Abstract：
BACKGROUND: Sorafenib and pazopanib, two tyrosine kinase inhibitors (TKI), are widely used in patients with progressive symptomatic desmoid tumors (DT). Limited real-word data is available on long-term outcomes of patients who progressed on, stopped, or continued TKIs.
METHODS: Patients diagnosed with DTs and treated with sorafenib or pazopanib between 2011 and 2022 at 11 institutions were reviewed. Patient history, response to therapy and toxicity were recorded. Statistical analyses utilized Kaplan-Meier and log-rank tests.
RESULTS: 142 patients with DT treated with sorafenib (n = 126, 88.7 %) or pazopanib (n = 16, 11.3 %) were analyzed. The median treatment duration was 10.8 months (range: 0.07- 73.9). The overall response rate and the disease control rate were 26.0 % and 95.1 %, respectively. The median tumor shrinkage was - 8.5 % (range -100.0 %- +72.5 %). Among responders, the median time to an objective response was 15.2 months (range: 1.1 to 33.1). The 1-year and 2-year progression-free survival rates were 82 % and 80 %. Dose reductions were necessary in 34 (23.9 %) patients. Grade 3 or higher adverse events were reported in 36 (25.4 %) patients. On the last follow-up, 55 (38.7 %) patients continued treatment. Treatment discontinuation (n = 85, 59.9 %) was mainly for toxicity (n = 35, 45.9 %) or radiological or clinical progression (n = 30, 35.3 %). For the entire cohort, 36 (25.4 %) patients required subsequent treatment. In the 32 responders, only 1 (3.1 %) patient required a subsequent treatment. In patients who discontinued TKI, 25 (44.6 %) with stable disease received subsequent treatment compared to 0 (0.0 %) of responders.
CONCLUSIONS: This retrospective study represents the largest cohort of DT patients treated with sorafenib or pazopanib to date. Discontinuation of treatment in responders is safe. The optimal treatment duration in patients with stable disease remains to be defined.
METHODS: Patients diagnosed with DTs and treated with sorafenib or pazopanib between 2011 and 2022 at 11 institutions were reviewed. Patient history, response to therapy and toxicity were recorded. Statistical analyses utilized Kaplan-Meier and log-rank tests.
RESULTS: 142 patients with DT treated with sorafenib (n = 126, 88.7 %) or pazopanib (n = 16, 11.3 %) were analyzed. The median treatment duration was 10.8 months (range: 0.07- 73.9). The overall response rate and the disease control rate were 26.0 % and 95.1 %, respectively. The median tumor shrinkage was - 8.5 % (range -100.0 %- +72.5 %). Among responders, the median time to an objective response was 15.2 months (range: 1.1 to 33.1). The 1-year and 2-year progression-free survival rates were 82 % and 80 %. Dose reductions were necessary in 34 (23.9 %) patients. Grade 3 or higher adverse events were reported in 36 (25.4 %) patients. On the last follow-up, 55 (38.7 %) patients continued treatment. Treatment discontinuation (n = 85, 59.9 %) was mainly for toxicity (n = 35, 45.9 %) or radiological or clinical progression (n = 30, 35.3 %). For the entire cohort, 36 (25.4 %) patients required subsequent treatment. In the 32 responders, only 1 (3.1 %) patient required a subsequent treatment. In patients who discontinued TKI, 25 (44.6 %) with stable disease received subsequent treatment compared to 0 (0.0 %) of responders.
CONCLUSIONS: This retrospective study represents the largest cohort of DT patients treated with sorafenib or pazopanib to date. Discontinuation of treatment in responders is safe. The optimal treatment duration in patients with stable disease remains to be defined.
摘要：
背景：索拉非尼和帕唑帕尼，两种酪氨酸激酶抑制剂（TKI），广泛用于进行性症状性硬纤维瘤（DT）患者。关于进展的患者的长期结果，可以获得有限的真实数据，停止,或继续TKIs。
方法：回顾了2011年至2022年在11个机构中诊断为DTs并接受索拉非尼或帕唑帕尼治疗的患者。患者病史,记录对治疗的反应和毒性.统计分析使用Kaplan-Meier和对数秩检验。
结果：分析了142例接受索拉非尼（n=126，88.7％）或帕唑帕尼（n=16，11.3％）治疗的DT患者。中位治疗时间为10.8个月（范围：0.07-73.9）。总有效率和疾病控制率分别为26.0%和95.1%,分别。中位肿瘤收缩率为-8.5％（范围-100.0％-72.5％）。在响应者中，达到客观缓解的中位时间为15.2个月(范围:1.1~33.1).1年和2年无进展生存率分别为82%和80%。34例（23.9%）患者需要减少剂量。36例（25.4%）患者报告了3级或更高的不良事件。在最后的后续行动中，55例（38.7%）患者继续治疗。停止治疗（n=85，59.9％）主要是因为毒性（n=35，45.9％）或放射学或临床进展（n=30，35.3％）。对于整个队列，36例（25.4%）患者需要后续治疗。在32名响应者中，只有1例(3.1%)患者需要后续治疗.在停用TKI的患者中，与0（0.0％）的应答者相比，25例（44.6％）的疾病稳定者接受了后续治疗。
结论：这项回顾性研究代表了迄今为止接受索拉非尼或帕唑帕尼治疗的最大的DT患者队列。在应答者中停止治疗是安全的。病情稳定患者的最佳治疗持续时间尚待确定。
方法：回顾了2011年至2022年在11个机构中诊断为DTs并接受索拉非尼或帕唑帕尼治疗的患者。患者病史,记录对治疗的反应和毒性.统计分析使用Kaplan-Meier和对数秩检验。
结果：分析了142例接受索拉非尼（n=126，88.7％）或帕唑帕尼（n=16，11.3％）治疗的DT患者。中位治疗时间为10.8个月（范围：0.07-73.9）。总有效率和疾病控制率分别为26.0%和95.1%,分别。中位肿瘤收缩率为-8.5％（范围-100.0％-72.5％）。在响应者中，达到客观缓解的中位时间为15.2个月(范围:1.1~33.1).1年和2年无进展生存率分别为82%和80%。34例（23.9%）患者需要减少剂量。36例（25.4%）患者报告了3级或更高的不良事件。在最后的后续行动中，55例（38.7%）患者继续治疗。停止治疗（n=85，59.9％）主要是因为毒性（n=35，45.9％）或放射学或临床进展（n=30，35.3％）。对于整个队列，36例（25.4%）患者需要后续治疗。在32名响应者中，只有1例(3.1%)患者需要后续治疗.在停用TKI的患者中，与0（0.0％）的应答者相比，25例（44.6％）的疾病稳定者接受了后续治疗。
结论：这项回顾性研究代表了迄今为止接受索拉非尼或帕唑帕尼治疗的最大的DT患者队列。在应答者中停止治疗是安全的。病情稳定患者的最佳治疗持续时间尚待确定。
