Neurodevelopmental Implications on Urological Self-management Among People Living With Spina Bifida: A Practical Guide for Urology Providers.

Although folic acid fortification and advances in prenatal repair have reduced Spina Bifida (SB) prevalence and the severity of comorbidities, individuals with SB remain at elevated risk for neurocognitive impairments that studies have shown can negatively impact, among other things, urological self-care. Identifying and addressing these impairments with practical interventions can meaningfully improve long-term outcomes for individuals with SB. We review neurocognitive impairments associated with SB and provide practical solutions to support improvement of long-term urological outcomes.

Improving Infant Hydrocephalus Outcomes in Uganda: A Longitudinal Prospective Study Protocol for Predicting Developmental Outcomes and Identifying Patients at Risk for Early Treatment Failure after ETV/CPC.

Infant hydrocephalus poses a severe global health burden; 80% of cases occur in the developing world where patients have limited access to neurosurgical care. Surgical treatment combining endoscopic third ventriculostomy and choroid plexus cauterization (ETV/CPC), first practiced at CURE Children's Hospital of Uganda (CCHU), is as effective as standard ventriculoperitoneal shunt (VPS) placement while requiring fewer resources and less post-operative care. Although treatment focuses on controlling ventricle size, this has little association with treatment failure or long-term outcome. This study aims to monitor the progression of hydrocephalus and treatment response, and investigate the association between cerebral physiology, brain growth, and neurodevelopmental outcomes following surgery. We will enroll 300 infants admitted to CCHU for treatment. All patients will receive pre/post-operative measurements of cerebral tissue oxygenation (SO2), cerebral blood flow (CBF), and cerebral metabolic rate of oxygen consumption (CMRO2) using frequency-domain near-infrared combined with diffuse correlation spectroscopies (FDNIRS-DCS). Infants will also receive brain imaging, to monitor tissue/ventricle volume, and neurodevelopmental assessments until two years of age. This study will provide a foundation for implementing cerebral physiological monitoring to establish evidence-based guidelines for hydrocephalus treatment. This paper outlines the protocol, clinical workflow, data management, and analysis plan of this international, multi-center trial.

Neuropsychological care guidelines for people with spina bifida.

While the neuropsychological profile for individuals with Spina Bifida (SB) can vary, often certain patterns of strengths and weaknesses are evident across the lifespan. Understanding variability related to neural structure, genetics, ethnicity, and the environment is key to understanding individual differences in outcomes and can be vital in planning interventions and tracking progress. This article outlines the SB Guideline for the Neuropsychological Care of People with Spina Bifida from the 2018 Spina Bifida Association's Fourth Edition of the Guidelines for the Care of People with Spina Bifida and acknowledges that further research in SB neurocognitive profiles is warranted.

Validation of a bowel dysfunction instrument for adolescents with spina bifida.

INTRODUCTION: Existing survey instruments for bowel dysfunction in the pediatric population are either parent-reported or focus on non-neurogenic bowel dysfunction. OBJECTIVE: The purpose of this study was to develop and validate an adolescent-reported survey to assess the severity of bowel dysfunction in spina bifida patients and examine its impact on quality of life (QOL). STUDY DESIGN: We performed a cross-sectional study of patients in our Myelodysplasia Program, aged 11-17 years, with a history of constipation and/or fecal incontinence (FI) from November 2010 to June 2013. Control patients, aged 11-17 years, were recruited from the stone clinic. Exclusion criteria were lack of English fluency, insufficient reading skills, or an incontinent fecal diversion. A 29-item version of the Adolescent Fecal Incontinence and Constipation Symptom Index (A-FICSI) was developed with five domains (Figure). Test re-test reliability and correlation with the total global health-related QOL score from the Parkin survey were measured using the Pearson correlation coefficient. A factor analysis model with four-fold correlations was tested. RESULTS: Of the 65 study-eligible individuals approached, 25 (11 boys) completed the A-FICSI (median age 12.6 years, IQR 11.8-14.7 years) and 17 completed another survey on urinary incontinence (64.6% response rate). Twenty-one control patients with nephrolithiasis completed the A-FICSI. Nine of the 25 completed a second administration of the survey. The mean correlation between repeated administrations of the survey was r = 0.43. There was a significant negative correlation between severity of constipation (r = -0.299, p < 0.05) and severity of FI (r = -0.316, p < 0.05) with the total Parkin QOL score. The Comparative Fit Index (CFI) was 0.864. Most items loaded significantly on their respective factors. Between-factor correlations were all significant (>0.30) in the predicted direction. Unstandardized residuals were 8.7% (95% CI 6.4-10.9%). Item reduction was performed on the 29-item instrument based on results of the factor analysis. The finalized instrument contained 21 items. DISCUSSION: This is the first adolescent-reported bowel dysfunction instrument to undergo formal psychometric assessment in the spina bifida population. The instrument demonstrated adequate reliability and the five-factor structure fit the data well. This study highlights the negative impact of bowel dysfunction on the QOL of spina bifida patients. It is limited by the moderate sample size that is a common issue in relatively rare diseases. CONCLUSION: The A-FICSI possesses desirable psychometric properties for the measurement of bowel dysfunction in the spina bifida population.

