Quality and Safety

DNA Molecule

Decoding cellular signals linked to hypospadias

DNA Molecule

“By advancing our understanding of the genetic causes and the anatomic differences among patients, the real goal of this research is to generate knowledge that will allow us to take better care of children with hypospadias,” Daniel Casella, M.D. says.

Daniel Casella, M.D., a urologist at Children’s National, was honored with an AUA Mid-Atlantic Section William D. Steers, M.D. Award, which provides two years of dedicated research funding that he will use to better understand the genetic causes for hypospadias.

With over 7,000 new cases a year in the U.S., hypospadias is a common birth defect that occurs when the urethra, the tube that transports urine out of the body, does not form completely in males.

Dr. Casella has identified a unique subset of cells in the developing urethra that have stopped dividing but remain metabolically active and are thought to represent a novel signaling center. He likens them to doing the work of a construction foreman. “If you’re constructing a building, you need to make sure that everyone follows the blueprints.  We believe that these developmentally senescent cells are sending important signals that define how the urethra is formed,” he says.

His project also will help to standardize the characterization of hypospadias. Hypospadias is classically associated with a downward bend to the penis, a urethra that does not extend to the head of the penis and incomplete formation of the foreskin. Still, there is significant variability among patients’ anatomy and to date, no standardized method for documenting hypospadias anatomy.

“Some surgeons take measurements in the operating room, but without a standardized classification system, there is no definitive way to compare measurements among providers or standardize diagnoses from measurements that every surgeon makes,” he adds. “What one surgeon may call ‘distal’ may be called ‘midshaft’ by another.” (With distal hypospadias, the urethra opening is near the penis head; with midshaft hypospadias, the urethra opening occurs along the penis shaft.)

“By advancing our understanding of the genetic causes and the anatomic differences among patients, the real goal of this research is to generate knowledge that will allow us to take better care of children with hypospadias,” he says.

Parents worry about lingering social stigma, since some boys with hypospadias are unable to urinate while standing, and in older children the condition can be associated with difficulties having sex. Surgical correction of hypospadias traditionally is performed when children are between 6 months to 1 year old.

When reviewing treatment options with family, “discussing the surgery and postoperative care is straight forward. The hard part of our discussion is not having good answers to questions about long-term outcomes,” he says.

Dr. Casella’s study hopes to build the framework to enable that basic research to be done.

“Say we wanted to do a study to see how patients are doing 15-20 years after their surgery.  If we go to their charts now, often we can’t accurately describe their anatomy prior to surgery.  By establishing uniform measurement baselines, we can accurately track long-term outcomes since we’ll know what condition that child started with and where they ended up,” he says.

Dr. Casella’s research project will be conducted at Children’s National under the mentorship of Eric Vilain, M.D., Ph.D., an international expert in sex and genitalia development; Dolores J. Lamb, Ph.D., HCLD, an established leader in urology based at Weill Cornell Medicine; and Marius George Linguraru, DPhil, MA, MSc, an expert in image processing and artificial intelligence.

Billie Lou Short and Kurt Newman at Research and Education Week

Research and Education Week honors innovative science

Billie Lou Short and Kurt Newman at Research and Education Week

Billie Lou Short, M.D., received the Ninth Annual Mentorship Award in Clinical Science.

People joke that Billie Lou Short, M.D., chief of Children’s Division of Neonatology, invented extracorporeal membrane oxygenation, known as ECMO for short. While Dr. Short did not invent ECMO, under her leadership Children’s National was the first pediatric hospital to use it. And over decades Children’s staff have perfected its use to save the lives of tiny, vulnerable newborns by temporarily taking over for their struggling hearts and lungs. For two consecutive years, Children’s neonatal intensive care unit has been named the nation’s No. 1 for newborns by U.S. News & World Report. “Despite all of these accomplishments, Dr. Short’s best legacy is what she has done as a mentor to countless trainees, nurses and faculty she’s touched during their careers. She touches every type of clinical staff member who has come through our neonatal intensive care unit,” says An Massaro, M.D., director of residency research.

For these achievements, Dr. Short received the Ninth Annual Mentorship Award in Clinical Science.

Anna Penn, M.D., Ph.D., has provided new insights into the central role that the placental hormone allopregnanolone plays in orderly fetal brain development, and her research team has created novel experimental models that mimic some of the brain injuries often seen in very preterm babies – an essential step that informs future neuroprotective strategies. Dr. Penn, a clinical neonatologist and developmental neuroscientist, “has been a primary adviser for 40 mentees throughout their careers and embodies Children’s core values of Compassion, Commitment and Connection,” says Claire-Marie Vacher, Ph.D.

For these achievements, Dr. Penn was selected to receive the Ninth Annual Mentorship Award in Basic and Translational Science.

The mentorship awards for Drs. Short and Penn were among dozens of honors given in conjunction with “Frontiers in Innovation,” the Ninth Annual Research and Education Week (REW) at Children’s National. In addition to seven keynote lectures, more than 350 posters were submitted from researchers – from high-school students to full-time faculty – about basic and translational science, clinical research, community-based research, education, training and quality improvement; five poster presenters were showcased via Facebook Live events hosted by Children’s Hospital Foundation.

Two faculty members won twice: Vicki Freedenberg, Ph.D., APRN, for research about mindfulness-based stress reduction and Adeline (Wei Li) Koay, MBBS, MSc, for research related to HIV. So many women at every stage of their research careers took to the stage to accept honors that Naomi L.C. Luban, M.D., Vice Chair of Academic Affairs, quipped that “this day is power to women.”

Here are the 2019 REW award winners:

2019 Elda Y. Arce Teaching Scholars Award
Barbara Jantausch, M.D.
Lowell Frank, M.D.

Suzanne Feetham, Ph.D., FAA, Nursing Research Support Award
Vicki Freedenberg, Ph.D., APRN, for “Psychosocial and biological effects of mindfulness-based stress reduction intervention in adolescents with CHD/CIEDs: a randomized control trial”
Renee’ Roberts Turner for “Peak and nadir experiences of mid-level nurse leaders”

2019-2020 Global Health Initiative Exploration in Global Health Awards
Nathalie Quion, M.D., for “Latino youth and families need assessment,” conducted in Washington
Sonia Voleti for “Handheld ultrasound machine task shifting,” conducted in Micronesia
Tania Ahluwalia, M.D., for “Simulation curriculum for emergency medicine,” conducted in India
Yvonne Yui for “Designated resuscitation teams in NICUs,” conducted in Ghana
Xiaoyan Song, Ph.D., MBBS, MSc, “Prevention of hospital-onset infections in PICUs,” conducted in China

Ninth Annual Research and Education Week Poster Session Awards

Basic and Translational Science
Faculty: Adeline (Wei Li) Koay, MBBS, MSc, for “Differences in the gut microbiome of HIV-infected versus HIV-exposed, uninfected infants”
Faculty: Hayk Barseghyan, Ph.D., for “Composite de novo Armenian human genome assembly and haplotyping via optical mapping and ultra-long read sequencing”
Staff: Damon K. McCullough, BS, for “Brain slicer: 3D-printed tissue processing tool for pediatric neuroscience research”
Staff: Antonio R. Porras, Ph.D., for “Integrated deep-learning method for genetic syndrome screening using facial photographs”
Post docs/fellows/residents: Lung Lau, M.D., for “A novel, sprayable and bio-absorbable sealant for wound dressings”
Post docs/fellows/residents: Kelsey F. Sugrue, Ph.D., for “HECTD1 is required for growth of the myocardium secondary to placental insufficiency”
Graduate students: Erin R. Bonner, BA, for “Comprehensive mutation profiling of pediatric diffuse midline gliomas using liquid biopsy”
High school/undergraduate students: Ali Sarhan for “Parental somato-gonadal mosaic genetic variants are a source of recurrent risk for de novo disorders and parental health concerns: a systematic review of the literature and meta-analysis”

Clinical Research
Faculty: Amy Hont, M.D., for “Ex vivo expanded multi-tumor antigen specific T-cells for the treatment of solid tumors”
Faculty: Lauren McLaughlin, M.D., for “EBV/LMP-specific T-cells maintain remissions of T- and B-cell EBV lymphomas after allogeneic bone marrow transplantation”

