Research Institute Archives

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

October 5, 2023

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:

Children’s National announces new professorships

October 2, 2023

Robert Keating, M.D., Brian Rood, M.D., and Catherine Bollard, M.D., M.B.Ch.B.

Children’s National Hospital named Robert Keating, M.D., as the McCullough Distinguished Professor of Neurosurgery. He serves as the chief of neurosurgery and co-director of the high-intensity focused ultrasound (HIFU) program at Children’s National.

Children’s National Hospital named Brian Rood, M.D., as the Kurt D. Newman, M.D., Professor of Neuro-Oncology. He serves as director of clinical neuro-oncology and medical director of the Brain Tumor Institute at Children’s National.

Children’s National Hospital elevated Catherine Bollard, M.D., M.B.Ch.B., to the Dr. Robert J. and Florence T. Bosworth Distinguished Professor of Cancer and Transplantation Biology Research. She is the Interim Executive Vice President and Chief Academic Officer and Interim Director, Children’s National Research Institute. She also serves as the director of the Center for Cancer and Immunology Research and director of the Program for Cell Enhancement and Technologies for Immunotherapy at Children’s National.

About the awards

Professorships at Children’s National support groundbreaking work on behalf of children and their families and foster new discoveries and innovations in pediatric medicine. These appointments carry prestige and honor that reflect the recipient’s achievements and donor’s forethought to advance and sustain knowledge. Children’s National is grateful for its generous donors, who have funded 47 professorships.

Dr. Keating is a longstanding leader in neurosurgery research and care. His areas of expertise include brain tumors, traumatic brain injuries, craniofacial anomalies, Chiari malformations and spinal dysraphism. With Dr. Keating’s leadership, the neurosurgery department is pioneering innovations such as HIFU, a non-invasive therapy using focused ultrasound waves to ablate a focal area of tissue. It can treat tumors located in difficult locations of the brain, movement disorders and epilepsy. Children’s National was one of the first pediatric hospitals in the nation to use HIFU for neuro-oncology patients.

“Our goal is to elevate our top-ranked program to even greater heights,” says Dr. Keating. “We will continue to use cutting-edge technology and non-invasive approaches to make the knife obsolete in pediatric neurosurgery and improve outcomes for children.”

Dr. Rood studies the biology of pediatric brain tumors. He focuses on protein signatures and biomarkers specific to different types of brain cancers. His study of neoantigens is informing the development of T-cell immunotherapies to target a tumor’s unique proteins.

“Immunotherapy is revolutionizing how we treat childhood brain tumors — safely, effectively and with the precision made possible by using a patient’s own cells,” says Dr. Rood. “This professorship enables our team to advance this revolution, which will save lives and improve lifetimes.”

Dr. Bollard received the Dr. Robert J. and Florence T. Bosworth Professor of Cancer and Transplantation Biology Research in 2018 to support her work to develop cell and gene therapies for patients with cancer and underlying immune deficiencies. Her professorship has been elevated to a distinguished professorship to amplify her research and celebrate her accomplishments in the field of immunotherapy.

About the donor

These appointments were made possible through an extraordinary $96 million investment from an anonymous donor family for rare pediatric brain tumor research and care. It is one of the hospital’s largest donations and will transform the hospital’s ability to give patients with rare brain cancer a better chance at healthy lifetimes.

The anonymous family brings a depth of compassion for children facing rare and often challenging diagnoses. Their partnership will immediately advance every aspect of our globally recognized leadership to create new, more effective treatments.

Their investment also endowed the Professorship in Molecular Neuropathology. We look forward to bestowing that honor on a Children’s National pediatric leader.

Andrea L. Gropman, M.D., FAAP, FACMG, FANA, named as the Margaret O’Malley Professor of Genetic Medicine

September 25, 2023

Children’s National Hospital named Andrea L. Gropman, M.D., FAAP, FACMG, FANA, as the Margaret O’Malley Professor of Genetic Medicine at Children’s National Hospital.

Dr. Gropman serves as Chief of the Division of Neurogenetics and Developmental Pediatrics at Children’s National Hospital. She is also a Professor of Pediatrics and Professor of Neurology at George Washington School of Medicine and Health Sciences.

About the award

Dr. Gropman joins a distinguished group of Children’s National physicians and scientists who hold an endowed chair. The Margaret O’Malley Professor of Genetic Medicine is one of 47 endowed chairs at Children’s National.

Professorships support groundbreaking work on behalf of children and their families and foster new discoveries and innovations in pediatric medicine. These appointments carry prestige and honor that reflect the recipient’s achievements and donor’s forethought to advance and sustain knowledge.

Dr. Gropman’s research focuses on neuroimaging, inborn errors of metabolism such as urea cycle disorders and mitochondrial disorders, and neurogenetics. She is the principal investigator of the Urea Cycle Disorders Consortium (UCDC) and the UCDC imaging consortium. She is the deputy clinical director of the Mito EpiGen Program.

Thomas and Mary Alice O’Malley, through their vision and generosity, are ensuring that Dr. Gropman and future holders of this professorship will launch bold, new initiatives to rapidly advance the field of pediatric genetic medicine, elevate our leadership and improve the lifetimes of children with genetic diseases.

