Arterial Chemoembolization for the Treatment of Desmoid Fibromatosis

Официальное название

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

Обзор

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.

Подробное описание

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.

Вмешательства

• Процедура Chemoembolization with Doxorubicin
  * Identification of the main vessels afferent to the metastasis to be treated. * Selective and super-selective catheterization of pathological arterial branches, responsible for the blood supply to the neoplasm, through the use of micro-catheters. * Injection of embolizing material (hydrogel microspheres with doxorubicin) * Control of technical success: the embolization will be followed by diagnostic arteriography that will evaluate the success of the procedure (occlusion of more than 90 percent

Первичные конечные точки

• Volume reduction in cm [Срок оценки: 1 year]
• Reduction of pain (VAS score) [Срок оценки: 1 year]
• Reduction of antalgic therapy (mg) [Срок оценки: 1 year]

Вторичные конечные точки (2)

• Improvement in quality of life (EORTC QLQ-C15-PAL questionnaire) [Срок оценки: 1 year]
• Improvement in quality of life (EORTC QLQ - BM22) [Срок оценки: 1 year]

Критерии участия

Критерии включения

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

Критерии исключения

• 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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

Италия · 1 центр

• Istituto Ortopedico Rizzoli — Bologna

Идентификаторы

NCT: NCT06268457 · CE-AVEC 789/2023/Oss/IOR

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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