At least 15 million children reside in Health Professional Shortage Areas (HPSAs) that average fewer than one health professional for every 3,500 people. In these health care deserts, time and transportation barriers prevent even children with health insurance have trouble getting timely care, particularly specialty care. Children in poor, rural areas are most at risk.
So health problems fester and get worse — and more expensive when finally addressed.
Telehealth can solve many of these problems. Through remote video/voice/data connections, dermatologists can view images of rashes and moles sent by primary care providers; cardiologists can patch into local emergency rooms and listen to heart sounds and read EKG tracings; critical care physicians and neonatologists can see and hear newborns in distress, listen to lung sounds, read their vital signs and view images. They can advise local clinicians and guide them through next steps.
Although there are more than 150 types of childhood cancer, pediatric cancer receives only a small fraction of NIH and National Cancer Institute funding, Williams writes. Yet, he points out, just as breakthroughs in adult cancer research can help children, breakthroughs in pediatric cancer can also benefit adults.
Williams and other members of the Coalition for Pediatric Medical Research recently met with the staff of Vice President Joseph Biden, leader of the federal government’s cancer moonshot. Their message? Make sure that pediatric cancer is represented on the moonshot.
What are the public health consequences of freely available weed — both acute and long-term? Are we making a big mistake here?
Concerned about potential harms to adolescents, Sharon Levy, MD, MPH and Elissa Weitzman, ScD, Msc, of Boston Children’s Hospital’s divisions of Developmental Medicine and Adolescent/Young Adult Medicine respectively, argue for a better, real-time marijuana surveillance system in this week’s JAMA Pediatrics. …
Most adult transplant centers require patients to walk a set distance in under six minutes to remain a good candidate for lung transplant. The thought is that if patients cannot meet this minimal threshold, then their chances of being able to rehabilitate after transplant are diminished. In pediatrics, this is also important. But Dawn Freiberger, RN, MSN, Boston Children’s Hospital’s Lung Transplant coordinator, says there are other factors that have to be considered.
“The walk test is just one piece of the pie,” says Freiberger.
“Precision medicine” looks to be heading down the same path as “big data” and “innovation”: The term is becoming so widely used that it threatens to detract from the real impact it is already having in patients’ lives.
But for children, who are still developing and have the most to gain, precision medicine is more than a bumper sticker. On the micro scale, early genetic testing—perhaps routinely, someday, in newborns—can help guide medical care, targeted therapies and preventive strategies based on a child’s genetic makeup. On a macro scale, big data from the larger population becomes a predictive tool, guiding medical decisions that could be life-altering in a still-malleable child.
“If you can make an early diagnosis, you can amplify the effects of what you do, rather than try to change the highways once they’re built,” said Wendy Chung, MD, PhD, of Columbia University Medical Center during a panel discussion last week at Boston Children’s Hospital’s Global Pediatric Innovation Summit + Awards (#PedInno15). …
Funding drives biomedical research, and research drives treatment innovation. Access to funds, particularly National Institute of Health (NIH) awards, is critical to move research forward. The 21st Century Cures Act, which passed the U.S. House on July 10, could give the NIH $8.75 billion more in new grants to disperse over the next five years, the largest increase since the Recovery Act of 2009.
How would those funds be used? Can research find a better way to treat patients? Prevent disease? Disseminate advances in medicine?
In 2014, Boston Children’s led the U.S. in NIH awards. Here’s a look at how a few research teams are leveraging NIH funding to improve care for both children and adults.
A report this April rocked the scientific world: scientists in China reported editing the genomes of human embryos using CRISPR/Cas9 technology. It was a limited success: of 86 embryos injected with CRISPR/Cas9, only 71 survived and only 4 had their target gene successfully edited. The edits didn’t take in every cell, creating a mosaic pattern, and worse, unwanted DNA mutations were introduced.
“Their study should give pause to any practitioner who thinks the technology is ready for testing to eradicate disease genes during [in vitro fertilization],” George Q. Daley, MD, PhD, director of the Stem Cell Transplantation Program at Boston Children’s Hospital, told The New York Times. “This is an unsafe procedure and should not be practiced at this time, and perhaps never.”
As Daley detailed last week in his excellent presentation at Harvard Medical School’s Talks@12 series, the report reignited an ethical debate around tampering with life that’s hummed around genetic and stem cell research for decades. What the Chinese report adds is the theoretical capability of not just changing your genetic makeup, but changing the DNA you pass on to your children. …
Severe social and emotional deprivation in early life is written into our biochemical stress responses. That’s the latest learning from the long-running Bucharest Early Intervention Project (BEIP), which began in 2000 and has been tracking severely neglected Romanian children in orphanages. Some of these children were randomly picked to be placed with carefully screened foster care families, and they’ve been compared with those left behind ever since.
While studies in rodents have linked early-life adversity with hyper-reactivity of the sympathetic nervous system and the hypothalamic–pituitary–adrenal (HPA) axis, the relationship has been harder to pin down in humans. BEIP’s study, involving almost 140 children around the age of 12, had children perform potentially stressful tasks, including delivering a speech before teachers, receiving social feedback from other children and playing a computer game that malfunctioned partway through.
Unlike the rodents, the institutionalized children had blunted responses in the sympathetic nervous system, which is associated with the “fight or flight” response, and in the HPA axis, which regulates production of the stress hormone cortisol. The researchers note that this dulled physiologic response has been linked to health problems, including chronic fatigue, pain syndrome and auto-immune conditions, as well as aggression and behavioral problems. …
Can sequencing of newborns’ genomes provide useful medical information beyond what current newborn screening already provides? What results are appropriate to report back to parents? What are the potential risks and harms? How should DNA sequencing information be integrated into patient care?