Newborn baby in a crib

Pioneering research center aims to revolutionize prenatal and neonatal health

  • Katherine Wisner
  • Catherine Stoodley
  • Daniel Licht
  • Katherine Ottolini
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Catherine Limperopoulos, Ph.D., was drawn to understanding the developing brain, examining how early adverse environments for a mother can impact the baby at birth and extend throughout its entire lifetime. She has widened her lens – and expanded her team – to create the new Center for Prenatal, Neonatal & Maternal Health Research at Children’s National Hospital.

“Despite the obvious connection between mothers and babies, we know that conventional medicine often addresses the two beings separately. We want to change that,” said Dr. Limperopoulos, who also directs the Developing Brain Institute. “Given the current trajectory of medicine toward precision care and advanced imaging, we thought this was the right moment to channel our talent and resources into understanding this delicate and highly dynamic relationship.”

Moving the field forward

Since its establishment in July 2023, the new research center has gained recognition through high-impact scientific publications, featuring noteworthy studies exploring the early phases of human development.

Dr. Limperopoulos has been at the forefront of groundbreaking research, directing attention to the consequences of maternal stress on the unborn baby and the placenta. In addition, under the guidance of Kevin Cook, Ph.D., investigators published a pivotal study on the correlation between pain experienced by premature infants in the Neonatal Intensive Care Unit and the associated risks of autism and developmental delays.

Another area of research has focused on understanding the impact of congenital heart disease (CHD) on prenatal brain development, given the altered blood flow to the brain caused by these conditions during this period of rapid development. Led by Josepheen De Asis-Cruz, M.D., Ph.D., a research team uncovered variations in the functional connectivity of the brains of infants with CHD. In parallel, Nickie Andescavage, M.D., and her team employed advanced imaging techniques to identify potential biomarkers in infants with CHD, holding promise for guiding improved diagnostics and postnatal care. Separately, she is investigating the impact of COVID-19 on fetal brain development.

In the months ahead, the team plans to concentrate its efforts on these areas and several others, including the impact of infectious disease, social determinants of health and protecting developing brains from the negative impacts of maternal stress, pre-eclampsia and other conditions prevalent among expectant mothers.

Assembling a team

Given its robust research plan and opportunities for collaboration, the center pulled together expertise from across the hospital’s faculty and has attracted new talent from across the country, including several prominent faculty members:

  • Daniel Licht, M.D., has joined Children’s National to build a noninvasive optical device research group to better care for children with CHD. Dr. Licht brings decades of experience in pediatric neurology, psychiatry and critical care and is recognized internationally for his expertise in neurodevelopmental outcomes in babies with CHD.
  • Katherine L. Wisner, M.S., M.D., has accumulated extensive knowledge on the impact of maternal stress on babies throughout her career, and her deep background in psychiatry made her a natural addition to the center. While Dr. Wisner conducts research into the urgent need to prioritize maternal mental health, she will also be treating mothers as part of the DC Mother-Baby Wellness Initiative — a novel program based at Children’s National that allows mothers to more seamlessly get care for themselves and participate in mother-infant play groups timed to align with their clinical appointments.
  • Catherine J. Stoodley, B.S., M.S., D.Phil., brings extensive research into the role of the cerebellum in cognitive development. Dr. Stoodley uses clinical studies, neuroimaging, neuromodulation and behavioral testing to investigate the functional anatomy of the part of the brain responsible for cognition.
  • Katherine M. Ottolini, M.D., attending neonatologist, is developing NICU THRIVE – a research program studying the effects of tailored nutrition on the developing newborn brain, including the impact of fortifying human milk with protein, fat and carbohydrates. With a grant from the Gerber Foundation, Dr. Ottolini is working to understand how personalized fortification for high-risk babies could help them grow.

Early accolades

The new center brings together award-winning talent. This includes Yao Wu, Ph.D., who recently earned the American Heart Association’s Outstanding Research in Pediatric Cardiology award for her groundbreaking work in CHD, particularly for her research on the role of altered placental function and neurodevelopmental outcomes in toddlers with CHD. Dr. Wu became the third Children’s National faculty member to earn the distinction, joining an honor roll that includes Dr. Limperopoulos and David Wessel, M.D., executive vice president and chief medical officer.

Interim Chief Academic Officer Catherine Bollard, M.D., M.B.Ch.B., said the cross-disciplinary collaboration now underway at the new center has the potential to make a dramatic impact on the field of neonatology and early child development. “This group epitomizes the Team Science approach that we work tirelessly to foster at Children’s National,” Dr. Bollard said. “Given their energetic start, we know these scientists and physicians are poised to tackle some of the toughest questions in maternal-fetal medicine and beyond, which will improve outcomes for our most fragile patients.”

desktop computer showing the CNRI Annual Report

Driving pediatric breakthroughs through 2023

desktop computer showing the CNRI Annual ReportThe Children’s National Research Institute released its 2022-2023 Academic Annual Report. In the report, a summary of the past academic year highlights the accomplishments of each of the institute’s research centers, provides research funding figures and exalts some of the institute’s biggest milestones.

The stories in the report are a testament to the hard work and dedication of everyone at the Children’s National Research Institute.

We celebrated five decades of leadership and mentorship of Naomi Luban, M.D., and her incredible accomplishments in the W@TCH program, which have been instrumental in shaping the future of pediatric research.

We also celebrated innovation, highlighting our recent FDA award to lead a pediatric device consortium, which recognizes our commitment to developing innovative medical devices that improve the lives of children.

Breakthroughs at the Research & Innovation Campus continued as our researchers worked tirelessly to develop new treatments and therapies that will transform the lives of children and families around the world.

Taking a look at the breakthroughs happening in our now six research centers, we spotlighted the following stories:

  • Reflecting on decades of progress in the blood, marrow and cell therapy programs at Children’s National. Our researchers have made significant strides in this field, and we are proud to be at the forefront of these life-saving treatments.
  • In genetic medicine, we continue to be a beacon of hope for families facing rare and complex conditions. Our researchers are making incredible breakthroughs that are changing the landscape of pediatric medicine.
  • We are also proud to share the $90 million award received from an anonymous donor to support pediatric brain tumor research. The predominant focus of this award is to develop new treatments that will improve outcomes for children with this devastating disease.
  • This year, we opened a new Center that enhances our research capabilities in the field of Prenatal, Neonatal & Maternal Health Research. We are excited about the possibilities this new center will bring and look forward to the discoveries that will emerge from it.
  • In addition, we are driving future pandemic readiness with the NIH funded Pediatric Pandemic Network. Our researchers are using cutting-edge technology and innovative approaches to prepare for the next pandemic and protect children.
  • We are also exploring the potential of artificial intelligence (AI) in pediatric breakthroughs. Our researchers are using machine learning and other AI techniques to develop new treatments and therapies that will transform the lives of children.
Boy lying in a hospital bed, surrounded by medical equipment

Black, Hispanic children at greater risk for complications during hospitalization

Boy lying in a hospital bed, surrounded by medical equipment

The research team found that patients who are Black and Medicaid-insured patients experienced the greatest disparities in postoperative sepsis, a rare complication in which patients suffer from infection that can cause multi-organ failure.

