In Brief Archives

Treatment of neuroblastoma with immunotherapy and vaccine combination shows promise

March 2, 2018

“Treatment options like these that help the body use its own immune system to fight off cancer are incredibly promising, and we look forward to continuing this work to understand how we can best help our patients and their families,” said Anthony Sandler, M.D.

Despite being the most common extracranial solid tumor found in children and having multiple modes of therapy, neuroblastoma continues to carry a poor prognosis. However, a recent cutting-edge pre-clinical study, PD-L1 checkpoint inhibition and anti-CTLA-4 whole tumor cell vaccination counter adaptive immune resistance: A mouse neuroblastoma model that mimics human disease, published in PLOS Medicine shows the first signs of success in treating high-risk neuroblastoma, a promising step not only for neuroblastoma patients, but potentially for other types of cancer and solid tumors as well. While the research was conducted on mouse models and is in the early stages, the lead author of the study, Anthony Sandler, M.D., senior vice president and surgeon-in-chief of the Joseph E. Robert, Jr., Center for Surgical Care at Children’s National, believes these findings are an encouraging development for the field.

The treatment method combines a novel personalized vaccine and a combination of drugs that target checkpoint inhibitors enabling the immune system to identify and kill cancer cells. When these checkpoints are blocked, it’s similar to taking the brakes off the immune system so that the body’s T cells can be primed by the vaccine, identify the tumor and allow for targeted tumor cell killing. The vaccine then brings in reinforcements to double down on the attack, helping to eradicate the tumor. The vaccine could also be used as a way to prevent recurrence of disease. After a patient has received the vaccine, the T cells would live in the body, remembering the tumor cells, and attack reemerging cancer in a similar way that a flu vaccine helps fight off the flu virus.

“Treatment options like these that help the body use its own immune system to fight off cancer are incredibly promising, and we look forward to continuing this work to understand how we can best help our patients and their families,” said Dr. Sandler.

New guidelines advance treatment approach for children with low-grade gliomas

March 1, 2018

“We believe our understanding of LGGs combined with novel therapies will soon lead to a new standard of care for children,” says Roger J. Packer, M.D. “We are optimistic about the future for patients with this disease.”

Patients with low-grade gliomas (LGGs) will benefit from new recommendations from a group led by Roger J. Packer, M.D., senior vice president for the Center for Neuroscience and Behavioral Medicine, as well as clinicians, researchers and industry leaders from around the world, that were recently published in Neuro-Oncology. The new framework for LGGs will significantly advance the future of care for patients with these complex diseases and set a new path to expedite the translation of scientific advances into clinical care. The recommendations build on a treatment approach developed more than 25 years ago by Dr. Packer and his colleagues that revolutionized care for LGGs.

LGGs are both common and complicated, and one treatment approach does not work for all cases. Until now, there has not been a standardized way to categories the tumors to prescribe more effective and personalized treatment options. The new guidelines will provide clinicians with one mutually agreed upon set of recommendations to further advance the field and better diagnose and treat patients with LGGs.

Topics within the framework include:

Implications of the growing understanding of genomics underlying these tumors and how to apply to clinical practice
The need for more and better model systems to assess the likely benefits of new treatments for LGGs before exposing patients to new therapy
A review and assessment of what is needed for the design of future clinical trials
Evaluation of current therapies and the steps needed to expedite molecularly targeted therapy into late-stage clinical trials, including in those newly diagnosed with the disease so as to avoid less-personalized chemotherapy or radiotherapy

“We believe our understanding of LGGs combined with novel therapies will soon lead to a new standard of care for children,” says Dr. Packer. “We are optimistic about the future for patients with this disease.”

Ricardo Muñoz, M.D., joins Children’s National as Chief of Cardiac Critical Care Medicine, Executive Director of Telemedicine and Co-Director of Heart Institute

February 28, 2018

Children’s National Health System is pleased to announce Ricardo Muñoz, M.D., as chief of the Division of Cardiac Critical Care Medicine and co-director of the Children’s National Heart Institute. Dr. Muñoz also will serve as the executive director of Telemedicine Services at Children’s National, working to leverage advances in technology to improve access to health care for underserved communities and developing nations.

Within the new division of Cardiac Critical Care Medicine, Dr. Muñoz will oversee the work of a multidisciplinary team, including critical care nurse practitioners and nurses, respiratory and physical therapists, nutritionists, social workers and pharmacists, in addition to a medical staff with one of the highest rates of double-boarded specialists in cardiology and critical care.

