Ethical statement

The Institutional Review Board, Committee for Protection of Human Subjects of The Children’s Hospital of Philadelphia, approved this study, and all parents or legal guardians gave written informed consent for their children (institutional review board number 2011-007977).

Patient population

Between January 1998 and February 2003, 58 patients met our institutional inclusion criteria described previously and underwent fMMC surgery, with 54 survivors. All infants were subsequently born by cesarean delivery and received standardized neonatal care at our institution. Details of the preoperative evaluation, selection process, surgical approach, and postnatal management have been extensively described elsewhere.

Data collected from maternal prenatal charts, postnatal hospital charts, and follow-up records included gestational age at fetal intervention, anatomical lesion level, and clinical outcomes. Operative reports of shunt placement, if performed, were reviewed to determine timing and indications for shunt placement. Neurosurgical criteria for ventriculoperitoneal shunt placement have been previously described.

Short-term follow-up assessment

As previously reported, families were asked to return for follow-up at 1, 2, 3, and 5 years of adjusted age. Each visit included evaluations by a pediatrician, physical therapist, developmental psychologist, radiologist, neurosurgeon, and urologist. During the first 3 years, neurodevelopmental outcome was assessed by the Bayley Scales of Infant Development-II (BSID, second edition) and the Preschool Language Scales-III for cognitive development.

At 5 years of adjusted age, the Wechsler Preschool and Primary Scale of Intelligence, third edition (WPPSI-III), was administered to assess cognitive function. Overall, 37 (68%) returned for follow-up evaluation at 1 year, 30 (56%) at 2 years, 29 (54%) at 3 years, and 30 (56%) at 5 years. These short-term results were used to evaluate potential correlations between early and long-term outcome.

Long-term follow-up assessment

Given that the most common reasons that precluded fMMC families to return for an onsite neurodevelopmental assessment during the first 5 years of life were travel distance and financial costs combined with our goal to capture long-term information of as many patients possible, we opted to mail out structured questionnaires.

The first questionnaire, designed by the authors, contained questions related to the overall ambulatory status, bladder and bowel function, the need for additional neurosurgical and/or orthopedic intervention, and the need for additional therapy and support (eg, enrollment in physical therapy, occupational therapy, etc).

The overall ambulatory status was classified according the Hoffer Functional Ambulation scale into community ambulators (ie, walks indoors and outside), household ambulators (ie, able to walk indoors only but requires equipment and/or wheelchair for outside mobility), and nonambulatory (ie, wheelchair dependent). The questions required mostly yes/no answers. If questions were answered with yes, we encouraged comments on the age of onset, severity, potential intervention, and follow-up. Families were contacted by phone if additional clarification was required.

In addition to the above-mentioned questionnaire, parents were also asked to complete the BRIEF and the ABAS-II. Both are standardized and validated parental questionnaires to assess executive function and behavioral adaptive skills, respectively.

The BRIEF is a questionnaire for parents of schoolage children (5–18 years) that enables professionals to assess executive function behaviors in the everyday home and school environments. The parent form contains 86 items within 8 theoretically and empirically derived clinical scales that measure different aspects of executive functioning (inhibit, shift, emotional control, initiate, working memory, plan/organize, organization of materials, and monitor) as well as 2 validity scales (inconsistency and negativity). Parents rate each behavior as occurring never, sometimes, or often.

Raw scores for each scale are converted to T-scores (mean, 50, SD, 10). Scores between 60 and 69 fall in the at-risk range, and scores of 70 or higher fall in the clinically significant range. The clinical scales form 2 broader indexes, the Metacognition Index (MCI; summarizes skills that are essential for self-regulation of cognitive processes) and the Behavioral Regulation Index (BRI; represents skills essential for self-regulation of behavior) as well as an overall score, the Global Executive Composite (GEC).

The ABAS-II provides a comprehensive, norm-referenced inventory that assesses aspects of adaptive functioning (the daily, functional skills of an individual) across multiple settings. The ABAS-II questionnaire can be used to assess an individual’s adaptive skills for the diagnosis and classification of disabilities and disorders and identification of strengths and limitations and to document and monitor an individual’s developmental progress over time.

The Parent/Primary Caregiver form is designed for individuals aged 5–21 years, and contains 232 items yielding individual domain scores related to communication, community use, functional academics, home living, health and safety, leisure, self-care, self-direction, social, and work (for individuals with full- or part-time employment), with 21–25 items per skill area. The domain scores are subsequently combined to form index scores of conceptual, social, and practical adaptive skills as well as a total score, called the General Adaptive Composite. These scores are based on a population mean of 100 and a SD of 15.

Statistical analysis

Categorical variables were compared with participants/nonparticipants, ambulation at preschool outcomes, normal bladder/bowel function, and BRIEF/ABAS-II scores, using χ ² or Fisher exact tests. Continuous variables were compared with the same outcomes using 2-sample Student t or Wilcoxon rank sum tests. Univariate logistic regression models were utilized to observe variables of interest by binary BRIEF and ABAS-II outcomes. Finally, χ tests were used to compare observed BRIEF/ABAS-II proportions with specified normal proportions. Data were analyzed using SAS 9.2 statistical software (SAS Institute, Cary, NC).

