Freire Andrea P Espejo, Skubitz Keith M
Department of Medicine, The Masonic Cancer Center, The University of Minnesota Medical School, University of Minnesota, Minneapolis, MN 55455, USA.
Cancers (Basel). 2025 Jan 17;17(2):293. doi: 10.3390/cancers17020293.
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. 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. 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. 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.
硬纤维瘤型纤维瘤病(DTF)是一种由肌成纤维细胞样细胞和胶原蛋白组成的局部侵袭性肿瘤;它不会发生转移,但可导致严重的局部病变。大多数散发性病例与编码β-连环蛋白的CTNNB1基因突变有关。已采用多种治疗方法,其疗效和毒性各不相同。在我们机构,聚乙二醇化脂质体阿霉素(PLD)已成为DTF患者的首选治疗方法。我们旨在描述我们在需要治疗的DTF患者中使用PLD的经验。对2000年至2023年间治疗的61例DTF患者(41例女性,20例男性)进行回顾性分析,以评估PLD的疗效和毒性。在接受PLD治疗的26例患者中,23例有随访临床数据以评估获益情况。21例显示临床获益,仅1例病情进展。2例患者因输注反应未从PLD中获益,选择了替代疗法。PLD的主要副作用是手足综合征(HFS),但通过降低剂量和延长给药间隔,大多数患者能够耐受治疗。其他治疗方法,如甲氨蝶呤、长春碱/长春瑞滨和索拉非尼,也显示出活性,但有显著毒性,包括严重的HFS、不适和高血压。有趣的是,61例患者中有31例既往有精神疾病史(主要是抑郁症和焦虑症),41例女性中有6例有个人或家族多囊卵巢综合征(PCOS)病史。此外,15例患者肥胖,4例患者甲状腺功能减退。PLD是一种治疗DTF有效且耐受性良好的方法,在较低的可耐受剂量下有良好的临床反应。既往存在的精神疾病诊断、PCOS和肥胖之间的关联值得进一步研究。