Nearly 20 years ago, two procedures—endoscopic third ventriculostomy (ETV) and choroid plexus cauterization (CPC)—were combined to successfully treat pediatric hydrocephalus in Africa. The procedure, referred to as ETV-CPC, reignited discussion in the United States about the safety, efficacy and long-term outcomes associated with CPC. 

In a new review article, Moise Danielpour, MD, and his coauthors Sarah Stricker, MD, Raphael Guzman, MD, and Thomas Blauwblomme, MD, make clear the need for additional research to fully understand potential risks and for increased, candid discussion with interested patient families. 

“Etiology of the patients who underwent the combined procedures was not necessarily the same as the majority of patients in the rest of the world. The cause of hydrocephalus in a lot of those patients may have been infection,” said Danielpour, director of Pediatric Neurosurgery at Cedars-Sinai Guerin Children’s and the Vera and Paul Guerin Family Chair in Pediatric Neurosurgery. “We need to ask ourselves if this procedure makes as much of a difference here as it did there. To make a significant difference, you have to ablate almost all of the choroid plexus, and we have to be apprehensive about any kind of destructive procedure.”

The choroid plexus (CP) has several functions in the brain: It secretes spinal fluid and acts as the entry point for the immune cells to fight intracranial infections. Additionally, recent studies suggest that the CP plays a large role throughout brain development and impacts brain homeostasis. There are even studies that suggest a role for the CP in conditions like Alzheimer's disease.

Because of its important roles, Danielpour cautions against being overeager to remove the CP. Although CPC can be beneficial in reducing the need for placing a shunt to treat hydrocephalus, there are not enough studies yet on long-term outcomes or risks of neurodegeneration and inflammatory changes. He believes the knowledge of long-term outcomes is crucial in the decision-making process. 

“I personally do not feel that I could confidently recommend ETV-CPC without a very detailed discussion of the possible long-term risks,” Danielpour said. “Oftentimes, parents of children with hydrocephalus will hear about ETV-CPC on the internet and then look for a doctor willing to perform it. As neurosurgeons, we have to be advocates for our patients—we’re not just providers of a service. You have to be thoughtful and judicial when you're recommending one treatment versus another.”

ETV-CPC is not the sole modern treatment for pediatric hydrocephalus. Traditionally, neurosurgeons have inserted a shunt to drain fluid or performed an ETV (without CPC) to install an internal bypass. Physicians are much more familiar with the potential risks, side effects and ongoing results of these options. 

Although ETV-CPC certainly has value for medically underserved communities where the shunt option may not be available, Danielpour does not recommend it being widely reintroduced without, at least, extensive discussion with patients and families.

“I would not feel comfortable recommending ETV-CPC to my patients based on what we know right now,” said Danielpour. “If a patient or family chooses ETV-CPC, they should do so informed of both everything we know about the potential risks and what we don’t yet know.”

He hopes that with time, research will provide more concrete answers to the unknowns of ETV-CPC, such as the success in hydrodynamic stabilization of hydrocephalus and long-term outcomes related to neurodegeneration, inflammatory changes and compensatory metabolic mechanisms. 

“There is some additional research underway—randomized studies looking at outcomes and potential cognitive deficits,” said Danielpour. “In the meantime, we as physicians need to remember that the newest thing is not always the best thing.”

“At an academic medical center like Cedars-Sinai, we are fortunate to have one foot in the clinical world and one in academics and research. We continue to focus on getting the best possible outcomes for patients while conducting research to find new cures and treatments,” he noted.