Prenatal surgery promises brighter future for babies with spina bifida
One year after becoming a standard of care at The Children’s Hospital of Philadelphia, fetal surgery for spina bifida is helping families from all over the United States
The Mulligans’ baby had myelomeningocele (MMC), the most common, yet most severe form of spina bifida, in which an opening in the back exposes the fetus’ developing spinal cord to progressive damage. They were told that a baby with this condition, even with surgery after birth, would likely face lifelong disabilities, including paralysis and cognitive impairment. But the Mulligans found hope in what was at the time an experimental fetal surgery available at The Center for Fetal Diagnosis and Treatment at The Children’s Hospital of Philadelphia (CHOP), an internationally recognized leader in fetal surgery and fetal care.
Led by Dr. N. Scott Adzick, surgeon-in-chief of CHOP and director of the Center, team members had pioneered a surgical procedure to repair MMC before birth and had been performing it at CHOP since 1998. The team’s nearly 30 years of work in the laboratory and with patients strongly suggested that operating on the baby in the womb, months before birth, could reduce the need to divert fluid from the brain, improve neurologic function and increase the likelihood that a child would be able to walk independently.
The Mulligans contacted CHOP, and after extensive testing and counseling, Katherine became the 29th patient for prenatal spina bifida repair at the center. For the surgery, a team of specialized doctors worked together to operate on the baby in the uterus. The spinal lesion was repaired and surgeons closed the skin to protect the spinal cord from additional exposure to the amniotic fluid. On Dec. 28, 2000 – his father’s birthday – Sean Mulligan was born, weighing a healthy six pounds, seven ounces, with nothing more than a scar where his MMC lesion had been. “He came out kicking and screaming, so to speak, and he’s been doing that ever since,” Katherine says.
Sean’s case was one of 58 fetal surgeries for spina bifida the CHOP center performed between 1998 and 2003. Their research helped lay the groundwork for the seven-year federally funded Management of Myelomeningocele Study (MOMS), a nationwide, multi-center, randomized prospective clinical trial, that compared prenatal surgery to postnatal surgery. Although the goal was to enroll 200 patients, the National Institutes of Health ended the trial in December 2010, after 183 surgeries because, by that point, the effectiveness of prenatal spina bifida surgery had been conclusively established. In February 2011 the results of the MOMS trial were published in the “New England Journal of Medicine” and fetal surgery for spina bifida became a standard of care at CHOP.
One year after the study’s publication, CHOP’s center had received more than 200 fetal spina bifida referrals from all over the world, and performed more than 100 evaluations and nearly 30 prenatal spina bifida surgeries. The team currently performs about three fetal surgeries for spina bifida each month.
“What was once just an idea is now a standard of care,” says Adzick, “The path to get here has been long and fraught with extraordinary challenges. But the beauty of medicine is that, with determination and perseverance, we can change and improve practice to offer new hope.”
Today, Sean is a healthy 11-year-old. He excels in school, and participates in Cub Scouts, soccer, basketball, tennis and swimming. He loves to play baseball and hang out with his three younger brothers. Sean’s progress is followed yearly through a spina bifida clinic near his home. He has never needed a shunt and his bowel and bladder function are good. Because of this important medical research and surgical advances, more children now have access to the fetal surgery for spina bifida that Sean received – and all the possibilities it provides.