“We are honored to welcome Dr. Ricardo Muñoz to Children’s National,” says David Wessel, M.D., executive vice president and chief medical officer of Hospital and Specialty Services. “He is a pioneer and innovator in the fields of cardiac critical care and telemedicine and will undoubtedly provide a huge benefit to our patients and their families along with our cardiac critical care and telemedicine teams.”

Dr. Muñoz comes to Children’s National from Children’s Hospital of Pittsburgh of UPMC. During his 15-year tenure there, he established the cardiac intensive care unit and co-led the Heart Center in a multidisciplinary effort to achieve some of the best outcomes in the nation. He also is credited with pioneering telemedicine for pediatric critical care, providing nearly 4,000 consultations globally.

“Children’s National has a longstanding reputation of excellence in cardiac critical care, and I am pleased to be able to join the team in our nation’s capital to not only deliver top-quality care to patients regionally, but also around the world,” says Dr. Muñoz. “The early identification and treatment of pediatric congenital heart disease patients has made rapid improvements in recent decades, but there is a shortage of intensivists to care for these children during what is often a complex recovery course.”

Dr. Muñoz attended medical school at the Universidad del Norte, Barranquilla, Colombia, and completed his residency in pediatrics at the Hospital Militar Central, Bógota, Colombia. He continued his training as a general pediatrics and pediatric critical care fellow at Massachusetts General Hospital, and as a pediatric cardiology fellow at Boston Children’s Hospital. He then joined the faculty at Harvard Medical School and served as an attending physician in the Cardiac Intensive Care Unit at Boston Children’s.

Dr. Muñoz is board certified in pediatrics, pediatric critical care and in pediatric cardiology. He is a fellow of the American Academy of Pediatrics, the American College of Critical Care Medicine and the American College of Cardiology. Additionally, he is the primary editor and co-author of multiple textbooks and award-winning handbooks in pediatric cardiac intensive care, including Spanish language editions.

Children’s National orthopaedic surgery experts prepare for the 2018 POSNA annual meeting

February 20, 2018

The Pediatric Orthopaedic Society of North America (POSNA) will hold its 2018 annual meeting May 9-12, 2018 in Austin, TX. POSNA is dedicated to improving the care of children with musculoskeletal disorders through education, research and advocacy. Along with 1,400 othopeadic surgeons, physicians and other health care professionals, experts from Children’s National will attend and participate in the following activities:

Matthew Oetgen, M.D., M.B.A., Division Chief of Orthopaedic Surgery and Sports Medicine, along with hospitalists Rita Fleming, M.D., and Anjna Melwani, M.D., will give a presentation on quality, safety and value titled, “Hospitalist co-management of pediatric orthopedic patients improves outcomes and quality processes.”
Danielle Putur, M.D., Miguel Pelton, M.D., Niharika Patel, M.P.H., and Emily Niu, M.D., will present a poster titled, “ACL growth with age in the skeletally immature: an MRI study.”
Benjamin Martin, M.D., will present a poster titled, “The effectiveness of intrathecal morphine compared to oral methadone for postoperative pain control after posterior spinal fusion for adolescent idiopathic scoliosis.”

Benjamin Martin, M.D., and Anjna Melwani, M.D., are among the experts from Children’s National who will be presenting at the POSNA annual meeting.

As a newly elected POSNA board member, Dr. Oetgen will also preside over the clinical award session, as well as chair the Spine Subspecialty Day, which is designed to update surgeons on current, cutting-edge topics and provide tips and tricks on a range of issues related to adolescent idiopathic scoliosis and moderate a discussion at this year’s meeting.

Additionally, Benjamin Martin, M.D., recently won the 2017 POSNA Clinical Trials Planning Grant – “The Treatment of Pediatric Diaphyseal Femur Fractures: A Clinical Trials Planning Grant.”

Visit the POSNA website to find out more information on this year’s conference.

New network will advance treatments for children

February 12, 2018

Three leaders from Children’s National Health System are among the investigators of a new FDA-funded program created to launch a global clinical trials network. The initial $1 million grant from the Food and Drug Administration (FDA) establishes a network among the Institute for Advanced Clinical Trials for Children (I-ACT for Children), the National Capital Consortium for Pediatric Device Innovation (NCC-PDI) (affiliated with Children’s National), PEDSnet, the James M. Anderson Center for Health Systems Excellence and the Critical Path Institute, to address the unmet medical needs of children by improving quality and efficiency in developing innovative pediatric drugs and devices.

