New study shows cheaper, less invasive treatment can improve brain growth, function in babies with hydrocephalus

North American research could have global impact

TORONTO – Turning conventional thinking about hydrocephalus on its head, new research shows that a less invasive, more cost-effective treatment for babies with fluid in the brain results in similar brain growth and function compared to the more complicated and expensive option. The study, co-led by The Hospital for Sick Children, Pennsylvania State University and Boston Children’s Hospital, opens the door to more accessible treatment for babies around the world, especially in Africa, where the condition is common in infants.

The research is published in the December 21 online edition of the New England Journal of Medicine.

Hydrocephalus, a condition in which brain fluid builds up and causes pressure on the brain, is often found in babies who have infections or were born premature. It is usually treated with a shunt, a type of rubber drainage tube that is permanently surgically placed in the baby’s brain to remove fluid, thus relieving the pressure. Another treatment that is less commonly used is endoscopic third ventriculostomy (ETV), which is a less invasive type of surgery where an endoscope (a tube with an attached camera) is used to guide the surgeon in creating a small hole at the base of the brain, which allows the fluid to flow more freely. While the ETV treatment has some clear benefits for patients – it does not require a permanent implant to be placed in the brain and causes fewer complications – it drains less fluid than a shunt. For this reason, it has thus far not been the preferred treatment for hydrocephalus in babies.

In a randomized clinical trial, the research team studied 100 babies in Uganda who had developed hydrocephalus after severe brain infection, comparing conventional shunt treatment with ETV. They found that there was no difference in how the brain grew or functioned, regardless of the treatment administered. Prior to treatment, many of the babies had smaller-than-average sized brains; however, many experienced “catch-up growth”, allowing their brains to grow to average size.

“For decades, the treatment of babies with hydrocephalus was based on how much fluid could be drained; the philosophy was that draining more fluid must be better for the brain. Our study shows that this is not the case and that substantial brain growth can happen even if more fluid is left behind, as we see with ETV treatment,” says the study’s co-lead author Dr. Abhaya Kulkarni, Staff Neurosurgeon and Senior Scientist at SickKids.

“We found what appears to be a major biological discovery: before treatment, almost all infants had brain growth arrest while the pressure in their brains was high, and an important group of these babies demonstrated catch-up growth after the pressure was relieved with treatment. The infants whose brains had the best growth response also demonstrated the best cognitive outcomes, and this was not dependent on the type of treatment that was administered,” says Kulkarni, who is also Professor in the Division of Neurosurgery at the University of Toronto.

He notes that this discovery could have global implications, as tens of thousands of babies with hydrocephalus in developing countries could be successfully treated with the less expensive ETV procedure, sparing them potentially life-threatening complications. Worldwide, hydrocephalus occurs in an estimated 400,000 paediatric cases every year.

Next steps in the research include following the participating children in Uganda up to age five and monitoring their continued brain growth and cognitive development. The research team will also repeat the study with North American babies to determine if similar results will be observed, given that the type of hydrocephalus that occurs in North American babies differs from the more severe form of the condition in Africa.

This study was supported by the National Institutes of Health and SickKids Foundation. It is an example of how SickKids is contributing to making Ontario Healthier, Wealthier and Smarter. www.healthierwealthiersmarter.ca.

About The Hospital for Sick Children

The Hospital for Sick Children (SickKids) is recognized as one of the world’s foremost paediatric health-care institutions and is Canada’s leading centre dedicated to advancing children’s health through the integration of patient care, research and education. Founded in 1875 and affiliated with the University of Toronto, SickKids is one of Canada’s most research-intensive hospitals and has generated discoveries that have helped children globally.  Its mission is to provide the best in complex and specialized child and family-centred care; pioneer scientific and clinical advancements; share expertise; foster an academic environment that nurtures health-care professionals; and champion an accessible, comprehensive and sustainable child health system. SickKids is proud of its vision for Healthier Children. A Better World. For more information, please visit www.sickkids.ca. Follow us on Twitter (@SickKidsNews) and Instagram (@SickKidsToronto).

Media contacts:

Suzanne Gold
The Hospital for Sick Children
416-813-7654, ext. 202059
suzanne.gold@sickkids.ca

Jessamine Luck
The Hospital for Sick Children
416-813-7654, ext. 201436
jessamine.luck@sickkids.ca