Behind the Nociometer: Q&A with Julia Finkel, MD

Julia Finkel

Julia Finkel, MD, is a pediatric anesthesiologist and the director of Pain Medicine Research at the Sheikh Zayed Institute for Pediatric Surgical Innovation.

What if doctors could measure pain as precisely as they measure blood pressure? That’s the vision driving Julia Finkel, MD, a pediatric anesthesiologist and director of Pain Medicine Research at the Sheikh Zayed Institute for Pediatric Surgical Innovation. Recently featured in The Washington Post, Dr. Finkel is pioneering the Nociometer, a new device that could transform how we understand and treat pain.

This new innovation uses a painless electrical current on a finger or toe to gently stimulate the body’s three main sensory nerve fibers. It then measures pupil response — linked to the brain’s pain centers — to identify the type and intensity of pain.

Now heading into clinical trials, the Nociometer shows how federal funding fuels innovation for patients. Here, Dr. Finkel shares the inspiration behind her work and the vital role of federal support in pediatric research.

Q: What started you on the path to developing this device?

A: My father is a theoretical physicist. He often talked in lay terms about everything he was thinking. When I was a child, we’d go for walks at night and look at the stars, and he’d explain the universe. My mother was an artist, but she struggled with constant pain from rheumatoid arthritis. There weren’t very good therapeutics at the time. Pain impacted her quality of life.

As an undergraduate, I was a chemistry major and interested in the mechanisms of how things worked. I planned to be a rheumatologist, but switching to anesthesia was transformative.

Q: What did you love about anesthesiology?

A: I love how it profoundly impacts patients. You induce unconsciousness, you induce wakefulness and you eliminate pain. When pain stops for a child, it also lessens the suffering of the parents. Our research is all about gaining a deeper understanding of the mechanisms of pain.

The Nociometer helps identify the mechanism behind the pain symptom so we can treat it appropriately. Research shows us that pain has a recognizable signature in the body. Because we can recognize it, we can treat it better. It’s like having an X-ray to look at before treating a broken leg.

Q: What is it like to work with children experiencing chronic pain? Why is measuring pain this way more effective than the typical scale?

A: Families become desperate. Often, they have seen many doctors before coming to a specialist. But pain is a single word for many different disease states. Not everyone has the right expertise. We often see children with chronic abdominal pain. Perhaps they struggle in school or can’t go at all. They don’t eat well, they have trouble engaging in regular activities. The Nociometer helps us determine the cause of the pain to better treat the problem. The device also gives us a way to validate pain while the pain scale tells us perception. The Nociometer provides objective data, very similar to a blood pressure reading.

Q: How has federal funding, and support from Children’s National, shaped your work?

A: Federal funding has been absolutely critical. Initially, we had grants from multiple agencies within the National Institutes of Health (NIH). This shows how ubiquitous and problematic pain is across the board. It’s the underpinning of many disease states and an upstream driver of the opioid epidemic.

We have grants from the Small Business Innovation Research program within the National Cancer Institute. We have funding from the National Institute of Arthritis and Musculoskeletal and Skin Diseases and an award from the Advanced Research Projects Agency for Health. This federal funding comes with scientific rigor and the ecosystem at Children’s National has allowed me to accomplish so much.

Q: What makes Children’s National the right place for this kind of innovation?

A: Children’s National has given me the space to think creatively and pursue unconventional ideas. In 2011, I became a founding member of the Sheikh Zayed Institute for Pediatric Surgical Innovation. With early institutional support, I was able to explore questions that led to the discovery of a physiological biomarker for pain — the basis of the Nociometer.

“Creative” isn’t a word you often hear in medicine, but here, it’s part of the process. I was able to spin out a company, secure funding and build a prototype — steps that wouldn’t have been possible elsewhere. The culture at Children’s National embraces discovery and fuels real impact.

Julia Finkel, MD, is a pediatric anesthesiologist and Director of Pain Medicine Research and Development, The Sheikh Zayed Institute for Pediatric Surgical Innovation and Professor of Anesthesiology, Pediatrics and Critical Care Medicine at The George Washington University.

Perspective: Rethinking racial benchmarks in pediatric safety events

Smiling boy in hospital bed

The research team estimated that 754 pediatric safety events could be avoided annually if all racial groups performed as well as the top-rated group for each type of safety event studied.

Quality and safety would improve in pediatric healthcare – for all races – if the practice of benchmarking outcomes and performance against white patients changed. Instead, investigators should consider using the best-performing group to improve healthcare disparities, according to a new Pediatrics perspective from a multi-center research team led by Children’s National Hospital.

The team examined seven pediatric safety indicators from their previous research, including postoperative infection and accidental lacerations. They determined the best-performing group was not always the white subset, but data in scientific research is often benchmarked against white patients. This can perpetuate racism and erroneously imply that white patients should define the standard for healthcare, the investigators found.

The patient benefit

The research team estimated that 754 pediatric safety events could be avoided annually if all racial groups performed as well as the top-rated group for each type of safety event studied. For some safety events, this would require benchmarking against Black, Hispanic, Asian and Pacific Islander populations.

“By looking at the data with a new lens, we believe that clinicians and researchers could make a major step forward in shrinking and eliminating disparities across pediatric healthcare,” said Kavita Parikh, M.D., MSHS, medical director of Quality & Safety Research and research director of the Division of Hospital Medicine at Children’s National. “If we can employ a more precise understanding of patient and family experience as they interact with the healthcare system, we can prevent negative outcomes that can impact entire lifetimes.”

What’s ahead

The multi-center team continues to work on ways to improve quality and safety at pediatric hospitals. With broad partnerships, the researchers hope to redesign national safety models to encourage hospitals to overcome health-equity hurdles, such as racism, language barriers and other social factors that lead to disparities.

“This call to action is not new, but it demands renewed urgency,” said Dr. Parikh, the first author of the perspective. “We propose future analyses that infuse antiracist principles with quality improvement and patient safety standards to design more effective interventions.”

