Teens with type 1 diabetes can download their blood glucose data and attend "virtual clinics" from home.
Most adolescents fight for the freedom to manage their own lives, especially when it comes to friends, curfews and hobbies. That excitement conspicuously slips away when they’re faced with managing something less glamorous—like diabetes.
Since diabetes is a chronic illness with potentially serious risks, it requires continuous management. But adolescents aren’t exactly lining up around the block for extra medical visits.
“Some adolescents forget to do things like take insulin or check their blood glucose level, and they could benefit from more frequent check-ins with their diabetes team,” says Erinn Rhodes, MD, MPH, director of the Type 2 Diabetes Program and Inpatient Diabetes Program at Boston Children’s Hospital. “But that’s not easy, especially if time is limited or if transportation is a challenge.”
So Rhodes has designed a study for adolescents 13 to 17 years old, to see if “televisits”—video conferences between teens and their diabetes care providers—can improve their diabetes while encouraging better self-management. Full story »
(Teak Sato/Wikimedia Commons)
Research on rare disorders, or in new fields, often follows a particular trajectory. It tends to start out fragmented, carried out by one or two isolated researchers at a few institutions.
But as researchers find each other, identify more patients and start to collaborate systematically, patterns of disease biology emerge, researchers start speaking the same language and new treatments materialize.
The field of complex vascular anomalies—a set of conditions characterized by blood vessels that have not developed normally—is in this kind of early days. In large part this is because they are relatively rare. In addition, few centers worldwide have the multidisciplinary experience to provide comprehensive care to these rare patients.
But a new coalition forming around vascular anomaly research and care could help unravel the biology of vascular anomalies and fashion better treatments for these children by bringing to bear the resources and knowledge of specialists from across the continent. Full story »
A research registry helped Inga Hofmann, MD, PhD, search the genomes of several patients with a rare blood disorder and reveal new mutations behind it. (Michael David Pedersen/Flickr)
To really understand rare conditions, you need a lot of data from a lot of patients. But no one hospital or center usually sees more than a few patients with any given rare disease, precisely because they’re rare.
This is where case registries become important. These research collaborations, which usually span several institutions, typically focus on a single rare disease or a few related conditions, serving as a data warehouse for collecting information from as many patients and as many places as possible.
One such registry based out of Dana-Farber/Boston Children’s Cancer and Blood Disorders Center—the Pediatric Myelodysplastic Syndromes (MDS) and Bone Marrow Failure (BMF) Registry—has recently started to bear fruit, finding that a unique set of mutations in a single gene may play a larger-than-realized role in a group of rare blood diseases. Full story »
Newborns like this child have a high risk of hypothermia, even in warm climates. An innovative warming pad could be one potential fix. (Courtesy of Anne Hansen)
In the United States, we rarely worry about newborn babies getting dangerously cold, but in poorer countries the basic provision of warmth can be extremely challenging. Although the World Health Organization (WHO) considers newborn thermal care
a critical part of neonatal care, hypothermia remains a leading cause of sickness and death globally.
Even in places with warm climates such as sub-Saharan Africa and South Asia, babies can quickly lose heat, and how hypothermia in newborns is treated reveals a dramatic contrast with the developed world.
The playing field may soon get more level, thanks to a device Boston Children’s Hospital’s Anne Hansen, MD, MPH, has been developing with collaborators at Lawrence Berkeley National Laboratory’s Institute for Globally Transformative Technology (LIGTT) since visiting Rwanda in 2010. That device is a warming pad that can keep a newborn warm for hours at a time with no electricity, and which can be used in a home, clinic, hospital or transport setting. Full story »
A Queens College gymnasium served as an evacuation center after Hurricane Sandy.
Shannon Manzi, PharmD, chief pharmacist for the Massachusetts–1 Disaster Medical Assistance Team, directs the Clinical Pharmacogenomics Service at Boston Children’s Hospital and is team leader for Emergency and Combined Services in the hospital’s Department of Pharmacy. With MA-1 DMAT, she has deployed to Louisiana after Hurricane Katrina in 2005 and Hurricane Gustav in 2008 and to Haiti after the 2010 earthquake.
As I watch the Arizona-1 and Texas-3 Disaster Medical Assistance Teams (DMATs) respond to the tornado in Moore, Okla., I know they will serve with great skill and caring. But I wish the Massachusetts-1 DMAT was the team on call this month. Although we’re unlikely to be deployed for this disaster, our hearts are with the people of Moore and all our fellow responders.
