Effectiveness and safety of Rituximab in demyelinating diseases spectrum: An Italian experience.

Highlights • Off-label use of Rituximab for Demyelinating disease of the central nervous system is increasing. • No guidelines on intervals and dosages of administration are available. • Disease control was obtained in about 65% of our patients. • Only one patient experienced severe adverse event, that is a severe lymphopenia. • About 25% of our patients were naïve to any treatment. Abstract Background Rituximab (RTX), a monoclonal antibody targeting the CD20+ B lymphocytes, deserves major attention as therapeutic option in the treatment of demyelinating disorders of the central nervous system (DDCNS). We reported our clinical experience with the use of RTX in terms of efficacy and safety in persons suffering from DDCNS. Methods An Italian single-center observational analysis of patients who underwent RTX treatment between 2011 and 2017 was performed at MS center of Catania, Italy. No evidence of disease activity (NEDA) was applied to evaluate the response to RTX. CD19+ and CD20+ counts were collected along therapy. RTX-related adverse events were recorded. Results Eleven patients with MS, four with NMOSD and two with NMO were enrolled. Out of them, 4/17 were naïve to previous treatments. According to NEDA status, 11/17 got NEDA3 status at the follow-up. Six patients had relapses (two had a single relapse and four had multiple relapses). One patient with primary progressive MS and one with relapsing remitting MS stopped RTX, the last one for severe lymphopenia. Conclusions RTX showed efficacy to impact DDCNS worsening with an acceptable safety profile. [ABSTRACT FROM AUTHOR]