You can read the complete perspective – “Analyzing Pediatric Safety Events Using Antiracist Principles” – in Pediatrics.

Ten sustainable steps to mitigate the climate crisis

Refinery in Corpus Christi , Texas

A new manuscript published in the Cambridge University Press outlines 10 actionable steps that infectious diseases professionals can take to reduce their environmental impact.

The U.S. healthcare sector contributes disproportionately to greenhouse gas emissions. A new manuscript published in the Cambridge University Press outlines 10 actionable steps that infectious diseases professionals can take to reduce their environmental impact, from better waste management to promoting telehealth and advocating for hospital-wide sustainability initiatives.

Shreya Doshi, M.D., pediatric fellow at Children’s National Hospital, shares some key highlights from this manuscript below:

Q: What’s been the hold-up in the field?

A: Despite the healthcare sector’s significant contribution to greenhouse gas emissions, we are not doing enough as a community, a nation and as an industry. Much more needs to be done, and a lack of awareness is a major barrier. To truly protect children’s health and keep them out of hospitals, we must focus on reducing harm to their environment. Currently, the healthcare industry is responsible for approximately 8.5 percent of total emissions in the U.S.

Q: How will this benefit patients?

A: By adopting sustainable practices, healthcare systems can reduce pollution and its related health effects. Simultaneously, the resource-efficient use of medical supplies and medications can also lead to more cost-effective care without compromising patient safety, ultimately leading to better health outcomes.

Q: How is Children’s National leading in this space? How unique is this work?

A: This work stands out by focusing on simple, actionable steps that everyone can take, while emphasizing the leadership role that infection preventionists, antimicrobial stewards, and other ID professionals can play in advancing healthcare sustainability. We also have a hospital-wide Sustainability Council that fosters interdisciplinary collaboration. To get involved, email Sustainabilitycouncil@childrensnational.org to join!

You can read the full study, 10 sustainable steps infectious disease professionals can take to mitigate the climate crisis, in the Cambridge University Press.

Marc Levitt, M.D., reflects on the Colorectal & Pelvic Reconstruction Program

Dr. Marc Levitt with a young patient

Dr. Levitt shares insights into the program’s journey, key successes and future goals.

This September, the Colorectal and Pelvic Reconstruction Program at Children’s National proudly marks five years of commitment to patient-centered care for children and their families. Under the guidance of Division Chief Marc Levitt, M.D., the program has achieved remarkable advancements in clinical care, improving patient outcomes and enhancing family experiences.

Dr. Levitt shares insights into the program’s journey, key successes and future goals, highlighting the importance of collaboration among specialists and the continuous pursuit of innovative treatments.

Q: What was your vision for the program when it first launched? Has the vision changed?

A: When the Colorectal Program first launched, we aimed to establish a comprehensive program for colorectal patient care. This included integrating specialists across multiple disciplines, such as pediatric general surgery, urology, gynecology, gastroenterology, nursing, neurosurgery, orthopedics, pathology, radiology, anesthesia, psychology and pelvic floor therapy. Our efforts have exceeded expectations; the team has developed a cohesive and collaborative dynamic where each specialist contributes effectively to patient care. This integrated approach enhances outcomes, as patients and their families are at the center of our model, surrounded by a network of dedicated caregivers and coordinators, all focused on improving the patients’ quality of life.

Q: Can you highlight some key successes or achievements of the program?

A: The creation of our integrated colorectal care program at Children’s National has been a significant success, bolstered by strong institutional support. While this model requires considerable effort, it ultimately attracts patients and leads to outstanding outcomes. We’ve received patient inquiries from 48 states (AK,AL, AR, AZ, CA, CO, CT, D.C., DE, FL, GA, HI, IA, ID, IL, IN, KS, KY, LA, MA, MD, MI, MN, MO, MS, MT, NC, ND, NE, NH, NJ, NM, NV, NY, OH, OK, OR, PA, RI, SC, TN, TX, UT, VA, VT, WA, WI, WV) and 68 countries (Australia, Bahamas, Bangladesh, Bulgaria, Canada, Chile, Croatia, Cyprus, Dominican Republic, Ecuador, Egypt, England, Ethiopia, France, Germany, Greece, Guatemala, Haiti, Hong Kong, India, Ireland, Israel, Jamaica, Jordan, Kazakhstan, Kenya, Kuwait, Lithuania, Maldives, Mauritania, Mexico, Monaco, Morocco, Myanmar, The Netherlands, New Zealand, Nepal, Nigeria, Northern Ireland, Norway, Oman, Palestine, Pakistan, Paraguay, Papua New Guinea, Peru, Philippines, Portugal, Romania, Russia, Saint Lucia, Saudi Arabia, Scotland, Serbia, Singapore, Slovakia, Slovenia, Spain, Sri Lanka, South Korea, St. Kitts & Nevis, Sudan, Sweden, Ukraine, Uzbekistan, United Arab Emirates, Venezuela, Wales).

Some key successes include the development of innovative surgical procedures for conditions such as imperforate anus and anorectal malformations, advanced repair techniques for cloacal malformations, integrated care strategies for colonic dysmotility, and novel approaches for the newborn care of patients with cloacal exstrophy. All these new approaches were developed at Children’s National over the past five years.

In addition to clinical innovations, we have significantly enhanced our academic environment. Over the last five years, our team has authored more than one hundred articles and three well-regarded books, helping health care professionals around the world improve the care they provide to their patients. We have also trained surgical fellows and nurses in colorectal care and welcomed clinicians from over 30 countries for specialized training. This outreach is especially fulfilling, as it enables us to influence the care of children around the globe whom we may never meet, by sharing valuable skills and knowledge with these practitioners. 

Q: Can you share examples of particularly challenging cases or high-profile cases that have influenced the program’s clinical approach?

