Department of Medicine, Memorial Sloan-Kettering Cancer Center, New York, New York 10065-6007, USA.
Cancer. 2010 May 1;116(9):2258-65. doi: 10.1002/cncr.25089.
In the current study, the authors examined the outcomes of patients with desmoid tumors who received systemic therapy at a single institution to provide a basis for the examination of newer agents.
Records of patients with desmoid tumors who were treated with chemotherapy at the study institution were reviewed. The activity of nonsteroidal anti-inflammatory drugs was not addressed. Patients without measurable disease and those receiving therapy could not be documented, and those receiving prophylactic therapy were excluded.
A total of 68 patients received 157 lines of therapy. At the time of last follow-up, 9 patients had died, 7 of progressive disease. The cohort was 62% female, with a median age of 32.5 years. Approximately 32% of the patients had Gardner syndrome. The median follow-up was 63 months, and patients received a median of 2 lines of therapy. An intra-abdominal primary tumor location was the most common (44%). The greatest Response Evaluation Criteria in Solid Tumors (RECIST) response rate was observed with anthracyclines and hormonal therapy and the lowest response was noted with single-agent dacarbazine/temozolomide or tyrosine kinase inhibitors, principally imatinib. On multivariate analysis, macroscopic nodular morphology and the presence of Gardner syndrome were the only tumor factors found to be associated with a greater time to disease progression.
Compared with other agents, antiestrogens and anthracycline-containing regimens appear to be associated with a higher radiological response rate against desmoid tumors. Systemic therapy can be successful in patients with desmoid tumors, and is a viable option in lieu of morbid or disabling surgery.
在本研究中，作者检查了在单一机构接受系统治疗的患者的硬纤维瘤的治疗结果，为检查新药物提供了依据。
回顾了在研究机构接受化疗治疗的硬纤维瘤患者的病历。未涉及非甾体类抗炎药的活性。无法记录无可测量疾病和正在接受治疗的患者，也排除了接受预防性治疗的患者。
共有 68 名患者接受了 157 条治疗线。在最后一次随访时，有 9 名患者死亡，其中 7 名死于疾病进展。该队列中女性占 62%，中位年龄为 32.5 岁。约 32%的患者有 Gardner 综合征。中位随访时间为 63 个月，患者接受了中位数为 2 线的治疗。最常见的是腹腔内原发性肿瘤部位（44%）。实体瘤反应评估标准（RECIST）的最大反应率观察到与蒽环类药物和激素治疗有关，而单药达卡巴嗪/替莫唑胺或酪氨酸激酶抑制剂（主要是伊马替尼）的反应率最低。多因素分析表明，宏观结节状形态和 Gardner 综合征的存在是与疾病进展时间延长相关的唯一肿瘤因素。
与其他药物相比，抗雌激素和含蒽环类药物的方案似乎与硬纤维瘤的放射学反应率更高相关。全身治疗可成功治疗硬纤维瘤患者，并且是一种可行的替代选择，可替代病态或致残性手术。
