Children’s of Alabama Leads Practice-Changing Spina Bifida Study

Dr. David Joseph is a pediatric urologist at Children’s of Alabama.

Spina bifida, a condition in which the neural tube doesn’t completely close, is one of the most common congenital malformations, affecting approximately one out of every 2,700 births. While neurosurgeons provide immediate care, it isn’t long before urologists and nephrologists get involved. That’s because damage to the spinal cord and nerves may keep brain signals from reaching the bladder. If that happens, urine can back up into the kidneys, possibly causing kidney damage. “Yet nearly all newborns with spina bifida show normal kidney function at birth,” Children’s of Alabama pediatric urologist David Joseph, M.D., said. “But over time, at least half will deteriorate to some degree.”

Which begs the question: How do you manage these children? A 10-year, nine-center initiative at Children’s of Alabama has been trying to answer that question. Called Urologic Management to Preserve Initial Renal Function Protocol for Young Children with Spina Bifida (UMPIRE), the initiative is designed to provide an evidence-based protocol for testing and monitoring kids with spina bifida to identify early kidney injury. The Centers for Disease Control and Prevention (CDC) funds the study, which is now following more than 500 children with the most severe form of spinal bifida, myelomeningocele, in which a sac of fluid containing part of the spinal cord and nerves protrudes through an opening in the baby’s back.

Unlike most clinical trials, where the outcomes are evaluated at the end, the UMPIRE investigators review the data every quarter and tweak the protocol accordingly. “Obviously, this is not as clean as a randomized control trial,” Joseph said. “But it’s an effective way to manage a small population without a control group.”

This approach has led to some important revelations. For example, the team learned that bringing newborns in for imaging every three months for the first year as they’d been doing had no benefit at the nine-month visit. They also recognized that prophylactic antibiotics to prevent infection weren’t needed in newborns. Children’s chief of pediatric urology Stacy Tanaka, M.D., discovered that each center assessed urodynamics (lower urinary tract function) differently, which was a serious problem given the reliance on those tests to classify a patient’s level of damage and determine treatment.

Finding participants for the study hasn’t been a problem, Joseph said. “I don’t think we’ve had a family in the past seven years that has turned down the opportunity to be in the study,”—something he attributes to Betsy Hopson, MSHA, coordinator of the Children’s of Alabama Comprehensive Spina Bifida Program. The goal is to follow the children for at least 10 years or for as long as the CDC continues to fund the study.

“The urologic community looks to the UMPIRE program for the protocol in anticipation that it will help direct future management,” Joseph said.

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