Midsection of a handicapped man and woman

Increasing the patient’s voice in osteogenesis imperfecta research and care

Midsection of a handicapped man and woman

The Osteogenesis Imperfecta Foundation Patient-Centered Outcomes Research Project brought patient voices into efforts to improve patient-centered outcomes research and education in osteogenesis imperfecta.

Rare bone diseases constitute about 5% of all known rare diseases. Osteogenesis imperfecta (OI) is a group of genetic disorders causing connective tissue dysfunction and bone fragility, affecting an estimated 25,000 to 50,000 people in the U.S. Many patients with rare diseases, including those with conditions like OI, suffer from delays in diagnosis and lack of access to the complex multidisciplinary care they require.

Children’s orthopaedic surgeon Laura Tosi, M.D., is the co-lead of a two-year program, Osteogenesis Imperfecta Foundation Patient-Centered Outcomes Research Project. The now completed program brought patient voices into efforts to improve patient-centered outcomes research and education in osteogenesis imperfecta.

Increasingly, the OI community has become more vocal about its need for information and options for evidence-based care. Research about this condition often lacks the patient’s voice. Collecting the patient’s perspective about natural history, clinical best practices, quality of life and research priorities is challenging because, like so many rare diseases, the affected population is relatively small and geographically dispersed.

“We were excited that so many members of the OI community (patients, caregivers, clinicians, and researchers) stepped up to help us with this work for the last two years,” said Dr. Tosi. “They served on committees, completed surveys, and attended training sessions to learn more about patient-centered outcomes research.”

The program accomplished many goals, including creating a community of stakeholders who are trained in patient-centered outcomes research, with specific attention to priority topics identified by the OI community.

“We believe that attention to the broad impact of OI on patients’ lives, from patients’ perspectives, is urgently needed to better inform assessment, care and comparative effectiveness recommendations across the life span,” said Dr. Tosi.

The program considered the patients’ perspective and encouraged a collaborative effort among Children’s National Bone Health Program staff, geneticists, endocrinologists and other specialists to achieve rapid, accurate diagnosis that facilitates individualized care.

“Advances in knowledge can and will happen faster when more people participate in research from the outset,” said Dr. Tosi. “This means not just shaping the questions but also gaining the ability to understand and interpret the impact of such investigations.”

The program also expanded the communications and education strategies related to patient-centered outcomes, even during COVID-19.

The pandemic positively influenced the program’s educational efforts, Dr. Tosi added. The difficulties that emerged in the early months of the pandemic helped capture the community’s attention as they discussed the importance of the program. Through a COVID-19 supplemental award, the program gained more resources to expand the scope of the original education efforts.

With the new challenges and the possible physical and mental health threats that the pandemic posed to people with OI, including isolation and social distancing, the team used engagement strategies to raise awareness of the COVID-19 hazards, disseminated best practices for responses, and enhanced communications between stakeholders. The researchers were able to:

  1. Expand virtual meetings, which allowed them to reach a wider range of patients.
  2. Create and disseminate COVID-19 content (e.g., prophylactic care, symptoms, diagnosis, treatment, outcomes).
  3. Use their existing OI Registry to document and track evolving issues such as more difficult access to medical care (or other necessary services), “daily living” concerns or burdens, and options for safe “return to work” and American Disabilities Act “work accommodations.”
  4. Gather information on therapeutic options (e.g., pros and cons of medicines, counseling, telemedicine) and their potential to mitigate the health problems that isolation and social distancing create.
  5. Develop tools to prepare the community for telemedicine, with emphasis on educating health professionals about patients’ higher vulnerability to coronavirus.

While the outcomes of this initiative were overwhelmingly positive, the researchers are also considering some changes, such as implementing “hybrid” models of in-person and remote meetings in the future.

Dr. Tosi served as project co-lead alongside colleagues Tracy Hart, project lead, from the Osteogenesis Imperfecta Foundation (OIF) and Bryce Reeve, Ph.D., co-project lead, director of the Center for Health Measurement at Duke University.

Matthew Oetgen

Matthew Oetgen, M.D., M.B.A, lauded for paper on treating Compartment Syndrome

Matthew Oetgen

For his work in advancing the care for children with complex orthopaedic needs, Matthew Oetgen, M.D., M.B.A., chief of Orthopaedics at Children’s National Hospital, recently accepted the award for Best Basic Science Paper at the 2021 Pediatric Orthopaedic Society of North America (POSNA) annual meeting. Dr. Oetgen co-authored the paper titled “Activation of A Central Immunosuppressive Cascade Prevents Ischemia Reperfusion Injury after Acute Compartment Syndrome in a Murine Model.”

Compartment Syndrome is rare and often difficult to diagnose. It occurs when interstitial pressure exceeds perfusion pressure and results in warm ischemia and cell death due to impaired aerobic metabolism. Following surgical decompression and reperfusion of the extremity, a robust innate inflammatory response results in further tissue injury due to the production of reactive oxygen species and local capillary dysfunction.

The authors described using varenicline, an FDA-approved medication for smoking cessation, to mitigate inflammation after ischemia reperfusion injury in murine models. Twenty-four hours after reperfusion, the treatment reduced acute leukocyte infiltrate, 7 days following reperfusion, the expression of pro-fibrotic genes was reduced and 14 days following treatment, histologic evidence of collagen deposition was also significantly reduced.

