News Briefs

Children’s National Fetal Medicine Institute hosts 2nd annual International Symposium on the Fetal Brain


The Children’s National Health System Fetal Medicine Institute hosted the 2nd annual International Symposium on the Fetal Brain in August 2017 in Washington, D.C.

Speakers at this year’s symposium focused on the following four areas:

  • Brain Development in an Unsupportive In Utero Environment – Diagnosis and Consequences
  • Supporting Brain Development in the Ex Utero Fetus: How Far Are We From Optimal?
  • Genomic and Epigenomic Mechanisms Underlying Differences in Brain Development
  • The Emergence of Consciousness and Pain Sensation

Adré J. du Plessis, M.B.Ch.B., M.P.H., Director of the Fetal Medicine Institute and Division Chief of Fetal and Transitional Medicine hosted the conference. In his opening remarks Dr. du Plessis noted “Our goal has been to gather together a diverse group from across the spectrum of disciplines focused on the well-being of the fetal brain and to engage all disciplines together.”

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Diana Bianchi, MD gives her keynote presentation on non-invasive fetal testing at the second annual International Symposium on the Fetal Brain.

Invited, internationally renowned speakers presented on diverse topics, including Diana Bianchi, Director of the Eunice Kennedy Shriver National Institute of Child Health and Human Development who spoke on the “Non-Invasive Fetal Testing Beyond Karyotype: What’s in it for the Fetal Brain?”

A new component to the symposium was the clinically-focused breakfast breakout sessions, created based on feedback from attendees of the 2016 Symposium. Sessions covered varied topics such as “Fetal Ultrasound: the Cornerstone of Fetal Neurodiagnosis,” “The Essentials of Neurogenetic Testing,” “Developing a Transitional Fetal-Neonatal Program” and “Using MRI to Advance Fetal Neurodiagnosis.”

The conference started with an exciting discussion by Alistair Gunn, M.B.Ch.B., Head of the Department of Physiology at the University of Auckland. His presentation “Fetal Heart Rate: What It Does and Does Not Tell Us” explored the considerable body of evidence that essentially all decelerations are mediated by chemoreflex responses to repeated hypoxia and that the parasympathetic autonomic nervous system is the critical regulator of both fetal heart rate and heart rate variation in labor.

Following a voting process from the symposium’s external speakers, the inaugural Andrea Poretti Abstract Award was presented to Katherine Ottolini for her poster titled: Breastmilk Feeds Improve Brain Microstructural Development in Very Premature Infants.

For more information about the sessions and speakers at the 2017 Symposium, please visit our website.

Nationally recognized immunotherapy and pathology experts take on new leading roles at Children’s National

Children’s National Health System recently made several exciting leadership announcements in the allergy, immunology and laboratory medicine fields, furthering the hospital’s ongoing commitment to providing the most comprehensive, innovative care for children.

Award-winning hematologist and immunotherapist Catherine Bollard, M.D., M.B.Ch.B., currently chief of the Division of Allergy and Immunology, has been chosen to serve as director of the Children’s Research Institute’s (CRI) Center for Cancer and Immunology Research (CCIR). CCIR includes more than 50 clinicians and scientists performing groundbreaking clinical and translational research in understanding the origins of, and developing and testing novel therapies for childhood cancers and immunologic disorders. The center receives more than $10 million annually from the National Institutes of Health and other external entities. In her new role on the leadership team of CCIR, Dr. Bollard will lead the advancement and oversight of cancer and immunology research performed at Children’s National.

“All of the progress made in cellular immunotherapy here at Children’s National can be attributed to Catherine and her leadership,” says Mark L. Batshaw, M.D., chief academic officer and director of CRI. “We are confident her impact will extend even further in her new role.”

Hemant Sharma, M.D., M.H.S., will assume the role of chief of the Division of Allergy and Immunology. In 2008, he joined the faculty at Children’s National and started the Food Allergy Program, which he directs today. His areas of interest include health disparities and community-based management of food allergy. He is also site principal investigator of novel clinical trials of immunotherapy for peanut allergy. He serves on the Medical Advisory Board of Food Allergy Research and Education (FARE), and was the recipient of the 2016 FARE Vision Award for his contributions to the national food allergy community. Dr. Sharma also serves as the site director of the allergy immunology fellowship program with the National Institutes of Health and has won various teaching awards.

In addition, nationally recognized laboratory medicine expert Meghan Delaney, D.O., M.P.H., has joined Children’s National as chief of pathology and lab medicine. An expert in the field of transfusion medicine, Dr. Delaney will lead efforts to unify Anatomic Pathology and Laboratory Medicine into a single division, while advancing cutting-edge practices in the lab to ensure the highest standard of quality and safety for patients. Dr. Delaney joins Children’s National from Seattle, where she held many leadership positions including serving as medical director at the Pediatric Apheresis Program at Seattle Children’s Hospital & Seattle Cancer Care Alliance, the blood bank at Seattle Children’s Hospital and the Immunohematology & Red Blood Cell Genomics Reference Laboratory at Bloodworks Northwest.

“Dr. Delaney brings extensive experience in laboratory medicine innovation and program-building, and we are confident she will make a lasting impact on our patients,” said Jeffrey Dome, M.D., Ph.D., vice president for the Center for Cancer and Blood Disorders at Children’s National. “Her leadership will bolster our commitment to providing top quality care for our patients through advancement of lab medicine research and treatments.”

Children’s National Health System advances sickle cell disease cure through Doris Duke Charitable Foundation grant

An innovative Children’s National Health System project aimed at improving the only proven cure for sickle cell disease – hematopoietic cell transplantation – will receive more than $550,000 in funding from the Doris Duke Charitable Foundation’s inaugural Sickle Cell Disease/Advancing Cures Awards, which provides grants to advance curative approaches for sickle cell disease. The study, a three-year, multi-center trial that will study a low intensity, chemotherapy-free transplantation approach to cure children with sickle cell disease using a matched related donor, is led by Allistair Abraham, M.D., blood and marrow transplantation specialist, and Robert Nickel, M.D., hematologist, and is one of seven projects receiving approximately $6 million total through the awards.

