Stories about: Pediatrics

One-time hydrocephalus operation, alternative to shunting, brings good outcomes for babies

infant hydrocephalus archival photos
(Flickr/Wikimedia Commons)

Hydrocephalus, literally “water on the brain,” is an abnormal build-up of cerebrospinal fluid in the brain cavities known as ventricles. In infants, it can be congenital (it often accompanies spina bifida, for example), or it can be caused by brain hemorrhage or infection. The usual treatment is surgery to implant a shunt, which drains the excess fluid into the abdomen, relieving pressure on the brain.

But over time, shunts nearly always fail, requiring emergency neurosurgery to repair or replace them. But emergency neurosurgery is not something that’s readily available outside of metropolitan areas. Untreated, hydrocephalus causes progressive brain damage and usually death.

What if a one-time operation could treat hydrocephalus permanently? In today’s New England Journal of Medicine, a randomized trial shows good results with a minimally invasive, relatively inexpensive shunt alternative called endoscopic third ventriculostomy with choroid plexus cauterization (ETV/CPC).

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Taking a sideswipe at high-risk neuroblastoma

Microscopy image of human neuroblastoma cells.
Human neuroblastoma cells.

Cancer and other diseases are now understood to spring from a complex interplay of biological factors rather than any one isolated origin. New research reveals that an equally-nuanced approach to treating high-risk neuroblastoma may be the most effective way to curb tumor growth.

One challenge in treating pediatric cancers like neuroblastoma is that they are not initiated from the same kinds of genetic mutations as adult cancers, which usually arise from mutations related to an accumulation of DNA replication errors or environmental factors. In contrast, childhood cancers more often stem from genetic duplications, deletions or translocations, the latter of which occurs when a gene sequence switches its location from one chromosome to another.

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Giving young organ transplant recipients a mobile reminder that might save their lives

Flow chart showing how the medication adherence app interacts with patients and a near-field-communication-enabled pillboxIn the U.S., more than 1,700 children receive organ transplants each year. Following transplantation, they must take immunosuppressants and steroids to protect their transplanted organ from being attacked by their own immune system.

But transplant teams know that kids are 60 percent more likely than adults to struggle with keeping a strict medication schedule. That puts the longevity of donated organs — and the lives of organ recipients — at unnecessary risk.

This challenge inspired a team of pediatric transplant experts at the Boston Children’s Hospital to develop a mobile application for smartphones that could serve as a portable reminder and a resource to support medication adherence.

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A “half-hearted” solution to one-sided heart failure

Illustration showing how the system supports a failing right ventricle
Illustration showing sectional view of a heart with the soft robotic system helping to draw blood into (left) and pump blood out (right) of the heart’s right ventricle.

Soft robotic actuators, which are pneumatic artificial muscles designed and programmed to perform lifelike motions, have recently emerged as an attractive alternative to more rigid components that have conventionally been used in biomedical devices. In fact, earlier this year, a Boston Children’s Hospital team revealed a proof-of-concept soft robotic sleeve that could support the function of a failing heart.

Despite this promising innovation, the team recognized that many pediatric heart patients have more one-sided congenital heart conditions. These patients are not experiencing failure of the entire heart — instead, congenital conditions have caused disease in either the heart’s right or left ventricle, but not both.

Read our Vector story on the soft robotic heart sleeve that mimics cardiac muscles.

“We set out to develop new technology that would help one diseased ventricle, when the patient is in isolated left or right heart failure, pull blood into the chamber and then effectively pump it into the circulatory system,” says Nikolay Vasilyev, MD, a researcher in cardiac surgery at Boston Children’s.

Now, Vasilyev and his collaborators — researchers from Boston Children’s, the Harvard John A. Paulson School of Engineering and Applied Sciences and the Wyss Institute for Biologically Inspired Engineering at Harvard University — have revealed their soft robotic solution. They describe their system in a paper published online in Science Robotics today.

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Breastfeeding protects against food allergy: We have evidence

Mother breastfeeding her baby. Is she helping her child avoid food allergy?

Eating allergenic foods during pregnancy can protect your child from food allergies, especially if you breastfeed, suggests new research. The findings, in a mouse model of allergy, underscore recent advice that pregnant or nursing mothers not avoid allergenic foods like eggs and peanuts.

The study is the first controlled investigation to demonstrate protection against food allergy from breast milk, while also pointing to a biological mechanism for inducing food tolerance. It was published online today in the Journal of Experimental Medicine.

“Whether mothers should eat allergenic foods during pregnancy or avoid them has been controversial,” says Michiko Oyoshi, PhD, of Boston Children’s Division of Allergy and Immunology, who led the study in collaboration with Richard Blumberg, MD, of Brigham and Women’s Hospital, her co-senior author.

