Abstract Happy 2022 New Year greeting card with light bulb

The best of 2022 from Innovation District

Abstract Happy 2022 New Year greeting card with light bulbA clinical trial testing a new drug to increase growth in children with short stature. The first ever high-intensity focused ultrasound procedure on a pediatric patient with neurofibromatosis. A low dose gene therapy vector that restores the ability of injured muscle fibers to repair. These were among the most popular articles we published on Innovation District in 2022. Read on for our full top 10 list.

1. Vosoritide shows promise for children with certain genetic growth disorders

Preliminary results from a phase II clinical trial at Children’s National Hospital showed that a new drug, vosoritide, can increase growth in children with certain growth disorders. This was the first clinical trial in the world testing vosoritide in children with certain genetic causes of short stature.
(2 min. read)

2. Children’s National uses HIFU to perform first ever non-invasive brain tumor procedure

Children’s National Hospital successfully performed the first ever high-intensity focused ultrasound (HIFU) non-invasive procedure on a pediatric patient with neurofibromatosis. This was the youngest patient to undergo HIFU treatment in the world.
(3 min. read)

3. Gene therapy offers potential long-term treatment for limb-girdle muscular dystrophy 2B

Using a single injection of a low dose gene therapy vector, researchers at Children’s National restored the ability of injured muscle fibers to repair in a way that reduced muscle degeneration and enhanced the functioning of the diseased muscle.
(3 min. read)

4. Catherine Bollard, M.D., M.B.Ch.B., selected to lead global Cancer Grand Challenges team

A world-class team of researchers co-led by Catherine Bollard, M.D., M.B.Ch.B., director of the Center for Cancer and Immunology Research at Children’s National, was selected to receive a $25m Cancer Grand Challenges award to tackle solid tumors in children.
(4 min. read)

5. New telehealth command center redefines hospital care

Children’s National opened a new telehealth command center that uses cutting-edge technology to keep continuous watch over children with critical heart disease. The center offers improved collaborative communication to better help predict and prevent major events, like cardiac arrest.
(2 min. read)

6. Monika Goyal, M.D., recognized as the first endowed chair of Women in Science and Health

Children’s National named Monika Goyal, M.D., M.S.C.E., associate chief of Emergency Medicine, as the first endowed chair of Women in Science and Health (WISH) for her outstanding contributions in biomedical research.
(2 min. read)

7. Brain tumor team performs first ever LIFU procedure on pediatric DIPG patient

A team at Children’s National performed the first treatment with sonodynamic therapy utilizing low intensity focused ultrasound (LIFU) and 5-aminolevulinic acid (5-ALA) medication on a pediatric patient. The treatment was done noninvasively through an intact skull.
(3 min. read)

8. COVID-19’s impact on pregnant women and their babies

In an editorial, Roberta L. DeBiasi, M.D., M.S., provided a comprehensive review of what is known about the harmful effects of SARS-CoV-2 infection in pregnant women themselves, the effects on their newborns, the negative impact on the placenta and what still is unknown amid the rapidly evolving field.
(2 min. read)

9. Staged surgical hybrid strategy changes outcome for baby born with HLHS

Doctors at Children’s National used a staged, hybrid cardiac surgical strategy to care for a patient who was born with hypoplastic left heart syndrome (HLHS) at 28-weeks-old. Hybrid heart procedures blend traditional surgery and a minimally invasive interventional, or catheter-based, procedure.
(4 min. read)

10. 2022: Pediatric colorectal and pelvic reconstructive surgery today

In a review article in Seminars in Pediatric Surgery, Marc Levitt, M.D., chief of the Division of Colorectal and Pelvic Reconstruction at Children’s National, discussed the history of pediatric colorectal and pelvic reconstructive surgery and described the key advances that have improved patients’ lives.
(11 min. read)

Societies for Pediatric Urology logo

Children’s National at the Societies for Pediatric Urology Fall Congress

Providers from the Department of Urology at Children’s National Hospital attended the Societies for Pediatric Urology Fall Congress in October 2022 and shared a series of abstracts.

The abstracts look at unplanned healthcare use and missed visits among children with spina bifida, as well as factors associated with these outcomes. The team collected data from chart review and patient interviews to complete these projects. They considered clinical information, as well as demographic information and the childhood opportunity index, which is a composite measure that proxies social determinants of health, while accounting for how far a family lives from the emergency department.

Clinically, patients with more complex spina bifida (e.g., ventricular shunt or wheelchair use) were more likely to use the emergency department. Genitourinary and gastrointestinal problems were the most common reasons for emergency department use. Within the Spina Bifida Program at Children’s National, our providers are aggressively trying to reduce gastrointestinal complaints with a nurse practitioner dedicated to bowel management and working collaboratively with the Division of Colorectal and Pelvic Reconstruction.

A sobering finding from this research was that non-white patients with spina bifida at Children’s National were more likely to have had an emergency department visit between 2016-2020 after accounting for all other variables. This indicates a clear need for improving access, communication and quality of care for minority patients with spina bifida. We are increasingly developing our community partnerships with pediatricians and school nurses, and we are accessible by a direct phone line and email to allow for prompt support. We have also established clinics in Prince George’s County in Maryland which will bring the program closer to some minority communities.

The team also found that families of children with spina bifida scheduled for telemedicine visits were much more likely to miss a visit than a child without spina bifida or if the visit was in-person. This has led to changes in how we deploy telemedicine in this population, while still remaining nimble with the option of telemedicine or in-person visits. By screening the family’s ability and desire to use this modality, we hope to reduce missed visits overall.

View the abstracts

poster conclusions

growth of the Children's National Spina Bifida Program

A look at the Children’s National Spina Bifida Program

The Spina Bifida Program at Children’s National Hospital, led by co-directors, Christina Ho, M.D., and Briony Varda, M.D., has seen impressive growth and there are plans to continue expanding the program. Drs. Ho and Varda share insights on the multidisciplinary care being provided, along with their plans for the future of the program.

Q: How would you describe the growth of the program?

A: The growth of the Spina Bifida Program has been remarkable! The graph below really shows it all (more than a 25% increase in patient visits in the past year). We currently hold multidisciplinary programs including providers from neurosurgery (Robert Keating, M.D.), orthopaedic surgery (Matthew Oetgen, M.D.), physical medicine and rehabilitation (Mi Ran Shin, M.D.), urology (Briony Varda, M.D. and Christina Ho, M.D.), and expanded to include bowel management (Celicia Little, N.P.), gynecology (Allison Mayhew, M.D.) for our adolescent patients, as well as a dedicated nurse, Nicole Allentuck, R.N., and program associate, Christine Scott.

