}fficacy and safety of entrectinib in children with
                      extracranial solid or central nervous system ({CNS}) tumours
                      harbouring {NTRK} or {ROS}1 fusions.},
      journal      = {European journal of cancer},
      volume       = {220},
      issn         = {0959-8049},
      address      = {Amsterdam [u.a.]},
      publisher    = {Elsevier},
      reportid     = {DKFZ-2025-00560},
      pages        = {115308},
      year         = {2025},
      abstract     = {Entrectinib, a central nervous system (CNS)-penetrant
                      TRK/ROS1 inhibitor, has demonstrated clinical activity in
                      children with NTRK1/2/3 or ROS1 fusion-positive extracranial
                      solid and CNS tumours. We present integrated data of
                      entrectinib in children with NTRK or ROS1 fusion-positive
                      tumours from the STARTRK-NG, TAPISTRY, and STARTRK-2
                      trials.Efficacy analyses were undertaken on TRK/ROS1
                      inhibitor-naïve patients aged <18 years with
                      metastatic/locally advanced NTRK1/2/3 or ROS1
                      fusion-positive extracranial solid or CNS tumours who
                      received ≥1 entrectinib dose and had ≥6 months of
                      follow-up from enrolment. Tumour responses were confirmed by
                      blinded independent central review (BICR) per RECIST
                      v1.1/RANO criteria.BICR-assessed confirmed objective
                      response rate (cORR). Key secondary endpoints: duration of
                      response (DoR); time to response (TtR); safety.As of 16 July
                      2023, out of 91 safety-evaluable patients, 64 (NTRK: n=44;
                      ROS1: n=20) were efficacy evaluable. In the NTRK and ROS1
                      subgroups, respectively, median age was 4.0 years and 7.5
                      years; median survival follow-up was 24.2 months and 27.6
                      months. cORR was 72.7 $\%$ (NTRK, 95 $\%$ confidence
                      interval [CI]: 57.2-85.0) and 65.0 $\%$ (ROS1, 95 $\%$ CI:
                      40.8-84.6). Median DoR was not reached (NTRK, 95 $\%$ CI:
                      25.4-not evaluable [NE]); ROS1, 95 $\%$ CI: 16.2-NE); median
                      TtR was 1.9 months in both subgroups. The most frequently
                      reported treatment-related adverse events included weight
                      gain (35.2 $\%)$ and anaemia (31.9 $\%).Integrated$ data
                      from three trials confirm entrectinib induces rapid and
                      durable responses in children with NTRK or ROS1
                      fusion-positive tumours. The increased duration of safety
                      monitoring does not demonstrate new or cumulative toxicity.
                      Registered clinical trials: STARTRK-NG: NCT02650401;
                      TAPISTRY: NCT04589845; STARTRK-2: NCT02568267.},
      keywords     = {CNS tumours (Other) / Entrectinib (Other) / NTRK fusions
                      (Other) / Paediatric tumours (Other) / ROS1 fusions (Other)
                      / ROS1 inhibitor (Other) / STARTRK-2 (Other) / STARTRK-NG
                      (Other) / TAPISTRY (Other) / TRK inhibitor (Other)},
      cin          = {B310 / HD01},
      ddc          = {610},
      cid          = {I:(DE-He78)B310-20160331 / 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:40086048},
      doi          = {10.1016/j.ejca.2025.115308},
      url          = {https://inrepo02.dkfz.de/record/299823},
}