1. Tumor response rate at 25 weeks was significantly greater in vimseltinib than placebo.

2. Most adverse events were mild-to-moderate with no treatment-related fatalities.

Evidence Rating Level: 1 (Excellent)

Study Rundown: Tenosynovial giant cell tumour (TGCT) is an aggressive neoplasm of the tendon sheath with limited treatment options. Vimseltinib, an oral CSF1R inhibitor, has shown promise for symptomatic TGCT patients, although further research is needed. This randomized controlled trial aimed to assess the safety and efficacy of vimseltinib compared to placebo among patients with TGCT not amenable to surgery. The primary outcome was Response Evaluation Criteria in Solid Tumors (RECIST) score at 25 weeks, while key secondary outcomes included the safety profile. According to study results, vimseltinib demonstrated a significantly increased objective response rate compared to the placebo group. Although this study was well done, it was limited by a relatively small sample size and short-term follow-up period.

Click to read the study in The Lancet

Relevant Reading: Pexidartinib versus placebo for advanced tenosynovial giant cell tumour (ENLIVEN): a randomised phase 3 trial

In-depth [randomized controlled trial]: Between Jan 21, 2022, and Feb 21, 2023, 146 patients were enrolled across 35 hospitals in 13 countries. Included were patients ≥ 18 years old with histologically confirmed TGCT ineligible for surgery. Altogether, 123 patients (80 in vimseltinib and 40 in placebo) were included in the final analysis. The primary outcome of response rate per RECIST at week 25 was significantly greater in vimseltinib than placebo (difference 40%, 95% confidence interval [CI] 29-51, p<0.0001). The majority of adverse events were mild-to-moderate in severity, with increased blood creatine phosphokinase being the predominant grade 3 or 4 event (10%) in vimseltinib-treated patients. Findings from this study suggest that vimseltinib offers an effective treatment for symptomatic TGCT patients not suitable for surgery, demonstrating clinical benefit in tumor response and symptom management.

Image: PD

©2024 2 Minute Medicine, Inc. All rights reserved. No works may be reproduced without expressed written consent from 2 Minute Medicine, Inc. Inquire about licensing here. No article should be construed as medical advice and is not intended as such by the authors or by 2 Minute Medicine, Inc.