Improvement in Stiffness & Physical functioning in patients
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Pexidartinib decreased tumor size statistically significantly more than placebo after 25 weeks in patients with symptomatic, advanced tenosynovial giant cell tumor (TGCT) for whom surgery was not recommended, according to the ENLIVEN study. The effect of pexidartinib on patient-reported physical function and stiffness in ENLIVEN is detailed in this study.

This analysis included adults with symptomatic, advanced TGCT treated with pexidartinib or placebo. Physical function was assessed using the Patient-Reported Outcomes Measurement Information System: PROMIS - physical function (PF), and worst stiffness was assessed using a numerical rating scale (NRS).

The improvements in PROMIS-PF and worst stiffness NRS scores between treatment groups were compared using a mixed model for repeated measures from baseline to week 25. On the basis of threshold estimates from accurate change index and anchor-based methods, response rates for the PROMIS-PF and worst stiffness NRS at week 25 were determined.

Results:
--Between baseline and week 25, greater improvements in physical function and stiffness were experienced by patients receiving pexidartinib than patients receiving placebo; change in worst stiffness NRS.

--Patients receiving pexidartinib had higher response rates than patients receiving placebo for meaningful improvements in physical function and stiffness.

--Improvements were sustained after 50 weeks of pexidartinib treatment.

Pexidartinib treatment provided sustained, meaningful improvements in physical function and stiffness for patients with symptomatic, advanced TGCT.

Source: https://www.tandfonline.com/doi/full/10.1080/17453674.2021.1922161?af=R
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