We are extremely proud of how the program has grown, particularly through the past two years, with the advent of our adolescent clinic and having a dedicated supportive team that ensures we have significant re-engagement of patients who had previously been lost to care. We are also working with the Prenatal Pediatrics Institute at Children’s National to enhance our prenatal care for patients diagnosed with spina bifida.

growth of the Children's National Spina Bifida Program

Q: What advancements is the spina bifida program making that benefit patients?

A: We have developed a transition clinic for our adolescent spina bifida patients to help address goals of care, independence with care and readiness for transition to adult providers. Our program coordinator, Celicia Little, N.P., also oversees our bowel management program to provide specialized bowel management care for our patients. We have been working on educational information specific to different areas within spina bifida, including how to catheterize, common medications, bowel management and anticipatory guidance. This helps to provide longevity for our patients as well.  We are evaluating ways to decrease visits to the emergency department and ensure appropriate antibiotic usage with proper diagnoses of urinary infections in patients who perform CIC.  To further our goal of providing longitudinal care within our program, we have engaged gynecology colleagues to see patients within our adolescent clinic and begin promoting independence through perpetuated discussions (e.g., are they able to schedule their own physician appointments or grocery shop for themselves?) in preparation for adult transitional care. Additionally, we readily offer telehealth visits for convenience and in hopes of easing access to care.

Q: Looking ahead, what’s next for the Spina Bifida Program? How will we continue to measure success?

A: We are excited to continue expanding our Spina Bifida Program. We currently care for more than 500 patients within the Washington, D.C., Maryland and Virginia areas. We want to continue to include patient narratives through our research initiatives to inform quality of care, including decreasing E.D. utilization and needless antibiotic use. We also want to expand our adolescent program to include sexual healthcare as they transition to adult care with our adult collaborators within MedStar and GWU. We would like to provide more comprehensive care, improve access to care, and ensure patients and families have utilization of the armamentaria of medical resources available at Children’s National.

DNA strands

Whole genome sequencing solves precocious puberty case

DNA strands

By conducting whole-genome sequencing, doctors were able to discover the cause of a patient’s severe precocious puberty.

A true medical anomaly — a patient with severe precocious puberty starting in infancy later developed bilateral testicular tumors. Despite extensive testing at multiple other hospitals, no one had been able to understand the underlying cause of his precocious puberty. That is until now, through a study led by Andrew Dauber, M.D., M.M.Sc., chief of Endocrinology at Children’s National Hospital.

The hold-up in the field

Before receiving care at Children’s National, the patient’s diagnostic workup was limited by genetic testing modalities and the ability to enroll him in an innovative research protocol.

Moving the field forward

“We were able to enroll the patient in a research protocol that allowed them to sequence his whole genome,” says Dr. Dauber. “Both in a DNA sample from his blood as well as in a sample from one of his testicular tumors, which was being removed surgically.”

Dr. Dauber then performed an analysis of the genome data and found that the patient had a mutation in the luteinizing hormone receptor (LHR), which was present in the testicle but not in his blood. This is called a somatic mutation. The LHR receives the signal from the pituitary gland, which tells the testicle to make testosterone. In this case, the LHR is always turned on, which makes him develop Leydig cell tumors in his testes, overproducing testosterone, causing him to have very early puberty.

By conducting whole-genome sequencing of the tumor and blood samples, the patient was confirmed to have bilateral, diffuse Leydig cell tumors harboring the somatic gain-of-function p.Asp578His variant in the LHCGR gene.

This mutation had been identified before in patients with isolated tumors but never in someone with diffuse bilateral tumors.

The patient benefit

By using cutting-edge genomic approaches, medical providers can identify unknown causes of endocrine disorders. It also stresses the importance of the clinical team working with translational researchers to determine answers for patients.

“With a more definitive diagnosis and understanding of what these tumors are, researchers can better counsel the family about the treatment options,” says Dr. Dauber. Other members of the Children’s National team that contributed to this work include Seth Berger, M.D., Ph.D.; Daniel Casella, M.D.; and Emmanuèle C Délot, Ph.D.

You can read the full study, Precocious Puberty in a Boy With Bilateral Leydig Cell Tumors due to a Somatic Gain-of-Function LHCGR Variant, in the Journal of the Endocrine Society.

girl playing basketball

Sphincter reconstruction offers new hope for children with Hirschsprung disease

girl playing basketball

After surgery, patients reported sleeping better, higher productivity, the ability to participate in sports and be away from home with confidence.

A new surgical procedure successfully addresses a common lingering challenge — incontinence – faced by some families with young children who have undergone surgery for Hirschsprung disease. This first-of-its kind procedure was pioneered by surgeons in the Division of Colorectal and Pelvic Reconstruction at Children’s National Hospital, led by Division Chief Marc Levitt, M.D.

The 12-month outcomes for early recipients of the procedure appear in the Journal of Pediatric Surgery.

Why it matters

“The goal of surgery for Hirschsprung disease is to give a child a chance to have normal bowel function, which plays a significant role in their quality of life,” says Dr. Levitt, who served as senior author on the study. “But sometimes after a child receives their pull-through operation — the first surgical step to treat Hirschsprung — they continue to be incontinent. This novel follow-up procedure is offering these kids and their families new hope for a more normal life.”

Hirschsprung disease is a congenital condition that affects one in 5,000 children each year. Children with this condition develop a host of health problems including intestinal blockage and severe constipation, caused by the inability of the colon, or large intestine, to work properly.

The pull-through procedure is the best treatment for children with Hirschsprung disease. The procedure removes the portion of the intestine that does not function so that the child can regain control.

However, after the procedure, some children continue to experience incontinence that requires routine enemas for their entire lives. Before now there was no solution to this problem.

Why we’re excited

The new pilot study shows long-term outcomes for seven patients between the ages of 2 and 18 who underwent the novel technical approach called sphincter reconstruction. Each of the seven were diagnosed with an anatomic or physical issue contributing to their incontinence. It was during a repair procedure for the first patient that Dr. Levitt and his team had the idea of a technique for sphincter reconstruction. After that, six more patients with similar anatomy were offered the procedure.

Four of the six now have normal bowel function. They reported sleeping better, higher productivity, the ability to participate in sports and be away from home with confidence. The remaining two have behavioral conditions that have delayed their progress but are on their way to similar success.

The patient benefit

“Parents and caregivers have told us repeatedly about how much enemas negatively impact their family quality of life,” says Dr. Levitt. “That’s what makes this exciting — this validated surgical approach offers new hope for families who previously thought their child would face a lifetime of issues that would limit their freedom. Now, we finally have something to offer.”

