Stories about: Pediatrics

Effective vaccination of newborns: Getting closer to the dream

 

newborn vaccines global health

In many parts of the world, babies have just one chance to be vaccinated: when they’re born. Unfortunately, newborns’ young immune systems don’t respond well to most vaccines. That’s why, in the U.S., most immunizations start at two months of age.

Currently, only BCG, polio vaccine and hepatitis B vaccines work in newborns, and the last two require multiple doses. But new research raises the possibility of one-shot vaccinations at birth — with huge implications for reducing infant mortality.

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Lack of drug testing in kids exposes them to off-label harm

drug testing in children
Loopholes in current legislation let drug companies defer testing their products in children, so the majority of prescriptions are off-label.

Florence T. Bourgeois MD, MPH, is assistant professor of Pediatrics and Emergency Medicine at Harvard Medical School, faculty in the Computational Health Informatics Program, and Scientific Director of the Biobank for Health Discovery at Boston Children’s Hospital

Every day, more than half of children seen in outpatient clinics are prescribed a medication that is not FDA approved for the child’s age or diagnosis. Such off-label prescribing is widespread across pediatric conditions and treatment settings and as many as 90 percent of pediatricians have knowingly prescribed off-label medications.

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Abraham Rudolph, MD: The path of a pediatric cardiology pioneer

Abraham Rudolph MD
Rudolph (left) at Boston Children’s Hospital with Cardiologist-in-Chief Alexander Nadas, MD, c. 1958.

Abraham Rudolph, MD, who recently turned 93, has watched his chosen corner of the medical profession — pediatric cardiology — grow from rudimentary beginnings into a robust, multivariate discipline. Yet while his name is known worldwide in pediatric cardiology circles, he entered cardiology more than 50 years ago only by happenstance.

Born in South Africa in 1924, Rudolph came to the United States in 1951 to train in cardiology by invitation of Charles Janeway, MD, then Physician-in-Chief at Boston Children’s Hospital. Concerned about providing for his wife and newborn daughter, he chose cardiology over hematology or neurology because it offered a salary; many other physician-training opportunities at the time were unpaid. That first year, as the hospital’s first cardiology fellow, he made $3,000 — thanks to a family donation.

He stayed on for nine years, becoming director of the cardiac catheterization laboratory. There he found his focus.

“I became more and more interested in the physiology of the circulation, particularly the problems surrounding infancy,” he said in a 1996 interview with the American Academy of Pediatrics. “At that time, there were relatively few places that were doing anything about infants with heart disease. Most of the emphasis was on older children.”

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2017 predictions for biomedicine

2017 predictions for biomedicine

David Williams, MD, is Boston Children’s Hospital’s newly appointed Chief Scientific Officer. He is also president of the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and director of Clinical and Translational Research at Boston Children’s. Vector connected with him to get his forecast on where biomedical research and therapeutic development will go in the year ahead.

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Why we’re glad the Cures Act passed: Its provisions benefit children

Cures Act children
Via @POTUS

Amy Judge DeLong is manager of Federal Government Relations at Boston Children’s Hospital.

In the midst of a seismic shift in Presidential administrations and anticipation of the incoming Congress, a landmark medical research bill with several provisions important to children cleared the lame duck session of Congress. The 21st Century Cures Act (Cures) is the end result of nearly three years of bipartisan Congressional activity. Last week, it was signed into law.

Cures includes scores of provisions aimed at strengthening National Institutes of Health funding for medical research and accelerating review efforts at the Food and Drug Administration. The law cleared Congress with overwhelming majorities, an example of bipartisanship that may be challenged in the months ahead.

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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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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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Topical antibiotics for otitis media: A one-squirt cure?

otitis media transtympanic gel
A single-application gel could revolutionize treatment of ear infections, reducing side effects and drug resistance. (Click to play animation.) Credit:Kohane group

Otitis media, or middle-ear infection, affects 95 percent of children and is the number one reason for antibiotic prescriptions in pediatrics. Typically, antibiotic treatment involves 7 to 10 days of oral medication — several times a day — a formidable task for parents of little kids.

“Force-feeding antibiotics to a toddler by mouth is like a full-contact martial art,” says Daniel Kohane, MD, PhD, a pediatrician and director of the Laboratory for Biomaterials and Drug Delivery at Boston Children’s Hospital.

A single-application bioengineered gel could be the answer to parents’ and pediatricians’ prayers. Described in a paper published today in Science Translational Medicine, the gel would provide an entire course of therapy through a single squirt into the ear canal. It was developed by Kohane’s team in collaboration with investigators at Boston Medical Center and Massachusetts Eye and Ear.

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The 21st Century Cures Act: Addressing unmet needs in children with rare disease

21st Century Cures Act and children
Among its other provisions, the Cures Act would advance implementation of the 2013 National Pediatric Research Network Act, boosting therapeutic development for rare childhood diseases.

Medical solutions often require countless hours of investigation, months of testing and monitoring, years of post-trial and market analysis and billions of dollars of investment — with no certainty of success.

Last year, after years of groundwork, the U.S. House of Representatives passed the 21st Century Cures Act. A companion measure is being developed in the Senate, and stakeholders are optimistic that agreement on a package — even a slimmed down bill — could happen this year.

While Congress has addressed research and medical product regulatory needs before, the Cures Act has been unique in its comprehensive approach, looking at all elements of the research spectrum — from basic discovery science to translational research to regulatory review. It would upgrade the National Institutes of Health’s research capabilities and update the Food and Drug Administration’s approval policies to get new drugs and devices to the clinic sooner.

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BCL11A-based gene therapy for sickle cell disease passes key preclinical test

sickle cell gene therapy coming
(unsplash/Pixabay)

Research going back to the 1980s has shown that sickle cell disease is milder in people whose red blood cells carry a fetal form of hemoglobin. The healthy fetal hemoglobin compensates for the mutated “adult” hemoglobin that makes red blood cells stiffen and assume the classic “sickle” shape.

Normally, fetal hemoglobin production tails off after birth, shut down by a gene called BCL11A. In 2008, researchers Stuart Orkin, MD, and Vijay Sankaran, MD, PhD, at the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center showed that suppressing BCL11A could restart fetal hemoglobin production; in 2011, using this approach, they corrected sickle cell disease in mice.

Now, the decades-old discovery is finally nearly ready for human testing — in the form of gene therapy. Today in the Journal of Clinical Investigation, Dana-Farber/Boston Children’s researchers report that a precision-engineered gene therapy vector suppressing BCL11A production overcame a key technical hurdle.

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