4CPS-190 Rituximab use in children, a single hospital experience

Background Rituximab is a monoclonal antibody directed against the CD20 antigen, expressed on the surface of B-lymphocytes, promoting the lyses of the cells. It is labelled for adult different indications, non-Hodgkin’s lymphoma, chronic lymphocytic leukaemia (CLL), rheumatoid arthritis and granulomatosis with polyangiitis and microscopic polyangiitis. Nowadays it is commonly used as an off-label treatment for many other diseases, including some paediatric disorders. Purpose To describe the patterns of rituximab use in a paediatric teaching hospital. Material and methods We conducted a retrospective observational study involving all patients treated in a paediatric hospital with rituximab from January 2001 to June 2018. Clinical data were collected from electronic patients’ medical records, including: patient age, prescribing services and indication. Results The study comprised 145 patients (39% males) with a median age of 15.4 years. The principal indications according to the prescribing services were: Forty–seven patients of the nephrology unit: resistant or refractory nephrotic syndrome (34) and transplants–rejects (13). Forty patients of the oncology unit: non–Hodgkin lymphoma (23), syndrome opsoclonus–myoclonus in neuroblastoma (14) and others (three). Twenty–five patients of the haematology unit: disease: haemolytic anaemia (11), leukaemia (four), haemophagocytic syndrome (four), thrombocytopenic purpura (two) and others (four). Thirteen patients of the rheumatologic diseases unit: juvenile idiopathic arthritis (four), systemic lupus erythematosus (four), vasculitis (two) and others (three). Twelve patients of the neurology unit: autoimmune encephalitis (nine), post–Herpes Simplex encephalitis (two) and others (one). Seven patients of the infectious unit: Epstein–Barr virus infection (seven). One dermatologic disease: Steven–Johnson disease (one). No unexpected side effects were observed outside those reported in the summary characteristics of the product. Conclusion In paediatrics, rituximab treatment is prescribed for off-label indications. Our study shows that rituximab is used in a wide variability of disorders, where the renal disease, specifically the nephrotic syndrome, is the most common indication as a second-line treatment. Although the utilisation of rituximab increases every year and some uses are well described, further studies for some indications are necessary to establish a correct safety and efficacy profile in children. References and/or acknowledgements To the pharmacy, oncology, haematology, immunology and nephrology staff. No conflict of interest.