Staff: Iman A. Abdikarim, BA, for “Timing of allergenic food introduction among African American and Caucasian children with food allergy in the FORWARD study”
Staff: Gelina M. Sani, BS, for “Quantifying hematopoietic stem cells towards in utero gene therapy for treatment of sickle cell disease in fetal cord blood”
Post docs/fellows/residents: Amy H. Jones, M.D., for “To trach or not trach: exploration of parental conflict, regret and impacts on quality of life in tracheostomy decision-making”
Graduate students: Alyssa Dewyer, BS, for “Telemedicine support of cardiac care in Northern Uganda: leveraging hand-held echocardiography and task-shifting”
Graduate students: Natalie Pudalov, BA, “Cortical thickness asymmetries in MRI-abnormal pediatric epilepsy patients: a potential metric for surgery outcome”
High school/undergraduate students: Kia Yoshinaga for “Time to rhythm detection during pediatric cardiac arrest in a pediatric emergency department”

Community-Based Research
Faculty: Adeline (Wei Li) Koay, MBBS, MSc, for “Recent trends in the prevention of mother-to-child transmission (PMTCT) of HIV in the Washington, D.C., metropolitan area”
Staff: Gia M. Badolato, MPH, for “STI screening in an urban ED based on chief complaint”
Post docs/fellows/residents: Christina P. Ho, M.D., for “Pediatric urinary tract infection resistance patterns in the Washington, D.C., metropolitan area”
Graduate students: Noushine Sadeghi, BS, “Racial/ethnic disparities in receipt of sexual health services among adolescent females”

Education, Training and Program Development
Faculty: Cara Lichtenstein, M.D., MPH, for “Using a community bus trip to increase knowledge of health disparities”
Staff: Iana Y. Clarence, MPH, for “TEACHing residents to address child poverty: an innovative multimodal curriculum”
Post docs/fellows/residents: Johanna Kaufman, M.D., for “Inpatient consultation in pediatrics: a learning tool to improve communication”
High school/undergraduate students: Brett E. Pearson for “Analysis of unanticipated problems in CNMC human subjects research studies and implications for process improvement”

Quality and Performance Improvement
Faculty: Vicki Freedenberg, Ph.D., APRN, for “Implementing a mindfulness-based stress reduction curriculum in a congenital heart disease program”
Staff: Caleb Griffith, MPH, for “Assessing the sustainability of point-of-care HIV screening of adolescents in pediatric emergency departments”
Post docs/fellows/residents: Rebecca S. Zee, M.D., Ph.D., for “Implementation of the Accelerated Care of Torsion (ACT) pathway: a quality improvement initiative for testicular torsion”
Graduate students: Alysia Wiener, BS, for “Latency period in image-guided needle bone biopsy in children: a single center experience”

View images from the REW2019 award ceremony.

Beth Tarini

Getting to know SPR’s future President, Beth Tarini, M.D., MS

Beth Tarini

Quick. Name four pillar pediatric organizations on the vanguard of advancing pediatric research.

Most researchers and clinicians can rattle off the names of the Academic Pediatric Association, the American Academy of Pediatrics and the American Pediatric Society. But that fourth one, the Society for Pediatric Research (SPR), is a little trickier. While many know SPR, a lot of research-clinicians simply do not.

Over the next few years, Beth A. Tarini, M.D., MS, will make it her personal mission to ensure that more pediatric researchers get to know SPR and are so excited about the organization that they become active members. In May 2019 Dr. Tarini becomes Vice President of the society that aims to stitch together an international network of interdisciplinary researchers to improve kids’ health. Four-year SPR leadership terms begin with Vice President before transitioning to President-Elect, President and Past-President, each for one year.

Dr. Tarini says she looks forward to working with other SPR leaders to find ways to build more productive, collaborative professional networks among faculty, especially emerging junior faculty. “Facilitating ways to network for research and professional reasons across pediatric research is vital – albeit easier said than done. I have been told I’m a connector, so I hope to leverage that skill in this new role,” says Dr. Tarini, associate director for Children’s Center for Translational Research.

“I’m delighted that Dr. Tarini was elected to this leadership position, and I am impressed by her vision of improving SPR’s outreach efforts,” says Mark Batshaw, M.D., Executive Vice President, Chief Academic Officer and Physician-in-Chief at Children’s National. “Her goal of engaging potential members in networking through a variety of ways – face-to-face as well as leveraging digital platforms like Twitter, Facebook and LinkedIn – and her focus on engaging junior faculty will help strengthen SPR membership in the near term and long term.”

Dr. Tarini adds: “Success to me would be leaving after four years with more faculty – especially junior faculty – approaching membership in SPR with the knowledge and enthusiasm that they bring to membership in other pediatric societies.”

SPR requires that its members not simply conduct research, but move the needle in their chosen discipline. In her research, Dr. Tarini has focused on ensuring that population-based newborn screening programs function efficiently and effectively with fewer hiccups at any place along the process.

Thanks to a heel stick to draw blood, an oxygen measurement, and a hearing test, U.S. babies are screened for select inherited health conditions, expediting treatment for infants and reducing the chances they’ll experience long-term health consequences.

“The complexity of this program that is able to test nearly all 4 million babies in the U.S. each year is nothing short of astounding. You have to know the child is born – anywhere in the state – and then between 24 and 48 hours of birth you have to do testing onsite, obtain a specific type of blood sample, send the blood sample to an off-site lab quickly, test the sample, find the child if the test is out of range, get the child evaluated and tested for the condition, then send them for treatment. Given the time pressures as well as the coordination of numerous people and organizations, the fact that this happens routinely is amazing. And like any complex process, there is always room for improvement,” she says.

Dr. Tarini’s research efforts have focused on those process improvements.

As just one example, the Advisory Committee on Heritable Disorders in Newborns and Children, a federal advisory committee on which she serves, was discussing how to eliminate delays in specimen processing to provide speedier results to families. One possible solution floated was to open labs all seven days, rather than just five days a week. Dr. Tarini advocated for partnering with health care engineers who could help model ways to make the specimen transport process more efficient, just like airlines and mail delivery services. A more efficient and effective solution was to match the specimen pick-up and delivery times more closely with the lab’s operational times – which maximizes lab resources and shortens wait times for parents.

Conceptual modeling comes so easily for her that she often leaps out of her seat mid-sentence, underscoring a point by jotting thoughts on a white board, doing it so often that her pens have run dry.

“It’s like a bus schedule: You want to find a bus that not only takes you to your destination but gets you there on time,” she says.

Dr. Tarini’s current observational study looks for opportunities to improve how parents in Minnesota and Iowa are given out-of-range newborn screening test results – especially false positives – and how that experience might shake their confidence in their child’s health as well as heighten their own stress level.

“After a false positive test result, are there parents who walk away from newborn screening with lingering stress about their child’s health? Can we predict who those parents might be and help them?” she asks.

Among the challenges is the newborn screening occurs so quickly after delivery that some emotionally and physically exhausted parents may not remember it was done. Then they get a call from the state with ominous results. Another challenge is standardizing communication approaches across dozens of birthing centers and hospitals.

“We know parents are concerned after receiving a false positive result, and some worry their infant remains vulnerable,” she says. “Can we change how we communicate – not just what we say, but how we say it – to alleviate those concerns?”

Johannes Van den Anker

Dr. Johannes van den Anker awarded 2019 Sumner J. Yaffe Lifetime Achievement Award

Johannes Van den Anker

Johannes van den Anker, M.D., Ph.D., division chief of Clinical Pharmacology and vice chair of Pediatrics for Experimental Therapeutics at Children’s National Health System, has been selected to receive the 2019 Sumner J. Yaffe Lifetime Achievement Award in Pediatric Pharmacology and Therapeutics by the Pediatric Pharmacy Advocacy Group (PPAG). Given annually, the Yaffe Award was established in 2002 by the PPAG Board of Directors and recognizes individuals with significant and sustained contributions toward the improvement of children’s health through the field of pediatric pharmacology and therapeutics.

