News Briefs

Children’s receives $3M research grant from Mallinckrodt

Mallinckrodt Pharmaceuticals has awarded a $3 million Healthcare Advancement Grant to Children’s National Health System, supporting a research initiative focused on pediatric patients in the intensive care setting.

In the U.S., 20 percent of hospitalized children are cared for in the pediatric intensive care unit (ICU). Yet this is an under-researched patient population with layers of complexity. These patients face a 2.5 to 5 percent mortality rate, with 5 to 10 percent serious morbidity rate, and the morbidity and mortality rates double within three years.

Children’s National is uniquely qualified to address this opportunity, with its level IV neonatal ICU, ranked third in the nation by U.S. News and World Report in its 2016-2017 Best Children’s Hospitals survey. Home to the Children’s Research Institute and the Sheikh Zayed Institute for Pediatric Surgical Innovation, Children’s National is one of the nation’s top National Institute of Health (NIH)-funded pediatric institutions. It is a member of the Collaborative Pediatric Critical Care Research Network of the NIH and enjoys strong partnerships with major universities in the Washington, DC, area, providing data-generation resources. The institution plans to mine data from this myriad of sources and more to unearth knowledge and improve outcomes.

Children’s National has identified three priorities to launch and execute this multi-year initiative, specifically:

  • Establish a Critical Care Outcomes Research Initiative team
  • Build on existing partnerships and expand to acquire additional data
  • Build outcomes research studies in the critical care arena

“We applaud Mallinckrodt for their forward thinking as we begin this important research initiative that will help meet the challenges faced by seriously ill pediatric patients,” said Robin Steinhorn, M.D., Senior Vice President for the Center for Hospital-Based Specialties. “We firmly believe the combination of this generous research grant, our many collaborative relationships and Children’s National’s renowned research enterprise will lead to improved outcomes for children in the future.”

David Wessel to speak at USN’s Healthcare of Tomorrow

David Wessel

The fourth annual U.S. News & World Report Healthcare of Tomorrow conference will take place on Nov. 2 in Washington, DC. The leadership forum, which examines challenges in health care and how we must evolve with policies, society, and technology, will hold children’s hospital sessions for the first time. The topics will include pediatric population health, patient safety, strategic partnerships, and genomic medicine. Children’s National’s David Wessel, M.D., executive vice president and chief medical officer, hospital and specialty services, is scheduled to speak during the event.

Pediatric surgical innovation symposium approaches

This year, there were a record number of entries (91) for the National Capital Consortium for Pediatric Device Innovation (NCC-PDI) competition, which will be held  Oct. 8 at the fourth annual Pediatric Surgical Innovation Symposium, hosted by the Sheikh Zayed Institute for Pediatric Surgical Innovation at Children’s National Health System. Twelve finalists have been selected to pitch their pediatric medical device innovation, and up to six innovations will be awarded up to $50,000 each, with awards based on presentations given before a panel of expert judges.

The finalists are:

  • PECA Labs, Pittsburgh, Pa. – synthetic vascular conduit for surgical repair of congenital heart defects that’s capable of minimally invasive, controlled expansion to grow with the patient
  • Maternal Life, Palo Alto, Calif. – low-cost closed system that captures and administers colostrum to newborns with zero percent loss
  • Magnamosis, Inc., San Francisco, Calif. – device to provide safer, less invasive repair of the esophagus in newborns with esophageal atresia/tracheoesophageal fistula, a condition requiring surgery that is currently performed by hand
  • JustRight Surgical, Louisville, Colo. – second generation surgical 5mm stapler sized for use with a wider range of pediatric surgical procedures and bringing the benefits of laparoscopy to patients
  • CareTaker Medical, Charlottesville, Va. – disposable, finger cuff for single patient use to continuously and non-invasively monitor neonatal  heart rate without adhesives, electrodes and wires
  • Nebula Industries, Melrose, Mass. – quick release medical tape to prevent neonatal and pediatric skin injuries
  • Lully, San Francisco, Calif. – moisture sensor and Smart Pod monitor, placed under the mattress, that are wirelessly connected to a smartphone app to prevent bedwetting episodes
  • Center for Advanced Sensor Technology, University of Maryland Baltimore County, Baltimore, Md. – low-cost, disposable multifunctional incubator for at-risk, low birth weight babies
  • Multisensor Diagnostics, Baltimore, Md. – non-invasive portable handheld device designed to perform rapid medical assessment of key vitals for pediatric patients
  • May & Meadow, Inc., Redwood City, Calif. – low-cost, mobile medical device for assessing feeding ability in infants at risk for feeding problems
  • PediaStent, Cleveland, Ohio – novel pediatric bioresorbable stent for use in repairing congenital heart lesions
  • Averia Health Solutions, Alexandria, Va. – low-cost concussion screening and management system that uses smartphone technology

