IL-6 Inhibition in Refractory Systemic Sclerosis Save

A small, single-center cohort analysis of difficult, refractory systemic sclerosis (SSc) patients showed that treatment with tocilizumab (TCZ), an interleukin-6 inhibitor, resulted in significant improvement in joint and skin involvement, regardless of SSc disease duration or subtype. 

Overall, 21 SSc patients (20 women; 16 diffuse cutaneous SSc;10 with early disease [< 5 yrs]) were studied. All had active joint and/or skin disease despite prior treatment with 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). All were given weekly subcutaneous TCZ (162 mg subcutaneously) and clinically assessed using the modified Rodnan skin score (mRSS), Disease Activity Score in 28 joints (DAS28), lung function tests (LFTs), and patient-reported outcomes (PROs) for the first 1 year of treatment.

While one patient discontinued TCZ after 3 months (lack of efficacy), 20 patients remained on therapy for 1 year. Notably there were significant improvements in:

• skin involvement (mean mRSS change: –6.9 ± 5.9, P < 0.001)
• polyarthritis (mean DAS28 change: –1.9 ± 0.8, P < 0.001),
• PROs (all P < 0.001);

In the second year, 16/20 remained on TCZ with 3 patients stopping TCZ (1 CMV infection, inefficacy in 2, 1 death), with clinical benefit still present after a mean of 2.2 ± 1.1 yrs.

Three patients did have LFT deterioration and another 3 patients had recurrent digital ulcer infections.

These 20 refractory SSc patientd did well with weekly SC TCZ, with improved skin and joint scores and long-term retention rates.