不可手术切除韧带样型纤维瘤伊马替尼治疗疗效分析

Efficacy of Imatinib for treatment of inoperable desmoid-type fibromatosis patients

目的韧带样型纤维瘤是一种极易复发的交界性肿瘤,对于不可手术切除的韧带样型纤维瘤患者可以考虑药物治疗。本研究探讨伊马替尼靶向治疗不可手术切除韧带样型纤维瘤患者的临床效果。方法选取2016-05-01-2017-09-30北京大学肿瘤医院骨与软组织肿瘤科收治的48例患者作为研究对象,均为不可手术切除的韧带样型纤维瘤患者。纳入的48例患者中,男21例,女27例;发病年龄1~57岁,平均年龄19.6岁;发病部位以下肢和臀部为最多(25例);肿瘤最大直径1.6~34cm,平均10.0cm。所有患者均为伊马替尼口服治疗,至少持续3个月。伊马替尼服用至少3个月后进行第一次随访复查,所有患者均获得随访,随访时间3~18个月,中位随访时间3个月。结果治疗3个月后疗效,PR1例,SD39例,PD8例,肿瘤控制率为83.3%。3个月无进展生存率(progress-free survival,PFS)为83.3%,6个月PFS为63.5%,9个月PFS为55.5%,12个月PFS为48.9%。治疗期间主要毒副作用为白细胞降低、腹泻、皮疹、水肿、转氨酶升高、脱发和肿瘤部位疼痛等,均为轻度,对症治疗后均不影响用药。结论对于不可手术切除的韧带样型纤维瘤患者,口服伊马替尼是一种可以改善局部控制安全有效的药物治疗方法。

OBJECTIVE Desmoid-type fibromatosis（DF） is a kind of borderline tumor which is easy to recrudesce. For inoperable DF, systematic treatment could be considered. This article is to assess the efficacy of Imatinib for inoperable DF. METHODS From May 2016 to September 2017,48 patients of inoperable desmoid type fibromatosis were treated in our department. The treatment plan was Imatinib administered oral daily for at least 3 months. Clinical data of those pa tients were analyzed retrospectively. Among all the patients, there were 21 males, 27 females. The age of onset was from 1 to 57 years,and the mean age was 19.6 years. The most frequent sites were lower limb and buttock（n=25 cases）. The tumor size（the maximum diameter） was from 1. 6 to 34 cm, the mean size was 10 cm. The follow up was from 3 to 18 months. The median follow up time was 3 months. Imatinib were taken for at least 3 months at the moment of first follow-up. RESULTS At the first follow up,1 cases with partial response（PR） ,39 cases with stable disease（SD） and 8 cases with progressive disease（PD）. The 3-month local control rate was 83.3%. 3-month, 6-month, 9-month and 12-month PFS was 83.3%, 63.5%, 55.5% and 48.9%, respectively. The main adverse effects during the treatment were leukocyte reduction,diarrhea, skin rashes, edema, increased aminotransferase, hair loss, and pain in the tumor site. All the adverse effects were mild,and did not affect the medication after symptomatic treatment. （X）NCLUSION For recurrent,inoperable and progressive desmoid-type fibromatosis,imatinib is a safe and optional choice for local control.