Evaluating more than 5 million pediatric hospital stays nationwide, researchers found children who are Black, Hispanic or insured with Medicaid face a greater risk of health events after surgeries than white patients, according to a new study published in the journal Pediatrics.

“We looked at the data, and we calculated the risks,” said Kavita Parikh, M.D., MSHS, medical director of Quality & Safety Research, research director of the Division of Hospital Medicine and first author on the multi-institute study. “Despite decades of focus on eliminating medical errors, we know that children continue to suffer substantial harms in hospital settings, and our study highlights where children who are Black, Hispanic or insured with Medicaid are at the greatest risk.”

The big picture

The study analyzed data from more than 5.2 million hospitalizations collected by the 2019 Kids’ Inpatient Database, a national repository of data on hospital stays. It includes a 10% sample of newborns and an 80% sample of other pediatric discharges from 4,000 U.S. hospitals. More than 80% of patients were younger than 1 year of age.

The research team found that patients who are Black and Medicaid-insured patients experienced the greatest disparities in postoperative sepsis, a rare complication in which patients suffer from infection that can cause multi-organ failure. Patients who are Hispanic experienced the greatest disparity in postoperative respiratory failure, a complication that can limit breathing and ventilation.

Plausible factors cited include structural racism in the U.S. healthcare system, clinician bias, insufficient cultural responsiveness, communication barriers and limited access to high-quality healthcare.

What’s ahead

The study – “Disparities in Racial, Ethnic, and Payor Groups for Pediatric Safety Events in U.S. Hospitals” – is foundational in understanding what is happening among pediatric patients. Dr. Parikh said that researchers now must conduct further studies into these alarming disparities and qualitative work to understand drivers, with the action-oriented goal of developing interventions to improve patient safety in the hospital for all children.

“We brought together leaders in pediatric medicine, health policy and public health to analyze this data, and we are committed to taking the next steps to improve outcomes for pediatric patients,” Dr. Parikh said. “It will take more patient-centered work and research, resources and multifaceted strategies to resolve these worrying disparities for our pediatric patients nationwide.”

doctor listening to child's heartbeat

Earlier detection of cardiometabolic risk factors for kids may be possible through next generation biomarkers

doctor listening to child's heartbeat

The next generation of cardiometabolic biomarkers should pave the way for earlier detection of risk factors for conditions such as obesity, diabetes and heart disease in children.

American Heart Association statement finds potential future measures, reiterates importance of heart-healthy lifestyle from birth through adulthood.

The next generation of cardiometabolic biomarkers should pave the way for earlier detection of risk factors for conditions such as obesity, diabetes and heart disease in children, according to a new scientific statement from the American Heart Association published in the journal Circulation.

“The rising number of children with major risk factors for cardiometabolic conditions represents a potential tsunami of preventable disease for our healthcare system,” says the statement’s lead author Michele Mietus-Snyder, M.D., a preventive cardiologist and clinical research scientist at Children’s National Hospital. “But by the time a child is identified based on today’s clinical biomarkers, it’s often too late to reverse the disease trajectory.”

The big picture

The scientific statement included biomarkers that met three criteria:

  • Early and precise clinical detection of metabolic abnormalities before a child begins to show the current clinical signs such as high body mass index (BMI), blood pressure or cholesterol.
  • Mechanistic intervention targets providing immediate risk measures and giving clinicians new targets to personalize and optimize interventions.
  • Modifiable biomarkers that are capable of tracking progression toward or away from cardiometabolic health.

The statement’s identified biomarkers included measures of:

  • Epigenetic, or environmental, factors
  • Gut microbiome health
  • Small particle metabolites in the body
  • Different types of lipids and their impacts on cell membranes
  • Inflammation and inflammatory mediators

The authors proposed these biomarkers with the goal of “expanding awareness to include a whole new realm of biomarkers that precede the traditional risk factors we currently rely upon, such as BMI, blood pressure, cholesterol and blood sugar,” says Mietus-Snyder. “Ideally, these new biomarkers will be added to the array of measures used in clinical research to better assess their value for earlier identification and prevention of global patterns of cardiometabolic health and risk.”

Why it matters

The next generation cardiometabolic biomarkers outlined by the authors are all currently used in research studies and would need to be validated for clinical use. However, Mietus-Snyder notes that the data already collected from these biomarkers in research can make a difference in clinical practice by enhancing our understanding of the deep metabolic roots for children at risk.

Evidence reviewed in the statement shows the risk factors children are exposed to, even before birth, can set the stage for cardiovascular and metabolic health across the lifespan.

Interestingly, all the different factors reviewed have been found to alter the functioning of the mitochondria — the complex organelles responsible for producing the energy for the body that every cell and organ system in turn needs to function. Every class of biomarkers reviewed is also favorably influenced by heart-healthy nutrition, a simple but powerful tool known to improve mitochondrial function.

What’s next

Even as the new so-called ‘omic’ biomarkers reviewed in this statement are developed for clinical applications, there are things clinicians can do to optimize them and improve mitochondrial function, according to Mietus-Snyder.

Most important is to strengthen the collective dedication of care providers to removing the barriers that prevent people, especially expecting mothers and children, from living heart-healthy lifestyles.

We have long known lifestyle factors influence health. Even as complicated metabolic reasons for this are worked out, families can reset their metabolism by decreasing sedentary time and increasing activity, getting better and screen-free sleep, and eating more real foods, especially vegetables, fruits and whole grains, rich in fiber and nutrients, with fewer added sugars, chemicals, preservatives and trans fats. Clinicians can work with their patients to set goals in these areas.

“We know diet and lifestyle are effective to some degree for everyone but terribly underutilized. As clinicians, we have compelling reasons to re-dedicate ourselves to advocating for healthy lifestyle interventions with the families we serve and finding ways to help them implement them as early as possible. The evidence shows the sooner we can intervene for cardiometabolic health, the better.”

child being evaluated for autism

Using a multisystem approach to improve access to autism care in Washington, D.C.

child being evaluated for autism

Children with autism face significant barriers to accessing evaluations and intervention services.

An article in the journal Pediatrics reviews the outcomes from a collective, targeted advocacy approach to improving access to autism supports and resources for children and their families in Washington, D.C. The effort was led by Children’s National Hospital and engaged a multidisciplinary team from within the hospital and across a wide range of community sectors.

What this means

Children’s National and DC Autism Parents worked collaboratively with a coalition of organizations from the broader District of Columbia community to address some of the biggest challenges and barriers that prevent autistic children and their families from receiving the resources and support they need in the nation’s capital.

Why it matters

Children with autism face significant barriers to accessing evaluations and intervention services often because of confusing referral processes, lack of centralized coordination across organizations serving children with autism, insurance coverage gaps, multiyear waitlists for diagnostic services and limited provider knowledge about autism. Racism and systemic inequities also persist in autism care across the United States.

Long and growing wait times in autism diagnostic clinics and lack of centralized care coordination for autistic children are prevalent across the District of Columbia, and as a result, many children and families in the region continue to lack access to the support they need.

What’s unique

The study describes multiyear efforts (2017–2022) to improve autism care throughout the District of Columbia using a collective impact framework to unite organizations from different sectors. This approach features the creation of a common agenda (including defining goals and priorities), shared measurement, mutually reinforcing activities, continuous communication and infrastructure support.

Together, the group members sought to address barriers and overcome challenges at multiple levels of the healthcare system at the same time by focusing advocacy in three specific areas:

  • Infrastructure-building initiatives/system-level approaches.
  • Population- and community-level services to build capacity and connect providers and families to needed resources.
  • Direct services that provide innovative, gap-filling supports to children and families as a stopgap until the necessary supports can be more sustainably provided across the board.

Bottom line

While more work is needed to continue expanding the availability of needed services, the findings from this initial effort can inform the next steps in Washington, D.C., and serve as a model for a collective framework approach for autism services in other parts of the United States.

You can read the full study “A Multisystem Approach to Improving Autism Care” in the journal Pediatrics.

More information and resources about these autism initiatives can be found at:

newborn baby with bandaid on heel

JAMA Pediatrics editorial: A better approach for newborn screening

The medical community has an opportunity to update its approach to newborn screening (NBS) to be prepared for emerging technological advancements that will help diagnose children with rare diseases from their first weeks of life, according to an editorial from a leading Children’s National Hospital researcher published in JAMA Pediatrics.

“In health care, we are seeing ways in which we can identify more children who have rare diseases even earlier, in the newborn period, rather than waiting for children to develop symptoms or experience irreversible changes,” said Beth Tarini, M.D., M.S., M.B.A., associate director of the Center for Translational Research. “We have continued innovations in screening technology – with more on the way – that can be added to the screening programs overseen by all 50 states. Updating how we approach newborn screening presents an incredible opportunity for doctors and their patient-families.”

Why it matters

Newborn screening happens before the baby leaves the hospital, generally with a prick of the heel to take a small sample of blood to look for several dozen rare, debilitating disorders such as sickle cell disease, congenital hypothyroidism and cystic fibrosis. The current screening system has grown successfully for roughly 60 years and creates a network of state programs. Along the way, researchers have had extensive debates about which disorders to include, based on whether there are treatments and options for patients.

Dr. Tarini, a pediatrician who has done extensive research on NBS and related policies, said that the existing screening programs across all 50 states should be modernized, with federal research support and funding, to create a unified “learning newborn screening system” that derives information from the 4 million babies born each year and provides feedback to the medical community about best practices for babies who are diagnosed with a rare disease or at risk for developing one.

“A new approach will require resources and infrastructure, but as the technology advances, we should change our system to leverage the experience of doctors, patients, and NBS programs across the country,” Dr. Tarini said. “We have the will, the experience and the ability to transform the care for children with rare disease.”

Read the full editorial in JAMA Pediatrics.

girl being examined by doctor

Pediatric hospitals underutilize systems to get at social challenges impacting health

 

girl being examined by doctor

Physicians treating hospitalized children rarely use a coding system established in 2015 for flagging social challenges and stressors that may be impacting patient health, according to new research from Children’s National Hospital.

 

Physicians treating hospitalized children rarely use a coding system established in 2015 for flagging social challenges and stressors that may be impacting patient health, according to new research from Children’s National Hospital published in Pediatrics. Known as social determinants of health (SDOH), these factors include food insecurity, homelessness and adverse childhood events like substance abuse at home, and they can greatly affect a child’s well-being.

“We only get so many touchpoints with our patients,” said Kaitlyn McQuistion, M.D., pediatric hospital medicine fellow at Children’s National and co-lead author of the paper. “Our research shows the screening itself provides valuable insight into our patients, making identification an important part of inpatient and post-discharge care. With this information, doctors can help families tap into social workers, community supports and other resources aimed at providing a more holistic approach to child health.”

The big picture

The American Academy of Pediatrics advises screening for unmet social needs and using the codes laid out in the International Classification of Disease, 10th Revision (ICD-10), to flag risk factors for some of a hospital’s most vulnerable patients. In 2018, the American Hospital Association (AHA) clarified that the coding can be added by any healthcare professional accessing the chart, including nurses, social workers, case managers and physicians. The study looked at data from 4,000 hospitals in 48 states and the District of Columbia and found that use of the codes has remained low, even with the AHA’s clarification.

In practice, physicians know that screening and documentation are the essential first steps to help families find resources. Yet less than 2% of pediatric inpatients were coded as needing support. Most commonly, “problems related to upbringing” – a broad category indicating social needs and adverse childhood events – was used.

What’s ahead

Some providers are using these SDOH codes, or Z Codes, more often to address and improve health disparities. “Our mental health colleagues and those working with the Native American population, in particular, are using these tools more often to capture and disseminate critical information related to their patients’ social needs,” said Stacey Stokes, M.D., a hospitalist at Children’s National and co-lead author on the paper.  “Their innovative approaches to address and improve health disparities may provide learning opportunities for institutions.”

The researchers said that more work needs to be done to take these successes to other populations, find ways to incentivize this work in billing and ensure that providers have community resources to address the needs that they uncover.

“The ultimate goal of this work is to identify patients with social needs affecting their health and connect them with resources,” said Kavita Parikh, M.D., director for the Research Division of Hospital Medicine. “There are many avenues to explore to find ways to better utilize this tool, including language learning models, improved training and stronger community resources.”

smiling young family

Survey: Teen parents with support report greater well-being

smiling young family

Teen parents in Washington, D.C., reported better physical and mental health when they had more social support, a community and people who understood their problems.

Teen parents in Washington, D.C., reported better physical and mental health when they had more social support, a community and people who understood their problems, according to a survey conducted as part of a novel city-wide program to bring together resources for young families.

“This should come as no surprise. Young parents say they do better – and their children do better – when they have the material and social supports they need,” said Yael Smiley, M.D., a pediatrician at Children’s National Hospital and lead author of research in BMC Public Health. “This work is the start of a roadmap to help young parents thrive.”

