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.

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©2024 The Authors; Published by the American Association for Cancer Research
2024
American Association for Cancer Research
This open access article is distributed under the Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International (CC BY-NC-ND 4.0) license.
This work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License .
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Supplementary data

Supplementary Data S1

Supplementary Data S1. Definition of response, Description of response criteria used by sites to evaluate tumor response, Definition of extend of resection, Definition of extend of resection used by sites.

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Supplementary Figure S1

Supplementary Figure S1. Diagram of population patients.

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Supplementary Figure S2

Supplementary Figure S2. A) Hazard ratio for progression or death according to clinical characteristics. B) Hazard ratio for death according to clinical characteristics. LGG: Low-grade glioma, HGG: High-grade glioma

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Supplementary Table S1

Supplementary Table S1. NTRK fusion type and histology.

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Supplementary Table S2

Supplementary Table S2. Responses based on clinical assessment for each line of therapy. Non-targeted therapy and TRKi given concurrently in a specific line were excluded.

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Supplementary Table S3

Supplementary Table S3. Survival outcome.

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Supplementary Table S4

Supplementary Table S4. Other molecular alterations reported.

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Supplementary Table S5

Supplementary Table S5. Method of NTRK fusion detection as reported by participating centers.

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