Neurologic complications after hematopoietic stem cell transplant (HSCT) in children are poorly described, but may contribute to morbidity and mortality. We retrospectively reviewed 261 patients who underwent allogeneic or autologous HSCT, of whom 57 had brain magnetic resonance imaging (MRI) post-transplant, and subsequently identified indications for imaging and determined correlation of findings with neurologic symptoms. Approximately half of imaging studies were done to investigate new neurologic symptoms; of those, 1/3 had acute findings on MRI that correlated with symptoms. Imaging done in patients at risk for neurologic processes but without active neurologic symptoms did not demonstrate acute or actionable findings.

Background: Pediatric patients who undergo hematopoietic cell transplant (HCT) are at risk for neurocognitive impairments; however, long-term studies are lacking. Procedure: Eligible survivors (HCT at age <21y and ≥1y post-HCT) completed a 60-question survey of neurocognitive function and quality of life, which included the Childhood Cancer Survivor Study Neurocognitive Questionnaire (CCSS-NCQ) and the Neuro-Quality of Life Cognitive Function Short Form (Neuro-QoL). Baseline demographic and transplant characteristics were retrieved from the institutional research database. Analyses of risk factors included univariate comparisons and multivariable logistic regression. Results: Participants (n=199, 50.3% female, 53.3% acute leukemia, 87.9% allogeneic transplants) were surveyed at median age of 37.8 years (range 18-61) at survey and median 27.6 years (range 1-46) from transplant. On the CCSS-NCQ, 18.9-32.5% of survivors reported impairments (Z-score >1.28) in task efficiency, memory, emotional regulation, or organization, compared with expected 10% in the general population (all p<0.01). Certain co-morbidities were associated with impaired CCSS-NCQ scores. However, survivors reported average Neuro-QoL (T-score 49.6±0.7) compared with population normative value of 50 (p=0.52). In multivariable regression, impaired Neuro-QoL (T-score <40) was independently associated with hearing issues (OR 4.79, 95% CI 1.91-12.0), history of stroke or seizure (OR 5.22, 95% CI 1.73-15.7), and sleep disturbances (OR 6.90, 95% CI 2.53-18.9). Conclusions: Although long-term survivors of pediatric HCT reported higher rates of impairment in specific neurocognitive domains, cognitive quality of life was perceived as similar to the general population. Subsets of survivors with certain co-morbidities had substantially worse neurocognitive outcomes.

Data for visual acuity (VA) after treatment of neurofibromatosis type 1-associated optic pathway gliomas (NF1-OPGs) is limited. We retrospectively collected VA, converted to logMAR, before and after targeted therapy with everolimus for NF1-OPG, and compared to radiologic outcomes (14/18 with NF1-OPG, 25 eyes [3 without quantifiable vision]). Upon completion of treatment, VA was stable in 19 eyes, improved in 4 eyes, and worse in 2 eyes; visual and radiologic outcome were discordant. In summary, the majority of children with NF1-OPG exhibited stabilization of their VA after everolimus treatment. A larger, prospective study will help delineate visual outcomes after targeted therapy.