Author: Nancy Fliesler

Clinical simulation training goes to the dogs

clinical simulation

Boston Children’s Hospital’s fast-growing Simulator Program, SIMPeds, creates medical scenarios for clinical teams to practice challenging procedures and situations in a risk-free environment. Now serving 27 departments and divisions at the hospital, SIMPeds’ customized simulations prepare clinicians for everything from a Code Blue to complex surgery to breaking difficult news to parents.

At the Simulation Center this week, there was one special team member being trained: Rafa, a Miniature Australian Shepherd auditioning to be part of Pawprints, Boston Children’s dog visitation program. Not all dogs are behaviorally up to the job when confronted with a hospital environment. So the SIM team created a mock intensive-care-unit patient room, fully equipped and complete with an overly enthusiastic child (overwhelming for some dogs), played by SIM engineer Katie Fitzpatrick. As Rafa interacted with the “patient,” the SIM staff set off alarms, had “doctors” and “nurses” come in and out and staged other hospital things that might distract or make a dog skittish.

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Zinc chelation: A better way to regenerate the optic nerve?

optic nerve zinc chelation

For more than two decades, researchers have tried to regenerate the injured optic nerve using different growth factors and/or agents that overcome natural growth inhibition. They’ve had partial success, sometimes even restoring rudimentary elements of vision in mouse models.

But at best, these methods get only about 1 percent of the injured nerve fibers to regenerate and reconnect the retina to the brain. That’s because most of the damaged cells, known as retinal ganglion cells (RGCs), eventually die, says Larry Benowitz, PhD, of the F.M. Kirby Neurobiology Center at Boston Children’s Hospital.

Benowitz and colleagues now show a surprising new approach that gets RGCs to live longer and regenerate robustly: using chelating agents to bind up zinc that’s released as a result of the injury.

These studies, too, were done in mice. If the findings hold up in human studies, they could spell hope for people with optic nerve injury due to trauma, glaucoma or other causes, and possibly even spinal cord injury.

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7 digital health predictions for 2017

digital health predictions

What does 2017 have in store for digital healthcare innovations? Vector connected with clinical, digital health and business experts from the Innovation & Digital Health Accelerator (IDHA) at Boston Children’s Hospital and asked for their predictions.

Overall? “Expect to see a reshaping of the patient journey, more patient-centric care and more clinically impactful technology in 2017,” says John Brownstein, PhD, Chief Innovation Officer at the hospital. “We’re also looking forward to digital health offerings being met by industry-wide adoption as patient-centric care is provided and reimbursed.”

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From math major to transplant pioneer: An interview with William Harmon, MD

William Harmon MD
William Harmon, MD, c. 1996

William Harmon, MD, a pioneer in pediatric dialysis and kidney transplantation, passed away on May 29, 2016 after 45 years at Boston Children’s Hospital. He was 72 years old. Starting as an intern in 1971, the year the hospital performed its first kidney transplant, he worked his way up to Nephrologist-in-Chief, a position he held for 25 years.

Harmon was passionate about caring for children with end-stage renal disease (ESRD), pioneering techniques and devices to adapt hemodialysis to infants and young children. He helped get NIH support for child-specific transplant research and led multiple clinical trials of treatment protocols to help children not only tolerate their transplants, but thrive. He also worked to ensure that government guidelines and legislation on ESRD and kidney transplant gave priority to children.

Harmon was the first chair of the pediatric committee of the United Network for Organ Sharing (UNOS) and the first pediatrician to chair the New England Organ Bank’s Board of Directors. He was passionate about teaching, training more than 65 pediatric nephrology fellows.

Below are lightly edited excerpts from interviews with Harmon, most recently in 2014.

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Real-time contextual information could help doctors interpret children’s brain scans

Radiologists who can tune in to the nuances of brain scans in children are a pretty rarified group. Only about 3 percent of U.S. radiologists, some 800 to 900 physicians, practice in pediatrics. Those specifically trained in pediatric neuroradiology are even scarcer.

To a less trained eye, normal developmental changes in a child’s brain may be misinterpreted as abnormal on MRI. Conversely, a complex brain disorder can sometimes appear normal. That’s especially true when the abnormality affects both sides of the brain equally (see sidebar).

It can be hard to find the cause of a child’s developmental delay without a proper read. “Pediatric brain scans of children under age 4 can be particularly tricky to read because the brain is rapidly developing during this period,” says Sanjay Prabhu, MBBS, a pediatric neuroradiologist at Boston Children’s Hospital. “If you’re looking at adult scans all the time, it’s incredibly difficult to transition to pediatric scans and understand what is considered ‘normal’ and ‘abnormal.’ Clinicians often wonder, ‘Should I repeat the scan? Should I send the patient to a specialist?’”

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Antisense drug for spinal muscular atrophy nears the clinic

spinal muscular atrophy Spinraza
Max High of South Carolina was diagnosed with SMA before the age of 1. (Ron Brinson/Flickr)

Update: Nusinersen received FDA approval on December 23, 2016, and will be marketed as Spinraza.