The big picture

Each year in D.C., 300 babies are born to teens, and the rate of births among teens ages 15 to 17 is higher than the national average. Dr. Smiley is a leader in a federally funded coalition run by D.C. Primary Care Association (DCPCA) to help support young parents called the D.C. Network for Expectant and Parenting Teens (DC NEXT!). This city-wide network brings together community partners to support teen mothers, fathers and their children.

A key feature of the program is its context team, a group of teen parents who provide insights into their experiences as young parents. They advise about research and support for their peers, and they also build a community and develop a platform to speak up about their issues. The context team recruited other parents to complete a well-being survey in 2022, which will guide programming and resources for families. An added benefit for the parents who participate in the context team: The survey data showed that a key facet of well-being for the young parents was feeling “like you were part of a group of friends or community” and having “someone who understood your problems.”

“The context team provides the type of community that the research shows is essential to the well-being of teen parents,” said Dr. Smiley. “Not only are the context team members helping us understand what teen parents need, but they are also helping each other thrive as young parents.”

The fine print

Among the findings of the survey of 107 teen parents in D.C.:

  • The most used resources were supplemental food programs – with 35% receiving Supplemental Nutrition Assistance Program (SNAP) benefits, 24% receiving support from the Special Supplemental Nutrition Program for Women, Infants and Children (WIC) and 18% accessing food pantries.
  • Also popular were Temporary Assistance for Needy Families (TANF), housing assistance and childcare.
  • Surprisingly, the largest group of respondents, 37%, reported receiving no resources.

Having higher social support was correlated with higher ratings for physical health, mental health and well-being. “With this research, we have even more evidence underscoring the need to connect young parents with programs and resources that will improve their well-being,” Smiley said. “I can’t wait to see what the leaders on the context team and in the DC NEXT! program do in the next phases of our work.”

sick boy with malaria

New guidance to optimize blood sugar monitoring in cerebral malaria

A Children’s National Hospital research team based in Malawi pinpointed the optimal duration and frequency for monitoring the blood glucose in children with cerebral malaria, providing a roadmap to improve the treatment and outcomes for young patients diagnosed with the life-threatening disease.

Published in the American Journal of Tropical Medicine and Hygiene, the findings analyzed data from 1,674 pediatric cases to recommend the best schedule for periodic bedside point-of-care laboratory testing in children with cerebral malaria (CM). Currently, World Health Organization (WHO) guidelines state that blood glucose should be monitored in all forms of severe malaria, but they do not include advice on the timing or duration of the measurements.

Children’s National neurologist Douglas Postels, M.D., M.S., led a team of trainees from Howard University, The George Washington University, the University of Washington and Children’s National to collect and analyze patient data that led to the creation of evidence-based recommendations for glucose monitoring.

“If blood glucose in children with severe malaria is too low, the child is at high risk of death,” Dr. Postels said. “What we found in this research study is both interesting and important, and we hope our study results will help the WHO in creating evidence-based guidelines for blood glucose monitoring in children with cerebral malaria.”

The big picture

In 2021, 247 million people contracted malaria worldwide, killing some 619,000 primarily in Africa. Almost 80% were children under 5 years old, making it one of the most virulent pediatric diseases in the world. Many who survive experience significant neurologic, cognitive and behavioral morbidities.

Dr. Postels works at Queen Elizabeth Central Hospital in Blantyre, Malawi, caring for patients on the Pediatric Research Ward and conducting research aimed at improving outcomes. The clinical team works without access to many medical tools that are considered standard throughout more advanced economies. Yet this team has one of the lowest mortality rates for cerebral malaria across Africa, thanks to their ongoing research to better understand the pathophysiology of malaria and improve its treatment.

The challenges are immense: During COVID-19, they battled supply chain issues that frustrated repairs on an aging MRI scanner. After the second tropical cyclone struck in the last two years, Blantyre was left without power for a week and without water for a month.

“You can imagine trying to run a hospital with no water,” Dr. Postels said. “During Cyclone Freddy, it was raining like crazy, and people were collecting water in bins—anything they could collect it in — to use for handwashing, as well as trying to clean instruments and supplies.”

What they’re saying

Using evidence-based guidelines to optimize care becomes important in sub-Saharan Africa, where resources are scarce. Running laboratory-based blood studies frequently is an inefficient use of supplies and laboratory reagents. Results of point-of-care testing are also available more rapidly and at less cost than studies performed in hospital labs.

An accompanying editorial in the journal said the new research on glucose monitoring in cerebral malaria “provides valuable data that could help clinicians in resource-limited settings improve CM management with more efficient use of available resources.” The work is increasing calls for further study and updates to international guidelines.

The bottom line

According to the team’s analysis, blood glucose should be measured in children with CM on admission and every six hours for the first 24 hours. If all results in the first 24 hours are normal, clinicians can stop testing.  If any blood sugar levels in the first 24 hours are low, the patient’s blood glucose should be checked for another 24 hours.

“This testing strategy captures 100% of the children who have a glucose level of 2.2 mmol/L or less, the definition of hypoglycemia in severe malaria,” Dr. Postels said.  “We want to do enough, but not too much.”

The overarching goal for Dr. Postels and the trainees who join him in Blantyre is improving care.  “If we can help clinicians better care for children with cerebral malaria, then hopefully we can make a small contribution to decreasing the death rate and improve neurologic outcomes of the children who survive,” he said. “That’s my hope.”

Panel members at the NIAID symposium

CN-NIAID Symposium seeks ways to promote child health amid challenges

Panel members at the NIAID symposium

More than 30 million children seek emergency care each year, but 80 percent of these visits happen at hospitals that aren’t designed for pediatrics — a daunting figure during pandemics and other crises in healthcare. This considerable hurdle is one of many challenges that leaders in pediatric health came to discuss during a two-day symposium on promoting child health, hosted by Children’s National Hospital, the National Institute of Allergy and Infectious Diseases and the Pediatric Pandemic Network (PPN).

The symposium laid out a multitude of issues facing children and their doctors: growing mental health diagnoses, shrinking access to care in rural areas, asthma and eczema, winter respiratory surges and more.

Joelle Simpson, M.D., chief of emergency medicine at Children’s National and PPN principal investigator, said the network is drawing on expertise from 10 pediatric hospitals to ensure communities are better prepared for whatever challenges lie ahead, through training and support, collaboration among pediatric specialists, education on best practices and the promotion of equity and inclusion.

Built on a Health Resources and Services Administration grant, the network is focusing on four key areas: infectious disease and disease outbreaks, emergency and disaster management, mental and behavioral health, and health equity and community engagement. “This year, we know we are boiling the ocean as we come together,” Simpson said.

Miss the symposium? Check out the recordings available on YouTube, including the closing Q&A with many of the panelists and Sheryl Gay Stolberg, health policy reporter with the New York Times.

