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Fetal surgery for myelomeningocele: history, research, clinical trials, and future directions
1University of California, Davis Health System, Sacramento, CA, USA - benjamin.keller@ucdmc.ucdavis.edu.
Insights
Prenatal surgery for myelomeningocele (spina bifida) significantly reduces the need for shunting and improves motor function. In utero repair is now the standard of care for eligible patients.
Area of Science:
- Medical Science
- Pediatric Surgery
- Neuroscience
Background:
- Myelomeningocele, or spina bifida, is the most common neural tube defect causing childhood paralysis.
- Traditional postnatal closure aimed to prevent infection and further spinal cord damage.
- Research indicated ongoing damage during gestation, prompting investigation into prenatal interventions.
Purpose of the Study:
- To review the evolution of myelomeningocele treatment from postnatal to prenatal repair.
- To discuss the research and clinical trials supporting in utero surgery for spina bifida.
- To highlight the current standard of care and future research directions.
Main Methods:
- Review of historical treatment standards for myelomeningocele.
- Analysis of early animal research on prenatal defect closure.
- Examination of clinical outcomes from human fetal repair, including the Management of Myelomeningocele Study (MOMS).
Main Results:
- Prenatal repair of myelomeningocele demonstrated improved lower extremity motor function compared to postnatal repair.
- The Management of Myelomeningocele Study (MOMS) showed a decreased need for ventriculoperitoneal shunting in the prenatal repair group.
- In utero surgery has become the standard of care for eligible myelomeningocele cases.
Conclusions:
- Prenatal repair of myelomeningocele offers significant benefits over traditional postnatal closure.
- The shift to in utero surgery represents a major advancement in managing spina bifida.
- Continued research is crucial for optimizing outcomes and exploring future therapeutic avenues.
Abstract:
Myelomeningocele, more commonly known as spina bifida, is the most common neural tube defect worldwide. In the United States, it is the primary cause of lifelong childhood paralysis with approximately four children born daily with this devastating disease. To minimize damage to the exposed spinal cord and prevent ascending central nervous system infections, postnatal closure of the spinal defect has been the standard of care for decades. Research into the mechanism of spinal cord injury in those with spina bifida revealed that damage continues to accrue during the gestational period. Prenatal defect closure via in utero surgery was proposed to prevent this early deterioration of the spinal cord, and early animal research demonstrated that prenatal repair was feasible and promising. This paved the way for the first human prenatal repairs in the mid-to-late 1990s. Following the promising outcomes observed during the first human cases, a randomized controlled trial, the Management of Myelomeningocele Study (MOMS), was conducted comparing postnatal repair of spina bifida to prenatal repair. The MOMS trial demonstrated that to those undergoing prenatal repair of spina bifida had a decreased need for ventriculoperitoneal shunting and improved lower extremity motor function. With the success of the MOMS trial, in utero repair is now considered the standard of care in those who meet the criteria for prenatal repair. This review will provide an overview of spina bifida and its impact, highlight the historical changes in care, describe the early research and theory that made prenatal repair an option, discuss the clinical experiences with human fetal repair and briefly touch on future research directions for those with myelomeningocele.
