Stories about: Pediatrics

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/stock.adobe.com)

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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SIDS associated with higher blood serotonin levels

A baby sleeping on its back, which is the safest sleeping position to prevent SIDS
The Safe to Sleep campaign has helped reduce SIDS deaths, but underlying causes for SIDS have largely remained mysterious.

Sudden infant death syndrome (SIDS) accounts for the greatest share of deaths in children between the ages of 1 and 12 months. What if a blood test could explain a third of SIDS deaths – and in the future, help prevent them? New findings by a Boston Children’s Hospital team show that an increased level of serotonin in blood serum may underpin some SIDS deaths and suggests the possibility that this biological vulnerability may one day be detected in the blood of living infants.

While there are known risk factors for SIDS — such as sleeping face-down or on soft surfaces — how and why such seemingly minor threats kill some children, and not others, remains a mystery.

“Research on the underlying pathology of SIDS is critical to further our understanding of the biological mechanisms contributing to a SIDS death,” says Robin Haynes, PhD, a researcher in the Department of Pathology at Boston Children’s Hospital.

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Can we improve neuropsychiatric outcomes in children with congenital heart disease?

Jane Newburger studies neurodevelopment in children with congenital heart defects
Jane Newburger, MD, has dedicated her career to helping children with heart defects reach their full potential.

About 1 out of 100 babies are born with a congenital heart defects. Thanks to medical and surgical advances, these children usually survive into adulthood, but they are often left with developmental, behavioral or learning challenges.

Children with “single-ventricle” defects — in which one of the heart’s two pumping chambers is too small or weak to function properly — are especially at risk for neurodevelopmental problems. “Single-ventricle physiology creates cerebrovascular hemodynamics that can reduce oxygen delivery to the brain,” explains Jane Newburger, MD, MPH, director of the Cardiac Neurodevelopmental Program at Boston Children’s Hospital.

How does this play out in adolescence? In three recent studies, Boston Children’s Heart Center collaborated with the departments of Neurology and Psychiatry to track neurodevelopmental outcomes after corrective Fontan operations. They evaluated preteens and teens as old as 19 — the longest follow-up to date.

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An unclassified tumor — with a precisely targeted therapy

Jesus (who received targeted therapy for his tumor) pictured with his father
Jesus and his father, Nathaneal

Early last year, at his home in San Juan, Puerto Rico, Jesus Apolinaris Cruz’s leg hurt so much he could barely sleep. “All day,” the 13-year-old recalls. “It was constant pain.” His parents took him to two local pediatricians, who examined him, drew blood, tested his platelets. No diagnosis. Finally, in April 2016, a physician ordered an MRI. No wonder Jesus’s leg hurt. He had a large, cancerous tumor lodged in his hip.

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Webchat to highlight what’s new in pediatric brain tumors

pediatric brain tumors, child MRI

Last September, the National Center for Health Statistics reported that brain tumors have overtaken the much more common leukemia as the leading cause of death from pediatric cancer. Although progress has been made and the promise of more progress is on the horizon, the cure rate for childhood brain tumors lags behind a number of other pediatric cancers.

As pediatric neuro-oncologist Peter Manley, MD, of Dana-Farber/Boston Children’s Cancer and Blood Disorders Center told Live Science, new research on cancer genomics “is so impressive that my feeling is that we will continue to see a decline in deaths.”

To mark Brain Tumor Awareness Month, Mark Kieran, MD, PhD, clinical director of the Brain Tumor Center at Dana-Farber/Boston Children’s, will host a webchat on Monday, May 22 (3:30 p.m. ET). The live chat will highlight the latest research and treatments for pediatric brain tumors. Here’s a look back at some recent developments:

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One family, one researcher: How Mikey’s journey is fueling an attack on DIPG

Picture of Mikey on 11th birthday, shortly after his DIPG diagnosis
Mikey and his family at his 11th birthday party, just one week after he was diagnosed with DIPG, a devastating tumor in his brain stem. Since Mikey’s passing in 2008, his family has been committed to supporting DIPG research.

“It’s a brutal disease; there’s just no other way to describe DIPG,” says Steve Czech. “And what’s crazy is that there aren’t many treatment options because it’s such a rare, orphan disease.”

Czech’s son, Mikey, was diagnosed with a diffuse intrinsic pontine glioma (DIPG) on Jan. 6, 2008. It was Mikey’s 11th birthday. The fast growing and difficult-to-treat brainstem tumors are diagnosed in approximately 300 children in the U.S. each year.

Sadly, the virtually incurable disease comes with a poor prognosis for most children. The location of DIPG tumors in the brainstem — which controls many of the body’s involuntary functions, such as breathing — has posed a huge challenge to successful treatment thus far.

“Typically, they give kids about nine months,” says Czech. “Our lives changed forever the day that Mikey was diagnosed.”

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GALLERY: Forecasting the future of pediatric hematology/oncology

Title image for pediatric hematology/oncology predictionsRecently, the annual ASPHO (American Society for Pediatric Hematology/Oncology) meeting brought together more than 1,100 pediatric hematologists and oncologists, including a team from the Dana-Farber/Boston Children’s Cancers and Blood Disorders Center. Some of the delegates from Dana-Farber/Boston Children’s included:

Based on their discussions with their peers, these are their key takeaways from the meeting:

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Can virtual reality headsets save vision in people with lazy eye?

Luminopia amblyopia virtual reality
IDHA’s Matt Murphy tries out Luminopia’s VR headset with Dean Travers (photo: Greb Weintraub)

Three to five percent of the population has amblyopia, a.k.a. lazy eye, in which a healthy eye never “learns” to see because isn’t used. This usually happens because of a focusing problem or subtle misalignment of that eye. The brain learns to ignore input from that eye, and unless this is noticed early, it weakens and can slowly go blind.

“When I can diagnose amblyopia early enough, I can treat it with an eye patch or eye drops to block the ‘good’ eye,” says David Hunter, MD, PhD, chief of ophthalmology at Boston Children’s Hospital. “This gives the eye with amblyopia time to catch up.”

Unfortunately, eye patching doesn’t work well at older ages, and kids hate the socially stigmatizing patches, which often need to be worn for more than a year. As Dean Travers, cofounder of Luminopia, put it at Boston Children’s Hospital’s Innovators’ Showcase last week, “Being a pirate isn’t cool for very long.”

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