Tocilizumab for systemic sclerosis: implications for future trials

Abstract

Systemic sclerosis is a chronic autoimmune disease that causes high morbidity and increases mortality. Although treatments have become available for some manifestations such as pulmonary arterial hypertension, there is still no approved therapy for fibrotic manifestations and the overall course of disease. 1 Kowal-Bielecka O Landewe R Avouac J et al. EULAR recommendations for the treatment of systemic sclerosis: a report from the EULAR Scleroderma Trials and Research group (EUSTAR). Ann Rheum Dis. 2009; 68: 620-628 Crossref PubMed Scopus (500) Google Scholar However, following findings about the pathogenesis of fibrotic diseases, several new targeted therapies are being tested in clinical studies. 2 Distler O Cozzio A Systemic sclerosis and localized scleroderma-current concepts and novel targets for therapy. Semin Immunopathol. 2016; 38: 87-95 Crossref PubMed Scopus (72) Google Scholar , 3 Ramming A Dees C Distler JH From pathogenesis to therapy—perspective on treatment strategies in fibrotic diseases. Pharmacol Res. 2015; 100: 93-100 Crossref PubMed Scopus (15) Google Scholar Safety and efficacy of subcutaneous tocilizumab in adults with systemic sclerosis (faSScinate): a phase 2, randomised, controlled trialTocilizumab was not associated with a significant reduction in skin thickening. However, the difference was greater in the tocilizumab group than in the placebo group and we found some evidence of less decline in forced vital capacity. The efficacy and safety of tocilizumab should be investigated in a phase 3 trial before definitive conclusions can be made about its risks and benefits. Full-Text PDF