重磅：复发难治性硬纤维瘤（侵袭性纤维瘤病）有了新疗法！

复发难治性硬纤维瘤（侵袭性纤维瘤病）的最新疗法

硬纤维瘤（侵袭性纤维瘤病）是一种临床少见的纤维性肿瘤，其中许多具有共同的分子改变，到目前为止，我们还没有非常有效的治疗方案。可以发生于FAP/Gardner综合征的患者，也可独立原发。最初的药物治疗主要包括非甾体抗炎药和三苯氧胺，但发表在新英格兰杂志的最新数据（索拉非尼2年无进展生存率与安慰剂相比为81%：36%）的出现令其成为新的标准治疗。其无进展生存曲线令人印象非常深刻。——Axel Grothey MD

TAKE-HOME MESSAGE

• In this double-blind, phase III trial, 87 patients with progressive, symptomatic, or recurrent desmoid tumors were randomly assigned to receive either sorafenib or matching placebo. Crossover to the sorafenib group was permitted for patients in the placebo group whose disease had progressed.

• Among patients with progressive, refractory, or symptomatic desmoid tumors, sorafenib significantly prolonged progression-free survival (2-year PFS 81% vs 36%) and induced durable responses.

– Jeffrey M. Wiisanen, MD

BACKGROUND

Desmoid tumors (also referred to as aggressive fibromatosis) are connective tissue neoplasms that can arise in any anatomical location and infiltrate the mesentery, neurovascular structures, and visceral organs. There is no standard of care.

METHODS

In this double-blind, phase 3 trial, we randomly assigned 87 patients with progressive, symptomatic, or recurrent desmoid tumors to receive either sorafenib (400-mg tablet once daily) or matching placebo. Crossover to the sorafenib group was permitted for patients in the placebo group who had disease progression. The primary end point was investigator-assessed progression-free survival; rates of objective response and adverse events were also evaluated.

RESULTS

With a median follow-up of 27.2 months, the 2-year progression-free survival rate was 81% (95% confidence interval [CI], 69 to 96) in the sorafenib group and 36% (95% CI, 22 to 57) in the placebo group (hazard ratio for progression or death, 0.13; 95% CI, 0.05 to 0.31; P<0.001). Before crossover, the objective response rate was 33% (95% CI, 20 to 48) in the sorafenib group and 20% (95% CI, 8 to 38) in the placebo group. The median time to an objective response among patients who had a response was 9.6 months (interquartile range, 6.6 to 16.7) in the sorafenib group and 13.3 months (interquartile range, 11.2 to 31.1) in the placebo group. The objective responses are ongoing. Among patients who received sorafenib, the most frequently reported adverse events were grade 1 or 2 events of rash (73%), fatigue (67%), hypertension (55%), and diarrhea (51%).

CONCLUSIONS

Among patients with progressive, refractory, or symptomatic desmoid tumors, sorafenib significantly prolonged progression-free survival and induced durable responses.