Clinical trial NCT06268457: Arterial Chemoembolization for the Treatment of Desmoid Fibromatosis
Official title: Chemioembolizzazione Arteriosa Per il Trattamento Della Fibromatosi Desmoide: Studio Osservazionale Prospettico
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Desmoid
Keywords: Chemoembolization
Lead sponsor: Istituto Ortopedico Rizzoli
Sponsor class: OTHER
First posted: 2024-02-20
Enrollment: 20
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients affected by desmoid fibromatoses eligible to be treated with chemoembolization with Doxorubicin

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.

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

Primary outcomes:
measure: Volume reduction in cm
timeFrame: 1 year
measure: Reduction of pain (VAS score)
timeFrame: 1 year
measure: Reduction of antalgic therapy (mg)
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: Emilia Romagna
Study officials:
References: