Originally published by 2 Minute Medicine® (view original article). Reused on AccessMedicine with permission.

1. Patients in the tofacitinib group reported significantly fewer flare-ups (29%) compared to placebo (53%) by week 44.

2. The occurrence of adverse events with tofacitinib was comparable to that with placebo and most events were mild in severity.

Evidence Rating Level: 1 (Excellent)

Study Rundown:

Juvenile idiopathic arthritis (JIA) is an auto-inflammatory condition affecting one or more joints in children below the age of 16. Currently, disease-modifying antirheumatic drugs (DMARDs), such as methotrexate, are used as first-line therapy for JIA. However, many patients have reported incomplete response. Janus kinase (JAK) inhibitors are an alternative class of anti-rheumatic medications that may be effective for non-responders to methotrexate. This randomized controlled trial aimed to compare the safety and efficacy of tofacitinib – a JAK inhibitor – in patients with polyarticular JIA. The study was divided into two parts; in part 1, all patients received tofacitinib for 18 weeks and during part 2, patients either continued tofacitinib or switched to placebo for 26 weeks. The primary endpoint was the rate of JIA flare-ups – defined as worsening by 30% or more in three core set variables – from baseline to 44 weeks. According to results, tofacitinib resulted in a significantly lower flare rate than placebo, with few serious adverse events. This study was strengthened by a large sample size with patients of various JIA subtypes.

In-depth [randomized controlled trial]:

Between June 10, 2016, and May 16, 2019, 286 patients were assessed for eligibility across 64 centers in 14 countries. Included were those aged 2 to 17 years with one of the following subtypes: extended oligoarthritis, RF-positive or RF-negative polyarthritis, systemic JIA, psoriatic arthritis, or enthesitis-related arthritis. Altogether, 225 patients were included, of which 82% had polyarticular JIA. Patients in the tofacitinib group reported significantly fewer flare-ups (29%) compared to placebo (53%) by 44 weeks (hazard ratio [HR] 0.46, 95% CI 0.27-0.79, p=0.0031). The rate of adverse events in both groups was comparable (77% in tofacitinib vs. 74% in placebo) and serious adverse events were rare (1% in tofacitinib vs. 2% in placebo). Overall, findings from this study suggest that tofacitinib may be both safe and effective in patients with polyarticular JIA. Given that it is orally administered, the use of tofacitinib may be preferred over more invasive and injectable therapies.

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