Thirteen years ago, I was asked to join the MA-1 DMAT as the pediatric pharmacist. It’s been one of the most grueling and difficult commitments of my life, but I’ve never looked back. I love it.
I have slept for weeks on the ground, not being able to shower or eat anything other than MREs (meals-ready-to-eat)—all while working 18- to 20-hour days. However, I hold no illusions that what we do is heroic. I can go home in two to three weeks to an intact house and family. This is not the case for the people we serve. Full story »
In the developing world, health care providers often don’t have access to diagnostic technologies like the automated lab tests taken for granted in the resource-rich United States. Specimens often have to be sent to a distant central lab, and it can be weeks before an answer wends its way back.
That’s a tough situation when you’re, say, trying to assess whether a patient is having liver toxicity from a drug, such as drugs used to treat tuberculosis (TB) and HIV. By the time the results come back and indicate you need to stop or switch medications, the patient may be long gone, unable to travel back to the clinic.
For the past four years, Nira Pollock, MD, PhD, associate medical director of the Infectious Diseases Diagnostics Lab at Boston Children’s Hospital, has been working with Diagnostics For All (DFA), a nonprofit organization based in Cambridge, Mass., to develop and test a low-cost diagnostic device that works on the spot, involving just a finger-stick and a square of paper. The technology is all in the paper square—using wax printing and microfluidics techniques Full story »
Alyssa Bianca Velasco, ScB, is a clinical data specialist for the Standardized Clinical Assessment and Management Plans (SCAMPs) program at Boston Children’s Hospital.
Engaging clinicians in change will require a cultural shift. (David Oliva/Wikimedia Commons)
Reducing health care costs doesn’t have to involve making sacrifices in patient safety or quality of care or holding clinicians to rigid guidelines. Over the past several years, Boston Children’s Hospital has rolled out a methodology known as Standardized Clinical Assessment and Management Plans (SCAMPs). Described in the May issue of Health Affairs, SCAMPs are based on the idea that clinicians should be able to diverge from established medical best practices, provided they document the reasons and track the results—in essence making continual data-driven modifications to practice.
The success of SCAMPs in reducing practice variability and costs and improving outcomes at Boston Children’s has led other institutions, one by one, to adopt them. In the next phase, we plan to expand SCAMPs much more broadly, creating a network of hospitals that will pool pertinent clinical data into a centralized non-profit institution, the Institute for Relevant Clinical Data Analytics (IRCDA).
I am part of a team that is providing training, analytics and IT support to help make that large-scale implementation happen. Full story »
Maude Tessier, PhD, is assistant director of business development and strategic initiatives in the Technology and Innovation Development Office at Boston Children’s Hospital. Her role is to initiate, develop and realize alliances between Boston Children’s and industry partners. She tweets from @maude_tessier.)
Psyché et l’Amour, François Gérard, 1798
I log on to the Web portal with excitement and set up my profile. I browse for potential matches, reading though all their interests to see if they match my own. I send out requests to meet face to face. I wait. Have I received favorable responses? Were my short email invite and profile enticing enough? Is my dance card getting full?
It’s not a dating website, but rather the prelude to a biotech business partnering conference. In my role as a leader of business development and marketing efforts at Boston Children’s Technology and Innovation Development Office, my objective is to quickly and effectively pitch our most promising work to industry contacts, in hopes of continuing conversations after the conference is over. Attending these conferences is a great way to “break the ice”—and it is key to my success in building relationships and developing partnerships and alliances with life sciences companies.
I liken it to speed and online dating combined. Full story »
Part 2 of a two-part series. (Read part 1.)
Joshua Frase, who died from X-linked myotubular myopathy (MTM), with his father, Paul Frase, in 2006.
Back in the 1990s, rheumatologist Richard Weisbart, MD, of University of California, Los Angeles (UCLA), was studying lupus in a mouse model and found that the mice were making an antibody that had the intriguing ability to get inside tissues and cells.
Weisbart shifted his work away from studying lupus to studying and refining the antibody, called 3E10, and he and others showed that proteins could be delivered into different tissues of the body simply by attaching them to a fragment of 3E10.
Dustin Armstrong, PhD, a postdoc at Novartis at the time, was trying to find molecules that could activate growth in weakened muscles—without activating possibly cancerous growth in other tissues. He saw Weisbart’s work and contacted UCLA. In 2008, he obtained seed money and founded a company around 3E10-based therapeutics for muscular diseases, now known as Valerion Therapeutics (formerly 4s3 Bioscience).
“There’s a huge need for therapies for genetic muscle diseases, and muscle was a tissue we could target well with our technology,” says Armstrong. Full story »