Summary of First-of-Its-Kind, In-Utero Procedure To Fix Deadly Vascular Malformation:
Researchers and clinicians from Brigham and Women’s Hospital and Boston Children’s Hospital have performed the first-ever in-utero cerebrovascular surgery in the U.S. The operation’s success means doctors can repair an aggressive vascular malformation in an unborn child’s brain, which can cause severe damage to the brain after birth. The surgery is part of a clinical trial that aims to correct the malformation before birth, potentially reducing long-term injury, disability, or death. The first treated patient has shown no adverse effects, indicating that this process has the potential to become a new standard for managing VOGM.
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Revolutionary In-Utero Surgery: Treating Vascular Malformation in Infant’s Brain
In a groundbreaking medical achievement, a team from Brigham and Women’s Hospital and Boston Children’s Hospital successfully treated an aggressive vascular malformation in an infant’s brain before birth, marking the first in-utero cerebrovascular surgery in the United States. The treatment was part of a clinical trial to address vein of Galen malformation (VOGM) in utero, a rare condition that can cause severe brain injury and is typically treated after birth. With this revolutionary approach, the team aims to repair the malformation before birth, potentially reducing the risk of long-term brain damage, disability, or death.
Prevention of Potentially Fatal Symptoms
Collaborating researchers and clinicians from Boston Children’s Hospital and Brigham and Women’s Hospital have prevented a deadly developmental condition by treating an aggressive vascular malformation in an infant’s brain before birth. The case, the first-ever in-utero cerebrovascular surgery in the United States, is described in a paper published today (May 4) in Stroke, the peer-reviewed flagship journal of the American Stroke Association, a division of the American Heart Association.
The Success of First Treated Patient
“In our trial, we use ultrasound-guided transuterine embolization to address the vein of Galen malformation before birth. In our first treated case, we were thrilled that the aggressive decline usually seen after birth did not appear. We are pleased to report that at six weeks, the infant is progressing remarkably well, on no medication, eating normally, gaining weight, and returning home. There are no signs of any negative effects on the brain,” said lead study author Darren B. Orbach, M.D., Ph.D., Co-Director of the Cerebrovascular Surgery & Interventions Center at Boston Children’s Hospital and an associate professor of radiology at Harvard Medical School.
Potential Paradigm Shift in Managing VOGM
“While this is only our first treated patient, and we must continue the trial to assess the safety and efficacy in other patients, this approach has the potential to mark a paradigm shift in managing vein of Galen malformation, where we repair the malformation before birth and head off the heart failure before it occurs rather than trying to reverse it after birth. This may reduce the risk of long-term brain damage, disability, or death among these infants.”
VOGM: A Rare Condition That Calls for Early Intervention
VOGM is rare when misshapen arteries in the brain connect directly to veins instead of capillaries. This slows blood flow and can lead to high-pressure blood flowing into the veins. This increase in pressure can prevent an infant’s brain from draining adequately and lead to widespread brain injury or severe loss of tissue in the brain. The standard of care is currently to treat infants with VOGM after birth, but brain damage has already occurred in many cases.
Promising Outcome
Because of the abnormally high blood flow in the malformation, children born with VOGM do not live long enough to have it diagnosed, with many dying within the first few days of heart failure. In this case, after in-utero treatment, the newborn did not require additional surgery after birth, and the brain MRI showed no strokes, fluid buildup, or hemorrhage often seen with VOGM. The baby, born in mid-March, was discharged after several weeks in the NICU and is hitting all her milestones.
The Importance of Teamwork
“In every fetal surgery, there are two patients: the baby and the mother, and caring for both the fetus and the mother is an important aspect of fetal procedures,” said co-author Carol Benson, MD, staff radiologist at BWH and former co-director of BWH’s High-Risk Obstetrical Ultrasound Service. “You need to ensure that everything is aligned perfectly, and we couldn’t do anything without the precise communication and teamwork of everyone involved.”
Continued Research
While the success of the first treated patient is a huge milestone for the clinical trial, the team still has a long way to go. The problem will continue to assess the safety and efficacy of other patients.
The 20-year history of fetal surgery at the Brigham underscores the commitment to continuous improvement and collaboration between Boston Children’s Hospital and Brigham and Women’s Hospital,” said co-author Louise Wilkins-Haug, MD, Division Director of Maternal-Fetal Medicine at Brigham and Women’s Hospital. “This proof of concept was a highly collaborative effort between our two organizations for many years now, and to see it be so successful is a testament to that mission.”
In Conclusion
In-utero cerebrovascular surgery marks a paradigm shift in the management of VOGM where, by repairing the malformation before birth, the risk of long-term brain damage, disability, or death among these infants can be markedly reduced. Collaborative efforts and continued research are vital to the success of this revolutionary approach that aims to provide a better outcome for infants with VOGM.
