Real-World Evidence Supports Tocilizumab in SSc - EMJ

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Real-World Data Support Tocilizumab for Systemic Sclerosis

Tocilizumab Shows Real-World Benefit in Systemic Sclerosis - EMJ

TOCILIZUMAB stabilises lung function and improved skin, joint, and vascular disease in people with systemic sclerosis, a 197-patient French-Italian study has found, strengthening the case for the drug as a multidomain, potentially disease-modifying treatment in this hard-to-treat condition.

Systemic sclerosis (SSc) is a complex autoimmune disease that can scar the lungs, skin, joints, and blood vessels. Tocilizumab, which blocks interleukin-6 signalling, is approved for SSc-related interstitial lung disease (ILD), but real-world evidence on how it performs across the disease’s many domains has remained limited.

Tracking Multiple Disease Domains Over Two Years

The researchers retrospectively analysed 197 patients with SSc treated with tocilizumab at 15 referral centres (88% women; median age 57 years; median disease duration nine years; 67% anti-topoisomerase I positive). Clinical data were gathered at 12 months before treatment, at baseline, and at 12 and 24 months, covering the modified Rodnan skin score (mRSS), pulmonary function tests, a 28-joint disease activity score using C-reactive protein, digital ulcers, and cardiac biomarkers. ILD progression was defined as a fall of at least 5 points in predicted forced vital capacity over roughly 12 months. Tocilizumab was given alone in 29% of patients, while methotrexate was the commonest add-on at 35%.

Tocilizumab Steadies the Lungs in Systemic Sclerosis

In SSc-associated ILD, predicted forced vital capacity had already fallen significantly in the year before treatment, from 81% to 77% (p=0.003). After tocilizumab began, lung function stabilised and the share of patients progressing dropped from 43% to 24% (p=0.015). Among those with diffuse cutaneous disease, mRSS fell significantly at both 12 and 24 months, while digital ulcers, arthritis activity, and cardiac biomarkers also improved. Infections were the most common adverse event, affecting 22.8% of patients. Tocilizumab was stopped in 32%, chiefly for lack of efficacy. Pulmonary arterial hypertension and older age predicted treatment failure, whereas raised C-reactive protein predicted a better response.

Refining Who Benefits Most

The authors concluded that, in this large real-world cohort, tocilizumab was safe and delivered consistent benefits across multiple domains of systemic sclerosis, supporting its potential as a disease-modifying option in selected patients. They suggested raised C-reactive protein could help identify those most likely to respond, and that combining tocilizumab with immunosuppressive or antifibrotic drugs warrants further study.

Reference

Buonsante G et al. Safety and effectiveness of tocilizumab in systemic sclerosis: a multicentre French-Italian study. RMD Open. 2026;12:e006688.

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