Day 1 of the 6th Annual Children’s National Hospital – NIAID Symposium

Day 2 of the 6th Annual Children’s National Hospital – NIAID Symposium

 

Borrelia burgdorferi bacteria

Most children diagnosed with Lyme disease recover fully after treatment

Borrelia burgdorferi bacteria

Lyme disease is caused by the bacterium Borrelia burgdorferi.

Experts found most children diagnosed with Lyme disease recovered within six months of completing antibiotic treatment, according to a new joint study published in Pediatric Research.

The study, which was supported through a partnership between Children’s National Research Institute (CNRI) at Children’s National Hospital and the National Institute of Allergy and Infectious Diseases (NIAID), also revealed that a notably small percentage of children took longer than six months to recover and experienced a significant impact on their daily functioning.

The big picture

This research studied the long-term outcomes of children with Lyme disease through a cross-sectional evaluation using validated surveys. The study collected survey responses from the parents of 102 children ages 5 to 18 years who had been diagnosed with Lyme disease between six months and 10 years before enrollment. Adolescents ages 10 to 18 years were also invited to complete adolescent-specific questionnaires.

According to these parent survey responses, the vast majority (87%) of fully recovered patients did so

within the initial 6-month period after completing antibiotic treatment, with approximately one third recovering within the first month post-treatment and an additional third recovering 1–3 months post-treatment. However, 13% of children who ultimately made a full recovery took longer to do so. At the time of study completion, 6% of children still experienced symptoms attributed to Lyme disease but only 1% experienced symptoms significant enough to impair daily functioning.

Why we’re excited

According to the authors, this study supports previous data showing an excellent overall prognosis for children with Lyme disease, which should help alleviate understandable parental stress associated with lingering non-specific symptoms among infected children.

“These findings can help clinicians manage families’ expectations about the varying post-treatment recovery times of pediatric Lyme disease patients,” says Roberta DeBiasi, M.D., chief of Infectious Diseases at Children’s National Hospital and one of the study’s authors. “It may also give parents relief in knowing that their child has an excellent chance of full recovery after treatment.”

What’s next

Common symptoms of Lyme disease include fever, headache, fatigue and a distinct skin rash called erythema migrans. Without treatment, the infection can spread to joints, the heart and the nervous system.

Antibiotic treatment resulting in full recovery is successful in most Lyme cases. For some, however, symptoms of pain, fatigue or difficulty thinking persist or return after antibiotic treatment. Symptoms that substantially reduce levels of activity and impact quality of life for more than six months after treatment are classified as post-treatment Lyme disease (PTLD) syndrome.

PTLD syndrome remains poorly understood in children and adults, and more research is needed to better understand these prolonged symptoms and identify treatment targets, according to the authors.

You can read the full study, Pediatric Lyme disease: systematic assessment of post-treatment symptoms and quality of life, in Pediatric Research.

Dalia Haydar

Harnessing children’s immune systems to fight their own brain tumors

Dalia Haydar, Pharm.D., Ph.D., principal investigator for the Program for Cell Enhancement and Technologies for Immunotherapies, recently joined Children’s National Hospital to help develop breakthrough treatments that hopefully will be a key in the fight against pediatric brain tumors. She brings her deep experience at St. Jude Children’s Research Hospital to the Center for Cancer and Immunology Research (CCIR) to help support the NexTGen team’s 10-year, $25-million Cancer Grand Challenges award.

Dalia – literally – has drive: She commutes 180 miles round trip from her home in Hershey, Pa., to her lab. She says she is grateful to be at one of the few research institutions in the world that is researching how to harness the power of CAR T-cell therapies to attack solid tumors in kids. While these therapies have been approved to treat leukemia and other blood cancers, solid tumors have proven far more stubborn. Haydar has tremendous hope that she and the team led by CCIR Director Catherine Bollard, M.D., M.B.Ch.B., will change that.

Q: Could you explain the importance of this research?

A: Unfortunately, once a patient is diagnosed with a brain tumor, especially a kid, there’s very little we can do. Using chemotherapy or radiation therapy has big disadvantages because of developmental delays and other side effects. We are hoping this kind of immunotherapy – where we take the patient’s own immune cells and engineer them in the lab to attack their cancer – will eradicate their very harsh and aggressive tumors, without causing significant adverse effects.

Q: How are researchers at Children’s National going to attack solid tumors with a treatment originally designed for blood cancers?

A: We have a lot of resources and expertise at Children’s National that we are trying to put together to develop a therapy that would cure brain tumors. Unfortunately, solid tumors are hard to treat and there are several challenges for any kind of immunotherapy. But right now, being at a place where all the necessary resources, support and expertise are available, we are hoping to address each of these challenges, and we are determined to do something in a meaningful timeframe to push that survival curve toward the advantage of those kids.

Q: How soon can this work be done?

A: Within two or three years, we are hopeful we’ll be able to identify the best working regimen of this CAR T-cell immunotherapy and investigate if it will work in a patient. I foresee, in the next 5 to 10 years, that we’re hopefully going to have such therapy for kids with brain tumors.

Q: What has surprised most you in your work?

A: There are so many challenges in developing immunotherapies for kids with brain tumors. First, if it works for adults, it doesn’t necessarily work for kids. Some of the tumors in kids are more aggressive. We need to understand the tumor itself, besides understanding the immunotherapy we’re developing.

The other challenge is CAR-T immunotherapy is not like a pill or taking radiotherapy that is standardized for several patients. It’s a very expensive therapy. It’s taking the patient’s own immune cell, like a bone marrow transplant. We put it in the lab, re-engineer the cells without transforming them into a cancer cell, enable those immune cells to attack the cancer, and then put them back into the patient. There are a lot of steps you need to take to make sure you don’t artificially harm those cells or introduce contamination.

One of the most intriguing challenges for me is how we make immunotherapy work for kids who have different kinds of brain tumors – a medulloblastoma versus a glioma versus an embryonal tumor. This is one of the challenges that keeps me on my toes, and I’m hoping to answer.

Q: What is the power of being in a multi-center environment like the Children’s National Research Institute?

A: We have to do enough science on the bench to support any proposal for the therapy to move to the clinic. The last thing we want to do is to investigate a drug or therapy in patients without really knowing how it works and the potential adverse effects. Being able to work with researchers at different stages of the bench-to-bedside spectrum, as well as being able to have access to patient samples and innovative preclinical models, helps push the science forward in a shorter time frame.

Young girl with paints

Autism Center of Excellence finds tools to avoid late diagnosis of women, others

Young girl with paints

Longitudinal data shows that girls and women are the most likely to be misdiagnosed or missed using traditional methods of assessment for autism.