While transplantation using a matched sibling donor today has a high cure rate (>90 percent) for sickle cell disease, traditional transplant approaches have many risks and side effects in both the short and long term. The study will examine if a chemotherapy-free approach can lead to a successful transplant without resulting in graft-versus-host disease (GVHD). GVHD is one of the most challenging complications of a transplant, in which the transplant immune cells attack the patient’s body. The researchers anticipate that this new transplant approach will be so well tolerated that patients’ quality of life will be maintained and improved throughout the process, with most of the care administered in a clinic setting.

“This approach has proven to be effective for adults with sickle cell disease, so we are grateful for the opportunity to begin this important trial for children thanks to the Doris Duke Charitable Foundation,” says Dr. Abraham. “Children with sickle cell disease are in need of innovative treatments, and we look forward to finding more solutions that improve the quality of life for these patients.”

“Advancing treatment for sickle cell patients to the point where they can live free of the disease is our top priority,” says Dr. Nickel, who is also an assistant professor of pediatrics at the George Washington University School of Medicine and Health Sciences. “This funding is critical to our study and it will accelerate the timeline to achieve the goal of a well-tolerated and safe cure for children with sickle cell disease.”

Matthew Hsieh, M.D., who helped pioneer this work at the National Institute of Health in adults, and Greg Guilcher, M.D., who has used this transplant approach in children, are key collaborators on the project.

The study is projected to begin in December 2018 and continue for three years. The Comprehensive Sickle Cell Disease Program at Children’s National is among the largest in the country, treating more than 1,400 children and young adults with all types of sickle cell disease. Children’s National also offers the largest, most comprehensive blood disorders team in the Washington, D.C., area.

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Promoting a culture of safety with 10,000 good catches

Doctor-putting-mask-on

In today’s fast-paced health care environment, it has become increasingly important to create a culture of safety where improvement opportunities are recognized and welcomed. With medical errors cited as the one of the leading causes of morbidity and mortality in the United States, health care organizations are working to rapidly identify and respond to errors before long-term issues develop.

Improving event reporting is a critical step. To create an effective culture of safety, employees from throughout a hospital or health system must be empowered. They must be educated and have the ability to easily raise awareness of potential problems and risks and they must be able to proactively resolve problems. With this mindset, Children’s National Health System set out to double the number of voluntary safety event reports submitted over a three-year period; the intent was to increase reliability and promote safety culture by hardwiring employee event reporting. With the goal of growing from 4,668 reports in fiscal year 2014 to 9,336 in 2017, the initiative became known as 10,000 Good Catches. And, the positive framing of the endeavor added to a sense of ownership and reporting among staff members.

Following a Donabedian quality improvement framework of structure, process and outcomes, Children’s National formed a multidisciplinary team and identified three key areas for improvement:

  1. Technology: Make reporting user-friendly, fast and easy
  2. Safe to Report: Create a non-punitive environment in which staff feel secure reporting safety events
  3. Makes a Difference: Develop a culture and system to provide feedback and advance meaningful improvements stemming from safety event reporting

Over the next three years, the team, via subcommittees, routinely solicited feedback from front-line users and met as a larger group monthly to propose interventions, review quantitative data and prioritize next steps. In tandem, employees were educated through internal communications on how, what and when to report. The primary outcome measure was the number of safety event reports submitted through the electronic reporting platform. Event report submission time, number of departments submitting events and percent of safety event reports submitted anonymously were also tracked.

These efforts paid off, as Children’s National more than doubled the number of voluntary safety event reports filed over the three-year period from 4,668 in fiscal year 2014 to 10,971 in 2017, with steady annual improvements. Other metrics included decreased event reporting time and anonymous reports. Interestingly, there was a marked increase in the number of departments submitting reports.

This successful initiative not only resulted in increased safety reporting and engagement, but was an important step toward improving organizational reliability and building a culture of safety first. Future steps will focus on how to sustain improvement, how to more efficiently leverage reporting data and how to apply the data to prevent future safety events.

$250K awarded to six winners presenting innovative pediatric medical devices

SZI Symposium Winners

Six companies presenting innovative medical device solutions that address significant unmet needs in pediatric health were awarded a total of $250,000 in grant money yesterday in San Jose, Calif. at the Fifth Annual Pediatric Device Innovation Symposium, organized by the Sheikh Zayed Institute for Pediatric Surgical Innovation at Children’s National Health System.

The “Make Your Medical Device Pitch for Kids!” competition is sponsored by the National Capital Consortium for Pediatric Device Innovation (NCC-PDI), an FDA-funded consortium led by Children’s National and the A. James Clark School of Engineering at the University of Maryland. Four companies were awarded $50,000 each and two were awarded $25,000. The six winners were selected from a field of twelve finalists. A record 98 total submissions from five countries were received for the competition this year.

“To improve care for children, it is imperative that we recognize and encourage relevant new solutions in pediatric medical devices, especially in light of the challenges innovators face in addressing this specialized market,” said Kurt Newman, M.D., president and CEO of Children’s National. “Children’s National is committed to fostering collaboration among innovators, clinicians, policy makers and investors to advance pediatric device development for the benefit of children everywhere.”

This year’s winning innovations receiving $50,000 awards are:

  • CorInnova, Houston, Texas – soft robotic, non-blood-contacting biventricular cardiac assist device for the treatment of heart failure in children
  • Green Sun Medical, Fort Collins, Colo. – novel device that provides necessary pressure for the correction of spinal deformity while providing real-time feedback to clinicians
  • Hub Hygiene and Georgia Institute of Technology, Atlanta, Ga. – low-cost, single-use cleaning technology to prevent central line-associated blood stream infections (CLABSI), a hospital-acquired infection by pediatric ICU patients
  • NAVi Medical Technologies, Houston, Texas – device to provide accurate information about the localization of an umbilical venous catheter (UVC) used in critically-ill newborns to reduce the risk of catheter malposition

Winning innovations receiving $25,000 awards are:

  • Prapela, LLC, Boston, Mass. – novel “baby box” that will allow for a non-pharmacological approach to help drug-exposed infants relax and sleep during withdrawal and post-withdrawal care
  • X-Biomedical, Inc., Philadelphia, Pa. – portable surgical microscope for use in surgeries for treatable causes of blindness in low-income countries and under-resourced setting

“We are honored to recognize these outstanding innovations with this funding,” said Kolaleh Eskandanian, Ph.D., executive director of the Sheikh Zayed Institute and NCC-PDI. “We are even more excited about welcoming this new cohort of companies to our family of pediatric device startups and entrepreneurs. Together we can move the needle a bit faster and safer to bring pediatric products to market.”

