Abstract
Background
Infantile fibrosarcoma is the most frequent soft tissue sarcoma in newborns or children under one year of age. This tumour often implies high local aggressiveness and surgical morbidity. The large majority of these patients carry the ETV6–NTRK3 oncogenic fusion. Hence, the TRK inhibitor larotrectinib emerged as an efficacious and safe alternative to chemotherapy for NTRK fusion-positive and metastatic or unresectable tumours. However, real-world evidence is still required for updating soft-tissue sarcoma practice guidelines.
Objective
To report our experience with the use of larotrectinib in pediatric patients.
Methods
Our case series shows the clinical evolution of 8 patients with infantile fibrosarcoma under different treatments. All patients enrolled in this study received informed consent for any treatment.
Results
Three patients received larotrectinib in first line. No surgery was needed with larotrectinib, which led to the rapid and safe remission of tumours, even in unusual anatomical locations. No significant adverse effects were observed with larotrectinib.
Conclusion
Our case series supports that larotrectinib may be a therapeutic option for newborn and infant patients with infantile fibrosarcoma, especially in uncommon locations.
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Data availability statement
Data are available at demand.
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Acknowledgements
The authors would like to thank the parents of the patients for allowing us to present their clinical data. The authors would also like to thank CRIS Cancer Foundation (https://criscancer.org/en/) for its continuous research support and Bayer Spain for financing medical writing assistance, which was provided by Neus Cantariño (Trialance SCCL, Spain).
Funding
Medical writing services were funded by Bayer Spain.
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MDCS and APM contributed to the acquisition, analysis and interpretation of data for the work and wrote the first draft of the manuscript. RJC, PRA, DPLS, ASU, JJP-K, JCLG, JMGC, and EJOC contributed to the acquisition, analysis and interpretation of data for the work. All authors contributed to the manuscript revision, and read and approved the submitted version.
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Patients enrolled in this study received informed consent for any treatment.
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Ethical approval of the study was not obtained because we have IC from the patiens.
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Corral Sánchez, M.D., Jiménez Carrascoso, R., Rubio Aparicio, P. et al. Therapeutic strategies and clinical evolution of patients with infantile fibrosarcoma: a unique paediatric case series. Clin Transl Oncol 25, 3307–3311 (2023). https://doi.org/10.1007/s12094-023-03175-9
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DOI: https://doi.org/10.1007/s12094-023-03175-9