“The promising results of this study show that this medication may have the potential to blunt the immune response resulting in better outcomes for children with compartment syndrome,” says Dr. Oetgen.

Watch Dr. Oetgen’s presentation here.

x-ray of a fracture

Genetic disorders, including osteogenesis imperfecta, rarely mimic child abuse

x-rays of fractures

In suspected child abuse cases, pediatric specialists are often called for clinical consultations or subpoenaed to courtrooms to discuss unexplained fractures and hemorrhage. During routine clinical practice, Children’s National Hospital (CNH) geneticists, child abuse pediatricians, orthopedists and radiologists use a multidisciplinary, stepwise approach that differentiates genetic disorders from cases of suspected child abuse. These teams provide a clear process for when clinical and radiological review suffices versus cases when judicious use of genetic testing and biochemical testing should be considered.

Every year, approximately 675,000 children suffer abuse or neglect, and at least 1,700 die from abuse — one of the leading causes of childhood morbidity and mortality nationwide.

Ill-intended arguments regarding genetic disorders often reach the court. Out of the 7,000 known rare diseases, rare bone diseases constitute about 5% of the cases. When there are true genetic disorders like osteogenesis imperfecta (OI), a group of genetic disorders that cause fractures, orthopedic specialists help tremendously with diagnosis and treatment.  The Children’s National Bone Health Program specializes in caring for healthy children, children with genetic bone conditions, and children whose bones have been damaged from illness or poor nutrition. Their team of experts enhances treatment to meet the needs of each child help us provide the best possible care for children with a broad range of bone health conditions  When a rare condition that causes bone fragility is suspected, these teams work together to provide proper diagnosis and management.

“OI is a diagnosis that can be made clinically with the help of geneticists, radiologists and orthopedists,” said Tanya Hinds, M.D., a child abuse pediatrician at Children’s National. “Outside of the newborn period, multiple unexplained fractures in infants with radiologically normal bones is suspicious for child physical abuse, not OI.”

When these regional cases reach the courtroom, Children’s National pediatricians often serve as clinician-educators and expert witnesses. According to Children’s National experts, clinicians must share the best available medical practices in both the hospital and courtroom.  Unfortunately, in some cases around the country, a handful of expert medical witnesses provide unique and unsubstantiated opinions, sometimes claiming the presence of a rare genetic disorder as a cause of fracture or hemorrhage, when this has not been diagnosed by mainstream genetics specialists.

“On the part of expert witnesses, scientifically sound explanations versus unfounded hypotheses can influence outcomes in civil proceedings, which determine a child’s placement and criminal proceedings, which determine judgment on the perpetrators,” said Natasha Shur, M.D., medical geneticist at Children’s National, and Nathaniel Robin, M.D., professor and clinical genetics director at the University of Alabama in an editorial published in Current Opinion in Pediatrics.

Dr. Hinds works on behalf of children to provide the best and most comprehensive work-up in cases of unexplained fractures or hemorrhage. As a board-certified child abuse pediatrician, she is responsible for implementing the evidence-based practice guidelines of the American Academy of Pediatrics and other similar societies.  Dr. Hinds mentioned that it is possible to use medical history, physical examination and diagnostic testing to differentiate traumatic causes of fractures and subdural hematomas from genetic causes, a belief she states is held by the vast majority of child serving clinicians.

“In cases of suspected child abuse, a multidisciplinary group of clinicians at Children’s National routinely provide comprehensive and top-rate care and consider alternative explanations for fractures,” said Eglal Shalaby-Rana, M.D., a radiologist at Children’s National who has partnered with the hospital’s Child and Adolescent Protection Center team on these challenging cases since 1991. “A multidisciplinary team is crucial to the evaluation and often includes additional specialists such as pediatric radiology, trauma surgery, hematology and in some cases genetics.”

Further, these clinician-educators and researchers at Children’s National call for increased publication and use of consensus guidelines such as the consensus statement on abusive head trauma published in Pediatric Radiology in 2018.  “Consensus guidelines synthesize the best available medical evidence and should be the basis for both clinical practice and education offered in the courtroom,” said Drs. Shur, Hinds and Shalaby-Rana.

 Distinguishing child abuse from genetic disorders

Drs. Hinds, Shalaby-Rana and Shur have served as expert witnesses and in turn wanted to come together to help develop frameworks that share scientifically sound information with peers who might encounter spurious arguments in courtrooms regarding genetic disorders as an explanation for physical abuse and inflicted fractures. Their 2021 literature review, published in Current Opinion in Pediatrics, addresses some of these issues.

To help distinguish child physical abuse from a genetic disorder, Drs. Shur, Hinds and Shalaby-Rana  worked as a multidisciplinary team to highlight best practices in six instances when genetic disorders were raised as explanations for inflicted fractures or hemorrhage, including Elhers-Danlos syndrome (EDS), osteogenesis imperfecta (OI), Menkes Disease and Glutaric Acidemia type I. In some cases, these explanations could be reasonable but should be diagnosed using routine clinical and radiological review, and when indicated, genetic and biochemical testing.