About the donors

Tom and Mary Alice O’Malley have partnered with Children’s National to improve the lives of patients with urea cycles disorders for more than two decades. In 2003, their transformational philanthropy helped launch the Urea Cycle Disorders Consortium. This pioneering network grew to include 16-sites worldwide. It garnered 20 years of funding from the NIH’s Rare Diseases Clinical Research Network — the only center to sustain continuous funding over this period. This consortium’s research has yielded multiple effective treatment strategies, including government approval of three lifesaving therapies.

“The O’Malley family’s steadfast generosity helped us grow into the robust community of investigators and families we are today,” says Dr. Gropman. “They transformed care for UCD patients everywhere.”

A destination for pediatric oncology care: Children’s National Hospital’s T-cell therapy trials

September 19, 2023

When children are diagnosed with pediatric cancer, most doctors are forced to reach for the same standard therapies that were available decades ago. Research oncologists at Children’s National Hospital are changing that with clinical trials that will hopefully train the body’s immune system – specifically its T cells – to fight the tumors.

Holly Meany, M.D., and her colleague Amy Hont, M.D., oncologists and research scientists at the Center for Cancer and Immunology Research, have put together a pair of clinical trials that are investigating two pathways for using T cells to go after solid tumors.

“At Children’s National, we have a novel immunotherapy to offer to patients with relapsed or refractory solid tumors,” said Meany, director of the Solid Tumor Program. “This is a patient population who has failed standard therapy, so new technologies and treatments are always needed in this group.”

Where we started

Meany’s trial laid the foundation. She began the center’s research using a patient’s own blood sample to develop a targeted therapy and evaluate the safety and efficacy of this approach. In her study, scientists isolated the T cells, grew millions in a lab and reinfused them into the patient. The cells were replicated in an environment that was rich in three proteins that are commonly found on the surface of solid tumor cancer cells.

“Our hope and hypothesis are that when we give the T cells back to the patient, those T cells circulate and hunt down the cancer cells that have the tumor proteins,” Meany said. “We are hoping to use the patient’s own immune system to attack the cancer in an enduring way.”

Where we are headed

Hont’s phase 1 trial, which is currently recruiting participants, builds on Meany’s work using a healthy donor whose T cells have not been impacted by chemotherapy or other treatments. The cells can be prepared, stored and readily available for patients who need them. They are also matched through specific proteins on the patient’s own cells to bolster their effectiveness. The participants in this trial have Wilms tumors, rhabdomyosarcoma, neurosarcoma, soft tissue sarcoma or neuroblastoma, but conventional therapies including chemotherapy, radiation or surgery were unable to fully treat the disease.

In both studies, Hont said that the T cells have been given in an outpatient setting with fewer side effects compared to other cancer treatments aimed at high-risk malignancies.

“This allows patients to really maintain a good quality of life during a particularly hard time,” Hont said. “Also, these T cells are designed to act in the body the way that our immune system acts in a physiologic way. This means patients typically don’t have the severe side effects that we think of with chemotherapy or other therapies.”

Children’s National leads the way

The team at Children’s National is one of the few in the country to offer this kind of T-cell therapy for solid tumors. “Immunotherapy has been challenging for this patient population because the tumors are adept at finding out ways to evade treatment,” Hont said. “Giving patients a chance to receive a targeted T-cell therapy, while also maintaining a high quality of life, is something that’s special here.”

Researchers publish first-ever atlas of cancerous mutations in histones

September 12, 2023

Leading genetic researchers at Children’s National Hospital have published the first pan-cancer atlas of key mutations that can drive molecular changes leading to tumors, creating a roadmap that could lead to new treatments for brain tumors and other cancers.

The research – published in npj Genomic Medicine – presents the first-of-its-kind atlas of histone mutations across pediatric, adolescent/young adult and adult cancers. The novel genetic work offers a framework allowing specific cancers to be redefined in the context of changes in histones, which are essential proteins that provide the structural support for chromosomes.

The big picture

“One of the major challenges that we face every day with pediatric, aggressive tumors, including pediatric high-grade gliomas, is that these tumors grow fast. Doctors often have to give patients 9 to 12 months from diagnosis,” said Javed Nazarian, Ph.D., scientific director of the Brain Tumor Institute at Children’s National and principal investigator at the Center for Genetic Medicine Research. “Children’s National has put together a team of clinicians that are truly devoted to finding a therapy for pediatric high-grade gliomas and aggressive pediatric brain tumors. Our dedicated team empowers translational research, from bench to bedside and reverse translation.”

In 2023, the American Cancer Society estimates that 9,910 children under age 15 will be diagnosed with cancer, making it the second leading cause of death among children. Because of treatment advances, 85% will survive, but many will be left with lifelong disabilities from their treatment. Nazarian and his team believe that identifying the underlying molecular alterations leading to cancers will be essential to finding new therapies that extend life expectancies and preserve quality of life.

