Background: Tocilizumab (TCZ) is a humanized anti-human IL-6R antibody, a novel therapy for rheumatoid arthritis (RA) patients who fail treatment with disease modifying anti-rheumatic drugs (DMARDs) or anti-tumor necrosis factor (anti-TNFs).
Objective: To assess the safety and efficacy of TCZ monotherapy or in combination with non-biologic DMARDs or anti-TNFs in moderate to severe active RA.
Design: Prospective, phase III, multi-center, open-label, single arm, 24-week trial.
Setting: Three centers in Saudi Arabia.
Patients and methods: The study included consecutive RA patients infused with TCZ (8 mg/kg) over 60 minutes every 4 weeks (up to 6 times), either alone or with non-biologic DMARDs. Patients were followed for 24 weeks. Patients with good/moderate European League Against Rheumatism responses, continued on TCZ as long as commerically available or for 1 year.
Main outcome measure(s): Disease activity measured by DAS28 score.
Results: Of 28 patients enrolled from 2 November 2011 to 12 May 2013 (18 months), 21 completed (77.8%) and 7 (25%) discontinued TCZ therapy. One patient was excluded from the intent-to-treat analysis. Efficacy analysis showed a significant difference (P < .0001) in the Disease Activity Score based on 28 joints and on swollen and tender joint counts. Three (10.7%) patients experienced at least one AE that was considered related to study drug (one probably and two possibly). Only one (3.6%) patient reported a severe adverse event (neutropenia and thrombocytopenia). No adverse events led to dose modification or death.
Conclusion: TCZ monotherapy or in combination with non-biologic DMARDs resulted in a significant effect on the endpoints in moderate to severe RA in Saudi Arabia, which is consistent with other published reports.
Limitations: No information on tapering of steroid therapy, lack of follow-up data of all 28 patients, lack of data on long-term effects of TCZ on lipid levels and the need for statins. (ClinicalTrials.gov identifier: NCT01326962).