For instance, EDS is a diagnosis that is sometimes erroneously used to explain multiple fractures in cases of suspected physical abuse and can be misused in courtrooms. The most common EDS type is hypermobile EDS, often found in late adolescence or early adulthood. In some cases, babies erroneously receive hypermobility exams, or clinicians perform hypermobility assessments on parents of children with unexplained fractures — neither practice is indicated. Instead, the Children’s National team points out that children should receive a medical evaluation using the standard guidelines set by the American Academic of Pediatrics, American College of Radiology and other professional societies.

Dr. Shur also collaborated with radiologists at Boston Children’s Hospital in a related review published in Pediatric Radiology. There, George et al. addressed the clinical and molecular diagnosis criteria for EDS to help radiologists prevent misdiagnosis and support clinicians when seeing patients with multiple fractures.

“It is disturbing that the unsubstantiated EDS infant bone fragility hypothesis continues to be advanced in civil and criminal child abuse proceedings when fractures are not part of the diagnosis criteria for EDS,” said George et al.

The clinicians noted that the Beighton score, which helps diagnose hypermobile EDS, is not intended for children younger than 8 years old. Additionally, since the score provides insufficient data, other EDS features must be present, such as skin findings and connective tissue abnormalities.

OI, known as ‘‘brittle bone disease,’’ is a group of disorders that rarely present only with unexplained fractures. The researchers emphasize that infants and children with mild OI do not present exclusively with multiple fractures, which are specific to physical abuse such as multiple, bilateral rib fractures and classic metaphyseal lesions. Drs. Shur, Hinds and Shalaby-Rana share that pediatric specialists could overcome the diagnostic challenges between OI and child abuse through the inclusion of a genetic team in some cases, during the medical evaluation while also considering various criteria, such as family history, physical examination and laboratory findings. Molecular testing may be required in some instances, but it cannot substitute traditional clinical and radiology evaluations, according to these clinician-researchers.

Similarly, while Menkes disease can present with intracranial hemorrhage and fractures like child physical abuse, there are other distinguishing characteristics unique to Menkes disease, such as hair and facial dysmorphism. In a third related case-review published in Pediatric Radiology, Shur, Hinds and Shalaby-Rana et al. emphasize that diagnostic difficulties may arises when a multidisciplinary evaluation is omitted. They call upon all clinicians to provide ethical testimony in civil or criminal proceedings and to continue to utilize a multidisciplinary approach during daily clinical practice.

Irresponsible testimony and predatory journals

According to George et al., in collaboration with Dr. Shur, there are flawed publications on EDS associated with infant bone fragility that do not follow the gold standard of the scientific community. They believe this hypothesis must be rejected by experts in the field of pediatric imaging to safeguard the scientific integrity of the discipline. The lack of scientific design, peer review process and transparency causes negative consequences in the courtroom and threatens the proper adjudication of cases of suspected child physical abuse.

“Irresponsible testimony increasingly enters medico-legal proceedings dealing with allegations of child abuse, and so-called expert witnesses regularly cite these deeply flawed publications — in addition to misquoting the medical literature, loosely interpreting medical findings, presenting fictitious findings, and excluding salient and widely accepted facts from consideration,” said George et al.

In these pieces of literature, our Children’s National multidisciplinary team members reviewed the best available evidence and their collective decades of patient experience to highlight standard processes, which differentiate child physical abuse as a cause of fractures and hemorrhage from rare disorders.  Rare does not mean mysterious, and with education and a multidisciplinary approach, every child can receive the best possible medical work-up and care, according to Drs. Shur, Hinds and Shalaby-Rana. They urge all physicians to share only mainstream clinical medicine in the courtroom to help ensure the best possible social outcomes for children and their families.

red flags for irresponsible testimony

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.

Boy with scoliosis during rehabilitation

Children’s National first-use of anterior vertebral body tethering system for idiopathic scoliosis

Boy with scoliosis during rehabilitation

On Monday, Feb. 1, 2021, Children’s National Hospital performed the first anterior vertebral body tethering procedure for a child in the Washington, D.C., area. The device is a recently approved option for treating children with idiopathic scoliosis, the most common type of scoliosis. It allows for gradual correction of a spinal deformity through the natural growth of the spine, leading to improvements in spinal alignment while maintaining spinal flexibility.

In August 2019, the U.S. Food and Drug Administration (FDA) approved the first spinal tether system for pediatric patients called The Tether – Vertebral Body Tethering System. This device is attached to the spine during a minimally invasive thorascopic procedure performed by a multidisciplinary medical team, which includes orthopaedic surgery, thoracic surgery and anesthesia.

“This promising technology may help maintain the flexibility of the spine as it grows straighter over time,” says Matthew Oetgen, M.D., chief of Orthopaedics at Children’s National. “Novel devices like The Tether offer additional treatment options for idiopathic scoliosis, which have the potential to improve pediatric surgical outcomes and quality of life for children and adolescents with significant spinal deformities.”

The procedure at Children’s National brought together some the region’s best pediatric orthopaedic and thoracic surgeons, including Dr. Oetgen, Shannon Kelly, M.D., associate chief of Orthopaedic Surgery, and Timothy Kane, M.D., chief of General and Thoracic Surgery.