The fine print

Histones are essential cellular structures, which prevent DNA from getting tangled. Nazarian and other researchers are investigating whether errors in histones could lead to cancers, including high-grade gliomas and other particularly sinister tumors that can strike young children. By mapping the mutations of the histone-encoding genes, Nazarian and his team believe they can find the drivers of tumors in many pediatric and adult cancers. In studying more than 12,000 tumors for the pan-cancer atlas, they cataloged patient ages, survival outcomes and tumor locations to reveal important trends among different cancers.

Overall, the team found that 11% of tumors had somatic histone mutations, with the highest rates observed among chondrosarcoma, a type of bone cancer (67%); pediatric high-grade glioma, a type of cancer that attacks glial cells in the brain and spinal cord (>60%); and lymphoma, a category of cancers in the lymph system (>30%).

“I think one implication of our study is that we are looking at the epigenomic changes of these mutations in a new light,” Nazarian said. “These mutations are not just specific to a particular tumor type, but they are indeed across a large spectrum of cancer types, and they come in different flavors that could potentially show a new avenue for treatments.”

“Mask up!” Soon, AI may be prompting healthcare workers

September 6, 2023

Researchers at Children’s National Hospital are embarking on an effort to deploy computer vision and artificial intelligence (AI) to ensure medical professionals appropriately use personal protective equipment (PPE). This strikingly common problem touches almost every medical specialty and setting.

With nearly $2.2 million in grants from the National Institutes of Health, the team is combining their expertise with information scientists at Drexel University and engineers at Rutgers University to build a system that will alert doctors, nurses and other medical professionals of mistakes in how they are wearing their PPE. The goal is to better protect healthcare workers (HCWs) from dangerous viruses and bacteria that they may encounter — an issue laid bare with the COVID-19 pandemic and PPE shortages.

“If any kind of healthcare setting says they don’t have a problem with PPE non-adherence, it’s because they’re not monitoring it,” said Randall Burd, M.D., Ph.D., division chief of Trauma and Burn Surgery at Children’s National and the principal investigator on the project. “We need to solve this problem, so the medical community will be prepared for the next potential disaster that we might face.”

The big picture

The World Health Organization has estimated that between 80,000 and 180,000 HCWs died globally from COVID-19 between January 2020 and May 2021 — an irreplaceable loss of life that created significant gaps in the pandemic response. Research has shown that HCWs had an 11-fold greater infection risk than the workers in other professions, and those who were not wearing appropriate PPE had a 1/3 higher infection risk, compared to peers who followed best practices.

Burd said the Centers for Disease Control and Prevention has recommended that hospitals task observers to stand in the corner with a clipboard to watch clinicians work and confirm that they are being mindful of their PPE. However, “that’s just not scalable,” he said. “You can’t always have someone watching, especially when you may have 50 people in and out of an operating room on a challenging case. On top of that, the observers are generally trained clinicians who could be filling other roles.”

What’s ahead

Bringing together the engineering talents at Drexel and Rutgers with the clinical and machine-learning expertise at Children’s National, the researchers plan to build a computer-vision system that will watch whether HCWs are properly wearing PPE such as gloves, masks, eyewear, gowns and shoe covers.

The team is contemplating how the system will alert HCWs to any errors and is considering haptic watch alerts and other types of immediate feedback. The emerging power of AI brings tremendous advantages over the current, human-driven systems, said Marius George Linguraru, D.Phil., M.A., M.Sc., the Connor Family Professor in Research and Innovation at Children’s National and principal investigator in the Sheikh Zayed Institute for Pediatric Surgical Innovation.

“Human observers only have one pair of eyes and may fatigue or get distracted,” Linguraru said. “Yet artificial intelligence, and computers in general, work without getting tired. We are excited to figure out how a computer can do this work – without ever blinking.”

Children’s National Hospital leads the way

Linguraru says that Children’s National and its partners make up the ideal team to tackle this vexing challenge because of their ability to assemble a multidisciplinary team of scientists and engineers who can work together with clinicians. “This is a dialogue,” he said. “A computer scientist, like myself, needs to understand the intricacies of complicated clinical realities, while a clinician needs to understand how AI can impact the practice of medicine. The team we are bringing together is intentional and poised to fix this problem.”

Cell therapy mitigates neurological impacts of cardiac surgery in pre-clinical model

September 6, 2023

Differences of cortical fractional anisotropy between cardiopulmonary bypass and control (left), cardiopulmonary bypass + mesenchymal stromal cells and cardiopulmonary bypass (center), and 3 groups (right).

A pre-clinical study in the journal JACC: Basic to Translational Science shows that infusing bone marrow-derived mesenchymal stromal cells (BM-MSCs) during cardiac surgery provides both cellular-level neuroprotection for the developing brain and improvements in behavior alterations after (or resulting from) surgery.

What this means

According to lead author Nobuyuki Ishibashi, M.D., Oxidative and inflammatory stresses that are thought to be related to cardiopulmonary bypass cause prolonged microglia activation and cortical dysmaturation in the neonatal and infant brain. These issues are a known contributor to neurodevelopmental impairments in children with congenital heart disease.

