Tocilizumab beneficial for adults with persistent polymyalgia rheumatica symptoms who receive steroid therapy

Reference

Devauchelle-Pensec V, Carvajal-Alegria G, Dernis E, et al. Effect of tocilizumab on disease activity in patients with active polymyalgia rheumatica receiving glucocorticoid therapy. A randomized clinical trial. JAMA 2022;328(11):1053-62.

Synopsis

We need effective alternative treatments for persistent polymyalgia rheumatica (PMR) that requires long-term glucocorticoids. These investigators identified adults, older than 50 years, who met the standard diagnostic criteria for PMR and had a C-reactive protein (CRP) level of at least 10 mg/L or an erythrocyte sedimentation rate (ESR) of at least 20 mm per hour at disease onset. Eligible patients (N = 101) included those who responded to 12 mg to 25 mg per day of prednisone to attain a CRP level less than 10 mg/L or an ESR less than 20 mm per hour, but subsequently developed glucocorticoid dependency with worsening disease activity after tapering to a prednisone dosage less than 10 mg per day. Patients randomly received (concealed allocation assignment) an intravenous infusion of tocilizumab (8 mg/kg) every 4 weeks for 24 weeks or matched placebo. The primary outcome was a composite of disease activity, defined as a PMR activity score computed using the CRP level of less than 10 (range 0-100; higher scores indicate greater disease activity; based on minutes of morning stiffness, ability to elevate upper limbs, physician global assessment, patient reported pain, and CRP level), and either a prednisone dosage of less than or equal to 5 mg per day or a prednisone dosage decrease by at least 10 mg versus baseline. At weeks 12, 16, and 20 prednisone dosages greater than 10 mg per day were tapered by 5 mg. For those patients already taking prednisone dosages equal to or less than 10 mg per day, if the disease activity score was less than 10, the prednisone dosage was decreased by 2 mg every 2 weeks. Individuals masked to treatment group assignment assessed outcomes. Complete follow-up occurred for all patients at 24 weeks. Using intention-to-treat analysis, at 24 weeks the primary end point occurred significantly more often in patients treated with tocilizumab than in patients treated with placebo (67.3% vs 31.4%, respectively; number needed to treat = 2.8; 95% CI 1.9 - 6.1). No significant group differences occurred in adverse events leading to treatment discontinuation.