Department of Oncology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Republic of Korea.
Division of Hematology-Oncology, Department of Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Republic of Korea.
Target Oncol. 2020 Aug;15(4):485-493. doi: 10.1007/s11523-020-00731-z.
Pazopanib is the only tyrosine kinase inhibitor approved for the treatment of patients with advanced soft tissue sarcoma (STS) who have received prior chemotherapy, but there have been limited real-world data on pazopanib for the treatment of advanced STS.
We aimed to evaluate clinical outcomes of pazopanib in patients with multiple histologic STS types in real-world settings.
We retrospectively analyzed clinical data of Korean patients with advanced STS treated with pazopanib between 2008 and 2019. Outcomes of interest included treatment response, survival according to histologic subtypes, and adverse events.
The analysis included 347 STS patients. The disease control rate for all pazopanib-treated patients was 54.8% (95% confidence interval (CI) 49.5-60.0); 54 patients (15.6%) achieved a partial response and 136 (39.2%) had stable disease. Patients with alveolar soft-part sarcoma (ASPS; 90%), solitary fibrous tumor (SFT; 88.2%), synovial sarcoma (66.7%), leiomyosarcoma (61.1%), and undifferentiated pleomorphic sarcoma (59.6%) showed higher disease control rates than those with other STS subtypes. Overall, median progression-free survival (PFS) and overall survival (OS) were 5.3 months (95% CI 4.5-6.0) and 12 months (95% CI 10-14), respectively. Noticeable survival outcomes occurred in patients with ASPS and SFT, with a median PFS of 24.5 (95% CI 2.5-30.0) and 13.0 (95% CI 3.0-21.3) months, respectively. The median OS of patients with ASPS and SFT was 48 (95% CI 17-52) and 32 (95% CI 19-66) months, respectively. Adverse drug reactions occurred in 170 patients (49.0%) but were not life-threatening.
This real-world data analysis showed acceptable efficacy and tolerability of pazopanib in patients pretreated with cytotoxic chemotherapy for advanced STS, with favorable treatment outcomes for ASPS and SFT.
帕唑帕尼是唯一一种被批准用于治疗接受过化疗的晚期软组织肉瘤（STS）患者的酪氨酸激酶抑制剂，但在真实世界中，关于帕唑帕尼治疗晚期 STS 的数据有限。
我们旨在评估帕唑帕尼在真实环境中治疗多种组织学 STS 类型患者的临床结局。
我们回顾性分析了 2008 年至 2019 年间接受帕唑帕尼治疗的韩国晚期 STS 患者的临床数据。主要观察指标包括治疗反应、根据组织学亚型的生存情况以及不良反应。
该分析共纳入 347 例 STS 患者。所有接受帕唑帕尼治疗的患者的疾病控制率为 54.8%（95%置信区间[CI]：49.5-60.0）；54 例（15.6%）患者获得部分缓解，136 例（39.2%）患者疾病稳定。腺泡软组织肉瘤（ASPS；90%）、孤立性纤维瘤（SFT；88.2%）、滑膜肉瘤（66.7%）、平滑肌肉瘤（61.1%）和未分化多形性肉瘤（59.6%）患者的疾病控制率高于其他 STS 亚型患者。总体而言，中位无进展生存期（PFS）和总生存期（OS）分别为 5.3 个月（95%CI：4.5-6.0）和 12 个月（95%CI：10-14）。ASPS 和 SFT 患者的生存结局明显，中位 PFS 分别为 24.5 个月（95%CI：2.5-30.0）和 13.0 个月（95%CI：3.0-21.3），中位 OS 分别为 48 个月（95%CI：17-52）和 32 个月（95%CI：19-66）。170 例（49.0%）患者发生药物不良反应，但无生命威胁。
这项真实世界数据分析显示，帕唑帕尼在接受过细胞毒性化疗的晚期 STS 患者中具有可接受的疗效和耐受性，ASPS 和 SFT 患者的治疗结局良好。
