Cardiac RESTORE: Decreasing medication dependency in the cardiac intensive care unit

child getting medicineChildren undergoing heart surgery need strong sedation and pain medications. Weaning them off these medications is complicated; many have withdrawal symptoms that require additional medications. Unfortunately, says Patricia Lincoln, RN, MS, CCRN, CNS-BC, “the medications we use to manage withdrawal may keep patients in the hospital longer.”

Last spring, Lincoln and her nursing colleagues in the Boston Children’s Hospital Cardiac Intensive Care Unit (CICU) launched an initiative called Cardiac RESTORE to help wean patients from pain and sedation medications according to a carefully designed algorithm.

“Cardiac RESTORE helps us continually assess what patients need and regulate their physiologic response to changes,” says Lincoln. “Medication doses are constantly being titrated or weaned unless the patient has an acute deterioration.”

Early results show decreased usage of pain and sedation medications with no ill effects.

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Q&A: Mosquitos, Zika virus and microcephaly in Brazil

mosquito-Thriving

As you may have heard, Brazil is facing a startling outbreak of microcephaly, a rare condition in which a child is born with a head and brain that are much smaller than normal. Microcephaly is almost always associated with neurologic impairment and can be life-threatening.

The epidemic has been linked to an influx of the mosquito-borne virus Zika, first detected in Brazil last April. This past Friday, January 16, the Centers for Disease Control and Prevention issued travel warnings advising pregnant women to avoid visiting El Salvador, French Guiana, Guatemala, Haiti, Honduras, Martinique, Mexico, Panama, Paraguay, Suriname, Venezuela and Puerto Rico. And over the weekend, the first U.S. case of microcephaly linked to Zika reportedly surfaced in Hawaii.

Why this virus, why now? And how can a virus affect someone’s head size? In this Q&A on our sister blog, Thriving, Ganeshwaran Mochida, MD, of Boston Children’s Brain Development and Genetics (BrDG) Clinic, who specializes in microcephaly, and Asim Ahmed, MD, an infectious disease specialist at Boston Children’s, offer their insights.

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Drug ‘cocktail’ could restore vision in optic nerve injury

regenerating nerves-cropped
Gene therapy achieved extensive optic nerve regeneration, as shown in white, but adding a potassium channel blocking drug was the step needed to restore visual function. In the future, it might be possible to skip gene therapy and inject growth factors directly. (Fengfeng Bei, PhD, Boston Children’s Hospital)

When Zhigang He, PhD, started a lab at Boston Children’s Hospital 15 years ago, he hoped to find a way to regenerate nerve fibers in people with spinal cord injury. As a proxy, he studied optic nerve injury, which causes blindness in glaucoma — a condition affecting more than four million Americans — and sometimes in head trauma.

By experimenting with different growth-promoting genes and blocking natural growth inhibitors, he was able to get optic nerve fibers, or axons, to grow to greater and greater lengths in mice. But what about vision? Could the animals see?

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When preclinical studies get dosing wrong, new drugs get lost in translation

Robin Kleiman, PhD, is Director of Preclinical Research at Boston Children’s Hospital’s Translational Neuroscience Center.

lab mice cropped shutterstock_6792013

Basic research investigators are increasingly conducting translational research studies to advance their therapeutic approaches to clinical trials. Unfortunately, when testing drugs in rodent models of human disease, these studies often do not measure drug levels from their animal subjects.

This is understandable, since collecting these data can be very expensive and requires specialized expertise. But as a consequence, a lot of preclinical literature is published without any consideration of what drug concentration was actually achieved in the organ of interest. This is undercutting our efforts to get new therapies to patients.

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Interpreting genomes in undiagnosed patients: Our CLARITY Challenge experience

Peter White BGC Director High ResPeter White, PhD, Assistant Professor of Pediatrics and Director of the Biomedical Genomics Core at Nationwide Children’s Hospital, led the winning team in CLARITY Undiagnosed, an international challenge that interpreted genomic data from five families with undiagnosed conditions. The team also took part in CLARITY’s first challenge in 2012, receiving special mention. Here, White describes the team’s approach to these “toughest of the tough” patients.

