THU0532 RITUXIMAB FOR RAPIDLY PROGRESSIVE JUVENILE SYSTEMIC SCLEROSIS
Authors: Roberto Dal Pozzolo, Alessandra Meneghel, Biagio Castaldi, Giovanni Civieri, Giorgia Martini, Renzo Marcolongo, Alida Linda Patrizia Caforio, Francesco Zulian
Journal: Annals of the Rheumatic Diseases (2019)

<h3>Background</h3> Juvenile Systemic Sclerosis (JSSc) is a rare multi-systemic disease characterized by fibrous changes of the skin and internal organs [1]. Patients with "rapidly progressive SSc" usually present rapid development of skin induration and important organ damage [3], leading to poor prognosis [2, 3]. Recently Rituximab (RTX), a monoclonal antibody against the CD20 antigen on B cells, has demonstrated to be a promising therapy for adult patients with SSc [4, 5]. <h3>Objectives</h3> We describe four pediatric patients with rapidly progressive JSSc treated with RTX. <h3>Methods</h3> Data on clinical, laboratory and instrumental parameters were collected from four patients with rapidly progressive JSSc treated with RTX for at least one year. Data were recorded at baseline and every 6 months after initiation of therapy. All patients underwent i.v. RTX therapy with four cycles (375 mg/m<sup>2</sup> every 2 weeks), at 3 months intervals. Oral prednisone (PDN, 0.5 mg/Kg/day) and oral mycophenolate mofetil (MMF, 500 mg/m<sup>2</sup>/day) were administered between RTX pulses. Skin changes were assessed by MRSS, changes on muscles involvement by CMAS. Variations on BMI, pulmonary function tests (FVC, FEV1, DLCO) and cardiac involvement (LVEF, LVEDV, GLS) were expressed as% change from baseline. J4S was used to assess the overall disease severity [6]. <h3>Results</h3> Four JSSc patients (3M, 1F), aged 8-17 years, entered the study. Three patients presented with prevalent cardiological involvement. One patient (Case 3) presented with severe pulmonary involvement. After one year RTX treatment, all patients showed significant decreased of number/duration of Raynaud Phenomenon attacks and 3 patients of cutaneous involvement. Two patients needed an implantable cardioverter defibrillator (ICD) because of episodes of severe ventricular tachycardia (VT). After 12 months of therapy one patient presented improvement LVEF (+19%) and J4S, the other showed a global cardiac improvement (LVEF +37%, LVEDV -18%) and J4S. Both underwent a second year-long treatment with RTX with no worsening of internal organs' involvement. Case 3 showed a significant improvement of the respiratory function (FVC +46%, FEV1 +33%, DLCO +30%) with decrease of J4S. Case 4 improved her arrhythmia and muscle strength (CMAS +17%). No major RTX-related side effects were reported. <h3>Conclusion</h3> Rapidly progressive JSSc still carries a high mortality rate. To the best of our knowledge this is the first case series of patients with JSSc successfully treated with RTX. Our experience, although in a small cohort, confirms the beneficial effect of this therapy on the life-threatening internal organ involvement, particularly on cardiac function. <h3>References</h3> [1] Martini G, et al. Arthritis Rheum 2006; 54(12): 3971. [2] Martini G, et al. Rheumatology (Oxford) 2009;48(2):119. [3] Joven BE, et al. Semin Arthritis Rheum. 2010; 39:285. [4] Daoussis D, et al. Semin Arthritis Rheum 2017; 46 (5): 625-31. [5] Jordan S, et al. Ann Rheum Dis, 2015; 74: 1188-94. [6] La Torre F, et al. Arthritis Rheum. 2012;64(12):4143-50 <h3>Disclosure of Interests</h3> None declared