This study found that, in a pre-clinical model, the innovative use of cardiopulmonary bypass to deliver these mesenchymal stromal cells minimizes microglial activation and neuronal apoptosis (cell death), with subsequent improvement of cortical dysmaturation and behavioral alteration after neonatal cardiac surgery.

Additionally, the authors note that further transcriptomic analyses provided a possible mechanism for the success: Exosome-derived miRNAs such as miR-21-5p, which may be key drivers of the suppressed apoptosis and STAT3-mediated microglial activation observed following BM-MSC infusion.

Why it matters

Significant neurological delay is emerging as one of the most important current challenges for children with congenital heart disease, yet few treatment options are currently available.

Applications of BM-MSC treatment will provide a new therapeutic paradigm for potential MSC-based therapies as a form of neuroprotection in children with congenital heart disease.

Children’s National Hospital leads the way

The Ishibashi lab is the first research team to demonstrate the safety, efficacy and utility of using cardiopulmonary bypass to deliver BM-MSCs with the goal of improving neurological impairments in children undergoing surgery for congenital heart disease. In addition to this pre-clinical research, a phase 1 clinical trial, MeDCaP, is underway at Children’s National.

Recent additional funding from the NIH will allow the team to identify molecular signatures of BM-MSC treatment and mine specific BM-MSC exosomes for unique cardiopulmonary bypass pathology to further increase understanding of precisely how and why this cell-based treatment shows success.

Children’s National joins team to use AI to expand health knowledge in Kenya

August 16, 2023

Children’s National Hospital is joining a team of global health researchers to use large language models (LLMs) like ChatGPT to help Kenyan youth learn about their health and adopt lifestyles that may prevent cancer, diabetes and other non-communicable diseases.

The work, which is one of nearly 50 Grand Challenges Catalyzing Equitable Artificial Intelligence (AI) Use grants announced by the Bill & Melinda Gates Foundation, will harness the emerging power of AI to empower young people with information that they can carry through adulthood to reduce rates of unhealthy behaviors including physical inactivity, unhealthy diet and use of tobacco and alcohol.

“We are thrilled to be part of this effort to bring our AI expertise closer to young patients who would benefit dramatically from technology and health information,” said Marius George Linguraru, D.Phil., M.A., M.Sc., a co-principal investigator for the project, the Connor Family Professor in Research and Innovation at Children’s National and principal investigator in the Sheikh Zayed Institute for Pediatric Surgical Innovation. “Using generative AI, we will build an application to enhance the knowledge, attitudes and healthy habits of Kenyan youth and use this as a foundation to improve health inequities around the globe.”

Why it matters

A lower middle-income country located on the east coast of Sub-Saharan Africa, Kenya is home to 50 million people and one of the continent’s fastest-growing economies. English is one of Kenya’s official languages, and the country has been recognized as a technology leader in Africa, with 82% of Kenyans having phone connectivity. Taken together, these factors make the country an ideal location to deploy an LLM-based platform designed to improve health information and attitudes.

The Gates Foundation selected this project from more than 1,300 grant applications. The nearly 50 funded projects are aimed at supporting low- and middle-income countries to harness the power of AI for good and help countries participate in the AI development process. The project’s findings will contribute to building an evidence base for testing LLMs that can fill wide gaps in access and equitable use of these tools. Each of the grants provides an opportunity to mitigate challenges experienced by communities, researchers and governments.

What’s next

The project development will be led by the National Cancer Institute of Kenya, with Linguraru and other global experts advising the effort from Kenyan institutions and Stanford University. Researchers plan to enroll youth from universities, shopping malls, markets, sporting events and other high-traffic locations. The study will look at participants’ risk factors and how their attitudes toward healthier lifestyles changed after engaging with the new LLM platform.

“The team is thrilled to be selected as one of the nearly 50 most promising AI proposals in the Gates Foundation Grand Challenge competition, and we look forward to seeing how our work can benefit the health of Kenyan youth,” said Dr. Martin Mwangi, principal investigator for the project and head of the Cancer Prevention and Control Directorate at the National Cancer Institute of Kenya. “If successful, we hope to share this model and the expertise we gain to expand health equity and knowledge to other regions.”

JAMA Pediatrics editorial: A better approach for newborn screening

August 7, 2023

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.

Pediatric hospitals underutilize systems to get at social challenges impacting health

July 31, 2023

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, 10^th 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.”

Survey: Teen parents with support report greater well-being

July 28, 2023

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.”

New Becker muscular dystrophy drug on the horizon

July 20, 2023

Muscular dystrophies are a collection of genetic disorders that affect boys and cause progressive loss of muscle strength and disability throughout childhood. They impact the protein dystrophin, and other proteins associated with it, which helps strengthen muscles and protect them from injury.

A new corticosteroid – vamorolone – improves symptoms of Becker muscular dystrophy (BMD) with fewer side effects than the off-label treatments currently offered to patients, according to new research from Children’s National Hospital published in iScience.

Currently, there are no drugs approved to treat BMD, an inherited disorder that causes progressive muscle weakness. In preclinical models, daily treatment with vamorolone improved muscle strength and structure with results comparable to prednisolone, which is sometimes prescribed to patients with BMD. Unlike prednisolone, vamorolone is not known to stunt growth, weaken bone and cause negative behaviors.

