Multiple sclerosis: treatment

Tuesday, May 31, 2011, 11:30 – 12:30

Safety and efficacy of rituximab in neuromyelitis optica spectrum disorder

M. Radaelli, L. Moiola, D. Privitera, M. Rodegher, B. Colombo, V. Barcella, F. Sangalli, F. Esposito, G. Comi, V. Martinelli (Milan, IT)

Objective: Neuromyelitis Optica (NMO) is an inflammatory demyelinating disease of the central nervous system which generally involves the optic nerve and the spinal cord. The recent discovery of a specific biomarker has brought to consider NMO a “spectrum of disorders “(NMOsd) rather than a single nosological entity. Till now there are no treatment approved for this disease. The aim of our study is to evaluate the safety and efficacy of Rituximab (RTX) in patients with NMOsd
Methods: We retrospectively evaluated patients affected by NMOsd treated with at least one cycle of i.v. Rituximab and with at least 6 months of follow-up.
Results: 17 patients (14 females) were treated with i.v. Rituximab between June 2006 and July 2009. Eight patients were affected by NMO and nine by recurrent longitudinally extensive transverse myelites (rLETM). Mean EDSS and ARR at baseline were 5,5 and 2,6, respectively. RTX was used as first line therapy in only two patients. Twelve out the 15 patients received at least two courses of RTX. Serious adverse events which led to drug discontinuation were reported by two patients: the first one, after the third cycle of RTX developed recurrent episodes of pneumonia and persistent leukopenia; she died for septicemia. The second patient after the first course of RTX developed a severe bedsore which required hospitalization. Another patient developed persistent leukopenia and hypogammaglobulinemia after the second course of RTX. Infusion related adverse events were observed in only one patient. At a mean follow-up of 28 months the EDSS was stable or improved in 14 patients. Ten patients were relapse-free. Other four patients presented only one relapse in concomitance of an increase of CD20 B cell count and achieved further remission after retreatment with RTX. We observed a significant decrease of ARR from 2,2 to 0,5 (p< 0,006).
Discussion and conclusion: Rituximab has shown a good safety profile in our study. Severe infective adverse events were presented only by two patients with high disability. We observed a significant ARR reduction after Rituximab treatment and an EDSS improvement or stabilization was achieved by most of the patients. Our study suggests that Rituximab can be a promising therapeutical option in patients affected by NMOsd. Larger randomized clinical trials with a longer follow-up are needed to confirm our results.

Read More: Neuromyelitis Optica Spectrum Disorder: the importance of the NMO IgG in clinical practice