“Children’s National can offer these types of procedures because the hospital is home to many talented pediatric surgeons across specialties, many of whom are experts in minimally invasive techniques for children,” says Dr. Kane. “We collaborate together, often, because we know that’s the best way to continually improve the care we provide to children and their families.”

Idiopathic scoliosis is the most common type of scoliosis and can occur in children between age 10 and 18 or until they are fully grown. Spinal fusion surgery is the most common treatment for children with the most severe spine curvatures and continues to be the “gold standard” for surgical treatment of this condition. It takes about 3 months for a child or adolescent to fully recover from the procedure. While spinal fusion produces excellent and reproducible outcomes, in certain patients with less severe deformities and significant skeletal growth remaining, vertebral body tethering may offer some benefits over spinal fusion. In clinical trials, the spinal tether was shown to shorten recovery time and increase range of motion. Early diagnosis is vital to dictate which treatment will be most beneficial.

Karun-Sharma-and-kids-MR-HIFU

FDA approves MR-HIFU system to treat osteoid osteoma

Karun-Sharma-and-kids-MR-HIFU

“This FDA approval encourages and further motivates our focused ultrasound program to continue to develop and expand clinical applications of MR-HIFU in the pediatric population,”  said Karun Sharma, M.D., Ph.D.

After garnering successful clinical trial results at Children’s National Hospital, the United States Food and Drug Administration (FDA) recently announced the approval of Profound Medical’s Sonalleve MR-guided High Intensity Focused Ultrasound (MR-HIFU) system for the treatment of osteoid osteoma (OO) in the extremities. OO is a benign, but painful bone tumor that occurs most commonly in children and young adults. This marks the first focused ultrasound regulatory approval that will directly impact pediatric patients and it is the sixth indication to earn approval in the United States.

Nine patients were treated in a pilot trial designed to evaluate the safety and feasibility of MR-HIFU ablation treatment in patients with painful OO. The procedure was performed without any technical difficulties or serious adverse events in all nine patients, and resulted in complete pain relief with no further pain medication usage in eight out of nine patients.

“This FDA approval encourages and further motivates our focused ultrasound program to continue to develop and expand clinical applications of MR-HIFU in the pediatric population,” said Karun Sharma, M.D., Ph.D., director of Interventional Radiology and associate director of clinical translation at the Sheikh Zayed Institute for Pediatric Surgical Innovation (SZI) at Children’s National. “This completely non-invasive and radiation-free aspects of this therapy are especially relevant for growing children.”

Researchers at Children’s National have moved beyond OO are also evaluating MR-HIFU treatment for patients with relapsed and refractory bone and soft tissue tumors. “This is especially important as these patients don’t have any other good treatment options,” said Dr. Sharma. “For these tumors, we are using not only thermal ablation, but also other modes and biomechanisms of focused ultrasound such as mild hyperthermia to facilitate targeted, enhanced drug delivery and histotripsy (i.e., mechanical tissue fractionation) to enhance cancer immunotherapy. We also hope to move into MR-HIFU brain application in pediatrics.”

At Children’s National, a multidisciplinary team of physicians and scientists use MR-HIFU to focus an ultrasound beam into lesions to heat and destroy the tissue in that region, with no incisions at all. In 2015, Children’s National doctors became the first in the U.S. to use MR-HIFU to treat pediatric osteoid osteoma. The trial, led by Dr. Sharma, demonstrated early success in establishing the safety and feasibility of noninvasive MR-HIFU in children as an alternative to the current, more invasive approaches to treat these tumors. Since then, the Children’s National team has built an active clinical trials program and become a leader in translation of focused ultrasound for the treatment of relapsed pediatric solid tumors.

Lee Beers

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

Lee Beers

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

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

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

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

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

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

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

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

insta-3D™ imaging from company nView medical

New innovative 3D imaging technology used in pediatric spine surgery

insta-3D™ imaging from company nView medical

Children’s National Hospital performed the first surgical use of breakthrough medical imaging technology designed specifically for kids. The innovation, insta-3D™ imaging from company nView medical, is designed to make 3D images available in the operating room quickly and safely.

Children’s National Hospital performed the first surgical use of breakthrough medical imaging technology designed specifically for kids. The innovation, insta-3D™ imaging from company nView medical, is designed to make 3D images available in the operating room quickly and safely. The 3D images provide surgeons with better visualization, allowing them to continue improving patient care and outcomes.

Matthew Oetgen, M.D., division chief of Orthopaedic Surgery at Children’s National, is overseeing the first use of this 3D imaging technology in orthopaedic procedures.

“Having a technology like this available in the operating room will potentially help make our surgeries even more precise with 3D imaging available quickly,” says Dr. Oetgen. “We anticipate this improved precision will lead to better outcomes and added value to what we do for our patients.”

Cristian Atria, nView medical’s CEO, commented for the first case.

“Seeing our imaging technology provide critical information during a kid’s surgery reminds us what the purpose of nView medical is all about,” says Cristian. “I would like to thank the surgeons, our backers, the team, and our clinical partners for making this first surgery a success. I couldn’t be more enthusiastic for what’s ahead!”