The National Institute of Mental Health awarded $12.5 million to three institutions, including Children’s National Hospital, to become an Autism Center of Excellence. The goal of the research is to help autistic adolescents and adults receive timely and appropriate services and supports to improve overall outcomes. It is co-led by Lauren Kenworthy, Ph.D., at the Center for Autism Spectrum Disorders at Children’s National, Kevin Pelphrey, Ph.D., at the University of Virginia, and Allison Jack, Ph.D., from George Mason University,

The research will focus on developing screeners to identify people for autism assessment who traditionally have a high risk of a late or missed diagnosis.

Why it matters

Late or missed diagnosis puts people with autism spectrum disorder at greater risk for depression, anxiety and self-harm. It can also prevent access to supports through schools or other community organizations. Some people are misdiagnosed with other mental health conditions such as bipolar or borderline personality disorder leading to inappropriate treatments.

Longitudinal data shows that girls and women are the most likely to be misdiagnosed or missed using traditional methods of assessment for autism.

The hold-up in the field

There are two big reasons why truly autistic people fail to be identified. First, previous work to understand and diagnose autistic people was done based on data from mostly white, young, male participants. The tools do a very good job identifying autism that presents similarly to those study participants.

Kenworthy says the research community took a very long time (too long, perhaps) to recognize that many people with autism have a wide range of experiences both positive and negative that can inform diagnosis.

This relates to the second big hold-up in the field: that researchers have also been slow to recognize the importance of listening to the experiences of autistic people. Dr. Kenworthy says that for years, clinicians have known that diagnosing anxiety means asking the person how they feel inside. That same approach was rarely used with autistic people. “We need to listen to the people who are experiencing this or we are going to miss a lot,” she points out.

What’s next

The new Autism Center of Excellence has three main aims for the 5 years of funding.

  • Collect large amounts of behavioral and cognitive phenotyping data
  • Conduct qualitative interviews with autistic people using those data
  • Validate the development of the Self-Assessment of Autistic Traits — a tool that seeks to do a better job accelerating identification of people who need to be assessed for autism spectrum disorders but don’t necessarily meet the criteria of the current screeners.

Children’s National leads the way

This collaboration continues previous work the Center for Autism Spectrum Disorders has done with neuroimagers including Pelphrey and Jack to understand how autism and autism interventions affect the brain and builds on it by adding the experience of researchers from the autistic community.

The neuroimaging teams will use technology such as functional magnetic resonance imaging (fMRI), data analysis and genetic tools to find biomarkers and phenotypes that reflect what is learned from people with autism who experienced a missed or late diagnosis.

The end result will be a validated tool developed with people who experience autism, that gives people with autism, clinicians and researchers a unique new tool for identifying autistic strengths and challenges.

Kenworthy says it’s the two pieces coming together that will be the game-changer. “The technology, the biomarkers and phenotypes are really important, but aren’t meaningful until we understand how that maps onto the lived experience of autism.”

baby getting heel prick

Researchers study murky findings in newborn screening panels with $3.7m NIH grant

baby getting heel prick

Children’s National received a grant to investigate the impact of newborn screening on families who receive an uncertain prognosis.

The National Institutes of Health (NIH) awarded Children’s National Hospital a $3.7 million grant to investigate the impact of newborn screening on the growing population of families who leave the testing with an uncertain prognosis.

Following the families longitudinally allows for a real-time view of the experiences of these children, sometimes referred to as “patients in waiting.”

Newborn screening is part of a universal, mandatory state health program that helps to identify inherited conditions that can affect a child’s health and survival. Millions of babies are screened annually for genetic, metabolic and endocrine disorders, using a few drops of blood from a prick to the heel; additional tests are done at the bedside such as hearing and heart screening. Sometimes, however, the results create medical odysseys and flag conditions that may never result in symptoms.

“For its first 50 years, newborn screening presented relatively consistent outcomes,” said principal investigator Beth Tarini, M.D., M.S., M.B.A., who serves as the associate director of the Center for Translational Research at Children’s National. “However, in the 21st century, new screening tests have created more ambiguous findings. As a result, we cannot accurately predict what type of symptoms a child may develop, when or if they will develop them, or how severe they will be. This is a lot to ask parents to deal with after the birth of a new child who appears otherwise healthy.”

Why it matters

The uncertainty can take a significant toll on parents by creating fear, anxiety and the medicalization of a child. However, to date, little long-term data exist to inform the care for these children. Ethically, that gap leaves clinicians unsure of how to weigh the benefit and harm of mandatory newborn screening programs. From a policy perspective, the drought of information leads to questions about how best to add disorders to newborn screening panels – an issue that will likely only grow as technology allows us to test for more conditions.

“We have a new group of children growing up and wondering when – or if – they will ever develop signs or symptoms of a disease,” Dr. Tarini said. “For some families, the information is an opportunity. For others, it becomes a burden. We owe it to these families to understand their experience and chart a sensible path forward to help them.”

What’s next

The four-year study will bring together researchers at Children’s National and Case Western University to analyze data and patient interviews from families in Virginia, Iowa and Oregon. The research team will include experts in newborn screening, genetics, health services, genetic counseling, psychology, bioethics and biostatistics.

Innovations for health equity: Food pharmacy app wins Hackathon

When families come into the endocrinology clinic, 66% of prediabetes and Type 2 diabetes patients screen positive for food insecurity. One remedy: a smartphone app envisioned by Children’s National Hospital researchers to communicate with families between visits and provide resources to help stock pantries with nutritious foods.

The Children’s National Food Pharmacy app is on its way from idea to reality, thanks to the inaugural Health Equity in Research Hackathon event at the Children’s National Research & Innovation Campus. This team-based, “Shark Tank”-like competition involved roughly 50 experts designing creative healthcare solutions that could be delivered through ubiquitous smartphones.

“It takes a village to raise a child, and we want to show that at Children’s National we are part of that community,” said food pharmacy founder and diabetes educator Alexis Richardson, M.S., R.D., L.D.N., C.D.C.E.S.

Why it matters

The rate of new-onset Type 2 diabetes increased by a staggering 182% during the first nine months of the COVID-19 pandemic. Now, the Children’s National food pharmacy provides families that screen positive for food insecurity during quarterly clinic appointments with a 50-pound bag of medically-tailored groceries.

The new app, as envisioned, would follow them home to connect them with food bank information and other nutritional resources, eliminating paper forms and other hurdles that get in the way of care.

What’s ahead

Children’s National leaders are committed to making the proposal a reality. “We are going to support today’s winner through the next steps to prepare them to enter the app development pipeline at the Sheikh Zayed Institute,” said Lisa Guay-Woodford, M.D., director for the Clinical and Translational Science Institute at Children’s National (CTSI-CN) and one of the main judges of the competition.

