Handrawn illustration of human Kidneys

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Presenter: Kaushal Amatya, Ph.D., psychologist

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

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

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


Marva Moxey Mims

Tackling bias – the power of one

Marva Moxey MimsIn the most recent edition of the American Society of Pediatric Nephrology’s Kidney Notes, Children’s National Hospital Chief of Nephrology, Marva Moxey-Mims, M.D., wrote a perspective piece asking other providers to join her in an effort to see patients as a whole person and try to put aside personal biases, thereby improving overall patient care.

In this personal commentary, Dr. Moxey-Mims reflects on challenging herself to better serve patients by making sure they feel seen and to understand them. “Just think of the ripple effect if we can do this with even a fraction of our patients,” said Dr. Moxey-Mims. “The goodwill that patients will feel knowing that we are trying to see them is immeasurable.”

You can read the entire article “Tackling Bias – The Power of One” here.

kidneys with science images

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

kidneys with science images

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

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

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

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

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

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

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

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

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

urine sample

Approaches to nephrotic syndrome and proteinuria

urine sample

Nephrologist Melissa Meyers, M.D., recently gave a Lunch and Learn presentation at Children’s National titled, “Urine the Know: General Pediatricians’ Approach to Nephrotic Syndrome and Proteinuria.”

Melissa Meyers, M.D., a nephrologist at Children’s National Hospital, recently gave a Lunch and Learn presentation at Children’s National titled, “Urine the Know: General Pediatricians’ Approach to Nephrotic Syndrome and Proteinuria.”

During the virtual presentation, Dr. Meyers detailed the definitions of proteinuria and nephrotic syndrome and discussed methods used to screen, test and manage the disorders.

She ended with a question-and-answer segment, which included a series of scenarios pediatricians may encounter while diagnosing and treating patients with the conditions.

The full presentation can be viewed here.

Dr. Meyers is a pediatric nephrologist with 10 years’ experience caring for children. She is passionate about kidney health and providing comprehensive medical care to her patients and their families.

colored illustration of kidney x-ray

Partnership with CMS and HRSA addresses national kidney shortage

colored illustration of kidney x-ray

Children’s National Hospital is proud to announce that it is participating in the Centers for Medicare & Medicaid Services (CMS) and Health Resources & Services Administration (HRSA)’s new End-Stage Renal Disease Treatment Choices Learning Collaborative (ETCLC). This effort will focus on addressing kidney disease prevention and treatment, including improved access to kidney transplants in the United States.

The ETCLC will engage transplant centers, Organ Procurement Organizations (OPOs), large donor hospitals, patients and donor family members to identify highly effective practices currently in use and spread the use of these practices throughout the organ procurement, kidney care and kidney transplant community to achieve the following three AIMs:

  • AIM #1: Increase the number of deceased donor kidneys transplanted
  • AIM #2: Decrease the current national discard rate of all procured kidneys
  • AIM #3: Increase the percentage of change for kidneys recovered for transplant in the 60-85 Kidney Donor Profile Index score group

The ETCLC brings the potential for collaboration, communication and innovation across geography into reality. By participating in the ETCLC, Children’s National will benefit by:

  • the creation of efficiencies and reduction of duplicative efforts in kidney patient care
  • exposure to new, innovative ideas regarding the kidney transplant process
  • the enhancement of communication and relationship building within the kidney care community
  • the application of substantive changes to improve the donation and transplantation system
cystic kidney disease

American Heart Association grant funds study of vascular complications in ADPKD

cystic kidney disease

Ashima Gulati, M.D., Ph.D., pediatric nephrologist at Children’s National Hospital recently was awarded a grant from the American Heart Association. Dr. Gulati’s work will aim to identify the genetic determinants of vascular complications in autosomal dominant polycystic kidney disease (ADPKD).

Cerebrovascular complications such as vascular aneurysms and anomalies are an important cause of morbidity in ADPKD that need to be studied. The goal of Dr. Gulati’s research is to contribute to knowledge towards using molecular medicine to inform individual genetic risk of clinically significant vascular complications in ADPKD.

This work addresses a clinically significant vascular complication in ADPKD, the most common inherited form of kidney failure world-wide.

Kristen Sgambat, Ph.D., and Asha Moudgil, M.D.

