Mantiero Mara, Bini Marta, Polignano Maggie, Porcu Luca, Sanfilippo Roberta, Fabbroni Chiara, Parma Gabriella, Lapresa Mariateresa, Calidona Carmelo, Silvestri Cecilia, Franza Andrea, Raspagliesi Francesco, Colombo Nicoletta, Ducceschi Monika
Department of Gynecologic Oncology, National Cancer Institute of Milan, Via Venezian 1, 20133 Milan, Italy.
Department of Medical Oncology, National Cancer Institute of Milan, Via Venezian 1, 20133 Milan, Italy.
Cancers (Basel). 2023 Dec 30;16(1):192. doi: 10.3390/cancers16010192.
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.
We present results of multicenter retrospective data on 38 patients with heavily pretreated metastatic uLMS who underwent oral pazopanib during their therapeutic journey.
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.
Pazopanib in everyday clinical practice is safe and shows a good disease control rate with prolonged survival.
子宫平滑肌肉瘤（uLMS）具有侵袭性，复发和死亡风险高。几种治疗药物已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、口服有效的小分子药物，通过抑制多种酪氨酸激酶发挥作用。迄今为止，对真实世界数据的研究较少。我们旨在评估该药物在日常临床实践中的有效性和安全性。
我们展示了38例接受过大量治疗的转移性uLMS患者在治疗过程中口服帕唑帕尼的多中心回顾性数据结果。
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。中位随访8.6个月时，无进展生存期为4个月，总生存期为19.8个月。最常见的3级药物相关不良事件是肝毒性、腹泻、高血压、恶心和呕吐（考虑整个研究队列，所有这些不良事件的发生率均为5%）。未发生4级不良事件。
在日常临床实践中，帕唑帕尼是安全的，并且显示出良好的疾病控制率和延长的生存期。