Patient population

Prior to the MOMS trial, 58 patients underwent fMMC closure at our institution. Four pregnancies were lost because of complications of severe prematurity following delivery for uncontrolled preterm labor at 25, 26, 27, and 27 weeks of gestation, respectively. Overall neonatal survival following fMMC surgery was 93%. Forty-two of the 54 survivors (78%) participated in this study. Median age at follow-up for the study group was 10 years (range, 8–14 years). Ten fMMC children (24%) were below 10 years of age. The remaining 32 (76%) were 10 years old and older at assessment.

In 9 of the 12 nonparticipating families (75%), we were unable to locate a current address and packages returned unopened and marked return to sender. Three families initially agreed to participate but never returned the questionnaires. Overall baseline characteristics and fMMC surgery details for the entire population as well as for the study and nonparticipating group are summarized in Table 1 . There were no statistical differences in time of fetal surgical intervention, MMC lesion level, degree of ventriculomegaly progression, incidence of ventriculoperitoneal shunt placement, and overall infancy ambulatory status between groups. However, mean birthweight was significantly lower in the fMMC children who did not participate compared with those who did ( P = .009). Mean gestational age at delivery tended to be lower for the nonparticipants but did not reach statistical significance ( P = .06).

Long-term follow-up outcome of fMMC participants

At follow-up a total of 33 fMMC children (79%) are community ambulators, 3 (9%) are household ambulators, and 6 (14%) are wheelchair dependent. Thirty-one of the 33 community ambulators (94%) are also able to run and jump without difficulties. Of the 6 wheelchair-bound patients, 2 children were wheelchair bound at the 5 year lower extremity neuromotor function analysis: one because of the development of severe spinal cord tethering at 6 months of age, leading to the loss of lower-extremity function, and the second because of the development of severe intracranial hemorrhage during shunt revision surgery at 18 months of life. The remaining 4 children who are now wheelchair dependent were ambulatory during the preschool assessment (independent ambulation, n = 2; assisted walking, n = 2).

Three children developed spinal cord tethering during their early school years, which resulted in a loss of neuromotor function. One child, able to walk very short distances in the household, opted to use the wheelchair inside and outside for better mobility. Overall, 28 fMMC children (67%) were participating in sports activity, both special needs and typical, ranging from wheelchair basketball and tennis as well as horseback riding to Little League baseball, swimming, ballet, gymnastics, soccer, basketball, skiing, 5 kilometer runs, bike riding, and others.

Ten patients (24%) required at least 1 additional orthopedic procedure including tendon release surgery, scoliosis correction, or hip dysplasia surgery. Per parental report, overall, 14 children (33%) developed spinal cord tethering, and in 8 of those (57%, 20% of total), spinal cord tethering was accompanied by an intradural dermoid cyst. Thus far, 11 required 1 untethering surgery, 2 required 2 surgeries, and 1 required 4 spinal cord release surgeries.

As demonstrated in Table 2 , fMMC children who were able to independently ambulate during preschool age were more likely to be community ambulators at follow-up ( P < .01). Similar to our initial report, children who required untethering surgery with or without the removal of an intradural dermoid cyst were more likely to lose neurofunction and were associated with persistent deterioration of overall ambulatory status ( P = .007).

When parents were asked about the urological and bowel function, 11 (26%) reported that their fMMC children have successfully toilet trained (ie, continent at all times) and 13 (31%) have normal bowel function. Additional pertinent urological and bowel function data are summarized in Table 3 .

Overall, 18 of the study population (43%) required ventriculoperitoneal shunt placement for worsening ventriculomegaly at a mean age of 5.5 ± 3.0 months of age (range, 1-12 months). Eleven of those (61%) required shunt revision during early childhood for infection, obstruction, or both.

Executive functioning at follow-up

Mean T-scores for the BRIEF subscales and indices are summarized in Table 4 . Mean fMMC percentile scores for GEC (64.4 ± 28.7), MCI (68.4 ± 27.2), and BRI (53.0 ± 31.0) were within or slightly above the 95th percentile confidence intervals of age-matched normal (53–59, 61–63, 47–55, respectively), suggesting that children with fMMC are able to successfully complete everyday tasks without or with only minimal difficulties. Nevertheless, although the majority scored within the average range for the GEC, MCI, and BRI, significantly more children who had fMMC surgery had deficits in executive functioning in all three BRIEF indices compared with population norms ( Table 5 ).

The fMMC patients who scored within the normal range for either the BSID-II or the WPPSI-III during the neurodevelopmental assessment at preschool age scored were more likely to have BRIEF-GEC ( P = .003), BRIEF-MCI ( P = .008), and BRIEF-BRI ( P < .0001) scores within the average range, compared with those with early neurodevelopmental deficits.

Adaptive behavior at follow-up

Mean adaptive functioning scores are presented in Table 6 . Although conceptual composite score and social composite score adaptive skills were more likely to follow the expected distribution of population norms, fMMC patients scored significantly lower than the general population means for the general adaptive composite and practical composite scores ( Table 7 ).

Table 6

Mean scores, SD, and ranges for the subdomain and the overall adaptive composite score of the ABAS-II questionnaire

Variables	fMMC children (n = 42)
CCS	91.7 ± 18.5 (49–120)
SCS	97.6 ± 17.3 (53–120)
PCS	81.7 ± 21.4 (40–120)
GACS	86.8 ± 20.3 (40–120)

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