“Different studies have found different results, in part because it’s hard in human studies to know when mothers and babies first encountered a specific food,” says Oyoshi. “But in a mouse model, we can control exposure to food.”

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Personalized care model enhances preterm babies’ development

NICU baby with his mother
Christian, born at 26 weeks gestation, has lived in the NICU since May. (Photos: Katherine C. Cohen, unless otherwise noted)

November 17, 2017 is World Prematurity Day.

From a cozy, dark and quiet existence, a preterm baby is forced out into a harsh, bright and noisy environment. Instead of being comforted and held securely by their parents, preemies are poked and prodded, hooked up to machines and exposed to jarring sights and smells as their developing brains struggle to realign.

Each year, an estimated 15 million babies around the world — 1 in 10 — are born prematurely. Medical advances enable more of them to live, but often with medical and developmental problems.

Heidelise Als, PhD, director of Neurobehavioral Infant and Child Studies at Boston Children’s Hospital, has worked for more than 30 years to create better outcomes, developing the Newborn Individualized Developmental Care and Assessment Program, or NIDCAP.

The NIDCAP model of care seeks to support the development of fragile newborns and reduce their stress. In a series of studies, Als and colleagues at other hospitals have documented its successes: improvements in lung function, feeding and growth; shorter lengths of stay; a reduction in brain hemorrhage and improved brain function and structure, with brain effects lasting until at least 8 years of age. Benefits have been documented even in medically fragile, very preterm infants and infants with severe intrauterine growth restriction.

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Meeting an unmet need: A surgical implant that grows with a child

Depiction of a growth-accommodating implant expanding in sync with a child's growing heart.
Artist’s rendering showing how a braided, tubular implant could grow in sync with a child’s heart valve. Credit: Randal McKenzie

Medical implants can save lives by correcting structural defects in the heart and other organs. But until now, the use of medical implants in children has been complicated by the fact that fixed-size implants cannot expand in tune with a child’s natural growth.

To address this unmet surgical need, a team of researchers from Boston Children’s Hospital and Brigham and Women’s Hospital have developed a growth-accommodating implant designed for use in a cardiac surgical procedure called a valve annuloplasty, which repairs leaking mitral and tricuspid valves in the heart. The innovation was reported today in Nature Biomedical Engineering.

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Pediatric devices wanted: Boston Children’s Hospital and the Boston Pediatric Device Consortium launch $250,000 challenge

Boston Pediatric Device Strategic Partner Challenge opens

There’s generally little incentive for industry to develop medical devices for children: The pediatric market is small (most children are healthy) and clinical trials are harder to do in children.

“Innovation in medical devices with the potential to improve the health of children and adolescents continues to lag in comparison to those for adults,” says Pedro del Nido, MD, leader of the Boston Pediatric Device Consortium and Chief of Cardiac Surgery at Boston Children’s Hospital. 

This week, the Innovation and Digital Health Accelerator (IDHA) at Boston Children’s Hospital and the Boston Pediatric Device Consortium (BPDC) announced a national challenge to try to remedy this problem. The Boston Pediatric Device Strategic Partner Challenge will award up to $50,000 to entrepreneurs and innovators seeking to create novel pediatric medical devices, from a total pool of up to $250,000.

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“Omics” study takes a comprehensive look at premature birth

Seven layers of omics study
Seven layers of “omics” included in the PREM-MAP study

Every year, one in 10 new babies in the United States is born preterm, or before 37 weeks of gestation. With the last few weeks of pregnancy crucial to proper development of the lungs and brain, prematurely born infants can suffer lifelong problems.

Now scientists at Boston Children’s Hospital and Beth Israel Deaconess Medical Center have launched a comprehensive study to understand the reasons and risk factors for premature births. Earlier this year, Olaf Bodamer, MD, PhD was awarded a grant for this work from uBiome, a microbial genomics company.

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From mice to humans: Genetic syndromes may be key to finding autism treatment

Boy and a mouse eye-to-eye
(Aliaksei Lasevich/

A beautiful, happy little girl, Emma is the apple of her parents’ eyes and adored by her older sister. The only aspect of her day that is different from any other 6-month-old’s is the medicine she receives twice a day as part of a clinical trial for tuberous sclerosis complex (TSC).

Emma’s mother was just 20 weeks pregnant when she first heard the words “tuberous sclerosis,” a rare genetic condition that causes tumors to grow in various organs of the body. Prenatal imaging showed multiple benign tumors in Emma’s heart.

Emma displays no symptoms of her disease, except for random “spikes” on her electroencephalogram (EEG) picked up by her doctors at Boston Children’s Hospital. The medication she is receiving is part of the Preventing Epilepsy Using Vigabatrin in Infants with TSC (PREVeNT) trial. Her mother desperately hopes it is the active antiepileptic drug, vigabatrin, rather than placebo.

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