Neuroblastoma (NBL) in infants has the potential to regress/mature spontaneously. The literature showed some cases, subjected to initial observation, with reasonable outcome. Deferring/avoiding active treatment was investigated in selected favorable NBL cases. Patients enrolled on the watch and see strategy (W&S) had small primary tumor, localized stages 1 to 2, uncomplicated stage 4s, or stage 3. Tissue biopsy was not mandatory for infants below 6 months with localized mass. On progression, active intervention was indicated according to disease stage and risk after biological characterization. In total, 32 patients were enrolled on W&S strategy; male/female:2.6/1. Twelve had stages 1 to 2, 16 had stage 4s, and 4 were stage 3. Primary adrenal site was reported in 85% patients, and 65% patients had small mass (≤5 cm). Five-year overall and event-free survival were 100% and 80.9±7%, respectively, with a 43-month median follow-up duration. Spontaneous total/near total resolution of mass occurred in 50% patients. Median time to regression was 1.7 months, and 20.7 months until resolution. Only 19% patients witnessed progression; median time to progression was 4.8 months. W&S is a reasonable approach for localized and uncomplicated stages 3 and 4S NBL. Extended tumor size is a newly investigated entity in the present study. All progressive cases were safely rescued with 100% survival outcome.

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.