Along with the fiscal 2017 funds, there is a potential for $1 million in funding each year for an additional four years to I-ACT for Children, contingent on annual appropriations and the availability of funding. I-ACT for Children is a new independent, nonprofit organization that works to improve the planning and completion of pediatric clinical trials. PEDSnet and the Anderson Center will serve as the network’s data and learning core, while the Critical Path Institute will serve as the regulatory science core and NCC-PDI will serve as the medical device core.

From Children’s National, the investigators include: Peter Kim, M.D., Ph.D., vice president of the Sheikh Zayed Institute for Pediatric Surgical Innovation; Kolaleh Eskandanian, Ph.D., executive director of the Sheikh Zayed Institute and NCC-PDI and Johannes van den Anker, M.D., Ph.D., division chief of Clinical Pharmacology and vice chair of Experimental Therapeutics.

“We are pleased that this grant addresses innovative reengineering of the pediatric device trials system,” says Eskandanian. “In contrast with drug trials, device trials are generally less optimally understood in academic medical centers and clinical sites.”

She explains that children have medical device needs that are considerably different from adults. Designing devices for children requires considerations such as growth and development, anatomical and physiological differences. Often, the lack of available devices for children forces clinicians to use an adult device off-label or to improvise. Off-label use may be the only option, but such use can bring risks of serious adverse events that could be avoided if there were more FDA–approved pediatric devices.

“Thanks to partnership with I-ACT we will be able to address the pressing need to improve clinical trials and post-market monitoring of pediatric devices,” says Eskandanian.

Leading the network as principal investigator is Edward Connor, M.D., president of I-ACT for Children and an emeritus professor of Pediatrics, Microbiology, Immunology, and Tropical Medicine at George Washington University School of Medicine and Children’s National.

Work has been initiated to integrate network components and engage public and private shareholders. Next steps include selecting priority projects for implementation in 2018 and beyond, and scaling the network in North America and abroad.

Funding for this work was made possible, in part, by the Food and Drug Administration through grant 1 U18 FD 006297. Views expressed in written materials or publications and by speakers and moderators do not necessarily reflect the official policies of the Department of Health and Human Services; nor does any mention of trade names, commercial practices, or organization imply endorsement by the United States Government.

Children’s National submissions make hackathon finals

February 8, 2018

This April, the Clinical and Translational Science Institute at Children’s National (CTSI-CN) and The George Washington University (GW) will hold their 2nd Annual Medical and Health App Development Workshop. Of the 10 application (app) ideas selected for further development at the hackathon workshop, five were submitted by clinicians and researchers from Children’s National.

The purpose of the half-day hackathon is to develop the requirements and prototype user interface for 10 medical software applications that were selected from ideas submitted late in 2017. While idea submissions were not restricted, the sponsors suggested that they lead to useful medical software applications.

The following five app ideas from Children’s National were selected for the workshop:

A patient/parent decision tool that could use a series of questions to determine if the patient should go to the Emergency Department or to their primary care provider; submitted by Sephora Morrison, M.D., and Ankoor Shah, M.D., M.P.H.
The Online Treatment Recovery Assistance for Concussion in Kids (OnTRACK) smartphone application could guide children/adolescents and their families in the treatment of their concussion in concert with their health care provider; submitted by Gerard Gioia, Ph.D.
A genetic counseling app that would provide a reputable, easily accessible bank of counseling videos for a variety of topics, from genetic testing to rare disorders; submitted by Debra Regier, M.D.
An app that would allow the Children’s National Childhood and Adolescent Diabetes Program team to communicate securely and efficiently with diabetes patients; submitted by Cynthia Medford, R.N., and Kannan Kasturi, M.D.
An app that would provide specific evidence-based guidance for medical providers considering PrEP (pre-exposure prophylaxis) for HIV prevention; submitted by Kyzwana Caves, M.D.

Kevin Cleary, Ph.D., technical director of the Bioengineering Initiative at Children’s National Health System, and Sean Cleary, Ph.D., M.P.H., associate professor in epidemiology and biostatistics at GW, created the hackathon to provide an interactive learning experience for people interested in developing medical and health software applications.

The workshop, which will be held on April 13, 2018, will start with short talks from experts on human factors engineering and the regulatory environment for medical and health apps. Attendees will then divide into small groups to brainstorm requirements and user interfaces for the 10 app ideas. After each group presents their concepts to all the participants, the judges will pick the winning app/group. The idea originator will receive up to $10,000 of voucher funding for their prototype development.