She added that in addition to the financial support and consultation services through NCC-PDI, the awardees can leverage the validation received through this highly competitive process to raise the additional capital needed for commercialization. Since inception in 2013, NCC-PDI has supported 67 pediatric devices and the companies and research labs owning these devices have collectively raised $55 million in additional funding.

The twelve finalists each made five-minute presentations to the symposium audience and then responded to judges’ questions. Finalists also included Anecare, LLC, Salt Lake City, Utah; ApnoSystems, Buenos Aires, Argentina; Deton Corp., Pasadena, Calif.; Kite Medical, Dublin, Ireland; Moyarta 2, LLC, The Plains, Va.; and Oculogica, Inc., New York, N.Y.

Serving on the distinguished panel of judges were Susan Alpert, M.D., of SFA Consulting, a former director of the FDA Office of Device Evaluation and former senior vice president and chief regulatory officer of Medtronic; Charles Berul, M.D., co-director, Children’s National Heart Institute; Andrew Elbardissi, M.D., of Deerfield Management; Rick Greenwald, Ph.D., of the New England Pediatric Device Consortium (NEPDC); James Love, J.D., of Oblon; Josh Makower, M.D., of NEA; Jennifer McCaney, Ph.D., of MedTech Innovator; Jackie Phillips, M.D., of Johnson & Johnson; and Tracy Warren of Astarte Ventures.

The pitch competition is a highlight of the annual symposium organized by the Sheikh Zayed Institute at Children’s National, designed to foster innovation that will advance pediatric healthcare and address the unmet surgical and medical device needs for children. New this year, the symposium co-located in a joint effort with The MedTech Conference powered by AdvaMed, the premier gathering of medtech professionals in North America.

Keynote speakers at the event included Daniel Kraft, M.D., faculty chair of Medicine & Neuroscience, Singularity University and executive director, Exponential Medicine; Vasum Peiris, M.D., chief medical officer, Pediatrics and Special Populations, FDA;  and Alan Flake, M.D., director of Center for Fetal Research, Children’s Hospital of Philadelphia.

Panel discussions focused on gap funding for pediatric innovation, the journey from ideation to commercialization, and the pediatric device needs assessment in the future regulatory environment.

Children’s National welcomes Marva Moxey-Mims, M.D., renowned nephrologist, as incoming Division Chief

A close-up of Dr. Marva Moxey Mims at Children's National.

Marva Moxey-Mims, M.D., a leading expert in chronic kidney disease and glomerular disease who has conceptualized and overseen multicenter clinical studies aimed at improving chronic kidney disease treatment, has been named Chief of Pediatric Nephrology at Children’s National Health System.

Dr. Moxey-Mims comes to Children’s National from the National Institute of Diabetes and Digestive and Kidney Diseases at the National Institutes of Health where she served as Deputy Director for clinical science and oversaw a research portfolio that included clinical trials for kidney disease and genitourinary dysfunction in adults and children. As Pediatric Nephrology Division Chief, Dr. Moxey-Mims plans to add new staff and restructure a division already ranked among the nation’s leaders by U.S. News & World Report in order to carve out dedicated time for research and improve care for children with kidney disease.

”Children’s National is honored to welcome Dr. Moxey-Mims as the new leader for our talented nephrology team,” says Robin Steinhorn, M.D., senior vice president of the Center for Hospital-Based Specialties. “She brings unparalleled expertise in this field, is a member of a number of influential national committees and has authored more than 90 scientific publications, including peer-reviewed articles and book chapters. Under her guidance, Pediatric Nephrology at Children’s is well-positioned to continue to lead the nation in clinical care and research.”

“I want to inspire the division,” Dr. Moxey-Mims says. “I want the faculty to be happy in their work here and to look forward to coming to work every day. I want them to have enough time to pursue their academic interests, so clinicians not only continue to provide excellent patient care but also can conduct research. All of the staff has potential projects in mind; it’s just a matter of finding the time to do them.”

From a pragmatic standpoint, Children’s pediatric nephrologists will start with what is feasible: Continuing and expanding current cross-disciplinary research projects.

“There are some research projects that will be important to pursue, but we just don’t have the building blocks in place right now to move in that specific direction,” Dr. Moxey-Mims says. “However, continuing ongoing collaborations with our colleagues in neonatologyoncologyhematology and urology are reasonable places to start. I agree with the cliché that success breeds success. If we have an established collaboration and can build on it, that is how we start expanding our research enterprise.”

To that end, the division is in the early stage of joining an existing consortium that is studying four types of glomerular disease, conditions caused by varying mechanisms that often lead to kidney failure. “Information that is gathered will inform care going forward,” she says. “Part of what is being done in these studies is obtaining a better understanding of how disease progresses in different groups of children and adults and quantifying the impact of varying treatment approaches. It’s very exciting for Children’s National to be a new player in this.”

Dr. Moxey-Mims received her undergraduate degree from McGill University in Montreal and her medical degree from Howard University in Washington, D.C. She completed her pediatric residency and clinical pediatric nephrology training at Children’s National and from 1994 to 1999 worked at Children’s National as a staff nephrologist.

“Returning to Children’s has been a wonderful homecoming,” Dr. Moxey-Mims says. “I wanted to return to the hospital setting and have direct exposure to patients. I missed that. In this new role, I can participate in patient care, as well as foster an environment that spurs even more research. It’s really the best of both worlds.”

Happy girl in hospital bed surrounded by doctors

Addressing MB-CLABSI through innovation – and dedication – to pediatric safety

Happy girl in hospital bed surrounded by doctors

With mucosal barrier central line-associated blood stream infections (MB-CLABSI) posing a serious risk to cancer and other immunocompromised patients, Children’s National Health System was intent on finding a way to prevent them. Through a focused initiative, the hospital experienced great success, cutting infection rates by more than half.

