Stories about: Pediatrics

Progress in the treatment of childhood leukemia

Although treatments for childhood cancer patients are improving, cancer remains the leading cause of death by disease in children. Doctors and researchers are also focused on decreasing the toxicity of these treatments, which can have side effects years after a child finishes treatment.

“The war against childhood cancer is hardly over,” says Kimberly Stegmaier, MD, a pediatric oncologist at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. “We need to do better.”

Stegmaier, who focuses her research on identifying new drug targets and new drugs for childhood leukemiaEwing sarcoma, and neuroblastoma, recently discussed advances in childhood cancer treatment in a Science, Innovation, and Discovery Talk (SID Talk) at Dana-Farber. During the TED Talk-style presentation, Stegmaier explained some of her research in the treatment of sub-microscopic acute lymphoblastic leukemia (ALL) as well as genetic targets in childhood cancers.

“What you can do in an environment where you have chemists, biologists, and clinicians adjacent and working collaboratively is very powerful,” says Stegmaier. “That’s why I’m here today—we need to cure 100 percent of kids, and we can’t do this alone.”

This story originally ran on Dana-Farber Cancer Institute’s Insight blog.

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The autism-GI link: Inflammatory bowel disease found more prevalent in ASD patients

brain gut connection autism IBD ASDReports from parents and a growing number of studies over the past 10 to 15 years suggest that children with autism spectrum disorder (ASD), especially more severe ASD, are prone to gastrointestinal disorders. Researchers have attributed the association to altered GI microbiota, abnormal intestinal physiology, immune alterations and other mechanisms. Some speculate that the connection results from unusual eating patterns in children with ASD.

A 2012 study led by bioinformatician Isaac Kohane, MD, PhD, of Boston Children’s Hospital and Harvard Medical School grouped autism patients according to the gene expression patterns in their blood, and one group had altered immunologic and inflammatory pathways. A more recent study went a step further, finding similar gene expression profiles in the intestines of children with ASD and those with inflammatory bowel disease (IBD).

Looking at IBD (Crohn’s and colitis) sets the bar a little higher, since IBD is uncommon and also unlikely to be caused by dietary factors (though it can certainly be aggravated by them). In a new study in the journal Inflammatory Bowel Disease, Kohane and colleagues crunched three large databases to create what they believe is the largest ASD/IBD study to date.

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New and improved device shows promise for pediatric heart surgery

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In the Norwood procedure for HLHS, a graft creates a conduit between the right ventricle and the aorta, diverting blood flow from the underdeveloped left ventricle. But that graft can wear out. (BLUE represents oxygen-poor blood; RED, oxygen-rich blood; PURPLE, a mixture of the two.)

Hypoplastic left heart syndrome (HLHS) is a rare but serious form of congenital heart disease that leaves the left pumping chamber (ventricle) of the heart severely underdeveloped. Children born with HLHS can’t pump enough oxygenated blood from their heart to the rest of their body and need surgery as soon as possible to survive. Treatment ultimately involves three corrective surgeries throughout the infant and toddler years.

The first surgery, known as the Norwood procedure, is the riskiest of the three. Ideally performed within the first week of life, the procedure re-routes the heart’s plumbing to ensure enough oxygenated blood is circulated while the child grows big enough for the second surgery. A device called a graft is used to connect the fully-functional right ventricle to the aorta, bypassing the stunted left ventricle, for proper blood flow. However, with each ventricular contraction, the graft gets squeezed, which can cause it to shift or lose its shape over time. Repeat interventions to adjust the graft are often needed.

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Does autism stress families’ chromosomes?

telomeres autismThe ends of our chromosomes have little caps called telomeres that keep our DNA from degrading as our cells divide. Telomere length is partly determined by genetics. However, telomeres also shorten as we age and as a result of health conditions, including stress—as in institutionalized children in Romania and women caring for children with chronic illnesses.

An intriguing new study in the July Journal of the American Academy of Child and Adolescent Psychiatry finds shortened telomeres not just in children with autism (confirming a recent study from China) but also in their infant siblings and their mothers. An effect was also seen in fathers, but it didn’t reach statistical significance.

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Power to the people: Citizen science meets precision medicine for rare disease

At this recent GoldLab Symposium presentation in Colorado, parent Matt Might shows how it’s done.

People credit rapid next-generation gene sequencing for the increased pace of medical discovery. But patients and their families—especially those with rare or undiagnosed conditions—are emerging as the true engines of precision medicine. Racing against the clock to save their children, parents are building databanks, connecting scientific dots and fueling therapeutic advances that could otherwise take a decade or more to happen.

“There’s a culture shift,” said Isaac Kohane, MD, PhD, chair of Harvard Medical School’s Department of Biomedical Informatics (DBMI), which hosted a conference titled Precision Medicine 2015: Patient Driven in late June. “A culture shift where patients feel empowered morally and intellectually to lead in precision medicine research and delivery.”