Pediatric Colorectal Surgery Tips and Tricks

New textbook offers pediatric colorectal surgery tips and tricks

Pediatric Colorectal Surgery Tips and Tricks

The cover of the new Pediatric Colorectal Surgery Tips and Tricks textbook, written and edited by Marc A. Levitt, M.D.

The first edition of Pediatric Colorectal Surgery Tips and Tricks, a textbook written and edited by Marc A. Levitt, M.D., is now available.

The book, Dr. Levitt’s fourth textbook, focuses on sharing the knowledge he’s earned as the most experienced active pediatric colorectal surgeon in the world. The book provides inside expert information about the surgical and medical care of children with colorectal conditions.

The book includes surgical techniques and tips for medical management of:

  • Anorectal and cloacal malformations
  • Hirschsprung disease
  • Function constipation & fecal incontinence
  • Post-PSARP and Post Hirschsprung disease pull-through problems
  • Bowel management
  • Nursing care and contributions

The Pediatric Colorectal Surgery Tips and Tricks, 1st edition textbook can be purchased here.

Dr. Levitt is chief of the Division of Pediatric Colorectal and Pelvic Reconstruction at Children’s National Hospital. He has cared for children from all 50 states and 76 countries and has performed more than 15,000 pediatric colorectal procedures, which is more surgeries covering the entire spectrum of problems involving the colon and rectum than any other surgeon in the world.

He is also a driving force around the world in enhancing the care of children with colorectal and pelvic reconstructive needs through the development of specialized, integrated and collaborative surgical centers. He is internationally recognized as an expert in conditions affecting the newborn, pediatric and adolescent population affected with anorectal malformations (imperforate anus), cloacal malformations, Hirschsprung disease, as well as a variety of conditions leading to fecal incontinence, such as spinal conditions and functional constipation.

He has written four textbooks and authored over 300 scientific articles on these subjects. His work extends beyond providing direct surgical care to educating medical students, medical doctors, surgeons and nurses, as well as developing collaborating centers throughout the world to ensure that all children have access to quality colorectal care. He has hosted hundreds of surgeons and nurses from hospitals across the globe, who have sought out education and training on advanced colorectal surgical techniques, management of patients and research outcomes. Dr. Levitt is the founder of the Colorectal Team Overseas (CTO), a group of international providers that travel to the developing world to provide care and teaching for patients with colorectal needs. He co-founded the Pediatric Colorectal and Pelvic Learning Consortium (PCPLC), which is an organization of collaborating colorectal centers across the globe.

pregnant woman at fertility consultant

Fertility preservation in sickle cell disease patients

pregnant woman at fertility consultant

Fertility is a long-standing concern for patients with sickle cell disease and their families.

In a recent review in the Journal of Clinical Medicine, researchers from Children’s National Hospital look at the current state of fertility preservation in patients with sickle cell disease and make recommendations for longitudinal post-treatment for these individuals.

Fertility is a long-standing concern for patients with sickle cell disease and their families. Current curative therapy for the disease requires gonadotoxic conditioning, which many patients resist because of the resulting risk of infertility. And, while standard fertility preserving interventions exist for pre- and postpubescent females and males, best practices for integrating these interventions into sickle cell disease care have not yet been established.

In their article, Children’s National hematologist Robert Sheppard Nickel, M.D., and co-authors review current fertility assessments, fertility considerations in pre- and post-transplant patients with sickle cell disease and fertility preserving interventions for patients. The authors conclude that in the future, less toxic curative approaches may make fertility preservation unnecessary, but at present, fertility preservation should be offered to patients with sickle cell disease pursing curative therapy.

Additional authors from Children’s National include Michael Hsieh, M.D., Ph.D., and Jacqueline Maher, M.D.

Read the full review article, Fertility after Curative Therapy for Sickle Cell Disease: a Comprehensive Review to Guide Care, in the Journal of Clinical Medicine.

US News Badges

Children’s National named to U.S. News & World Report’s Best Children’s Hospitals Honor Roll

US News BadgesChildren’s National Hospital in Washington, D.C., was ranked No. 5 nationally in the U.S. News & World Report 2022-23 Best Children’s Hospitals annual rankings. This marks the sixth straight year Children’s National has made the 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 sixth year in a row.

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

“In any year, it would take an incredible team to earn a number 5 in the nation ranking. This year, our team performed at the very highest levels, all while facing incredible challenges, including the ongoing pandemic, national workforce shortages and enormous stress,” said Kurt Newman, M.D., president and chief executive officer of Children’s National. “I could not be prouder of every member of our organization who maintained a commitment to our mission. Through their resilience, Children’s National continued to provide outstanding care families.”

“Choosing the right hospital for a sick child is a critical decision for many parents,” said Ben Harder, chief of health analysis and managing editor at U.S. News. “The Best Children’s Hospitals rankings spotlight hospitals that excel in specialized care.”

The annual rankings are the most comprehensive source of quality-related information on U.S. pediatric hospitals and recognizes the nation’s top 50 pediatric hospitals based on a scoring system developed by U.S. News.

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.

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

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

Everyone Poops Book Cover

2022: Pediatric colorectal and pelvic reconstructive surgery today

Everyone Poops book coverAdapted from Levitt MA. New and exciting advances in pediatric colorectal and pelvic reconstructive surgery – 2021 update. Semin Pediatr Surg. 2020 Dec;29(6):150992.

As demonstrated in the popular children’s book by Taro Gomi, Everyone Poops, the physiology of stooling is a focus of early childhood development and a subject of concern for both parents and children. References to pediatric colorectal problems go back many thousands of years. In fact, the Babylonian Talmud, written in the year 200 CE, recommends that “an infant whose anus is not visible should be rubbed with oil and stood in the sun… and where it shows transparent the area should be torn crosswise with a barley grain.” Surgical techniques to manage such patients have certainly evolved since that time, but the basic principles of care remain the same.

How we got here

The modern story of the care of patients with anorectal malformations (ARMs) began in the 1940s in Melbourne, Australia, when Henry Douglas Stephens worked to define the anatomy of children with ARMs by analyzing the anatomy of twelve deceased patients with these conditions. He continued to focus on this specialty for the rest of his career and published two books on the topic in 1963 and 1971. Prior to his groundbreaking work, the anatomy of such patients was only a concept that existed in surgeons’ minds – without anatomic precision – since no one had actually seen the anatomy. These concepts were depicted in the bible of pediatric surgery in North America, the Gross textbook, which in retrospect was both oversimplified and inaccurate.