This was a daunting proposition. Historically MB-CLABSI has not been viewed as a preventable infection due to the side effects typically associated with bone marrow transplants in this patient population. Sores and mucosal disruption that develops in the oral cavity post-transplant are fairly common and make it exceedingly difficult to keep the mouth clean and clear of bacteria. Without regimented oral hygiene in this type of environment, the mouth can quickly develop bacteria putting the patient at risk of a MB-CLABSI.

“We challenged the notion that we could not prevent MB-CLABSI and set out on a journey to try to prevent these types of infections from occurring,” says vice president and chief quality and safety officer, Rahul Shah, M.D. “With leadership from our nursing teams and the infection control and prevention group working together with the physicians, we were able to approach this issue from a unique perspective.”

In 2013, Children’s National launched a MB-CLABSI prevention program focused around saline rinses to improve oral hygiene. The goal was to keep the mouth cleaner to avoid bacteria from forming and ultimately entering the blood stream.

Children’s National put the plan into action through the following measures:

Provider

  • Simplified ordering of saline rinses to increase accountability and compliance with the practice and make it easier for providers
  • Implemented reminders to order saline rinses during daily rounds
  • Added saline rinses to the Medication Administration Record to drive compliance in administration of the task

Administrative

  • Saline rinses were chosen as an indicator to be displayed on public-facing quality boards throughout the hospital
  • Implemented daily audits of the quality board to track opportunities for improving compliance and reducing omissions and errors
  • Standardized daily medical rounds to include review of the quality boards

Patient/Caregiver

  • Implemented discussion of saline rinses of the mouth for oncology and bone marrow transplant patients during daily rounds
  • Standardized education for caregivers of children with central lines

“Through strategic programs like this, our patients are safer and Children’s National continues to be a national pediatric quality leader,” says Dr. Shah.

Gerard Martin

European workgroup creates recommendations for CCHD pulse oximetry screening

Gerard Martin

Several experts, including Gerard R. Martin, M.D., recently published recommendations for the use and standardization of pulse oximetry screening for critical congenital heart defects in newborns.

The European Pulse Oximetry Screening Workgroup recently published recommendations for the use and standardization of pulse oximetry screening for critical congenital heart defects in newborns. Children’s National Medical Director of Global Services Gerard R. Martin, M.D., was among the experts that compiled the recommendations.

Approximately 1 in 500 babies is born with a critical congenital heart defect (CCHD). Because these conditions can cause serious, life-threatening symptoms, early detection and intervention is essential. Pulse oximetry screening (POS) – a method that measures oxygen saturation – is regarded as a simple, quick and reliable tool for early detection of CCHD, and was recommended for use in screening by the American Academy of Pediatrics and the American Heart Association in 2011.

In Europe, although POS is being used by an increasing number of hospitals, few countries have issued national guidelines recommending universal POS. To remedy this situation, neonatologists, experts in CCHD screening, and representatives from major scientific pediatric societies across Europe came together to create recommendations for the use and standardization of POS for early detection of CCHD across Europe.

Their recommendations, which were published in The Lancet, are as follows:

  • POS for critical congenital heart defects should be recommended for all European countries
  • POS should be done with new-generation equipment that is motion tolerant
  • Screening should occur after 6 hours of life or before discharge from the birthing centre (preferably within 24 hours after birth)
  • Screening should be done in two extremities: the right hand and either foot
  • Each country should consider the advantages and disadvantages of the two available protocols and use that which best suits their population

Children’s National Chief of Allergy and Immunology helps move gene therapy forward

Catherine Bollard

Catherine Bollard, M.D., MBChB, Chief of the Division of Allergy and Immunology, recently shared her expertise on an FDA panel that unanimously expressed its support for a pediatric cancer T-cell therapy called CTL019.

On July 12, 2017, a U.S. Food and Drug Administration advisory committee unanimously expressed its support for CTL019 – a pediatric cancer T-cell therapy. If the FDA follows the advice from the 10-member Oncologic Drug Advisory Committee (ODAC) – which included Children’s National Health System’s Catherine Bollard, M.D., MBChB, Chief of the Division of Allergy and Immunology and Director of the Program for Cell Enhancement and Technologies for Immunotherapy – CTL019 will become the first gene therapy to hit the market.

“Many of these children with recurrent cancer are out of other options to treat their illness,” said Dr. Bollard. “We are encouraged by these findings and the potential for this therapy to improve outcomes and quality of life.”

CTL019 is a chimeric antigen receptor (CAR) T-cell therapy, comprised of genetically modified T cells that target CD19, an antigen expressed on the surface of B cells. Also known as tisagenlecleucel, the therapy targets a single type of cancer called acute lymphoblastic leukemia and was created by Novartis.

In clinical trials, CTL019 showed unparalleled effectiveness. Of the 68 patients who received the drug, 52 responded almost immediately, and their cancer disappeared within the first three months. Seventy-five percent of those patients remained cancer-free six months after treatment. The therapy has one main side effect: an immune reaction called cytokine release syndrome, which can be deadly, with extended spiking fevers and other symptoms.

However, because of CTL019’s high efficacy, FDA scientists asked the ODAC panel to focus on the therapy’s safety and manufacturing challenges rather than whether or not it works.

Several committee members, including Dr. Bollard, expressed apprehension about the T-cell subpopulations’ heterogeneity, which could affect safety and efficacy. Another issue for consideration by the ODAC panel was the long-term side effects of CTL019 and the possibility that the T-cell modification could go awry, causing secondary cancers in the future.

Despite these concerns, the committee concluded that the strong efficacy data and the near-term benefits of CAR-T therapy more than tipped the scales in favor of the therapy. ODAC members were also pleased with Novartis’ plan to minimize risk, which includes limiting CTL019 distribution to selected centers with CAR T-cell therapy experience, and extensive, long-term post-marketing surveillance plans.

The FDA is not required to follow the ODAC panel’s advice when making its final decision, but it often does so. A final decision by the FDA is anticipated by late September.

Read more about the story in the Philadelphia Inquirer, Medpage Today and Healio.com.

Children’s National expert joins a national discussion on Zika and other emerging threats

Roberta DeBiasi

“Our goal is to provide the earliest and most accurate information to women affected by Zika exposure and infection during pregnancy, including capability for fetal MRI,” says Roberta DeBiasi, M.D., M.S., chief of the Division of Pediatric Infectious Diseases and co-director of the Congenital Zika Virus Program at Children’s National Health System.

