Stories about: Pediatrics

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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Acetaminophen does not aggravate young children’s asthma

asthma
A head-to-head comparison with ibuprofen refutes a link between acetaminophen and asthma exacerbations.

Your toddler is screaming in pain. Her forehead is burning. You rush to your local drugstore. What do you get — Tylenol or Motrin? And by the way, she also has asthma.

Recently, many parents have been under the impression that acetaminophen (Tylenol, etc.) may do more harm than good in young children with asthma.

“There’s been a lot of ‘smoke’ about this, based on a lot of retrospective observational data,” says Wanda Phipatanakul, MD, MS, of Boston Children’s Hospital’s Division of Allergy and Immunology.

The studies in question concluded that the common over-the-counter remedy can cause asthma exacerbations. Reviewing these studies, one author concluded, “Until future studies document the safety of this drug, children with asthma or at risk for asthma should avoid the use of acetaminophen.”

The Acetaminophen Versus Ibuprofen in Children with Asthma (AVICA) trial, led by Phipatanakul for the National Heart, Lung and Blood Institute’s AsthmaNet now sets the record straight.

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More clinical trials in kids? Nearly half are unfinished or unpublished

pediatric trials clinical trials
Of 559 interventional trials in children, 19 percent were stopped early and 30 percent of completed trials remained unpublished several years later, finds a new study. (Vmenkov/Wikimedia Commons)

Recent laws like the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act are encouraging clinical trials in children. Yet, as with adult trials, these trials commonly stall out or, if completed, remain unpublished several years later, finds a study published online today in Pediatrics.

“Our findings may speak to how commonplace discontinuation and non-publication are in medical research in general,” says Natalie Pica, MD, PhD, a senior resident at Boston Children’s Hospital and the study’s coauthor. “We need to make sure that when children participate in clinical trials, their efforts are contributing to broader scientific knowledge.”

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Can asthma be nipped in the bud?

asthma
A multicenter randomized trial is testing omalizumab (Xolair) in wheezy toddlers. (FDA/Wikimedia Commons)

Worldwide, asthma affects an estimated 300 million people, and is expected to surpass 400 million by 2025, according to the World Health Organization. About 1 in 10 U.S. children have asthma, and research shows that the vast majority of them also have allergy. Could that provide a clue to its prevention?

Starting at 2 to 3 years of age, susceptible children start to become sensitized to pollens, mold spores and other airborne allergens. They begin to produce IgE antibodies, which not only trigger allergic reactions but also impair their anti-viral immune responses — potentially leading to more viral infections that can further hasten their progression to asthma.

A multicenter clinical trial, led by Wanda Phipatanakul, MD, MS, of the Division of Allergy & Immunology at Boston Children’s Hospital, now aims to test whether the anti-IgE drug omalizumab (Xolair) can short-circuit this process.

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News Notes: Pediatric science roundup

A quick look at recent research Vector finds noteworthy.

Tracking infants’ microbiomes

cute microbes-shutterstock_317080235-croppedMicrobiome studies are blooming as rapidly as bacteria in an immunocompromised host. But few studies have been done in children, whose microbiomes are actively forming and vulnerable to outside influences. Two studies in Science Translational Medicine on June 15 tracked infants’ gut microbiomes prospectively over time. The first, led by researchers at the Broad Institute and Massachusetts General Hospital, analyzed DNA from monthly stool samples from 39 Finnish infants, starting at 2 months of age. Over the next three years, 20 of the children received at least one course of antibiotics. Those who were repeatedly dosed had fewer “good” bacteria, including microbes important in training the immune system. Overall, their microbiomes were less diverse and less stable, and their gut microbes had more antibiotic resistance genes, some of which lingered even after antibiotic treatment. Delivery mode (cesarean vs. vaginal) also affected microbial diversity. A second study at NYU Langone Medical Center tracked 43 U.S. infants for two years and similarly found disturbances in microbiome development associated with antibiotic treatment, delivery by cesarean section and formula feeding versus breastfeeding.

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Stem cells and birth defects: Could gastroschisis be treated in utero?

gastroschisis birth defects
Although Gianna was treated surgically, Dario Fauza, MD, hopes to someday use stem cells from the amniotic fluid, multiplied and returned to the womb, to naturally heal gastroschisis and other birth defects. (Courtesy Danielle DeCarlo)

Except when spreading awareness about her condition, 6-year-old Gianna DeCarlo prefers not to wear two-piece bathing suits because of the long vertical scar on her stomach. “Even though nobody’s said anything, she feels like she’ll be made fun of,” says her mother, Danielle. “I do what I can to make her love her body.”

Gianna doesn’t remember her three surgeries or the nasogastric tube she needed as an infant, before she was able to eat normally. She was born with gastroschisis, a striking birth defect in which the abdominal wall doesn’t seal fully during fetal development. As a result, her intestines developed outside her body. She was fed through an IV for several weeks, and was finally stitched fully shut at age 2.

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