LAUSR

Juvenile dermatomyositis (JDM) is the most common idiopathic inflammatory myopathy (IIM) of childhood, but the involvement of muscle also can be complication of systemic lupus erythematosus (SLE), mixed connective tissue disease (MCTD) and different overlap syndromes. The presence of myopathy can significantly affect the prognosis of the disease and the quality of life. Despite of the absence of official indication for biological therapy fo IIM, it`s may be needed in severe course of disease.To analyze in retrospective study the efficacy and safety of Biologics (B) in patients (pts) with IIM in our pediatric rheumatology center.The study included all pts with rheumatic diseases (RD), who had IIM and received B during past 10 years.A total of 17 pts (10 females) were identified: 23.5% with JDM, 11.8% - SLE, 35.3% - MCTD, 29.4% - overlap syndromes. The median age at the onset of RD was 9.75 years [interquartile range (IQR) 6.9; 13.5]. The median disease duration at the onset of myopathy was 7.0 months [3.0; 10.0], in all pts with JDM – at onset. All pts had myopathic syndrome. The rash tipical for JDM was in 8 pts (47%). The lung involvement observed in 6 pts (35.3%). Levels of serum muscle enzymes (CK, ALT, AST and lactate dehydrogenase [LDH]) were elevated in 88.2% of pts: CK in 52.9% (mean ± std 624.35±1007.2 U/L), ALT – 82.4% (mean ± std 142.18±148.16U/L), AST – 82.4% (mean ± std 159.6±201.1 U/L), LDH – 88.2% (mean ± std 492.5±229.2 U/L). Abnormal EMG findings were in 10 pts (59.4%). Nailfold capillaroscopic changes tipical for IIM had 10 pts (59.4%). Before B 94.1% of pts received corticosteroids (CS), 58.8% - methotrexate, 29.4% - hydroxychloroquine, 17.6% - cyclophosphamide, 11.8% - cyclosporine, 5.9% - mycophenolate mofetil, 5.9% - azathioprine, 47% - IVIG. B therapy was started due to insufficient efficacy of previous therapy. The median age at start of B was 12.3 years [IQR 9.5; 15.0]. The median disease duration prior to treatment with B was 2.25 years [IQR 0,8; 3.6]. 58.8% of pts received rituximab (RTX), 41.2% - abatacept (ABA). The median time between each course of RTX was 182 days [IQR 156–315]. 80% of pts underwent more than one course of RTM therapy, with a maximum of 5 courses. The median duration of ABA therapy was 2.8 years [IQR 1.6; 5.0]. Discontinuation of B due to serious AE was observed in 1 patient with overlap syndrome (5.9%) – macrophage activation syndrome (MAS) 8 day after RTX infusion (5th course, 1st infusion). One patient (female, 12.3 yo) died from MAS (at onset of SLE, developed before B), the others achieved remission. B therapy allowed to reduce the dose of CS to maintenance in all pts, decrease of calcinosis in 2 pts and improve the quality of life.Our study demonstrated that B is highly effective in children with IIM and have the acceptable safety. Early diagnosis and initiation of intensive therapy are important in reducing symptoms of myopathy in RD. It seems that the development of IIM in other RDs, not JDM, indicates the need for B and its good efficacy. The further extended studies are needed.None declared