An expert roundtable discussion, “Facing the Zika Crisis and Other Emerging Threats,” organized in collaboration with Purdue University, the Gallup Organization and the Bipartisan Policy Center, was recently held at the U.S. Capitol. Chief of the Division of Pediatric Infectious Diseases and Co-Director of the Congenital Zika Program at Children’s National Health System, Roberta DeBiasi, M.D.,M.S., weighed in on the discussion, sharing knowledge on the challenges to the United States health system and the continuous research and work that the Children’s National Congenital Zika program does.

Eighteen months ago, Children’s National received its first referral for a Washington, D.C. woman who had a Zika infected pregnancy in January 2016. This case prompted the development of the Congenital Zika program to serve as a consultation resource for affected women and infants, and to perform research to address the knowledge gaps concerning Zika infection during pregnancy: Young researchers tackle Zika’s unanswered questions.

“Our goal is to provide the earliest and most accurate information to women affected by Zika exposure and infection during pregnancy, including capability for fetal MRI,” says Dr. DeBiasi.

Since then, the Zika team has evaluated 65 mother-fetus/infant pairs. Researchers are actively learning the best methods for detection of infection and neurologic injury by continually conducting research and obtaining new and useful information that can be shared with others. The research mission of the Congenital Zika program is now focusing on several areas, which include the study of biomarkers to predict if the infant could be affected by the disease, the utility of a fetal MRI in conjunction with ultrasound, genetic risk factors in mothers and infants that might explain why some infants become infected and some do not, long term neurodevelopment of babies that are infected, and neuropathologic evaluations of brains from fetuses that have died from Zika.

The challenges and concerns that were presented for the United States health system include the willingness and ability to share information, the acceptance of the need for data sharing between institutions and determining if testing resources are adequate and appropriate.

Dr. DeBiasi says, “Institutions have become much better at looking at how to prepare for emerging infectious diseases on a broader level.” Proactively thinking, Dr. DeBiasi finds it useful for health systems to use their own task forces, such as the Ebola Response Task force at Children’s National, as a cohesive existing team that will be prepared for additional infectious disease threats that may arise.

Eric Vilain explores “Disorders/differences of sex development: A world of uncertainty”

Eric Vilain

In his keynote address at Children’s National’s Research and Education Week, Eric Vilain, M.D., Ph.D., explored the genetics of sex development and sex differences.

After announcing he would be joining Children’s National as the new director of the Center for Genetic Medicine Research late last year, internationally-renowned geneticist Eric Vilain, M.D., Ph.D., gave a keynote address entitled “Disorders/Differences of Sex Development: A World of Uncertainty” during Children’s National’s Research and Education Week.

Dr. Vilain explored the genetics of sex development and sex differences – specifically differences of sex development (DSD), congenital conditions in which the development of chromosomal, gonadal or anatomical sex is atypical.

“The first step in sex development is looking at genetic sex and how it results in gonadal sex,” Dr. Vilain said. “From a scientific perspective, we are trying to take a step back and assess how cells become more typically male or female.”

He explained that, at conception, the fundamental difference between male and female embryos exists in the sex chromosome complement. Both XX and XY embryos have bipotential gonads capable of differentiating into a testis or an ovary, though embryos are virtually indistinguishable from a gender perspective up until six weeks in utero.

Eric Vilain - sex differences

According to Dr. Vilain, the fundamental difference between male and female embryos exists in the sex chromosome complement, though embryos are virtually indistinguishable until six weeks in utero.

Whether or not a bipotential gonad forms is largely left up to the genetic makeup of the individual. For example, a gene in the Y chromosome (SRY) triggers a cascade of genes that lead to testis development. If there is no Y chromosome, it triggers a series of pro-female genes that lead to ovarian development.

However, genetic mutations can alter the subsequent steps of sex differentiation. Dr. Vilain explained that, depending on the genotype, an individual may experience normal gonadal development, but abnormal development of the genitalia.

He also noted that these genes are critical to determining the differences between men and women in non-gonadal tissues as well.

In addition to exploring the genetics of sex development and sex differences, Dr. Vilain’s research explores the biological bases of sex variations in predisposition to disease. His clinic at Children’s National is completely devoted to caring for patients with a wide array of genetic and endocrine issues, particularly cases dealing with variations of sex development.

For seven years, Children’s National’s Research and Education Week has celebrated the excellence in research, education, innovation and scholarship at Children’s National and around the world. This year, the annual event focused how “Collaboration Leads to Innovation” and celebrated the development of ideas that aim to transform pediatric care.

Panel: Significant Zika risks linger for pregnant women and developing fetuses in US

Roberta DeBiasi

The threat from Zika “is not over. It is just beginning for the families who are affected by this,” says Roberta L. DeBiasi, M.D., M.S., chief of the Division of Pediatric Infectious Diseases and co-director of the Congenital Zika Virus Program at Children’s National Health System.

The Zika virus epidemic may have fallen off the radar for many media outlets, but significant risks continue to linger for pregnant women and developing fetuses, a panel of experts told staff working for U.S. Congressional leaders.

“The threat of this virus is real, and the threat continues,” Margaret Honein, Ph.D., M.P.H., of the Centers for Disease Control and Prevention’s (CDC) pregnancy and birth defects task force, said during the July 13 briefing held in the Russell Senate Office Building.

Dr. Honein told about 100 attendees that more than 200 Zika-affected babies have been born in the United States suffering from serious birth defects, such as rigid joints, inconsolable distress that causes them to cry continuously and difficulties swallowing. Some of these infants experience seizures that cause further brain damage.

Predicting what Zika will do next in the United States is very difficult, Dr. Honein said, adding that local outbreaks could occur “at any time.” A map she displayed showed Zika’s impact in shades of blue, with Zika infections documented in nearly every state and the highest number of infections – and deepest shade of blue­ – for California, Florida and Texas.

The threat from Zika “is not over. It is just beginning for the families who are affected by this,” agreed Roberta L. DeBiasi, M.D., M.S., chief of the Division of Pediatric Infectious Diseases and co-director of the Congenital Zika Virus Program at Children’s National Health System.