Children’s receives NIH grant to study use of stem cells in healing CHD brain damage

February 2, 2018

“Bone marrow stem cells are used widely for stroke patients, for heart attack patients and for those with developmental diseases,” explains Nobuyuki Ishibashi, M.D. “But they’ve never been used to treat the brains of infants with congenital heart disease. That’s why we are trying to understand how well this system might work for our patient population.”

The National Institutes of Health (NIH) awarded researchers at Children’s National Health System $2.6 million to expand their studies into whether human stem cells could someday treat and even reverse neurological damage in infants born with congenital heart disease (CHD).

Researchers estimate that 1.3 million infants are born each year with CHD, making it the most common major birth defect. Over the past 30 years, advances in medical technology and surgical practices have dramatically decreased the percentage of infants who die from CHD – from a staggering rate of nearly 100 percent just a few decades ago to the current mortality rate of less than 10 percent.

The increased survival rate comes with new challenges: Children with complex CHD are increasingly diagnosed with significant neurodevelopmental delay or impairment. Clinical studies demonstrate that CHD can reduce oxygen delivery to the brain, a condition known as hypoxia, which can severely impair brain development in fetuses and newborns whose brains are developing rapidly.

Nobuyuki Ishibashi, M.D., the study’s lead investigator with the Center for Neuroscience Research and director of the Cardiac Surgery Research Laboratory at Children’s National, proposes transfusing human stem cells in experimental models through the cardio-pulmonary bypass machine used during cardiac surgery.

“These cells can then identify the injury sites,” says Dr. Ishibashi. “Once these cells arrive at the injury site, they communicate with endogenous tissues, taking on the abilities of the damaged neurons or glia cells they are replacing.”

“Bone marrow stem cells are used widely for stroke patients, for heart attack patients and for those with developmental diseases,” adds Dr. Ishibashi. “But they’ve never been used to treat the brains of infants with congenital heart disease. That’s why we are trying to understand how well this system might work for our patient population.”

Dr. Ishibashi says the research team will focus on three areas during their four-year study – whether the stem cells:

Reduce neurological inflammation,
Reverse or halt injury to the brain’s white matter and
Help promote neurogenesis in the subventricular zone, the largest niche in the brain for creating the neural stem/progenitor cells leading to cortical growth in the developing brain.

At the conclusion of the research study, Dr. Ishibashi says the hope is to develop robust data so that someday an effective treatment will be available and lasting neurological damage in infants with congenital heart disease will become a thing of the past.

ER attending clinician named Presidential Leadership Scholar

February 1, 2018

Children’s Pediatric Emergency Medicine Attending Lenore Jarvis M.D., M.Ed., FAAP, has been accepted to the fourth annual class of 2018 Presidential Leadership Scholars (PLS). PLS serves as a catalyst for a diverse network of leaders brought together to collaborate and make a difference in the world as they learn about leadership through the lens of the presidential experiences of George W. Bush, Bill Clinton, George H.W. Bush and Lyndon B. Johnson.

The incoming scholars were selected after a rigorous application and review process. Scholars were selected based on their leadership growth potential and the strength of their personal leadership projects aimed at improving the civic or social good by addressing a problem or need in a community, profession or organization.

Scholars will travel to each participating presidential center to learn from former presidents, key former administration officials and leading academics. They will study and put into practice varying approaches to leadership, develop a network of peers and exchange ideas with mentors and others who can help them make an impact in their communities. The program kicks off in Washington on Feb. 6, 2018.

“I am deeply honored to have been selected for this prestigious program,” Dr. Jarvis says. “I look forward to continuing to work collaboratively with social workers and community stakeholders to provide interventions to mothers who screen positive for postpartum depression more expeditiously. We know from our research in the pediatric emergency department that postpartum depression is reported by about one in four mothers. Providing real-time interventions can help improve the quality of care we provide new mothers and their infants.”

Improved procedures, reduced harm: Moving the needle on spinal fusion

January 29, 2018

In many cases of pediatric scoliosis, a surgical posterior spinal fusion – a life-changing yet complicated process – is needed to straighten the spine.

As part of its ongoing transition to value-based care, Children’s National is constantly reevaluating systems and processes across specialties and proactively seeking ways to deliver the highest quality care. This includes treatments for everything from the rarest of diseases to more frequent conditions, such as pediatric scoliosis.

