Tenosynovial giant cell tumour (TGCT) is a locally aggressive neoplasm of the tendons and cannot always be managed surgically. TGCT is driven by dysregulated CSF1 production and CSF1R-dependent inflammatory macrophages, and the CSF1R-directed multitargeted tyrosine-kinase inhibitor (TKI) pexidartinib is an FDA-approved systemic treatment for this disease but can cause potentially fatal cholestatic hepatoxicity. Now, the phase III MOTION trial presents the CSF1R-selective TKI vimseltinib as an efficacious and possibly safer alternative.

In MOTION, 123 patients with TGCT not amenable to resection were randomly assigned (2:1) to receive twice-weekly vimseltinib or placebo, regardless of prior treatment (excepting CSF1R-selective agents). Objective response rate (ORR) at week 25 was the primary end point.

腱鞘巨细胞瘤（TGCT）是一种局部侵袭性肌腱肿瘤，不能总是通过手术治疗。 TGCT 是由 CSF1 产生失调和 CSF1R 依赖性炎症巨噬细胞驱动的，针对 CSF1R 的多靶点酪氨酸激酶抑制剂 (TKI) 培西达替尼 (pexidartinib) 是 FDA 批准的治疗该疾病的全身疗法，但可能导致潜在致命的胆汁淤积性肝毒性。现在，III 期 MOTION 试验将 CSF1R 选择性 TKI vimseltinib 作为一种有效且可能更安全的替代方案。

在 MOTION 中，123 名不适合切除的 TGCT 患者被随机分配 (2:1) 接受每周两次的 vimseltinib 或安慰剂，无论先前的治疗情况如何（CSF1R 选择性药物除外）。第 25 周的客观缓解率 (ORR) 是主要终点。