Treatment of Desmoid Fibromatosis With Arterial Embolization (TAE-DESMO)

Treatment of Desmoid Fibromatosis With Arterial Embolization: Retrospective and Prospective Observational Study

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.

Study Overview

• Status: Recruiting
• Conditions: Desmoid Fibromatosis
• Intervention / Treatment: Procedure: embolization therapy

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.

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.

• Study Type: Observational
• Enrollment (Estimated): 20

Contacts and Locations

• Study Contact: Name: Giancarlo Facchini, Medicine and Surgery; Phone Number: +39 0516366836; Email: giancarlo.facchini@ior.it

Study Locations

• Italy
  • BO
    • Bologna, BO, Italy, 40136
      • Recruiting
      • Istituto Ortopedico Rizzoli
      • Contact: Giancarlo Facchini, Medicine and Surgery; Phone Number: +39 0516366836; Email: giancarlo.facchini@ior.it
      • Principal Investigator: Giancarlo Facchini, Medicine and Surgery

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Patients affected by desmoid fibromatoses eligible to be treated with transarterial embolization

Description

Inclusion Criteria:

• 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

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
• Incompatibility to performing MRI examination.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort

Intervention / Treatment

Performed embolization

Procedure: embolization therapy; Identification of the main vessels afferent to the tumor 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) - 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).

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Reduction of pain (VAS score)	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.	1 year
Reduction of antalgic therapy (mg)	Mean reduction in the use of pain medications evaluated in mg of active ingredient taken daily before the treatment and at follow-up.	1 year
Volume reduction in cm³	Reduction of the lesion evaluated with MRI, measuring the diameter of the lesion in cm³.	1 year

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Improvement in quality of life (EORTC QLQ - BM22)	Clinical assessment regarding quality of life by EORTC QLQ - BM22 questionnaire. The questionnaire consists of several scales, covering different aspects related to palliation. The maximum and minimum scores for each scale of the questionnaire can range from 0 to 100. The interpretation of scores depends on the specific scale in the questionnaire, and a higher value may indicate either greater negative impact (e.g., more symptoms or more suffering) or greater positive impact (e.g., better quality of life or less symptomatology). The overall assessment requires a detailed analysis of individual scores and related scales.	1 year
Improvement in quality of life (EORTC QLQ-C15-PAL questionnaire)	Clinical assessment regarding quality of life by EORTC QLQ-C15-PAL questionnaire. The questionnaire consists of several scales, covering different aspects related to palliation. The maximum and minimum scores for each scale of the questionnaire can range from 0 to 100. The interpretation of scores depends on the specific scale in the questionnaire, and a higher value may indicate either greater negative impact (e.g., more symptoms or more suffering) or greater positive impact (e.g., better quality of life or less symptomatology).	1 year

Collaborators and Investigators

• Sponsor: Istituto Ortopedico Rizzoli

Study record dates

Study Major Dates

• Study Start (Actual): July 1, 2025
• Primary Completion (Estimated): February 1, 2028
• Study Completion (Estimated): February 1, 2029

Study Registration Dates

• First Submitted: April 18, 2025
• First Submitted That Met QC Criteria: April 18, 2025
• First Posted (Actual): April 27, 2025

Study Record Updates

• Last Update Posted (Estimated): September 12, 2025
• Last Update Submitted That Met QC Criteria: September 11, 2025
• Last Verified: September 1, 2025

More Information

Terms related to this study

• Keywords: Embolization; Desmoid fibromatosis
• Additional Relevant MeSH Terms: Neoplasms; Neoplasms by Histologic Type; Neoplasms, Connective and Soft Tissue; Neoplasms, Connective Tissue; Neoplasms, Fibrous Tissue; Fibroma; Desmoid Tumors; Therapeutics; Hemostatic Techniques; Therapeutic Occlusion; Embolization, Therapeutic
• Other Study ID Numbers: CE AVEC: 49/Oss/IOR

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No