The potential of nView medical’s insta-3D™ imaging is especially exciting for Children’s National as nView medical is a 2019 Winner of the National Capital Consortium for Pediatric Device Innovation (NCC-PDI) competition “Make Your Medical Device Pitch for Kids!” NCC-PDI is led by the Sheikh Zayed Institute for Pediatric Surgical Innovation at Children’s National and the A. James Clark School of Engineering at the University of Maryland with support from partners MedTech Innovator, BioHealth Innovation, and design firm Archimedic.

NCC-PDI is one of five members in the FDA’s Pediatric Device Consortia Grant Program created to support the development and commercialization of medical devices for children in areas of critical need where innovation can significantly improve children’s health care.

“Children deserve to benefit from our most advanced medical technologies and we know that improvements in pediatric care can make a positive difference over the lifetime of a child,” says Kolaleh Eskandanian, Ph.D., M.B.A, P.M.P, vice president and chief innovation officer at Children’s National and principal investigator of NCC-PDI. “Pediatric hospitals must lead the way in supporting innovation for children’s care. That’s why, through NCC-PDI and our innovation institute, Children’s National helps to provide promising new pediatric devices with resources and expertise that support their journey to the market.”

Dr. Laura Tosi talks to a patient

Refining criteria for childhood skeletal fragility and osteoporosis

Dr. Laura Tosi talks to a patient

Orthopaedic surgeon Laura Tosi, M.D., presented information about bone fractures and skeletal fragility in children at this year’s POSNA Annual Meeting.

It’s true that broken bones are often a typical part of childhood, says international bone health expert Laura Tosi, M.D., an orthopaedic surgeon at Children’s National Hospital. But for some children, a single bone fracture under the right circumstances may be a signal that a child needs a closer look to rule out underlying skeletal fragility.

Dr. Tosi presented on this topic as part of the Pediatric Orthopaedic Society of North America’s (POSNA) 2020 Annual Meeting. The presentations were conducted virtually this year due to COVID-19.

“We know that between 27 and 40% of girls, and 42 to 51 percent of boys will have at least one fracture during childhood,” she says. “What we have also seen over time is that almost 40 percent of children who have one fracture will have more. How do we tell which children with a fracture may need our help to avoid future ones?”

During her session, Dr. Tosi discussed how adding more nuance to clinical evaluation criteria for childhood fractures can help identify which children should be evaluated for conditions affecting bone density.

To widen the scope and make sure an underlying bone density issue is detected and treated as early as possible, Dr. Tosi says there are some specific findings that should suggest the need for further exploration:

  • Does the child have an a priori risk for a fragility fracture due to a genetic bone disorder(such as osteogenesis imperfects (aka brittle bone disease) or immobility caused by a disorder such as spina bifida, cerebral palsy or muscular dystrophy?
  • Is there a mismatch between the fracture severity and level of trauma that led to the injury?
  • Does the child’s history include any of four factors known to be associated with increased fracture risk: early age at the time of the first fracture, intolerance to cow’s milk, low dietary calcium intake or high BMI values.
  • Does the child have a vertebral compression fracture?
  • Is there a family history of frequent fractures (which may indicate a previously unidentified genetic condition)

Dr. Tosi also laid out specific evaluation steps for a skeletal fragility condition when a child’s fracture meets criteria, including:

  • Family, nutrition and exercise histories
  • A detailed physical exam
  • Complete radiograph review, including previously existing films and bone densitometry
  • Rule out rickets and child abuse
  • A complete lab work up

“It can be extremely challenging to identify if a child’s first bone fracture is a result of typical childhood activity or something else,” says Dr. Tosi. “But the risks of waiting to evaluate a fracture that meets some of the criteria above may mean we are delaying a treatment that might improve bone density and prevent a future fracture altogether — which is always what we’d hope to do.”

In the past, bone health experts felt that the word “osteoporosis” should not be used in children and pushed for the term “low bone density for age.”  That perspective has begun to change thanks to important advances in our understanding of the genetic basis of bone fragility, the important role of chronic conditions and how the use of bone-active medications can significantly reduce fracture risk and improve function in certain conditions.

She then spoke about the benefits of early detection for conditions causing skeletal fragility by presenting compelling evidence of the resiliency of a child’s bones when they are managed appropriately.

She noted that she’s seen significant bone remodeling in patients with serious bone degeneration due to osteogenesis imperfecta and leukemia, for example, thanks to early detection and treatment.

“Our knowledge of bone density and bone health is improving, but is still imperfect,” she concluded. “But as we learn more, and are able to appropriately identify and treat kids with skeletal fragility or osteoporosis earlier, we can continue to refine how we evaluate and care for all of them.”

Research & Innovation Campus

Boeing gives $5 million to support Research & Innovation Campus

Research & Innovation Campus

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

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

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

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

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

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

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

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

NCC-PDI device competition

Medical device competition announces six winners to share in $250K

Judges award grants for pediatric medical devices that address cardiovascular, NICU, and orthopaedic and spine device innovations.

orthopaedics infographic

2020 at a glance: Orthopaedic Surgery and Sports Medicine at Children’s National


The Children’s National Division of Orthopaedics is consistently recognized by U.S. News & World Report as one of the top programs in the nation.

US News Badges

Children’s National ranked a top 10 children’s hospital and No. 1 in newborn care nationally by U.S. News

US News Badges

Children’s National Hospital in Washington, D.C., was ranked No. 7 nationally in the U.S. News & World Report 2020-21 Best Children’s Hospitals annual rankings. This marks the fourth 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 fourth year in a row.

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

“Our number one goal is to provide the best care possible to children. Being recognized by U.S. News as one of the best hospitals reflects the strength that comes from putting children and their families first, and we are truly honored,” says Kurt Newman, M.D., president and CEO of Children’s National Hospital.

“This year, the news is especially meaningful, because our teams — like those at hospitals across the country — faced enormous challenges and worked heroically through a global pandemic to deliver excellent care.”

“Even in the midst of a pandemic, children have healthcare needs ranging from routine vaccinations to life-saving surgery and chemotherapy,” said Ben Harder, managing editor and chief of Health Analysis at U.S. News. “The Best Children’s Hospitals rankings are designed to help parents find quality medical care for a sick child and inform families’ conversations with pediatricians.”

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 surgery, gastroenterology and gastro-intestinal surgery, and urology.

Matt Oetgen and patient

Periop procedures improve scoliosis surgery infection rates

Matt Oetgen and patient

Matthew Oetgen, M.D., MBA, chief of orthopaedics and sports medicine at Children’s National Hospital, presented findings from a study aimed at improving quality and safety for pediatric spinal fusion procedures by reducing surgical site infection rates.

Pediatric orthopaedic surgery as a field is focused on improving quality and value in pediatric spine surgery, especially when it comes to eliminating surgical site infections (SSI). Many studies have documented how and why surgical site infections occur in pediatric spinal fusion patients, however, there is very little data about what approaches are most effective at reducing SSIs for these patients in a sustainable way.

At the Pediatric Orthopaedic Society of North America’s 2020 Annual Meeting, Matthew Oetgen, M.D., MBA, chief of orthopaedic surgery and sports medicine at Children’s National Hospital, presented findings from a long-term single institution study of acute SSI prevention measures.

“These findings give us specific insight into the tactics that are truly preventing, and in our case sometimes even eliminating, SSIs for pediatric scoliosis surgery,” says Dr. Oetgen, who also served on the annual meeting program committee. “By analyzing patient records across more than a decade, we were able to see that some strategies are quite effective, and others, that we thought would move the needle, just don’t.”

The team reviewed medical records and radiographs dating back to 2008 for 1,195 patients who had spinal fusion for scoliosis, including idiopathic scoliosis as well as other forms such as neuromuscular or syndromic scoliosis. Over that period of time, the division of orthopaedics and sports medicine at Children’s National was collaborating with the hospital’s infection control team to achieve several programmatic implementation milestones, including:

  • January 2012: Standardized infection surveillance program
  • July 2013: Standardized perioperative infection control protocols including those for pre-operative surgical site wash, surgical site preparation and administration of antibiotics before and after surgery
  • March 2015: Standardized comprehensive spinal care pathway including protocols for patient temperature control, fluid and blood management, and drain and catheter management

Over the study time period, the team found that SSIs did decrease, but interestingly, the rate did not progressively decrease with each subsequent intervention.

“Instead, we found that the rate went down and was even eliminated for some subgroups when the perioperative infection control protocols were implemented in 2013 and sustained through the study period end,” says Dr. Oetgen. “The other programmatic efforts that started in 2012 and 2015 had no impact on infection rates.”

He also notes that the study’s findings have identified a crucial component in the process for infection control in pediatric spinal surgery—perioperative protocols. “A relatively uncomplicated perioperative infection control protocol did the best job decreasing SSI in spinal fusion. Future efforts to optimize this particular protocol may help improve the rates even further.”

Vittorio Gallo and Mark Batshaw

Children’s National Research Institute releases annual report

Vittorio Gallo and Marc Batshaw

Children’s National Research Institute directors Vittorio Gallo, Ph.D., and Mark Batshaw, M.D.

The Children’s National Research Institute recently released its 2019-2020 academic annual report, titled 150 Years Stronger Through Discovery and Care to mark the hospital’s 150th birthday. Not only does the annual report give an overview of the institute’s research and education efforts, but it also gives a peek in to how the institute has mobilized to address the coronavirus pandemic.

“Our inaugural research program in 1947 began with a budget of less than $10,000 for the study of polio — a pressing health problem for Washington’s children at the time and a pandemic that many of us remember from our own childhoods,” says Vittorio Gallo, Ph.D., chief research officer at Children’s National Hospital and scientific director at Children’s National Research Institute. “Today, our research portfolio has grown to more than $75 million, and our 314 research faculty and their staff are dedicated to finding answers to many of the health challenges in childhood.”

