UK Multicentre Study of Children with Opsoclonus Myoclonus Syndrome (UMSCOM)
Opsoclonus Myoclonus Syndrome / Posted 2 years ago
Opsoclonus myoclonus syndrome (OMS) is a rare disorder of the nervous system (incidence 1/5 million/year) with onset usually in the second year of life. It presents as jerky movements of the eye (opsoclonus) and body (myoclonus), with ataxia, irritability and sleep disturbance, and is associated with subsequent learning, movement and behavioural problems. About 50% of children with OMS have an underlying neuroblastoma and it seems likely that it is an immune-mediated, sometimes paraneoplastic, condition. Steroids, often supplemented with other immunosuppressants, are the primary treatment but there is limited evidence for drug choice and dosage and little knowledge of the relationship between early symptomatic response and later cognitive outcome. This study will examine drug response of OMS children, with and without NB. 100 children (15 from the UK), recruited over 3 years across 8 European countries, will be treated with an escalating 3-step schedule. All will receive a pulse of dexamethasone (3 consecutive daily doses) at diagnosis and then at a further eleven 4-weekly intervals. Children who fail to show marked benefit after three pulses of dexamethasone will additionally receive cyclophosphamide at each of the next three or six dexamethasone pulses. Those who show inadequate improvement after 3 doses of cyclophosphamide will in its place receive two doses of rituximab at 2-weekly intervals. The primary outcome measure will be remission of OMS symptoms/signs as recorded by the clinician. For each treatment group the final statistic will be percentage of participants in disease remission. Secondary outcome measures will be improvement in OMS symptom score and cognitive and behavioural outcome. A European bio-bank will be established.