Abstract

Background: Systemic sclerosis (SSc) is a rare autoimmune disease characterized by endothelial dysfunction and progressive fibrosis leading to organ failure. The increasing endothelial damage is one of the hallmarks of the disease [1]. Aminaphtone is a synthetic molecule registered to treat microvascular disorders that interferes with the expression of vasoactive mediators released by activated endothelial cells [2,3]. Nailfold videocapillaroscopy (NVC) is the standardized tool to assess microvascular damage and may be considered a morphological biomarker for detection of SSc diagnosis and progression [4]. Objectives: The aim of our study was to evaluate the safety and the possible effects of aminaphtone on microvascular morphology when added to standard treatment (ST) in SSc patients in a time frame of five years. Methods: Seventy-six SSc patients (68 females and 8 males, mean age 69–15 years) according to 2013 ACR/EULAR criteria with active secondary Raynaud's phenomenon started aminaphtone treatment (75 mg BID) in addition to stable ST for SSc. As control group we used a cohort of forty-two age and sex matched SSc patients treated only with ST. All patients provided written informed consent to manage their clinical data. Possible side effects related to aminaphtone treatment were carefully checked every six months. NVC was assessed at baseline, after 1 and 5 years of treatment, adopting the SSc-pattern classification ("Early", "Active", "Late") [4,5]. The timing of transition of the scleroderma-pattern was compared between patients treated with aminaphtone and patients only treated with ST. Results: In our cohort of patients no serious side-effect has been reported during the observational time frame of 5 years. Nevertheless, 10% of SSc patients presented mild intolerance due to well-known reversable adverse events (headache 2.7%, itching 1.3%, others 6%) leading to aminaphtone discontinuation. NVC patterns in SSc patients treated with aminaphtone remained stable during the observational time frame in 91% of cases; in 9% of patients was observed a transition from "Early" to "Active" or from "Active" to "Late" SSc-pattern (6.6% and 3.4%, respectively). Of note, SSc patients treated with aminaphtone showed a significantly slower time of transition between NVC patterns compared to the control group of patients treated with ST alone (from "Active" to "Late" 120±64 months vs 50±26 months, p=0.05), while a similar transition time from "Early" to "Active" (36±30 months vs 36±42 months; p>0.05) was observed in both groups. Conclusion: Aminaphtone was shown to be safe and well tolerated in SSc patients with secondary RP during long term treatment (5 years). The scleroderma-pattern stability over five years follow-up, observed at NVC examination, suggests the potential supplementary therapeutic effect by aminaphtone when used alongside standard therapy in SSc patients, at least on vascular tissue damage. Further investigations to confirm this outcome are ongoing. REFERENCES: [1] Sulli A et al. Rheumatology (Oxford) 2020 1;59(5):1051-1058. [2] Gotelli E et al. Pharmaceuticals (Basel) 2023;16(4):569. [3] Ruaro B et al. Front Pharmacol 2019;10:293. [4] Cutolo M et al. Nat Rev Rheumatol 2010;6, 578–87. [5] Smith V et al. Autoimmun Rev. 2020;19(3):102458. Acknowledgements: NIL. Disclosure of Interests: None declared.