TY - JOUR
T1 - Long-term efficacy and safety of subcutaneous tocilizumab in clinical trials of polyarticular or systemic juvenile idiopathic arthritis
AU - PRINTO and PRCSG Investigators
AU - Brunner, Hermine I
AU - Ruperto, Nicolino
AU - Ramanan, Athimalaipet V
AU - Horneff, Gerd
AU - Minden, Kirsten
AU - Calvo Penades, Inmaculada
AU - Alexeeva, Ekaterina
AU - Cleary, Gavin
AU - Stern, Sara M
AU - Kone-Paut, Isabelle
AU - Maldonado Velázquez, María Del Rocío
AU - Rabinovich, C Egla
AU - Remesal, Agustin
AU - Silva, Clovis Artur
AU - Nikishina, Irina
AU - Zucchetto, Mauro
AU - Brockwell, Laura
AU - Gordon, Oliver
AU - Nagel, Sandra
AU - De Benedetti, Fabrizio
N1 - © The Author(s) 2024. Published by Oxford University Press on behalf of the British Society for Rheumatology.
PY - 2024/9/1
Y1 - 2024/9/1
N2 - 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 or sJIA).METHODS: Patients with pJIA or sJIA from two open-label, 52-week phase 1b 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 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.CLINICAL TRIAL REGISTRATION: clinicaltrials.gov, NCT02165345.
AB - 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 or sJIA).METHODS: Patients with pJIA or sJIA from two open-label, 52-week phase 1b 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 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.CLINICAL TRIAL REGISTRATION: clinicaltrials.gov, NCT02165345.
KW - Humans
KW - Antibodies, Monoclonal, Humanized/therapeutic use
KW - Arthritis, Juvenile/drug therapy
KW - Child
KW - Female
KW - Male
KW - Treatment Outcome
KW - Injections, Subcutaneous
KW - Adolescent
KW - Child, Preschool
KW - Antirheumatic Agents/therapeutic use
KW - C-Reactive Protein/metabolism
KW - Receptors, Interleukin-6/antagonists & inhibitors
KW - Interleukin-6/antagonists & inhibitors
U2 - 10.1093/rheumatology/keae180
DO - 10.1093/rheumatology/keae180
M3 - Article (Academic Journal)
C2 - 38552315
SN - 1462-0324
VL - 63
SP - 2535
EP - 2546
JO - Rheumatology
JF - Rheumatology
IS - 9
ER -