Ecker Nóra, Aranyi Marietta, Kiss Edina, Kiss Nóra, Lahm Erika, Nagy Zsófia, Sikter Márta, Szabó Ádám, Szentesi Anikó Szászné, Takács Klára, Uhlyarik Andrea, Vachaja József, Sebők Barbara, Pápai Zsuzsanna
Department of Medical Oncology, Central Hospital of Northern Pest-Military Hospital, Budapest, Hungary.
Department of Pediatric Traumatology, Dr. Manninger Jenő Accident Center, Budapest, Hungary.
Pathol Oncol Res. 2025 Apr 1;31:1611965. doi: 10.3389/pore.2025.1611965. eCollection 2025.
Pazopanib is a tyrosine-kinase inhibitor also used for the treatment of advanced soft tissue sarcomas. Our retrospective study analyzed real-world data of stage 4 sarcoma patients treated with pazopanib in our department in the past 10 years. Data were collected from the Medworks medical system, which is used for daily work in our center. A total of 99 patients were included: 46 men and 53 women The median age at the diagnosis was 49.8 years. The most common histological subtypes were leiomyosarcoma and synovial sarcoma. All patients received 800 mg of pazopanib per day, which was reduced to 400 mg in the event of toxicity. Treatment was continued until disease progression or unmanageable toxicity. The primary endpoint of the study was progression-free survival and the secondary endpoints were overall survival, overall response rate and disease control rate. The results in relation to demographic data, previous treatments, localizations of primary tumors and metastasis and histological subtypes were analyzed. In our center pazopanib was most frequently used in the third line. In total, 61 patients received perioperative therapy; the most common regimen used in the metastatic setting was VIP. Median PFS and OS were 3 months and 7 months, respectively. ORR was 14% and DCR was 40.45%. Dose reductions were necessary during the treatment of 56 patients. Hematological toxicity was detected in 23% of cases, with the most frequent events being grade 1 thrombocytopenia and grade 2 leukocytopenia. Non-hematological adverse events were documented in half of the patients. Pazopanib was more effective in earlier lines of treatment. Compared to the PALETTE phase 3 trial more patients received perioperative therapy, median PFS and OS were shorter (3 months vs. 4.6 months and 7 months vs. 11.9 months) and ORR was higher (14% vs. 9%) in our patient population. Dose reductions were more frequent in our center. Pazopanib is a therapeutic option for the treatment of advanced soft tissue sarcoma, also according to real-world data. Further investigations are needed to select patients who can benefit the most from pazopanib and to determine the most appropriate sequence of therapy.
帕唑帕尼是一种酪氨酸激酶抑制剂，也用于治疗晚期软组织肉瘤。我们的回顾性研究分析了过去10年在我科接受帕唑帕尼治疗的IV期肉瘤患者的真实世界数据。数据从用于我们中心日常工作的Medworks医疗系统中收集。共纳入99例患者，其中男性46例，女性53例。诊断时的中位年龄为49.8岁。最常见的组织学亚型是平滑肌肉瘤和滑膜肉瘤。所有患者每天服用800mg帕唑帕尼，如出现毒性则减至400mg。治疗持续至疾病进展或出现无法控制的毒性。该研究的主要终点是无进展生存期，次要终点是总生存期、总缓解率和疾病控制率。分析了与人口统计学数据、既往治疗、原发肿瘤和转移灶的部位以及组织学亚型相关的结果。在我们中心，帕唑帕尼最常用于三线治疗。共有61例患者接受了围手术期治疗；转移情况下最常用的方案是VIP。中位无进展生存期和总生存期分别为3个月和7个月。总缓解率为14%，疾病控制率为40.45%。56例患者在治疗期间需要减量。23%的病例检测到血液学毒性，最常见的事件是1级血小板减少症和2级白细胞减少症。一半的患者记录了非血液学不良事件。帕唑帕尼在早期治疗中更有效。与PALETTE 3期试验相比，我们的患者群体中更多患者接受了围手术期治疗，中位无进展生存期和总生存期更短（3个月对4.6个月和7个月对11.9个月）且总缓解率更高（14%对9%）。在我们中心减量更频繁。根据真实世界数据，帕唑帕尼也是治疗晚期软组织肉瘤的一种治疗选择。需要进一步研究以选择能从帕唑帕尼中获益最大的患者，并确定最合适的治疗顺序。
