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Clinical Characteristics and Outcomes of Central Nervous System Tumors Harboring NTRK Gene Fusions

Lamoureux, A.-A. ; Øra, I. LU orcid and Perreault, S. (2025) In Clinical Cancer Research 31(3). p.561-572
Abstract
Purpose: Tropomyosin receptor kinase (TRK) fusions are detected in less than 2% of central nervous system tumors. There are limited data on the clinical course of affected patients. Experimental Design: We conducted an international retrospective cohort study of patients with TRK fusion–driven central nervous system tumors. Results: A total of 119 patients were identified. The median age at the time of diagnosis was 4.5 years. The majority were reported to have a histology consistent with a diagnosis of high-grade glioma (HGG; 57.1%) followed by low-grade glioma (LGG; 27.7%). Pediatric patients had a better prognosis, with a median overall survival of 185.5 months compared with 24.8 months in adults (P < 0.0001). Patients with LGG also... (More)
Purpose: Tropomyosin receptor kinase (TRK) fusions are detected in less than 2% of central nervous system tumors. There are limited data on the clinical course of affected patients. Experimental Design: We conducted an international retrospective cohort study of patients with TRK fusion–driven central nervous system tumors. Results: A total of 119 patients were identified. The median age at the time of diagnosis was 4.5 years. The majority were reported to have a histology consistent with a diagnosis of high-grade glioma (HGG; 57.1%) followed by low-grade glioma (LGG; 27.7%). Pediatric patients had a better prognosis, with a median overall survival of 185.5 months compared with 24.8 months in adults (P < 0.0001). Patients with LGG also had a better outcome when compared with HGG (P = 0.0012). The objective response was 68.8% with larotrectinib compared with 38.1% for nontargeted treatment. Conclusions: Children with LGG had a favorable outcome compared with adult glioma and HGG. TRK inhibitors seem to improve tumor control. ©2024 The Authors. (Less)
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Contribution to journal
publication status
published
subject
keywords
entrectinib, larotrectinib, Neurotrophic tyrosine receptor kinase, protein kinase, selitrectinib, tropomyosin, unclassified drug, adolescent, adult, Article, cancer chemotherapy, cancer control, central nervous system, central nervous system tumor, child, cohort analysis, controlled study, diagnostic test accuracy study, female, gene fusion, glioma, high throughput sequencing, histology, human, juvenile xanthogranuloma, major clinical study, male, metastasis, neuroblastoma, overall response rate, overall survival, pathological complete response, progression free survival, retrospective study
in
Clinical Cancer Research
volume
31
issue
3
pages
12 pages
publisher
American Association for Cancer Research Inc.
external identifiers
  • scopus:85217005317
  • pmid:39625867
ISSN
1078-0432
DOI
10.1158/1078-0432.CCR-24-0581
language
English
LU publication?
yes
id
e011202a-3ae0-4658-b429-620378e81091
date added to LUP
2025-12-17 10:20:08
date last changed
2025-12-18 03:00:16
@article{e011202a-3ae0-4658-b429-620378e81091,
  abstract     = {{Purpose: Tropomyosin receptor kinase (TRK) fusions are detected in less than 2% of central nervous system tumors. There are limited data on the clinical course of affected patients. Experimental Design: We conducted an international retrospective cohort study of patients with TRK fusion–driven central nervous system tumors. Results: A total of 119 patients were identified. The median age at the time of diagnosis was 4.5 years. The majority were reported to have a histology consistent with a diagnosis of high-grade glioma (HGG; 57.1%) followed by low-grade glioma (LGG; 27.7%). Pediatric patients had a better prognosis, with a median overall survival of 185.5 months compared with 24.8 months in adults (P &lt; 0.0001). Patients with LGG also had a better outcome when compared with HGG (P = 0.0012). The objective response was 68.8% with larotrectinib compared with 38.1% for nontargeted treatment. Conclusions: Children with LGG had a favorable outcome compared with adult glioma and HGG. TRK inhibitors seem to improve tumor control. ©2024 The Authors.}},
  author       = {{Lamoureux, A.-A. and Øra, I. and Perreault, S.}},
  issn         = {{1078-0432}},
  keywords     = {{entrectinib; larotrectinib; Neurotrophic tyrosine receptor kinase; protein kinase; selitrectinib; tropomyosin; unclassified drug; adolescent; adult; Article; cancer chemotherapy; cancer control; central nervous system; central nervous system tumor; child; cohort analysis; controlled study; diagnostic test accuracy study; female; gene fusion; glioma; high throughput sequencing; histology; human; juvenile xanthogranuloma; major clinical study; male; metastasis; neuroblastoma; overall response rate; overall survival; pathological complete response; progression free survival; retrospective study}},
  language     = {{eng}},
  number       = {{3}},
  pages        = {{561--572}},
  publisher    = {{American Association for Cancer Research Inc.}},
  series       = {{Clinical Cancer Research}},
  title        = {{Clinical Characteristics and Outcomes of Central Nervous System Tumors Harboring NTRK Gene Fusions}},
  url          = {{http://dx.doi.org/10.1158/1078-0432.CCR-24-0581}},
  doi          = {{10.1158/1078-0432.CCR-24-0581}},
  volume       = {{31}},
  year         = {{2025}},
}