A: The way we care for a patient with a cloaca is unique – the entire team joins together in the operating room to assess the anatomy, and every member then gives their thoughts on how to approach the reconstruction. The very next day we perform that surgery, with the understanding of the anatomy in mind, and what plays out in that operating room is quite magical. This collaborative approach frequently leads to exceptional outcomes, as our diverse ideas come together to form a cohesive plan. Through this teamwork, we have developed creative ways to solve complex anatomical problems that no one individual surgeon would have thought of on their own.

Q: How have patient outcomes improved over time?

A: We have optimized the collaborative experience so that patients only need a single visit to see all the specialists they require. Our outcomes for complex colorectal surgeries have significantly improved, particularly for conditions like cloaca, Hirschsprung disease and anorectal malformations. Advances in surgical techniques and enhancements in nursing care have led to dramatically better results. We have reduced, and in some cases eliminated, complications from these surgeries, while also seeing improvements in bowel continence and kidney health. Our nursing teams — outpatient, operating room and inpatient — play a crucial role, offering unique insights into pre- and post-operative care that are vital for achieving successful outcomes.

Q: What have been the biggest challenges or barriers faced by the program and how have you addressed these challenges?

A: One of the biggest challenges we face is ensuring that patients can get to us. Many patients encounter obstacles with insurance companies that restrict travel outside their network, or they deal with the difficulties of traveling from far away for their surgery. We have worked diligently to address these issues by improving our insurance approval process and making families comfortable during their stay in Washington, D.C., including assistance with accommodations, necessary medical supplies, etc. If we can remove these barriers, we can care for more patients and make the experience easier for them and their families.

Q: How does the colorectal program collaborate with other departments or services? How has the program integrated from various specialties (e.g., urology, gynecology, GI) to enhance patient care?

A: We have specialists who are fully integrated in their roles on the colorectal and pelvic reconstruction team. General pediatric surgeons, urologists, gynecologists and gastroenterologists work both in the colorectal program and in their home program in a uniquely integrated fashion. For example, our urologists each spend half of their time in general urology and the other half in colorectal, handling the urologic aspects of colorectal patients’ care. In this way collaboration is enhanced and encouraged.

Q: What are the future goals or plans for the colorectal program?

A: Our future goals for the colorectal program are focused on expanding our impact and enhancing patient care. First and foremost, we aim to help as many patients as possible. We plan to collaborate with other colorectal centers worldwide to share knowledge and best practices. Training surgeons and nurses remains a priority, as we want to equip as many healthcare professionals as possible with the skills needed in this field. Additionally, we are committed to traveling to the developing world to provide care for children who cannot access services at Children’s National. Finally, we intend to enhance our research efforts, leveraging basic science to investigate and address conditions related to the colon, ultimately seeking to reduce or eliminate associated illnesses.

Celebrating excellence: Children’s National Spina Bifida Program earns SBA accreditation

Spina Bifida Association

We are thrilled to announce that the Children’s National Spina Bifida Program has received official accreditation from the Spina Bifida Association (SBA).

We are thrilled to announce that the Children’s National Spina Bifida Program has received official accreditation from the Spina Bifida Association (SBA). This is a significant milestone that underscores our commitment to delivering the highest standards of clinical care and reflects the dedication of our entire team.

To gain a deeper understanding of what this accreditation means, we spoke with Christina Ho, M.D., Co-Director of the Children’s National Spina Bifida Program. Dr. Ho provided valuable insights into the significance of this achievement and its impact on the team and Spina Bifida Program.

Q: What does this accreditation mean to you and our program? 

A: Achieving accreditation from the SBA signifies that our program is aligned with the best clinical practices and standards for spina bifida care. Over the past few years, we have made substantial strides, particularly in enhancing our adolescent transition program, implementing quality improvement projects and expanding community outreach.

This accreditation is a testament to the relentless efforts of our multidisciplinary team, each member of whom is committed to providing exceptional patient care. It acknowledges the hard work and dedication we invest daily to ensure the highest quality outcomes for our patients.

Q: How will this affect patient care?

A: As an accredited clinical care partner, we will have increased opportunities to collaborate on research projects with the SBA and other clinical care sites. This partnership will enable us to continue pushing the envelope on research-focused practices and cutting-edge care for all our patients. Our passion for excellence in patient care remains unwavering, and this accreditation will help us expand the breadth and quality of services and support we provide.

Q: What does this mean for patient families?

A: For families seeking comprehensive spina bifida care, this accreditation is a mark of assurance. By visiting the SBA website, families can now see that Children’s National Hospital is a recognized clinical care partner. This means they can access specialized, state-of-the-art spina bifida care right here in the Washington DC, Maryland and Virginia area. We are excited about the prospect of reaching more families in our region and welcoming those who seek the very best in spina bifida care.

A note on our team

Our program’s success is a direct result of the passion and dedication of our team members. Each individual brings an unwavering commitment to improving the lives of families living with spina bifida. It is this collective enthusiasm and expertise that truly distinguishes our program.

We look forward to continuing our mission of providing exceptional care and making a positive impact on the lives of those we serve. Thank you for your ongoing support and dedication.

Learn more about Children’s National Spina Bifida program.

Children’s National at the 104th Annual AATS Meeting

Attending the American Association for Thoracic Surgery’s 104th Annual Meeting this weekend? Stop by the Children’s National Booth #1315! Here is a look at the topics that our Childrens National Heart Center will be presenting on. We look forward to connecting with you in Toronto.

