AB0743 SINGLE CENTER EXPERIENCE OF BIOLOGICAL THERAPY IN PATIENTS WITH JUVENILE SYSTEMIC SCLERODERMA
Authors: D. Alexseev, I. Nikishina, L. Ananieva
Journal: Annals of the Rheumatic Diseases (2021)

Background: Juvenile systemic scleroderma (jSSС) is a rare multisystem disease with a poor prognosis. The current therapy is imperfect. Biologics, as a modern efficient option for treatment of rheumatic diseases don`t have official indication for jSSС, but it seems to be useful in severe cases, especially lung and heart damage. Objectives: To analyze the existing experience of Biologics therapy in children with jSSС in single center in order to improve the understanding of the indications for use and the results of using these drugs. Methods: The retrospective study include 9 patients with jSSC with severe course of disease, 7 of them were girls. Results: Among this group of patients the age at disease onset was 9.1 years (min-max 1.1-15.1). Clinical manifestations: Raynaud’s syndrome (n = 9), ulcers (n = 3), skin changes (n = 9), joints (n = 8), muscles (n = 4), interstitial lungs disease (n = 6), heart (n = 3), esophagus (n = 5), calcification (n = 3). The main therapy prior to the use of biological agents: glucocorticoids (GC) (n = 9), cyclophosphamide (CF) (n = 2), hydroxychloroquine (HC) (n = 5), therapy for the microcirculation improvement. Biologics were prescribed in the case of insufficient effectiveness of previously conducted therapy, primarily in relation to lung damage (interstitial disease) and heart (with reduced contractility, arrhythmia), as well as changes in the skin and musculoskeletal system. In addition, the possibility to reduce the dose of GC (n=6), withdrawal of CF (n=2) was taken into account. Our experience consist of follows: rituximab (RTM) (n=7) at a dose of 375 mg/m2 interval 3-6 months, abatacept i.v.(ABT) (n=2) 10 mg/kg every 4 weeks, tocilizumab i.v.(TCZ) (n=2) 8 mg/kg. In all cases Biologics was prescribed not earlier than 1.5 year after disease onset (4.6 years in average). By present most patients continued to use biological therapy; of them - RTM was used for 2.2 (0.5-6.0) years, in 1 patient – was canceled after 6 years due to the loss of efficasy; ABT is used in 2 children for 0.5 and 3.6 years, TCZ - in 2 children for 0.3 (withdrawal) and 2.0 years (continues to use)., TCZ was prescribed in 1 patient after 6 years of RTM use. In 1 patient TCZ was withdrawal due to an infusion reaction, and ABT was prescribed. As a result of the therapy, it has been found to improve habitus, quality of life and well-being, reduce skin tightening, improve joint function; no progression of changes in the lungs according to CT and functional tests (FVC, DLCO), improvement in cardiac lesions. It seems that disease improvement became notable at least 3-6 months after the Biologics start. In most patients there were no serious adverse events (AE). No significant infections have been reported. But we met an unexpected case of substantial increasing of neutrophil’s count just after each TCZ infusions up to 21,000 and reversed by following infusions for a one year of treatment from 2 years of total duration of using. Conclusion: Our data shown that Biologics could be efficient and well-tolerated option for severe and resistant to «traditional» therapy course of jSSC. Because this kind of condition is extremely rare further multicenter study is needed. Disclosure of Interests: None declared