The CLARITY Undiagnosed challenge was markedly harder than the first CLARITY challenge. This time around, we were given whole-genome sequence datasets for five families and asked to produce clinically useful results through improved interpretation and reporting. It turned out to be a fantastic learning experience for all of us, and we will be using the collaborative approach we developed to solve genomics challenges in our own patients.

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Digital health, innovation and partnerships: A Q&A with Boston Children’s Chief Innovation Officer

Brownstein
Brownstein

During the last decade or so, health care has been rapidly transforming from a reactive, paper-based system to a responsive digital model.

Massachusetts, under Gov. Charlie Baker’s leadership, has launched a comprehensive public-private partnership to accelerate the state’s digital health care sector. The partnership has identified multiple ways to drive investment and growth in the state.

Technology transfer from universities to private companies is just one example. In the past, each transfer required completely new agreements. Three new standardized templates for licensing, technology transfer and sponsored research will help facilitate these processes. In 2016, the partnership will expand its Mentorship Speakers Series with a stronger focus on digital health care. Finally, the Digital Healthcare Innovation Hub and Accelerator will provide a space to support and grow new digital health companies in Boston.

Vector visited with John Brownstein, PhD, Boston Children’s Hospital’s Chief Innovation Officer, to better understand the background and potential impact of this new initiative.

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9 science and innovation predictions for 2016

Everett Collection/Shutterstock
Everett Collection/Shutterstock

What does 2016 have in store in the realm of science and clinical innovation? Vector asked clinical, digital and business leaders from around Boston Children’s Hospital to offer their forecasts.

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How can we make personalized therapy for childhood cancer a reality?

For some pediatric cancers, such as acute lymphoblastic leukemia, older forms of therapy — and older ways of defining who receives which therapy — have served well over the last few decades. But that approach is no longer sufficient. Revolutionary gains have been made in adult oncology using personalized genomic therapy — therapy based on matching treatments to the genetic makeup of a patient’s tumor. The time has come to take them to the pediatric space.

But how will pediatric oncology get there? A panel discussion at Boston Children’s Hospital’s Global Pediatric Innovation Summit about personalized cancer genomics — moderated by Bloomberg News’s John Lauerman and featuring Katherine Janeway, MD, clinical director of the Solid Tumor Center at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center — took on this question. Panelists raised four overarching concepts to consider: numbers, sharing, collaboration and incentives.

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In case you missed it: A look back at 2015

Dog looking back-cropped-Kim Britten-shutterstock_306257615It’s been another exciting year in science and innovation at Boston Children’s Hospital. Read on for a few Vector and audience favorites in science and technology.

 

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Genetic factors linked with neurodevelopmental delays in children with congenital heart disease

brain geneticsAs survival has improved dramatically for children with even the most serious forms of heart disease, neurodevelopmental disabilities have been increasingly recognized. These can affect not only school performance, but also future employment, quality of life and social relationships.

“We’ve known for a while that children with congenital heart disease (CHD) have a higher risk of developmental delays,” says Amy Roberts, MD, a genetic cardiologist at the Boston Children’s Hospital Heart Center. “There are multiple hypotheses as to why that might be, and they’re not mutually exclusive.”

The side effects of surgery, such as oxygen deprivation during bypass, are commonly thought to be to blame. Others suspect problems with the in utero environment. But these factors are not the whole story.

“Even in studies that have measured every known risk factor, only one third of neurodevelopmental disabilities in children with CHD can be explained by factors related to the child’s heart disease, medical history or family factors,” notes Jane Newburger, MD, MPH, director of the Cardiac Neurodevelopment Program at Boston Children’s.

Perhaps there is a genetic component?

In a recent study published in Science, a team of researchers from seven hospitals (Boston Children’s, Brigham and Women’s Hospital, Children’s Hospital of Philadelphia, Columbia, Mount Sinai, Yale and University of California Los Angeles), examined the whole genomes of 1,213 patients with complex CHD, looking for genetic indicators that a child will have developmental delays alongside his or her CHD.

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