Dr. van den Anker was selected as this year’s recipient for his contributions to the field of pediatric pharmacology and therapeutics, which have expanded and enhanced medical knowledge about the use of drugs in children and about the treatment of disease. He has also played an integral role in training the next generation of clinical pharmacists and pharmacologists.

“This award means a lot to me as it recognizes the importance of the field I am so passionate about and to which I have dedicated my career,” says Dr. van den Anker.

Dr. van den Anker joined Children’s National in 2002 and has become a leader in the discipline of pediatric pharmacology and therapeutics with significant contributions to research in this field. Some of his work includes changes in the dosing guidelines for frequently used antibacterial agents in newborns, the optimization of the dosing of pain medications in newborns and young infants and studies addressing the pharmacology of drugs in obese pediatric and adolescent patients.

“I am excited about being the 2019 recipient of this award” Dr. van den Anker says, “I am enthusiastic about future developments in the field of pediatric pharmacology and therapeutics with multiple ongoing studies with my colleagues, ranging from antibiotic dosing to the management of muscular dystrophy with novel drugs.”

The award will be presented at the 28th PPAG Annual Meeting on Friday, April 12 in Oklahoma City, OK., where he will also present the 2019 Yaffe Award Lecture to the attendees. The title of his lecture is “The Evolution of Neonatal Pharmacology and Therapeutics:  A Story of Resistance, Resilience and Revelation”.

Congratulations to Dr. Johannes van den Anker for this highly deserved honor!

Meghan Delaney

Pathology chief appointed to board of pathology advisory committee

Meghan Delaney

The American Board of Pathology (ABPath) has appointed the chief of Pathology and Laboratory Medicine at Children’s National Health System, Meghan Delaney, D.O., M.P.H., to its Test Development and Advisory Committee (TDAC) for blood banking and transfusion medicine. As a member of the committee, Dr. Delaney will play a role in the development and review of the American Board of Pathology certification exam questions. Physicians selected to serve on the TDAC are established subject matter experts in their subspecialty, with knowledge on the latest advances in the field of pathology and patient care.

“As TDAC members, these physicians play a critical role in the development of the exams and are entrusted with maintaining the integrity of the board-certified designation. The appointment to a TDAC indicates the physician is highly regarded in the field of pathology and exemplifies the utmost standards of care,” states Rebecca L. Johnson, M.D., CEO of the American Board of Pathology.

Dr. Delaney joined Children’s National as Chief of Pathology and Laboratory Medicine in 2017. A diplomate of the American Board of Pathology, with certification in transfusion medicine/blood banking and clinical pathology, she is an active member of several professional societies. She serves as chair of the AABB Transfusion Medicine Subsection Pediatric Subcommittee and as chair of the American Society of Apheresis Applications Committee Pediatric Subcommittee.  Dr. Delaney is also a scientific member of the BEST Collaborative, an associate editor for the journal Transfusion Medicine and a member of the editorial board of Transfusion.

The mission of the American Board of Pathology, as a member of the American Board of Medical Specialties, is to serve the public and advance the profession of pathology, by setting certification standards and promoting lifelong competency of pathologists. Founded in 1936, the ABPath accomplishes this mission by establishing certification and continuing certification standards, as well as, assessing the qualifications of those seeking to obtain voluntary certification in the specialty of pathology.  Since 1971, the ABPath has appointed test committees for each specialty area of pathology. The committee consists of ABPath trustees and other pathologists, or specialty physicians, who are recognized experts in their respective disciplines.

ACC19 attendees from Children's National

ACC.19: A focus on pediatric cardiology

ACC19 attendees from Children's National

Dr. Gerard Martin, center, accepts an award before delivering the 2019 Dan G. McNamara Keynote lecture at ACC.19.

“Innovation meets tradition,” is how many attendees and journalists described the American College of Cardiology’s 68th Scientific Sessions (ACC.19), which took place March 16-18, 2019 in New Orleans, La.

Gerard Martin, M.D., F.A.A.P., F.A.C.C., F.A.H.A., a pediatric cardiologist and the medical director of Global Services at Children’s National, supported this narrative by referencing both themes in his 2019 Dan G. McNamara keynote lecture, entitled “Improved Outcomes in Congenital Heart Disease through Advocacy and Collaboration.” Dr. Martin highlighted advancements in the field of pediatric cardiology that took place over the past 15 years, while touting modern advancements – such as pulse oximetry screenings for critical congenital heart disease – that were a result of physician-led advocacy and collaboration.

Dr. Martin’s message was to continue to invest in research and technology that leads to medical breakthroughs, but to remember the power of partnerships, such as those formed by the National Pediatric Cardiology Quality Improvement Collaborative. These alliances, which generated shared protocols and infrastructure among health systems, improved interstage mortality rates between surgeries for babies born with hypoplastic left heart syndrome.

A dozen cardiologists and clinicians from the Children’s National Heart Institute also participated in CME panel discussions or delivered poster presentations to support future versions of this template, touching on early-stage innovations and multi-institution research collaborations. The themes among Children’s National Heart Institute faculty, presented to a diverse crowd of 12,000-plus professional attendees representing 108 countries, included:

Personalized guidelines:

  • Sarah Clauss, M.D., F.A.C.C., a cardiologist, presented “Unique Pediatric Differences from Adult Cholesterol Guidelines: Lipids and Preventive Cardiology,” before Charles Berul, M.D., division chief of cardiology and co-director of the Children’s National Heart Institute, presented “Unique Pediatric Differences from Adult Guidelines: Arrhythmias in Adults with Congenital Heart Disease,” in a joint symposium with the American Heart Association and the American College of Cardiology.
  • Berul, who specializes in electrophysiology, co-chaired a congenital heart disease pathway session, entitled “Rhythm and Blues: Electrophysiology Progress and Controversies in Congenital Heart Disease,” featuring components of pediatric electrophysiology, including heart block, surgical treatment of arrhythmias and sudden death risk.

Early detection:

  • Anita Krishnan, M.D., associate director of the echocardiography lab, presented “Identifying Socioeconomic and Geographic Barriers to Prenatal Detection of Hypoplastic Left Heart Syndrome and Transposition of the Great Arteries” as a moderated poster in Fetal Cardiology: Quickening Discoveries.
  • Jennifer Romanowicz, M.D., a cardiology fellow, and Russell Cross, M.D., director of cardiac MRI, presented the “Neonatal Supraventricular Tachycardia as a Presentation of Critical Aortic Coarctation” poster in FIT Clinical Decision Making: Congenital Heart Disease 2.
  • Pranava Sinha, M.D., a cardiac surgeon, presented the poster “Neuroprotective Effects of Vitamin D Supplementation in Children with Cyanotic Heart Defects: Insights from a Rodent Hypoxia Model” in Congenital Heart Disease: Therapy 2.

Coordinated care:

  • Ashraf Harahsheh, M.D., F.A.C.C., F.A.A.P., a cardiologist with a focus on hyperlipidemia and preventive cardiology, co-presented an update about BMI quality improvement (Q1) activity from the American College of Cardiology’s Adult Congenital and Pediatric Quality Network – BMI Q1 leadership panel.
  • Niti Dham, M.D., director of the cardio-oncology program, and Deepa Mokshagundam, M.D., cardiology fellow, presented the poster “Cardiac Changes in Pediatric Cancer Survivors” in Heart Failure and Cardiomyopathies: Clinical 3.
  • Nancy Klein, B.S.N., R.N., C.P.N., clinical program coordinator of the Washington Adult Congenital Heart program at Children’s National, presented the poster “Improving Completion of Advanced Directives in Adults with Congenital Heart Disease” in Risks and Rewards in Adult Congenital Heart Disease.

Innovation:

  • Jai Nahar, M.D., a cardiologist, moderated “Future Hub: Augmented Cardiovascular Practitioner: Giving Doctors and Patients a New Voice.” The session focused on technical aspects of artificial intelligence, such as language processing and conversational artificial intelligence, as well as how applications are used in patient-physician interactions.
  • Nahar also participated in a key event on the Heart-to-Heart stage, entitled “Rise of Intelligent Machines: The Potential of Artificial Intelligence in Cardiovascular Care.”