Kristen Sgambat, Ph.D., R.D. and Asha Moudgil, M.D. receive Editors’ Choice Award

Kristen Sgambat, Ph.D., and Asha Moudgil, M.D.

Children’s National Hospital researchers Kristen Sgambat, Ph.D., and Asha Moudgil, M.D., were presented with the 2021 AJKD Editors’ Choice Award.

The American Journal of Kidney Disease (AJKD) announced the selection of the 2021 AJKD Editors’ Choice Award, recognizing outstanding articles published in their journal this year.

Children’s National Hospital researchers Kristen Sgambat, Ph.D., and Asha Moudgil, M.D., were presented with the 2021 AJKD Editors’ Choice Award for their July 2021 study, Social determinants of cardiovascular health in African American children with chronic kidney disease: An analysis of the chronic kidney disease in children (CKiD).

The study is the first to investigate the relationship between race, socioeconomic factors and cardiovascular health in children with chronic kidney disease. Dr. Sgambat, Dr. Moudgil and their collaborators found that African American children with chronic kidney disease had increased evidence of socioeconomic challenges, including food insecurity, reliance on public insurance, lower household incomes and lower levels of maternal education. These children had worse cardiovascular outcomes than Caucasian children with the same chronic kidney conditions. Notably, the cardiovascular outcomes of the two groups became more alike when statistical analysis was applied to equalize their socioeconomic factors. This suggests that these socioeconomic indicators do play a role in adverse cardiovascular health outcomes observed among African American children with chronic kidney disease.

“The findings of this study are important because they highlight the urgent need to shift the clinical research paradigm to investigate how social, rather than biological, factors contribute to racial differences in health outcomes,” said Dr. Sgambat. “Future studies should focus on the impact of systemic racism on cardiovascular health among children with chronic kidney disease, an area not well-studied so far.”

Denver Brown

New grant to conduct single center pilot trial of alkali therapy in children with CKD

Denver Brown

Denver D. Brown, M.D., recipient of the Child Health Research Career Development Award.

Linear growth (i.e., height) impairment is commonly observed in children with chronic kidney disease (CKD). Several studies have suggested metabolic acidosis, a frequent consequence of mild to moderate CKD in children, as a contributing factor to linear growth failure in these patients. Grant awardee Denver D. Brown, M.D., aims to conduct a pilot trial in children with mild metabolic acidosis and CKD, comparing differences in linear growth between an observation period versus a period of supplementation with alkali therapy (i.e., treatment for metabolic acidosis).

“This grant is so important because there has never been a clinical trial of alkali therapy in children with CKD despite its frequent use in this population” says Dr. Brown. “This research has the potential to better inform treatment practices with the aim of improving the care of our young, vulnerable patients.”

The Child Health Research Career Development Award (CHRCDA) of $125,000 will support Dr. Brown in her efforts to carry out this pilot trial.

“Funding for this pilot study could lay the groundwork for a large, randomized controlled clinical trial, which would help fill a major gap in knowledge as to the precise benefits of alkali therapy, especially regarding growth in children with impaired kidney function.”

colored x-ray showing kidneys and spine

New report advances improved way to diagnose kidney disease

colored x-ray showing kidneys and spine

The findings outline a new race-free approach to diagnose kidney disease, recommending the adoption of the new eGFR 2021 CKD EPI creatinine equation.

Patients with kidney disease will benefit from an improved approach, according to a new report.

The findings outline a new race-free approach to diagnose kidney disease, recommending the adoption of the new eGFR 2021 CKD EPI creatinine equation. This calculation estimates kidney function without a race variable. The report also recommends increased use of cystatin C combined with serum creatinine as a confirmatory assessment of eGFR or kidney function.

The effort is being spearheaded by a team of national nephrology experts that includes Marva Moxey-Mims, M.D., chief of the Division of Nephrology at Children’s National Hospital.

“This final report is important in recommending a uniform approach to the calculation of eGFR without the inclusion of race,” Dr. Moxey-Mims says. “This will avoid a piecemeal approach where eGFR is calculated differently at different health care facilities, potentially causing confusion.”

