Response to rituximab in 3 children with opsoclonus-myoclonus syndrome resistant to conventional treatments

We report the 1 year follow-up of 3 children affected by non-paraneoplastic Opsoclonus-Myoclonus Syndrome (OMS) resistant to conventional therapies (steroids, ACTH and intravenous immunoglobulins) who were treated with an anti CD20 monoclonal antibody (rituximab). Treatment response was recorded on the basis of an international score at 0, 3, 6, 9 and 12 months. Despite the long disease duration and the numerous previously administered treatments, all patients underwent rapid and persistent neurological recovery following rituximab administration, thus suggesting a potential role of this drug even in pre-treated patients.

► Experience with rituximab in opsoclonus-myoclonus-ataxia syndrome is limited.
► We report 1 year follow-up of 3 affected children treated with rituximab.
► Children were previously treated with conventional immunotherapies without efficacy.
► No acute or late effects were reported.
► Persistent neurological recovery followed rituximab administration.