“While I enjoyed the significant representation of Children’s National faculty at the meeting and all of the presentations this year, one research finding that I found particularly compelling was Dr. Krishnan’s poster about geographical disparities in detecting congenital heart disease,” says Dr. Berul. “Her research finds obstetricians providing care to women in the lowest quartile of socioeconomic areas were twice as likely to miss a diagnosis for a critical congenital heart defect during a fetal ultrasound, compared to obstetricians providing care for women in the highest quartiles.”

Dr. Krishnan’s study was the collaborative effort of 21 centers in the United States and Canada, and investigated how socioeconomic and geographic factors affect prenatal detection of hypoplastic left heart syndrome and transposition of the great arteries.

“We studied over 1,800 patients, and chose these diseases because they require early stabilization by a specialized team at a tertiary care center,” says Dr. Krishnan, who led the research in conjunction with the Fetal Heart Society Research Collaborative. “We hope that by understanding what the barriers are, we can reduce disparities in care through education and community-based outreach.”

Nichole Jefferson and Patrick Gee

African American stakeholders help to perfect the APOLLO study

Nichole Jefferson and Patrick Gee

Nichole Jefferson and Patrick O. Gee

African Americans who either donated a kidney, received a kidney donation, are on dialysis awaiting a kidney transplant or have a close relative in one of those categories are helping to perfect a new study that aims to improve outcomes after kidney transplantation.

The study is called APOLLO, short for APOL1 Long-Term Kidney Transplantation Outcomes Network. Soon, the observational study will begin to enroll people who access transplant centers around the nation to genotype deceased and living African American kidney donors and transplant recipients to assess whether they carry a high-risk APOL1 gene variant.

The study’s Community Advisory Council – African American stakeholders who know the ins and outs of kidney donation, transplantation and dialysis because they’ve either given or  received an organ or are awaiting transplant – are opening the eyes of researchers about the unique views of patients and families.

Already, they’ve sensitized researchers that patients may not be at the same academic level as their clinicians, underscoring the importance of informed consent language that is understandable, approachable and respectful so people aren’t overwhelmed. They have encouraged the use of images and color to explain the apolipoprotein L1 (APOL1) gene. The APOL1 gene is found almost exclusively in people of recent African descent, however only 13 percent of these people carry the high-risk APOL1 variant that might cause kidney problems.

One issue arose early, during one of the group’s first monthly meetings, as they discussed when to tell patients and living donors about the APOLLO study. Someone suggested the day of the transplant.

“The Community Advisory Council told them that would not be appropriate. These conversations should occur well before the day of the transplant,” recalls Nichole Jefferson.

“The person is all ready to give a kidney. If you’re told the day of transplant ‘we’re going to include you in this study,’ that could possibly stop them from giving the organ,” Jefferson says. “We still remember the Tuskegee experiments. We still remember Henrietta Lacks. That is what we are trying to avoid.”

Patrick O. Gee, Ph.D., JLC, another Community Advisory Council member, adds that it’s important to consider “the mental state of the patient and the donor. As a patient, you know you are able to endure a five- to eight-hour surgery. The donor is the recipient’s hero. As the donor, you want to do what is right. But if you get this information; it’s going to cause doubt.”

Gee received his kidney transplant on April 21, 2017, and spent 33 days in the hospital undergoing four surgeries. His new kidney took 47 days to wake up, which he describes as a “very interesting journey.” Jefferson received her first transplant on June 12, 2008. Because that kidney is in failure, she is on the wait list for a new kidney.

“All I’ve ever known before APOLLO was diabetes and cardiovascular issues. Nobody had ever talked about genetics,” Gee adds. “When I tell people, I tread very light. I try to stay in my lane and not to come off as a researcher or a scientist. I just find out information and just share it with them.”

As he spoke during a church function, people began to search for information on their smart phones. He jotted down questions “above his pay grade” to refer to the study’s principal investigator. “When you start talking about genetics and a mutated gene, people really want to find out. That was probably one of the best things I liked about this committee: It allows you to learn, so you can pass it on.”

Jefferson’s encounters are more unstructured, informing people who she meets about her situation and kidney disease. When she traveled from her Des Moines, Iowa, home to Nebraska for a transplant evaluation, the nephrologist there was not aware of the APOL1 gene.

And during a meeting at the Mayo Clinic with a possible living donor, she asked if they would test for the APOL1 gene. “They stopped, looked at me and asked: ‘How do you know about that gene?’ Well, I’m a black woman with kidney failure.”

Patrick O. Gee received his kidney transplant on April 21, 2017, and spent 33 days in the hospital undergoing four surgeries. His new kidney took 47 days to wake up, which he describes as a “very interesting journey.”

About 100,000 U.S. children and adults await a kidney transplant. APOLLO study researchers believe that clarifying the role that the APOL1 gene plays in kidney-transplant failure could lead to fewer discarded kidneys, which could boost the number of available kidneys for patients awaiting transplant.

Gee advocates for other patients and families to volunteer to join the APOLLO Community Advisory Council. He’s still impressed that during the very first in-person gathering, all researchers were asked to leave the table. Only patients and families remained.

“They wanted to hear our voices. You rarely find that level of patient engagement. Normally, you sit there and listen to conversations that are over your head. They have definitely kept us engaged,” he says. “We have spoken the truth, and Dr. Kimmel is forever saying ‘who would want to listen to me about a genotype that doesn’t affect me? We want to hear your voice.’ ”

(Paul L. Kimmel, M.D., MACP, a program director at the National Institute of Diabetes and Digestive and Kidney Diseases, is one of the people overseeing the APOLLO study.)

Jefferson encourages other people personally impacted by kidney disease to participate in the APOLLO study.

“Something Dr. Kimmel always says is ‘You’re in the room.’ We’re in the room while it’s happening. It’s a line from Hamilton. That’s a good feeling,” she says. “I knew right off, these are not necessarily improvements I will see in my lifetime. I am OK with that. With kidney disease, we have not had advances in a long time. As long as my descendants don’t have to go through the same things I have gone through, I figure I have done my part. I have done my job.”

Kinsley and Dr. Timothy Kane

Case study: Diagnosing a choledochal cyst in utero

Kinsley and Dr. Timothy Kane

The Feigel family worked with Timothy Kane, M.D., the division chief of general and thoracic surgery at Children’s National, to ensure an accurate diagnosis, coordinate a corrective procedure and support a strong recovery for Kinsley, who just celebrated a 5-month milestone.

On Sept. 30, 2018, Elizabeth Feigel gave birth to a healthy baby girl, Kinsley Feigel. Thirty-two days later, Elizabeth and her husband, Steven Feigel, delighted in another hospital moment: Kinsley, who developed a choledochal cyst in utero, was recovering from a surgical procedure to remove an abnormal bile duct cyst, which also required the removal of her gallbladder.

While the series of events, interspersed with multiple hospital visits, would likely create uneasiness in new parents, the Feigel family worked with Vahe Badalyan, M.D., a gastroenterologist at Children’s National Health System, and with Timothy Kane, M.D., the division chief of general and thoracic surgery at Children’s National, to ensure an accurate diagnosis, coordinate a corrective procedure and support a strong recovery for Kinsley, who just celebrated a 5-month milestone.

One of the keys to Kinsley’s success was close communication between her parents and providers.

Dr. Badalyan and Dr. Kane listened to Elizabeth and Steven’s concerns, explained complex medical terms in lay language, and provided background about Kinsley’s presenting symptoms, risk factors and procedures. Instead of second-guessing the diagnosis, Elizabeth and Steven put their trust into and remained in contact with the medical team, sharing updates about Kinsley at home. This parent-physician partnership helped ensure an accurate diagnosis and tailored treatment for Kinsley.

Here is her story.

An early diagnosis

During a 12-week prenatal ultrasound, Elizabeth discovered that Kinsley had an intra-abdominal cyst. Before Elizabeth came to Children’s National for an MRI, she met with several fetal medicine specialists and had a variety of tests, including an amniocentesis to rule out chromosomal abnormalities, such as Down syndrome.