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Crisis Care: A unique suicide prevention app

teen with phone

More than 100,000 smartphone apps are currently categorized as “health apps.” There are apps for physical health—apps that log work-outs, track nutritional intake, and monitor sleeping patterns. And there are apps for mental health—apps that identify your mood, guide meditation and alleviate depression. But can an app tackle a public health problem as serious as teen suicide?

Turns out, mobile phones and suicide prevention may not be such strange bedfellows.

Elizabeth Wharff, PhD, and Kimberly O’Brien, PhD, clinician-researchers from the Department of Psychiatry at Boston Children’s Hospital, specialize in working with adolescents who struggle with suicidal thoughts. Noting that teens are already turning to their phones whenever they need something, they believe a mobile app may be the perfect platform to support them through tough times. Wharff feels that existing apps designed to help with depression and anxiety lack something crucial: parent mode.

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Early adversity and the brain: Bangladeshi children may provide lessons

neuroimaging adversity Bangladesh
Children from the neighborhood around the neuroimaging lab

Dhaka, Bangladesh, is a megacity, one of the world’s fastest growing. By 2025, the U.N. predicts, Dhaka will be home to more than 20 million people as rural migrants swell its population. Many residents live in extreme poverty, crowded into dense, hot, chaotic slums with open sewers and corrugated housing.

While traditional global health programs have focused on curbing infectious disease, low-resource settings like Dhaka are also coming to be seen as “living laboratories” for investigating how adversity affects children’s brain development. Last year, the Bill & Melinda Gates Foundation awarded a two-year, $2.5 million grant to Charles Nelson, PhD, to bring the first fully equipped neuroimaging facility to Bangladesh.

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‘Huggable’ robot may ease kids’ hospital stress


Can a robotic talking bear have therapeutic value? “The Bear,” part of a New York Times video series called Robotica, offers a glimpse of Huggable’s potential when Beatrice Lipp, a child with a chronic medical condition, visits the hospital, nervous about what’s to come.

“We want to offer kids one more way of helping them to feel OK where they are in what’s otherwise a really stressful experience,” explains Dierdre Logan, PhD, director of Psychological Services for Pain Medicine at Boston Children’s Hospital.

Huggable, a creation of the MIT Media Lab’s Personal Robots Group and the Boston Children’s Simulator Program, comes into Beatrice’s room to chat, play games like “I Spy” and tell jokes. The session is recorded on video, and a bracelet called a Q Sensor collects Beatrice’s physiologic data–changes in skin conductance, temperature and motion that may indicate distress. Researchers at Northeastern University are analyzing these data to gauge the robot’s effect. Eventually, Huggable will be able to react to the data and respond accordingly—offering relaxation exercises and guided imagery, for example, if a child remains anxious.

Currently, Huggable is voiced by Child Life staff, but the ultimate goal is for it to work autonomously. Beatrice is part of a 90-child study comparing Huggable, an ordinary teddy bear and a tablet Huggable image.

I admit: My immediate thought on seeing Huggable was that kids would immediately see him (her?) as a fake, but the bear’s robotic nature doesn’t seem to faze them. As Logan says in the video:

I think there’s a way of connecting with kids that’s different than what grownups have to offer. They have incredible imaginations. And they can really suspend disbelief. There can be a true relationship that develops between Huggable and a patient.

See another child interacting with Huggable.

 

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The diagnostic odyssey: Parents shed light on their experience

the diagnostic journey
Robert Salmon: Storm at sea (Wikimedia Commons)

Nikkola Carmichael, MS, CGC, is a parent and a genetic counselor in the adult genetics clinic at Brigham and Women’s Hospital. Her research was conducted as part of her master’s degree in genetic counseling in conjunction with colleagues at Boston Children’s Hospital.

When a parent or provider first becomes concerned about a child’s development, a diagnostic odyssey begins. It may be brief or can stretch for years as a child undergoes multiple procedures and medical appointments in the search for a diagnosis.

This is a challenging time for families. While learning to address their child’s health needs and fearing for the future, parents may have difficulty accessing support services due to the lack of a diagnosis. Against this backdrop of emotional turmoil, parents strive to support their child through medical procedures that can be painful or frightening.

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What we’ve been reading: Week of May 18, 2015

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From cancer to feet: the power of Twitter in healthcare (MedCity News)
Why does Twitter care about the healthcare industry? Craig Hashi, one of two Twitter engineers dedicated to healthcare, details the opportunities.

MIT’s implantable device could help docs determine best cancer medicine (Boston Business Journal)
Removing the trial and error associated with cancer drug treatments is high on oncologists’ wish lists. Heeding that call, MIT has developed an implantable device (about the size of a grain of rice) that can carry up to 30 different drug doses to a cancerous tumor, and then be removed to test responses.

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