In many cases of pediatric scoliosis, a surgical posterior spinal fusion – a life-changing yet complicated process – is needed to straighten the spine. The procedure involves permanently fusing bones over the curved part of the spine and requires expert coordination among physicians, nurses and therapists. To improve the procedure and make it as safe and efficient as possible, experts at Children’s National developed a first-of-its-kind pediatric spinal fusion surgical home, an innovative, family-centered approach that is making a real impact.

Prior to this initiative, patients who underwent posterior spinal fusion to treat scoliosis spent multiple days across multiple units in the hospital. Thanks to a comprehensive care pathway with input from all care providers treating these patients, overall recovery time has been reduced as well as days in the hospital. This in turn decreased the costs to both the families and Children’s National.

In the first six months of implementation, changes included decreasing the average length of stay from approximately five days to three and a half days, decreased blood transfusion rate and less use of opioid pain medications. Each of these pieces directly contributes to the safety of a child and decreased costs across the board. Ultimately, implementing cutting-edge practices like these brings the organization closer to zero harm and helps move the needle on patient care across the industry.

Children’s National to host 28th Annual Pediatric Neurology Update

January 19, 2018

The Children’s National Health System Center for Neuroscience and Behavioral Medicine is proud to host the 28th Annual Pediatric Neurology Update course.

This year’s course will be focused on new understandings, molecular pathogenesis, novel treatment and outcomes of infections which affect the central nervous system; as well as the often indistinct boundaries between CNS infections and neuro immunologic diseases of the nervous system.

We invite you to join us for presentations from renowned experts in the field in this full-day, CME accredited event on May 3, 2018 at the Children’s National main campus in Washington, D.C.

For more information and to register, visit ChildrensNational.org/NeurologyUpdate.

Q&A with leading blood and marrow transplantation specialist

January 19, 2018

Children’s National Health System is proud to be the home of some of the world’s leading hematology experts, including Allistair Abraham, M.D., blood and marrow transplantation specialist within the Center for Cancer and Blood Disorders, who was recently selected to participate in the American Society of Hematology-Harold Amos Medical Faculty Development Program (ASH-AMFDP). Designed to increase the number of underrepresented minority scholars in the field of hematology, the ASH-AMFDP has awarded Dr. Abraham $420,000 that includes an annual stipend and research grant over the next four years. Here, Dr. Abraham tells us more about his research and what it means for the future of patients with sickle cell disease.

Q: What does this award mean to you?
A: This award comes at a critical time in my early career as I learn how to become an independent grant-funded researcher. It gives me an opportunity to dedicate 70 percent of my time to research for the next four years, during which I will hone my research skills and have access to highly accomplished mentors at Children’s National and from the ASH-AMFDP faculty.

Q: Your research for this grant focuses on improving curative hematopoietic stem cell transplantation for sickle cell disease. Why do they need to be improved?

A: Sickle cell disease causes significant health problems for children, which can worsen as they become adults, and even shorten their lifespan. Curative therapies to date are limited for many patients since most do not have a suitably matched donor for a curative bone marrow transplant. Many of us in the field hope we can provide a safe option for as many patients as possible so they can be cured in childhood and not have to face the negative impacts of the disease as they grow older.

Q: You will also be evaluating virus-specific T-cell (VST) recovery after transplantation. What will this mean for patients?

A: As we explore more transplant donor options such as unrelated donors and mismatched family donors, we have observed delayed immune system recovery. Viral infections are particularly problematic, as they can be life-threatening and respond poorly to available medications. Ultimately, a recovered immune system would address the infection problem. We hope to generate immune cells that are protective against viruses from the transplant donor and give them to patients as part of their transplant procedure.

Q: How do you envision your research improving the future of treatment for sickle cell patients?

A: My hope is that we get closer to having a safer transplant option for most patients who, despite optimal therapy, continue to suffer from complications of sickle cell disease. Ideally, these transplants would not only be widely available, but the treatment would also be simplified to the point where most of the therapy could take place in an outpatient setting.

Q: Why did you decide to work in this field?

A: Sickle cell disease has lagged behind other disorders in terms of new treatment strategies for quite some time. I experienced this as a medical trainee and struggled when parents would ask me to “do something” for their child when most of the time all I could offer was pain medication. In the last five years or so, there has been more focus on sickle cell disease from the field and the community, so now is the time to work toward developing a widely available cure.

