Adalimumab Monotherapy or Combination Therapy With Methotrexate in Paediatric Uveitis: Data From the AIDA Network Uveitis Registry

Abstract

Background: The study objective was to compare the effectiveness of adalimumab (ADA) in monotherapy and in combination with methotrexate (MTX) for paediatric noninfectious uveitis (NIU).

Methods: Registry-based observational study. Children receiving ADA for active uveitis were divided into the ADA monotherapy group (group 1) and the ADA plus MTX combination group (group 2).

Results: Eighty four children were enrolled (146 eyes): 22 in group 1 (26.2%) and 62 in group 2 (73.8%). ADA effectiveness was complete in 48 children (57.1%), partial in 23 (27.4%) and absent in 4 (5.3%), without any differences across the groups (p = 0.89). Fewer relapses per 100 PY occurred after ADA treatment both in group 1 (280.0 vs. 23.0, p = 0.005) and in group 2 (297.9 vs. 86.0, p < 0.001). The final BCVA was similar between groups 1 and 2 [median 1.0 (IQR 0.3) and 1.0 (IQR 0.3), respectively, p = 0.55]. A statistically significant steroid-sparing effect was observed in the entire cohort and in group 2 at the 6-month (p = 0.01 and p = 0.01), 12-month (p = 0.02 and p = 0.02), and last follow-up (p = 0.045 and p = 0.045). The estimated ADA retention rate was 97.1% at 12 months, 87.7% at 24 months, and 82.6% at 36 months, without a statistically significant difference among the groups (p = 0.77).

Conclusions: ADA monotherapy could be equally effective as its combination with MTX in both preventing uveitis relapses and preserving visual acuity in paediatric NIU, with comparable retention rates over 36 months of treatment. The steroid-sparing effect of ADA monotherapy warrants further extensive evaluation to define its optimal placement in the therapeutic strategy for paediatric NIU.

Keywords: adalimumab; methotrexate; monotherapy; noninfectious uveitis; paediatric ophthalmology.

FIGURE 1

Visualisation of the distribution of adalimumab treatment duration in the entire cohort (A) and in the two study groups (B). List of abbreviations: ADA, adalimumab; MTX, methotrexate.

FIGURE 2

Percentage of children with complete, partial, or no effectiveness of adalimumab treatment across the study groups. No statistically significant differences were found between the groups (p = 0.89). List of abbreviations: ADA, adalimumab; MTX, methotrexate.

FIGURE 3

Percentage of children receiving corticosteroids at different timepoints after the start of adalimumab. Percentages are calculated on the total number of children with available information at each timepoint in the two groups. Statistical significance has been evaluated between the baseline and each timepoint. List of abbreviations: ADA, adalimumab; MTX, methotrexate.

FIGURE 4

Adalimumab survival curve for the whole cohort (A) and stratified by study groups (B). The Kaplan–Meier curves display survivorship up to 36 months. Patients with shorter follow‐up durations are censored at their last observation. The number of patients at risk of discontinuation and the number of patients discontinuing adalimumab – for reasons other than remission – are reported at each timepoint. List of abbreviations: ADA, adalimumab; MTX, methotrexate.