Name Session & Role Topic Type Date Time Location
Can Yerebakan, M.D. Congenital Scientific Session: Strategies for Management of the Borderline Heart

(Speaker)

 

Debate: Hybrid is the Default Pathway- Pro Oral Presentation Saturday, April 27, 2024 8:50 a.m. Room 716
Yves d’Udekem, M.D., Ph.D. AATS/CHSS Summit: Hypoplastic Left Heart Syndrome

(Commentator)

 

Are We Getting better? Ongoing Challenges of Atrioventricular Valve Repair in Single Ventricle Patients with Right Ventricular Morphology Oral Presentation Saturday, April 27, 2024 1:30 – 3:56 p.m. Room 716
Yves d’Udekem, M.D., Ph.D. Congenital Rapid Fire Orals – Theater Session I

(Case Video Presenter)

Early Pulmonary veins Channeling in a Complex Heterotaxia Heart wit Oral Presentation Sunday, April 28, 2024 9:12 a.m. Theater 1
Eiri Kisamori, M.D. Congenital Rapid Fire Orals

(Presenter)

Alarming Rate of Liver Cirrhosis After the Small Conduit Extracardiac Fontan.

A Comparative Analysis With the Lateral Tunnel.

Abstract Presentation Sunday, April 28, 2024 11:43 a.m. Room 716
Rittal Mehta, MS, BDS Congenital Poster Session II

(Presenter)

Navigating the Future of Pediatric Cardiovascular surgery: Insights and Innovation powered by ChatGPT. Poster Presentation Monday, April 29, 2024 8:00 a.m. Poster Area
Can Yerebakan, M.D. Congenital Poster Session II

(Presenter)

Can Delayed Norwoods in High Risk Patients Achieve Similar Results than Primary Norwood for Low Risk Patients. Poster Presentation Monday, April 29, 2024 8:00 a.m. Poster Area
Arif Selcuk, M.D. Congenital Rapid Fire Orals – Theater Session III

(Presenter)

Initial Rescue of a High-Risk Newborn by Atrial Kissing Procedure’ for Left Atrium Decompression and Bilateral Pulmonary Artery Banding. Case Video Presentation Monday, April 29, 2024 3:51 p.m. Theater 1
Yves d’Udekem, M.D., Ph.D. Congenital Disasters and Rescues

(Speaker)

Mitral Valve Repair Techniques in Neonates and Infants’ Rather than a Specific Situation? Oral Presentation Tuesday, April 30, 2024 8:45 a.m. Room 716

Supporting breakthroughs in at-home pediatric monitoring

Baby wearing Gabi SmartCare device

The child-sized, lightweight, wearable band enables at-home monitoring of patient vital signs.

As their infant daughter recovered from a life-threatening virus, tech executive Jonathan Baut and his wife lived in a constant state of alert for any signs of a change in her condition. That experience prompted Baut to look for an at-home vital signs monitoring solution for parents facing medical challenges at home.

He located the technology in Belgium and found the clinical support to advance the innovation at Children’s National Hospital, tapping into its leadership of a pediatric device consortium funded by the Food and Drug Administration (FDA), the Alliance for Pediatric Device Innovation (APDI).

The technology platform, called Gabi SmartCare, features a tiny, lightweight, wearable band made for children that enables at-home monitoring of a patient’s vital signs including oxygen saturation,  and heart rate movement. It also supports the collection of other data about health conditions through health assessment.

The big picture

Wireless home-based monitoring could reduce hospital stays while improving the transitional care provided to patients at home. It could also aid in the home care of chronically ill patients.

Recently, Gabi SmartCare received its FDA clearance as a class II device for monitoring use. Baut says the multifaceted support he received from Children’s National and APDI helped him achieve this critical milestone, including the hospital’s role as one of two U.S. clinical trial sites for the device.

He explained that the APDI team also connected him with experts in device trials, regulatory processes and reimbursement, which helped him better understand the U.S. market and the nuances of the pediatric continuum of care.

Gabi SmartCare monitoring screens

Physicians can remotely monitor patient’s oxygen saturation and heart rate movement.

As he focuses on getting his product into hospitals, Baut already has additional monitoring features in development for Gabi SmartCare including respiratory rate, actigraphy, skin auto-calibration and sleep phases.

Why it matters

Reliable at-home monitoring tools can expand a pediatric hospital’s capabilities to support patients beyond the hospital setting. They can also help reduce trips to the emergency room and reduce the stress and anxiety parents experience when providing home care. Helping to advance devices like these can deliver those benefits to patient families at Children’s National and beyond.

“The pandemic underscored a great need for technologies that improve remote monitoring for children,” said Kolaleh Eskandanian, Ph.D. M.B.A., P.M.P., Children’s National vice president and chief innovation officer and program director of APDI. “At-home monitoring devices enable remote and continuous surveillance of pediatric patients, ensuring timely intervention and optimal care delivery even in remote or resource-limited settings. These technologies are needed now, and even more during public health emergencies.”

The division of urology creates tailored orientation program

The Division of Urology at Children’s National has developed a first-of-its-kind orientation program for pediatric urology advanced practice providers (APPs). This program was created based on real feedback from pediatric urology APPs in the United States and Canada.

The primary purpose of the orientation program is to increase the knowledge and confidence of pediatric APP’s in transition to independent practice.  The program prepares the next group of advanced practice providers in the Division of Urology to achieve the same high quality of care that the current group provides to our patients.

“I think what makes our orientation program unique is how involved leadership is in training the orientee. We are with our orientee every step of the way every week. We are there making sure that they are attaining their skills and their knowledge. We’re checking in on them very regularly, and we also are trying to make it very individualized to that person.” said Katie Scarpaci, nurse practitioner and creator of the APP orientation program.

Global summit to address complex pediatric scoliosis

ICEOS logoThe 2023 International Congress on Early Onset Scoliosis and the Growing Spine (ICEOS) was a three-day conference for surgeons and health professionals from around the world. The sessions focused on solutions for pediatric spine deformities. Matthew Oetgen, M.D., chief of Orthopaedic Surgery and Sports Medicine at Children’s National Hospital, was a member of the faculty.

Session highlights included:

  • Pre-Course: Pediatric Spine Study Group (PSSG) Mini-Research Symposium – Considerations for your biosketch, regulatory and IRB
  • How Do You Propel Your Research Team

“Overall this was a great meeting with valuable content,” says Dr. Oetgen. “Learning and teaching alongside fellow orthopaedic leaders is a fantastic opportunity for everyone involved.”

