ETA2023 Poster Presentations Thyroid Eye Disease (9 abstracts)
Long-Term outcome of moderate-to-severe, active graves’ orbitopathy following treatment with sirolimus (Ramamycin): results in a case series
Simone Comi 1 , Giulia Lanzolla 1 , Giada Cosentino 2 , Francesca Menconi 3 , Maria Novella Maglionico 4 , Chiara Posarelli 5 , Lorenzo Leni 1 , Michele Figus 5 , Rossella Elisei 6 , Ferruccio Santini 7 & Michele Marinò 8
1University of Pisa, Department of Clinical and Experimental Medicine, Pisa, Italy; 2University of Pisa, Department of Clinical and Experimental Medicine, Endocrinology, Pisa, Italy; 3Endocrinology Unit, University of Pisa, University of Pisa, Department of Clinical and Experimental Medicine, Pisa, Italy; 4University of Pisa, Department of Surgical, Medical and Molecular Pathology and Critical Care Medicine, Pisa, Italy; 5University of Pisa, Ophthalmology Unit I, Department of Surgical, Medical and Molecular Pathology, Pisa, Italy; 6Oncology Section of the Endocrine Unit, Department of Clin and Exp Medicine, University Pisa, Pisa, Italy; 7Dipartimento DI Endocrinologia, University de Pisa, Pisa, Italy; 8Department of Clinical and Experimental Medicine, University of Pisa, Pisa, Italy., Department of Clinical and Experimental Medicine, Pisa, Italy
Objectives: Sirolimus is an immunosuppressive drug with anti-fibrotic and anti-proliferative activities. In a recent study, sirolimus (given off-label as a second-line treatment) was found to be associated with a better outcome of Graves’ orbitopathy (GO) at 6 months compared to the standard treatment (intravenous glucocorticoids). Here we investigated the effects of sirolimus over a longer period of time.
Methods: The study design entailed data analysis of 10 consecutive patients [2 men and 8 women, age: 60.7 (10) yr.] with moderate-to-severe and active GO, given sirolimus off-label as a second-line treatment at relatively low dosage (2 mg orally on first day, followed by 0.5 mg/day for 12 weeks). Primary outcome was the overall outcome (composite evaluation) of GO over time (12, 24 and 48 weeks). Secondary outcomes were: 1) outcome of single eye features; 2) outcome of quality of life (GO-QoL); 3) TSH-receptor antibodies (TRAbs) across the observational period.
Results: The proportions of overall GO responders at 12, 24 and 48 weeks were 50%, 70% and 60%, respectively. Proptosis responders (reduction by at least 2 mm) were 50% at 12 and 24 weeks and 30% at 48 weeks. Clinical activity score responders (reduction by at least one point on a 5-point scale) were 40%, 50% and 60% (12, 24 and 48 weeks, respectively). An improvement of diplopia (disappearance or improvement in Gorman’s score) was observed in 50% of patients at 12 weeks, 60% at 24 weeks, and 70% at 48 weeks. GO-QoL improved by at least 6% in 50% of patients at all time points. TRAb decreased over time, although not to a statistically significant extent. Ten mild adverse events were recorded in 6 patients, 4 of which possibly related to sirolimus.
Conclusions: Treatment with sirolimus is followed by a fairly good overall response of GO and of single eye features and the effect appears to be sustained over time. Thus, sirolimus may represent a valid and safe alternative treatment for moderate-to-severe and active GO. Further, randomized clinical trials are needed to confirm the efficacy and safety of sirolimus.
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Endocrine Abstracts
ISSN 1470-3947 (print) | ISSN 1479-6848 (online)
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