The app development will happen in the months ahead. Kevin Cleary, Ph.D., technical director of the Sheikh Zayed Institute of Pediatric Surgical Innovation, said the Hackathon planted the seeds. “It really depends on the drive of the individual to see the idea to fruition,” Cleary told competitors.

Other app entries were encouraged to continue their work:

  • The Surgical Checklist, led by Brian K. Reilly, M.D., co-director of the Cochlear Implant Program: this app would help patients and providers successfully navigate the often-confusing pre-operative checklist, including required physical exams, lab work, imaging and pre-procedure fasting. Reilly said the hospital handles about 15,000 cases a year, and about 10% are rescheduled, often for reasons that could be avoided with digital organization and reminders for families.
  • More than Determined, led by Pediatrician Jessica Lazerov, M.D., M.B.A.: this app aims to give time-strapped providers a platform to better understand and address social determinants of health – such as access to safe housing, education and jobs – that can promote better preventative care outcomes.

The Health Equity in Research Hackathon was created by the new Health Equity in Research Unit, a joint initiative between the CTSI-CN and the Center for Translational Research within the Children’s National Research Institute.

Dr. Lisa Guay-Woodford and the winners of the Health Equity in Research Hackathon

Dr. Lisa Guay-Woodford, director for the Clinical and Translational Science Institute, joins the winners of the inaugural Health Equity in Research Hackathon: the Children’s National Food Pharmacy. The team’s proposed app will connect families facing food insecurity with resources and guidance for nutritious eating.

Monika Goyal

Researchers to address pain management inequities with over $4M NIH award

Monika Goyal

Over the years, research led by Dr. Goyal documented racial and ethnic inequities in the ED.

The National Institutes of Health (NIH) awarded Children’s National Hospital with over $4.2 million to address inequities in pain management for children that come into the emergency department (ED).

Why this matters

The ED is a strategic venue for addressing health inequities, where children account for more than 30 million visits annually.

“There are widespread inequities in the quality-of-care delivery for children. Because the factors contributing to these disparities arise on both individual and systemic levels, it is imperative that we develop interventions to achieve health equity,” said Monika Goyal, M.D., M.S.C.E., associate chief of Emergency Medicine at Children’s National Hospital and recipient of the award.

Over the years, research led by Dr. Goyal documented racial and ethnic inequities in the ED management of pain among children with long bone fractures and appendicitis and disparities in the management of pain reduction for minoritized children.

“These findings indicate there are differences in health care quality even in settings with universal access,” she added.

The research goal

Dr. Goyal and her team aim to mitigate, and ultimately eradicate, health care inequities through evidence-based interventions. With the research support from the NIH, the team will advance this goal by:

  • Measuring how clinician (physician and nursing) implicit bias is associated with quality of care for pain management in children presenting to the ED with appendicitis or long bone fractures.
  • Using a stakeholder-engaged approach to develop patient- and caregiver-informed quality metrics related to pain management.
  • Develop and measure the impact of ‘Equity Report Cards’ and electronic health record (EHR)-embedded clinical decision support (CDS) tools to mitigate inequities in care delivery.

The bottom line

Most research to date has focused on documenting disparities. This research has the potential to move the needle in equity research by developing and testing interventions that seek to eradicate inequities in care delivery.

2022 Hackathon logo

Addressing health equity issues through an app innovation competition

Children’s National Hospital launches The Health Equity in Research Hackathon — a team-based collaborative competition that empowers researchers to address health equity issues in the community through innovative apps. A panel of expert judges will select winning app ideas for full development, including finding grant opportunities, access to mentors and collaborators.

The big picture

“This Hackathon is a great chance for our research community to address larger issues related to advancing health equity within translational and clinical research,” said Patrick O’Keefe, administrative director for the Clinical and Translational Science Institute at Children’s National (CTSI-CN). “We are thrilled to see how people collaborate to create solutions for big problems that have traditionally slowed research and contributed to the vast inequities in health we see today.”

Additional details

The 2022 hackathon will be a two-part event. During Part I, slated for June 17, participants will gather in diverse teams to discuss and refine the selected app ideas. They will learn from technical and scientific experts and brainstorm app-based approaches to address health equity.

Each team will pitch their ideas to a panel of judges, and the winning app(s) will advance to Part II of the hackathon planned for Fall 2022, where app developers will build the selected apps.

“We encourage anyone at Children’s or George Washington University to submit an app idea – even if it is not fully formed — as long as they think it would help reduce health disparities through improving the research process,” said Lisa Guay-Woodford, M.D., director for Clinical and Translational Science Institute and Center for Translational Research at Children’s National. “We also hope researchers, staff and students who don’t have app ideas at this time will consider attending anyway to participate in the lively development process of the Hackathon.”

Anyone within the Children’s National and George Washington University research communities can submit an app idea for consideration. No app development experience is necessary to enter.

Why it matters

Health equity also means bringing the community into the research process. Thus, in part II, Children’s National will partner with high schools and universities in the area to incorporate voices who are often under-represented in the science and technology field.

“Our community is home to brilliant young minds at our local high schools and universities,” said Chaya Merrill, Dr.P.H., director for Child Health Data Lab at Children’s National. “We are excited to create an opportunity for them to work alongside experienced app builders – at the intersection of health equity and technology – by engaging in Part II of the Hackathon to build the winning apps!”

The hackathon will take place at Children’s Research and Innovation Campus in partnership with CTSI-CN and the Center for Translational Research.

The app idea submission deadline is on May 20, 2022, by 5pm. You may apply here. If you have questions about completing this submission, please email Patrick O’Keefe at pokeefe@childrensnational.org.

2022 Hackathon logo

Handrawn illustration of human Kidneys

Children’s National Nephrology team presents virtually at IPTA’s 11th Congress

Handrawn illustration of human KidneysThe International Pediatric Transplant Association (IPTA) is hosting their 11th Congress meeting March 26-29, 2022, and many Children’s National Hospital providers will be presenting throughout the conference. We hope you will join us!

Diversity, inequity and inclusivity in the practice of pediatric transplantation in the U.S.

Presenter: Marva Moxey-Mims, M.D., division chief, Nephrology

Dr. Moxey-Mims will review the trends in pediatric kidney transplantation over the past decade, focusing on differences by race and ethnicity, whether the gaps have narrowed over that time and steps that can be taken to increase equity.

Prevalence of mycophenolate mofetil discontinuation and subsequent outcomes in pediatric kidney transplant recipients: A PNRC study

Presenter: Asha Moudgil, M.D., medical director, Kidney Transplant

Mycophenolate mofetil (MMF) is a common maintenance immunosuppressant in children receiving kidney transplants but is often discontinued for various reasons. In this multi-center Pediatric Nephrology Research Consortium study, researchers sought to determine the prevalence and reasons for MMF discontinuation and its association with patient and allograft outcomes. They will be discussing their methodology and results from this study.

