Author: Justine Varieur

PVNH: Could this genetic disorder have a ‘butterfly’ effect?

The butterfly effect is defined as “the sensitive dependence on initial conditions, where a small change at one place in a deterministic nonlinear system can result in large differences to a later state.” In medicine, the identification of a rare disease or a genetic mutation may provide insights that spread well beyond the initial discovery.

And in genetics, scientists are learning just how widespread the effects are for mutations in one gene: filaminA (FLNA).

FLNA is a common cause of periventricular nodular heterotopia (PVNH), a disorder of neuronal migration during brain development. The syndrome was first described by the late Peter Huttenlocher, MD, and the gene was identified by Christopher Walsh, MD, PhD, of Boston Children’s Hospital.

In normal brain development, neurons form in the periventricular region, located around fluid-filled ventricles near the brain’s center, then migrate outward to form six onion-like layers. In PVNH, some neurons fail to migrate to their proper position and instead form clumps of gray matter around the ventricles.

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Skin-like material can ‘sense’ if surgeons get off track

Experimental setup for calibrating the sensing skin. Each sensor pad is lowered onto the indentation surface placed on a weight-measuring scale. Changes in the channel resistance are processed through analog signal conditioning and a DAC board, and recorded on a computer. (J Neurosurgery: Pediatrics)
Experimental setup for calibrating the sensing skin. Each sensor pad is lowered onto the indentation surface placed on a weight-measuring scale. Changes in the channel resistance are processed through analog signal conditioning and a DAC board, and recorded on a computer. (Images: J Neurosurgery: Pediatrics)

When surgeons perform image-guided minimally invasive procedures using an endoscope, some aspects of visualization and image quality are typically compromised as compared with open surgeries in which the physician can peer into the body. However, a new pressure-sensing material, placed over an endoscope, may someday provide surgeons with additional guidance and protect healthy tissue during these procedures.

“Neurosurgeons, especially pediatric neurosurgeons, are increasingly using neuroendoscopy to perform minimally invasive brain and spine surgery,” notes Patrick Codd, MD, from the Department of Neurosurgery at Boston Children’s Hospital, who was the lead author on a study evaluating this new material.

“Whenever you move to image-guided minimally invasive surgery, there is typically a tradeoff between the resolution of the image and the field of view,” where you have one but not the other, says Pierre Dupont, PhD, chief of Pediatric Cardiac Bioengineering at Boston Children’s and senior author on the study.

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Sticky heart: Novel surgical glue provides promising alternative for heart procedures

Surgical glue interacting with tissue
A close-up view of the adhesive (pink) interacting with collagen tissue (blue). Images courtesy Karp Lab.

A safe and effective adhesive, or glue, that can be used internally in the body has been a pressing need in medicine. Its creation has faced major hurdles—not the least of which is ensuring the glue is nontoxic and capable of repelling fluids—but a new study published today in Science Translational Medicine offers a potential breakthrough.

Congenital heart defects occur in nearly 1 in 100 births, and those that require treatment are plagued with multiple surgeries to deliver or replace implants that do not grow along with the child. Currently, therapies are invasive and challenging due to an inability to quickly and safely secure devices inside the heart. Sutures take too much time to stitch and can cause stress on fragile heart tissue, and the available clinical adhesives are subpar.

“Current glues are either toxic or easily wash out in the presence of blood or react immediately upon contacting water,” says Pedro del Nido, MD, chief of Cardiac Surgery at Boston Children’s Hospital and senior co-author of the study. “The available options also tend to lose their sticking power in the presence of blood or under dynamic conditions, such as in a beating heart.”

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FDA gets serious about improving medical device innovation for kids

FDA entrance-ShutterstockAssisted by a congressional mandate, the FDA has taken a new approach to helping clinical innovators overcome barriers to moving pediatric medical devices from the research stage to commercialization. So says Linda Ulrich, MD, director of the Pediatric Device Consortia Grant Program at the FDA’s Office of Orphan Products Development, who recently spoke at an Innovators’ Forum cosponsored by her office and Boston Children’s Hospital’s Innovation Acceleration Program.

Regulation and reimbursement are the largest barriers to medical device innovation. “The time it takes to develop a medical device and get it to the U.S. market can take a range from 18 months to 10 years,” says Ulrich.

The stages of device development are concept, prototype, preclinical and clinical testing, manufacturing and marketing—only for the device to become obsolete within as little as 18 months after commercialization. Inventors need to consider the return on investment after their products receive regulatory approval, given the time and funding it takes to get them to market, says Ulrich.

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Guidelines urging more cholesterol screening do not result in more kids on drugs

heart_screening cropped ShutterstockDespite recent national pediatric guidelines recommending identification and treatment of children with familial hypercholesterolemia, the use of lipid-lowering treatment has been flat over the past decade in real-world pediatric practice, finds a large multicenter study.

Justin Zachariah, MD, MPH, a pediatric cardiologist at Boston Children’s Hospital, presented the findings this week at the 2013 American Heart Association (AHA) Scientific Sessions. He believes they dispel some critiques of the recent guidelines, particularly concerns that more screening would result in overmedicating the pediatric population.

Extending beyond 2008 recommendations from the American Academy of Pediatrics, the 2011 National Heart, Lung and Blood Institute’s pediatric guidelines call for universal lipid screening and medical treatment for children at highest risk for early cardiovascular disease. One such high-risk condition is familial hypercholesterolemia, a genetic disorder characterized by high blood cholesterol levels, specifically very high levels of low-density lipoprotein (LDL, or “bad” cholesterol) and early coronary events.

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In the ICU, nurse experience and education can mean life or death

Morning rounds on the pediatric cardiac ICU.
Morning rounds on the pediatric cardiac intensive care unit.

