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This case report describes the outcomes of tropomyosin receptor kinase (TRK) inhibitor treatment in an infant with an infantile fibrosarcoma (IFS) with a TPM3–NTRK1 gene fusion. The IFS on the left foot was refractory to chemotherapy and was partially resected. After 5 months, there was local recurrence and further surgery was performed. The patient developed pulmonary and central nervous system metastases. Pan-TRK antibody staining was positive and genomic profiling with next-generation sequencing confirmed a TPM3–NTRK1 gene fusion. The patient started treatment with larotrectinib in February 2019, with a durable response, including a clear reduction in brain metastases. Research on novel gene fusions and molecular testing to direct the use of targeted therapy should be encouraged, especially in refractory solid tumors.

clinical oncology and research

Targeted Therapy Using the Selective Tropomyosin Kinase Receptor Inhibitor Larotrectinib in an Infant with Infantile Fibrosarcoma with a TPM3–NTRK1 Gene Fusion with Lung and Central Nervous System Metastases: Case Report