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Authors Ami Vijay Desai, Giles W. Robinson, Ellen M. Basu, Jennifer Foster, Karen Gauvain, Amit Sabnis, Suzanne Shusterman, Margaret E Macy, Luke Maese, Janet Yoon, Thomas Cash, Mohamed Abdelbaki, Kellie Nazemi, Brian D. Weiss, Saibah Chohan, Alison Cardenas, Katherine Hutchinson, Guillaume Bergthold, Amar J. Gajjar, Elizabeth Fox
Title Updated entrectinib data in children and adolescents with recurrent or refractory solid tumors, including primary CNS tumors.
Journal Journal of Clinical Oncology
Date May 2020
Abstract Text Background: The phase 1/2 STARTRK-NG trial (NCT02650401) is evaluating entrectinib, a CNS-penetrant oral inhibitor of TRK, ROS1 and ALK tyrosine kinases, in children and adolescents < 21 years old with recurrent/refractory solid tumors, including primary CNS tumors. Methods: After determining the recommended dose as 550mg/m2/day in all-comers, expansion cohorts with gene-fusion-positive CNS/solid tumors (NTRK1/2/3 and ROS1) are being enrolled. Results: As of 1 July 2019 (data cut-off), 34 patients (4.9 months to 20 years old; median age 7 years) have been evaluated for response to treatment with entrectinib. Responses were classified as complete response (CR), partial response (PR), stable disease (SD), or progressive disease (PD) using RANO for CNS tumors, RECISTv1.1 for solid tumors, or Curie score for neuroblastomas. Responses in fusion-positive patients were assessed by blinded independent central review (BICR), and occurred at doses ≥400mg/m2. Best responses in patients with fusion-positive CNS tumors (n = 8) were four CR (ETV6-NTRK3, EML1-NTRK2, GOPC-ROS1, and TPR-NTRK1), two PR (KANK1-NTRK2 and EEF1G-ROS1), and two PD (EML4-ALK and PARP6-NTRK3). In patients with fusion-positive solid tumors (n = 6) best responses were three CR (DCTN1-ALK, ETV6-NTRK3, and ETV6-NTRK3), and three PR (TFG-ROS1, EML4-NTRK3, and KIF5B-ALK). Responses (Investigator-assessed) in patients with non-fusion tumors (n = 20) were one CR (ALK F1174L mutation), four SD, ten PD, and five patients were unevaluable or had no data. The objective response rate (defined as the total number of CR and PR) in fusion-positive patients was 86% (12/14) versus 5% (1/20) in non-fusion patients. Similarly, PFS was 17.5 months (95% CI 7.4–NE) in fusion-positive patients versus 1.9 months (1.8–5.7; p = 0.0002) in non-fusion patients. Most commonly reported treatment-related adverse events included weight gain (n = 14 [5 Grade 3/4]), elevated creatinine (n = 13), anemia (n = 13), nausea (n = 11), increased ALT (n = 10 [1 Grade 3/4]), increased AST (n = 10 [1 Grade 3/4]), decreased neutrophils (n = 9 [6 Grade 3/4]), and bone fractures (n = 7, of which 4 were treatment related). Conclusions: In children and adolescents < 21 years old, entrectinib has produced striking, rapid, and durable responses in solid tumors with target gene fusions, especially in high-grade CNS neoplasms


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Molecular Profile Treatment Approach
Gene Name Source Synonyms Protein Domains Gene Description Gene Role
Therapy Name Drugs Efficacy Evidence Clinical Trials
Drug Name Trade Name Synonyms Drug Classes Drug Description
Gene Variant Impact Protein Effect Variant Description Associated with drug Resistance
Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
EML4 - NTRK3 Advanced Solid Tumor predicted - sensitive Entrectinib Case Reports/Case Series Actionable In a Phase Ib/II trial (STARTRK-NG), a patient with an advanced solid tumor harboring EML4-NTRK3 demonstrated a partial response when treated with Rozlytrek (entrectinib) (Journal of Clinical Oncology 38, no. 15_suppl (May 20, 2020) 107-107; NCT02650401). detail...