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Andrew Dauber

Growth disorder study starts by analyzing DNA

The National Institutes of Health has awarded Andrew Dauber, M.D., MMSc, the chief of endocrinology at Children’s National Health System, a five-year grant that will allow four pediatric health systems to compile and study clinical and genetic markers of severe pediatric growth disorders.

The study will use the electronic health records of large health systems combined with DNA samples from dozens of children, with the goal of enabling endocrinologists to detect children with previously undiagnosed severe genetic growth disorders.

“If you’re a pediatrician treating an 8-year-old patient who has stopped growing, the first thing you’ll want to do is determine the underlying cause, which could be due to many factors including a genetic mutation,” says Dr. Dauber. “There are many reasons why children grow poorly and it is often very difficult to figure out what is causing the problem. However, the various causes may be treated quite differently and may alert us to other medical issues that we need to watch out for. We need to be able to identify clues from the patient’s clinical presentation that may point us to the right diagnosis.”

Dr. Dauber and endocrinology researchers from Children’s National Health System, Cincinnati Children’s Hospital Medical Center, Boston Children’s Hospital and The Children’s Hospital of Philadelphia will use electronic health records to identify children who likely have rare genetic growth disorders. They will then use cutting-edge DNA sequencing technologies, whole exome sequences, to identify novel genetic causes of severe growth disorders. Patients with growth hormone resistance, resistance to insulin-like growth factor 1 (IGF-I) and severe short stature inherited from a single parent will be recruited for the initial phases of the study.

“It’s rare to find patients meeting criteria for each of these subgroups, which is why it’s critical to work collaboratively across institutions,” says Dr. Dauber. “This type of genetic sorting and sharing brings us closer to identifying new markers for severe or treatment-resistant growth disorders, which will help alert pediatricians and parents to potential risks earlier on in a child’s life.”

In addition to assessing genetic markers for short stature, the endocrinologists will conduct pilot studies of targeted interventions, such as IGF-I therapy in patients with mutations in the growth hormone pathway, based on these genetic underpinnings.

“Ideally, by identifying markers of severe growth disorders first, we’ll be able to provide targeted treatments and therapies later on to help patients throughout their lifespan,” adds Dr. Dauber.

Typical treatments for atypical growth patterns include growth hormone or less commonly insulin-like growth factor, or IGF-1, for short stature and hormone-inhibiting treatments for precocious puberty.

The multicenter clinical trial is funded by the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), under grant Ro1HD093622, and runs through June 30, 2023.

Stephen Teach does an asthma exam

Stephen J. Teach, M.D., MPH, inaugural holder of new endowed chair

Stephen Teach does an asthma exam

Stephen J. Teach, M.D., M.P.H., has been named the inaugural Wendy Goldberg Professor in Translational Research in Child Health and Community Partnerships. This professorship comes with an endowed chair at Children’s National Health System.

The prestigious honor is given for the duration of Dr. Teach’s (and future chair holders’) employment at Children’s National. The award’s namesake, Wendy Goldberg, and her husband, Fred T. Goldberg Jr., are among the brightest stars in the constellation of Children’s National supporters, says Dr. Teach, Associate Dean for Pediatric Academic Affairs and Chair of the Department of Pediatrics at The George Washington University School of Medicine & Health Sciences.

In addition to serving on many Children’s boards, in the mid-2000s the Goldbergs made a $250,000 gift that benefited Improving Pediatric Asthma Care in the District of Columbia (IMPACT DC), Dr. Teach’s award-winning program to improve clinical care, empower patients and families, and conduct new research to improve patients’ outcomes.

“In recognition of the anchor aims of Children’s new strategic plan, the Goldbergs wanted this new gift to focus on the intersection of community health and research,” Dr. Teach says. “Thanks to their generosity, my team will work with community partners to use data to drive improvements in population health.”

With the dedicated funding Dr. Teach was able to hire a new staffer, Caitlin Munoz, to help mine electronic health records to create disease-specific registries that include 15,000 children and adolescents – the lion’s share of kids younger than 17 who live in Washington and have asthma.

