Researchers perform intrauterine surgery to repair potentially fatal developmental condition

For the first time, researchers repair a potentially fatal developmental condition by treating an aggressive vascular malformation called Galen’s vein in the fetal brain before birth, according to a new study published in the uterus. I had a successful internal surgery. 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. , can lead to pulmonary hypertension, heart failure, or other potentially life-threatening conditions. VOGM is most often first seen on prenatal ultrasound and definitively diagnosed by MRI late in the second or third trimester of pregnancy.

The researchers performed intrauterine embolization on fetuses 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, in the United States. Conducted under supervision. Food and Drug Administration.

An ongoing clinical trial is using ultrasound-guided transuterine embolization to address the veins of Galen’s malformation prenatally. I was thrilled to see that in the first treated case, the aggressive decline normally seen after birth simply did not appear. I am happy to report that I am eating normally, gaining weight and coming home. There are no signs of adverse effects on the brain. “

Darren B. Orbach, MD, Ph.D., lead study author, co-director of the Center for Neurovascular Surgery and Intervention, Boston Children’s Hospital, associate professor of radiology at 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.

“This is the first patient we have treated and it is important that the trial continues to assess safety and safety. Effectiveness In other patients, this approach may represent a paradigm shift in the management of Galen malformation, repairing the malformation prenatally rather than trying to reverse heart failure postnatally, before heart failure develops. “This could significantly reduce the risk of long-term brain injury, disability, or death in these infants,” Orbach said.

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. said FAHA’s Gary M. Satou, M.D., Ph.D. He is the director of pediatric echocardiography at UCLA Mattel Children’s Hospital and co-director of the UCLA Fetal Cardiology Program and was not involved in this study. Mr. Sato, a pediatric cardiologist who has served as chairman of the Congenital Heart Diseases Committee of the Young Hearts Council of the American Heart Association, and David of UCLA, is also a clinical professor of pediatrics at the Geffen Medical School.

“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 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 the money. N. was Chief of the Department of Radiology and Chair of the Department of Radiology at the University of Iowa Health Care-Carver School of Medicine, and former Chair 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. Surprising aspects of this case report. 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.