During his autopsy dissections, Stephens came to the key anatomic conclusion that the puborectalis muscle (the sphincters) lay behind the urethra. He devised an operation based on this concept: first, the urethra was identified, then a space between it and the puborectalis was dissected, and then the rectosigmoid was pulled through that space. A small incision in the perineum for the pulled-through bowel was made, within which the new anus was created. The perineal dissection was a blind maneuver. During the same time period, William Kiesewetter in Pittsburgh proposed his version of the sacral abdominoperineal pull-through using similar anatomic principles.

Justin Kelly was one of Stephens’ trainees in Australia who learned how to do this operation. At Boston Children’s Hospital in the late 1960’s, he taught what he had learned from Stephens to the surgeons there, including another trainee, Alberto Pena. Pena and his fellow surgical residents benefitted from exposure to Kelly as well as master surgeon Hardy Hendren, who operated on patients across town at Massachusetts General Hospital. Hendren, the pioneer in the care of children with cloacal anomalies, passed away this year at the age of 96.

Pena completed his training in Boston and went to Mexico City in 1972 at the age of 34 to become the head of surgery at the National Pediatric Institute. He tells the story that when he asked his new pediatric surgery faculty to choose an area of specialization, no one chose colorectal, so he decided to take on that group of patients and thus embarked on his revolutionary colorectal career. Pena at first applied the technique he had learned from Kelly to repair anorectal malformations, but he became increasingly frustrated by the procedure. He felt that the maneuvers offered very poor exposure to the anatomy, and over time his incision grew longer and longer. In 1980, Pena’s collaboration with Pieter de Vries – who had come to Mexico City to work on these cases with Pena – culminated in the first posterior sagittal anorectoplasty [Figure 1]. That same year, Pena presented his findings at a meeting of the Pacific Association of Pediatric Surgeons.

imperforate anus repair

Figure 1: Original diagrams of the posterior sagittal approach to repair imperforate anus

This posterior sagittal approach opened a beneficial Pandora’s Box in surgery. It allowed for a true understanding of the pelvic anatomy and led to the care of many conditions that were previously – to use Pena’s words – “too difficult to reach from above (via laparotomy) and too difficult to reach from below (perineally).” This new approach influenced the repair of cloacal malformations, urogenital sinus, pelvic tumors, urethral problems, reoperations for imperforate anus and Hirschsprung disease (HD), a transpubic approach for complex genitourinary problems, and a comprehensive strategy for the management of cloacal exstrophy. In addition to his surgical contributions, Pena also conceived of the intervention that has arguably improved patients’ quality of life the most: a focused approach to the bowel management of fecal incontinence. Thanks to such bowel management programs, now available at many centers across the world, thousands of children are no longer wearing diapers and have said goodbye to their stomas. The impact of bowel management is perhaps comparable to that of intermittent catheterization for patients with urinary incontinence.

My personal journey in this field began in 1992, when I was an eager medical student and signed up for an elective in pediatric surgery with Alberto Pena. This experience changed my career trajectory in a very dramatic and positive way. Medicine was becoming increasingly complex, and fields such as neonatal care, transplantation, and cardiology were benefitting from a collaborative approach. It became clear that the colorectal field needed the same approach. As a comparative example, consider the project of building a bridge. How does such a project start? The cement layers do not show up one day and lay cement prior to the steel team deciding where to place the beams. The project must begin with all parties meeting together to develop a comprehensive plan. Amazingly, however, that type of collaborative planning does not often happen in the care of medically complex patients. It most assuredly needs to.

The value of multi-disciplinary care

Medical complexity requires integrated and collaborative care because all the anatomic structures that need to be managed are located right next to each other and because each has a unique and complex physiology that can affect the other systems. To achieve success, patients with anorectal malformations, Hirschsprung disease, fecal incontinence (related to a variety of conditions), and colonic motility disorders require care from a variety of specialists throughout their lives. These include providers in the fields of colorectal surgery, urology, gynecology, gastroenterology, motility, orthopedics, neurosurgery, anesthesia, pathology, radiology, psychology, social work, nutrition, and many others. Perhaps most important to the achievement of a good functional result, however, is a patient’s connection to superb nursing care. A complex colorectal operation takes about four hours to perform, but to get a good result, it takes an additional 96 hours of work, the vast majority of which involves nursing care. The value of good nursing partners to ensure successful surgery cannot be overemphasized. They have unique skills in identifying and solving problems, a willingness to get down in the weeds, and are always striving to fill the gaps in care.

What parents want and need

Having met many parents with newborns diagnosed with colorectal problems, I have made several observations. First, it seems that no parent has ever imagined that their child could have a problem with stooling – this is a physiologic ability that is taken for granted. When they are told about the problem with their baby, they are uniformly shocked that something like this could happen. Second, when discussing that their child will need surgery to correct their colorectal anatomy, parents don’t focus on the surgical technique and elegance of the reconstruction, as surgeons tend to do. Instead, parents dwell on whether the surgery will create a working reconstructed anatomy that will allow their child to stool without difficulty or embarrassing accidents. As surgeons we need to remember this. We always need to understand what it is that the family and patient wishes us to deliver, and we need to strive to achieve those goals. As proud of our surgical skills as we are, it is the functional outcome that matters most.

Where we are in 2022

In 2022, the advances in the field of pediatric colorectal and pelvic reconstruction are significant. They include new techniques and ideas that over time have made a dramatic and positive impact on the care and quality of life of children who suffer from colorectal problems. Here are a few such advances:

  1. Prenatal diagnosis of anorectal and cloacal malformations has been progressively improving. Perinatologists have learned to look for specific findings, such as a pelvic mass in a female with a single kidney, and consider that it could be a cloaca. Assessment of perineal anatomy, pubic bone integrity, sacral development, abnormalities of the radius bone, as well as cardiac, spinal, and renal anomalies may lead the clinician to consider that a fetus may have an anorectal malformation.
  2. Management of the newborn, particularly in the fields of newborn radiology and neonatal care, has dramatically improved as neonatal techniques have advanced. Specific to the colorectal patient have been advances in radiology such as assessments of hydronephrosis, 3D reconstruction of cloacal anomalies, and ultrasound-guided distal colostography. Further advances include improved techniques in the management of hydrocolpos and stoma care, to name a few.
  3. The treatment of associated urologic anomalies has diminished chronic renal disease, and proactive bladder management is reducing the need for bladder augmentations and renal transplantation.
  4. Understanding the gynecologic collaboration has helped clinicians define the Mullerian anatomy and better plan for menstruation and future obstetric potential.
  5. Prediction of continence, even in the newborn period, requires an understanding of the associated problems with the sacrum and spine. This knowledge has allowed clinicians to have more robust conversations with families about their child’s future.
  6. The decision of whether to do a newborn repair versus a colostomy must be guided by the surgeon’s experience and the clinical circumstances in which they find themselves.
  7. The defining of anatomy allows patients to be compared across medical centers, and for treatment options and outcomes to be uniformly analyzed. Keeping track of one’s outcomes and always striving to improve should be basic tenets of surgical practice.
  8. Recognizing the value of laparoscopy and knowing for which cases this approach should be applied. Morbidities associated with a laparoscopic approach for a rectum in an ARM patient well below the peritoneal reflection have been noted. In HD cases, laparoscopy can limit the stretching of the sphincters which occurs during the transanal rectal dissection.
  9. Development of a treatment algorithm for the management of cloacal malformations which considers the importance of their common channel and urethral lengths.
  10. Recognizing key complications after ARM and Hirschsprung surgery, knowing when and how to do a reoperation, determining the outcomes of such reoperations, and ultimately figuring out how to avoid complications altogether.
  11. Understanding the causes of fecal incontinence, the amount of incontinence that can be anticipated, and the surgical contributors to achieving continence.
  12. Development of bowel management programs in multiple centers and committing to following these patients in the long term.
  13. Learning the pathophysiology of motility disorders and developing treatment protocols, as a result of the vital collaboration between surgery and GI/motility. Medical treatments with laxatives, rectal enemas, botox injection of the anal canal, and surgical adjuncts such as antegrade colonic flush options and sometimes colon resections are key aspects of the treatment armamentarium.
  14. Developing a collaboration between colorectal surgery and urology allows the clinical team to know when the colon can be used for a bladder augment (which not only offers an augment option but also can improve antegrade flushes of the colon) or if the appendix can be shared between Malone and Mitrofanoff. The collaboration with urology to plan the management of both urinary and fecal continence during the same operation is a very valuable trend. This proactive planning has improved the lives of many patients and has reduced the numbers of operations they need as well as their hospital stays.
  15. Sacral Nerve Stimulation (SNS) has shown promise in the management of urinary incontinence and seems to have a role in improving fecal continence and promoting motility, as an adjunct to treatments for constipation.
  16. Development of dedicated colorectal centers that are integrated and collaborative allows a team to tabulate their results and strive for better outcomes. The value of a collaborative model for the care of such patients cannot be overstated, not only for patient convenience, but also for creating an integrated plan for their care. These exist now in many parts of the country and care is available in nearly every region, reducing a family’s need to travel great distances away from their home to access care.
  17. Establishing a transition to adult programs, as with congenital heart disease and cystic fibrosis. Colorectal surgeons are obligated to develop a transition plan for their patients as they enter adulthood.
  18. Using Basic Science to advance the field, including tissue engineering and genetics, will be revolutionary. We should be able to imagine the day when cloacal reconstruction could be based on a previously tissue-engineered segment of vagina, produced by the patient’s own stem cells. In addition, the genetics of anorectal malformations as well as Hirschsprung disease are being vigorously pursued which will impact parental counseling and potential therapies.
  19. Real time data used to follow outcomes is needed to keep track of complications as well. That information can be used to adjust protocols which will improve results.
  20. Development of international consortiums will help patients in a way that is not achievable by a single institution. Consortiums allow ideas to be spread rapidly which will dramatically affect how many patients can be helped and how quickly. The Pediatric Colorectal and Pelvic Learning Consortium, pcplc.org, is well on its way to achieving these goals.
  21. Bringing complex care to all corners of the world because there is a great deficiency in advanced colorectal care in the developing world. The care of colorectal patients in a resource-limited setting has unique challenges, but creative solutions by innovative surgeons in those areas have a led to dramatic improvements in care.
  22. Parent/patient organizations provide education, advocacy, and support for families at all stages of their child’s care. With internet access readily available, colorectal patients and their families can now access the welcoming environment of these organizations, and no longer feel as lost and alone as in previous years.

Why it matters

Given all these wonderful advances, we must continue to reaffirm the key principles stated by Sir Dennis Browne that “the aim of pediatric surgery is to set a standard, not to seek a monopoly.” With an integrated approach to the care of this complex group of patients, great things can be achieved. I am hopeful that the caregivers and parent/patient group organizations who commit to the care of children with colorectal problems and understand the daily struggle of improving a patient’s quality of life will learn the skills and tricks necessary to achieve good results. If they do, they will help many children.

Finally, I will share a humorous piece written by my daughter, Jess Levitt, regarding the value of bringing order to chaos. Its message is particularly relevant to the care of children with colorectal problems in 2022, as we build on our efforts to improve, streamline, and transform the formerly chaotic process through collaboration and education. As we endeavor to advance this field, we need to remember what Dr. Pena often said: “It is not the unanswered questions, but rather the unquestioned answers that one must pursue.”

“A” must come before “B,” which must come before “C,” everybody knows that. But what if the Millercamps of this world did not have to sit next to the Millerchips when it comes to seating arrangements? Can Pat Zawatsky be called before Jack Aaronson when the teacher is taking attendance? Do those 26 letters that make up all the dialogue, signs, thoughts, books, and titles in the English-speaking department of the world need their specific spots in line? Everyone can sing the well-known jingle from A to Z, but not many people can tell you why the alphabet is the way it is.

For almost as long as humans have had the English language, they have had the alphabet. The good ol’ ABCs. However, the alphabet represents the human need for order and stability. I believe that the same thinking that went into the construct of time and even government went into the alphabet. Justifiably, lack of order leads to chaos. Knife-throwing, gun-shooting chaos, in the case of lack of governmental order. Listen to me when I tell you that there is absolutely no reason that the alphabet is arranged the way that it is. Moreover, the alphabet is simply a product of human nature and how it leads people to establish order for things that do not require it.

Now I know this sounds crazy, but bear with me. Only if you really peel away the layers of the alphabet will you find the true weight it carries. People organized the letters of our speech into a specific order simply because there wasn’t already one. Questioning this order will enlighten you on the true meaning of it. Really dig deep into the meaning behind the social construct that is the alphabet. Short and sweet as it may be, the order of the ABCs is much less than meets the eye. There is no reason that “J” should fall before “K”! Understand this. Very important as order is, it is only a result of human nature.

What’s next? X-rays become independent of Xylophones in children’s books of ABCs?

You know what the best part is? Zero chance you even noticed that each sentence in this essay is in alphabetical order.

