Background Efficacy and good tolerability of Rituximab (Rtm) in some refractory to standard care systemic autoimmune rheumatic diseases (SARD) has been demonstrated in several registers (1-3). Although, available evidence is not yet sufficient, thus RTM is used as an off label therapy in systemic lupus erythematosus (SLE), systemic sclerosis (SSc), primary SjÖgren's syndrome (pSS) and polydermatomyositis (PDM).
Objectives To evaluate efficacy and tolerability of Rtm in patients with severe SARD when standard of care (SOC) therapy is ineffective or contraindicated, or not tolerated – as it happens in real life clinical setting.
Methods Prospective study included 229 pts with SARD: 97 (42,4%) SLE pts; 50 (21,8%) ANCA-associated systemic vasculitis (SV) pts; 40 (17,5%) SSc pts, 23 (10%)- pSS and 19 (8,3%) - PDM. All pts were administered Rtm and conventional therapy consisting of glucocorticoids (GC) and/or immunosuppressants (IS). Median follow up period after first Rtm administration was 23 mo (1-60). The groups were compared based on response to treatment, classified as: complete response, partial response, no response (1).
Results Totally participating pts received 1-5 Rtm courses during the follow up period. Average Rtm dose received varied from 1,6±0,84 to 3,1±1,75. Improvement was documented in 80,6% pts, complete response was achieved in 50,6% pts and partial response in 30%. The greatest proportion of non-responders was seen in SLE and DMP cohorts (18% and 16%, respectively). GCs were tapered following patients' improvement and/or immunosuppressants were discontinued. Rtm demonstrated accumulation of effect and enhancing efficacy with longer duration of therapy (i.e. repeated courses). In SLE patients repeated Rtm administration allowed to improve the responder's rate, as compared to a single Rtm course (p=0,05). Repeated Rtm course allowed to decrease the relapse rate in ANCA-SV pts (p =0,002). Rtm efficacy in SSc pts inversely correlated with disease duration. Adverse drug reactions (ADRs) were registered in 101 (44%) pts, and the majority of them (73 pts) were coming from SLE and ANCA-SV cohorts. Infectious complications were the most common ADRs, including severe infections (from 2,5% in SSc cohort to 17% in pSS cohort). There were 20 deaths out of 229 participants (8,7%), caused by progression of the disease or infectious complications.
Conclusions Complete or partial response to Rtm therapy was achieved in 80% of SARD patients, refractory to standard of care therapy or having contraindications to SOC. Repeated Rtm courses resulted in sustainable and long term efficacy of therapy allowing to taper or even discontinue conventional therapy agents. Rtm tolerability was satisfactory
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Disclosure of Interest None declared