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采用培昔替尼治疗腱鞘巨细胞瘤的前景:综述。

Prospects of Treating Tenosynovial Giant Cell Tumor through Pexidartinib: A Review.

机构信息

Department of Pharmaceutical Chemistry & Analysis, ISF College of Pharmacy, G.T. Road, Moga-142 001, Punjab, India.

出版信息

Anticancer Agents Med Chem. 2021;21(12):1510-1519. doi: 10.2174/1871520620999201102123555.

Abstract

BACKGROUND

Tenosynovial giant cell tumor refers to a group of rarely occurring tumors that are formed in the joints, which are characterized by pain, swelling, and limitation of movement of the joint. Surgery is the main treatment strategy, but the tumor is likely to recur, especially in pigmented villonodular synovitis, which is the diffuse-type giant cell tumor. Pexidartinib was approved in August 2019 by the Food and Drug Administration (FDA) with a brand name TURALIO as the first systemic approved therapy for patients having Tenosynovial Giant Cell Tumors (TGCT).

OBJECTIVE

In this review, different aspects pertaining to pexidartinib have been summarized, including the pathophysiology of TGCT, chemistry, pharmacokinetics and pharmacodynamics of pexidartinib. Special attention is given to various reported clinical trials of pexidartinib.

METHODS

A comprehensive literature search was conducted in the relevant databases to identify studies published in this field during recent years.

CONCLUSION

Pexidartinib acts by inhibiting the Colony-Stimulating Factor (CSF1)/CSF1 receptor pathway, which leads to the inhibition of the cell lines proliferation and promotes the autophosphorylation process of the ligand-induced CSF1 receptor. Pexidartinib emerged as a potential drug candidate for the treatment of TGCT.

摘要

背景

腱鞘巨细胞瘤是一组罕见发生于关节的肿瘤,其特征为关节疼痛、肿胀和活动受限。手术是主要的治疗策略,但肿瘤可能会复发,尤其是在弥漫型巨细胞肿瘤即色素绒毛结节性滑膜炎中。pexidartinib 于 2019 年 8 月被美国食品和药物管理局(FDA)批准,商品名为 TURALIO,成为首个用于治疗腱鞘巨细胞瘤(TGCT)患者的全身性批准疗法。

目的

本综述总结了 pexidartinib 的各个方面,包括 TGCT 的病理生理学、化学、药代动力学和药效学。特别关注了 pexidartinib 的各种已报道临床试验。

方法

在相关数据库中进行了全面的文献检索,以确定近年来该领域发表的研究。

结论

pexidartinib 通过抑制集落刺激因子(CSF1)/CSF1 受体途径发挥作用,从而抑制细胞系增殖,并促进配体诱导的 CSF1 受体的自动磷酸化过程。pexidartinib 作为治疗 TGCT 的潜在药物候选物出现。

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