– Jess Levitt


Jacob Smith

Q&A with Pediatric Surgical Innovation Fellow Jacob Smith, M.D.

Jacob Smith

Jacob Smith, M.D.

Jacob Smith, M.D. is currently a fellow with The Joseph E. Robert, Jr. Fellowship in Pediatric Surgical Innovation at Children’s National Hospital. The fellowship provides is an exciting and dynamic post-graduate research experience focused on biomedical innovation. Participants can focus their work on specific areas of interest. Here, Dr. Smith shares some information on his work with the Urology Department.

Q: How has the Joseph E. Robert, Jr., Fellows in Pediatric Surgical Innovation program allowed you to expand your experience in pediatric urology?

A: The Roberts Fellow program has provided me the ability to work with Michael Hsieh, M.D., who has years of experience in UTI and schistosomiasis research. The UTI research has been a focus of mine and we are working on ways to diagnosis and treat UTI faster in patients. One population that this can benefit are those that deal with recurrent UTIs, such as patients in our spina bifida population.

Q: Talk a little about your work on spina bifida and areas of opportunity for research.

A: Briony Varda, M.D. is heavily involved in our spina bifida program here at Children’s National. I am working with Dr. Varda to develop a database that looks at the use of the emergency department (ED) by children with spina bifida in the Washington, D.C. area. We are also conducting interviews with families and other stakeholders in this population to better understand the factors that drive this population to utilize the ED. We hope that this research can give us a better understanding of the spina bifida care in our area and inform us to provide improvement in care.

Q: How will your work to develop methods to diagnose urinary tract infections faster and how will this benefit our patient population?

A: As mentioned before, a common patient population that deals with recurrent UTIs is the spina bifida population. The protocol that we are attempting to develop would provide us quicker results than the standard urine culture and sensitivity testing that can take anywhere between 48-72 hours to result on average. We hope by providing a quicker result, we can better identify the necessary treatment these patients need to treat their infections. Another scenario that we hope this can be utilized in is for those patients that have recurrent UTI symptoms but negative urine cultures. We hope we can determine if there are viable cells that may be too few to culture but still could cause these symptoms.

Q: What are you currently working on that you are most excited about?

A: As you can tell, a lot of my research is conducted around the spina bifida population. I am excited to dive into the data we have collected on this population and determine if there are ways we could improve the care of these patients.

Q: What made you interested in specializing in pediatric urology?

A: Pediatric urology is a great field. It has a very diverse patient population. There are a multitude of problems that could arise from multiple congenital malformations to stones and voiding dysfunctions. There is also a range of cases from minor and major reconstruction, robotics, endourology and laparoscopy. One thing that does differentiate pediatric urology is that many of the problems that we repair are congenital and it is a privilege to be able to help these children and families. In fact, I was a patient myself as a child with a ureteral malformation which required surgery. The concept of helping other children with urology concerns brings an obvious “pay-it-forward” mentality that also attracted me to the field. I am excited to obtain the knowledge and skills necessary to treat my future patients.

medical symbol on a map of the world

Observership program builds worldwide expertise to treat colorectal issues

medical symbol on a map of the worldPediatric colorectal specialists are in short supply, and this is particularly true in many areas of the developing world. When Marc Levitt, M.D., travels abroad, he consistently finds eager surgeons and nurses who wish to obtain advanced colorectal skills to help their patients. To meet this need Dr. Levitt has established an international observership program that brings leading physicians and nurses from around the globe to participate in one- to three-month-long observerships.

During 2022, the program will welcome participants from 13 different countries including Spain, Belgium, Vietnam, Indonesia, Chile, Pakistan, Uganda, Iraq, Mexico, Brazil, Saudi Arabia and Singapore.

Under the colorectal team’s instruction, participating physicians and nurses will learn how to diagnose and care for children with complex colorectal conditions. They will learn about the wide range of malformations and successful treatment options so they can bring these skills to patients in their home countries. Those selected for these observerships are among the most promising providers in their communities who currently work to improve treatment for children with colorectal issues.  The patients they care for are those who would otherwise have no or limited access to this specialty care.

For information about applying for the observership program, please contact the colorectal department at ColorectalVisitors@childrensnational.org.

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.

surgeon doing laparoscopic surgery

Autonomous robotic laparoscopic surgery for intestinal anastomosis

surgeon doing laparoscopic surgery

Children’s National Hospital in collaboration with the University of North Carolina Wilmington and Johns Hopkins University developed an enhanced autonomous strategy for laparoscopic soft tissue surgery.

A new approach to soft tissue surgery could simplify autonomous surgical planning and enable collaborative surgery between an autonomous robot and human, a new study published in Science Robotics finds. This is the first time a robot can complete an autonomous soft tissue surgical task under laparoscopic conditions, forming the foundation for future soft tissue surgeries.

Children’s National Hospital in collaboration with the University of North Carolina Wilmington and Johns Hopkins University developed an enhanced autonomous strategy for laparoscopic soft tissue surgery. The multi-institutional effort made it possible to perform a robotic laparoscopic small bowel anastomosis in phantom and in vivo intestinal tissues. The findings further suggest that autonomous robot-assisted surgery has the potential to provide more efficacy, safety and consistency independent of an individual surgeon’s skill and experience.

The hold-up in the field

Autonomous anastomosis is known to be a challenging soft tissue surgery task. And in the laparoscopic setting, surgeries like these prove to be more challenging because of the need for high maneuverability and repeatability under motion and vision constraints – especially in pediatric patients.

“This work represents the first time autonomous soft tissue surgery has been performed using a laparoscopic technique and is the first step in bridging the gap between human and machine towards completing autonomous surgical tasks in soft tissue surgeries,” says Hamed Saeidi, Ph.D., assistant professor at University of North Carolina Wilmington and lead author of the study.

To overcome the unpredictable motions of the tissue, the experts used machine learning based techniques to track the dynamic motions of the soft tissue during the surgery. These methods also pave the way for markerless methods for tracking the tissue motion in future surgeries.

“Until now, laparoscopic autonomous surgeries were not possible in soft tissue due to the unpredictable motions of the tissue and limitations on the size and capabilities of surgical tools,” says Justin Opfermann, M.S., Ph.D., student and Johns Hopkins University and co-author.

What’s unique

Performing autonomous surgery would require the development of novel suturing tools, imaging systems and robotic controls to visualize a surgical scene, generate an optimized surgical plan and then execute that surgical plan with the highest precision.

The autonomous robot takes its skill one step further when performing surgical tasks on soft tissues by enabling a robot-human collaboration to complete more complicated surgical tasks where preoperative planning is not possible.