Since Children’s National launched its Zika program in May 2016, the multidisciplinary team has consulted on 65 mother-fetus/infant pairs, Dr. DeBiasi said. Because in utero Zika infection can result in a wide range of side effects, the Children’s team includes pediatric infectious diseases experts, fetal/neonatal neurologists to consult on seizures, audiologists to assess hearing, physical therapists and orthopaedists to contend with limb contractures, pulmonologists to relieve breathing problems and ophthalmologists to diagnose and treat vision disorders – among other specialists.

“You really need a program that has all of these areas of expertise available for a family,” Dr. DeBiasi told attendees. “It is not possible for a family to organize 27 different appointments if you have a child with these needs.”

Children’s Zika experts also collaborate with researchers in Colombia to gauge the ability of magnetic resonance imaging to produce earlier Zika diagnoses, to assess the role of viral load as biomarkers and to document Zika’s long-term impact on children’s neurodevelopment. The Colombia study has enrolled an additional 85 women/infant pairs.

In one presentation slide, Dr. DeBiasi showed sharp magnetic resonance imaging scans from their research study of a fetal brain at 18 and 22 weeks gestation that indicated clear abnormalities, including abnormal cortical folding. Ultrasound images taken at the exact same time points did not detect these abnormalities, she said.

Asked for advice by an attendee whose clinic treats women who regularly travel between California and Mexico, Dr. DeBiasi underscored the fact that Zika infection poses a risk to developing fetuses even if the pregnant woman has no symptoms of infection. “Whether or not they’re symptomatic, the risk is the same. It’s hard for people to understand that. That is No. 1,” she said.

Another challenge is for women who scrupulously follow the CDC’s guidance on lowering their infection risk while traveling. Upon return, those women may be unaware that they could still be exposed to Zika through unprotected sex with their partner who also has travelled, for as long as six months after travel.

US News Honor Roll 2017-18

Children’s National is #1 in Neonatology and Top 10 overall in U.S. News & World Report Survey

US News Honor Roll 2017-18Children’s National is proud to be named #1 in Neonatology in the U.S. News & World Report 2017-18 Best Children’s Hospitals survey. Also, Children’s National was once again named to the coveted Honor Roll, placing them among the Top 10 children’s hospitals in the country.

Being the #1 ranked Neonatology program reflects the quality of care throughout Children’s National because it requires the support and partnership of many other specialties, including cardiology, neurology and surgery. In addition to this honor, Children’s National ranked in the Top 10 in four additional services: Cancer (#7), Neurology and Neurosurgery (#9), Orthopedics (#9) and Nephrology (#10).  For the seventh year in a row, Children’s National has ranked in all ten services, a testament to the pediatric care experts across the organization and their commitment to children and families.

“This recognition is a great achievement for Children’s National, affirming our place as a premier destination for pediatric care, and the commitment of our people, partners and supporters to helping every child grow up stronger,” said Kurt Newman, M.D., President and CEO of Children’s National. “I’m particularly proud of our #1 ranking in Neonatology as, in many ways it reflects the quality of care across our hospital. Treating these tiny patients often encompasses many other specialties, including our Fetal Medicine Institute.”

Children’s National is dedicated to improving the lives of children through innovative research, expert care and advocacy on behalf of children’s needs. In addition to being recognized among the “best of the best” by U.S. News & World Report, Children’s National is a Magnet® designated hospital for excellence in nursing and is a Leapfrog Group Top Hospital. As a top NIH-funded pediatric health system, Children’s National marries cutting-edge research with the highest quality care, to deliver the best possible outcomes for children today and in the future.

Children’s National Heart Institute experts partner with FDA and nation’s leading cardiology organizations to advance pediatric drug development

New joint health policy statement offers roadmap for immediate changes in clinical trial design to save children’s lives

Families with children suffering from rare and difficult-to-treat cardiovascular diseases may soon have better access to drugs to treat their often life-threatening conditions. For the first time, experts from the U.S. Food and Drug Administration (FDA), the American College of Cardiology, the American Heart Association and the American Academy of Pediatrics are working together to describe the challenges and opportunities to improve pediatric drug research as shared in a joint statement published online June 29 in the Journal of the American College of Cardiology and Circulation: Cardiovascular Quality and Outcomes.

“Children should have access to the latest advances in treatment and the best care. By challenging the status quo and designing new, safe and effective alternative study designs, we can give them the best opportunity to grow up stronger,” notes David Wessel, M.D., executive vice president and chief medical officer, Hospital and Specialty Services at Children’s National Health System. Dr. Wessel is internationally recognized for his pioneering work in caring for children with heart disease. As the senior author of the new joint statement and principal investigator of the STARTS-1 trial, which is the catalyst for this collaboration, he says he is “optimistic about this forward progress.”

According to the statement, less than 50 percent of drugs approved for use in the United States have sufficient data to support labeling for dosing, safety and efficacy in children. Additionally, a 2008 report by Pasquali et al, which reviewed more than 30,000 records of hospitalized children with cardiovascular disease, found that 78 percent received at least one off-label medication and 31 percent received more than three.

There are numerous challenges in the development and approval of medications for children – especially those with rare diseases – but the paper’s lead author, Craig Sable, M.D., associate division chief of cardiology at Children’s National, says we can and need to do better.

“While randomized clinical drug trials remain the gold standard in advancing care for adults with cardiovascular disease, relying solely on these types of trials for children unnecessarily limits the drugs approved for use in children,” says Dr. Sable. “Through this unique collaboration that unifies the voice of leaders in pediatric cardiology and the FDA, our goal is to provide a framework to better define which drugs are needed and how we can create novel study designs to overcome the current trial barriers.”

To read more about the barriers and ideas presented, please find the full statement here.

