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

BACKGROUND: Opsoclonus-myoclonus-ataxia syndrome (OMAS) is a rare autoimmune disorder of the nervous system presenting with abnormal eye and limb movements, altered gait, and increased irritability. Two to four percent of children diagnosed with neuroblastoma have neuroblastoma-associated OMAS (NA-OMAS). These children typically present with non-high-risk neuroblastoma that is cured with surgery, with or without chemotherapy. Despite excellent overall survival, patients with NA-OMAS can have significant persistent neurological and developmental issues. OBJECTIVE: This study aimed to describe long-term neurocognitive and adaptive functioning of patients with NA-OMAS treated with multimodal therapy, including intravenous immunoglobulin (IVIG) on Children's Oncology Group (COG) protocol ANBL00P3. METHODS: Of 53 children enrolled on ANBL00P3, 25 submitted evaluable neurocognitive data at diagnosis and at least one additional time point within 2 years and were included in the analyses. Adaptive development was assessed via the Vineland Adaptive Behavior Scale, and validated, age-appropriate measures of intellectual function were also administered. RESULTS: Twenty-one of the 25 patients in this cohort ultimately received IVIG. Descriptive spaghetti plots suggest that this cohort demonstrated stable long-term cognitive functioning and adaptive development over time. This cohort also demonstrated decreased OMAS scores over time consistent with improved OMAS symptoms. CONCLUSIONS: While statistical significance is limited by small sample size and loss to follow-up over 10 years, findings suggest stable long-term cognitive and adaptive functioning over time in this treated cohort.

Developmental and adaptive functioning of very young children with solid tumors and brain tumors.

PURPOSE: Infancy/toddlerhood is a period of rapid development. All infants/toddlers (0-36 months-of-age) undergoing cancer-directed treatment at one hospital are offered developmental assessments and related services. Yet, literature comparing development of infants/toddlers with brain tumors to those with non-CNS solid tumors is sparse. DESIGN AND METHODS: Developmental assessment data were abstracted from electronic health records of infants/toddlers undergoing treatment for a brain tumor (n = 36; mean age = 21.83 ± 9.96 months) or a solid tumor (n = 40; mean age = 17.35 ± 8.50). Z-scores compared obtained data with age expectations. Chi-square analyses assessed whether a greater proportion of participants scored within the clinical range than normative expectations. Multivariate analysis of variance and chi-square analyses compared developmental outcomes between groups. RESULTS: Compared with age expectations, the overall group demonstrated significantly less well-developed skills. Infants/toddlers with solid tumors demonstrated clinical deficits at rates higher than expected for most domains; the rate of impairment for the solid tumor group did not differ significantly from that of the brain tumor group across most subtests. CONCLUSIONS: Like young patients with brain tumors, the developmental functioning of infants/toddlers with solid tumors should be studied across time to determine the trajectory of functioning for these young patients and to inform future developmental intervention studies. PRACTICE IMPLICATIONS: Infants/toddlers with a malignant solid tumor may be at increased risk for delayed development. These very young patients would likely benefit from developmental assessment, early intervention services during and after treatment, and ongoing monitoring of development across time.