In the first intrauterine brain surgery, doctors eliminated the symptoms of a dangerous condition

Research highlights:

• Using ultrasound guidance, researchers have successfully repaired a potentially fatal vascular malformation deep in the prenatal fetal brain called Galen’s malformation. This malformation of excessive blood flow often leads to heart failure, severe brain damage, or death shortly after birth.
• The first intrauterine embolism repair was successfully performed in a fetus at 34 weeks 2 days of gestation. Fetal ultrasonography showed an immediate reduction in abnormal blood flow through the brain malformation, and fetal echocardiography showed significant improvement in cardiac function the day after surgery.
• Since birth, the infant required no medication to treat heart failure, nor postnatal surgery to treat malformations. Postnatal repeat echocardiography showed marked improvement in cardiac output, and her MRI of the brain showed no brain damage and a normal neurological examination.

Embargoed until Thursday, May 4, 2023, 4:00 AM CT / 5:00 AM ET

Dallas, May 4, 2023 — Repairing a potentially fatal developmental condition in the prenatal fetal brain by treating an aggressive vascular malformation called the Galen’s vein, according to new research First intrauterine surgery was successful.A study published today in strokethe peer-reviewed flagship journal of the American Stroke Association, a division of the American Heart Association.

Galen Vein Malformation (VOGM) is a rare prenatal condition in which an artery carrying high-flow, high-pressure blood from the heart to the brain connects directly to one of the major collecting veins deep at the base of the brain. rather than the capillaries needed to slow blood flow and deliver oxygen to the surrounding brain tissue. Due to changes in the infant’s vascular physiology during and after birth, the malformed high flow rate has even more severe effects on the postnatal heart and brain, putting greater pressure on the neonatal heart and lungs. , pulmonary hypertension, heart failure or other potentially life-threatening conditions. VOGM is most often first seen on prenatal ultrasound and definitively diagnosed by her MRI late in the second or third trimester of pregnancy.

The researchers performed intrauterine embolization on a fetus with VOGM at 34 weeks and 2 days gestation as the first treatment patient in an ongoing clinical trial at Boston Children’s Hospital and Brigham and Women’s Hospital, USA. conducted under the supervision of Food and Drug Administration.

“Ongoing clinical trials are using ultrasound-guided transuterine embolization to address Galen malformation veins prenatally. At 6 weeks, the infant is doing very well, taking no medications, eating normally, gaining weight, and going home. We are pleased to report that there have been no indications of adverse effects on the brain,” said lead study author Darren B. Orbach, MD, Ph.D. He is co-director of the Center for Cerebrovascular Surgery and Intervention at Boston Children’s Hospital and Harvard Medical School.

The infant was delivered 2 days later by induced labor due to premature rupture of the membrane during intrauterine embolization. Postnatal echocardiography showed a progressive normalization of cardiac output. In this case, the neonate did not require cardiovascular support or surgery after intrauterine treatment and was observed in the NICU for several weeks after birth due to prematurity before being sent home. , MRI of the brain showed no stroke, fluid accumulation, or hemorrhage.

“While this is our first treatment patient and it is important that we continue the trial to assess safety and efficacy in other patients, this approach is the first of its kind in Galen malformation, which pre-repairs the malformation. “It may represent a paradigm shift in venous management: rather than trying to reverse heart failure after birth, it’s about giving birth and avoiding heart failure before it occurs,” Orbach said. This may significantly reduce the risk of long-term brain injury, disability, or death in these infants.”

VOGM, the most common congenital vascular brain malformation, is estimated to occur in as many as 1 in 60,000 live births. The current standard of care for VOGM is postnatal treatment with embolization. This is a catheter-based procedure that blocks direct connections between malformed arteries and veins, blocking excess blood flow to the brain and heart. However, embolization itself is risky and is not always successful in reversing heart failure. In addition, severe brain damage may already have occurred, which can lead to lifelong cognitive impairment, life-threatening conditions in young children, and even death.

“The Fetal Intervention Team at Boston Children’s Hospital and Brigham and Women’s Hospital has successfully devised another intrauterine procedure that could be highly impactful for a specific group of patients diagnosed with Galen’s malformation. Sato, director of pediatric echocardiography at UCLA Mattel Children’s Hospital and co-director of the UCLA Fetal Cardiology Program, was not involved in the study. He has served as Chair of the Hearts Council Congenital Heart Diseases Committee and is a Clinical Professor of Pediatrics at UCLA’s David Geffen School of Medicine.

“As always, many of these fetal cases need to be conducted and followed to establish a clear pattern of improvement in neurological and cardiovascular outcomes,” Sato said. “Therefore, national clinical trials will be very important to obtain adequate data and hopefully successful results.”

The procedure is not without limitations, says Colin P. Derdeyn, a neurointerventional radiologist at the University of Iowa College of Health Care who performs VOGM embolization in neonates and was not involved in the study. , MD said.

“The key advance here is to intervene before the physiological events of childbirth can lead to life-threatening heart failure. We do not have sufficient experience to conclude that it is worth it.Safety issues may arise in future procedures, and this intravenous approach is unlikely to be consistently successful in preventing heart failure. No. The procedures described here are designed to reduce flow through the deformity, not to cure it,” said a radiologist in the Department of Radiology at the University of Iowa Health Care Carver College of Medicine. Derdeyn, department professor and director of the radiology department, said: Former chairman of the American Heart Association’s Stroke Council.

“However, the hemodynamic changes they observed in utero and postnatally – reduced flow, reduced size of the outflow vein, reversal of abnormal regurgitation in the aorta – are truly encouraging. “It’s some of the most exciting and surprising aspects,” he said. “This is pioneering work being done in a very careful and responsible way.”

Co-author is Louise E. Wilkins-Haug, MD. Carol B. Benson, MD. Wayne Tworetzky, MB Ch.B. Shivani D. Rangwala, MD. Stephanie H. Guseh, MD. Nicole K. Gately, RN. Dr. Jeffrey N. Stout. Arielle Mizrahi-Arnaud, MD. and Alfred P. Disclosures of the MD Authors are provided in the manuscript.

This study was funded by a grant from the Sage Schermerhorn Chair for Image-Guided Therapy (DBO) in the Department of Radiology, Boston Children’s Hospital.

Research statements and conclusions published in the American Heart Association scientific journal are those of the study authors only and do not necessarily reflect the policies or positions of the association. The Association makes no representations or warranties as to their accuracy or reliability. The association receives funding primarily from individuals. Foundations and companies (including pharmaceuticals, device manufacturers, and other companies) also make donations to fund specific programs and events of the association. The Society has strict policies to prevent these relationships from affecting the content of science. Income from pharmaceutical companies, biotech companies, device manufacturers, health insurers, and the association’s overall financial information are available. here.

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