Journal of Rheumatology, The Journal of Rheumatology, p. jrheum.210273, 2021
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Objective To examine the efficacy and safety of interleukin-6 inhibition by tocilizumab in difficult-to-treat real-world patients with Systemic Sclerosis (SSc). Methods Twenty-one patients [20 women, 16 diffuse SSc, mean age: 52±10 years, 10 with early (<5 years) and 11 with long-standing disease (mean disease duration: 6.4±3.7 years)] with active joint and/or skin involvement refractory to corticosteroids (n=21), methotrexate (n=19), cyclophosphamide (n=10), mycophenolate (n=7), rituximab (n=1), leflunomide (n=2), hydroxychloroquine (n=2), and hematopoietic stem cell transplantation (n=2) who received weekly tocilizumab (162 mg subcutaneously) in an academic center, were monitored prospectively. Changes in modified Rodnan skin score (mRSS), disease activity score (DAS)28, lung function tests (LFTs) and patient reported outcomes (PROs) were analyzed after one year of treatment and at follow-up end. Results One patient discontinued tocilizumab after 3 months due to inefficacy. During the first year of treatment, improvement was evident in the remaining 20 patients regarding skin involvement (mean mRSS change: -6.9±5.9,p<0.001), polyarthritis (mean DAS28 change: -1.9±0.8,p<0.001) and PROs (all p<0.001); LFTs stabilization was observed in 16/20 patients. During the second year, 3 patients discontinued tocilizumab (cytomegalovirus infection in 1, inefficacy in 2) and one died. Beneficial effects were sustained in all 16 patients at follow-up end (2.2±1.1 years), except LFTs deterioration in 3. Apart from recurrent digital ulcer infection in 3 patients, tocilizumab was well-tolerated. Conclusion Tocilizumab was effective in refractory joint and skin involvement irrespective of SSc disease duration or subtype. Long-term retention rates and disease stabilization for most real-world patients suggest that tocilizumab might be a valuable choice for difficult-to-treat SSc.