ABSTRACT
Opsoclonus myoclonus ataxia syndrome (OMAS) is a rare disorder that causes significant neurodevelopmental sequelae in children. Approximately half of pediatric OMAS cases are paraneoplastic, typically associated with localized neuroblastic tumors. Since early persistence or relapse of OMAS symptoms is common even after tumor resection, OMAS relapses may not routinely prompt reevaluation for recurrent tumors. We report a 12-year-old girl with neuroblastic tumor recurrence associated with OMAS relapse a decade after initial treatment. Providers should be aware of tumor recurrence as a trigger for distant OMAS relapse, raising intriguing questions about the role of immune surveillance and control of neuroblastic tumors.
Subject(s)
Opsoclonus-Myoclonus Syndrome , Female , Humans , Child , Opsoclonus-Myoclonus Syndrome/etiology , Opsoclonus-Myoclonus Syndrome/therapy , Neoplasm Recurrence, Local , Ataxia/therapy , Ataxia/complicationsABSTRACT
The coronavirus disease 2019 (COVID-19) crisis confronted us, like many researchers worldwide, with an unforeseen challenge during the final stages of a randomized controlled trial involving ataxia patients. Institutional guidelines suddenly no longer allowed regular follow-up visits to take place, impeding the clinical evaluation of long-term outcomes. Here, we discuss the various scenarios that we considered in response to these imposed restrictions and share our experience of home video recording by dedicated, extensively instructed family members. Albeit somewhat unconventional at first glance, this last resort strategy enabled us to reliably assess the study's primary endpoint at the predefined point in time and hopefully encourages researchers in other ongoing ataxia trials to continue their activities. Remote assessments of ataxia severity may serve as a reasonable substitute in interventional trials beyond the current exceptional situation generated by the COVID-19 pandemic, but will require further investigation.