The final report, published in the American Journal of Kidney Diseases and the Journal of the American Society of Nephrology, was drafted with considerable input from hundreds of patients, family members, medical students, clinicians, scientists, health professionals and other stakeholders. This will help achieve consensus for an unbiased and most reasonably accurate estimation of GFR so that laboratories, clinicians, patients and public health officials can make informed decisions to ensure equity and personalized care for patients with kidney diseases.

“Patients, professionals and other stakeholders can have confidence in this estimate that is relying solely on biologic measures. Hopefully, these can evolve even further as the science progresses,” Dr. Moxey-Mims says. “My hope is that health systems and labs will adopt these changes expeditiously.”

US News badges

For fifth year in a row, Children’s National Hospital nationally ranked a top 10 children’s hospital

US News badges

Children’s National Hospital in Washington, D.C., was ranked in the top 10 nationally in the U.S. News & World Report 2021-22 Best Children’s Hospitals annual rankings. This marks the fifth straight year Children’s National has made the Honor Roll list, which ranks the top 10 children’s hospitals nationwide. In addition, its neonatology program, which provides newborn intensive care, ranked No.1 among all children’s hospitals for the fifth year in a row.

For the eleventh straight year, Children’s National also ranked in all 10 specialty services, with seven specialties ranked in the top 10.

“It is always spectacular to be named one of the nation’s best children’s hospitals, but this year more than ever,” says Kurt Newman, M.D., president and CEO of Children’s National. “Every member of our organization helped us achieve this level of excellence, and they did it while sacrificing so much in order to help our country respond to and recover from the COVID-19 pandemic.”

“When choosing a hospital for a sick child, many parents want specialized expertise, convenience and caring medical professionals,” said Ben Harder, chief of health analysis and managing editor at U.S. News. “The Best Children’s Hospitals rankings have always highlighted hospitals that excel in specialized care. As the pandemic continues to affect travel, finding high-quality care close to home has never been more important.”

The annual rankings are the most comprehensive source of quality-related information on U.S. pediatric hospitals. The rankings recognize the nation’s top 50 pediatric hospitals based on a scoring system developed by U.S. News. The top 10 scorers are awarded a distinction called the Honor Roll.

The bulk of the score for each specialty service is based on quality and outcomes data. The process includes a survey of relevant specialists across the country, who are asked to list hospitals they believe provide the best care for patients with the most complex conditions.

Below are links to the seven Children’s National specialty services that U.S. News ranked in the top 10 nationally:

The other three specialties ranked among the top 50 were cardiology and heart surgerygastroenterology and gastro-intestinal surgery, and urology.

plate of food

Looking back one year later – Keeping it Renal: Global Cuisine for Kids

plate of food

The cookbook introduces a variety of culturally diverse kidney-friendly recipes that kids of all ages love.

It has been one year since the Children’s National Hospital Department of Nephrology released their cookbook “Keeping it Renal: Global Cuisine for Kids” and we are still receiving requests for this collection of recipes. In order to stay healthy, most children with kidney disease have to limit or avoid foods that are high in certain minerals including sodium, potassium and phosphorus. “Children on dialysis have to give up a lot of what they like to eat. This cookbook introduces a variety of culturally diverse kidney-friendly recipes that kids of all ages love. By learning to cook these recipes, our patients can take an active role in their own healthcare and learn some fun new skills,” said Kristen Sgambat, Ph.D., R.D., and Asha Moudgil, M.D., medical director of transplant.

It is often challenging for children and their families to balance these dietary restrictions with proper nutrition and enjoyable mealtimes. “This cookbook offers novel and exciting recipes that patients and families may not be aware of. Seeing these options can help patients see that a renal diet does not have to be bland or repetitive and thus improve patients’ outlook on treatment and motivate them to adhere to the dietary restrictions,” said Kaushalendra Amatya, Ph.D., pediatric psychologist for Nephrology and Cardiology at Children’s National.

As an innovative way to facilitate adherence to these limitations, our nephrology department collaborated with our patient families to create the cookbook “Keeping it Renal: Global Cuisine for Kids,” a compilation of their favorite kidney-friendly recipes.

Children’s National is one of the top pediatric hospitals in NIH funding, and our nephrology program ranks number 7 in the country, according to U.S. News & World Report. The Kidney Transplantation Program is the only one of its kind in the Washington, D.C., area focused on the needs of children and teens with kidney disease. Committed to providing the best quality care to all of our pediatric dialysis and transplant patients, we are always looking for new ways to support our patient families.