Dr. Oetgen participated in the following sessions:

  • Timing of Hemivertebra Excision
  • Let’s Debate! nView Workshop – 3D imaging and guidance, available technologies for pediatric spine and when to use them
  • Case-Based Learning – Congenital Scoliosis

The 2024 ICEOS Meeting will be November 13-15 in Phoenix, AZ.

Hip preservation: Moving the needle in patient care

x-ray showing a hip

Hip preservation treats a wide variety of conditions such as hip dysplasia, hip impingement, hip torsion/rotation abnormalities, hip synovitis, snapping hip and hip labrum tears.

The Hip Preservation Program at Children’s National Hospital offers advanced care to children and adults with hip pain, providing a continuum of care that extends well into adulthood. Jeffrey Peck, M.D., an orthopaedic surgeon with subspecialty interests in pediatric and young adult hip preservation, discusses the program and what Children’s National is doing to move the needle forward in patient care.

Q: What is hip preservation and what are common conditions it addresses?

A: The field of hip preservation is dedicated to diagnosing and treating hip conditions that result in hip pain and can potentially result in hip degeneration.

There are several potential conditions that can lead to hip pain and eventual hip joint degeneration.  These include atypical anatomy in and around the hip joint, which can be congenital, developmental or traumatic in origin. Strains and sprains of the muscles and ligaments around the hip may also cause hip joint pain. Hip preservation treats a wide variety of conditions such as hip dysplasia, hip impingement, hip torsion/rotation abnormalities, hip synovitis, snapping hip and hip labrum tears.

Q: Who is affected and when should a patient seek out an orthopaedic expert?

A: The people most often affected by conditions treated through hip preservation are adolescents and young adults up to age 45 years old who develop hip pain. When hip pain does not subside, it can be due to a condition that has resulted in hip joint inflammation that may eventually begin to cause hip joint degeneration leading to arthritis if the condition is not treated. If a patient has hip pain that lasts for multiple months without improving, it is prudent to seek evaluation with a hip preservation orthopaedic surgeon specialist.

Q: What is Children’s National doing to move the needle for hip preservation patients?

A: Children’s National cares for patients with hip pain using an integrated care team, featuring a Hip Preservation fellowship trained surgeon, physiatry experts, sports psychologists, radiologists and physical therapists with a focus on adolescents and young adults. We work in a collaborative environment with the shared goal of helping patients with hip pain return to living life without the pain that has held them back.

The Children’s National team also collects patient reported outcome scores to better understand the severity of patient symptoms and, later, how surgery has improved those symptoms. Additionally, Children’s National is engaged in research endeavors evaluating hip conditions and how patients have responded to treatment. By pursuing these efforts, we not only assist the patients we see today, but also contribute to advancing care to better help future patients.

Q: What excites you about the future for the field of hip preservation?

A: The field of Hip Preservation is a young specialty within orthopaedic surgery that has demonstrated consistently positive outcomes for patients who previously had very limited treatment options for their hip pain. With this comparative newness comes continual innovation and advancement as we learn more about how hip pain can occur and what we can do to best treat that pain. Procedures that we perform at Children’s National such as the Ganz Periacetabular Osteotomy (PAO) and the use of hip arthroscopy to treat hip impingement are relatively new innovations in the field of orthopaedics that can provide immense relief to patients having hip pain.

Revolutionizing pediatric nephrology one patient at a time

Marva Moxey-Mims

“With each kidney transplant, we’re not just restoring the health of children, we’re pioneering progress and setting new benchmarks for the field of pediatric nephrology,” says Marva Moxey-Mims, M.D., chief of Nephrology at Children’s National Hospital.

“With each kidney transplant, we’re not just restoring the health of children, we’re pioneering progress and setting new benchmarks for the field of pediatric nephrology,” says Marva Moxey-Mims, M.D., chief of Nephrology at Children’s National Hospital. “We offer not just treatment, but a chance for our patients to flourish and live their best life.”

Big picture

The Kidney Transplantation Program at Children’s National is the only one of its kind in the Washington, D.C., region focused on the needs of children and teens with kidney disease.

Performing an average of 15-20 kidney transplants per year, the program’s latest one- and three-year graft and patient survival data from the Scientific Registry for Transplant Recipients is 100%.

“Our program surpasses national numbers, reflecting a commitment to exceptional care, superior patient outcomes and a focus on setting a new standard for excellence in transplant support,” says Dr. Moxey-Mims.

Children’s National leads the way

At the forefront of groundbreaking treatment, the nephrology team is pioneering progress by providing innovative care and resources. One example is the hospital’s collaboration with the National Kidney Registry and MedStar Georgetown Transplant Institute’s Living Donor Program. This provides a greater chance of finding more suitable donors for difficult-to-match children and has resulted in altruistic donations.

“Through living kidney donations, we redefine the timeline for pediatric transplants. Children experience shorter waits and swifter paths to renewed health compared to the national wait list,” says Asha Moudgil, M.D., medical director of the Kidney Transplantation Program at Children’s National. “It’s a testament to our commitment to making a difference in every child’s life.”

That commitment is also seen in the hospital’s focus on making sure families have the resources they need during their medical journey.

“Caring for a child with renal disease is an immense challenge, not only for the young patients but also for their families. The demanding schedule of hospital visits, often three times a week, adds an extra layer of stress and disruption to daily life,” says Dr. Moudgil.

Through grant funding from donors and national programs, families with children undergoing kidney transplants and dialysis can receive financial assistance. In some cases, families receive up to $2,000 of mortgage or rental payment assistance. Families can also receive funds for medication co-pays and other expenses incurred around the time of the transplant.

“In the face of medical adversity, these funds become a source of support,” says Dr. Moudgil. “We make sure every family, regardless of financial constraints, can access the care and resources their child needs.”

