Treatment of Desmoid Fibromatosis With Arterial Embolization: Retrospective and Prospective Observational Study
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Reduction of pain (VAS score)
研究概览
简要总结
Desmoid fibromatoses are rare and locally aggressive mesenchymal tumors. The current scientific evidence regarding the efficacy and safety of the treatment of desmoid fibromatosis by arterial embolization is constituted by several retrospective and prospective studies. These studies report promising results through the use of chemoembolization, that is, arterial embolization using particles loaded with chemotherapy.
Instead, the type of treatment we propose would consist of injection of embolizing material without the use of chemotherapy, based on the positive results we have consistently reported over the years on arterial embolization of musculoskeletal tumors.
详细描述
Desmoid fibromatoses are rare (1-2 cases/million per year) and locally aggressive, characterized histologically by monoclonal myoblasts present in abundant stromal tissue.The current therapeutic strategy has abandoned primary resection, as recurrences after resection are common and often their phenotype is more infiltrative. Nonsurgical approaches remain suboptimal. For asymptomatic disease, current guidelines suggest an initial period of active surveillance. The current scientific evidence regarding the efficacy and safety of the treatment of desmoid fibromatosis by arterial embolization is constituted by several retrospective and prospective studies. These studies report promising results through the use of chemoembolization, that is, arterial embolization using particles loaded with chemotherapy.
Instead, the type of treatment we propose would consist of injection of embolizing material without the use of chemotherapy, based on the positive results we have consistently reported over the years on arterial embolization of musculoskeletal tumors.
Doxorubicin is routinely used in the treatment of soft tissue sarcomas and other mesenchymal malignancies. Its use against desmoid fibromatosis is effective but associated with hematologic, gastrointestinal, and cardiac toxicity. Consequently, this drug is reserved for symptomatic, nonresponsive, rapidly growing, or life-threatening fibromatoses.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients of both sexes aged ≥ 12 years
- •Fibromatosis demsoid symptomatic and in active phase (documented growth at last follow-ups)
- •Patients not eligible for surgery or cryoablation
- •Patients who have had embolization surgery for fibromatosis desmoide from 01/01/2023 to date and all new patients listed for this type of treatment.
- •Signature of informed consent to the study
排除标准
- •Patients with life expectancy <3 months or severely impaired status functional status (ASA 4)
- •Patients with fibromatosis not in active phase, documented clinically and by investigations imaging (MRI, CT)
- •Patients with coagulation deficiency or plateletopenic disease
- •Patients with documented active infection
- •Incompatibility to performing MRI examination.
结局指标
主要结局
Reduction of pain (VAS score)
时间窗: 1 year
Clinical assessment regarding pain by Visual Analogue Scale (VAS) score (0-100 mm), in which 0 represents no pain, and 100 represents maximum pain imaginable.
Reduction of antalgic therapy (mg)
时间窗: 1 year
Mean reduction in the use of pain medications evaluated in mg of active ingredient taken daily before the treatment and at follow-up.
Volume reduction in cm³
时间窗: 1 year
Reduction of the lesion evaluated with MRI, measuring the diameter of the lesion in cm³.
次要结局
- Improvement in quality of life (EORTC QLQ-C15-PAL questionnaire)(1 year)
- Improvement in quality of life (EORTC QLQ - BM22)(1 year)