If you would like to receive a copy of the Keeping it Renal: Global Cuisine for Kids cookbook, please send your request to:


Denver Brown, M.D., and Celina Brunson, M.D.

Children’s National expands its nationally ranked nephrology division

Children’s National Hospital has added five physicians to its nationally ranked Nephrology Division. Denver Brown, M.D., Celina Brunson, M.D., Ashima Gulati, M.D., Melissa Meyers, M.D., Catherine Park, M.D., all have joined the department over a span of the past two years.

“These physicians are incredible additions to our nephrology division,” said Marva Moxey-Mims, M.D., chief of the Division of Nephrology at Children’s National. “We are excited about the expertise these young physicians already contribute to our division and can’t wait to share more as we continue to expand our efforts as one of the top nephrology programs in the country. We are elated to have them on our team.”

Each of the new faculty members has specific areas of clinical and research interests. Dr. Brown’s focus is chronic kidney disease (CKD) and the impact of acidosis on growth and disease progression. Dr. Brunson’s interest is dialysis, health disparities and social determinants of health for children with CKD. She is a JELF Advocacy scholar through the American Society of Nephrology. Dr. Gulati is an expert in inherited kidney diseases with a particular focus on polycystic kidney disease for which she has external grant support. Dr. Meyers’ interest is kidney transplantation and Dr. Park, our newest addition, is interested in systemic inflammatory diseases, especially lupus nephritis.

The nephrology team will continue to provide comprehensive inpatient and outpatient care to children throughout the full spectrum of kidney diseases.

Denver Brown, M.D., Celina Brunson, M.D., Ashima Gulati, M.D., Melissa Meyers, M.D., Catherine Park, M.D.

Denver Brown, M.D., Celina Brunson, M.D., Ashima Gulati, M.D., Melissa Meyers, M.D. and Catherine Park, M.D., recently joined the nephrology division.

light micrograph of wilms tumor

Evolution of risk stratification for Wilms tumor

light micrograph of wilms tumor

Light micrograph of Wilms tumor.

Wilms tumor is a rare kidney cancer that primarily affects children. Also known as nephroblastoma, it is the most common malignant renal tumor in children. Advances in the treatment of Wilms tumor are some of the great achievements in the field of oncology, improving survival to 90% and decreasing the burden of therapy.

A key factor in the success of Wilms tumor treatment has been improved risk stratification, enabling augmentation or reduction of therapy depending on a patient’s risk of relapse. In a review article in Current Opinion in Pediatrics, Jeffrey Dome, M.D., Ph.D., vice president of the Center for Cancer and Blood Disorders at Children’s National Hospital, Marie V. Nelson, M.D., assistant professor of pediatrics in the Division of Oncology, and their colleagues look at the evolution of clinical and biological factors that have been adopted for Wilms tumor.

The authors found that the original National Wilms Tumor Study Group (NWTSG) and International Society of Pediatric Oncology (SIOP) studies relied solely on tumor stage to define treatment. Over time, however, additional factors were incorporated into the risk stratification schema, allowing for a multifactorial precision medicine approach.

The authors conclude that “the application of new clinical and biological prognostic factors has created unprecedented ability to tailor therapy for Wilms tumor, accompanied with improved outcomes. Current and future trials will continue to enhance precision medicine for Wilms tumor.”

Read the full study in Current Opinion in Pediatrics.

Pediatric Transplantation Journal Cover

Special issue of Pediatric Transplantation features Children’s National experts

Pediatric Transplantation Journal Cover

While much has been written about advances in the field of pediatric transplantation, there have been relatively few publications that address the social, psychological and day‐to‐day struggles faced by pediatric transplant recipients and their families. A special February 2021 issue of the journal Pediatric Transplantation, guest edited by Children’s National Hospital nephrologist and medical director of transplant Asha Moudgil, M.D., features a compilation of articles from a diverse group of professionals who share their expertise on topics related to healthy living for pediatric solid organ transplant patients. Among these leaders in their fields are several clinicians from Children’s National, including Jonathan Albert, M.D. (Infectious Diseases fellow), Benjamin Hanisch, M.D. (Transplant Infectious Diseases), Kristen Sgambat, Ph.D., R.D. (Renal Dietician), Melissa R. Meyers, M.D. (Nephrologist) and Kaushalendra Amatya, Ph.D. (Psychologist).