Dr. Laura Olivieri holding a 3D printed heart

Cardiology and radiology experts to participate in CMR 2018

January 17, 2018

Later this month, the international cardiovascular magnetic resonance (CMR) community will gather in Barcelona, Spain, for CMR 2018, a joint meeting organized by the European Association of Cardiovascular Imaging (EACVI) and the Society for Cardiovascular Magnetic Resonance (SCMR). Among the many attendees will be several cardiology and radiology experts from Children’s National Heart Institute:

Pediatric cardiology fellow Ashish Doshi, M.D., will be giving a talk titled, “Subendocardial resting perfusion defect in a case of acute fulminant myocarditis,” and will also present a poster titled, “Native T1 measurements in pediatric heart transplant patients correlate with history of prior rejection episodes.”
Pediatric cardiology fellow Rohan Kumthekar, M.D., will present a poster titled, “Native T1 values can identify pediatric patients with myocarditis.”
Cardiologist Laura Olivieri, M.D., will present two posters: “Native T1 measurements from CMR identify severity of myocardial disease over time in patients with Duchenne muscular dystrophy on therapy,” and “Feasibility of noncontrast T1 and T2 parametric mapping in assessment of acute ventricular ablation lesions in children.”
Pediatric cardiology fellow Neeta Sethi, M.D., will present a poster titled, “Cardiac magnetic resonance T2 mapping in the surveillance of acute allograft rejection in pediatric cardiac transplant patients.”

Additionally, Drs. Doshi and Sethi and Ileen Cronin, FNP-BC, a nurse practitioner in the Cardiac Catheterization Laboratory/Interventional Cardiac Magnetic Resonance (ICMR) Program, received travel awards to attend the conference.

CMR 2018 will be held January 31-February 3, 2018 and will focus on the theme of “Improving Clinical Value by Technical Advances.” The meeting’s emphasis will be on the common goal of improving clinical outcomes in cardiovascular disease through innovation in basic MR development and medical engineering.

Newborn screening leader selected to advisory committee on heritable disorders in newborns and children

January 11, 2018

Sarah Viall, PPCNP, coordinator for the Newborn Screening Program at the Children’s National Rare Disease Institute (CNRDI), has been invited to serve on the Education and Training Workgroup of the Health Resources & Services Administration’s (HRSA) Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC).

Established under the Public Health Service Act, the ACHDNC focuses on reducing morbidity and mortality in newborns and children who have, or are at risk for, genetic disorders. The Committee currently recommends that all newborn screening programs include a Uniform Screening Panel that monitors for a total of 34 core disorders and another 26 secondary disorders.

In addition to developing recommendations on national newborn screening guidelines, the ACHDNC also advises the U.S. Department of Health and Human Services Secretary on the most appropriate application of newborn screening technologies, tests, policies and standards. The Committee provides technical information that helps develop Heritable Disorders Program policies and priorities that enhance the ability of local and state health agencies to provide screening, healthcare services and counseling for newborns and children affected by genetic disease.

Viall had previously spent a year observing meetings for the ACHDNC Education and Training Workgroup.

“I am thrilled to be an official member that can contribute to the important work of educating communities about newborn screening,” says Viall.

$3M Retrophin gift establishes Rare Disease Network at Children’s National

January 10, 2018

“This is an exciting first step toward a new era of rare disease care and innovation,” says Marshall Summar, M.D., director of the CNRDI. “We are grateful for this gift from Retrophin that will help us accelerate progress for our patients and families and pursue work that will have a far-reaching impact on both children and adults across the country and around the world thanks to the support of Retrophin.”

Children’s National Health System and Retrophin, Inc. have announced the creation of the Retrophin Rare Disease Network at Children’s National. Retrophin, a biopharmaceutical company specializing in identifying, developing and delivering life-changing therapies to people living with rare diseases, has committed $3 million over the next six years to support the work of the Children’s National Rare Disease Institute (CNRDI). Retrophin’s commitment marks the first corporate gift to CNRDI.

“One of the chief challenges of 21st century pediatric medicine is our continued inability to provide more help to those born with rare genetic diseases,” says Marshall Summar, M.D., director of the CNRDI. “This is an exciting first step toward a new era of rare disease care and innovation. We are grateful for this gift from Retrophin that will help us accelerate progress for our patients and families and pursue work that will have a far-reaching impact on both children and adults across the country and around the world thanks to the support of Retrophin.”

As a dedicated source of funding, the Retrophin Rare Disease Network will advance the CNRDI’s efforts to create a global “hub and spoke” model for disseminating and streamlining patient access to optimal care methods and among national and international peer institutions. The network will enhance the field of rare disease medicine by standardizing care models and establishing world-wide best practices in diagnosis and treatment.

