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Efficacy (paediatrics)

Data from the phase 1/2 SCOUT trial in paediatric patients and adolescents/young adults with advanced solid or primary CNS tumours were analysed separately and included patients with and without identified NTRK gene fusions [1]. The objective response rate was 93% (14/15) in evaluable patients with cancers with TRK fusion cancer, compared with 0% (0/7) without TRK fusion cancer. Larotrectinib was detectable in the cerebrospinal fluid (CSF) of two patients that underwent standard-of-care sampling with CSF to plasma concentrations of 28% via Ommaya and 123% via lumbar puncture.


  1. Laetsch TW, DuBois SG, Mascarenhas L et al. Larotrectinib for paediatric solid tumours harbouring NTRK gene fusions: phase 1 results from a multicentre, open-label, phase 1/2 study. Lancet Oncol 2018; 19: 705-714.

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Last update: 24 June 2019