Stylianos T Panopoulos1, Maria G Tektonidou2, Vasiliki-Kalliopi Bournia2, Aikaterini Arida2, Petros P Sfikakis2. 1. S.T. Panopoulos, MD, PhD, M.G. Tektonidou, MD, PhD, Professor, V.K. Bournia, MD, PhD, A. Arida, MD, PhD, P.P. Sfikakis, MD, PhD, Professor, Department of First Propaedeutic and Internal Medicine, Joint Rheumatology Program, Medical School of Athens, General Hospital of Athens "LAIKO", Athens, Greece. sty.panopoulos@gmail.com. 2. S.T. Panopoulos, MD, PhD, M.G. Tektonidou, MD, PhD, Professor, V.K. Bournia, MD, PhD, A. Arida, MD, PhD, P.P. Sfikakis, MD, PhD, Professor, Department of First Propaedeutic and Internal Medicine, Joint Rheumatology Program, Medical School of Athens, General Hospital of Athens "LAIKO", Athens, Greece.
Abstract
OBJECTIVE: To examine the efficacy and safety of interleukin-6 inhibition by tocilizumab (TCZ) in difficult-to-treat, real-world patients with systemic sclerosis (SSc). METHODS: Twenty-one patients (20 women; 16 diffuse cutaneous SSc; mean age: 52 ± 10 yrs; 10 with early disease [< 5 yrs]; and 11 with long-standing disease [mean disease duration 6.4 ± 3.7 yrs]) with active joint and/or skin involvement refractory to corticosteroids (n = 21), methotrexate (n = 19), cyclophosphamide (n = 10), mycophenolate mofetil (n = 7), rituximab (n = 1), leflunomide (n = 2), hydroxychloroquine (n = 2), and hematopoietic stem cell transplantation (n = 2), who received weekly TCZ (162 mg subcutaneously) in an academic center, were monitored prospectively. Changes in modified Rodnan skin score (mRSS), Disease Activity Score in 28 joints (DAS28), lung function tests (LFTs), and patient-reported outcomes (PROs) were analyzed after 1 year of treatment and at end of follow-up. RESULTS: One patient discontinued TCZ 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); LFT stabilization was observed in 16/20 patients. During the second year, 3 patients discontinued TCZ (cytomegalovirus infection in 1, inefficacy in 2) and 1 died. Beneficial effects were sustained in all 16 patients at end of follow-up (2.2 ± 1.1 yrs), except LFT deterioration in 3 patients. Apart from recurrent digital ulcer infection in 3 patients, TCZ was well tolerated. CONCLUSION: TCZ was effective in refractory joint and skin involvement regardless of SSc disease duration or subtype. Long-term retention rates and disease stabilization for most real-world patients suggest that TCZ might be a valuable choice for difficult-to-treat SSc.
OBJECTIVE: To examine the efficacy and safety of interleukin-6 inhibition by tocilizumab (TCZ) in difficult-to-treat, real-world patients with systemic sclerosis (SSc). METHODS: Twenty-one patients (20 women; 16 diffuse cutaneous SSc; mean age: 52 ± 10 yrs; 10 with early disease [< 5 yrs]; and 11 with long-standing disease [mean disease duration 6.4 ± 3.7 yrs]) with active joint and/or skin involvement refractory to corticosteroids (n = 21), methotrexate (n = 19), cyclophosphamide (n = 10), mycophenolate mofetil (n = 7), rituximab (n = 1), leflunomide (n = 2), hydroxychloroquine (n = 2), and hematopoietic stem cell transplantation (n = 2), who received weekly TCZ (162 mg subcutaneously) in an academic center, were monitored prospectively. Changes in modified Rodnan skin score (mRSS), Disease Activity Score in 28 joints (DAS28), lung function tests (LFTs), and patient-reported outcomes (PROs) were analyzed after 1 year of treatment and at end of follow-up. RESULTS: One patient discontinued TCZ 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); LFT stabilization was observed in 16/20 patients. During the second year, 3 patients discontinued TCZ (cytomegalovirus infection in 1, inefficacy in 2) and 1 died. Beneficial effects were sustained in all 16 patients at end of follow-up (2.2 ± 1.1 yrs), except LFT deterioration in 3 patients. Apart from recurrent digital ulcer infection in 3 patients, TCZ was well tolerated. CONCLUSION: TCZ was effective in refractory joint and skin involvement regardless of SSc disease duration or subtype. Long-term retention rates and disease stabilization for most real-world patients suggest that TCZ might be a valuable choice for difficult-to-treat SSc.
Authors: Anca Cardoneanu; Alexandra Maria Burlui; Luana Andreea Macovei; Ioana Bratoiu; Patricia Richter; Elena Rezus Journal: Biomedicines Date: 2022-01-29