The brevity of the email the National Institutes of Health sent Dec. 7, 2010, to investigators in the Management of Myelomeningocele Study trial contrasted with the enormous impact of the message. After seeing results on 183 patients, the NIH was stopping the study on the basis of overwhelming evidence of efficacy of the fetal surgery.
The landmark clinical trial began in 2003 to compare patient outcomes for children with the most severe form of spina bifida who received prenatal versus postnatal surgery to cover the myelomeningocele exposed by the birth defect.
“Fetal surgery was working so well that we did not have to enroll the 200 patients we were scheduled to enroll,” said Lori J. Howell, RN, MS, executive director of the Center for Fetal Diagnosis and Treatment at the Children’s Hospital of Philadelphia, and one of the investigators in the study.
Clinicians and statisticians — independent of the surgical teams and unaware of whether children receive prenatal or postnatal surgery — compiled the results from the prospective, randomized clinical trial. The study, published online by the New England Journal of Medicine in February, found that at 12 months of age, 40% of children in the fetal surgery group had received a shunt compared with 83% in the postnatal group. At 30 months, 42% of children in the prenatal surgery group could walk without assistive devices compared with 21% in the control group.
“In the big view, it offers hope for children with prenatally diagnosed spina bifida,” said lead author N. Scott Adzick, surgeon-in-chief at CHOP and director of the Center for Fetal Diagnosis and Treatment. “In the history of treatment of spina bifida, one of the most common birth defects in the world, this is a huge breakthrough.” While not a cure, fetal surgery can greatly improve the quality of life for children with spina bifida, he said.
Howell understands that well. She has worked with countless children with spina bifida and their families over the course of her three and a half decades in nursing. While neither she nor Adzick knew the results of the MOMS trial until the NIH stopped the study, they knew fetal surgery had proved successful for children treated as early as 1998, well before the start of the study.
They had observed that as the children grew up, some of them were now trying to decide whether to take jazz or ballet classes rather than evaluating which wheelchair to use. “It’s extremely dramatic. It’s probably the most important effort that I’ve been involved with in my professional career,” she said.
Adzick said Howell played an integral role in the study. He describes her as disciplined, caring, and indefatigable. “To do a trial such as the MOMS trial, we had to standardize everything, for both postnatal therapy and prenatal therapy. She was really the crucial person who got that done. You can’t have three different medical centers doing things in three different ways. She helped standardize the medications we used for mothers after the operation to prevent preterm labor. She helped us craft the inclusion criteria and the exclusion criteria for the trial,” Adzick said. “She was the glue that has held the MOMS trial together.”
The multidisciplinary care team involved numerous other nurses. Nurses provided postpartum and antepartum care, assisted in the OR and helped provide counseling to parents. “The nurses were vitally important,” Adzick said. “We couldn’t have done it without them.”
He said that because of the results of the trial, fetal surgery by experienced multidisciplinary teams now can be offered for those diagnosed prenatally with spina bifida, which affects about 1,500 babies born each year. “This is no longer experimental therapy,” Adzick said. “Just recently the national Blue Cross Blue Shield organization has said that as part of their policy, this can be offered as a standard of care.”
Anne Federwisch is a freelance writer.