The Retrophin Rare Disease Network will also provide funding for new dedicated positions at the CNRDI and build on the Institute’s existing digital and telemedicine programs, to extend the reach of its researchers and caregivers in areas where there is currently limited care available for children and adults living with rare diseases.

CNRDI is a first-of-its-kind center focused exclusively on advancing the care and treatment of children and adults with rare genetic diseases. The first National Organization for Rare Disorders (NORD) Center of Excellence, it aims to provide a medical home for patients and families seeking the most advanced care and expertise for rare genetic conditions that remain largely unknown to the general medical community.

Stephen Teach, M.D., M.P.H., named associate dean at GW School of Medicine and Health Sciences

January 10, 2018

Stephen J. Teach, M.D., M.P.H., chair of the Department of Pediatrics at Children’s National Health System, was named associate dean for Pediatric Academic Affairs at The George Washington University (GW) School of Medicine and Health Sciences.

Dr. Teach is director and principal investigator of Improving Pediatric Asthma Care in the District of Columbia (IMPACT DC), a care, research and advocacy program focused on helping under-resourced and largely minority children who suffer from asthma. He also serves as principal investigator for the Washington site for the Inner City Asthma Consortium, funded by the National Institutes of Health.

At GW, Dr. Teach will play a critical role in supporting and enhancing education and training relationships between the university and Children’s National and will support the academic advancement of Children’s National faculty. Read more.

Leading blood disorder experts from Children’s National convene in Atlanta for 59th American Society of Hematology annual meeting

January 9, 2018

In early December 2017, more than 25,000 attendees from around the world, including several experts from Children’s National Health System, convened in Atlanta for the American Society of Hematology’s annual meeting and exposition, the world’s premiere hematology event. For four days, physicians, nurses and other healthcare professionals attended sessions, listened to speakers and collaborated with each other, focusing on enhancing care and treatment options for patients with blood disorders and complications, including leukemia, sickle cell disease and transplants.

As nationally recognized leaders in the field, the Children’s National team led educational sessions and gave keynote speeches highlighting groundbreaking work underway at the hospital, which sparked engaging and productive conversations among attendees. Highlights from the team include:

Catherine Bollard, M.D., M.B.Ch.B., Director of the Center for Cancer and Immunology Research, educating global experts on cellular immunotherapy for non-Hodgkin lymphoma.
Kirsten Williams, M.D., bone and marrow transplant specialist, presenting novel work utilizing TAA-specific T cells for hematologic malignancies with Dr. Bollard, the sponsor of this first-in-man immunotherapy; moderating sessions on immunotherapy and late complications and survivorship after hematopoietic stem cell transplantation (HSCT).
Allistair Abraham, M.D., blood and marrow transplantation specialist, moderating a session on hemoglobinopathies.
David Jacobsohn, M.D., ScM, Division Chief of Blood and Marrow Transplantation, moderating a session on allogeneic transplantation results.
Naomi Luban, M.D., hematologist and laboratory medicine specialist, introducing a plenary speaker on the application of CRISPR/Cas 9 technology for development of diagnostic reagents for diagnosis of alloimmunization from stem cells.

Additional presentations from the Children’s National team included an oral abstract on the hospital’s work to improve hydroxyurea treatment for sickle cell disease by pediatric resident Sarah Kappa, M.D., who also received an ASH Abstract Achievement Award; another key session on hemoglobinopathies moderated by Andrew Campbell, M.D., director of the Comprehensive Sickle Cell Disease Program; an abstract on the clinical use of CMV- specific T-cells derived from CMV-native donors, presented by Patrick Hanley, Ph.D.; a leukemia study presented by Anne Angiolillo, M.D., oncologist; and a presentation about pain measurement tools in sickle cell disease by Deepika Darbari, M.D., hematologist.

Combating ENT wrong patient errors

January 9, 2018

A recent article published in ENTtoday highlights specific ways ENT physicians can improve quality and care for patients to work towards eliminating wrong patient errors and achieving a zero-harm environment.

In the article, Rahul Shah, M.D., Vice President and Chief Quality & Safety Officer at Children’s National Health System, points out that ENTs are especially vulnerable to wrong patient errors (WPEs) due to the wide variety of settings in which they see patients. He asserts that with this knowledge in mind, ENTS can find ways to “block and tackle” to prevent WPEs from occurring. Key to success is the development of a supportive culture of reporting where all staff are encouraged and empowered to speak up.

