Clinical trial NCT06945887: Treatment of Desmoid Fibromatosis With Arterial Embolization
Official title: Treatment of Desmoid Fibromatosis With Arterial Embolization: Retrospective and Prospective Observational Study
Study type: OBSERVATIONAL
Overall status: NOT_YET_RECRUITING
Conditions: Desmoid Fibromatosis
Keywords: Desmoid fibromatosis
Embolization
Lead sponsor: Istituto Ortopedico Rizzoli
Sponsor class: OTHER
First posted: 2025-04-27
Enrollment: 20
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients affected by desmoid fibromatoses eligible to be treated with transarterial embolization

Brief summary: 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.

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Reduction of pain (VAS score)
timeFrame: 1 year
measure: Reduction of antalgic therapy (mg)
timeFrame: 1 year
measure: Volume reduction in cm³
timeFrame: 1 year
Secondary outcomes:
measure: Improvement in quality of life (EORTC QLQ-C15-PAL questionnaire)
timeFrame: 1 year
measure: Improvement in quality of life (EORTC QLQ - BM22)
timeFrame: 1 year
Other outcomes:
Locations:
city: Bologna
country: Italy
facility: Istituto Ortopedico Rizzoli
state: BO
Study officials:
References: