Summary

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• Turalio (pexidartinib) is indicated for the treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT) associated with severe morbidity or functional limitations and not amenable to improvement with surgery.
• This summary is based on the review of four randomized controlled trial(s). ^[1-4]
• Tumor Response: In the ENLIVEN Phase 3 Study, pexidartinib showed a significant objective tumor response in adults with tenosynovial giant cell tumors (TGCT) not amenable to surgery, with 39% of patients achieving overall tumor shrinkage by RECIST 1.1 criteria at week 25, compared to 0% in the placebo group (absolute difference of 39%; 95% CI 27-53; p<0.0001).
• Pain Relief: The ENLIVEN Phase 3 Study indicated a modest reduction in pain with pexidartinib. Primary assessment of pain response showed no significant difference (31% vs. 15%; one-sided p = 0.03), though exploratory analyses revealed some improvement with pexidartinib (26% vs. 10%; one-sided p = 0.02 at the 50% threshold, and 31% vs. 14%; one-sided p = 0.02 using the MCID threshold). The mean difference in weekly mean worst-pain NRS score was -2.2 (95% CI -3.0 to -1.4; p<0.001).
• Physical Function and Stiffness: Patients treated with pexidartinib in the ENLIVEN Phase 3 Study demonstrated greater improvements in physical function and stiffness compared to placebo. Changes in PROMIS-PF (physical function) were 4.1 (95% CI 1.8-6.3) for pexidartinib versus -0.9 (95% CI -3.0 to 1.2) for placebo, while changes in worst stiffness NRS were -2.5 (95% CI -3.0 to -1.9) for pexidartinib versus -0.3 (95% CI -0.9 to 0.3) for placebo.
• The most frequent adverse events associated with pexidartinib in the ENLIVEN Phase 3 Study were hair color changes (67%), fatigue (54%), increased aspartate aminotransferase (AST) (39%), nausea (38%), increased alanine aminotransferase (ALT) (28%), and dysgeusia (25%). Serious adverse events occurred in 13% of patients in the pexidartinib group compared to 2% in the placebo group.
• Hepatotoxicity was a significant safety concern in the ENLIVEN Phase 3 Study, with three patients experiencing aminotransferase elevations three or more times the upper limit of normal along with elevated total bilirubin and alkaline phosphatase levels, indicative of mixed or cholestatic hepatotoxicity. This led to the halting of enrollment by the data monitoring committee, with one case confirmed by biopsy lasting 7 months.
• There is no population types or subgroups information available in the reviewed studies.