“The impressive number of well qualified applications we received from all over the US as well as from other countries speaks to the enthusiasm of Medtech innovators to develop and test devices specifically for children,” said Kolaleh Eskandanian, Ph.D., M.B.A., P.M.P., Executive Director of the Sheikh Zayed Institute for Pediatric Surgical Innovation and NCC-PDI. “We are committed to building on this momentum and keeping the conversation going with all who applied and will provide consultation services if needed.”

Read more.

First 3D bioprinted placenta model for study of preeclampsia created

Drs. DeBiasi and du Plessis

Scientists at the Sheikh Zayed Institute for Pediatric Surgical Innovation at Children’s National, in partnership with the University of Maryland, are the first to create a 3D bioprinted placenta model and use it to study preeclampsia, a life-threatening pregnancy complication. Bioprinting is the three-dimensional printing of biological tissue and organs through the layering of living cells, with cell function and viability preserved within the printed structure. Because the Institute’s bioprinted placenta model mimics the organ’s complex cellular structure, the model creates unprecedented opportunities to understand and develop new treatments for life-threatening maternal conditions involving the placenta.

Read the full press release. 

Cancer update: tumor targeting, neurofibromatosis type 1 symptoms

June 6, 2016Targeting tumors more precisely, with fewer lasting side effects for kids
Pediatric patients with cancer are often treated with a cocktail of therapies to attack the disease through a variety of mechanisms. While this approach has been instrumental in saving children’s lives, the life-saving therapies can be accompanied by acute side effects, and the treatments may have lingering impacts as cancer survivors enter adulthood. Magnetic resonance-guided high-intensity focused ultrasound (MR-HIFU) holds the promise of surgically removing large tumors without exacting the same array of harsh side effects. Ultrasound relies on high-frequency sound waves to make diagnostic images, and those same sound waves can be used therapeutically to destroy tumors. Layering on MR imaging gives clinicians the ability to precisely guide the ultrasound therapy in real time. A study led by Children’s National Health System researchers and clinicians is using MR-HIFU for the first time in children to examine its safety and feasibility.

May 11, 2016 – Quantitative MRI criteria for optic pathway enlargement in neurofibromatosis type 1
Symptoms of neurofibromatosis type 1 (NF1) vary widely, but the condition is characterized by changes in skin pigmentation and growth of tumors along nerves. The research team sought to determine quantitative size thresholds for enlargement of the optic nerve, chiasm, and tract in children aged 0.5 to 18.6 years with NF1. The study, published in Neurology, found that quantitative reference values for anterior visual pathway enlargement will enhance development of objective diagnostic criteria for optic pathway gliomas secondary to NF1.

Neurology and neurosurgery update: whole exome sequencing, Sesame Workshop

May 9, 2016 WES yields clinical diagnoses for 42 percent, ending ‘diagnostic odyssey’.
Whole exome sequencing (WES), a method to look at all the genes in the genome at once, yielded clinical diagnoses for 42 percent of patients whose white matter abnormalities had been unresolved an average of eight years, ending families’ “prolonged diagnostic odyssey.”  White matter disorders, which affect 1 in 7,000 children born each year, are progressive and involve age-related weakness in the region of the nerves that connect various parts of the brain to each other and to the spinal cord. Nine of 28 named authors of the study, published online May 9, 2016 in Annals of Neurology, are affiliated with Children’s National Health System.

