阿帕替尼治疗复发性和进展性韧带样纤维瘤的疗效与安全性分析

Efficacy and safety of apatinib for progressive and recurrent desmoid fibromatosis

目的:分析阿帕替尼治疗复发性和进展性韧带样纤维瘤(desmoid fibromatosis,DF)的疗效和安全性。方法:收集2017年10月至2020年9月就诊于四川大学华西医院的复发性或进展性韧带样纤维瘤患者37例,接受靶向药物阿帕替尼治疗,起始剂量250 mg/d,根据药物不良反应调整剂量。结果:无完全缓解病例,17例(45.9%)为部分缓解,18例(48.6%)为疾病稳定,2例(5.4%)为疾病进展,客观有效率为45.9%,疾病控制率为94.6%。患者的关节活动度(rang of motion,ROM)、视觉模拟评分(visual analog scale,VAS)、上肢功能评定表(disabilities of the arm,shoulder and hand,DASH)评分、髋关节Harris评分及美国特种外科医院(HSS)评分均较治疗前有明显改善,且差异具有统计学意义(P<0.05)。常见的不良反应为手足综合征(37.8%)、胃肠反应(29.7%)和月经紊乱(21.6%)。结论:靶向药物阿帕替尼对复发性和进展性DF有一定的疗效,且不良反应可耐受,可作为复发性和进展性韧带样纤维瘤治疗的新途径。

Objective:To investigate the efficacy and safety of apatinib in recurrent and progressive desmoid fibromatosis(DF).Methods:From October 2017 to September 2020,all patients who presented with recurrent or progressive DF at West China Hospital,Sichuan University were included in this study and initialy received a daily dose of 250 mg of apatinib.We adjusted the dose based on drug-related toxicity.Results:Seventeen patients showed partial response(45.9%),18 patients had stable disease(48.6%),and 2 patients had progressive disease(5.4%).The objective response rate was 45.9%and disease control rate was 94.6%.After apatinib treatment,the range of motion(ROM),visual analog scale(VAS)score,Disabilities of the Arm,Shoulder,and Hand(DASH)score,Harris score,and Hospital for Special Surgery(HSS)score of the patients significantly improved compared to those prior to treatment.The most common adverse events were hand-foot syndrome[14/37(37.8%)],gastrointestinal reaction[11/37(29.7%)],and paramenia[8/37(21.6%)].Conclusions:Apatinib was efficacious in the treatment of progressive or recurrent DF with mild adverse events.Thus,it can likely be considered as a novel option for the treatment of DF.