A clinical trial conducted by Boston Children’s Hospital and Brigham and Women’s Hospital, under the supervision of the US Food and Drug Administration, has performed brain surgery on a fetus inside the womb to correct potentially life-threatening vessel damage.
This was the first patient to undergo the procedure. The condition, known as Vein of Galen malformation (VOGM), is a rare prenatal abnormality in which arteries carrying high-pressure blood connect to a major vein deep at the base of the brain instead of smaller capillaries, which should be the case in normal fetal development. This results in a rapid blood flow that can cause heart failure or stroke after birth.
The team in the United States utilized ultrasound technology to perform the procedure on a woman who was 34 weeks pregnant, and it was a success.
Two days later, the baby was delivered through a typical labor process after the mother’s membrane had broken, and the child was placed in the neonatal intensive care unit for several weeks. The mother and baby are now reunited and at home.
“In our first treated case, we were thrilled to see that the aggressive decline usually seen after birth simply did not appear,” said lead author Professor Darren Orbach of Boston Children’s Hospital.
Follow-up echocardiograms conducted after the child’s birth indicated a significant improvement in cardiac output. Scans also revealed normal functioning of the heart and brain.
Dr. Orbach stated in a statement that they are delighted to report that the baby is progressing remarkably well at six weeks, and is not taking any medications, eating normally, gaining weight, and is now back home.
“There are no signs of any negative effects on the brain.”
According to an article in the peer-reviewed journal of the American Stroke Association, Stroke, experts have praised the groundbreaking procedure and the positive outcome of the surgery. The premature baby who underwent the surgery did not require any cardiovascular assistance or further surgery after the procedure. Additionally, neurological exams showed no signs of stroke, fluid buildup or hemorrhage in brain MRI scans.
Experts have hailed the procedure as a “paradigm shift” in managing Vein of Galen malformation, a rare pre-natal condition that can lead to heart failure or other life-threatening complications after birth. The surgery aims to repair the malformation before birth to prevent heart failure and other related conditions after birth, potentially reducing the risk of long-term brain damage, disability or death.
VOGM, which is the most common congenital vascular brain malformation, occurs in approximately one in every 60,000 births. It is typically detected during late second or third trimester pregnancy via prenatal ultrasound scan and then confirmed through MRI diagnosis.
Currently, the standard of care involves post-birth treatment using embolization, a catheter-based procedure that blocks excess blood flow to the brain and heart by closing off the direct artery-to-vein connections. However, this procedure is high-risk and not always effective at reversing heart failure. Furthermore, severe brain damage may have already occurred at birth, which can cause lifelong cognitive disabilities, life-threatening conditions, or even death for the infant.
According to Professor Gary Satou, a fetal cardiologist at the University of California, Los Angeles, who was not involved in the study, the intervention may have a significant impact on a specific group of patients. He noted that a sufficient number of fetal cases need to be performed to establish a clear pattern of improvement in both neurological and cardiovascular outcomes. Therefore, the national clinical trial will be critical in achieving adequate data and, hopefully, successful outcomes.
Prof. Colin Derdeyn, a neuro-interventional radiologist at the University of Iowa who was not involved in the study, cautiously welcomed the breakthrough and acknowledged the key advancement of intervening before the physiologic events of birth can cause life-threatening heart failure.
However, he also emphasized that one successful case is not enough experience to conclude that the risks of the procedure are worth the benefits. Despite this, he found the positive hemodynamic changes observed both in utero and after birth, such as the reduction in flow and size of the draining vein, and reversal of abnormal flow in the aorta, encouraging.
According to him, these are some of the most exciting and surprising aspects of this case report and represent pioneering work being done in a very careful and responsible way.
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