April 6, 2016 Georgetown, Children’s National researchers to evaluate Sesame Workshop’s Autism Initiative.
Sesame Workshop, the nonprofit educational organization behind Sesame Street, has selected Georgetown University Medical Center and Children’s National Health System researchers to lead an evaluation of  “Sesame Street and Autism: See Amazing in All Children,” an initiative developed to reduce stigma and build understanding about autism spectrum disorder. Sesame Workshop launched a new phase of its autism initiative in early April. Last year, Sesame Street introduced Julia, an autistic preschool digital Muppet, and accompanying resources, such as an app, videos, storybooks, and daily routine cards as part of the Sesame Street and Autism: See Amazing in All Children initiative. Now ready for evaluation, Sesame Workshop awarded a grant for real-world testing of the materials to Bruno Anthony, PhD, deputy director of the Georgetown Center for Child and Human Development in collaboration with his wife and research collaborator Laura Anthony, PhD, at the Center for Autism Spectrum Disorder at Children’s National.

Fetal medicine update: fetal brain development, zika virus

May 2, 2016 -Impaired global and tissue-specific brain development in the growth-restricted fetus.
A team of researchers applied an advanced imaging technique, three-dimensional MRI, to study brain development in high-risk pregnancies and are the first to report regional, tissue-specific volume delays for the developing fetal brain in FGR-affected pregnancies. The team compared overall fetal brain volume as well as regional brain volumes for a control group of healthy young pregnant women with a group of young women whose pregnancies were complicated by FGR. While fetuses in both groups grew exponentially as pregnancies progressed, the researchers began to see dramatic differences when they compared the volumes of specific regions of the brain, including the cerebellum, which coordinates balance and smooth movement; the deep gray matter, which also is involved in complex functions, such as memory and emotion; and the white matter, which is made up of millions of nerve fibers that connect to neurons in different regions.

March 30, 2016Congenital Zika viral infection linked to significant fetal brain abnormalities, despite ‘normal’ ultrasounds.
Infectious Zika virus was isolated from the brain of a 21-week-old fetus after causing extensive damage to brain tissue – despite ultrasounds that showed no sign of microcephaly at weeks 13, 16, and 17, according to a report published online in The New England Journal of Medicine. “While this is a single case, it poses troubling questions that could inform future research,” says the study’s co-senior author, Adre du Plessis, M.B.Ch.B., Director of the Fetal Medicine Institute and Chief of the Fetal and Transitional Medicine Division at Children’s National Health System. “Evidence is mounting that the Zika virus can persist in pregnant women’s bloodstreams weeks after their initial infection, arguing for changes to how these pregnancies are monitored,” Dr. du Plessis said. Six of the named authors are affiliated with Children’s National, where the pregnant woman sought more thorough assessment after testing positive for the Zika virus herself following international travel.

Neonatology updates: U.S. News ranking, skin-to-skin snuggling

June 21, 2016Children’s National ranked in top 20 in every specialty
U.S. News & World Report 2016-17 Best Children’s Hospital Survey ranks Children’s National Health System in the top 20 in every specialty, which makes Children’s one of just four pediatric hospitals in the country—and the only one in the region—to earn this recognition. Children’s ranked among the top 10 in three specialties: Neonatology (No. 3), neurology/neurosurgery (No. 8), and orthopaedics (No. 9).

Oct. 23, 2015Parental stress before and after skin-to-skin contact in the NICU
While stable parent-child bonds are key to healthy child development, achieving such bonding can be complicated for parents of babies born prematurely. Interim results from an ongoing study conducted in the neonatal intensive care unit indicate that skin-to-skin “snuggling” between mothers and babies can lower maternal stress levels.

Allergy and immunology update: asthma care, microbial signatures

June 16, 2016 – Increased identification of the primary care provider as the main source of asthma care among urban minority children
The research team used electronic communication between an asthma specialty clinic and short-term care coordination to encourage parents of urban youth with asthma to identify their primary care provider as the key source for episodic asthma care – rather than the emergency department. Guardians of 50 children were enrolled in the prospective cohort study, whose findings were published in Journal of Asthma. The youths’ median age was 5.8 years; 64 percent were male, 98 percent were African American. At three and six months after the intervention, 85 percent and 83 percent, respectively, reported that the primary care provider was their child’s primary asthma healthcare provider, compared with 70 percent at baseline. 

