TY  - JOUR
AU  - Desai, Ami V
AU  - Bagchi, Aditi
AU  - Armstrong, Amy E
AU  - van Tilburg, Cornelis M
AU  - Basu, Ellen M
AU  - Robinson, Giles W
AU  - Wang, Huanmin
AU  - Casanova, Michela
AU  - André, Nicolas
AU  - Campbell-Hewson, Quentin
AU  - Wu, Yeming
AU  - Cardenas, Alison
AU  - Ci, Bo
AU  - Ryklansky, Carolina
AU  - Devlin, Clare E
AU  - Meneses-Lorente, Georgina
AU  - Wulff, Jade
AU  - Hutchinson, Katherine E
AU  - Gajjar, Amar
AU  - Fox, Elizabeth
TI  - Efficacy and safety of entrectinib in children with extracranial solid or central nervous system (CNS) tumours harbouring NTRK or ROS1 fusions.
JO  - European journal of cancer
VL  - 220
SN  - 0959-8049
CY  - Amsterdam [u.a.]
PB  - Elsevier
M1  - DKFZ-2025-00560
SP  - 115308
PY  - 2025
AB  - 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 
KW  - CNS tumours (Other)
KW  - Entrectinib (Other)
KW  - NTRK fusions (Other)
KW  - Paediatric tumours (Other)
KW  - ROS1 fusions (Other)
KW  - ROS1 inhibitor (Other)
KW  - STARTRK-2 (Other)
KW  - STARTRK-NG (Other)
KW  - TAPISTRY (Other)
KW  - TRK inhibitor (Other)
LB  - PUB:(DE-HGF)16
C6  - pmid:40086048
DO  - DOI:10.1016/j.ejca.2025.115308
UR  - https://inrepo02.dkfz.de/record/299823
ER  -