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A newborn with a large NTRK fusion positive infantile fibrosarcoma successfully treated with larotrectinib
Kenneth J Caldwell1, Esther De La Cuesta2, Cara Morin3
1Department of Oncology, St Jude Children's Research Hospital, Memphis, Tennessee.
Abstract:
Infantile fibrosarcoma (IFS) is a rare pediatric cancer that typically presents early in life. Surgical resection is commonly curative; however, resection is sometimes not possible requiring additional multimodal treatment. IFS commonly harbors a fusion in one of the neurotrophic receptor tyrosine kinase (NTRK) genes. Larotrectinib, a highly selective inhibitor of tropomyosin receptor kinase (TRK), has been shown to be well tolerated and effective in children as young as 1-month old. We report a case of IFS in a newborn treated with larotrectinib. The patient experienced a rapid clinical and radiographic response demonstrating the potential to treat newborns with larotrectinib.
Insights
Infantile fibrosarcoma (IFS) is a rare pediatric cancer. Larotrectinib effectively treated a newborn with IFS, showing rapid clinical and radiographic response, highlighting its potential for treating this condition in newborns.
Area of Science:
- Pediatric Oncology
- Molecular Oncology
Background:
- Infantile fibrosarcoma (IFS) is a rare pediatric malignancy.
- Surgical resection is the primary curative treatment, but is not always feasible.
- Many IFS cases harbor neurotrophic receptor tyrosine kinase (NTRK) gene fusions.
Observation:
- A case of infantile fibrosarcoma in a newborn is presented.
- The patient was treated with larotrectinib, a tropomyosin receptor kinase (TRK) inhibitor.
- Larotrectinib is known for its safety and efficacy in young children.
Findings:
- The newborn with infantile fibrosarcoma experienced a rapid clinical and radiographic response to larotrectinib.
- This response demonstrates the drug's effectiveness in a neonatal patient.
- The treatment was well-tolerated by the infant.
Implications:
- Larotrectinib shows promise as a treatment option for infantile fibrosarcoma in newborns.
- Targeted therapy with TRK inhibitors may be a viable strategy for neonatal cancers.
- Further investigation into larotrectinib for neonatal IFS is warranted.
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