Related Experiment Video
Updated: Dec 10, 2025
04:44
Imaging Features of Systemic Sclerosis-Associated Interstitial Lung Disease
Published on: June 16, 2020
20.7K
Tocilizumab in systemic sclerosis: a randomised, double-blind, placebo-controlled, phase 3 trial
Dinesh Khanna1, Celia J F Lin2, Daniel E Furst3
1University of Michigan, Ann Arbor, MI, USA.
The Lancet. Respiratory Medicine
|September 1, 2020
Summary
Tocilizumab did not significantly improve skin fibrosis in systemic sclerosis patients. However, it showed potential in preserving lung function for those with SSc-ILD, with a safety profile consistent with prior use.
Area of Science:
- Rheumatology
- Immunology
- Pulmonology
Background:
- Systemic sclerosis (SSc) is a chronic autoimmune disease characterized by skin fibrosis and potential organ involvement.
- Tocilizumab, an anti-interleukin-6 receptor antibody, demonstrated preliminary efficacy in a phase 2 trial for SSc.
- This phase 3 trial aimed to further evaluate tocilizumab's safety and efficacy in treating SSc.
Purpose of the Study:
- To assess the efficacy of tocilizumab in reducing skin fibrosis in patients with diffuse cutaneous systemic sclerosis.
- To evaluate the effect of tocilizumab on systemic sclerosis-associated interstitial lung disease (SSc-ILD).
- To determine the safety profile of tocilizumab in this patient population.
Main Methods:
- A multicentre, randomized, double-blind, placebo-controlled phase 3 trial involving 210 adults with early diffuse cutaneous SSc.
- Participants received weekly subcutaneous tocilizumab or placebo for 48 weeks.
- Primary endpoint: change in modified Rodnan skin score (mRSS); secondary endpoints included FVC% predicted and time to treatment failure.
Main Results:
- The primary endpoint (reduction in mRSS) was not met; no significant difference between tocilizumab and placebo.
- Tocilizumab significantly favored preservation of lung function (FVC% predicted) compared to placebo (nominal p=0.0002).
- Time to treatment failure showed a trend favoring tocilizumab (nominal p=0.08). Safety was consistent with tocilizumab's known profile, with infections being the most common adverse events.
Conclusions:
- Tocilizumab did not meet the primary endpoint for skin fibrosis in early diffuse cutaneous SSc.
- Tocilizumab may help preserve lung function in patients with SSc-ILD, particularly those with elevated acute-phase reactants.
- The safety profile of tocilizumab in this trial was consistent with its established safety data.
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