Tocilizumab Effective for Resistant pJIA in Real-life Setting, Study Reports

Therapy with Actemra (tocilizumab) yields high survival rates in patients with hard-to-treat polyarticular juvenile idiopathic arthritis (pJIA), and enables many patients to reach low disease activity, a real-world study in Finland reports.

The study, “Efficacy and safety of tocilizumab in a real-life observational cohort of patients with polyarticular juvenile idiopathic arthritis,” was published in the journal Rheumatology.

Polyarticular juvenile idiopathic arthritis (pJIA) develops in children younger than 16 and is characterized by inflammation in five or more joints. The disease usually manifests in finger and hand joints, but can also occur in weight-bearing joints such as the knees, and joints surrounding the jawbone.

Children with pJIA tend to have more treatment-resistant disease than those with fewer joints affected, and longer periods of active disease.

The current recommendations suggest the use of TNF inhibitors — adalimumab (brand names Humira and Cyltezo, among others), etanercept (sold as Enbrel, among others), and golimumab (Simponi; approved for pJIA in the European Union but not in the United States) — as a first choice for pJIA patients when synthetic (chemical) disease-modifying antirheumatic drugs (DMARDs) have failed.

Actemra (marketed by Genentech, a Roche subsidiary) is another biological agent approved for pJIA, which acts through a different mechanism than TNF inhibitors. It blocks an inflammatory protein called IL-6.

Actemra is approved for the treatment of children age 2 and older and can be used alone or in combination with methotrexate, an immunosuppressant conventionally used for arthritis. However, in the EU, the medicine is sold as RoActemra (marketed by Roche) and is restricted to pJIA patients in whom prior treatment with methotrexate has not worked well.

Since Actemra’s approval, there is limited information about how well it works for pJIA patients in a real-world setting.

To fill this gap in knowledge, researchers conducted a nationwide observational study in Finland to detect the patterns of usage, efficacy, and safety of Actemra in pJIA patients during routine clinical practice.

The study involved 56 patients ages 2–17 who started on Actemra upon decision of their doctor. Researchers reviewed patients’ charts to determine the medicine’s efficacy and safety over two years after its initiation.

Actemra was started on average as a third-line biological agent, at a median disease duration of 5.2 years — a reflection that the medicine was prescribed to children who had treatment-resistant pJIA.

At one year after Actemra initiation, patient survival rates were 82%, decreasing to 64% at two years. Half of the patients withdrew treatment due to inadequate therapeutic effect. Some patients dropped out due to both adverse events and inadequate treatment effect (37.5%) and a smaller number due to adverse events alone (12.5%).

At one year of treatment, 58% of patients had reached low disease activity (defined as JADAS-10 score of 3.9 or lower). This increased to 84% at the end of two years of treatment.

A significant number of patients could go into a state of inactive disease (defined as a JADAS-10 score of 0.7 or lower), namely 19% and 44% following one and two years of treatment, respectively.

The same was true for a state of clinically inactive disease, as defined by Wallace’s criteria, which also takes into account uveitis, an inflammatory condition that can appear associated with arthritis and causes swelling and destruction of eye tissues. Using this criteria, inactive disease was achieved by 28% and 46% of the patients after one and two years of treatment, respectively.Treated patients also used significantly fewer synthetic DMARDs, and systemic or intra-articular glucocorticoids during the two-year follow-up period.The rate of adverse events was 200.9 per 100 patient years, meaning that 200.9 unwanted effects were seen if one would follow 100 patients on treatment during one year. The rate of serious events was much lower — 12.9 per 100 patient years.These rates were higher than in previous register studies but lower than those of the original clinical trial evaluating Actemra in pJIA.Infections were the most common adverse event (54 cases), detected in 24 patients (43%), most commonly upper respiratory, ear, and skin infections. Gastrointestinal events, such as nausea, aphthous ulcers, and abdominal pain, were also frequent (31 events in 18 patients).

“In conclusion, our study showed that survival of [Actemra] was good and a large proportion of the hard-to-treat pJIA patients reached [low disease activity] at 12 months of treatment. The rate of LDA continued to increase among those remaining on [Actemra] throughout 24 months,” the researchers said.

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