The team at Children’s National didn’t want to prematurely confirm Kinsley’s choledochal cyst in utero, but additional ultrasounds and an MRI helped narrow the diagnosis to a few conditions.

After Kinsley was born, and despite looking like a healthy, full-term baby, she was transported to the neonatal intensive care unit (NICU) at Children’s National. Dr. Badalyan and Dr. Kane analyzed Kinsley’s postnatal sonogram and found the cyst was bigger than they previously thought. Over a five-day period, the medical team kept Kinsley under their close watch, running additional tests, including an additional sonogram. They then followed up with Kinsley on an outpatient basis to better understand and diagnose her cyst.

Outpatient care

Over the next few weeks, Kinsley, Elizabeth and Steven returned to Children’s National to coordinate multiple exams, ranging from an MRI to a HIDA scan. During this period, Elizabeth and Steven remained in contact with Dr. Badalyan. They heard about Kinsley’s lab results and sent updates about her symptoms, including her stool, which helped the medical team monitor her status.

Meanwhile, Dr. Badalyan and Dr. Kane worked closely with the lab to measure Kinsley’s bilirubin levels. Her presenting symptoms and risk factors, she had jaundice and is a female baby of Asian descent, are associated with both choledochal cysts and biliary atresia.

Over time and with the help of Elizabeth, Steven and the pediatric radiologists, Dr. Badalyan and Dr. Kane confirmed Kinsley had a type 1 choledochal cyst, the most common. Originally, the plan was to operate at three to six months, but Dr. Kane needed to expedite the procedure and operate on Kinsley at one month due to a rise in her bilirubin, a sign of progressive liver disease.

Higher bilirubin levels are common in newborns and remain elevated at about 5 mg/dL after the first few days of birth, but Kinsley’s levels peaked and remained elevated. Instead of her bile flowing into her intestine, her choledochal cyst reduced the flow of bile, which accumulated and started to pour back into her liver. The timing of the surgery was as important as the procedure.

The surgery

On Oct. 31, Halloween, Kinsley had laparoscopic surgery to remove the choledochal cyst. Approximately five to seven patients per year undergo choledochal cyst removal at Children’s National. Smaller infants typically undergo removal of a choledochal cyst using a large incision (or open procedure). Kinsley was the smallest baby at Children’s National to have this type of surgery performed by minimally invasive laparoscopic surgery, which required a few 3-mm incisions – the size of coriander seeds.

Some hospitals use the da Vinci robot, which starts at 8-mm incisions, the size of a small pearl, to conduct this procedure on infants, but this method cannot effectively be done in very small infants. Instead, Dr. Kane prefers to stitch sutures by hand. This technique keeps the incisions small and is technically demanding, but Dr. Kane doesn’t mind (he views this as an advanced technical skill). The goal for this surgery was to cut out the abnormal piece of Kinsley’s common bile duct, comprised of the cyst, remove  this and then sew the bile duct to the small intestine (duodenum), creating a digestive pathway. The new digestive tube allows for bile to flow from her liver through the common hepatic duct, in place of the pathway where the cyst formed, and into her intestine.

Like other surgeries, Dr. Kane needed to adapt the procedure, especially with Kinsley’s size: Taking too much from the bile duct would create a tight space, and could create obstruction, blocking bile, while leaving too much room could create leakage and spilling of the bile, requiring a follow-up surgical procedure within a week or two of the original operation.

Dr. Kane had a few options in mind before he operated. He didn’t know which would be most suitable until the operation, but he remained open and prepared for all three. Adopting this mindset, instead of having one procedure in mind, has helped Dr. Kane with precise and tailored surgeries, which often result in the best procedure and a stronger recovery period for young patients.

After 4.5 hours, the surgery, a two-part procedure – removing the cyst and recreating a functional bile duct – was complete.

Kinsley moved into the recovery unit, where she rested and recovered under close medical supervision for five days. During the first few days, she didn’t have liquids or milk, but she did have two bedside nurses monitoring her status in addition to surgeons making regular rounds. Elizabeth and Steven were relieved: The diagnosis and surgery were over.

Managing risk factors

Before Kinsley left the hospital, Elizabeth and Steven scheduled a follow-up visit to ensure Kinsley was recovering well and avoided risk of infection, such as cholangitis, which can occur suddenly and become chronic.

Following Kinsley’s post-surgical bloodwork in early November, Dr. Badalyan noticed Kinsley’s white blood count was high, signaling infection, and he immediately brought the family back to the hospital. To help her body fight the infection, Kinsley received antibiotics and intravenous fluids. She stayed in the hospital for five days. Fortunately, cholangitis is easy to treat with antibiotics; the key is to detect it early.

Kinsley returned home in time for Thanksgiving. She came back to the hospital for biweekly visits. At this point, she was filling out, reaching a 2-month milestone and nearing a full recovery. She returned for follow-up visits in December and January – and has been healthy ever since. She will continue to make routine visits during her first year to ensure her white blood count remains in a healthy range.

Investing in youth resilience

Dr. Badalyan and Dr. Kane envision a healthy future for Kinsley. They don’t expect she’ll need additional operations. Her parents are also looking on the bright side: Since gallbladders aren’t essential for survival or long-term health outcomes, and since many people can easily live without them, Kinsley may be at an advantage. Elizabeth thinks Kinsley may be more cautious about lifestyle choices to support living without a gallbladder, which also support longevity.

Another perspective noted by Dr. Badalyan and Dr. Kane is Kinsley’s resilience factor. Having the surgery earlier brought unique challenges, but her age makes it easier for Kinsley to bounce back as her body rapidly develops. Her tissues were healthy, compared to adult patients undergoing surgery with chronic liver problems or heart disease, which puts her at an advantage for a faster healing process. Dr. Badalyan also mentions that while it’s good for her Kinsley and her family to continue to monitor risks for infections, she won’t have gallstones.

Elizabeth also started to notice something that Kinsley’s doctors likely wouldn’t pick up on: Her personality seems to be a result of her hospital experience and stay. Kinsley’s an easy baby. She eats well and sleeps well, which Elizabeth credits to being around clinicians and to learning the art of self-soothing, a skill she likely acquired while recovering from surgery.

This month, Kinsley has another adventure. She’ll travel with her parents to visit extended family in Seattle, Napa Valley, Calif. and West Virginia. She has several relatives and family friends, all of whom are looking forward to meeting her.

Prescription for a healthy heart: pediatric-driven partnerships

Dr. Martin and a patient share a smile after a visit at Children’s National Health System.

For pediatric cardiologists, February, National Heart Month, is a special time. We share health tips in the hospital and talk about heart health with those looking for advice, especially with patients and families impacted by congenital heart disease (CHD). It’s also a time to look back at what’s worked well in the field, while accelerating advancements for CHD treatment.

To start, congenital heart disease, a structural abnormality of the heart or of the blood vessels surrounding it, is the most common birth defect and occurs in about one in every 100 live births, affecting 40,000 babies born in the U.S. each year. One million children and 1.4 million adults in the U.S. have CHD. Over the past 15 years, pediatric cardiologists have cut mortality rates for CHD in half. Gratefully, now instead of saving children’s lives, the emphasis is on improving them. The catalyst for this paradigm shift isn’t simply due to a medical breakthrough, but is also the result of collaboration and advocacy.

Pediatric cardiologists worked together with other stakeholders – nurses, neonatologists, parents, state and federal agencies – to implement newborn screening methods in hospitals, with the introduction pulse oximetry screenings for critical congenital heart defects (CCHD). The screening, which measures blood oxygen levels in newborns, focuses on screening babies for CCHD before they leave the hospital. The concept and a national protocol for screening began with a small project in 2002, was endorsed by medical associations by 2012 and required by all states in 2018. The impact of CCHD screening of newborns is remarkable. Data published in JAMA showed a 33 percent reduction in CCHD infant deaths associated with states that required CCHD screening.

The pulse oximetry screening’s impact on the number of lives saved goes beyond identifying newborns with CCHD. Worldwide, though the detection of secondary conditions, such as hypothermia, pneumonia, and sepsis, the pulse oximetry screening is estimated to save roughly 772,000 lives by 2030.