Additionally, the robot used in this work uses a novel shared control scheme called “conditional autonomy,” whereby the robot performs the majority of the surgical task, which the surgeon oversees.

Bottom line

“Combining all of these features into a single system is non-trivial,” Opfermann adds. “In 2016, we were the first group to demonstrate feasibility of semi-autonomous small bowel anastomosis with a robot in soft tissue, and now we can perform autonomous laparoscopic anastomosis.”

The resulting anastomosis had more consistency and achieved higher burst strength than surgeons suturing with manual technique, resulting in less anastomotic leak.

In laparoscopic surgeries – and pediatric patients especially – these challenges are even more difficult due to the small size of the patient. Robotic anastomosis is one way to ensure that surgical tasks that require high precision and repeatability can be performed with more accuracy and precision in every patient independent of surgeon skill.

“As a surgeon, I can attest to the potential benefits of improving how we perform surgery on our patients,” says Michael Hsieh, M.D., Ph.D., director of Transitional Urology at Children’s National Hospital. “Working with my engineering colleagues at Johns Hopkins, we’ve been able to develop prototypes of supervised, autonomous suturing robots that may be a step towards such improvements.”

Hans Pohl

Q&A with Hans Pohl, M.D., on the future of pediatric urology

Hans Pohl

Hans Pohl, Division Chief, Urology

The Urology team at Children’s National Hospital is led by Hans Pohl, M.D., and includes seven other fellowship-trained pediatric urologists and three nurse practitioners. Dr. Pohl has experience in treating patients with spina bifida and bladder exstrophy, in addition to the other more common diagnoses and in using laparoscopy to perform surgery through minimally invasive techniques.

Here, Dr. Pohl tells us more about the department he is leading and what it means for the future of pediatric urology patients at Children’s National.

What excites you most about current research in the field of urology?

The most cutting-edge research these days pertains to understanding the complex and diverse role played by bacteria in the urinary tract. We previously thought the urinary tract is sterile but that turns out to not be the case. Just like the friendly bacteria living in our bodies, there are bacteria that live within our urinary tracts. It is thought that when the normal function of the urinary tract is disrupted, the balance of healthy to unhealthy bacteria is disrupted. Our faculty at the Children’s National Urology Division are looking at urinary tract infection (UTI) from various aspects. Michael Hsieh, M.D., is investigating the role of bacteria in causing inflammation; Daniel Casella, M.D., has investigated how a drug called varenicline might reduce inflammation in infected kidneys; and Briony Varda, M.D., will be investigating the socioeconomic factors affecting how people living with spina bifida manage their urinary tracts and UTIs. By looking at UTIs at all levels from bacteria factors to host factors to treatment we will understand better how to reduce the impact of a very common problem on human beings.

What are some of the most valuable changes or advancements for the program you hope to see in the next couple of years?

I believe that our next step in program evolution will focus on improving the quality of life of children with urination abnormalities and UTIs. Everyone has experienced that their child has urinary symptoms at some point during childhood, some also have UTIs. Pediatric urologists have learned that normal lower urinary tract function is critically important in reducing a child’s risk for a UTI. Even if a child does not have UTIs, there can be significant social stigma amongst a child’s peers when loss of urine control happens.

What makes the Urology Division at Children’s National unique from other programs in the country?

We have grown considerably over the past several years, adding to our faculty surgeons with complimentary skills outside of the operating room. For instance, we have pediatric urologists who are also basic scientists, translational scientists, systems scientists, a clinical informaticist and minimally invasive surgeons. These varied qualities create a dynamic group of people who bring diverse perspectives to treating patients’ problems and generating creative solutions. We believe that our democratic process of complex care management where all surgeons can openly think about how to optimize patient management is unique. Patients don’t get one opinion from their surgeon, they get ten.

two doctors perform surgery

Can complex pediatric surgery interventions be standardized to facilitate telementoring?

two doctors perform surgery

The study’s authors write, “These discussions are particularly relevant to surgeons in small or rural practices who provide much-needed care to underserved populations and have decreased exposure to these index cases. Conversely, in some developing countries where prevalence of rare congenital surgical conditions is higher, there is a shortage of adequately trained pediatric surgeons. Each of these scenarios involves a mismatch in experience and exposure, which can result in poor patient outcomes and inadequate healthcare delivery.”

How does a surgeon-in-training get enough exposure to rare or complex cases to serve the patients who need them? How does a practicing surgeon perform enough cases each year to maintain proficiency at such index cases?

The authors of a study in the Journal of Pediatric Surgery, including Marc Levitt, M.D., chief of the Division of Colorectal and Pelvic Reconstruction at Children’s National Hospital, write that, “These discussions are particularly relevant to surgeons in small or rural practices who provide much-needed care to underserved populations and have decreased exposure to these index cases. Conversely, in some developing countries where prevalence of rare congenital surgical conditions is higher, there is a shortage of adequately trained pediatric surgeons. Each of these scenarios involves a mismatch in experience and exposure, which can result in poor patient outcomes and inadequate healthcare delivery.”

Telementoring is one strategy being explored by the American College of Surgeons’ Telementoring Task Force initiative. Pediatric anorectal malformations (ARM), pediatric colorectal surgical procedure, posterior sagittal anorectoplasty (PSARP) were the “index” areas for the pilot study. Once the expert established the areas of great need, they will test the feasibility of a curriculum and training program using telementoring in pediatric surgery. The ACS Task Force notes that these conditions are relatively rare and require a particular skill level to manage appropriately, making them good candidates for the study.

The Journal of Pediatric Surgery study presents a process for mapping out a standardized curriculum for these procedures. First, the authors sought expert consensus on three interoperative checklists that form a de facto curriculum for teaching, learning and performing ARM and PSARP procedures. Second, a multidisciplinary team of medical educators and pediatric surgery experts drafted the checklists. The authors then sought review and input from pediatric colorectal surgery experts at 10 institutions worldwide, who comprised the study’s colorectal pediatric surgery subject matter expert panel. To be considered “expert,” participants had to meet or exceed several strict inclusion criteria related to years in practice and experience with these case types.

Institutions of the colorectal pediatric surgery subject matter expert panel.

Institutions of the colorectal pediatric surgery subject matter expert panel.

The process led to a successful set of consensus documents. “To our knowledge, this is the first study to establish and standardize key intraoperative objectives using a modified-Delphi method in pediatric surgery,” the authors write. “Although this process can be quite time consuming, it provides an incredible opportunity to standardize intraoperative teaching and expectations of trainees. Future studies will expand these checklists into developing a competency assessment tool involving assessment for validity and reliability in a clinical setting to ultimately improve patient safety through standardization.”

