派西达替尼对东亚腱鞘巨细胞瘤患者疗效和安全性的3期研究。

IF 3.2 Q2 ONCOLOGY

Oncology and Therapy Pub Date : 2025-08-21 DOI:10.1007/s40487-025-00365-z

Hairong Xu, Po-Kuei Wu, Zhaoming Ye, Zhengfu Fan, Tao Li, Tom Wei-Wu Chen, Jingnan Shen, Wangjun Yan, Issei Achiwa, Kazukoh Yoh, Kunika Kikumori, Yoshiharu Hiruma, Jonathan Greenberg, Xiaohui Niu

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引用次数: 0

摘要

本研究的目的是评估培西达替尼（一种集落刺激因子1受体抑制剂）在东亚腱鞘巨细胞瘤（TGCT）患者中的疗效和安全性。方法：这项多中心、单组、3期研究（NCT04488822）纳入了有症状的TGCT患者，接受培西达替尼400mg，每日2次。主要疗效终点是在第25周时每个中心评价的实体肿瘤反应评价标准（RECIST v1.1）的总缓解率（ORR）。次要终点包括肿瘤体积评分（TVS）的ORR，受影响关节的活动范围变化（ROM），最佳总体反应（BOR）和治疗后出现的不良事件（teae）。结果：共有40名东亚TGCT患者接受了培西达替尼治疗。RECIST v1.1 ORR为22.5%(95%可信区间[CI] 10.8-38.5)，无完全缓解（cr）， 9例部分缓解（pr）， 21例病情稳定（Sd）， 1例病情进展（PD）。TVS的ORR为47.5% (95% CI 31.5-63.9)，无cr， 19例pr， 12例Sd，无PD。在第25周，ROM的平均（标准误差[SE]）变化为23.05(3.207)，在第13周有所改善（15.64[3.312]）。RECIST v1.1 BOR为30.0% (95% CI 16.6-46.5)， TVS BOR为47.5% （95% CI 31.5-63.9）。所有患者均发生teae， 16例（40.0%）患者报告teae≥3级。最常见的teae是发色改变（77.5%）；丙氨酸转氨酶（60.0%）、天冬氨酸转氨酶（57.5%）、血乳酸脱氢酶（47.5%）升高；瘙痒症（47.5%）。结论：在东亚有症状的TGCT患者中，培西达替尼显示出临床益处，其安全性与先前的报道相当。试验注册：ClinicalTrials.gov识别码NCT04488822。

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A Phase 3 Study of the Efficacy and Safety of Pexidartinib in East Asian Patients with Tenosynovial Giant Cell Tumor.

Introduction: The aim of this study was to assess the efficacy and safety of pexidartinib, an inhibitor of colony-stimulating factor 1 receptor, in East Asian patients with tenosynovial giant cell tumor (TGCT).

Methods: This multicenter, single-arm, phase 3 study (NCT04488822) enrolled patients with symptomatic TGCT to receive pexidartinib 400 mg twice daily. The primary efficacy endpoint was overall response rate (ORR) by Response Evaluation Criteria in Solid Tumors (RECIST v1.1) per central review at week 25. Secondary endpoints included ORR by tumor volume score (TVS), change in range of motion (ROM) of the affected joint, best overall response (BOR), and treatment-emergent adverse events (TEAEs).

Results: A total of 40 East Asian patients with TGCT received pexidartinib. RECIST v1.1 ORR was 22.5% (95% confidence interval [CI] 10.8-38.5), with no complete responses (CRs), 9 partial responses (PRs), 21 patients with stable disease (Sd), and 1 patient with progressive disease (PD). TVS ORR was 47.5% (95% CI 31.5-63.9), with no CRs, 19 PRs, 12 patients with Sd, and none with PD. At week 25, mean (standard error [SE]) change in ROM was 23.05 (3.207), with improvement noted at week 13 (15.64 [3.312]). RECIST v1.1 BOR was 30.0% (95% CI 16.6-46.5), and TVS BOR was 47.5% (95% CI 31.5-63.9). All patients had TEAEs, and grade ≥ 3 TEAEs were reported in 16 (40.0%) patients. The most common TEAEs were hair color changes (77.5%); increases in alanine aminotransferase (60.0%), aspartate aminotransferase (57.5%), and blood lactate dehydrogenase (47.5%); and pruritus (47.5%).

Conclusions: In East Asian patients with symptomatic TGCT, pexidartinib demonstrated clinical benefits, with a safety profile comparable to previous reports.

Trial registration: ClinicalTrials.gov identifier NCT04488822.

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来源期刊

Oncology and Therapy ONCOLOGY-

CiteScore

3.40

自引率

0.00%

发文量

审稿时长

6 weeks

期刊介绍： Now indexed in PubMed Aims and Scope Oncology and Therapy is an international, peer reviewed, rapid-publication (peer review in 2 weeks, published 3–4 weeks from acceptance) journal dedicated to the publication of high-quality pre-clinical, clinical (all phases), observational, real-world, and health outcomes research around the discovery, development, and use of therapeutics and interventions (including devices) across all therapeutic areas. Studies relating to diagnostics and diagnosis, pharmacoeconomics, public health, epidemiology, quality of life, and patient care, management, and education are also encouraged. The journal is of interest to a broad audience of healthcare professionals and publishes original research, reviews, communications and letters. The journal is read by a global audience and receives submissions from all over the world. Oncology and Therapy will consider all scientifically sound research be it positive, confirmatory or negative data. 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