Purpose: Pazopanib has been approved for treating soft tissue sarcomas (STS) after chemotherapy. We aimed to evaluate the prognostic factors, clinical outcomes, and tolerability of pazopanib in patients with STS.
Patients and Methods: Forty-five patients treated between June 2015 and August 2019 were reviewed. Clinical outcome was measured by assessing the disease control rate (DCR) using Response Evaluation Criteria in Solid Tumors (version 1.1). Progression-free survival (PFS) and overall survival (OS) were estimated using the Kaplan–Meier method. Adverse effects were assessed using the Common Terminology Criteria for Adverse Events (version 5.0).
Results: The median age of patients at diagnosis was 28 (interquartile range (IQR), 23– 45) years. Pazopanib was used as the second-line treatment in 46.7% and the subsequent line in 53.3% of patients. The overall DCR was 55.6%, and at 8 and 12 weeks, it was 52.3% and 35.5%, respectively; the median duration of response was 7 (IQR: 2– 18) months. Pazopanib-induced hypothyroidism was associated with DCR, with an odds ratio of 7 (95% confidence interval [95% CI: 1.7– 27.5], p< 0.01). The median PFS and OS were 4.1 (95% CI: 0.85– 7.42) and 12.4 months (95% CI: 6.5– 18.36), respectively. Hypothyroidism and response to pazopanib, better ECOG PS, histological subtypes desmoid tumor/aggressive fibromatosis (DT/AF), and alveolar soft part sarcoma (ASPS) were favorable prognostic factors for PFS. Hypothyroidism and response to pazopanib were significant favorable factors for OS. There was no statistical difference in the OS between patients using pazopanib as the second-line therapy and those using it as the subsequent-line therapy.
Conclusion: Pazopanib is an effective treatment for STS. However, it showed variability in the clinical outcome in favor of ASPS and an outstanding response in the DF/AT subtype. Pazopanib-induced hypothyroidism is a good prognostic factor for disease control and is associated with prolonged PFS and OS.

Keywords: STS, a tyrosine kinase inhibitor, pazopanib, DT/AF, UPS, LMS, ASPS, Saudi Arabia


中文翻译：

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帕唑帕尼治疗晚期软组织肉瘤的真实结局和预后因素

目的：帕唑帕尼已被批准用于治疗化疗后的软组织肉瘤（STS）。我们旨在评估帕唑帕尼在 STS 患者中的预后因素、临床结果和耐受性。
患者和方法：回顾了 2015 年 6 月至 2019 年 8 月期间治疗的 45 名患者。通过使用实体瘤反应评估标准（1.1 版）评估疾病控制率 (DCR) 来测量临床结果。使用 Kaplan-Meier 方法估计无进展生存期 (PFS) 和总生存期 (OS)。使用不良事件通用术语标准（5.0 版）评估不良反应。
结果：诊断时患者的中位年龄为 28 岁（四分位距 (IQR)，23-45）岁。帕唑帕尼在 46.7% 的患者中被用作二线治疗，在 53.3% 的患者中被用作后续治疗。总体 DCR 为 55.6%，8 周和 12 周时分别​​为 52.3% 和 35.5%；中位反应持续时间为 7 (IQR: 2-18) 个月。帕唑帕尼诱导的甲状腺功能减退与 DCR 相关，优势比为 7（95% 置信区间 [95% CI：1.7-27.5]，p<0.01）。中位 PFS 和 OS 分别为 4.1（95% CI：0.85–7.42）和 12.4 个月（95% CI：6.5–18.36）。甲状腺功能减退和对帕唑帕尼的反应、更好的 ECOG PS、组织学亚型硬纤维瘤/侵袭性纤维瘤病 (DT/AF) 和肺泡软部分肉瘤 (ASPS) 是 PFS 的有利预后因素。甲状腺功能减退和对帕唑帕尼的反应是 OS 的显着有利因素。使用培唑帕尼作为二线治疗的患者与使用它作为后续治疗的患者之间的 OS 没有统计学差异。
结论：帕唑帕尼是治疗STS的有效方法。然而，它显示出有利于 ASPS 的临床结果的变异性和 DF/AT 亚型的显着反应。帕唑帕尼诱导的甲状腺功能减退是疾病控制的良好预后因素，并与延长 PFS 和 OS 相关。

关键词： STS，酪氨酸激酶抑制剂，帕唑帕尼，DT/AF，UPS，LMS，ASPS，沙特阿拉伯