“Patients with muscular dystrophy can fall anywhere on the spectrum from asymptomatic to facing life-threatening cardiac complications,” said Christopher Heier, Ph.D., principal investigator at the Center for Genetic Medicine Research at Children’s National. “We are excited to have our eye on a drug that may help manage the disease progression, without all the harmful side effects of the steroids currently being offered.”

The big picture

Muscular dystrophies are a collection of genetic disorders that affect boys and cause progressive loss of muscle strength and disability throughout childhood. They impact the protein dystrophin, and other proteins associated with it, which helps strengthen muscles and protect them from injury.

The FDA has approved four drugs to help mitigate the impact of Duchenne muscular dystrophy (DMD), the most severe and most common form of the disease, with dozens more drugs in the research pipeline for that disease subtype. In some cases, these drugs convert DMD into BMD, which is less severe but still greatly affects the quality of life. As a result, the number of patients living with BMD is growing, yet only two drugs are being studied to treat the Becker form of the disease.

Why we’re excited

The Food and Drug Administration is nearing approval for vamorolone to treat DMD. Researchers including Nikki McCormack, Ph.D., a postdoctoral fellow at Children’s National, found it has an added characteristic that makes it particularly helpful to BMD. “Excitingly, by reducing inflammatory signaling in the muscle, we find vamorolone can actually help to correct the underlying dystrophin protein deficiency in BMD through a newly discovered RNA-targeting mechanism.”

Investigators at Children’s National have been interested in expanding vamorolone’s possible use to BMD. Their work builds upon research finished late last year, when they created the first preclinical model to study drugs that could treat BMD. The model provides tremendous hope for those suffering from BMD around the world.

“By creating a pre-clinical model to test possible treatments, we are creating hope for boys living with this life-changing disorder,” said Alyson Fiorillo, Ph.D., principal investigator at the Center for Genetic Medicine Research at Children’s National. “This model, and the drugs it will lead to, will revolutionize how we treat those children living with this disorder.”

Marius George Linguraru, D.Phil., M.A., M.Sc., named as Connor Family Professor of Research and Innovation

July 17, 2023

“Artificial Intelligence may be the greatest tool we have for improving the quality of and access to medical care for children, especially those most vulnerable to health system inequities,” said Dr. Linguraru. “This professorship will help me extend our leadership in this vital field. The tools and care strategies we develop will benefit children worldwide.”

Children’s National Hospital named Marius George Linguraru, D.Phil., M.A., M.Sc., as the Connor Family Professor of Research and Innovation at Children’s National Hospital.

Dr. Linguraru is a principal investigator in the Sheikh Zayed Institute for Pediatric Surgical Innovation at Children’s National. He directs the award-winning Precision Medicine Imaging Group. He is also a professor of Radiology and Pediatrics and secondary professor of Biomedical Engineering at George Washington University.

About the award

Dr. Linguraru joins a distinguished group of 42 Children’s National physicians and scientists who hold an endowed chair. Professorships at Children’s National support groundbreaking work on behalf of children and their families and foster new discoveries and innovations in pediatric medicine. These appointments carry prestige and honor that reflect the recipient’s achievements and donor’s forethought to advance and sustain knowledge.

Dr. Linguraru is a global leader in harnessing the power of quantitative imaging and machine learning to rapidly and positively impact children’s health. Dr. Linguraru and his team use artificial intelligence (AI) and digital technology innovations to improve access to healthcare and the understanding of rare and newborn diseases. Their work enables clinicians to deliver care faster, evaluate responses to treatments and prevent health complications. They have positioned Children’s National as an international leader in the development of pediatric AI to ensure equitable care for all children.

“Artificial Intelligence may be the greatest tool we have for improving the quality of and access to medical care for children, especially those most vulnerable to health system inequities,” said Dr. Linguraru. “This professorship will help me extend our leadership in this vital field. The tools and care strategies we develop will benefit children worldwide.”

About the donors

The Connor family, through their vision and generosity, are ensuring that Dr. Linguraru and future holders of this professorship will launch bold, new initiatives to rapidly advance the field of pediatric research and innovation, elevate our leadership and improve the lifetimes of children.

“We strongly believe in the power of academic entrepreneurship to improve the health and wellbeing of children,” said Ed and Chris Connor, who are longtime donors and members of the Children’s National community. “This endowment is our way of supporting Children’s National’s work in research and innovation and recognizing Dr. Linguraru’s international leadership in using AI to benefit child health.”

x-ray of child with congenital heart disease

Cell therapy research for neuroprotection in congenital heart disease receives another $3.3 million from NIH

July 11, 2023

Significant neurological delay is emerging as one of the most important current challenges for children with congenital heart disease, yet few treatment options are currently available.

The research lab of Nobuyuki Ishibashi, M.D., at Children’s National Hospital, recently received $3.3 million in additional funding for research into cell therapy for neuroprotection in children with congenital heart disease. The new support comes from the National Heart, Lung and Blood Institute (NHLBI) of the National Institutes of Health.

