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临床试验/NCT06268457
NCT06268457招募中不适用

Chemioembolizzazione Arteriosa Per il Trattamento Della Fibromatosi Desmoide: Studio Osservazionale Prospettico

Istituto Ortopedico Rizzoli2 个研究点 分布在 1 个国家目标入组 20 人开始时间: 2024年2月20日最近更新:
适应症

试验速览

阶段
不适用
状态
招募中
入组人数
20
试验地点
2
主要终点
Volume reduction in cm

研究概览

简要总结

Desmoid fibromatoses are rare (1-2 cases/million per year) and locally aggressive mesenchymal tumors. 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. Embolization of desmoid tumors alone, without chemotherapy, on the contrary, has been shown to be inefficient. Using Doxorubicin in desmoid fibromatosis is effective but associated with systemic toxicity. Consequently, this drug is reserved for symptomatic, nonresponsive, rapidly growing or life-threatening tumors. The intrinsic hypervascularity of desmoid tissue can be exploited as a conduit to achieve local distribution of Doxorubicin by navigation of a catheter endovascular.

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. Embolization of desmoid tumors alone, without chemotherapy, on the contrary, has been shown to be inefficient. 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 tumors. The intrinsic hypervascularity of desmoid tissue can be exploited as a conduit to achieve local distribution of Doxorubicin by navigation of a catheter endovascular. Doxorubicin contains a protonated amine group which can establish an ionic bond with the sulfonate present on the surface of microbeads of hydrogel, ensuring embolization and elution of the drug. This process allows high concentrations of Doxorubicin in the target tissue and low concentrations in the systemic circulation.

研究设计

研究类型
Observational
观察模型
Cohort
时间视角
Prospective

入排标准

年龄范围
18 Years 至 —(Adult, Older Adult)
性别
All
接受健康志愿者

入选标准

  • FIbromatosis demsoid symptomatic and in active phase (documented growth at last follow-ups)
  • Patients who are not candidates for surgery or cryoablation

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

结局指标

主要结局

Volume reduction in cm

时间窗: 1 year

Reduction of the lesion evaluated with MRI, measuring the diameter of the lesion in cm.

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.

次要结局

  • Improvement in quality of life (EORTC QLQ - BM22)(1 year)
  • Improvement in quality of life (EORTC QLQ-C15-PAL questionnaire)(1 year)

研究者

申办方类型
Other
责任方
Sponsor

研究点 (2)

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