In recent months, two Phase III clinical trials have shown a clear benefit of nusinersen in children with spinal muscular atrophy (SMA), a genetic motor neuron disease that robs children of muscle control and is the leading genetic cause of infant mortality. The ENDEAR trial, involving infants with the more severe SMA Type 1, was first to terminate randomization in August 2016. The CHERISH trial, involving older children with milder Type 2 SMA, was halted on November 8, 2016, because it also met its efficacy target.

Both trials are now open-label, and the FDA has granted nusinersen a priority review. The drug, formerly called SMNRx and now brand-named Spinraza, is an antisense oligonucleotide works by altering gene splicing (see sidebar).

Vector asked Basil Darras, MD, director of the Spinal Muscular Atrophy Program at Boston Children’s Hospital, to put these developments in perspective. Darras is site principal investigator at Boston Children’s for both trials. The hospital was the first in the world to enroll a child with SMA Type 1 in the ENDEAR study, in 2014.

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Rainbow-hued blood stem cells shed new light on cancer, blood disorders

color-coded blood stem cells
These red blood cells bear color tags made from random combinations of red, green and blue fluorescent proteins. Same-color cells originate from the same blood stem cell (Nature Cell Biology 2016, Henninger et al).

A new color-coding tool is enabling scientists to better track live blood stem cells over time, a key part of understanding how blood disorders and cancers like leukemia arise, report researchers in Boston Children’s Hospital’s Stem Cell Research Program.

In Nature Cell Biology today, they describe the use of their tool in zebrafish to track blood stem cells the fish are born with, the clones (copies) these cells make of themselves and the types of specialized blood cells they give rise to (red cells, white cells and platelets). Leonard Zon, MD, director of the Stem Cell Research Program and a senior author on the paper, believes the tool has many implications for hematology and cancer medicine since zebrafish are surprisingly similar to humans genetically.

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Parents are generally open to placebo use in children – with caveats

placebo use in children

Placebos are a key ingredient of any controlled clinical trial, the yardstick against which experimental drugs are measured. Placebos are also increasingly used as a treatment in their own right, as studies show that they make people feel better through a “mind-body” effect. But do parents find placebos acceptable for their children? A study published today by The Journal of Pediatricsled by Boston Children’s Hospital, found the answer is mostly yes, provided ethical guidelines are followed.

“The question of placebos is more complex when it comes to children, since parents must make medical decisions on their behalf,” says Vanda Faria, PhD, a research fellow at Boston Children’s Hospital’s Center for Pain and the Brain and first author on the study. “Large placebo responses have been seen in a variety of pediatric conditions, and parent’s perceptions can influence how well placebos work. At the same time, little is still known about the potential harms of prolonged drug therapy on children’s development. Sometimes, the best intervention might not involve pharmacotherapy.”

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If I have the mutation, will I get the disease? New research looks at genetic ‘penetrance’

genetic penetranceRecently announced preliminary results of the BabySeq study included pathogenic or “likely pathogenic” variants linked to heart conditions in three apparently healthy babies. Two are being followed at Boston Children’s Hospital and have had cardiac testing. But is this testing necessary, and are these infants truly at risk? It’s too soon to tell.

Then, last week, a report from the Mayo Clinic raised an alarm about overzealous use of genetic testing in healthy individuals. After a 13-year-old boy died from a heart syndrome, about two dozen family members had genetic testing. All tested positive for variants in a gene linked to long-QT syndrome and were diagnosed with the disease. Yet none had cardiac symptoms, and only one had a positive EKG at any point — the boy’s brother, who had a defibrillator implanted. When the Mayo team reanalyzed the test results using a more up-to-date genetic database, they concluded the variant is harmless.

And this week, in Science Translational Medicine, researchers at Brigham and Women’s Hospital, Boston Children’s and Massachusetts General Hospital address the question: If people carry a genetic mutation linked to a condition, what are the chances they will develop that condition over time? As part of the genomes2people project, the researchers tested participants in two long-term population studies — the Framingham Heart Study and the Jackson Heart Study — for 56 genes representing 24 hereditary cancer and cardiac syndromes. They did not know the participants’ actual health status. As it turned out, carrying a mutation increased risk for the related disease 4.7-fold in African Americans and 6.4-fold in European Americans, who had longer follow-up. This was true regardless of family history.

Vector sat down with Nina Gold, MD, a senior resident in Pediatrics and Medical Genetics at Boston Children’s, for her perspective. Gold is a first author on this week’s report.

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It’s not just how long teens sleep, but when, that’s important to self-regulation

teen sleep

Chronic insufficient sleep is at epidemic levels in U.S. teens. It’s been associated with depression, substance use, accidents and academic failure. But according to a survey of some 2,000 7th to 12th graders in Fairfax County, VA, the number of hours of sleep isn’t the core problem. It’s being a “night owl” — unable to fall asleep until late at night.

Forced to get up early for school, night owls are in a state of chronic “jet lag” on school days. And that can lead to poor self-regulation, or an inability to alter thinking, emotions and behaviors to meet varying social demands, finds the study, published last week by Pediatrics.

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