In an editorial co-written with Priya Verghese, M.D., of Ann & Robert H. Lurie Children’s Hospital of Chicago, Dr. Moudgil writes, “It is widely acknowledged by those practicing in the field of transplant medicine that taking care of pediatric transplant recipients is a complex endeavor for all parties involved, including patients, families, and providers. In this compendium, we bring you expertise from a diverse group of professionals — including physicians, psychologists, social workers, and nutritionists. These authors provide a concise summary of the literature and evidence when available, and offer personal insight where there is paucity of literature in topics related to healthy living in pediatric transplantation.”

Dr. Albert, Dr. Hanisch and Sgambat provide their expertise in an article titled “Approaches to safe living and diet after solid organ transplantation,” which reviews the risks that pediatric and adolescent solid organ transplant recipients encounter through exposures such as household contacts, outdoor activities, travel, animal exposures and dietary choices.

Like their peers, transplant recipients go through challenges of sexual development, but are at greater risk for sexually transmitted diseases due to their chronic immunosuppression. To address this need, Dr. Meyers and colleagues provide an introductory sexual preventive care resource for adolescent and young adult solid organ transplant recipients in their article “Promoting safe sexual practices and sexual health maintenance in pediatric and young adult solid organ transplant recipients.

And, in an article titled “Psychological functioning and psychosocial issues in pediatric kidney transplant recipients,” Dr. Amatya and colleagues analyze psychological and psychosocial factors related to medical outcomes and overall well‐being post‐transplant.

Pediatric Transplantation articles written by experts from Children’s National in the 2021 February issue:

Asha Moudgil examines patient

Social determinants of cardiovascular health in African American children with CKD

Asha Moudgil examines patient

In a recent study, Asha Moudgil, M.D., and colleagues looked at differences in socioeconomic factors and subclinical cardiovascular disease markers by race in chronic kidney disease patients.

Children with chronic kidney disease (CKD) are known to have an increased risk for cardiovascular (CV) disease. African American children with CKD are also disproportionately affected by socioeconomic disadvantages related to systemic racism.

In a recent analysis of 3,103 visits from 628 children enrolled in the Chronic Kidney Disease in Children (CKiD) study, Children’s National Hospital researchers Kristen Sgambat, Ph.D., and Asha Moudgil, M.D., and their colleagues found that African American children with CKD had increased left ventricular mass index, more ambulatory hypertension and differences in lipid profile compared with Caucasian children. After adjusting for socioeconomic factors (public health insurance, household income, maternal education, food insecurity, abnormal birth history), a trend towards attenuation of the differences in these CV markers was observed.

The authors of the study conclude that, “as many social determinants of health were not captured by our study, future research should examine effects of systemic racism on CV health in this population.”

Read the full study in the American Journal of Kidney Diseases.

close up of an IV bag

Carnitine may improve heart function in children receiving CRRT

close up of an IV bag

A first-of-its-kind study demonstrated that IV carnitine supplementation is associated with improvement in myocardial strain and repletion of plasma total and free carnitine in children with AKI receiving CRRT.

Supplementation of a special nutrient could help improve heart function in children receiving continuous dialysis in critical care units. The nutrient carnitine plays an essential role in producing energy for use by heart and skeletal muscles. Critically ill children with acute kidney dysfunction often need a continuous dialysis therapy (also known as CRRT, continuous renal replacement therapy) to help remove toxins while kidneys are not working. An unintended consequence of this CRRT is removal of carnitine. Often these critically ill children are unable to eat by mouth and therefore can’t receive carnitine unless it is supplemented. Children’s National Hospital researchers have proven that intravenous carnitine supplementation is associated with repletion of the body’s carnitine supply and may cause improvement in heart function as shown by heart strain analysis (which detects subclinical cardiac dysfunction that may not be apparent by traditional echocardiography).

In a first-of-its-kind study, the Children’s National researchers, Asha Moudgil, M.D., Kristen Sgambat, M.D., and Sarah Clauss, M.D., investigated carnitine deficiency in children receiving CRRT. They demonstrated for the first time that these children become severely deficient in carnitine after being on CRRT for >1 week, and that carnitine supplementation is associated with carnitine repletion and improved heart function. This knowledge can help to guide clinical care, as carnitine can be easily added to the IV nutritional formulations that are typically given to these patients.