“For the first time, we will be able to describe in granular detail the near-universe of local children who have this chronic respiratory disease,” he says. “We will be able to describe many of the most clinically meaningful aspects of nearly every child with asthma who lives in D.C., including mean age, gender, ethnicity and mean number visits to the emergency department.”

Such a richly textured database will help identify children who should be prescribed daily controller medications to help them avoid missing school days due to asthma exacerbations, he says. The next pediatric chronic disease they will track via registry will be pediatric obesity via elevated body mass index.

“That, in and of itself, is insightful data. But the enduring impact of this applied research is it will inform our continuous quality-improvement efforts,” he adds.

By querying the registries the team will be able to tell, for example, how Children’s primary care centers rank comparatively by asking such questions as which percentage of kids with asthma actually take the medicines they had been prescribed the year prior.

“Increasingly, clinical research falls into one of two buckets. You can either do better things: That’s discovering new drugs or processes, like our ongoing clinical trial to desensitize kids to asthma allergens. Or, you can do things better. We often know what to do already. We know that guideline-based asthma care works well. We don’t need to prove that again. We just need to do things better by getting this care to the kids who need it. That’s where this line of research/quality improvement comes in: It’s getting people to do things better.”

Andrew Dauber

Andrew Dauber, M.D., joins Children’s National as Chief of Endocrinology

Andrew Dauber

“Researchers, clinicians and medical trainees are pressed for time,” says Andrew Dauber, M.D. “Merging these three arenas into a joint infrastructure powers institutional collaboration and fuels transformative, cutting-edge care.”

Imagine an endocrinology division staffed with endowed researchers, clinicians and specialists, that serves as an engine of innovation, making it easy for pediatricians to make the right referrals, based on the best research, to endocrinologists who can provide families with cutting-edge care.

Andrew Dauber, M.D., MMSc, the new chief of endocrinology at Children’s National, is turning this dream into a reality. Over the next few years, Dr. Dauber will work with a nationally-ranked endocrinology and diabetes center to build a clinical endocrinology research program, housing specialty clinics for Turner’s syndrome, thyroid care and growth disorders, amongst others.

“Researchers, clinicians and medical trainees are pressed for time,” notes Dr. Dauber. “Merging these three arenas into a joint infrastructure powers institutional collaboration and fuels transformative, cutting-edge care.”

To put his real-life hypothesis of providing an engine for innovation into practice, Dr. Dauber led the interdisciplinary growth center at Cincinnati Children’s Hospital Medical Center and organized a Genomics First for Undiagnosed Diseases Program to study genetic clues for undiagnosed diseases. At Boston Children’s Hospital, he was the assistant medical director for the clinical research unit and held academic appointments with Harvard Medical School.

Dr. Dauber finds it’s critically important to merge clinical practice with research and education. He received his medical degree and a Master’s of Medical Sciences in Clinical Investigation from Harvard Medical School. He has published more than 65 studies examining genetic clues to endocrine disorders, with a focus on short stature and growth disorders.

Dr. Dauber conducted the majority of his research – ranging from studying genetic clues for rare growth disorders and causes of precocious puberty to genes that regulate the bioavailability of IGF1, insulin-like growth factor – while counseling patients, advising students and fellows, managing grants, reviewing studies and speaking at international pediatric endocrinology conferences.

He’s harnessing this data by combining genomic insights with electronic health records and patient registries. While some of this information can be used immediately to identify a high-risk patient, other conditions may take years to understand. Dr. Dauber views this as an investment in the future of pediatric endocrinology.

“I’m excited to join Children’s National and to work in Washington, where we can power our city and the nation with premier partnerships and collaboration,” adds Dr. Dauber. “In addition to using genetic clues to investigate growth disorders, we’re just as enthusiastic about investing in and expanding access to youth-focused diabetes education and care.”

The Division of Diabetes and Endocrinology works with the National Institutes of Health, conducts independent research and received support from the Washington Nationals Dream Foundation for its diabetes program, the largest pediatric diabetes program in the region, which provides community education and counsels 1,800 pediatric patients each year.