Long-term efficacy and safety of subcutaneous tocilizumab in clinical trials of polyarticular or systemic juvenile idiopathic arthritis.

Objective

To investigate the safety and efficacy of subcutaneous tocilizumab (SC-TCZ) treatment in a long-term extension (LTE) of clinical trials in polyarticular or systemic juvenile idiopathic arthritis (pJIA, sJIA).

Methods

Patients with pJIA or sJIA from two open-label, 52-week phase 1 b core trials of SC-TCZ who had adequate response per investigator assessment entered the LTE and continued SC-TCZ treatment according to body weight-based dosing regimens until commercial availability or up to 5 years. Pharmacokinetics, pharmacodynamics, and efficacy were assessed for up to 3 years and safety for up to 5 years in the LTE.

Results

Forty-four patients with pJIA and 38 patients with sJIA entered the LTE. Tocilizumab trough concentrations were maintained within the range expected to provide clinical benefit (mean values: pJIA, ∼10 μg/ml; sJIA, ∼75 μg/ml over 3 years). Pharmacodynamic parameters (interleukin-6, soluble interleukin-6 receptor, erythrocyte sedimentation rate, C-reactive protein) were maintained throughout the LTE at levels achieved in the core trials. Inactive disease per American College of Rheumatology provisional criteria was reported for 90% (17/19) and 53% (8/15) of patients with pJIA and 91% (10/11) and 92% (12/13) of patients with sJIA in the <30 kg and ≥30 kg body weight groups, respectively. Serious adverse events in the LTE were reported in six patients with pJIA (13.6%; five serious infections) and five patients with sJIA (13.2%; one serious infection).

Conclusion

Patients with pJIA or sJIA experienced long-term disease control with SC-TCZ treatment. Long-term safety was consistent with the known tocilizumab safety profile.

© The Author(s) 2024. Published by Oxford University Press on behalf of the British Society for Rheumatology.

Overview publication

TitleLong-term efficacy and safety of subcutaneous tocilizumab in clinical trials of polyarticular or systemic juvenile idiopathic arthritis.
Date2024-03-29
Issue nameRheumatology (Oxford, England)
Issue numberpubmed:38552315
DOI10.1093/rheumatology/keae180
PubMed38552315
AuthorsBrunner HI, Ruperto N, Ramanan AV, Horneff G, Minden K, Calvo Penades I, Alexeeva E, Cleary G, Stern SM, Kone-Paut I, Maldonado Velázquez MDR, Rabinovich CE, Remesal A, Silva CA, Nikishina I, Zucchetto M, Brockwell L, Gordon O, Nagel S & De Benedetti F
KeywordsAutoinflammatory conditions, Biologic therapies, Juvenile idiopathic arthritis, Paediatric/juvenile rheumatology, Pharmacology, clinical trial, interleukin-6, long-term extension, pharmacovigilance, tocilizumab
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