Highlights from the Children’s National Research Institute annual report

  • In 2018, Children’s National began construction of its new Research & Innovation Campus (CNRIC) on 12 acres of land transferred by the U.S. Army as part of the decommissioning of the former Walter Reed Army Medical Center campus. In 2020, construction on the CNRIC will be complete, and in 2012, the Children’s National Research Institute will begin to transition to the campus.
  • In late 2019, a team of scientists led by Eric Vilain, M.D., Ph.D., director of the Center for Genetic Medicine Research, traveled to the Democratic Republic of Congo to collect samples from 60 individuals that will form the basis of a new reference genome data set. The researchers hope their project will generate better reference genome data for diverse populations, starting with those of Central African descent.
  • A gift of $5.7 million received by the Center for Translational Research’s director, Lisa Guay-Woodford, M.D., will reinforce close collaboration between research and clinical care to improve the care and treatment of children with polycystic kidney disease and other inherited renal disorders.
  • The Center for Neuroscience Research’s integration into the infrastructure of Children’s National Hospital has created a unique set of opportunities for scientists and clinicians to work together on pressing problems in children’s health.
  • Children’s National and the National Institute of Allergy and Infectious Diseases are tackling pediatric research across three main areas of mutual interest: primary immune deficiencies, food allergies and post-Lyme disease syndrome. Their shared goal is to conduct clinical and translational research that improves what we know about those conditions and how we care for children who have them.
  • An immunotherapy trial has allowed a little boy to be a kid again. In the two years since he received cellular immunotherapy, Matthew has shown no signs of a returning tumor — the longest span of time he’s been tumor-free since age 3.
  • In the past 6 years, the 104 device projects that came through the National Capital Consortium for Pediatric Device Innovation accelerator program raised $148,680,256 in follow-on funding.
  • Even though he’s watched more than 500 aspiring physicians pass through the Children’s National pediatric residency program, program director Dewesh Agrawal, M.D., still gets teary at every graduation.

Understanding and treating the novel coronavirus (COVID-19)

In a short period of time, Children’s National Research Institute has mobilized its scientists to address COVID-19, focusing on understanding the virus and advancing solutions to ameliorate the impact today and for future generations. Children’s National Research Institute Director Mark Batshaw, M.D., highlighted some of these efforts in the annual report:

  • Eric Vilain, M.D., Ph.D., director of the Center for Genetic Medicine Research, is looking at whether or not the microbiome of bacteria in the human nasal tract acts as a defensive shield against COVID-19.
  • Catherine Bollard, M.D., MBChB, director of the Center for Cancer and Immunology Research, and her team are seeing if they can “train” T cells to attack the invading coronavirus.
  • Sarah Mulkey, M.D., Ph.D., an investigator in the Center for Neuroscience Research and the Fetal Medicine Institute, is studying the effects of, and possible interventions for, coronavirus on the developing brain.

You can view the entire Children’s National Research Institute academic annual report online.

Laura Tosi

Technology bridges knowledge gaps in rare bone disease care

Laura Tosi

Dr. Tosi and colleagues presented on the NIH Rare Disease Day 2020 panel, Nontraditional Approaches to Improving Access for Rare Diseases.

As part of the global observance of Rare Disease Day in February, the NCATS and NIH Clinical Center hosted a special event to raise awareness about rare diseases, the people they affect and NIH research collaborations under way to address scientific challenges and advance new treatments.

This year, Children’s National Hospital orthopaedic surgeon Laura Tosi, M.D., took part in an afternoon panel, Nontraditional Approaches to Improving Access for Rare Diseases, where she outlined her work as the faculty chair of the Rare Bone Disease TeleECHO, a virtual meeting that allows care providers and experts to come together via the Zoom platform, discuss diagnosis of specific disorders and present cases for group input.

Dr. Tosi and the Rare Bone Disease Alliance have called Project ECHO Rare Bone Disease a game changer for care of these complex conditions. Rare bone disorders are only about 5% of all birth defects but include 461 skeletal disorders caused by 437 genes – making it difficult for any physician to see enough cases of any one disorder to correctly diagnose and treat it.

“Most doctors are like me, a pediatric orthopaedic surgeon. I need to know a lot of different rare diseases and it’s hard to keep everybody on the cutting edge,” Dr. Tosi says. “Even though we have found the genes for most of the disorders, the phenotypic overlaps, shortage of specialists and the multi-disciplinary needs of so many of the patients add to the challenges.”

So 7 months ago, Dr. Tosi joined together with colleagues at the Rare Bone Disease Alliance and the Osteogenesis Imperfecta Foundation to launch Project ECHO Rare Bone Disease. The now monthly telehealth meeting engages a distinguished faculty of experts from around the world and from across the spectrum of care for these rare bone disorders, including specialists in genetics, endocrinology, orthopaedics and others.

Project ECHO is a specific model for bridging distance and creating a network of professionals, with the goal of leveling the playing field for all by making vital information accessible to everyone, regardless of their location. In healthcare the model transcends traditional “telemedicine,” however. The program, launched from the University of New Mexico, self-describes itself as “telementoring, a guided practice where the participating clinician retains responsibility for the patient” but is able to discuss diagnosis and therapeutic recommendations with a set of esteemed faculty via a regular virtual meeting series.

In the case of the Rare Bone Disease TeleECHO, the ECHO’s faculty decided on two major foci for the curriculum. Half of the content is about how to make the right diagnosis and the other half shares the latest information about specific diseases. The sessions also offer free CME to attendees.

Dr. Tosi says that while finding cases to discuss can sometimes be challenging when it comes to rare bone diseases, she takes responsibility on herself to make sure the content is robust each month. So far the meetings have attracted between 40 and 90 participants per session – a great engagement rate for such a young teleECHO program.

