Objectives: To compare real-world effectiveness of two adaptive treatment strategies of disease-modifying antirheumatic drugs (DMARDs) in treating children with newly diagnosed polyarticular course juvenile idiopathic arthritis (pcJIA): early aggressive use of biologic DMARDs (bDMARDs) in combination with conventional synthetic DMARDs (csDMARDs) versus conservative delayed use of bDMARDs following the initial csDMARD prescription.
Methods: A single-centre newly diagnosed DMARD-naive pcJIA patient database (n=465) was derived from the electronic medical records between 1 January 2009 and 31 December 2018. The primary study endpoints were clinical Juvenile Arthritis Disease Activity Score (cJADAS) at 6 and 12 months following the first DMARD prescription. The secondary study endpoint was Pediatric Quality of Life Inventory (PedsQL) generic total score at 12 months. Averaged causal treatment effects were assessed using a Bayesian non-parametric casual inference method.
Results: Both cJADAS and PedsQL improve over time, regardless of the treatment strategies. Compared with the conservative approach, early aggressive approach is more effective in reducing cJADAS (mean -2.17, 95% CI -3.77 to -0.56) by 6 months. Adding bDMARD after 6 months to the initial treatment provides very little added benefit. The averaged treatment effect was 6.35 (95% CI -5.89 to 18.58) improvement in PedsQL at 12 months.
Conclusions: Timing matters-early aggressive use with bDMARDs is more effective than conservative delayed treatment in lowering disease activity after 6 and 12 months of treatment.
Keywords: DMARDs (biologic); DMARDs (synthetic); disease activity; juvenile idiopathic arthritis; outcomes research.
© Author(s) (or their employer(s)) 2020. Re-use permitted under CC BY. Published by BMJ.