The goal of this observational study is to learn about the effects of Electrochemotherapy (ECT) in patients with Desmoid Fibromatosis. The main question it aims to answer is: Is ECT effective in reducing the size of the lesion and improving patients' symptomatology? Patients will be followed with MRI and Quality of Life questionnaires at 3, 6 and 12 months after the intervention.
Desmoid fibromatoses are rare 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. Other minimally invasive local treatments such as cryoablation have been shown to be effective; however, certain patients are not candidates for such treatment because of lesion location/characteristics. An additional minimally invasive treatment with currently little evidence in the literature is arterial embolization. However, this procedure cannot be performed in the case of superficial lesions, nor in the presence of shared vasculature between the lesion and other healthy tissues or organs. In the absence of other treatments in use, electrochemotherapy treatment can be performed, which has proven effective on several other types of soft tissue and bone tumors, malignant or benign, treated at our Institute. Electrochemotherapy is based on the principle whereby reversible electroporation causes the application of short, high-voltage electrical pulses to temporarily align water molecules on the hydrolipid layer of the cell membrane, leading to a reversible local increase in permeability by increasing the intracellular concentration of Bleomycin and its toxicity on tumor cells by a factor of up to several thousand.
Study Type
OBSERVATIONAL
Enrollment
30
Electrochemotherapy (ECT) with bleomycin is a localized treatment approach used for managing desmoid fibromatosis, a benign but locally aggressive tumor. This technique combines electrical pulses with the administration of bleomycin, a chemotherapeutic agent, to enhance drug uptake into tumor cells. The electrical pulses create temporary pores in the cell membranes, allowing bleomycin to enter more effectively, leading to increased cytotoxicity. ECT is typically performed on patients who have not responded to conventional therapies or for whom surgery is not feasible. ECT can lead to significant tumor shrinkage and improvement in symptoms, making it a promising option in the multidisciplinary management of desmoid fibromatosis.
IRCCS Istituto Ortopedico Rizzoli
Bologna, Italy
RECRUITINGVolume reduction in cm
Change of the lesion evaluated with MRI, measuring the diameter of the lesion in cm.
Time frame: 1 year
Change 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.
Time frame: 1 year
Change in quality of life ( EuroQol-5D questionnaire)
Clinical assessment regarding quality of life by EuroQol-5D questionnaire. EQ-5D score can range from -0.594 to 1.0. The minimum value represents the worst possible health condition. The maximum value of 1.0 represents the best possible health condition. A value of 1.0 indicates that the patient has no problem in any of the five dimensions assessed by the questionnaire.
Time frame: 1 year
Feasibility of in vitro viability analysis of biopsy material
Evaluation of the feasibility of in vitro viability analysis of biopsy material for culturing explants and stabilizing primary cultures. Success is defined as the establishment of primary culture in 70% of samples. Because desmoid tumors are not aggressive, it may not be easy to maintain them in culture in vitro.
Time frame: 1 year
Evaluation of the role of inflammatory cytokines
Evaluation of the prognostic or predictive role of inflammatory cytokines detected in the blood of patients collected on the day of treatment and after 3 months
Time frame: 3 months
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