Although little was previously known about carnitine status in patients with acute kidney injury (AKI) receiving CRRT, iatrogenic carnitine deficiency related to chronic hemodialysis (HD) in patients with end stage renal disease is a well-known phenomenon. It was theorized that given the continuous removal of solutes by CRRT in combination with lack of dietary intake and impaired production of endogenous carnitine by the kidney in critically ill children with AKI, carnitine would be rapidly depleted.

The latest controlled pilot study (NCT01941823) of 48 children hypothesized that carnitine supplementation would improve left ventricular function in children receiving CRRT. Children ages 1-21 years with AKI requiring CRRT, who were admitted to the pediatric intensive care unit at Children’s National Hospital from 2015 to 2018 were eligible to prospectively enroll in the “CRRT Intervention group,” if they were total parenteral nutrition (TPN)-dependent and not receiving any enteral or IV carnitine prior to enrollment.

The researchers say that “An exciting collaborative effort between nephrology and cardiology made it possible to use a sophisticated technology known as speckle tracking imaging to study the effects of carnitine on heart in this population.” This technology can identify early changes in heart motion, also known as cardiac strain that may not be detected using standard heart imaging techniques.

This is the first study to demonstrate that IV carnitine supplementation is associated with improvement in myocardial strain and repletion of plasma total and free carnitine in children with AKI receiving CRRT. A cohort of pediatric chronic HD patients demonstrated similar benefits in a prior study conducted by Drs. Moudgil and Sgambat. Compared with chronic HD, carnitine is even more rapidly depleted by CRRT, with losses approximating 80% of intake. The effect of carnitine deficiency and supplementation on cardiovascular function in patients receiving CRRT had not been previously investigated.

The pilot study by Drs. Moudgil, Sgambat, and Clauss was single center and limited by small sample size. The small sample size may have limited the ability to detect significant differences in demographics and clinical characteristics and multivariable analyses could not be performed. However, given that it is a pilot study, the findings provide a solid launching point for future investigations to show how supplementation can be best utilized to optimize cardiac outcomes in children receiving CRRT.

Lee Beers

Lee Beers, M.D., F.A.A.P, begins term as AAP president

Lee Beers

“The past year has been a stark reminder about the importance of partnership and working together toward common goals,” says Dr. Beers. “I am humbled and honored to be taking on this role at such a pivotal moment for the future health and safety of not only children, but the community at large.”

Lee Savio Beers, M.D., F.A.A.P., medical director of Community Health and Advocacy at the Child Health Advocacy Institute (CHAI) at Children’s National Hospital, has begun her term as president of the American Academy of Pediatrics (AAP). The AAP is an organization of 67,000 pediatricians committed to the optimal physical, mental and social health and well-being for all children – from infancy to adulthood.

“The past year has been a stark reminder about the importance of partnership and working together toward common goals,” says Dr. Beers. “I am humbled and honored to be taking on this role at such a pivotal moment for the future health and safety of not only children, but the community at large.”

Dr. Beers has pledged to continue AAP’s advocacy and public policy efforts and to further enhance membership diversity and inclusion. Among her signature issues:

  • Partnering with patients, families, communities, mental health providers and pediatricians to co-design systems to bolster children’s resiliency and to alleviate growing pediatric mental health concerns.
  • Continuing to support pediatricians during the COVID-19 pandemic with a focus on education, pediatric practice support, vaccine delivery systems and physician wellness.
  • Implementation of the AAP’s Equity Agenda and Year 1 Equity Workplan.

Dr. Beers is looking forward to continuing her work bringing together the diverse voices of pediatricians, children and families as well as other organizations to support improving the health of all children.

“Dr. Beers has devoted her career to helping children,” says Kurt Newman, M.D., president and chief executive officer of Children’s National. “She has developed a national advocacy platform for children and will be of tremendous service to children within AAP national leadership.”

Read more about Dr. Beer’s career and appointment as president of the AAP.

Research & Innovation Campus

Boeing gives $5 million to support Research & Innovation Campus

Research & Innovation Campus

Children’s National Hospital announced a $5 million gift from The Boeing Company that will help drive lifesaving pediatric discoveries at the new Children’s National Research & Innovation Campus.

