Fetal Surgery Is Changing Lives for Kids With Spina Bifida
Spina bifida is a diagnosis no parents-to-be want to hear as they await their child's birth, and the idea of performing surgery on a baby while it is still in the womb can be terrifying. But new research shows that performing the delicate procedure before the baby is born, and not after, is worth it.
The findings showed that children with myelomeningocele (the most severe form of spina bifida) who had surgery while in utero were more likely to later be able to walk independently and go up and down stairs than children who had the surgery after they were born. Their leg muscles were stronger and they could walk faster. They also were likely to be able to do self-care tasks for themselves, including brushing their teeth, holding a fork and washing their hands.
"First of all, it's a durable finding that the need for a ventriculoperitoneal [brain] shunt to treat hydrocephalus is much, much less in the fetal surgery group compared to the conventional postnatal surgery group," said study co-author Dr. N. Scott Adzick. He is surgeon-in-chief at Children's Hospital of Philadelphia and director of the hospital's Center for Fetal Diagnosis and Treatment.
"Secondly, that motor function is not only durable at 1 and 2½ years of age, but also in the school-age group, 5 to 10 [years]. In that group, there are many more patients who can walk or have better motor function than those who are treated after their birth," Adzick added.
Not only that, but upper extremity coordination was nearly normal in the fetal surgery group, where it was not in the postnatal surgery group, Adzick said.
Spina bifida is a birth defect in which an area of the spinal column doesn't form properly. In severe cases, a section of the spinal cord and the spinal nerves are exposed through an opening in the back. This can lead to weakness or paralysis, an inability to walk without assistance and loss of bladder control and other motor skills. It affects one in 1,500 births.
Though there were previously raised concerns that the advantages of the fetal surgery might decrease over time, that doesn't appear to be the case, Adzick said. Instead, there appears to be a long-term benefit from neural protection in utero, he added.
The knowledge around the efficacy of fetal surgery has grown tremendously since the early days of that treatment, about four decades ago. Almost 10 years ago, researchers released data from what is known as MOMS (Management of Myelomeningocele Study). A randomized clinical trial, it compared the skills of children aged 1 to 2½ who had surgery while in utero with those who hadn't had surgery until after they were born. Children from 183 families participated in this first phase.
The latest results are from MOMS2, in which researchers focused on motor skills and neurocognitive outcomes from the children at 5 to 10 years of age. The second phase included 78 children with postnatal repair and 76 with prenatal repair.
Nearly 91% of the fetal surgery group performed age-appropriate self-care, the researchers said, compared to 85.5% of the postnatal group. They were 70% more likely to walk independently, at higher speeds and with better walking style. They were more than twice as likely to walk without braces on their legs. They also were six times more likely to go to the bathroom on their own.
About two in three fetuses with spina bifida are not candidates for the fetal surgery, sometimes because of other fetal abnormalities or serious maternal health or psychosocial concerns, Adzick said.
In the original MOMS trial, three centers were involved, but those teams have taught many others. There are now about 30 fetal surgery centers in North America.
"That's the way ... for these rare conditions, we can pool information and ideas to lend scale, so we can really help families in the future," Adzick explained.
The study was published online Feb. 8 in JAMA Pediatrics. Adzick is planning a MOMS3 study, which will follow the children to ages 15 to 18.
Dr. Tim Brei, medical director for the Spina Bifida Association (SBA) and a developmental pediatrician at Seattle Children's Hospital, said the improved mobility shown in the study may make engaging in community activities easier for families.
Brei shared excitement on behalf of SBA that research is providing better clarity on long-term outcomes, but also cautioned that good tracking of cases and outcomes needed to continue as surgery techniques evolve and more surgery centers perform fetal surgery.
"There is still much more we don't know about spina bifida and improving care, or improving treatments, than we do," Brei said.
While some outcomes may be better as a result of surgery, others may be unchanged, noted Brei, who was born with spina bifida.
"I do want families to know that while it's an option, women should not feel guilty if for some reason they choose not to do fetal surgery," Brei said. "There are lots of decisions that go into that for any given family or individual."
The U.S. Centers for Disease Control and Prevention has more information on spina bifida.
SOURCES: N. Scott Adzick, MD, surgeon-in-chief, Children's Hospital of Philadelphia (CHOP) and director, CHOP Center for Fetal Diagnosis and Treatment, Philadelphia; Tim Brei, MD, medical director, Spina Bifida Association, and developmental pediatrician, Seattle Children's Hospital; JAMA Pediatrics, Feb. 8, 2021, online