Defibrotide and peripheral obliterative arterial disease: preliminary data

Area of Science:

• Vascular Medicine
• Pharmacology
• Clinical Trials

Background:

• Arterial occlusive disease of the lower limbs significantly impacts patient mobility and quality of life.
• Current treatment options for advanced stages remain limited, necessitating exploration of novel therapeutic agents.

Purpose of the Study:

• To evaluate the efficacy and safety of defibrotide in patients with arterial occlusive disease of the lower limbs.
• To assess the impact of defibrotide on walking distance, pain, and relevant physiological markers.

Main Methods:

• A pilot study involving 22 patients with Fontaine 2nd and 3rd stage arterial occlusive disease.
• Administration of defibrotide, with assessments of walking distance, pain, Doppler velocimetry, Winsor index, photoplethysmography, and laboratory parameters.
• Evaluation of beta-thromboglobulin levels as a marker of platelet activation.

Main Results:

• Significant improvement in walking distance observed in Fontaine 2nd stage patients (p < 0.001), sustained post-treatment.
• Pain reduction reported in Fontaine 3rd stage patients, with resting pain eliminated in 4 individuals.
• Statistically significant reduction in beta-thromboglobulin levels (p < 0.001) observed during and after defibrotide treatment.
• No adverse effects on hepatic, renal, hemopoietic, or hemocoagulative functions were noted.

Conclusions:

• Defibrotide demonstrates significant efficacy in improving walking distance for Fontaine 2nd stage arterial occlusive disease patients.
• The drug shows promise in pain management for advanced stages and is well-tolerated, with no observed organ system toxicity.
• Defibrotide is a suitable treatment option for arterial occlusive disease stages where collateral circulation can be activated.