Background Rituximab is a chimeric anti-CD20 antibody approved in a few indications apart from oncology (rheumatoid arthritis, polyangiitis and Wegener’s granulomatosis). The French funding system allows the off-label use of rituximab if the therapeutic indication is argued by the prescriber (scientific societies’ guidelines, case series, case reports, patient’s clinical data). This information is discussed with hospital pharmacists before the therapeutic decision, on a case to case basis.

Purpose The aim of this study was to analyse the off-label use of rituximab, outside of oncology indications, in a French university hospital.

Material and methods A retrospective study of rituximab prescriptions was performed between 1 December 2015 and 1 June 2016 by extracting data from computerised physician order entry and pharmacy management software (Pharma, Computer Engineering). Off-label prescriptions were analysed through literature analysis and clinical evidence provided by physicians. In parallel, clinical trials involving off-label use of rituximab in progress in France were identified from the European Union clinical trials register.

Results Between 1 December 2015 and 1 June 2016, 188 patients were treated with rituximab for indications apart from oncology (286 courses). 175 (93%) patients were treated for off-label indications of this drug (264 courses). For 128 patients (68%), medical justification was documented, with pertinent bibliography in 98% of cases. The most common indications were multiple sclerosis (83 patients; 50%) and neuromyelitis optica (23 patients; 13%). In total, 26 off-label indications were used during this period. 14 clinical trials were in progress in France including 7 for those indications. None of the patients studied here was included in a clinical trial.

Conclusion Rituximab is an innovative drug with restricted approved indications, besides oncology. In this context, in our university hospital, we observed wide use (more than 90%) of rituximab off-label, especially in multiple sclerosis. The process of drug delivery applied in our hospital enables follow-up of a massive cohort of patients treated with rituximab off-label, providing real life data on efficacy and toxicity. The introduction of temporary use recommendations approved by the French Health Agency could enable this data collection, facilitating approval of new indications.

References and/or acknowledgements IGAS, 2012‘Evaluation du dispositif de financement des médicament en sus’.

No conflict of interest