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Trametinib-induced Left Ventricular Dysfunction in a Child With Relapsed Neuroblastoma.

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Trametinib, a MEK inhibitor, caused cardiac dysfunction in a pediatric neuroblastoma patient with an NRAS mutation. While reversible, the drug did not control tumor progression, highlighting potential risks in children.

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Area of Science:

  • Pediatric oncology
  • Pharmacology
  • Genetics

Background:

  • Trametinib is approved for BRAF-mutated melanoma.
  • Its use in pediatric patients is not reported.
  • Neuroblastoma is a challenging pediatric cancer.

Observation:

  • An 11-year-old male with relapsed neuroblastoma had an NRASQ61K mutation.
  • He received trametinib after normal cardiac function.
  • Cardiac dysfunction developed on day 13.

Findings:

  • Trametinib was discontinued on day 15.
  • Left ventricular function recovered after stopping trametinib.
  • Neuroblastoma progression continued despite treatment.

Implications:

  • Trametinib may pose cardiac risks in pediatric patients.
  • NRAS-mutated neuroblastoma may not respond to trametinib.
  • Further research is needed on MEK inhibitors in pediatric cancers.