Bottom line

Children’s National is at the forefront of pediatric kidney transplantation. “Our unique approach ensures every facet of a patient’s journey is considered,” says Dr. Moxey-Mims. “We’ve created an environment where success is not just a medical outcome but a comprehensive and sustained improvement in a child’s overall quality of life.”

Learn more about our latest advances in pediatric Nephrology.

Cell & Gene Therapy in the DMV: Experts collaborate for cures

Leaders in medicine, academia, industry and state and local government came together for the first annual Cell and Gene Therapy in the DMV Symposium, hosted at the Children’s National Research & Innovation Campus. The mission: Connect the local scientific community – bursting with expertise and collaboration potential – to develop these cutting-edge therapies for cancers, sickle cell disease and immune-mediated disorders.

The daylong event drew over 100 experts from a range of organizations in the D.C, Maryland and Virginia region, sometimes called the DMV: Children’s National Hospital, the Food and Drug Administration, the National Institute of Standards and Technology, the National Institutes of Health, the General Accounting Office, Virginia Tech, MaxCyte, AstraZeneca, Kite Pharma, Montgomery College, the Maryland State Department of Commerce and more. Together, they unraveled a host of topics including the regulatory environment, workforce development and training, research standards and the promise of these therapies.

“Our Cell & Gene Therapy Symposium brings together our current collaborators and future partners in the D.C., Maryland and Virginia space, which is an incredibly rich area. We see tremendous opportunity and breakthroughs in our future,” said Catherine Bollard, M.D., M.B.Ch.B., interim chief academic officer and chief of Pediatrics at Children’s National Hospital. “Many different diseases rely on the immune system to either be ramped up or to be controlled, and we can seize on these biological processes. Cell and gene therapies are at the heart of where medicine is going.”

The big picture

For decades, oncologists largely have turned to the same menu of treatments to fight cancer, including surgery, chemotherapy and radiation. Cell and gene therapies offer the promise of training the immune system to fight diseases with fewer side effects and potentially higher success rates. Early work has shown progress in liquid cancers, like leukemia, raising the possibility that the therapies could be used on solid tumors and other disorders, such as lupus and sickle cell disease. However, many disciplines must come together to yield discoveries.

“Nobel Prize-winning work doesn’t necessarily translate into available therapies for patients. It takes a whole community like this to make it happen,” said Cenk Sumen, chief scientific officer at MaxCyte Inc., an international cell engineering company based in Rockville, Md. “It has been exciting to see this diverse group of stakeholders come together, which is probably unmatched anywhere on the planet.”

Why we’re excited

Symposium host Patrick Hanley, Ph.D., chief and director of the Cellular Therapy Program at Children’s National, said the goal was to cement the region as the No. 1 location for this highly technical research and development. He believes Children’s National can offer essential elements to this success, given its clinical and research expertise, workforce training opportunities and geographic proximity to the scientific leadership of the federal government. “What makes us unique is our proximity to all the players who can help create new treatment options for patients. We truly are the biomedical capital of the world,” he said.

Michael Friedlander, vice president for health sciences at Virginia Tech, notes that the earliest stages of invention will emanate from academic labs including those at Virginia Tech and Children’s National. “You have basic scientists who are doing fundamental research on properties and procedures that will lead to the new therapies of tomorrow,” he said. “We are putting in place the fundamental pieces to advance children’s health in all dimensions.”

What’s ahead

One challenge is developing a workforce to help prepare cell therapies for patients, following precise standards to ensure the therapy works as designed. Children’s National does this training, as do others in the region. Lori Kelman, Ph.D., M.B.A., biotechnology coordinator and professor at Montgomery College, said that the area is full of people who want to help people and who like science.

“The thing that people might not know is that you don’t need a Ph.D. to work in cell and gene therapy,” she said. “There are opportunities at all levels, including the entry level, which is where a great career often starts.”

A team approach to complex bronchopulmonary dysplasia

“By the time a baby is diagnosed with bronchopulmonary dysplasia, families have already had a long journey with prematurity in the neonatal intensive care unit (NICU),” says Hallie Morris, M.D., neonatologist and lead of the Complex Bronchopulmonary Dysplasia (BPD) Program at Children’s National Hospital. “To be able to have a team that is focused on the holistic health of their child in the context of this diagnosis makes a world of difference to these families.”

The big picture

Some premature infants with BPD experience more severe respiratory disease with comorbidities associated with their underlying disease processes, but also factors related to their lengthened intensive care unit (ICU) stay. This includes delayed development with neurodevelopmental impairment, ICU delirium, pulmonary hypertension, airway disease, gastroesophageal reflux disease, feeding difficulties, retinopathy of prematurity and more.

The Complex BPD Program at Children’s National encompasses a group of specialists dedicated to improving the care of infants with BPD and other chronic lung disease of infancy. BPD places extreme demands on families. Education is a critical component for families and our team works to make sure they are well informed, have realistic expectations and understand their care plan.

What they’re saying

  • “Our program is unique in that it has the ability to follow the patient for several months in the NICU as well as after discharge,” says Maria Arroyo, M.D., pulmonologist and co-lead of the Complex BPD Program at Children’s National. “This includes a subacute facility where some of our NICU patients transfer to for continued respiratory weaning and rehabilitation with parent education and outpatient visits once families are home.”
  • “Since this program was created, we have improved patient care and outcomes with this interdisciplinary approach,” says John Berger, M., medical director for the Pulmonary Hypertension Program at Children’s National. “We expect that with our consistent and personalized care, patients will continue have better overall outcomes, less readmissions and improved neurodevelopmental outcomes.

Learn more about the Complex BPD Program.

State of Celiac Disease in the United States

USA line art map with a wheat plant iconVahe Badalyan, M.D., director of the Children’s National Celiac Disease Program, discusses the current state of celiac disease in the United States, including diagnosis and care among pediatric populations.

