Arterial Chemoembolization for the Treatment of Desmoid Fibromatosis

NCT ID: NCT06268457

Last Updated: 2025-04-02

Basic Information

• Recruitment Status: RECRUITING
• Total Enrollment: 20 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2024-02-20
• Study Completion Date: 2028-02-01

Brief Summary

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.

Detailed Description

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.

Conditions

Desmoid

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

Performed chemoembolization

Chemoembolization with Doxorubicin

Intervention Type: PROCEDURE

• 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 of the pathologic vasculature).

Interventions

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 of the pathologic vasculature).

Intervention Type: PROCEDURE

Eligibility Criteria

Inclusion Criteria

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

Exclusion Criteria

• 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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Istituto Ortopedico Rizzoli

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Istituto Ortopedico Rizzoli

Bologna, Emilia-Romagna, Italy

Site Status: RECRUITING

Countries

Italy

Facility Contacts

Giancarlo Facchini, MD

Role: primary

giancarlo.facchini@ior.it

+39 333 650 0944

Other Identifiers

CE-AVEC 789/2023/Oss/IOR

Identifier Type: -

Identifier Source: org_study_id