Children’s National Hospital announced a $5 million gift from The Boeing Company that will help drive lifesaving pediatric discoveries at the new Children’s National Research & Innovation Campus. The campus, now under construction, is being developed on nearly 12 acres of the former Walter Reed Army Medical Center. Children’s National will name the main auditorium in recognition of Boeing’s generosity.

“We are deeply grateful to Boeing for their support and commitment to improving the health and well-being of children in our community and around the globe,” said Kurt Newman, M.D., president and CEO of Children’s National “The Boeing Auditorium will help the Children’s National Research & Innovation campus become the destination for discussion about how to best address the next big healthcare challenges facing children and families.”

The one-of-a-kind pediatric hub will bring together public and private partners for unprecedented collaborations. It will accelerate the translation of breakthroughs into new treatments and technologies to benefit kids everywhere.

“Children’s National Hospital’s enduring mission of positively impacting the lives of our youngest community members is especially important today,” said Boeing President and CEO David Calhoun. “We’re honored to join other national and community partners to advance this work through the establishment of their Research & Innovation Campus.”

Children’s National Research & Innovation Campus partners currently include Johnson & Johnson Innovation – JLABS, Virginia Tech, the National Institutes of Health (NIH), Food & Drug Administration (FDA), U.S. Biomedical Advanced Research and Development Authority (BARDA), Cerner, Amazon Web Services, Microsoft, National Organization of Rare Diseases (NORD) and local government.

The 3,200 square-foot Boeing Auditorium will be the focal point of the state-of-the-art conference center on campus. Nationally renowned experts will convene with scientists, medical leaders and diplomats from around the world to foster collaborations that spur progress and disseminate findings.

Boeing’s $5 million commitment deepens its longstanding partnership with Children’s National. The company has donated nearly $2 million to support pediatric care and research at Children’s National through Chance for Life and the hospital’s annual Children’s Ball. During the coronavirus pandemic, Boeing fabricated and donated 2,000 face shields to help keep patients and frontline care providers at Children’s National safe.

High magnification micrograph of focal segmental glomerulosclerosis

Reducing urinary protein for patients with FSGS slows kidney decline

High magnification micrograph of focal segmental glomerulosclerosis

High magnification micrograph of focal segmental glomerulosclerosis (FSGS).

Reducing the amount of protein in the urine of patients with focal segmental glomerulosclerosis (FSGS), a rare disease in which scar tissue forms on the parts of the kidneys that filter waste from the blood, can significantly slow declines in kidney function and extend time before patients’ kidneys fail, a new analysis by a Children’s National Hospital researcher and her colleagues shows. These findings, published online Aug. 10, 2020, in the American Journal of Kidney Disease, could provide hope for patients who are able to lower their urinary protein with available treatments but aren’t able to achieve complete remission, the researchers say.

FSGS affects about seven per every million people in the general population. However, in the United States, it’s responsible for between 5 and 20% of all cases of end stage kidney disease (ESKD), a condition in which the kidney function declines enough that patients can’t survive without dialysis or a kidney transplant. There are no proven treatments specifically targeting FSGS, but steroids and other immunosuppressants have shown promise in clinical trials.

One characteristic sign of FSGS is proteinuria, in which too much protein is present in patients’ urine. Most clinical trials of FSGS treatments have focused on complete remission of proteinuria as a sign that the intervention is working. However, says Marva Moxey-Mims, M.D., researcher and chief of the Children’s National Division of Nephrology, only a fraction of patients meet that end goal. Instead, many patients achieve some reduction in proteinuria, but it’s been unclear whether those reductions lead to significant benefits for kidney health.

To investigate this question, Dr. Moxey-Mims and her colleagues used data from the National Institutes of Health-funded FSGS clinical trial that took place between November 2004 and May 2008. Participants in this study — 138 patients who developed proteinuria due to FSGS between the ages of 2 and 40 and didn’t respond to steroids — received one of two different immunosuppressant regimens. They received frequent checkups including urinary protein tests during the duration of the study and were followed for a maximum of 54 months.

