TriangleHEALTH PROFESSIONAL · SOURCE READING
Larotrectinib
Source: Thyroid Cancer Treatment (PDQ®)–Health Professional Version, National Cancer Institute.
Source updated: May 9, 2025 · Captured 2026-09-09.
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Larotrectinib is a potent and highly selective small-molecule inhibitor of the TRKA, TRKB, and TRKC proteins.
Evidence (larotrectinib):
An expanded pooled efficacy analysis included 159 patients with TRK fusion−positive cancers treated with larotrectinib. The safety analysis included 260 patients treated with larotrectinib, regardless of TRK gene fusion status.[40]
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An expanded pooled efficacy analysis included 159 patients with TRK fusion−positive cancers treated with larotrectinib. The safety analysis included 260 patients treated with larotrectinib, regardless of TRK gene fusion status.[40]
For the 24 patients with thyroid cancer, the overall response rate was 79% (95% CI, 58%−93%). The median duration of response was NE (14.8 months−NE).[40][Level of evidence C2]
Source links and citations
An expanded pooled efficacy analysis included 159 patients with TRK fusion−positive cancers treated with larotrectinib. The safety analysis included 260 patients treated with larotrectinib, regardless of TRK gene fusion status.[40]
Of the 260 patients in the safety analysis, 13% had grade 3 treatment-related adverse events and less than 1% had grade 4 treatment-related adverse events.
An expanded pooled efficacy analysis included 159 patients with TRK fusion−positive cancers treated with larotrectinib. The safety analysis included 260 patients treated with larotrectinib, regardless of TRK gene fusion status.[40] › Of the 260 patients in the safety analysis, 13% had grade 3 treatment-related adverse events and less than 1% had grade 4 treatment-related adverse events.
The most common grade 3 or 4 treatment-related adverse events were increased alanine aminotransferase (3%), anemia (2%), and decreased neutrophil count (2%).
An expanded pooled efficacy analysis included 159 patients with TRK fusion−positive cancers treated with larotrectinib. The safety analysis included 260 patients treated with larotrectinib, regardless of TRK gene fusion status.[40] › Of the 260 patients in the safety analysis, 13% had grade 3 treatment-related adverse events and less than 1% had grade 4 treatment-related adverse events.
Doses were reduced in 8% of patients with TRK fusion–positive cancer because of adverse events. The drug was discontinued in 2% of patients because of treatment-related adverse events.
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Preserved source evidence · Not medical advice