“With any size of practice, you need to talk about safety and quality. If doesn’t have to be formal, and don’t overthink it. Something as easy as a safety huddle a couple of times a week goes a long way toward shaping the culture.”

Read the full article here.

Internationally renowned pediatric nephrologist named to NIH advisory council

January 4, 2018

Pediatric nephrologist Lisa M. Guay-Woodford, M.D., has been named to a three-year term as adviser serving on the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) Kidney, Urologic and Hematologic Diseases subcouncil.

Dr. Guay-Woodford, Director of the Center for Translational Science at Children’s National, is an internationally recognized expert in the mechanisms that modulate the clinical severity of certain inherited renal disorders, such as autosomal recessive polycystic kidney disease. She holds the Richard L. and Agnes F. Hudson Professorship in Health Services Research at Children’s National.

NIDDK, like other grant-awarding institutes within the National Institutes of Health (NIH), looks to its advisory councils for feedback on procedures that govern staff and manage its grant portfolios. The institute, the fifth largest at the NIH, supports clinical research about internal medicine and related subspecialties for many of the most common chronic health conditions.

“It is a tremendous honor to be asked to serve on this important council. I look forward to providing advice and perspective on the exciting portfolio of NIDDK-funded projects,” Dr. Guay-Woodford says.

Population Strategies for Children’s Health Summit

January 3, 2018

Children’s National, with sponsorship from Cerner Corporation, is excited to announce the first Population Strategies for Children’s Health Summit on February 19 – 20, 2018 at The Westin in Washington, D.C. This is the first summit focused exclusively on comprehensive population health management approaches that can help children reach their highest levels of health and potential.

Join us in developing new ideas and best practices that engage millennial healthcare consumers and address challenges pediatric providers face in transitioning to value-based care. You’ll learn how population health management strategies can improve care quality for an entire pediatric population in a way that supports your health system’s bottom line.

Speakers at the summit will focus on topics such as:

Health policy
Care coordination
Physician engagement
Registries and risk stratification
Telehealth
Health disparities
Taking on risk

Get a sneak peek of the featured Millennial Panel discussion on February 20:

The current and future state of health care from a consumer’s perspective

Health care is a dynamic, constantly evolving entity. This three-person panel plus moderator takes on the consumer point of view to discuss what is and isn’t working in health care today. The panel consists of a pediatrician and mom of a child with Type 1 diabetes and a 15-year-old Type 1 diabetes patient. They’ll share their experiences and thoughts about how they believe health care will progress in the future.

For more information about the 2018 Population Strategies for Children’s Health Summit, please visit our website.

William D. Gaillard, M.D., elected Second Vice President of the American Epilepsy Society

December 8, 2017

William Davis Gaillard, M.D., has been elected second vice president of the American Epilepsy Society (AES), a medical and scientific society with 4,000 members. Dr. Gaillard’s term started at the end of the society’s annual meeting, December 1-5, in Washington, D.C.

“The AES is the largest multidisciplinary professional and scientific society dedicated to the understanding, treatment and eradication of epilepsy and associated disorders, and I am honored to serve as the new Second Vice President.” Dr. Gaillard said.

Dr. Gaillard, an internationally recognized expert in pediatric epilepsy and imaging, is chief of Neurology, Epilepsy and Neurophysiology at Children’s National. He is also the associate director of the DC-IDDC and director of the of the Intellectual and Developmental Disabilities Research Center (DC-IDDRC) imaging core and associate director of the Center for Neuroscience Research, Children’s Research Institute. His academic appointments include professor of Pediatrics and Neurology at George Washington University and professor of Neurology at Georgetown University.

As division chief of Child Neurology, Epilepsy and Neurophysiology, Dr. Gaillard directs a team of pediatric specialists who see thousands of patients each year. Dr. Gaillard has worked throughout his career to care for children and young adults with epilepsy from the onset of seizures through novel therapeutic interventions, medication trials and, when appropriate, surgery. Treatment at Children’s National addresses the full range of the condition, including problems of difficult-to-control epilepsy. Additionally, treatment includes the concurrent social, educational and emotional issues faced by children with the condition and their families.

An active participant in AES activities, Dr. Gaillard has served as treasurer and as chair of the Clinical Investigator Workshop and Pediatric Content Committees. He also serves as an associate editor for the journal Epilepsy Research, and as a regular reviewer on AES and Epilepsy Foundation study sections. Dr. Gaillard will service as first vice president in 2019 and accede to the presidency of AES in 2020.