In addition to newborn screening recommendations for CCHD, a group of cardiologists, including myself, worked for the Joint Council on Congenital Heart Disease (JCCHD) to form and support the National Pediatric Cardiology Quality Improvement Collaborative (NPC-QIC). We developed measures to see how we could improve survival rates between surgeries for infants born with hypoplastic left heart syndrome (HLHS), one of the most common and severe forms of CCHD.

Babies born with HLHS require two heart surgeries within the baby’s first six months. Babies that survived the first operation had a significant mortality rate (15 percent) and frequent growth failure, while waiting for the second operation. Our focused aims were to both decrease the death rate and improve growth in these children. We analyzed data from medical centers, utilized quality improvement principals from the Institute for Health Care Improvement, talked with doctors and families, and invited teams from across the U.S. to partner with us to put quality and safety measures into place.

We emphasized the following points:

  1. Clear communication. Parents leaving the hospital received consistent messages about CHD, the type of surgery their baby had, next steps and how to care for their child at home.
  2. Improved nutrient intake. Parents received clear guidelines about how many calories babies needed to consume, were asked to weigh their baby each day, and taught how to augment feeding.
  3. Warning signs.Parents received a list of typical infant behaviors and HLHS red flags to watch out for, such as if a baby isn’t gaining a certain amount of weight. They received monitors to measure oxygen saturation levels at home. If oxygen saturation dropped significantly or if parents noticed a problem, they called their doctor immediately.

The implementation of these procedures reduced interstage mortality rates and the number of growth failures for HLHS patients. In 2008, six centers participated in the NPC-QIC pilot. By 2018, 65 medical centers in the U.S. and Canada used these methods. Similar to the pulse oximetry screening guidelines, this new method wasn’t the result of a medical breakthrough, but the result of shared learning and shared infrastructure.

Now, we’re referring more adult congenital heart patients to board-certified adult congenital heart disease (ACHD) specialists, a better fit than internists or pediatric cardiologists. Adults with congenital heart defects should have their heart examined at least once by a specialist and those with complex needs should meet with a specialist at least every two years. More than 300 board-certified ACHD specialists practice in the U.S. and the field is growing. The third ACHD board exam takes place this year.

Over the next few decades, I hope we’ll make even more progress with understanding, diagnosing and treating CHD.

Emerging research examines genetic clues for congenital heart defects, which were once thought to account for 8 percent of cases and may now account for 30 percent of conditions. We’re working with neurologists to examine the timing and pathway of potential oxygen inefficiencies that occur as the brain develops in utero, infancy, and after neonatal surgery. We’ve come a long way, but we continue looking at new frontiers and for innovative solutions.

Fortunately, as cardiologists, we’re good at fixing problems. We work with surgeons and medical teams to repair holes in hearts, or replace them, and reroute blood from an underdeveloped left ventricle to improve circulation. For almost every heart defect, we have evidence-based solutions. However, to continue to help children worldwide, it’s imperative that we don’t forget about what works well: good science, tracking data, sharing best practices, active listening, transparency and constant collaboration.

Gerard Martin, M.D., F.A.A.P., F.A.C.C., F.A.H.A., is a cardiologist and the medical director of global services at Children’s National Health System. Dr. Martin has practiced pediatric cardiology for 34 years and is the Dan G. McNamara keynote speaker at the American College of Cardiology’s 2019 Scientific Sessions. Follow Dr. Martin on Twitter @Gerard_MD.

This article first appeared on KevinMD.com.

DNA Molecule

Test your knowledge of APOL1’s role in kidney health

Nikki Gillum Posnack

What are the health effects of plastics?

Nikki Gillum Posnack

Nikki Posnack, Ph.D., assistant professor at the Children’s National Heart Institute, is an early-stage investigator examining the impact plastic chemical exposure has on the developing hearts of newborns and young children.

For newborns or children in the pediatric intensive care unit, plastic tubing is part of daily life. It delivers life-sustaining blood transfusions, liquid nutrition and air to breathe. But small amounts of the chemicals in the plastic of this tubing and other medical devices can leak into the patient’s bloodstream. The potential effects of these chemicals on the developing hearts of newborns and very young children are not well understood.

One researcher, Nikki Posnack, Ph.D., an assistant professor at the Children’s National Heart Institute, aims to change that and shares her early insights, funded by the National Center for Advancing Translation Science (NCATS), in an NCATS news feature.

“While plastics have revolutionized the medical field, we know chemicals in plastics leach into the body and may have unintended effects,” Posnack said. “The heart is sensitive to toxins, so we want to look at the effect of these plastics on the most sensitive patient population: kids who are recovering from heart surgery and already prone to cardiac complications.”

Matthew Oetgen, M.D., discusses an image of a patient’s spine.

Eliminating unnecessary radiation exposure from spinal radiography

Matthew Oetgen, M.D., discusses an image of a patient’s spine.

Chief of Orthopaedics and Sports Medicine Matthew Oetgen, M.D., discusses an image of a patient’s spine.

If a child arrives at the pediatric orthopaedic specialist for an idiopathic scoliosis evaluation without an adequate radiographic image of his or her spine, it’s often necessary to order yet another imaging study for accurate assessment.

A study published in the Journal of the American Academy of Orthopaedic Surgeons found that in a 6 month period, almost half (43 percent) of patients referred for evaluation required a repeat radiograph due to missing or poor quality existing images.

“Repeating the radiograph means these kids received another exposure to radiation, too,” says Matthew Oetgen, M.D., the study’s lead author and chief of Orthopaedic Surgery and Sports Medicine at Children’s National Health System. “It’s frustrating because in many cases, a simple change in how the initial radiograph was taken could have prevented the need for more imaging studies.”

Dr. Oetgen and the study authors note that there is currently no standardized protocol for spinal radiography of suspected idiopathic scoliosis. However, a few basic criteria could greatly reduce the number of repeat images necessary. Radiographic images that allow for proper evaluation of idiopathic scoliosis and reduce radiation exposure include:

  • A full coronal view of the spine including skull base and pelvis
  • The iliac crest as an indicator of skeletal maturity
  • A full-length lateral view of the spine

Study authors also reinforced the need to do everything possible to reduce radiation exposure for children through proper use of protective shielding for reproductive organs and digital radiograph technology.

“Orthopaedic surgeons and pediatricians share the responsibility to ensure children are exposed to as little iatrogenic radiation as possible,” Dr. Oetgen concludes. “All physicians should be sure that the radiology facilities they refer patients to for spinal radiography employ every technology and safety measure available to limit radiation exposure. Additionally, we can and should work with radiologists to define evaluation criteria and improve what’s captured by radiography on the first try.”

Sangeeta Sule

Sangeeta Dileep Sule, M.D., Ph.D., joins Children’s National as Chief of Rheumatology

Sangeeta Sule

Sangeeta Dileep Sule, M.D., Ph.D., the new chief of rheumatology at Children’s National Health System, shares a similar vision and mission of all pediatric specialists: treat the whole child. Dr. Sule looks forward to supporting and expanding the Division of Rheumatology, while introducing clinics tailored for specific diseases – such as arthritis and lupus.

Dr. Sule has focused her clinical expertise and research efforts on providing multidisciplinary resources to pediatric patients diagnosed with rheumatologic disease. From integrating nephrology appointments into lupus clinics and exercise prescriptions into juvenile arthritis treatments, Dr. Sule is passionate about fusing the latest research into routine practice. For example, kidney disease affects up to 80 percent of children with lupus so it’s important for rheumatologists to work with nephrologists to conduct blood screenings and early detections, while practicing aggressive interventions, such as monitoring a child every one to three months following a diagnosis.

For arthritis, biological therapies are beneficial for treating targeted sections of the immune system where the disease manifests. Other therapies, such as exercise, can boost a child’s overall quality of life. Some patients may also need additional counseling to navigate weekly injections.

“The amount of innovation happening in pediatric health care is exciting,” says Dr. Sule, who mentors and teaches pediatric rheumatology residents and fellows. “Investing in pediatric rheumatology is critical. For example, children metabolize medicine differently than adults and this is one reason why they need extra support and care.”