Q: In your opinion, how would you classify the state of Celiac Disease among children in the United States as it compares to 10 years ago?

A: Celiac disease in the United States is increasingly recognized as a common disorder of childhood. While this can partly be attributed to improved recognition and more testing by primary care providers, “true incidence” of this immune-mediated disease is also said to be rising, independent of the screening practices.

While there is a larger variety of gluten-free food options available today, these options are often more costly than their gluten-counterparts. Additionally, gluten-free food options are not covered by insurance companies. With inflation and rising food prices, there is a real concern for increased economic burden and food insecurity for the families of children with celiac.

Q: Are there any misnomers about Celiac Disease that you think are important to address?

A: Classic symptoms of celiac disease, including diarrhea and malnutrition, now occur in a minority of celiac patients. There is a rising proportion of milder or asymptomatic presentation at the time of diagnosis. Paired with the fact that most celiac patients identified through mass-screening did not have a family history of celiac disease (~90%) or gastrointestinal symptoms (70%), one may need to have a high index of suspicion for children with atypical presentation, such as neurologic symptoms, iron and vitamin D deficiency.

Q: What excites you about the future of treatment for Celiac Disease?

A: I am encouraged that researchers across the globe are testing new, pharmacologic therapies for celiac disease. Some of the tested compounds, such as latiglutenase or TAK-062 , involve breaking down gluten-using enzymes, while others, such as TAK-101 aim to induce immune tolerance. These compounds are still at the early stages of the long and arduous process of drug development and approval. Current randomized trials typically include adults, although some recently opened enrollment to adolescents. While there are no guarantees, I am cautiously optimistic that in the future we will have pharmacologic, non-dietary treatment options for celiac disease.

Q: How is Children’s National a leader in the field of pediatric Celiac Disease?

A: Concerns about gluten exposure can be quite stressful for our patients and families, which can lead to hypervigilance, anxiety and even depression. We are fortunate to have a full-time psychologist, Shayna Coburn, Ph.D., as part of our Multidisciplinary Celiac Disease Program team. This allows us to treat not only physiological symptoms and concerns associated with celiac disease, but to also evaluate the psychological problems that may arise as a result of the condition.

The future of Wilms tumor therapies: Q&A with Jeffrey Dome, M.D., Ph.D.

illustration of diseased kidneys

Dr. Dome’s mission is to come up with other therapies to treat Wilms tumor.

Conducting Wilms tumor research has placed Children’s National Hospital as the leader in this space. Jeffrey Dome, M.D., Ph.D., talks about the future of Wilms tumor therapies and what excites him about being able to offer different treatment options to this group of patients.

Q: What’s next for Wilms tumor therapy treatments?

A: Something we’ve learned is that we’re reaching the maximum benefit that we can achieve with conventional chemotherapy and have hit a plateau with some of the high-risk Wilms tumors.

If you look at the history of Wilms tumor, we’ve ratcheted up the cure rate by adding more and more chemotherapy agents. We’re now at a point in which patients can’t tolerate having more toxic chemotherapy added to their regimen. Our mission is to come up with other therapies that work differently.

Q: What have you learned that can lead to new therapy options?

A: I was a co-investigator on a National Cancer Institute-funded project called the TARGET study, which entailed sequencing several hundred Wilms tumors to identify genetic mutations that could be druggable. While a wealth of knowledge was gained on the nature of the biology of Wilms tumor, only a minority of Wilms tumors have targetable mutations. We understand what the mutations are, but most are not mutations that lend themselves to drug therapy.

Therefore, we must think about other forms of therapy for Wilms tumor, such as immunotherapy. That’s where the work with the T cells targeting the WT1 protein Wilms tumor cells come into play.

Q: What do the new therapies look like?

A: I think the future of Wilms tumor therapy will be combining the classic chemotherapy and radiation therapy that we’ve used for many years but also adding another component for the patients with the highest risk of relapse, such as immunotherapy.

Up until now, the studies that we’ve conducted using immunotherapy have introduced this treatment very late as a last resort after everything else has failed. We have seen early signals of efficacy and if we can corroborate this in current studies, I believe the future will be to introduce immunotherapy earlier in the treatment course.

There has been success using immunotherapy in other pediatric solid tumors such as neuroblastoma. I believe the prospects for Wilms tumor are also exciting.

 

Leading Wilms tumor research nationwide: Q&A with Jeffrey Dome, M.D., Ph.D.

histological image of Wilms Tumor

Children’s National has become a resource for patients and families with Wilms tumor.

During the past year, Children’s National Hospital saw nearly 100 patients with Wilms tumor and other less common kidney cancers of childhood, far more than most centers in the country. This is largely due to the reputation the hospital has established for specializing in these diseases. While most patients with Wilms tumor have excellent outcomes, a significant minority of children with kidney cancer do not fare well. Children’s National has become a resource for patients and families with these challenging cancers.

Behind this reputation is Jeffrey Dome, M.D., Ph.D., senior vice president of the Center for Cancer and Blood Disorders and division chief of Oncology at Children’s National, and the team of researchers he leads. For over a decade, he chaired the Children’s Oncology Group Renal Tumor Committee, an opportunity that gave him and his work great exposure.

Dr. Dome shares more on how Children’s National is leading in this space and what the future holds for new, exciting Wilms tumor treatment options.

Q: How is Children’s National leading in this space?

A: The good news is that for the most common type of childhood kidney cancer, Wilms tumor with “favorable histology,” the survival rate is more than 90%, which is an incredible success story. But approximately 25% of children and teens with other types of Wilms tumor and other kidney cancers do not fare as well. We specialize in kidney cancers that are harder to treat, such as anaplastic Wilms tumor, relapsed favorable histology Wilms tumor, bilateral Wilms tumor, clear cell sarcoma of the kidney, malignant rhabdoid tumor and renal cell carcinoma. Because we see a relatively large number of patients, we can draw on our prior experience and observations to recommend the best treatment options.

