BACKGROUND: The effect of chemotherapy in metastatic bone sarcomas is poor and the condition is invariably fatal. Therefore, new treatment modalities are intensely needed. Pazopanib is a selective multitargeted tyrosine kinase inhibitor that has proven to be effective in the treatment of metastatic soft tissue sarcomas. The objective of this study was to evaluate the off-label use of pazopanib in patients with metastatic bone sarcomas who failed standard chemotherapy. METHODS: All patients with metastatic bone sarcomas treated with pazopanib between October 1st, 2011 and October 1st, 2017 at the Department of Oncology, Aarhus University Hospital were evaluated. Demographics, treatment, and survival outcomes were collected and analyzed. RESULTS: Nineteen patients were identified. The median age was 38 years (range 18–62). Most of the patients (50%) were diagnosed with osteosarcoma. All patients had documented disease progression at the time of initiating pazopanib treatment. The median overall survival was 11 months. Median progression free survival was 5.4 months. Out of 19 patients, 13 (68%) had either partial response or stable disease. In five patients, the dose of pazopanib was reduced because of toxicity. CONCLUSION: Off-label use of pazopanib is effective in the treatment of metastatic bone sarcomas of different histologies. Pazopanib was well tolerated in the treatment of patients with refractory bone sarcomas. Studies examining the effect of pazopanib alone or in combination with chemotherapy or other targeted therapies are needed.

背景：化学疗法在转移性骨肉瘤中效果不佳，而且病情总是致命的。因此，迫切需要新的治疗方式。帕唑帕尼是一种选择性的多靶点酪氨酸激酶抑制剂，已被证明可有效治疗转移性软组织肉瘤。这项研究的目的是评估帕唑帕尼在标准化疗失败的转移性骨肉瘤患者中的标签外使用。方法：对2011年10月1日至2017年10月1日在奥胡斯大学医院肿瘤科接受帕唑帕尼治疗的所有转移性骨肉瘤患者进行评估。人口统计学，治疗和生存结果被收集和分析。结果：确定了19名患者。中位年龄为38岁（范围18-62）。大多数患者（50％）被诊断出患有骨肉瘤。在开始帕唑帕尼治疗时，所有患者均已记录疾病进展。中位总生存期为11个月。中位无进展生存期为5.4个月。在19例患者中，有13例（68％）有部分反应或疾病稳定。在五名患者中，由于毒性降低了帕唑帕尼的剂量。结论：非处方使用帕唑帕尼可有效治疗不同组织学转移性骨肉瘤。帕唑帕尼在难治性骨肉瘤患者的治疗中耐受性良好。需要研究单独检查帕唑帕尼或与化学疗法或其他靶向疗法联用的效果。