Neuroblastoma-associated Opsoclonous Myoclonous Ataxia Syndrome: Profile and Outcome Report on 15 Egyptian Patients

Abstract

Opsoclonous myoclonous ataxia syndrome (OMAS) is a rare primarily immune-mediated disease in children. The current study aim was to find out the patterns and outcome of OMAS associated with neuroblastoma (NBL) among Children’s Cancer Hospital—Egypt patients. Data was reviewed for 15 eligible patients enrolled between 2007 and 2016. OMAS treatment included prednisolone and cyclophosphamide with/without intravenous immunoglobulin; NBL treatment was given according to risk-corresponding protocol. Patients’ age ranged from 0.75 to 12 years at presentation with male/female: 1.1/1. Concurrent diagnosis of OMAS and NBL occurred in 6 patients (40%). OMAS preceded NBL within 0.25 to 2 years in 33%, while NBL preceded OMAS within 0.5 to 1.5 years in 27%. Full OMAS picture was present in 10/15 patients, while 20% presented with truncal ataxia and myoclonus, 1 with truncal ataxia and opsoclonus, and 1 had opsoclonus and myoclonus. Median time till improvement of manifestations was 5 months. The 5-year OMAS progression-free survival was 33.3%, where 10 patients needed second-line therapy due to relapse/progression of OMAS. The median time to progression was 28 months measured from OMAS diagnosis. All patients remained alive with NBL 5-year overall survival of 100% and event-free survival of 85.7% for. However, 73% of the patients showed late sequelae ranging from ocular to cognitive, behavioral and motor disorders; rarely seizures and hemolytic anemia.

Review of Opsoclonus-Myoclonus Ataxia Syndrome in Pediatric Patients

“…Therefore, timely workup and treatment were especially important for her prognosis. While the symptoms of OMAS may resolve with time and appropriate treatment, neurological, behavioral, and cognitive deficits are common in patients [ 86 , 87 , 88 , 89 ]. Sun et al, in a retrospective review of 14 children with OMAS and neuroblastoma, reported that approximately 93% children had some neurological sequelae, with behavioral changes being the most common, followed by language impairments, motor retardation, and cognitive disorders.…”

Late cognitive and adaptive outcomes of patients with neuroblastoma‐associated opsoclonus‐myoclonus‐ataxia‐syndrome: A report from the Children's Oncology Group

“…10 Given the rarity of this disease, however, the limited research on the neurocognitive functioning of patients with NA-OMAS has been largely based on retrospective studies involving small samples and case reports. [11][12][13][14][15][16][17][18] There have been no prospective trials that included serial assessment of cognitive or psychological outcomes.…”