The research goal

The overarching goal of the award is to establish detailed molecular signatures from critical cell populations for tissue repair and regeneration at single cell resolution after bone marrow-derived mesenchymal stromal cell (BM-MSC) delivery. The team has shown cellular, structural and behavioral improvements in pre-clinical models after delivery of BM-MSCs through cardiopulmonary bypass for children with congenital heart disease. However, the mechanisms underlying the therapeutic action of BM-MSCs still remain largely unknown. This R01 renewal will address the key knowledge gap.

Why it matters

Significant neurological delay is emerging as one of the most important current challenges for children with congenital heart disease, yet few treatment options are currently available.

The Ishibashi lab has demonstrated the efficacy and utility of using cardiopulmonary bypass to deliver BM-MSCs to improve neurological impairments in children undergoing surgery for congenital heart disease. Most notably, this included development of a phase 1 clinical trial, MeDCaP, at Children’s National.

The big picture

Together with the ongoing clinical trial established from the previous award, identifying molecular signatures of BM-MSC treatment and mining specific BM-MSC exosomes for unique cardiopulmonary bypass pathology will significantly improve understanding of this cell-based treatment. This work will also provide a new therapeutic paradigm for potential cell-free MSC-based therapies for neuroprotection in children with congenital heart disease.

Attendees at the inaugural symposium on AI in Pediatric Health and Rare Diseases

AI: The “single greatest tool” for improving access to pediatric healthcare

June 26, 2023

The daylong event drew experts from the Food and Drug Administration, Pfizer, Oracle Health, NVIDIA, AWS Health and elsewhere to start building a community aimed at using data for the advancement of pediatric medicine.

The future of pediatric medicine holds the promise of artificial intelligence (AI) that can help diagnose rare diseases, provide roadmaps for safer surgeries, tap into predictive technologies to guide individual treatment plans and shrink the distance between patients in rural areas and specialty care providers.

These and dozens of other innovations were contemplated as scientists came together at the inaugural symposium on AI in Pediatric Health and Rare Diseases, hosted by Children’s National Hospital and the Fralin Biomedical Research Institute at Virginia Tech. The daylong event drew experts from the Food and Drug Administration, Pfizer, Oracle Health, NVIDIA, AWS Health and elsewhere to start building a community aimed at using data for the advancement of pediatric medicine.

“AI is the single greatest tool for improving equity and access to health care,” said symposium host Marius George Linguraru, D.Phil., M.A., M.Sc., principal investigator at the Sheikh Zayed Institute for Pediatric Surgical Innovation. “As a population, kids are vastly underrepresented in scientific research and resulting treatments, but pediatric specialties can use AI to provide medical care to kids more efficiently, more quickly and more effectively.”

What they’re saying

Scientists shared their progress in building digital twins to predict surgical outcomes, enhancing visualization to increase the precision of delicate interventions, establishing data command centers to anticipate risks for fragile patients and more. Over two dozen speakers shared their vision for the future of medicine, augmented by the power of AI:

Keynote speaker Subha Madhavan, Ph.D., vice president and head of AI and machine learning at Pfizer, discussed various use cases and the potential to bring drugs to market faster using real-world evidence and AI. She saw promise for pediatrics. “This is probably the most engaging mission: children’s health and rare diseases,” she said. “It’s hard to find another mission that’s as compelling.”
Brandon J. Nelson, Ph.D., staff fellow in the Division of Imaging, Diagnostics and Software Reliability at the Food and Drug Administration, shared ways AI will improve diagnostic imaging and reduce radiation exposure to patients, using more advanced image reconstruction and denoising techniques. “That is really our key take-home message,” he said. “We can get what … appear as higher dose images, but with less dose.”
Daniel Donoho, M.D., a neurosurgeon at Children’s National, introduced the audience to the potential of “Smart ORs”: operating rooms where systems can ingest surgery video and provide feedback and skill assessments. “We have to transform the art of surgery into a measurable and improvable scientific practice,” he said.
Debra Regier, M.D., chief of Genetics and Metabolism at Children’s National, discussed how AI could be used to diagnose and treat rare diseases by conducting deep dives into genetics and studying dysmorphisms in patients’ faces. Already, Children’s National has designed an app – mGene – that measures facial features and provides a risk score to help anyone in general practice determine if a child has a genetic condition. “The untrained eye can stay the untrained eye, and the family can continue to have faith in their provider,” she said.

What’s next

Linguraru and others stressed the need to design AI for kids, rather than borrow it from adults, to ensure medicine meets their unique needs. He noted that scientists will need to solve challenges, such as the lack of data inherent in rare pediatric disorders and the simple fact that children grow. “Children are not mini-adults,” Linguraru said. “There are big changes in a child’s life.”

The landscape will require thoughtfulness. Naren Ramakrishnan, Ph.D., director of the Sanghani Center for Artificial Intelligence & Analytics at Virginia Tech and symposium co-host, said that scientists are heading into an era with a new incarnation of public-private partnerships, but many questions remain about how data will be shared and organizations will connect. “It is not going to be business as usual, but what is this new business?” he asked.

