Background/Objectives:Desmoid-type fibromatosis (DTF) is a locally invasive tumor composed of myofibroblast-like cells and collagen; it does not metastasize but can cause significant local morbidity. Most sporadic cases are associated with mutations in the CTNNB1 gene, which encodes beta-catenin. Various treatments have been used with differing efficacy and toxicity profiles. At our institution, pegylated liposomal doxorubicin (PLD) has become the preferred treatment for patients with DTF. We aim to describe our experience using PLD in patients with DTF who require treatment.Methods:A retrospective review of 61 DTF patients (41 females, 20 males) treated between 2000 and 2023 was conducted to assess the efficacy and toxicity of PLD.Results:Of the 26 patients treated with PLD, 23 had follow-up clinical data to assess benefit. Twenty-one showed clinical benefit, and only one progressed. Two patients did not benefit from PLD due to infusion reactions and chose alternative therapies. The primary side effect of PLD was hand-foot syndrome (HFS), but dose reduction and extended intervals allowed most patients to tolerate treatment. Other treatments, such as methotrexate, vinblastine/vinorelbine, and sorafenib, also showed activity but had significant toxicities, including severe HFS, malaise, and hypertension. Interestingly, 31 out of 61 patients had a pre-existing history of psychiatric conditions (primarily depression and anxiety), and 6 of 41 women had personal or family history of polycystic ovary syndrome (PCOS). Additionally, 15 patients had obesity, and 4 had hypothyroidism.Conclusions:PLD is an effective and well-tolerated treatment for DTF, with good clinical responses at lower, tolerable doses. The association of pre-existing psychiatric diagnoses, PCOS, and obesity warrants further investigation.
背景/目的：硬纤维瘤型纤维瘤病是一种由肌成纤维细胞样细胞和胶原构成的局部侵袭性肿瘤，虽不发生转移但可导致显著的局部病变。大多数散发病例与编码β-连环蛋白的CTNNB1基因突变相关。现有多种治疗方案在疗效和毒性特征上存在差异。在本机构，聚乙二醇化脂质体阿霉素已成为硬纤维瘤型纤维瘤病患者的首选治疗方案。本研究旨在总结PLD在需要治疗的DTF患者中的应用经验。 方法：回顾性分析2000年至2023年间收治的61例DTF患者（女性41例，男性20例），评估PLD的疗效与毒性特征。 结果：在26例接受PLD治疗的患者中，23例具有可评估疗效的随访临床数据。其中21例显示临床获益，仅1例出现疾病进展。另有2例患者因输注反应未能获益而选择替代疗法。PLD的主要副作用为手足综合征，但通过剂量调整和延长给药间隔，多数患者能够耐受治疗。其他治疗方案如甲氨蝶呤、长春碱/长春瑞滨及索拉非尼虽也显示疗效，但存在显著毒性反应，包括重度手足综合征、全身乏力及高血压。值得注意的是，61例患者中有31例存在精神疾病史（主要为抑郁和焦虑），41例女性患者中有6例具有多囊卵巢综合征个人或家族史。此外，15例患者伴有肥胖症，4例患有甲状腺功能减退。 结论：PLD是治疗DTF有效且耐受性良好的方案，在较低耐受剂量下即可获得良好临床应答。既存精神疾病诊断、多囊卵巢综合征与肥胖的关联性值得进一步研究。
肿瘤(癌症)患者之家