Importance Desmoid tumor (DT) is a rare and locally aggressive monoclonal, fibroblastic proliferation characterized by a variable and often unpredictable clinical course. Previously, surgery was the standard primary treatment modality; however, within the past decade, a paradigm shift toward less-invasive management has been introduced and an effort to harmonize the strategy among clinicians has been made. To update the 2020 global evidence-based consensus guideline on the management of patients with DT, the Desmoid Tumor Working Group convened a 1-day consensus meeting in Milan, Italy, on June 30, 2023, under the auspices of the European Reference Network on Rare Adult Solid Cancers and Sarcoma Patient Advocacy Global Network, the Desmoid Foundation Italy, and the Desmoid Tumor Research Foundation. The meeting brought together over 90 adult and pediatric sarcoma experts from different disciplines as well as patients and patient advocates from around the world. Observations The 2023 update of the global evidence-based consensus guideline focused on the positioning of local therapies alongside surgery and radiotherapy in the treatment algorithm as well as the positioning of the newest class of medical agents, such as γ-secretase inhibitors. Literature searches of MEDLINE and Embase databases were performed for English-language randomized clinical trials (RCTs) of systemic therapies to obtain data to support the consensus recommendations. Of the 18 full-text articles retrieved, only 4 articles met the inclusion criteria. The 2023 consensus guideline is informed by a number of new aspects, including data for local ablative therapies such as cryotherapy; other indications for surgery; and the γ-secretase inhibitor nirogacestat, the first representative of the newest class of medical agents and first approved drug for DT. Management of DT is complex and should be carried out exclusively in designated DT referral centers equipped with a multidisciplinary tumor board. Selection of the appropriate strategy should consider DT-related symptoms, associated risks, tumor location, disease morbidities, available treatment options, and preferences of individual patients. Conclusions and Relevance The therapeutic armamentarium of DT therapy is continually expanding. It is imperative to carefully select the management strategy for each patient with DT to optimize tumor control and enhance quality of life.

中文翻译：

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硬纤维瘤的当前管理：综述。


重要性硬纤维瘤 （DT） 是一种罕见且局部侵袭性的单克隆成纤维细胞增生，其特征是临床病程多变且通常不可预测。以前，手术是标准的主要治疗方式;然而，在过去十年中，已经引入了向微创管理的范式转变，并努力在临床医生之间协调该策略。为了更新 2020 年 DT 患者管理的全球循证共识指南，硬纤维瘤工作组在欧洲罕见成人实体癌参考网络和肉瘤患者倡导全球网络、意大利硬纤维瘤基金会的主持下，于 2023 年 6 月 30 日在意大利米兰召开了为期 1 天的共识会议。 和硬纤维瘤研究基金会。会议汇集了来自不同学科的 90 多名成人和儿童肉瘤专家，以及来自世界各地的患者和患者权益倡导者。观察结果全球循证共识指南的 2023 年更新侧重于局部治疗与手术和放疗在治疗流程中的定位，以及最新一类药物治疗的定位，例如 γ-分泌酶抑制剂。对 MEDLINE 和 Embase 数据库进行文献检索，以查找全身治疗的英语随机临床试验 （RCT），以获得支持共识建议的数据。在检索到的 18 篇全文文章中，只有 4 篇文章符合纳入标准。 2023 年共识指南以许多新方面为依据，包括局部消融疗法（如冷冻疗法）的数据;其他手术适应症;以及 γ-分泌酶抑制剂 nirogacestat，这是最新一类药物的第一个代表，也是第一个获批的 DT 药物。DT 的管理很复杂，应仅在配备多学科肿瘤委员会的指定 DT 转诊中心进行。选择合适的策略应考虑 DT 相关症状、相关风险、肿瘤位置、疾病发病率、可用的治疗选择和个体患者的偏好。结论和相关性 DT 疗法的治疗武器库不断扩大。必须为每位 DT 患者仔细选择管理策略，以优化肿瘤控制并提高生活质量。