$96 million philanthropic investment will transform rare pediatric brain tumor research and care

June 22, 2023

Children’s National Hospital announced a $96 million investment from an anonymous donor family to transform rare childhood brain tumor research and care. The donation, which strengthens our globally recognized leadership in the field, is one of the largest in the hospital’s history.

Children’s National will harness the investment to recruit more top talent and advance the most promising research. This will produce safer, more effective treatments. It also will elevate standards of care to help children with rare brain tumors thrive for a lifetime.

The big picture

Brain tumors are the most common solid tumors affecting children. They are especially challenging in kids because their brains are still developing. The disease and current treatments can put them at risk for lifelong complications.

The anonymous family’s investment provides new hope for patients who face rare and often challenging brain tumor diagnoses — in the Washington, D.C., community and around the world.

“This incredible partnership will lift up one of the nation’s top pediatric brain tumor programs into the stratosphere,” said Kurt Newman, M.D., president and CEO of Children’s National. “It will immediately propel our best-in-class research and care, allowing us to bring new therapies to children with brain tumors. This fundamentally changes the healthcare journey and long-term outcomes for children and their families.”

Why it’s important

This transformational investment will have a far-reaching impact on our ability to save and improve the lives of children with brain tumors. Funds will fuel collaborative breakthroughs across a range of scientific and psychosocial approaches.

The partnership will supercharge highly individualized and promising treatments for children with brain tumors. We will radically transform the research landscape with a focus on:

Low intensity focused ultrasound (LIFU) – Advancing laboratory research and a clinical program designed to treat childhood brain tumors with LIFU therapy
Cellular immunotherapy – Testing new gene-engineered immune cell products and accelerating their integration into standards of care
Rare Brain Tumor Program – Propelling new clinical trials through the hospital’s national and global leadership in pediatric brain tumor consortia. Already, Children’s National is leading a new collaborative with hospitals in North America, South America and Europe to better understand and find novel treatments for these rare diseases
Neurosurgery innovation – Exploring multiple ways to perform safer, more effective neurosurgery and developing new methods to enhance drug/agent delivery
Precision medicine – Recruiting leading scientists to advance biology-informed therapies that can be targeted for children across a spectrum of brain tumors
Good Manufacturing Practices (GMP) facility – Expanding our GMP, one of the first standalone facilities at a children’s hospital in the country, to translate new discoveries into clinical trials more rapidly
Additional priorities including expansion of clinical research infrastructure and growth of bioinformatics, brain tumor repository and molecular diagnostics initiatives

The partnership also transforms how we approach care. It will power our pursuit of psychosocial, behavioral health and neuroscientific initiatives to help kids live well and cope with the unique circumstances of their diagnosis. We will focus on:

Lifetime health and wellness – Building a world-class research and clinical care program to shape a new paradigm for supporting a child’s physical and emotional health during and long after cancer treatment
Child Mental Health & Behavioral Brain Tumor Lab – Establishing a robust neuro-oncology mental health program that delivers timely interventions and specialized psychiatric care for patient well-being
Additional priorities including a new Neuroscience Nursing Excellence Program and growth of psychosocial support activities that bring comfort and encouragement to children during their treatment journey

Children’s National is proud to lead the way to a better future for pediatric rare brain tumor patients and expand our internationally recognized capabilities for neuro-oncology care.

Community-wide program to support teen parents serves as a model for engagement

June 14, 2023

More than 500 adolescent mothers, caregivers and community members benefitted from a coordinated “collective impact” model to provide support aimed at addressing the litany of strains faced by teen parents, according to a case study published Wednesday in the journal Pediatrics.

Known as the District of Columbia Network for Parenting and Expectant Teens (DC NEXT), the model used well-tested pillars of community organization to provide services and care that bolstered the well-being of pregnant and expectant teens in the city. The case study found that the 3-year-old program, which included teen advisors to help guide the mission, could serve as a model in other areas with high rates of teen parents.

“There’s no system of care for teen parents in Washington, D.C., or really anywhere in our country,” said Yael Smiley, M.D., Children’s National Hospital pediatrician and the study’s lead author. “Our coalition came together to connect the resources and the people who care about young parents and their families to improve their health, their outcomes, their well-being, and set them up for success. We fuse representatives from healthcare, education and housing policy — people who care about creating the very best outcomes possible.”

The patient benefit

More than 140,000 teens became parents in 2021 in the United States. In Washington, D.C., more than 300 babies are born to teens each year, and the rate of births to young teens ages 15 to 17 is double the national average. These young parents often face barriers to childcare and education and poor birth outcomes, often stemming from a lack of access to prenatal care.

To address these obstacles, the network assembled a city-wide network in 2020 to follow the five pillars of an evidence-based “collective impact” model:

Create a common agenda
Design shared measurement systems
Host mutually reinforcing activities
Foster continuous communication
Rely on trusted backbone support

What’s unique

DC NEXT was housed at the District of Columbia Primary Care Association (DCPCA), which received the 3-year, $4.5 million federal grant and provided the leadership. At Children’s National, Dr. Smiley led a team that partnered with DCPCA and Howard University to direct a network of community clinics, nonprofits and other organizations whose mission was to support adolescent parents. Hundreds of client-facing staff members were trained to provide trauma-informed, human-centered care. DC NEXT also engaged directly with teen mothers through well-being surveys that led to improved access to essential programs, including resources for housing and food security. Over 550 young parents and caregivers have been impacted by the program.