Children’s welcomes hematology leaders, expands expertise

The Center for Cancer and Blood Disorders at Children’s National is emerging as a leader in Pediatric Hematology, and the recruitment of two prominent physician-scientists to our Division of Hematology and Sickle Cell Disease Program is evidence of that growth and presence on the national platform. Joining the faculty in June are:

Suvankar (Seve) Majumdar, M.D., Suvankar (Seve) Majumdar, M.D.
Division Chief, Hematology
Dr. Majumdar was born in Zambia, attended the University of Zimbabwe College of Health Sciences and conducted his postdoctoral medical education at the University of Mississippi. Dr. Majumdar is currently the director of the Comprehensive Pediatric Sickle Cell Program at the University of Mississippi Medical Center. He previously directed the Mississippi Hemophilia Treatment Center and is a recognized leader in hematology and sickle cell disease. In addition to his broad clinical expertise, Dr. Majumdar is an accomplished researcher, and a principal investigator of NIH-funded studies.

Andrew Campbell, M.D.Andrew (Drew) Campbell, M.D.
Director, Sickle Cell Disease Program
Dr. Campbell’s distinguished training and career path began at Morehouse College. He continued medical school at Case Western Reserve University and completed post graduate training at Massachusetts General Hospital (Harvard) and Lurie Children’s Hospital (Northwestern University). He has been director of the Comprehensive Sickle Cell Center at the University of Michigan since 2005. His research interests span several topics in sickle cell disease including pulmonary complications, fetal hemoglobin switching in transgenic sickle cell mice, phenotype/genotype relationships and renal complications.

The Children’s National Division of Hematology includes the most comprehensive pediatric blood disorders team in the Washington, D.C., area. The Sickle Cell Disease Program is among the largest in the country, treating more than 1,400 children and young adults with all types of sickle cell disease.

Children’s National Diabetes Program Honored at SAMHSA’s National Children’s Mental Health Awareness Day

Maureen Monaghan and Fran Cogen at SAMHSA

The Substance Abuse and Mental Health Services Administration (SAMHSA) spotlighted the Children’s National diabetes program as an exemplar of integrated care for children and adolescents. Maureen Monaghan, Ph.D., CDE, (left) and Fran Cogen, M.D., CDE, interim co-chief of the Division of Endocrinology and Diabetes and director of the Childhood and Adolescent Diabetes Program, were in attendance.

On May 4, Maureen Monaghan, Ph.D., CDE, clinical and pediatric psychologist and certified diabetes educator in the Childhood and Adolescent Diabetes Program at Children’s National, participated in a panel emphasizing the importance of integrating physical and mental health in the care of young patients as part of the Substance Abuse and Mental Health Services Administration’s (SAMHSA) National Children’s Mental Health Awareness Day. SAMHSA also spotlighted the Children’s National diabetes program as an exemplar of integrated care for children and adolescents.

“Many of our families start out knowing nothing about the disease, and they now have a child whose care requires day-to-day management for the rest of their lives,” says Dr. Monaghan. “It’s not a disease you ever get a break from – which can take both a physical and emotional toll on children and their families.”

Maureen Monaghan at SAMHSA National Children’s Mental Health Awareness Day

Dr. Monaghan participated in a panel emphasizing the importance of integrating physical and mental health in the care of young patients with diabetes.

To combat this issue and reduce barriers and stigma related to seeking mental health care, the program brings a dedicated, multidisciplinary care team together in one convenient location.

From the initial diagnosis, patients have access to care from a comprehensive team, including six physicians, three nurse practitioners, eight nurse educators, three psychologists, a physical therapist, dietitian and social worker. Each expert counsels the patient and the family, helping them navigate all aspects of living with the disease – from overcoming stress and anxiety to offering healthy meal-planning guides and exercise routines.

“We aren’t just concerned about how they are doing medically or what emotions they are experiencing,” says Dr. Monaghan. “Instead, our team’s integration allows us to focus on the whole child and his or her total quality of life, which is so important for patients and families with chronic disease.”

To learn more, watch this short video, featuring employees and patients of the Children’s National Childhood and Adolescent Diabetes Program, which was presented during the events surrounding the SAMHSA National Children’s Mental Health Awareness Day.

Lenore Jarvis

Firearm-related injuries are leading causes of child and adolescent deaths and are preventable

Lenore Jarvis

Lenore Jarvis, M.D., M.Ed., F.A.A.P. addressed Congressional staff on behalf of the American Academy of Pediatrics in an effort to reduce gun injuries and deaths in the home by encouraging parents to ask about safe firearm storage.

“I see firsthand in my emergency department practice children getting shot: Unintentional, accidental injuries and shootings, homicides, suicides. And it’s terrible. If I never had to treat another child … for a gun-related injury, I would be so happy,” Lenore Jarvis, M.D., M.Ed., F.A.A.P., Pediatric Emergency Medicine Attending at Children’s National Health System, told Congressional staff. “I will never forget … a 5-year-old shot and killed by a family member who mistook him for a home intruder.”

Dr. Jarvis’ comments on behalf of the American Academy of Pediatrics came during briefings for Congressional staff working in the U.S. Senate and U.S. House of Representatives just days after a gunman targeted Republicans practicing for a Congressional baseball game wounding five, including House Majority Whip Steve Scalise.

The June 16, 2017 Congressional briefings were intended to draw attention to National ASK Day, an annual effort to reduce gun injuries and deaths in the home by encouraging parents to ask about safe firearm storage.

“It’s pretty simple,” Dr. Jarvis told attendees. “I think that there is a mutual recognition about what a public health problem this is: Firearms … are leading causes of deaths in children and adolescents through homicide and suicide by firearms. And they are preventable.”

Her comments echoed a recent firearms review paper published May 23, 2017, by a Children’s National research team that found firearms are present in 18 percent to 64 percent of U.S. homes, and 20,000 U.S. children are transported to Emergency Departments each year for firearm-related injuries. According to the study authors, pediatricians can play a pivotal role in helping to reduce gun violence by encouraging safe storage of firearms in the home and supporting research into firearm-related injury prevention.

In addition to Dr. Jarvis, speakers during the Congressional briefings included a Johns Hopkins Hospital surgeon who decided to pursue a medical career after surviving a gunshot wound to the throat while he was a teenager, a Missouri state representative who co-founded the Children’s Firearm Safety Alliance and a Brady Campaign strategist.

