Successful treatmentof refractory systemic' onset juvenile idiopathic arthritis with tocilizumab: a retrospective analysis of 25 cases

Abstract

Objective To investigate the efficacy and safety of tocilizumab inpatients with refractory systemic'onset juvenile idiopathic arthritis (SoJIA), and to provide a new option for the treatment of this severe disease. Methods We retrospectively studied 25 cases of hospitalized patients with refractory SoJIA treated withtocilizumab, of whom 22 had data that fit for analysis, from May 2005 to February 2016. Data of 22 cases were collected retrospectively from physicians in charge of the patients. Children with SoJIA were treated with nonsteroidal antiinflammatory drugs (NSAIDs), Glucocorticoid (GC), methotrexate, cyclosporin A, etanerceptetc before, but still in high disease activity due to inadequate response were involved. Weretrospective analyzedthe laboratory test results like C' reactive protein(CRP), Erythrocyte sedimentation rate(ESR), Ferritin and other inflammatory index. Improvement of pain,fever, rash, hepatosplenomegaly and lymphadenectasis of active SoJIA (disease course ≥6 months, and inadequate response to NSAIDs and GC) after tocilizumab treatment (Body weight ≥30 kg, 8 mg/kg; Body weight<30 kg, 12 mg/kg, per 4 weeks) were analyzed. Safety data of 22 cases were collected throughout the treatment period including neutropenia, infections, anaphylaxis and elevated liver enzymes etc. We also retrospectively analyzedthe dose change of GC and the long' term effect. Dichtomous paramenters were compared teween groups using the χ2 test. Continuous parameters were compared using the analysis of uariance. Results In comparison to the indices before the treatment, the level of CRP [(8.7±2.2) mg/L vs (111.6±74.4) mg/L, F=5.192, P=0.002], ESR [(6.4±6.3) mm/1 h) vs (65.6±24.3) mm/1 h, F=50.393, P=0.000], white blood cell (WBC) [(8.4±2.5)×109/L vs (17.6±8.6)×109/L, F=9.321, P=0.000], Neutrophil count[(4.9±2.4)×109/L vs (14.4±8.7)×109/L, F=10.541, P=0.000], blood platelet (PLT) [(269.5±79.2)×109/L vs (405.4±145.3)×109/L, F=5.704, P=0.000] and globulin [(19.2±4.1) g/L vs (30.1±3.8) g/L, F=22.896, P=0.000] dec-reased rapidly and hemoglobin [(118.3±9.0) g/L vs (108.5±9.8) g/L, F=4.693, P=0.002] increased significantly at 24 weeks after Tocilizumab (TCZ) treatment. Clinical manifestationssuch as fever, rash, hepatosplenomegaly, joint swelling and pain were significantly improved. GC dose [(1.25±3.8) mg·kg-1·d-1vs (16.2±12.8) mg·kg-1·d-1, F=8.21, P=0.000] were significantly reduced after TCZ treatment (P<0.05); American College of Rheumatology (ACR) Pedi 30/50/70/90 was improved after TCZ treatment. Adverse events occurred in 3 cases of 25 children, who were not included in the statistical analysis group. Conclusion This retrospective case series has demonstrated the efficacy of tocilizumab in SoJIA, low incidence of adverse reactions. Further studies are needed to be developed because this case series haslimited sample size. Key words: Arthritis, juvenile rheumatoid; Treatment; Tocilizumab; Children