The leadership quickly realized that the teen parents needed a voice and created a “context team” of paid teen advisors who provided insights into their unique experiences and needs as parents. They help set the agenda, choose program offerings and tailor communications to their peers.

“If the network can continue to grow and support young parents to achieve their health and well-being goals, the impact will be felt across generations,” said Dr. Smiley.

Germ cell tumor of testicle under microscopy

New research: Genes that drive testicular cancer identified

June 7, 2023

In the largest sequencing study to date on testicular cancer, researchers at Children’s National Hospital have identified genes that contribute to testicular germ cell tumors (TGCT), the most common cancer among young, white men.

The findings, published in European Urology, provide direction for future screening and treatment of this disease, which can strike during the teen years and often runs in families. While treatable when identified early, testicular cancer leads to infertility, mental health issues and sometimes death, making its identification crucial for young adults.

“Testicular cancer is really a young person’s disease,” said Louisa Pyle, M.D., Ph.D. , a pediatrician, medical geneticist and research geneticist at the Children’s National Rare Disease Institute. “Most folks who have testicular cancer are between the ages of 15 and 45. Even though testicular cancer is relatively rare in the cancer world, it results in the greatest number of years lost among all adult cancers.”

What we hope to discover

Dr. Pyle led a research team that included experts at the National Cancer Institute and the University of Pennsylvania to study families with multiple members diagnosed with testicular cancer. They used whole exome sequencing to identify variants in many genes that predisposed patients to TGCT. Their work suggests that multiple variants – inherited together – increased the risk for the disease and provides potential routes for drugs that could be used for prevention and treatment.

“We found many genes that help us understand how testicular cancer happens,” Dr. Pyle said. “Our hope is that we can use that to try to come up with better treatments or better ways to preserve fertility for people with testicular cancer or gonadal differences.”

The patient benefit

Testicular cancer most often strikes men of European ancestry. It is also more common among intersex patients and those with differences in sex development, which is a clinical and research focus for Dr. Pyle. Medically, these are children who have a change in the biological characteristics of sex, including their chromosomes, hormones, gonads or physical body parts.

By studying a more common version of testicular cancer, the team learned about the underlying genetics in a way that will benefit intersex patients.

“One of the things we do in medicine is study a common version of the rare thing,” Dr. Pyle said. “Through this research, we learned that the same genes that cause intersex traits in some patients are also changed in subtle ways for people with testicular cancer. This is a way to study something that could improve care for those kids, by studying a group that has greater numbers.”

Children’s National Research & Innovation Campus

Research campus joins Global Network of Innovation Districts

May 30, 2023

At the RIC’s 12-acre campus in Northwest Washington, D.C., experts from Children’s National work alongside public and private partners in industry, universities, federal agencies, start-up companies and academic medical centers to find solutions to some of science’s most vexing challenges.

The Children’s National Research & Innovation Campus (RIC) has become the first science ecosystem dedicated to pediatric health to join a network of over three dozen innovation districts worldwide that integrate research space with sustainable communities to create models for urban work and living.

Known as the Global Network of Innovation Districts (GNID), the community was conceived to unlock the design of campuses like the RIC to create collaborations among highly trained professionals. At the RIC’s 12-acre campus in Northwest Washington, D.C., experts from Children’s National work alongside public and private partners in industry, universities, federal agencies, start-up companies and academic medical centers to find solutions to some of science’s most vexing challenges. The campus is surrounded by mass transit, open spaces, retail and housing, and it’s built on deep historic roots in the city as the former home of the Walter Reed Army Medical Center.

Kerstin Hildebrandt, vice president of research administration at the Children’s National Research Institute, said the team at the Research & Innovation Campus is excited to maximize its potential by joining this global network of economic drivers that are enhancing their communities and cities.

“We look forward to sharing our best practices, and we want to learn about how our national and international colleagues are tackling complex issues,” she said. “For example, we can learn how others are leveraging their assets to improve their communities and their response to health crises, climate change and other significant challenges.”

The GNID was launched in March of last year by The Global Institute on Innovation Districts (GIID), an international nonprofit focused on the advancement of innovation districts. With an initial group of 23 districts. GIID is now expanding the network to include approximately 20 additional districts that extend across Europe, North America, Latin America, Australia and Asia.

GIID’s Founder Julie Wagner said innovation districts have become a worldwide phenomenon. She said their leaders are recognizing that working and collaborating with their peers — from Melbourne to Medellin — is a powerful strategy to help these complex geographies leverage their assets in new ways.

“We are finding that innovation districts are willing to execute impactful strategies after holding highly curated exchanges with their peers,” Wagner said. “These are the places armed to solve some of the world’s most vexing challenges. From where I sit, we all need to give them as many tools as possible to help them get there.”

New guidance to optimize blood sugar monitoring in cerebral malaria

May 25, 2023

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.”