Additional Children’s National staff involved in the study include:

Changes in diastolic function and cardiac geometry after pediatric kidney transplantation: A longitudinal study

Presenter: Kristen Sgambat, Ph.D., clinical assistant professor

Children with end stage kidney disease are at high risk for cardiovascular morbidities. Indicators of systolic function, such as ejection fraction and fractional shortening, are often preserved and may not reveal cardiac dysfunction until it is severe. Longitudinal changes in diastolic function and cardiac geometry have not been well studied.

Additional Children’s National staff involved in the study include:

COVID19 in pediatric kidney transplant recipients: Incidence, outcomes, and response to vaccine

Presenter: Asha Moudgil, M.D., medical director, Kidney Transplant

At the start of the pandemic, no information was available on the outcomes of pediatric kidney transplant recipients with COVID-19. When the COVID-19 vaccine became available, response of immunosuppressed children to the vaccine was not known. While more information has become available in adult transplant recipients, information on pediatric transplant recipients remains limited.

The team will discuss their methodology of collecting information and their results and conclusions.

Additional Children’s National staff involved in the study include:

Poster presentation: Psychological functioning and adaptive behavior in pediatric patients awaiting renal transplantation.

Presenter: Kaushal Amatya, Ph.D., psychologist

Psychosocial functioning of children with chronic kidney disease (CKD) at pretransplant evaluation is associated with transplant readiness and post-transplant outcomes. Higher prevalence of emotional/behavioral issues is noted in children with CKD compared to healthy counterparts. Although issues with functional impairment is often reported, research on adaptive functioning using a validated measure is lacking. The study aimed to explore psychological and adaptive functioning in pediatric pre-transplant patients to identify areas in need of intervention.

Additional Children’s National staff involved in the study include:

  • Asha Moudgil, M.D., medical director, Kidney Transplant
  • Paige Johnson, Psychology resident

 

Jacob Smith

Q&A with Pediatric Surgical Innovation Fellow Jacob Smith, M.D.

Jacob Smith

Jacob Smith, M.D.

Jacob Smith, M.D. is currently a fellow with The Joseph E. Robert, Jr. Fellowship in Pediatric Surgical Innovation at Children’s National Hospital. The fellowship provides is an exciting and dynamic post-graduate research experience focused on biomedical innovation. Participants can focus their work on specific areas of interest. Here, Dr. Smith shares some information on his work with the Urology Department.

Q: How has the Joseph E. Robert, Jr., Fellows in Pediatric Surgical Innovation program allowed you to expand your experience in pediatric urology?

A: The Roberts Fellow program has provided me the ability to work with Michael Hsieh, M.D., who has years of experience in UTI and schistosomiasis research. The UTI research has been a focus of mine and we are working on ways to diagnosis and treat UTI faster in patients. One population that this can benefit are those that deal with recurrent UTIs, such as patients in our spina bifida population.

Q: Talk a little about your work on spina bifida and areas of opportunity for research.

A: Briony Varda, M.D. is heavily involved in our spina bifida program here at Children’s National. I am working with Dr. Varda to develop a database that looks at the use of the emergency department (ED) by children with spina bifida in the Washington, D.C. area. We are also conducting interviews with families and other stakeholders in this population to better understand the factors that drive this population to utilize the ED. We hope that this research can give us a better understanding of the spina bifida care in our area and inform us to provide improvement in care.

Q: How will your work to develop methods to diagnose urinary tract infections faster and how will this benefit our patient population?

A: As mentioned before, a common patient population that deals with recurrent UTIs is the spina bifida population. The protocol that we are attempting to develop would provide us quicker results than the standard urine culture and sensitivity testing that can take anywhere between 48-72 hours to result on average. We hope by providing a quicker result, we can better identify the necessary treatment these patients need to treat their infections. Another scenario that we hope this can be utilized in is for those patients that have recurrent UTI symptoms but negative urine cultures. We hope we can determine if there are viable cells that may be too few to culture but still could cause these symptoms.

Q: What are you currently working on that you are most excited about?

A: As you can tell, a lot of my research is conducted around the spina bifida population. I am excited to dive into the data we have collected on this population and determine if there are ways we could improve the care of these patients.

Q: What made you interested in specializing in pediatric urology?

A: Pediatric urology is a great field. It has a very diverse patient population. There are a multitude of problems that could arise from multiple congenital malformations to stones and voiding dysfunctions. There is also a range of cases from minor and major reconstruction, robotics, endourology and laparoscopy. One thing that does differentiate pediatric urology is that many of the problems that we repair are congenital and it is a privilege to be able to help these children and families. In fact, I was a patient myself as a child with a ureteral malformation which required surgery. The concept of helping other children with urology concerns brings an obvious “pay-it-forward” mentality that also attracted me to the field. I am excited to obtain the knowledge and skills necessary to treat my future patients.

kidneys with science images

PMA-based PCR amplifies DNA from only live bacteria in urine

kidneys with science images

The question of why urinary tract infection (UTI) symptoms can persist in some patients who have been seemingly appropriately treated with antibiotics and have negative post-antibiotic urine cultures is one that urologists have long sought to answer.

Experts at Children’s National Hospital have successfully developed propidium monoazide (PMA)-based polymerase chain reaction (PCR) methods that amplify DNA from only live bacteria in urine. The study’s results, published in Frontiers, suggest that non-PMA bound DNA from live bacteria can be present in urine, even after antibiotic treatment.

PMA has been shown to differentiate between non-viable and viable bacteria in various settings. However, its effectiveness in urine has not been previously studied.

The question of why urinary tract infection (UTI) symptoms can persist in some patients who have been seemingly appropriately treated with antibiotics and have negative post-antibiotic urine cultures is one that urologists have long sought to answer.

“One theory is that very low levels of bacteria that don’t show up on cultures may be the cause,” says Michael Hsieh, M.D., director of Transitional Urology at Children’s National and senior author of the study.

Although PCR has previously been used to try and confirm this theory, the use of this method has been criticized because PCR can amplify DNA from dead bacteria (which obviously don’t cause UTI).

The authors developed a PCR test that selectively detects DNA from live bacteria. In a preclinical setting, results show that with the use of antibiotics, cultures collected can be negative but urine can contain DNA from live bacteria, as detected using the PCR test.

“We think something similar can occur in patients and we show some data in the paper confirming the PCR test can work with patient samples,” Dr. Hsieh adds. “I’m excited that we might finally have an explanation as to why some patients have persistent UTI symptoms after antibiotic treatment.”

Namely, he adds, that these patients still have a UTI. This may lead to better follow-up management of patients with UTI. The next step is to confirm the theory in patients.