Investigators from the Children’s Hospital of Philadelphia sought to evaluate the long-term outcomes in a cohort of patients who have been followed since undergoing in utero repair of myelomeningocele (MMC) at their institution from 1998–2003. Parents completed questionnaires to measure patients’ functional and developmental status. Functional status was assessed by including questions about ambulatory status (classified as community ambulator [walks indoors and outside], household ambulator [able to walk indoors only but requires equipment and/or wheelchair for outside mobility], and nonambulatory [wheelchair dependent]), bladder and bowel function, and need for additional surgeries. Developmental status was assessed by measuring executive functioning using the Behavior Rating Inventory of Executive Function (BRIEF), and behavioral adaptive skills using the Adaptive Behavioral Assessment System II (ABAS-II). BRIEF and ABAS-II scores were categorized as average, borderline, or impaired, and the proportion of patients in each score category was compared to population norms.
Among 54 patients who underwent in utero MMC repair, 42 (78%) were included in analysis. At a median follow-up of 10 years (range, 8–14 years), 79% of participants were community ambulators, 9% household ambulators, and 14% nonambulatory. Normal bladder function was reported in 26% and 31% had normal bowel function. A ventriculoperitoneal shunt was required in 43% of participants, all by 12 months of age.
Although the proportion of participants who had average BRIEF scores was not significantly different than population norms, there were significantly more participants who had borderline and/or impaired BRIEF scores compared to population norms. There were also significantly fewer participants who had an average composite ABAS-II score compared to population norms. The need for a ventriculoperitoneal shunt was associated with lower ABAS-II composite scores.
The authors conclude that in utero MMC repair probably improves functional outcomes but difficulties with adaptive skills and executive functioning in these patients remain considerable.
Dr Iqbal has disclosed no financial relationship relevant to this commentary. This commentary does not contain a discussion of an unapproved/investigative use of a commercial product/device.
The management of myelomeningocele study (MOMS), a prospective, multicenter, randomized trial, demonstrated a significant decrease in the need for ventriculoperitoneal shunting by 12 months of age with in utero repair compared to “standard” postnatal therapy.1 The investigators for the MOMS trial also studied secondary outcomes at 30 months, such as mental development and motor function, all of which favored the fetal repair group.1 This study is important, as the fetal repair of MMC is the first non-life-threatening indication for a fetal surgical procedure that has been shown to dramatically impact the postnatal course.
In most cases of fetal surgery, a life-threatening condition exists for the baby that justifies the maternal risk of the procedure. Utilizing percutaneous or minimally invasive techniques, risk to the mother can often be minimized....