RTI Health Solutions, Research Triangle Park, NC, USA.
Medical Affairs, SpringWorks Therapeutics, Inc., Stamford, CT, USA.
Expert Rev Pharmacoecon Outcomes Res. 2023 Jul-Dec;23(6):607-618. doi: 10.1080/14737167.2023.2203915. Epub 2023 Apr 26.
Desmoid tumors (DT) are soft-tissue tumors that infiltrate into surrounding structures with ill-defined margins. Although surgery is a potential treatment option, complete excision with negative margins is not often possible, the postsurgery recurrence rate is high, and surgery can result in disfigurement and/or loss of function.
We conducted a literature review to assess the burden of surgery in patients with DT, focusing on recurrence rates and functional deficits resulting from surgeries. Since economic data related to DT surgery is lacking, reviews of surgery costs in soft-tissue sarcomas and of general costs of amputations were conducted. Risk factors for DT recurrence after surgery are young age (<30 years), tumor location (extremities), tumor size (>5 cm in greatest diameter), positive resection margins, and history of trauma in the area of the primary tumor. Tumors in the extremities have the highest risk of recurrence (30%-90%). Lower rates of recurrences have been reported when radiotherapy was used after surgery (14%-38%).
Although effective in specific cases, surgery may be associated with poor long-term functional outcomes and higher economic costs. Therefore, it is imperative to find alternative treatments with acceptable efficacy and safety profiles that do not adversely affect functional aspects in patients.
硬纤维瘤（desmoid tumors，DT）是一种软组织肿瘤，其边界模糊，向周围结构浸润。尽管手术是一种潜在的治疗选择，但通常无法实现完全切除且切缘阴性，术后复发率高，且手术可能导致毁容和/或功能丧失。
我们进行了文献回顾，以评估手术在 DT 患者中的负担，重点关注手术相关的复发率和功能缺陷。由于缺乏与 DT 手术相关的经济数据，我们还对软组织肉瘤的手术成本和截肢的一般成本进行了回顾。手术后 DT 复发的风险因素包括年龄较小（<30 岁）、肿瘤位置（四肢）、肿瘤大小（最大直径>5cm）、切缘阳性和原发性肿瘤部位的外伤史。四肢的肿瘤复发风险最高（30%-90%）。术后放疗可降低复发率（14%-38%）。
尽管在某些特定情况下有效，但手术可能与较差的长期功能结果和更高的经济成本相关。因此，迫切需要寻找具有可接受的疗效和安全性特征且不会对患者功能方面产生不利影响的替代治疗方法。