Q: What’s unique about this research?

A: We have several early-phase clinical trials that are of interest for children with relapsed kidney tumors. Some of these trials are part of research consortia, such as the National Cancer Institute-funded Pediatric Early Phase Clinical Trials Network (PEP-CTN). Other studies have been developed in-house at Children’s National, including a couple of studies using T cells to target pediatric solid tumors. The T cells that have been engineered by the Children’s National Cellular Therapy Laboratory are of particular interest for Wilms tumor because they target a protein called WT1, which is expressed in most Wilms tumors. In fact, WT1 was named after Wilms tumor. We have now had more than 25 patients with relapsed Wilms tumor come from around the country to participate in these studies. Based on early successes, we are continuing this line of research and trying to improve the technology in the current generation of studies.

In the news: People v. pediatric cancer

“I just want to hammer home the fact that, if you have a child with a pediatric solid tumor who relapses, most likely the chemotherapy that will be treating that child will be the same chemotherapy that a child diagnosed 20 years ago would have received. This is how little progress has been made…. This is what we are trying to change.”

Catherine Bollard, M.D., M.B.Ch.B., director of the Center for Cancer and Immunology Research at Children’s National Hospital, pulled the curtain back on her work fighting pediatric brain tumors at The Atlantic’s People V. Cancer summit. This annual event brings together leading voices from the front lines for in-depth conversations about how to stop this complex and lethal disease. Dr. Bollard discussed the unique importance of collaboration among pediatric oncologists and the optimism she has for using a patient’s immune system to go after solid tumors with CAR T therapies.

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

 

Increasing access to autism spectrum disorder services through enhanced training

Hands holding letters that spell autismMany service providers struggle to keep pace with advances in autism-specific knowledge and tend to refer children to autism specialty clinics when the diagnosis of autism spectrum disorder (ASD) is in question. Unfortunately, it is in these settings where children most often wait for months or, worse, experience barriers to accessing any care at all. This has resulted in an access crisis for children and families with ASD concerns contributing to delays in diagnosis and treatment, particularly for children of color and for under-resourced families. Service disruptions and challenges related to the COVID-19 pandemic have only added to delays. As the need for autism-related services continues to grow, innovative models must be used to enhance competence among frontline medical, behavioral health and community-based providers who currently serve these children and families on a regular basis.

Children’s National Hospital has initiated a number of endeavors focused on increasing access to ASD services through enhanced training experiences, mentorship of allied mental health and frontline professionals and utilization of multidisciplinary approaches. These approaches enhance the skills and knowledge of treatment providers, which allows them to accurately address the needs of autistic patients while they await more comprehensive evaluations and sometimes reduce the need for additional evaluation. The following are efforts currently underway.

Virtual ECHO (Extension Community Healthcare Outcomes) Autism Clinics

The Center for Autism Spectrum Disorders (CASD) is hosting virtual ECHO (Extension Community Healthcare Outcomes) Autism Clinics aimed at building autism knowledge and competencies amongst community providers by creating shared learning forums with a multidisciplinary group of autism specialists for dissemination of knowledge and mentorship.

Clinics run in 6-month sessions on a bimonthly basis and target professionals in medical, community and educational/early intervention settings. There is no requirement for prior autism-related knowledge or training. The emphasis in learning stems from case-based discussions primarily, along with targeted autism specific didactics.

We have found good satisfaction with the program overall, as well as self-report of gains in ASD-specific knowledge and care competencies as a result of participation in ECHO. To date, CASD’s ECHO Autism program has reached 290 professionals and trainees serving autistic children and their families.

Integration of autism evaluations into primary care sites

The Community Mental Health (CMH) CORE (Collaboration, Outreach, Research, Equity) within the Children’s National Hospital Child Health Advocacy Institute (CHAI) has been working collaboratively with several other divisions, including CASD, to integrate autism evaluations into primary care sites for young children with high concern about ASD. We aim to increase capacity and access to autism services by training embedded psychologists in primary care settings in autism diagnostics.

By increasing behavioral health provider capacity and integrating in primary care, this clinic has been able to drastically decrease waits for ASD services by months to years. Families served by the program were predominately Black (81%) or Latinx (10%), and most (87%) had public insurance. Nearly one third (32%) were not primary English speakers. An ASD diagnosis was provided in 68% of all cases.

All referring PCPs surveyed indicated that they were “satisfied” or “very satisfied” with the program, that they “strongly like the integrated clinic model,” and that the program “is increasing equitable access to ASD. Currently, CHAI-supported ASD-focused embedded clinics in primary care have served 94 children and their families.

Changing the surgical evaluation of epilepsy

brain network illustrationThe choice between stereoelectroencephalography (SEEG) and subdural evaluation is not mutually exclusive, according to a new opinion piece published in JAMA Neurology.

In their article, Chima Oluigbo, M.D., pediatric epilepsy neurosurgeon, William D. Gaillard, M.D., division chief of Epilepsy and Neurophysiology and Neurology, both at Children’s National Hospital, and Mohamad Z. Koubeissi, M.D., M.A., from The George Washington University Hospital, discuss how the practicing epileptologist requires a profound understanding of the roles of different technologies. It also looks at how to integrate both traditional and emerging paradigms to optimize seizure control. This issue is particularly relevant to choosing the best method of invasive intracranial electroencephalography monitoring in individual cases.

Noting that despite the dramatic increase in SEEG use in recent years, the authors talk about how many patients still benefit from invasive monitoring using subdural grids. Therefore, it is important to define the considerations that should guide decision-making on the choice of SEEG versus subdural monitoring in each patient. The authors expand on their statement explaining that it is critical to define the roles of SEEG vs subdural grid investigation in each patient as subdural grid evaluations are still indicated in specific circumstances.

Additionally combined hybrid deployment of both techniques may be indicated in specific situations. Accommodation should be made to allow customization of the technique chosen to available technical expertise and equipment as well as patient preference.