Neuropsychological outcomes of standard risk and high risk patients treated for acute lymphoblastic leukemia on Dana-Farber ALL consortium protocol 95-01 at 5 years post-diagnosis.

BACKGROUND: Children treated for acute lymphoblastic leukemia (ALL) as High Risk (HR) patients may be more vulnerable to neurocognitive late effects because of the greater intensity of their therapy. We compared neuropsychological outcomes in children treated for Standard Risk (SR) or HR ALL on Dana-Farber Cancer Institute (DFCI) Consortium ALL Protocol 95-01. We also evaluated their performance relative to normative expectations. PROCEDURE: Between 1996 and 2000, 498 children with newly diagnosed ALL were treated on Protocol 95-01, 298 of whom were eligible for neuropsychological follow-up. A feature of this protocol was modification of risk group criteria to treat more children as SR rather than HR patients, intended to minimize toxicities. Testing was completed at a median of 5.3 years post-diagnosis for 211 patients (70.8%; ages 6-25 years; 45.5% male; 40% HR), all of whom were in continuous complete remission. RESULTS: Test scores for both groups were generally at or above normative expectation, with the exception of verbal working memory, processing complex visual information, and parent ratings of metacognitive skills. After adjusting for covariates, the SR group performed better on measures of IQ and academic achievement, working memory and visual learning. Effect sizes, however, were only in the small to moderate range. CONCLUSIONS: HR patients exhibited neuropsychological deficits relative to SR patients, though the differences were modest in degree. Modification of the risk group criteria to treat more children on the SR protocol therefore likely afforded some benefit in terms of neurocognitive late effects.

Neurobehavioral side effects of corticosteroids during active treatment for acute lymphoblastic leukemia in children are age-dependent: report from Dana-Farber Cancer Institute ALL Consortium Protocol 00-01.

BACKGROUND: Although corticosteroids remain a mainstay of treatment for acute lymphoblastic leukemia (ALL), they can cause troublesome neurobehavioral changes during active treatment, especially in young children. We evaluated acute neurobehavioral side effects of corticosteroid therapy in preschool versus school-age children by obtaining structured reports weekly for 1 month. PROCEDURE: Parents of 62 children (2-17 years) treated on Dana-Farber Cancer Institute (DFCI) ALL Consortium Protocol 00-01 participated during the continuation phase of treatment. Patients received cyclical twice-daily 5-day courses of prednisone (PRED; 40 mg/m(2) /day) or dexamethasone (DEX; 6 mg/m(2) /day). Parents completed behavior rating scales about their child weekly during one steroid cycle [baseline (Day 0), active steroid (Day 7), post-steroid (Days 14 and 21)]. RESULTS: Behavioral side effects increased significantly (P < 0.001) during the steroid week for preschool children (<6 years) on measures of emotional control, mood, behavior regulation, and executive functions, returning to baseline during the two "off-steroid" weeks. In contrast, school-age children (≥ 6 years) did not demonstrate an increase in side effects during the steroid week. Steroid type (PRED vs. DEX) was not a significant predictor of neurobehavioral side effects. CONCLUSIONS: Preschool children are at greater risk for neurobehavioral side effects during active steroid treatment for ALL than school-age children and adolescents. DEX was not associated with more neurobehavioral side effects than PRED. Counseling of families about side-effects should be adapted according to age. The observed effects, moreover, were transient, reducing concerns about longer-term neurobehavioral toxicities.