Background: Uterine leiomyosarcoma (uLMS) is characterized by aggressive behavior associated with a high risk of relapse and mortality. Several therapeutic agents have been employed in the treatment of metastatic disease, with a poor objective response rate. Pazopanib, approved in 2012, is a multi-targeted, orally active small molecule that exerts its effects by inhibiting several tyrosine kinases. To date, poor research on real-life data has been conducted. We aimed to assess the effectiveness and safety of the drug in everyday clinical practice. Methods: We present results of multicenter retrospective data on 38 patients with heavily pretreated metastatic uLMS who underwent oral pazopanib during their therapeutic journey. Results: At a median follow-up of 8.6 months, the disease control rate was 55.2%, with 17% partial responses and 15 patients (39.5%) with stable disease. At a median follow-up of 8.6 months, median progression-free survival was 4 months, and median overall survival was 19.8 months. The most common grade 3 adverse events (AEs) drug-related were hepatic toxicities, diarrhea, hypertension, nausea, and vomiting (all of them with an incidence of 5% considering the whole study cohort). No grade 4 AEs occurred. Conclusions: Pazopanib in everyday clinical practice is safe and shows a good disease control rate with prolonged survival.
背景：子宫平滑肌肉瘤（uLMS）具有侵袭性行为特征，复发和死亡风险较高。多种治疗药物已被用于转移性疾病的治疗，但客观缓解率较低。帕唑帕尼于2012年获批，是一种多靶点口服活性小分子药物，通过抑制多种酪氨酸激酶发挥作用。迄今为止，关于真实世界数据的研究较少。本研究旨在评估该药物在日常临床实践中的有效性和安全性。 方法：本研究呈现了38例经过多线治疗的转移性uLMS患者的多中心回顾性数据结果，这些患者在其治疗过程中接受了口服帕唑帕尼治疗。 结果：中位随访8.6个月时，疾病控制率为55.2%，其中部分缓解率为17%，15例患者（39.5%）病情稳定。中位无进展生存期为4个月，中位总生存期为19.8个月。最常见的3级药物相关不良事件为肝毒性、腹泻、高血压、恶心和呕吐（在整个研究队列中发生率均为5%）。未发生4级不良事件。 结论：帕唑帕尼在日常临床实践中安全性良好，显示出较高的疾病控制率和较长的生存期。
肿瘤(癌症)患者之家