Background Adalimumab (ADA) is approved for use in moderate to severe JIA in patients (pts) ≥4 yrs old in the US, EU, and Japan. ADA has not been studied in pts <4 yrs old, and limited data are available in pts ≥4 yrs old weighing <15 kg.
Objectives To assess safety and effectiveness of ADA in pts 2 to <4 yrs old or ≥4 yrs old weighing <15 kg with moderately to severely active polyarticular JIA or polyarticular course JIA.
Methods This is a multicenter, open-label, Phase 3b study in pts 2 to <4 yrs old or ≥4 yrs weighing <15 kg with moderately to severely active JIA in the US and EU. ADA was given subcutaneously every other wk, 24 mg/m2 BSA up to 20 mg/dose, for a minimum of 24 wks or until pts reached 4 yrs old and weighed 15 kg. Concomitant use of DMARDs was allowed. Key effectiveness endpoints were the proportion of pts achieving PedACR30/50/70/90 at wk 24. Other outcomes were tender joint count (TJC), swollen joint count (SJC), Pain on Passive Motion (POM75), Limitation on Passive Motion (LOM69), Active Joint Count (AJC73), Child Health Assessment Questionnaire (DICHAQ), Physician’s Global Assessment of Disease (PhGA), and Parent’s Global Assessment (PaGA) of Disease and Pain. Adverse events (AE) are being collected throughout the treatment period and include safety data up to 96 wks.
Results 88% of the pts were female. At baseline, mean age was 3 yrs, mean weight=13 kg, mean JIA duration=12 months, and 39% had elevated CRP (≥0.9 mg/dL). Of 32 pts enrolled, 31 completed 24 wks of treatment. At wk 24, 90% of pts had achieved PedACR30 (Table 1). Improvements in other JIA outcomes were also seen at wk 24 of ADA treatment (Table 2).
AE incidence rates included: any AEs (84%, 27/32), serious AEs (16%, 5/32), infectious AEs (69%, 22/32), and serious infections (9%, 3/32); no deaths, malignancies, opportunistic infections/TB, congestive heart failure, demyelinating disease, allergic reactions, lupus-like syndrome, or blood dyscrasias were reported.
Conclusions Adalimumab was safe and effective in younger JIA patients (2 to <4 yr old or ≥4 yrs old weighing <15 kg) with active polyarticular disease, with a safety profile similar to that seen in older pediatric patients with JIA.
Disclosure of Interest D. Kingsbury Grant/Research support from: Abbott, P. Quartier Grant/Research support from: Abbott, Novartis, Pfizer, Consultant for: Abbott, S. Santra Shareholder of: Abbott, Employee of: Abbott, H. Kupper Shareholder of: Abbott, Employee of: Abbott, N. Mozaffarian Shareholder of: Abbott, Employee of: Abbott