Registered nurses (RNs) remain the largest group of health care providers and typically account for the greatest share of most U.S. hospitals’ operating budgets, about 60 percent. In adult hospitals, research has shown a consistently positive effect of increasing percentages of nurses with baccalaureate educations, and linked increased RN staffing and healthy work environments with improved patient outcomes.

However, this assessment has not been conducted in children’s hospitals—until now.

In a study in the Journal of Nursing Administration, nursing leaders from 38 free-standing children’s hospitals explored which nursing and organizational characteristics influence mortality for children undergoing congenital heart surgery.

The study, involving 20,407 pediatric patients and 3,413 pediatric critical care nurses, was led by Patricia Hickey, PhD, MBA, RN, from the Heart Center at Boston Children’s Hospital.

In pediatrics, congenital heart disease is the most common birth defect requiring surgical intervention for survival. Due to their critical care needs, these patients consume a disproportionate share of U.S. hospital resources.

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Blood pressure, cholesterol screening in kids: Guidelines vs. real world?

Primary care visit
Is universal cardiovascular screening supported by the data, and are clinicians ready?
In 2011, the National Heart, Lung, and Blood Institute (NHLBI) guidelines for cardiovascular risk reduction in pediatrics reinforced the recommendation that primary care pediatricians (PCPs) should screen children and adolescents for cholesterol and blood pressure elevations. However, as PCPs try to incorporate it into their well childcare routine, questions are being raised about the practical implications of implementing that recommendation.

Last month, the U.S. Preventive Services Task Force (USPSTF) published its finding that there is not enough evidence to recommend for or against routine screening for primary hypertension in asymptomatic children and teens, repeating its suggestions from 2003. It has issued similar statements about lipid screening.

At this week’s 2013 American Academy of Pediatrics (AAP) conference, Sarah de Ferranti, MD, MPH, director of the Preventive Cardiology Clinic at Boston Children’s Hospital, gave a presentation titled “Universal Lipid Screening: Are Pediatricians Doing It and How Is It Working?” She spoke with Vector about screening both for cholesterol and blood pressure in children.

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Children’s hospital CEOs: Pediatric health care is at a ‘tipping point’

A moment of levity as the panelists discuss health care challenges.
A moment of levity as the panelists discuss challenges in pediatric care and care delivery.
To summarize the state of pediatric health care today, Steven Altschuler, MD, president and CEO of Children’s Hospital of Philadelphia (CHOP), quoted the 1963 movie The Leopard: “Everything must change, so that everything can stay the same.”

He spoke at a panel discussion with two other children’s hospitals’ CEOs—Boston Children’s Hospital’s outgoing CEO James Mandell, MD, and Herman Gray, CEO of Children’s Hospital of Michigan (CHM)—during Boston Children’s Pediatric Health Innovation Summit + Awards on September 27 in Boston. Erik Halvorsen, PhD, director of Boston Children’s Technology and Innovation Development Office, moderated the session on the top challenges in pediatric health care and care delivery.

“For us to continue to support our missions in the traditional manner and for us to continue to advance pediatric health care, we need to change everything, including the reimbursement and research-funding models, as well as the education of our new caregivers,” Altschuler said. In his estimation, medical education has “unprepared new doctors and nurses to practice appropriate medicine in a safe, effective manner. The education is completely out of touch with reality.”

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Industry-researcher partnerships accelerate innovation, but funding remains Achilles’ heel

InnoAccelEarly-stage researchers face a stark economic reality: decreasing available dollars. To address this barrier, a panel of experts at Boston Children’s Hospital’s National Pediatric Innovation Summit + Awards 2013 discussed the shifting of funding from venture capitalists to larger medical device, informatics and pharmaceutical companies.

Alan Crane, general partner at venture capital (VC) firm Polaris Partners, said it is a very difficult time for early-stage life sciences innovation, especially with respect to resource generation. “There were periods when innovation was stronger,” he said. “In 2000, there were 1,000 VC firms, now there are 350. Also, the size of the funds has decreased substantially, particularly for early-stage life sciences. While some biotech firms are going public, this trend hasn’t trickled to the earlier stages.”

To garner funding, Crane said, researchers need a proof of concept—for a clinical model and a business model. This proof of concept will vary based on the product or the nature of the innovation.

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How do you define innovation? Part VIII

innovation-box-cropped-purchased-no-creditOver the past few months, the Vector team has been collecting definitions from varied thought leaders—inside and outside Boston Children’s—and the responses have reflected the varied nature of their respective fields. In this series, the term has been called “clichéd” at one extreme to “necessary” for the evolution of care delivery at the other. This week’s respondents range from former FDA leaders to informatics experts to critical care specialists to bioengineers. Follow our continuing coverage of innovative efforts through this week’s National Pediatric Innovation Summit + Awards.

Innovation is the belief that we have an obligation to move the world forward through positive and lasting change, leaving it better than we found it. —Jeffrey P. Burns, MD, MPH, Chief of Critical Care Medicine; Director, Medical/Surgical Intensive Care Unit, Boston Children’s Hospital

Innovation is the process of significantly and meaningfully changing the way things are done, operate or perform. Most people think of innovation in terms of the individual developing a novel idea and inspiring or causing change. I think what is often overlooked is the power of a large organization to provide meaningful innovation on a broad scale—it’s not just the single individual that can innovate. Great examples of this are the Apollo mission to the moon, Google, the Internet, the Boeing 747 or, in health care, the development of advanced medical devices. The key to innovation, either as an individual or as a large organization, is to provide transformative change. —Robert “Chip” Hance, CEO of Creganna-Tactx Medical; former Entrepreneur-in-Residence at the FDA

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