Children’s National earns five awards at the 2017 SPR Annual Meeting

Radiology PULSE Suite

Several technologists, fellows and faculty in the Division of Diagnostic Imaging and Radiology at Children’s National Health System were recognized at the 2017 Society of Pediatric Radiology (SPR) Annual Meeting in Vancouver, Canada, May 16-20. Each year, the international conference recognizes society members for outstanding research and education in pediatric care on the topics of imaging and image-guided care. Out of 15 major awards, Children’s National staff earned five, including two Caffey Awards – SPR’s most prestigious awards for academic excellence.

The awards received are as follows:

The Society of Pediatric Radiology Caffey Award for Best Clinical Research Paper went to attending radiologist, Dorothy Bulas, M.D., for her clinical research paper titled, “CXR Reduction Protocol in the Neonatal Intensive Care Unit (NICU) – Lessons Learned,” which highlighted collaboration with the NICU team to reduce the reliance on x-rays to monitor neonates. This method decreases the radiation dose with no risk to the patient.

The Society of Pediatric Radiology Caffey Award for Educational Exhibit was given to Benjamin Smith, M.D., a pediatric radiology fellow, for his educational poster “Sonographic Evaluation of Diaphragmatic Motion: A Practical Guide to Performance and Interpretation.” The exhibit displayed a unique technique for examining the motion of the diaphragm using ultrasound to make an accurate diagnosis of diaphragm paralysis or motion. Dr. Smith’s exhibit was also recognized by The American Academy of Pediatrics and was given the Outstanding Clinical Education Poster Award along with radiologist Hansel Otero, M.D.; sonographer Tara K. Cielma, R.D.M.S, R.D.C.S, R.V.T.; and faculty member Anjum N. Bandarkar, M.D.

The Society of Pediatric Radiology Radiographer Best Poster Award was given to Dr. Bandarkar for her poster titled, “Infantile Hypertrophic Pyloric Stenosis: Value of measurement technique to avoid equivocal exam.” The World Federation of Pediatric Imaging also awarded Dr. Bandarkar, Adebunmi O. Adeyiga, M.D. and Tara Cielma the 2017 Outstanding Radiographer Educational Poster Award for their collaborative poster on, “A Sonographic Walk‐Through: Infantile Hypertrophic Pyloric Stenosis.”

Division Chief of Diagnostic Imaging and Radiology, Raymond Sze, M.D., remarked, “This is a major win not only for the department but also for the entire hospital. The support and collaboration of our Children’s National colleagues across many departments allowed us to advance the field of pediatric imaging and earn national recognition for the high-quality and impactful research and education that’s happening at our institution.”

STAR Team

STAR robot is finalist in NASA iTech challenge

STAR Team

Children’s National Health System’s proprietary robotic surgical technology Smart Tissue Autonomous Robot (STAR) has been named one of the top ten finalists in the 2017 NASA iTech call for ideas challenge.

The Sheikh Zayed Institute for Pediatric Surgical Innovation’s intelligent Smart Tissue Autonomous Robot (STAR) has been named one of the top ten finalists in the 2017 NASA iTech challenge.

The team will present the project at the NASA iTech Forum on July 12-13, 2017 at the National Institute of Aerospace in Hampton, VA, where leaders from NASA and prospective stakeholders will evaluate the 10 finalists and select three top solutions.

“We’re honored to be selected as a finalist in this prestigious challenge,” said Peter C. Kim, M.D., vice president and associate surgeon in chief at Sheikh Zayed Institute at Children’s National. “Our technology is capable of many solutions that would be useful as part of NASAs deep space exploration, including intelligent pods capable of common intelligent autonomous surgical procedures.”

A cutting-edge system, STAR was the first to perform a successful autonomous robotic soft tissue surgery on a live subject in May 2016 and is licensed to Omniboros.

Promoting diversity and inclusion in pediatric academic medicine

Mary Ottolini

Mary Ottolini, M.D., M.P.H., ME.d., highlighted the Academic Pediatric Association’s efforts to promote more diversity and inclusion within pediatric academic medicine.

Data from the Association of American Medical Colleges on faculty promotion show a very low percentage of diverse assistant professors being promoted to associate professors, and a low percentage of diverse associate professors rising through the ranks to become full professors within academic medicine. Mary Ottolini, M.D., M.P.H., ME.d., vice chair for Medical Education at Children’s National Health System, professor of pediatrics at George Washington University School of Medicine and president of the Academic Pediatric Association (APA) addressed this problem at the recent Pediatric Academic Societies annual meeting. In her presentation, “APA approach to diversity-inclusion,” Dr. Ottolini explained various APA initiatives in place to assist underrepresented minority (URM) residents, fellows and junior faculty to advance academically in pediatric medicine.

The APA’s core value and strategic goal for diversity and inclusion is to increase diversity and engagement of its membership. To execute this initiative, New Century Scholars was created in 2004 as a national mentorship program to increase racial and ethnic diversity of academic pediatric medicine. The two-year program collaborates with the American Pediatric Society and utilizes junior and senior mentors to provide support to our URM residents with a special interest in health disparities, social determinants of health, cultural competency and minority child health and development.

Dr. Ottolini believes, “it’s important for our URM faculty to have early, strong mentorship that provides an idea of what it takes to be academically successful, by networking and collaborating with others.” She went on to say, “By forming these collaborations, they can transform an idea into a project that will be published, which strengthens their ability to achieve promotions.”

Research Academic Pediatrics Initiative on Diversity (RAPID), is another APA national program working to recruit, retain, and provide career development for diverse junior faculty in general academic pediatrics that are pursuing careers in the National Institute of Diabetes and Digestive and Kidney Diseases mission areas. RAPID targets applicants who are members of an underrepresented minority group and are disabled or from a socially, culturally, economically or educationally disadvantaged background.

“Diversity and inclusion is an issue that is important for patients and the field of academic pediatrics because we need to have a physician workforce that resembles the patient population that we are entrusted to take care of,” Dr. Ottolini says.

Dr. Ottolini also explained APA’s current special interest groups were put in place to bring awareness to the role of race in the practice of medicine, and to provide resources by which members can support the healthy development and optimal care for U.S. youth of color. The ultimate goal is to develop strategies for increasing diversity and retention among academic medical faculty across the United States, and develop best practices for caring for youth of color in the primary care setting.

The presentation concluded with a question-and-answer session and further discussion from the audience. Since her presentation, Dr. Ottolini has received offers from other doctors and national organizations to fund these initiatives.