小剂量甲氨蝶呤联合长春瑞滨治疗不可手术切除的硬纤维瘤的疗效及预后因素分析

Low-dose methotrexate combined with vinorelbine for inoperable desmoid tumor:efficacy and the prognostic factors

目的研究硬纤维瘤的临床特点,探讨对于不可手术切除的硬纤维瘤患者进行系统性治疗的有效性并分析其预后因素。方法纳入2009年8月~2013年12月间北京大学肿瘤医院骨与软组织肿瘤科连续收治的52例不可手术切除的硬纤维瘤患者,治疗方法为长春瑞滨联合小剂量甲氨蝶呤化疗。结果确诊的52例患者中,男性22例,女性30例。发病年龄2~46岁,平均发病年龄18.7岁。发病部位以下肢最多,占36.5%（19/52）。肿瘤最大径2.7~37 cm,平均9.5 cm。随访时间7~64月,中位随访时间29月。化疗持续时间4~30月,中位化疗时间12月。化疗结束时评效：1例CR,18例PR,27例SD,6例PD。总有效率（CR＋PR＋SD）为88.5%。PFS时间为4~63月,中位PFS时间26.5月。2年PFS为76.7%,5年PFS为41.9%。化疗持续时间大于12月者预后较好,不同性别、发病年龄、化疗时年龄、肿瘤部位、肿瘤大小组间PFS无统计学差异。结论足够疗程的长春瑞滨联合小剂量甲氨蝶呤的化疗,在肿瘤反复复发、不可切除且肿瘤持续进展的情况下,是一种安全满意的治疗选择。

Objective To characterize the clinical features of desmoid tumor, assess the efficacy of conservative chemotherapy for inoperable desmoid tumor and analyze the prognostic factors. Methods From August 2009 to December 2013, 52 patients with inoperable desmoid tumor were treated in our department and received chemotherapy with vinorelbine combined with low-dose methotrexate. The clinical data of the patients were analyzed retrospectively. Results The patients studied included22 male and 30 female patients with the age of disease onset ranging from 2 to 46 years（mean 18.7 years）. The lesions occurred most frequently in the lower limbs（36.5%, 19/52） and the tumor size ranged from 2.7 to 37 cm（mean 9.5 cm）. The patients were followed up for a median of 29 months（7 to 64 months）. The chemotherapy lasted for 4 to 30 months（median 12months）. After completion of the chemotherapy, 1 patient had a complete response（CR）, 18 showed partial responses（PR）, 27 cases had stable disease（SD）, and 6 had progressive disease（PD）, with an overall response rate（ORR） of 88.5%. The progression-free survival（PFS） time of the patients ranged from 4 to 63 months（median 26.5 months） with a 2-year PFS rate of76.7% and 5-year PFS rate of 41.9%. A longer chemotherapy duration（over 12 months） was associated with a more favorable prognosis. No significant differences in PFS were found between the patients stratified by gender, age of disease onset, age when receiving chemotherapy, tumor site, or tumor size. Conclusion For recurrent, inoperable and progressive desmoid tumor, long enough cycles of vinorelbine combined with low-dose methotrexate can be an effective and safe option for tumor control.