Dr. Sule started her career as a pediatric rheumatologist by studying cellular and molecular biology at Tulane University. She attended medical school at Baylor College of Medicine and completed a doctorate in philosophy in clinical investigation from Johns Hopkins University School of Public Health. After completing a postdoctoral fellowship in rheumatology at Johns Hopkins University School of Medicine, Dr. Sule taught courses in pediatrics and internal medicine while practicing in the field.

Dr. Sule merged her research and clinical interests to support specialty clinics as the director of the Pediatric Lupus Center and of the Pediatric Arthritis Center within the Division of Pediatric Rheumatology at Johns Hopkins University School of Medicine.

She has published more than 24 peer-reviewed studies, and is certified by the American Board of Internal Medicine, with a sub-board certification of Rheumatology, and by the American Board of Pediatrics.

Preemie Baby

Getting micro-preemie growth trends on track

Preemie Baby

According to Children’s research presented during the Institute for Healthcare Improvement 2018 Scientific Symposium, standardizing feeding practices – including the timing for fortifying breast milk and formula with essential elements like zinc and protein – improves growth trends for the tiniest preterm infants.

About 1 in 10 infants is born before 37 weeks gestation. These premature babies have a variety of increased health risks, including deadly infections and poor lung function.

Emerging research suggests that getting their length and weight back on track could help. According to Children’s research presented during the Institute for Healthcare Improvement 2018 Scientific Symposium, standardizing feeding practices – including the timing for fortifying breast milk and formula with essential elements like zinc and protein – improves growth trends for the tiniest preterm infants.

The quality-improvement project at Children’s National Health System targeted very low birth weight infants, who weigh less than 3.3 pounds (1,500 grams) at birth. These fragile infants are born well before their internal organs, lungs, brain or their digestive systems have fully developed and are at high risk for ongoing nutritional challenges, health conditions like necrotizing enterocolitis (NEC) and overall poor development.

The research team measured progress by tracking the micro-preemies’ mean delta weight Z-score for weight gain, which measures nutritional status.

“In this cohort, mean delta weight Z-scores improved by 43 percent, rising from -1.8 to the goal of -1.0, when we employed an array of interventions. We saw the greatest improvement, 64 percent, among preterm infants who had been born between 26 to 28 weeks gestation,” says Michelande Ridoré, MS, Children’s NICU quality-improvement program lead who presented the group’s preliminary findings. “It’s very encouraging to see improved growth trends just six months after introducing these targeted interventions and to maintain these improvements for 16 months.”

Within Children’s neonatal intensive care unit (NICU), micro-preemies live in an environment that mimics the womb, with dimmed lighting and warmed incubators covered by blankets to muffle extraneous noise. The multidisciplinary team relied on a number of interventions to improve micro-preemies’ long-term nutritional outcomes, including:

  • Reducing variations in how individual NICU health care providers approach feeding practices
  • Fortifying breast milk (and formula when breast milk was not available), which helps these extra lean newborns add muscle and strengthen bones
  • Early initiation of nutrition that passes through the intestine (enteral feeds)
  • Re-educating all members of the infants’ care teams about the importance of standardized feeding and
  • Providing a decision aid about feeding intolerance.

Dietitians were included in the daily rounds, during which the multidisciplinary team discusses each infant’s care plan at their room, and used traffic light colors to describe how micro-preemies were progressing with their nutritional goals. It’s common for these newborns to lose weight in the first few days of life.

  • Infants in the “green” zone had regained their birth weight by day 14 of life and possible interventions included adjusting how many calories and protein they consumed daily to reflect their new weight.
  • Infants in the “yellow” zone between day 15 to 18 of life remained lighter than what they weighed at birth and were trending toward lower delta Z-scores. In addition to assessing the infant’s risk factors, the team could increase calories consumed per day and add fortification, among other possible interventions.
  • Infants in the “red” zone remained below their birth weight after day 19 of life and recorded depressed delta Z-scores. These infants saw the most intensive interventions, which could include conversations with the neonatologist and R.N. to discuss strategies to reverse the infant’s failure to grow.

Future research will explore how the nutritional interventions impact newborns with NEC, a condition characterized by death of tissue in the intestine. These infants face significant challenges gaining length and weight.

Institute for Healthcare Improvement 2018 Scientific Symposium presentation

  • “Improved growth of very low birthweight infants in the neonatal intensive care unit.”

Caitlin Forsythe, MS, BSN, RNC-NIC, NICU clinical program coordinator, Neonatology, and lead author; Michelande Ridoré, MS, NICU quality-improvement program lead; Victoria Catalano Snelgrove, RDN, LD, CNSC, CLC, pediatric clinical dietitian; Rebecca Vander Veer, RD, LD, CNSC, CLC, pediatric clinical dietitian; Erin Fauer, RDN, LD, CNSC, CLC, pediatric clinical dietitian; Judith Campbell, RNC, IBCLC, NICU lactation consultant; Eresha Bluth, MHA, project administrator; Anna Penn, M.D., Ph.D., neonatologist; Lamia Soghier, M.D., MEd, Medical Unit Director, Neonatal Intensive Care Unit; and Mary Revenis, M.D., NICU medical lead on nutrition and senior author; all of Children’s National Health System.

Photo of nurses in the cardiac intensive care unit at Children's National

Can pyruvate support metabolic function following heart surgery?

Photo of nurses in the cardiac intensive care unit at Children's National

Nurses rush a child to the cardiac intensive care unit at Children’s National Health System.

Can pyruvate, the end product of glycolysis, help improve cardiovascular function in children who have cardiopulmonary bypass surgery and suffer from low cardiac output syndrome (LCOS)? This question is one that Rafael Jaimes, Ph.D., a staff scientist at Children’s National Heart Institute, a division of Children’s National Health System, is studying, thanks to a two-year grant from the American Heart Association.

The competitive grant awards Dr. Jaimes with $110,000 to study how pyruvate may help improve cardiac output among pediatric patients with LCOS. The compound aims to stimulate metabolic function, now treated by inotropic agents, such as dobutamine and milrinone. These agents ensure optimal delivery of oxygen from the heart to the brain, as well as to other organs in the body, following heart surgery. While these agents help patients with cardiac dysfunction, there is still a critical need for safe and effective therapies.

“If there’s any detriment in cardiac output, the heart’s function begins to degrade,” explains Dr. Jaimes. “You see a downward spiral effect with reduced cardiac output because the heart is dependent on its own perfusion. It needs to pump blood throughout the body to survive.”

This is where the pyruvate study, and the grant, will be applied: Can pyruvate target the essential muscle of the heart and reverse this cardiac destabilization – and as soon as possible?

“By increasing the metabolic output of the heart’s local muscle, cardiac output increases,” Dr. Jaimes explains. “That’s going to lead to better recovery.”

Better recovery could be measured by how fast a child recovers from heart surgery as well as how much time they spend in the hospital, clinically referred to as throughput. A faster recovery could also influence a child’s quality of life and reduce overall health care costs.

Based on preliminary data that shows pyruvate improves cardiac function in experimental models after ischemic insult, which is what happens when pediatric patients undergo cardiac surgery, Dr. Jaimes believes the results will likely replicate themselves in his preclinical models.

To start, he’ll test pyruvate using 100 blood samples and discarded tissue from patients. The blood samples will be tested for metabolic markers, including measured pyruvate levels.

Part of what encouraged Dr. Jaimes to study how this compound could complement or replace standard therapies was the encouragement he received from his mentors in the field.

“Nobody has looked into using pyruvate for almost 30 years,” says Dr. Jaimes. “It’s not commercially favorable, there’s no patent on it, it doesn’t have a lot of marketability and there are no financial incentives, so it’s been put aside.”

As part of a discussion with cardiologists at a medical conference in Washington, Dr. Jaimes brought up the idea of using pyruvate for pediatric heart surgeries and received positive feedback.

“Once everyone’s eyes lit up, I knew I was on to something,” says Dr. Jaimes about the encouragement he received to pursue this study.

“You put lactate and glucose in your IV solutions,” adds Dr. Jaimes. “Pyruvate is an essential nutrient. It’s almost an essential sugar so there’s no reason not to put it in. If these cardiologists are intrigued by the project, maybe the American Heart Association will be, too.”

