gms | German Medical Science

Introduction: Tofacitinib was recently approved for juvenile idiopathic arthritis (JIA). To date only limited longitudinal ‚real-life‘ data was reported. We describe our experience including analyses if treatment response had associations to pretreatment or time of start.

Methods: We performed an internal search for patients with JIA with tofacitinib therapy at any time to 30/11/2023. Demographic, clinical and laboratory features were collated. Differences in disease activity (DA, JADAS-10, CRP, ESR), treatment duration and frequency of therapy change due to side effects (SE) or ineffectivity were analysed in patients with early (<48 months since JIA diagnosis) versus late (>48 months) Tofacitinib begin.

Results: 50 patients (86% female, 88% ANA-positive and 12% HLA-B27 positive) with predominantly polyarticular JIA, diagnosed at age 7.2 years (median, IQR 8.4) were identified. Visits at 3–4 (T1), 5–9 (T2) and 10–14 (T3, n=20) months were evaluated. Tofacitinib was started at a median 7.2 (IQR 7.4) years. The demographic, clinical, laboratory and tofacitinib-specific outcomes are summarised in Table 1 [Tab. 1]. Age at tofacitinib start was 15.4 (median, IQR 6.4) years, and duration of therapy 13 (IQR 15) months, which did not significantly differ between the subgroups. At tofacitinib start, 44% had concomittant methotrexate and 34% systemic corticosteroid therapy. Under tofacitinib, decrease in JADAS-10 (and achievement of minimal disease activity), CRP and ESR at each follow-up did not differ significantly according to early or late begin, or if few (<1) or many (>2) biological therapies were previously used. Only one patient stopped therapy due to side effects, and one due to personal wish. However, 30% of patients discontinued therapy, which was not associated with the time of tofacitinib start or previous number of BMARD therapies.

Conclusion: In this cohort, Tofacitinib was well tolerated, however the rate of discontinuation was high but reflected previously reported rates, and was mainly discontinued due to ineffectiveness. CRP, ESR, JADAS-10 and discontinuation did not differ when analysed according to early or late tofacitinib begin. However, only 20 patients were followed up to T3. Continued follow-up is therefore recommended.