Background/Purpose:

To assess the efficacy and safety of tocilizumab (TCZ) monotherapy for the induction therapy of adult onset Still’s disease (AOSD) in a prospective, single-arm, single-center, cohort, pilot study.

Methods: Eight AOSD patients (male 2, female 6) who had agreed with our prospective trial since April 2010 till May 2015 were enrolled. Patients received 8 mg/kg of intravenous TCZ fortnightly for the first two months (five courses), then monthly for the next 5 months and after that TCZ was discontinued and patients were followed up for another 6 month with careful monitoring of clinical symptoms and signs related to AOSD relapses. In this report, we evaluated the efficacy and safety at the sixth month. Efficacy was evaluated by serum markers (WBC, CRP and serum ferritin), clinical symptoms and ratio of patients who required additional therapy, and safety was evaluated by adverse events for six months.

Results: The mean age was 45.2. Fever, arthralgia, rash and sore throat were observed in 100%(n=8/8), 100%(n=8/8), 87.5%(n=7/8) and 75.0%(n=6/8) respectively. LOCF analysis revealed that WBC, CRP and serum ferritin level decreased significantly from 14075 ± 4732/μl to 7042 ± 2939/μl, from 12.2 ± 7.4 mg/dl to 0.32 ± 0.62mg/dl and from 9176 ± 8077ng/ml to 3380 ± 5615ng/ml in 6 month respectively (each, P<0.01). The improvement rate of fever, arthralgia and eruption were 100%(n=8/8), 75.0%(n=6/8) and 71.4%(n=5/7). Only 2 patients required additional therapy (prednisolone).The reason of cessation consisted of lack of efficacy (25%, n=2) and adverse event (12.5%, n=1). An adverse event was UTI. There were no other significant adverse events.

Conclusion: TCZ monotherapy may be an alternative treatment strategy for AOSD.

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ACR Meeting Abstracts - https://acrabstracts.org/abstract/corticosteroid-free-tocilizumab-monotherapy-for-adult-onset-stills-disease-results-in-six-month/