AB0925 TOCILIZUMAB AS A TREATMENT OPTION FOR PATIENTS WITH JUVENILE SYSTEMIC SCLEROSIS
Authors: Amra Adrovic, Sezgin Sahin, Mehmet Yildiz, Oya Koker, Kenan Barut, Ozgur Kasapcopur
Journal: Annals of the Rheumatic Diseases (2019)

<h3>Background</h3> Juvenile systemic sclerosis (JSS) is a rare autoimmune disease characterized by skin stiffness and fibrosis of internal organs (eg. lung, hearth, gastrointestinal system). The efficacy and safety of tocilizumab (TCZ), an interleukin 6 receptor-α inhibitor, in adult patients have been studied in a few recent studies. Reports on its efficacy among children have been reported among patients with localized disease form. This is a first report on treatment of JSS patients with TCZ. <h3>Objectives</h3> We aimed to evaluate efficacy of tocilizumab as a treatment option for patients with juvenile systemic sclerosis. Consequently, we sought to explore its influences on internal organ involvement, skin stiffness and patient global assessment score. <h3>Methods</h3> We retrospectively evaluated patients diagnosed with JSS according to EULAR criteria. Among 30 patients diagnosed with JSS, 7 (23%) were treated with TCZ. Demographic data are taken from patients' charts. Data on clinical characteristics and treatment outcome are recorded during the patients' last visit. <h3>Results</h3> Seven patients treated with TCZ were analyzed: 6 (85.71%) girls, one boy. Median age of patients was 17.5 years (range:11-12 years), at disease onset 10 years (range 6-15 years) and at diagnosis 11 years (range 7.5-17 years). Median disease duration was 8 years (min. 3-max.11 years). Median duration of TCZ treatment was 10 months (min. 1-max. 21 months). One (14.28%) patient had gastro-intestinal involvement, 4 (57.14%) patients had lung involvement and 2 (28.57%) patients had both gastro-intestinal and lung involvement. Improvement in mRSS was detected in 5 (71.43%) patients. DLCO improved in 4 (57.14%) patients. Four (57.14%) patients had radiologically confirmed improvement in lung findings. Data on radiological findings of one patient treated with TCZ for 6 moths were nor available. Five patients (71.43%) reported improvement in patient global assessment (PGA) score after the TCZ treatment. PGA score of two remaining patients were not applicable due to short treatment duration (Table 1). None of 7 patients had recorded adverse effect of TCZ treatment. <h3>Conclusion</h3> JSS is rare condition characterized with internal organ involvement. Tocilizumab represents an efficacy treatment options for patients unresponsive to standard treatment. Long-term prospective studies with higher number of patients are needed to provide more relevant data. <h3>References</h3> [1] Adrovic A, şahin S, Barut K, Kasapçopur ö. Juvenile Scleroderma: A Referral Center Experience. Arch Rheumatol. 2018;33(3):344-351. [2] Adrovic A, Sahin S, Barut K, Kasapcopur O. Juvenile Scleroderma-What has Changed in the Meantime?Curr Rheumatol Rev. 2018;14:219-225. [3] Arnold MB, Khanna D, Denton CP, et al. Patient acceptable symptom state in scleroderma: results from the tocilizumab compared with placebo trial in active diffuse cutaneous systemic sclerosis. Rheumatology (Oxford). 2018;57(1):152-157. [4] Khanna D, Denton CP, Lin CJF, et al. Safety and efficacy of subcutaneous tocilizumab in systemic sclerosis: results from the open-label period of a phase II randomized controlled trial (faSScinate). Ann Rheum Dis. 2018;77(2):212-220. <h3>Disclosure of Interests</h3> None declared