Results showed that about 49% of the study participants’ proteinuria improved by 26 weeks of treatment on either regimen. More importantly, says Dr. Moxey-Mims, these patients retained significantly better kidney function over time, determined by a test called estimated glomerular filtration rate (eGFR), compared to those whose urinary protein remained high. The greater the reduction in proteinuria, the better their kidney function remained, and the longer their kidneys remained active before they developed ESKD.

“Even a modest reduction in proteinuria, as small as 20 or 30%, had an impact on these patients’ kidney health,” Dr. Moxey-Mims says.

Dr. Moxey-Mims notes that the finding could impact the design of clinical trials for FSGS treatments. Currently, these trials typically must include large numbers of patients to show a benefit if complete remission of proteinuria — which only occurred in about 20% of patients in the National Institute of Diabetes and Digestive and Kidney Diseases trial — is used as the end point.

If researchers use a range of proteinuria reduction as end points, she says, it could be easier to see if a drug or other intervention is working.

Similarly, she says, patients with FSGS and their doctors should view any proteinuria reduction as a positive.

“They shouldn’t be discouraged if they can’t reach full remission,” Dr. Moxey-Mims says. “Doctors and patients alike can feel reassured that if they’re reducing protein in the urine to some degree, then patients are getting some benefit.”


cystic kidney disease

NIH $4 million grant funds new core center for childhood cystic kidney disease

cystic kidney disease

The University of Alabama at Birmingham (UAB), in collaboration with Children’s National Hospital has received a five-year, $4 million grant from the National Institute of Diabetes and Digestive and Kidney Diseases, part of the National Institutes of Health (NIH) to create a core center for childhood cystic kidney disease (CCKDCC). The UAB-CCKDCC will conduct and facilitate research into the causes of and possible treatments for cystic kidney diseases, particularly those that present in childhood.

The UAB/Children’s National grant is a U54 center grant, an NIH funding mechanism to develop a multidisciplinary attack on a specific disease entity or biomedical problem area. With this grant, UAB joins with investigators at the University of Kansas and the University of Maryland-Baltimore as part of the NIH Polycystic Kidney Disease Research Resource Consortium. The NIH describes the consortium as a framework for effective collaboration to develop and share research resources, core services and expertise to support innovation in research related to polycystic kidney disease.

“Infants with childhood cystic kidney disease may develop kidney failure within a few years after birth and some need dialysis and kidney transplantation before they reach adulthood,” said Lisa Guay-Woodford, M.D., director of the Clinical and Translational Science Institute at Children’s National and co-director of the UAB-CCKDCC. “In many cases, the earlier the onset of symptoms, the more severe the outcome.”

“The intent is to accelerate the science and advance research into new therapies for cystic kidney disease through enhanced sharing of resources and the establishment of a robust research community,” said Bradley K. Yoder, Ph.D., professor and chair of the UAB Department of Cell, Developmental and Integrative Biology and co-director of the UAB-CCKDCC. “Childhood polycystic disease can be a devastating condition for children and their families.”

The UAB-CCKDCC will focus primarily on childhood polycystic kidney disease, a condition that affects about one in 20,000 infants in the United States. The center’s primary goals are:

  • Provide the Polycystic Kidney Disease Research Resource Consortium members with access to phenotypic, genetic and clinical information and biomaterials from CCKD patients
  • Analyze pathways involved in cyst pathogenesis through the generation of verified genetic model systems and biosensor/reporter systems
  • Assess the impact of patient variants on cystic disease proteins through generation and validation of innovative models
  • Provide ready access to biological materials from genetic CCKD models
  • Develop efficient pipelines for in vitro and in vivo preclinical testing of therapeutic compounds

Dr. Guay-Woodford is an internationally recognized pediatric nephrologist with a research program focused on identifying clinical and genetic factors involved in the pathogenesis of inherited renal disorders, most notably autosomal recessive polycystic kidney disease (ARPKD). Her laboratory has identified the disease-causing genes in several experimental models of recessive polycystic kidney disease and her group participated in the identification of the human ARPKD gene as part of an international consortium. In addition, her laboratory was the first to identify a candidate modifier gene for recessive polycystic kidney disease. For her contributions to the field, she was awarded the Lillian Jean Kaplan International Prize for Advancement in the Understanding of Polycystic Kidney Disease, given by the Polycystic Kidney Disease Foundation and the International Society of Nephrology.