Abstract：
OBJECTIVE: Desmoid fibromatosis (DF) is a locally aggressive tumor with low mortality but significant morbidity. There is a lack of standard of care, and existing therapies are associated with significant barriers including access, cost, and toxicities. This study aimed to explore the efficacy and safety of the metronomic therapy (MT) in DF in a large, homogenous cohort from India.
METHODS: This study involved histologically confirmed DF cases treated with MT comprising vinblastine (6 mg) and methotrexate (15 mg) both once a week, and tamoxifen (40 mg/m2) in two divided doses once daily between 2002 and 2018.
RESULTS: There were 315 patients with a median age of 27 years; the commonest site was extremity (142 of 315; 45.0%). There were 159 (50.1%) male patients. Of the 123 (39.0%) prior treated patients, 119 had surgery. Of 315 patients, 263 (83.5%) received treatment at our institute (MT-151, 77-local treatment, 9-tyrosine kinase inhibitor, and 26 were observed). Among the MT cohort (n = 163, 61.2%), at a median follow-up of 36 (0.5-186) months, the 3-year progression-free and overall survival were 81.1% (95% CI, 74.3 to 88.4) and 99.2% (95% CI, 97.6 to 100), respectively. There were 35% partial responses. Ninety-two patients (56.4%) completed 1-year therapy, which was an independent prognosticator (P < .0001; hazard ratio, 0.177 [95% CI, 0.083 to 0.377]). MT was well tolerated. Predominant grade ≥3 toxicities were febrile neutropenia, 12 (7.4%) without any chemotoxicity-related death. The annual cost of MT was $130 US dollars.
CONCLUSIONS: The novel, low-cost MT qualifies as one of the effective, less toxic, sustainable, standard-of-care options for the treatment of DF with global reach and merits wide recognition.
METHODS: This study involved histologically confirmed DF cases treated with MT comprising vinblastine (6 mg) and methotrexate (15 mg) both once a week, and tamoxifen (40 mg/m2) in two divided doses once daily between 2002 and 2018.
RESULTS: There were 315 patients with a median age of 27 years; the commonest site was extremity (142 of 315; 45.0%). There were 159 (50.1%) male patients. Of the 123 (39.0%) prior treated patients, 119 had surgery. Of 315 patients, 263 (83.5%) received treatment at our institute (MT-151, 77-local treatment, 9-tyrosine kinase inhibitor, and 26 were observed). Among the MT cohort (n = 163, 61.2%), at a median follow-up of 36 (0.5-186) months, the 3-year progression-free and overall survival were 81.1% (95% CI, 74.3 to 88.4) and 99.2% (95% CI, 97.6 to 100), respectively. There were 35% partial responses. Ninety-two patients (56.4%) completed 1-year therapy, which was an independent prognosticator (P < .0001; hazard ratio, 0.177 [95% CI, 0.083 to 0.377]). MT was well tolerated. Predominant grade ≥3 toxicities were febrile neutropenia, 12 (7.4%) without any chemotoxicity-related death. The annual cost of MT was $130 US dollars.
CONCLUSIONS: The novel, low-cost MT qualifies as one of the effective, less toxic, sustainable, standard-of-care options for the treatment of DF with global reach and merits wide recognition.
摘要：
目的：纤维瘤病（DF）是一种局部侵袭性肿瘤，死亡率低，但发病率高。缺乏护理标准，和现有的治疗与重大障碍相关，包括获取，成本,和毒性。本研究旨在探讨节拍疗法(MT)在DF中的疗效和安全性,来自印度的同质队列。
方法：本研究包括经组织学证实的DF病例，接受含有长春碱（6mg）和甲氨蝶呤（15mg）的MT治疗，每周一次。和他莫昔芬（40mg/m2），在2002年至2018年之间每天一次分两次剂量。
结果：有315例患者，中位年龄为27岁；最常见的部位是四肢（315例中的142例；45.0%）。男性患者159例（50.1%）。在123名(39.0%)先前接受过治疗的患者中，119做手术。315名患者中,263（83.5%）在我们研究所接受治疗（MT-151，77-局部治疗，9-酪氨酸激酶抑制剂，和26个被观察到)。在MT队列中(n=163，61.2%)，在中位随访36(0.5-186)个月时，3年无进展生存率和总生存率分别为81.1%(95%CI,74.3~88.4)和99.2%(95%CI,97.6~100),分别。有35%的部分反应。92例患者（56.4%）完成1年治疗，这是一个独立的预测因素(P<0.0001；危险比，0.177[95%CI，0.083至0.377]）。MT耐受性良好。主要≥3级毒性为发热性中性粒细胞减少症，12例（7.4%）无任何化学毒性相关死亡。MT的年度成本为130美元。
结论：小说，低成本MT有资格成为有效的，毒性较小,可持续,治疗DF的标准护理方案具有全球影响力，值得广泛认可。
方法：本研究包括经组织学证实的DF病例，接受含有长春碱（6mg）和甲氨蝶呤（15mg）的MT治疗，每周一次。和他莫昔芬（40mg/m2），在2002年至2018年之间每天一次分两次剂量。
结果：有315例患者，中位年龄为27岁；最常见的部位是四肢（315例中的142例；45.0%）。男性患者159例（50.1%）。在123名(39.0%)先前接受过治疗的患者中，119做手术。315名患者中,263（83.5%）在我们研究所接受治疗（MT-151，77-局部治疗，9-酪氨酸激酶抑制剂，和26个被观察到)。在MT队列中(n=163，61.2%)，在中位随访36(0.5-186)个月时，3年无进展生存率和总生存率分别为81.1%(95%CI,74.3~88.4)和99.2%(95%CI,97.6~100),分别。有35%的部分反应。92例患者（56.4%）完成1年治疗，这是一个独立的预测因素(P<0.0001；危险比，0.177[95%CI，0.083至0.377]）。MT耐受性良好。主要≥3级毒性为发热性中性粒细胞减少症，12例（7.4%）无任何化学毒性相关死亡。MT的年度成本为130美元。
结论：小说，低成本MT有资格成为有效的，毒性较小,可持续,治疗DF的标准护理方案具有全球影响力，值得广泛认可。