In addition to funding the study, which could support future research about how metabolic makers in the blood can be stimulated to fast-track recovery following heart surgery, the American Heart Association grant is specific to pediatric health outcomes.

“The current state of pharmaceutical treatment for patients recovering from cardiac surgery is designed and created for adults,” says Dr. Jaimes. “From our research in pediatrics, we know that children aren’t small adults.”

Dr. Jaimes explains that children are different on an anatomical and physiological level. Their cells even look and function different, compared to adult cells, because they haven’t matured yet.

While congenital heart defects are rare, they affect 1 percent, or 40,000 births worldwide, they often require multiple surgeries throughout a child’s lifespan. LCOS impacts 25 percent of patients following cardiopulmonary bypass and the timing of treatment is important. In severe cases, insufficient cardiac output following surgery could impact a child’s long-term development, ranging from reasoning, learning, attention and executive function, to developing age-appropriate language and social skills.

“The metabolic insufficiencies I’m looking at, which may help improve the muscle function of the heart, are just one piece of a bigger puzzle in pediatric cardiology,” notes Dr. Jaimes about ongoing research at Children’s National Heart Institute. “We already know pyruvate is safe. We just have to see if it’s effective in supporting a patient’s recovery in the intensive care unit.”

Dr. Jaimes will work with his research mentor Nikki Posnack, Ph.D., assistant professor at the Children’s National Heart Institute, on this preclinical study throughout the grant’s lifecycle, which starts in early January 2019 and ends in late December 2020.

Top Children’s Hospital logo

The Leapfrog Group names Children’s National Health System a 2018 “Top Children’s Hospital”

Top Children’s Hospital logo

On December 4, 2018, Children’s National Health System was named a 2018 “Top Children’s Hospital” by The Leapfrog Group, an independent watchdog organization. The highest performing hospitals on the Leapfrog Hospital Survey are recognized annually for this prestigious award. Children’s National is proud to have been recognized 10 times as “Top Children’s Hospital.” Top hospitals are given the designation for having distinct achievements in patient safety and quality, including lower infection rates, decreased length of stay, fewer readmissions and higher survival rates for high-risk procedures.

“We’re particularly proud of this acknowledgment as it recognizes our unwavering commitment to high-quality patient care,” said Kurt Newman, M.D., president and CEO of Children’s National. “It highlights our exceptional clinicians and support teams who work to constantly improve the safety of the care we provide.”

The Top Children’s Hospitals are selected based on The Leapfrog Group’s annual survey of nearly 1,900 hospitals across the country which measures hospitals’ performance in many areas of hospital care, including reduced medication rates and capacity to prevent medication errors.

“Being acknowledged as a Top Children’s Hospital is an incredible feat achieved by less than six percent of eligible hospitals nationwide,” said Leah Binder, president and CEO of The Leapfrog Group. “With this honor, Children’s National has established its commitment to safer and higher quality care. Providing this level of care to patients in Washington, D.C. requires motivation and drive from every team member. I congratulate the board, staff and clinicians, whose efforts made this honor possible.”

QUILT conference

Children’s National hosts Quality Improvement Leadership Training Course

QUILT conference

In October 2018, Children’s National hosted 20 neonatologists from 15 hospitals in China for a 10 day Quality Improvement Leadership Training Course focused on quality improvement principles and methodology. The course also featured presentations on hospital-wide quality improvement work and included speakers from the Quality & Safety Department, Nursing Quality, and the Neonatal Intensive Care Unit (NICU). The Performance Improvement team worked with the attendees on their own projects, such as reducing antibiotic use and increasing family-centered care in the NICU. The attendees then presented at the end of the course to their colleagues, as well as to five hospital presidents visiting from China.

Matthew Oetgen examines a patient

Surgical home program for spinal fusion achieves long-term success

Matthew Oetgen examines a patient

“Our primary goal was to improve the value of care for children with scoliosis and their families,” says Dr. Oetgen. “Even better, we’ve shown that this model can be used consistently over time to maintain the benefits it delivers to this patient population.”

“Creating an effective process that benefits patients, is sustainable long term and doesn’t increase costs is one of the most challenging parts of any new procedure, both in health care and beyond,” says Matt Oetgen, M.D., chief of Orthopaedic Surgery and Sports Medicine at Children’s National.

Dr. Oetgen’s team accomplished this feat when building the Children’s National Spinal Fusion Surgical Home. The team used LEAN process mapping at the outset to engage a broad group of care providers who established a collaborative environment that empowered and engaged everyone to take ownership over a new care pathway for every patient who undergoes posterior spinal fusion surgery at the hospital.

This unique model designed using proven business process development tools has allowed patients require fewer pain medications after surgery and have shorter stays in the hospital. Even better, the team has maintained the integrity of the pathway consistently over a longer period of time than any other pediatric spinal fusion care model to date.

“Our primary goal was to improve the value of care for children with scoliosis and their families,” says Dr. Oetgen, who was the study’s lead author. “Even better, we’ve shown that this model can be used consistently over time to maintain the benefits it delivers to this patient population.”

The team conducted a retrospective analysis of prospective data from all patients (213) undergoing posterior spinal fusion at Children’s National Health System from 2014 to 2017, a period of time that captures nearly one year  before implementation of the new pathway and 2.5 years after implementation. The outcomes were reported in the Journal of Bone and Joint Surgery.

As pressure builds to increase the value of care, many hospital systems are trying standardized care pathways for many complex conditions, in an effort to decrease care variability, improve outcomes and decrease cost. Previous research has shown the effectiveness of a variety of standardized pathways with wide ranging goals for spinal fusion procedures, however, most published studies have focused only on the initial success of these pathways. This study is the first to look at the implementation over a period of 2.5 years to gauge whether the process and its effectiveness could be maintained long term.

The authors attribute physician buy-in across disciplines and strict adherence to pathway processes as key to the success of this model. In addition, the team created standardized educational procedures for onboarding new care providers and implemented standardized electronic order sets for both orthopaedic and anesthesia services to make the pathway easy to maintain with little deviation over time. Lean process mapping at the outset included a broad group of care providers who established a collaborative environment that empowered and engaged the entire team to take ownership over the new process.

“We used proven business models for culture change that were critical to the success of this program,” Dr. Oetgen says. “We’re proud of the model we have created and think it would work well in other pediatric hospitals with similar patient populations.”

Rebecca Cady

Rebecca Cady named Health Care Risk Management Professional of the Year by ASHRM

Rebecca Cady

Rebecca Cady, vice president and chief risk officer at Children’s National Health System, is the recipient of the 2018 American Society for Health Care Risk Management (ASHRM ) Professional of the Year award. Cady’s dedication to advancing the risk management industry has been demonstrated through her commitment to innovation in the face of industry challenges.

Recently, she directed the implementation process for reporting safety incidents via a mobile app created by RL Solutions. Children’s National was one of the early adopters of the mobile app technology that has allowed staff to instantaneously provide feedback about unsafe conditions directly from their smartphones.

“The mobile app enables staff to report an event quickly so that it doesn’t get lost or forgotten and something can be done about it,” Cady says. “We already were working on a project to increase incident reporting, so integrating the mobile app was an important addition for providers looking to report issues of concern.”

Cady is in charge of the Children’s National enterprise risk management department and management of their litigation program; including serving as counsel to the compliance officer and Human Resources. She also oversees operations of Children’s clinical risk management program, ombudsman program, workers’ compensation program and the insurance program; including managing the organization’s captive insurance company.

Her approach consistently applies diverse tools and strategies of risk management, such as enterprise risk management (ERM), strategic risk management, risk financing and insurance. At Children’s National, she has built an ERM infrastructure that enables faster understanding of risk management and adoption by staff at all levels. This led to adopting incident reporting on mobile devices and resulted in overall improvements in hospital performance.

Previously, Cady served as interim vice president, chief compliance and privacy officer at Children’s National. Prior to joining Children’s National, she was a partner at Grace Hollis Lowe Hanson & Schaeffer LLP.

Stephen Teach does an asthma exam

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

Stephen Teach does an asthma exam

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

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

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

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

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

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

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

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

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

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