For the first time, surgeons have successfully operated on an unborn baby to correct a life-threatening brain condition.
Using ultrasound to guide them, the team repaired a malformation called vein of Galen, in which arteries carrying high-pressure blood connect directly to veins carrying blood away from the brain – instead of surrounding capillaries which help deliver oxygen to the organ.
If left untreated, the high flow in the malformation has even more serious effects after birth, putting enormous pressure on a newborn’s heart and lungs.
The foetus was operated on at 34 weeks and two days, four weeks after the condition was detected. Six weeks after birth, the team reports the baby is doing ‘remarkably well’.
‘In our ongoing clinical trial, we are using ultrasound-guided transuterine embolisation [a procedure to block blood flow] to address the vein of Galen malformation before birth,’ said lead author Darren Orbach, a co-director at Boston Children’s Hospital.
‘In our first treated case, we were thrilled to see that the aggressive decline usually seen after birth simply did not appear. We are pleased to report that at six weeks, the infant is progressing remarkably well, on no medications, eating normally, gaining weight and is back home.
‘There are no signs of any negative effects on the brain.’
Due to ruptured membranes that occurred during surgery the infant was delivered two days after the procedure, but showed no symptoms associated with the condition. The newborn did not require any cardiovascular support or surgery following the treatment, and after several weeks under observation in the neonatal intensive care unit, was sent home.
During that time the newborn had a normal neurological exam, and MRI showed no strokes, fluid build-up or hemorrhages.
‘While this is only our first treated patient and it is vital we 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 prior to birth and head off the heart failure before it occurs, rather than trying to reverse it after birth,’ said Mr Orbach.
‘This may markedly reduce the risk of long-term brain damage, disability or death among these infants.’
Research suggests VOGM is the most common congenital vascular brain malformation, occurring in as many as one in 60,000 births.
Until now, cases have been treated after birth, using catheters to block the direct artery-to-vein connections. However, the procedure is high risk, and the delay in corrective treatment means brain damage may already have occurred.
Gary M Satou, director of paediatric echocardiography at UCLA Mattel Children’s Hospital who was not involved in the study, said: ‘As always, a number of these foetal cases will need to be performed and followed in order to establish a clear pattern of improvement in both neurologic and cardiovascular outcomes.
‘Thus, the national clinical trial will be crucial in order to achieve adequate data and, hopefully, successful outcomes.’
The study was published in the journal Stroke.
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