June 16, 2016 – Two sampling methods yield distinct microbial signatures in the nasopharynges of asthmatic children
The nasopharynx acts as an anatomical reservoir from which pathogenic microbes spread to the lower and upper respiratory airways, causing respiratory infections. A team led by Children’s National researchers used targeted 16S rRNA MiSeq sequencing and two techniques – nasal washes and nasal brushes – to characterize the nasopharyngeal microbiota in 30 children with asthma aged 6 to 17. The authors report in Microbiome that the children’s nasopharyngeal microenvironments contain microbiotas with different diversity and structure.

Nov. 30, 2015 – Alex’s Lemonade Stand Foundation grant to develop immune-based therapy
Physician-scientist Conrad Russell Y. Cruz, MD, PhD, was awarded a $450,000, grant from the Alex’s Lemonade Stand Foundation to develop novel cell-based therapies to combat pediatric cancer. The “A” grant encourages scientists to develop innovative treatments and cures that impact children with cancer and will provide Dr. Cruz and his team funding for three years.

Feasibility of home-based computerized working memory training with sickle cell disease patients

Children with sickle cell disease are at heightened risk for neurocognitive deficits. The research team sought to fill a gap in the research by evaluating the feasibility of using a home-based computerized working memory (WM) training intervention for children aged 7 to 16 years with sickle cell disease. Study participants used loaner iPads and were asked to work on Cogmed five days a week for five weeks – or a maximum of 25 sessions. According to research published by Pediatric Blood and Cancer, girls were more likely to complete the assignments, compared with boys. The mean number of sessions completed was 15.83.

Cardiology and heart surgery update: fetal magnetic resonance imaging, chest pain

July 20, 2016Utility of fetal magnetic resonance imaging in assessing the fetus with cardiac malposition
Abnormal cardiac axis and/or malposition can trigger an evaluation of fetuses for congenital heart disease. A research team led by Mary T. Donofrio, MD, director of the Fetal Heart Program at Children’s National Health System, sought to examine how fetal magnetic resonance imaging (fMRI) – might complement obstetrical ultrasound or fetal echocardiography (echo) – in defining etiology. The team reviewed 42 fetuses identified as having abnormal cardiac axis and/or malposition by fetal ultrasound and echo. While 55 percent of cases (23) had extracardiac anomalies, 29 percent (12) were reassigned by fMRI. fMRI findings were confirmed in 8 of these 12 cases postnatally.

June 13, 2016 – Targeted echocardiographic screening for latent rheumatic heart disease in Northern Uganda
Echocardiographic screening to detect latent rheumatic heart disease (RHD) has the potential to reduce the burden of disease, however additional research is needed to develop sustainable public health strategies. Some 33 million people, many living in low-resource environments, have RHD. What’s more, relatives of children with latent RHD may be at risk for developing the chronic heart condition. The research team found that siblings of children who were RHD-positive were more likely to have RHD, underscoring the importance of screening brothers and sisters of a child with confirmed RHD.

April 3, 2016 – Chest pain in children – the charge implications of unnecessary referral
While pediatricians are responsible for triaging chest pain complaints, questions linger about the best approach to reassure patients whose conditions are benign as well as how to best identify patients whose chest pain warrants further evaluation and testing. The study sought to assess how many patients with chest pain were inappropriately referred and found that chest pain due to cardiac disease is very rare in children. Thus, children whose chest pain is not accompanied by cardiac red flags can be managed safely by their pediatrician.

April 2, 2016Hemodynamic consequences of a restrictive ductus arteriosus and foramen ovale in fetal transposition of TGA
Dextro-transposition of the great arteries (d-TGA) occurs when the position of the main pulmonary artery and the aorta – the two main arteries that carry blood out of the heart – are switched. Newborns with d-TGA are at risk for compromise due to foramen ovale (FO) closure and pulmonary vascular abnormalities. One such fetus seen at 22 weeks of gestational age had a hypermobile, unrestrictive FO and small ductus arteriosus (DA) with bidirectional flow. By the 32 week, however, the DA was small with restrictive bidirectional flow. Doppler imaging showed reversed flow in the left pulmonary artery. By the 38th gestational week, the FO was closed, the left atrium/ventricle were dilated, and the DA was tiny. Within 30 minutes after birth, a balloon atrial septostomy was performed, and the infant later underwent surgical repair.