Tofacitinib in juvenile idiopathic arthritis: a double-blind, placebo-controlled, withdrawal phase 3 randomised trial

Tofacitinib, an oral, small-molecule Janus kinase inhibitor, was shown to have potential efficacy as induction therapy for ulcerative colitis in a phase 2 trial. We further evaluated the efficacy of tofacitinib as induction and maintenance therapy.

To develop preliminary criteria for inactive disease and clinical remission for select categories of juvenile idiopathic arthritis (JIA), and to decide what such clinical states should predict in terms of probability of disease recurrence. A Delphi serial questionnaire consensus-formation approach was used initially to gather criteria in use by pediatric rheumatologists (PR) for defining clinical remission in oligoarticular (persistent and extended), rheumatoid factor (RF) positive and negative polyarticular, and systemic JIA. Results from sequential questionnaires provided an agenda for a nominal group technique (NGT) conference to reach consensus on unresolved questions. One hundred and thirty PR from 34 countries responded to the questionnaires and 20 PR from 9 countries attended the conference. Draft criteria for inactive disease include the following: no active arthritis; no fever, rash, serositis, splenomegaly, or generalized lymphadenopathy attributable to JIA; no active uveitis; normal erythrocyte sedimentation rate or C-reactive protein; and a physician's global assessment of disease activity rated at the best score possible for the instrument used. According to consensus vote, 6 continuous months of inactive disease on medication defines clinical remission on medication, while 12 months of inactive disease off all anti-arthritis (and anti-uveitis) medications defines clinical remission off medication. The finalized criteria for remission off medication ideally should predict that a patient has