Autoinflammatory & rare systemic diseases

Long-term safety and efficacy of tocilizumab in adult-onset Still’s disease: open-label, long-term extension of the phase III trial

Rheumatology (Oxford)Published Open-label long-term extension study of a phase III trial

Authors' conclusion

During the observation period, no new safety findings were observed with the long-term use of tocilizumab. Response to tocilizumab was sustained even with an extended dosing interval, with substantial glucocorticoid dose reduction or discontinuation.

Study context

Population
Patients with AOSD who completed the precedent phase III trial of tocilizumab16 completed
Participants
22 patients
Outcome
Safety and tolerability
Methodological detailssingle-group, Mean observation period 168.9 ± 10.8 weeks, Efficacy assessed every 12 weeks
  • Single-group
  • Mean observation period 168.9 ± 10.8 weeks
  • Efficacy assessed every 12 weeks

Main finding

As reported in the abstract

Patients with serious AEs: 3

13.6%

Secondary outcomes

ACR70 response rate · last visit

ACR70 response rate
68.2%

GC dose reduction from start of phase III trial · last visit

Reduction in GC doses
95.2%

GC-free remission · last visit

GC-free remission
40.9%

Tocilizumab dosing interval · final visit

Dosing interval successfully extended
63.6%
Overall mean tocilizumab interval (weeks)
3.4

Laboratory markers (CRP, ferritin)

CRP and ferritin remained well controlled

Safety

Patients with serious AEs: 3
13.6%
Withdrawn from the trial due to serious AEs: 2 patients
No new safety signal detected

Original publication

Long-term safety and efficacy of tocilizumab in adult-onset Still’s disease: open-label, long-term extension of the phase III trial. Rheumatology (Oxford). Published 29 January 2026. doi:10.1093/rheumatology/keag054

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