Safety and efficacy of pazopanib as a second-line treatment and beyond for soft tissue sarcomas: A real-life tertiary-center experience in the MENA region.
Safety and efficacy of pazopanib as a second-line treatment and beyond for soft tissue sarcomas: A real-life tertiary-center experience in the MENA region.
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DOI：
10.1016/j.ctarc.2020.100275
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发表时间：
2021-01-01
影响因子：
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通讯作者：
Assi, Hazem I
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文献类型：
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作者：
Halim, Nour Abdul;Sayed, Rola El;Assi, Hazem I
肉瘤是一种罕见的恶性肿瘤。尽管多方努力，最近仍未取得进展。Pazopanib是一种安全有效的酪氨酸激酶抑制剂，用于治疗化疗失败后的软组织肉瘤（STS）。然而，它在发展中国家的使用是有限的，并且没有来自我们区域的疗效数据。我们的目的是研究帕唑帕尼在我们人群中的疗效，以化疗难治性晚期STS患者接受帕唑帕尼的缓解率为特征。次要终点包括无进展生存期（PFS）、总生存期（OS）和毒性特征。材料与方法：纳入2014年1月1日至2018年10月31日在黎巴嫩的一个三级中心接受帕唑帕尼作为≥二线治疗的15例（年龄≥18岁）诊断为晚期STS，一线化疗难治性患者。从回顾性图表中提取患者和疾病特征、疾病评估以及对治疗的耐受性。统计学分析采用SPSS version 24。结果：患者平均年龄48.6岁[19-66]。11名患者（73.3%）在二线接受了pazopanib，而4名患者（26.7%）在三线接受了pazopanib。13例（86.7%）进展，2例（13.3%）病情稳定。中位PFS为3个月[1-19]，平均OS为25.4个月[17.2-33.6]。5例患者因耐受性差需要减量。结论：由于患者数量少，不能得出结论。然而，考虑到3个月的PFS， 13%的患者保持疾病稳定，以及可耐受的安全性，将帕唑帕尼纳入STS治疗是合理的。需要在黎巴嫩进行更多有针对性的研究和更大的患者群体。
INTRODUCTION: Sarcomas are uncommon malignancies. No advances have been recently achieved despite multiple efforts. Pazopanib is a safe and effective tyrosine kinase inhibitor used in managing soft tissue sarcomas (STS) after chemotherapy failure. However, its use is limited in developing countries and no efficacy data exist from our region. We aimed to study the efficacy of pazopanib in our population, characterized by response rates of patients with chemotherapy-refractory advanced STS receiving pazopanib. Secondary endpoints included progression-free survival (PFS), overall survival (OS) and toxicity profile.MATERIALS AND METHODS: 15 patients (age≥18 year) diagnosed with advanced STS, refractory to first-line chemotherapy, receiving pazopanib as ≥second-line therapy in one tertiary center in Lebanon were included betweenJanuary 1st, 2014 and October 31st, 2018. Patient and disease characteristics, disease evaluation, as well as tolerance to treatment, were extracted from charts retrospectively. Statistical analysis was done using SPSS version 24.RESULTS: The mean age was 48.6 [19-66] years.Eleven patients (73.3%) received pazopanib in second-line, whereas four patients (26.7%) received it in third-line.Thirteen patients (86.7%) progressed, and two patients (13.3%) had stable disease. The median PFS was three months [1-19] and the mean OS was 25.4 months [17.2-33.6]. Five patients required dose-reductions due to poor tolerance.CONCLUSION: Conclusions cannot be drawn due to small patient numbers. However, given the 3-month PFS, 13% of patients maintaining stable disease, and tolerable safety profile, it is reasonable to incorporate pazopanib in STS treatment. More focused studies with larger patient populations need to be done inLebanon.