Department of Surgery, Sarcoma Service, Fondazione IRCCS Istituto Nazionale dei Tumori, Milano, Italy.
Division of Medical Oncology, Candiolo Cancer Institute, FPO-IRCCS, Candiolo (TO), Italy.
Clin Cancer Res. 2022 Sep 15;28(18):4027-4032. doi: 10.1158/1078-0432.CCR-21-4205.
To prospectively assess the behavior of primary sporadic (not familial adenomatous polyposis-associated) desmoid fibromatosis (DF) managed by active surveillance (AS).
This is an Italian prospective, multicenter, observational study (NCT02547831) including patients ≥16 years with primary sporadic DF at any site. Patients were assessed by Response Evaluation Criteria in Solid Tumor (RECIST) version 1.1. Primary endpoint was progression-free survival (PFS) at 3 years. Treatment-free survival (TFS) was also analyzed. PFS and TFS were calculated by Kaplan-Meier plots and compared by log-rank test. Cox proportional hazard multivariable regression analyses were performed.
From 2013 to 2018, 108 consecutive patients were included (82% female); median age was 39 years; median size was 51 mm. CTNNB1 mutations were T41A (50%), S45F (12%), other (19%), wild-type (19%). At 32.3-month median follow-up, 42 of 108 (39%) showed RECIST progression. Spontaneous regression was initially observed in 27 of 108 (25%), while it followed dimensional progression in another 33 of 108 (31%). PFS at 36 months was 54.5% [95% confidence interval (CI), 44.9%-66.1%]. Thirty-five of 108 (32%) patients received active treatment, 18 of 108 (17%) after RECIST progression and 17 of 108 (15%) after symptomatic progression. TFS at 36 months was 65.9% (95% CI, 57.3%-75.9%). Larger tumor size and extremity location were associated to shorter TFS and a trend for S45F mutation was also observed (P = 0.06), while none of the mentioned variables was significantly associated with PFS.
In primary DF, AS can be proposed, because disease stabilization and spontaneous regression frequently occur. However, extra care should be taken for patients with tumors of larger size, extremity location, and S45F mutation. See related commentary by Greene and Van Tine, p. 3911.
前瞻性评估主动监测（AS）管理的原发性散发（非家族性腺瘤性息肉病相关）硬纤维瘤病（DF）的行为。
这是一项意大利前瞻性、多中心、观察性研究（NCT02547831），纳入了任何部位患有原发性散发 DF 的≥16 岁患者。患者采用实体瘤反应评价标准（RECIST）1.1 进行评估。主要终点为 3 年无进展生存期（PFS）。还分析了无治疗生存期（TFS）。通过 Kaplan-Meier 图计算 PFS 和 TFS，并通过对数秩检验进行比较。进行了 Cox 比例风险多变量回归分析。
2013 年至 2018 年，共纳入 108 例连续患者（82%为女性）；中位年龄为 39 岁；中位肿瘤大小为 51mm。CTNNB1 突变类型为 T41A（50%）、S45F（12%）、其他（19%）、野生型（19%）。中位随访 32.3 个月时，108 例患者中有 42 例（39%）出现 RECIST 进展。108 例患者中有 27 例（25%）最初观察到自发消退，而另外 33 例（31%）则在出现肿瘤进展后发生自发消退。36 个月时 PFS 为 54.5%（95%CI，44.9%-66.1%）。108 例患者中有 35 例（32%）接受了积极治疗，其中 18 例（17%）在出现 RECIST 进展后接受治疗，17 例（15%）在出现症状性进展后接受治疗。36 个月时 TFS 为 65.9%（95%CI，57.3%-75.9%）。肿瘤较大、位于四肢与 S45F 突变与较短的 TFS 相关，S45F 突变也与较短的 TFS 相关（P=0.06），但上述变量均与 PFS 无显著相关性。
在原发性 DF 中，可采用 AS 方案，因为疾病稳定和自发消退经常发生。然而，对于肿瘤较大、位于四肢和 S45F 突变的患者，应更加谨慎。参见 Greene 和 Van Tine 的相关评论，第 3911 页。