Dr. Levitt says the overarching goal of this work is “to improve the surgical technique everywhere [to] thereby help as many kids as we can, even those we will never meet.”

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.

3D Illustration Concept of Human Urinary System

Predicting surgery risk through single diuretic renogram

3D Illustration Concept of Human Urinary System

In a new study published in the Journal of Pediatric Urology, Aaron Krill, M.D., urologist at Children’s National Hospital and leading author, shows that halftime from a single initial diuretic renogram in children with antenatally detected UPJO is predictive not only of worsening future drainage, but also of future surgical repair (pyeloplasty).

The findings of a new study show promising results that will help to further subdivide the indeterminate drainage range and will help practitioners identify those children at increased risk for worsening drainage and future surgery.

So far, we know that spontaneous resolution of antenatally detected kidney obstruction, also known as ureteropelvic junction obstruction (UPJO), is relatively common. However, it can take several years and require frequent surveillance ultrasounds and diuretic renograms before patients reach a clinical outcome.

The study, which published in the Journal of Pediatric Urology, and was led by Aaron Krill, M.D., urologist at Children’s National Hospital, shows that halftime (T1/2) from a single initial diuretic renogram in children with antenatally detected UPJ obstruction is predictive not only of worsening future drainage, but also of future surgical repair (pyeloplasty).

“Among infants with UPJO, 52 to 79% can be expected to undergo spontaneous improvement,” Dr. Krill said.

Initial nonoperative management has become the standard of care for cases with indeterminate drainage patterns with preserved differential renal function (DRF). Diuretic renography has traditionally been the gold standard for diagnosis and surveillance of this condition. Identification of patients with very good drainage who can safely be discharged and those with very poor drainage who require early surgical repair has never been difficult. However, patients with indeterminate drainage have posed a unique diagnostic and therapeutic problem. Previously published ranges for indeterminate drainage were either too wide or too narrow to be clinically useful.

In the study, recent data shows a five-year surgery-free survival probability for patients with t1/2 of 5-20, 21-40 and 41-60 minutes to be: 79.7%, 46.7% and 33.3%. This suggests that patients with t1/2 of more than 21 minutes are at moderate to high risk of requiring surgery within the first five years of life while those with t1/2 20 minutes or less are at relatively low risk.

“This would allow us to concentrate our efforts appropriately on those who are at high risk of progressing to surgery and minimize the burden of testing and that of radiation exposure for children who are at low risk and likely to improve spontaneously,” Dr. Krill added.

Researchers identified patients younger than 18 months at presentation with unilateral, isolated moderate to severe hydronephrosis who underwent diuresis renography from 2000-2016. This group was sub-divided into three T1/2 intervals: 5-20, 21-40 and 41-60 minutes. Endpoints were pyeloplasty and pyeloplasty free survival. Indications for surgery were loss of DRF, worsening T1/2, family preference and/or pain.

“Being told that your newborn has an obstructed kidney and may require surgery in the future is typically a very stressful event for families,” Dr. Krill said. “Now after only a single diuretic renogram, we can provide families with accurate probability estimates of their child’s lifetime risk of surgery and risk of surgery within their first five years. This should allow us to appropriately set expectations and customize our surveillance routine for each patient.”

For decades, Children’s National has been working to refine and standardize diuretic renography. Some accomplishments over the years include establishing the safety of the test in infants, verifying its diagnostic utility, identifying new drainage parameters, and coupling it with machine learning to improve accuracy. This most recent manuscript capitalizes on a large database spanning 20 years of the team’s clinical experience to further improve the ability to predict who will need surgical repair and better counsel our patients.

Other authors include Briony K. Varda, M.D., M.P.H., Nicholas A. Freidberg, Md Sohel Rana, M.B.B.S., M.P.H., Eglal Shalaby-Rana, M.D., Bruce M. Sprague and Hans G. Pohl, M.D.

Micrograph of human parasite Schistosoma mansonii

Diagnosing and monitoring of urogenital schistosomiasis

Micrograph of human parasite Schistosoma mansonii

Urogenital schistosomiasis (UGS) is caused by egg-laying S. haematobium worms dwelling within the veins draining the main pelvic organs, including the bladder, uterus, and cervix.

Although urogenital schistosomiasis remains a major global challenge, Michael Hsieh, M.D., Ph.D., director of Transitional Urology at Children’s National Hospital, and other experts including Eglal Shalaby Rana, M.D., from Children’s National, show in a new study published in Advances in Parasitology that newer refinements in associated technologies may lead to improvements in patient care.

In addition, application of investigational imaging methods, such as confocal laser endomicroscopy and two-photon microscopy in urogenital schistosomiasis, are likely to contribute to the understanding of this infection’s pathogenesis.

Read the full study in Advances in Parasitology.


An analysis of articles on pediatric COVID-19 cases


In a recent editorial, Dr. Briony Varda commented on a systematic review and meta-analysis of articles reporting on pediatric cases of COVID-19.

In a recent editorial, Children’s National Hospital Pediatric Urologist Briony Varda, M.D., M.P.H., and Emilie K. Johnson, M.D., M.P.H., from Ann & Robert H. Lurie Children’s Hospital of Chicago, comment on a systematic review and meta-analysis of articles reporting on pediatric cases of coronavirus disease 2019 (COVID-19) due to severe acute respiratory syndrome coronavirus-2 (SARS-CoV-2) infection.

Their take home messages were that although COVID-19 is typically milder in children than in adults, children (particularly infants) do appear to have cardiac damage from COVID-19 which may be a consideration for preoperative evaluation among surgeons. They also note the MIS-C is another emerging concern for children following an infection with COVID-19.

Read the full editorial in the Journal of Pediatric Urology.

conceptual image of bladder cancer

Sensitivity to physical versus chemical factors in CAP

conceptual image of bladder cancer

To date, reactive oxygen species and reactive nitrogen species have been regarded as the key factors causing the observable cellular death of cold atmospheric plasma (CAP)-treated cancer cells. The chemical basis of the conventional CAP treatment highlights apoptosis as the main CAP-triggered cell death mechanism.

However, in a recent study published in the Journal of Physics, Michael Hsieh, M.D., Ph.D., director of Transitional Urology at Children’s National Hospital, and other experts demonstrated a strong anti-melanoma effect based on physically-based CAP treatment. The study, which also tested bladder cancer, compared the anti-cancer effect of chemically-based versus physically-based CAP treatment on four typical cancer cell lines in vitro.