“I believe ECHO advances knowledge of healthcare and democratizes it by offering universal accessibility across the globe,” Tosi notes.

The Rare Bone Disease Alliance, which consists of 12 organizations, experts and patient families working together, is now deciding what’s next for the Rare Bone Disease TeleECHO. They may develop disorder-specific ECHOs, are studying the frequency of the sessions and how best to improve participation for all sessions. The idea is to increase access to this expertise even further, as it could have critical impacts on patients worldwide living with these rare diseases.

In terms of key take-aways from the panel of experts at Rare Disease Day, the hope is that more disease groups might leverage this type of technology to connect people in nontraditional ways. Doing so has the potential to ensure that everyone with a rare disease receives the best support and care possible because their doctors have the knowledge they need when they need it.

Watch more sessions from the NIH’s Rare Disease Day 2020.

little girl in wheelchair

A holistic and proactive approach to the management of the patients with cerebral palsy

little girl in wheelchair

The cerebral palsy program at Children’s National Hospital takes a comprehensive approach to meet children’s needs from infancy through young adulthood.

Though children with cerebral palsy (CP) often require significant rehabilitative and surgical support, most often each service is provided in the individual specialty itself. Patients and their families frequently experience a great deal of stress coordinating care, getting to appointments, keeping track of medications and managing treatments on their own.

However, the CP program at Children’s National Hospital, co-led by an orthopaedic surgeon, Sean Tabaie, M.D., and a pediatric rehabilitation specialist, Olga Morozova, M.D., working together and in collaboration with Shannon Kelly, M.D., (Orthopaedics) and Jeff Rabin, D.O., (Physical Medicine and Rehabilitation) has evolved into a truly comprehensive approach. The program is designed to meet the needs of these children from infancy through young adulthood with the goal of improving function and preventing musculoskeletal deformities and complications.

Providing children and their families with a single point of care coordination allows the care team to track and anticipate a multitude of potential challenges for each child as early as possible and intervene in smaller ways before they bring pain and long-term complications or require major surgical interventions. Key highlights of this collaborative program include:

  • Hip surveillance: Patients with CP are followed closely to identify hip pathology prior to serious subluxation or dislocation. Early identification allows for the use of medical or surgical interventions to prevent a minor issue from becoming a major one.
  • Serial casting for children with early signs of muscle contractures: Dr. Morozova uses agents to relax the muscles and Dr. Tabaie applies the cast in the operating room followed by continued weekly serial casting in the clinic. “Proper medical management and bracing at regular intervals can improve muscle function and prevent the need for larger surgeries and more intense rehabilitation later,” says Dr. Morozova.
  • Advanced coordination between physical therapy in the hospital and outpatient services in the region, building on the hospital’s partner agreement with the HSC Health Care System.
  • Single event multi-level surgeries (SEMLS): Ensuring that surgical procedures capture all surgical needs at one time by assessing the entire anatomy and scheduling multiple surgical or pharmaceutical interventions to occur in a single session.

The doctors point out that offering these services in one cohesive location and combining treatments into the same appointment or procedure date is something that many patients with CP and their families truly appreciate.

“I think families of children with CP will travel great distances if the care they receive is comprehensive and eliminates some of the back and forth travel they do now,” says Dr. Tabaie.

Today, the team sees close to 100 patients with CP per month and hopes to expand to reach as many families in the region and beyond who need them.

Dr. Tabaie says, “Our goal is to identify patients early and start managing them to help their quality of life today, prepare them to grow as healthily and in as little pain as possible and set them up to be as healthy as they can possibly be as adults, too.”

Dr. Lauri Tosi examines a patient

Building patient-centered outcomes research in osteogenesis imperfecta

Dr. Lauri Tosi examines a patient

Children’s orthopaedic surgeon Laura Tosi, M.D., is the co-lead on a program to improve patient-centered outcomes research and education in osteogenesis imperfecta that recently received a Eugene Washington Engagement Award of $250,000 from the Patient-Centered Outcomes and Research Institute (PCORI).

Children’s orthopaedic surgeon Laura Tosi, M.D., is the co-lead on a program to improve patient-centered outcomes research and education in osteogenesis imperfecta (OI) that recently received a Eugene Washington Engagement Award of $250,000 from the Patient-Centered Outcomes and Research Institute (PCORI). Dr. Tosi serves as project co-lead alongside colleagues Tracy Hart, project lead, from the Osteogenesis Imperfecta Foundation (OIF) and Bryce Reeve, Ph.D., co-project lead, director of the Center for Health Measurement at Duke University.

The project, which will be housed at the Osteogenesis Imperfecta Foundation, will run for two years and seeks to:

  • Create a community of stakeholders (patients/caregivers/clinicians/researchers) who are trained or training in patient-centered outcomes research, with specific attention to priority topics identified by the OI community.
  • Expand communications and education strategies related to patient-centered outcomes research to enhance the care of the OI community.
  • Establish and extend the capacity among patients, caregivers, clinicians and researchers in OI to participate in both patient-centered outcomes research and comparative effectiveness activities.
  • Develop an OI-specific toolkit focused on disseminating evidence-based clinical care recommendations to stakeholders and care providers, based on sustainable input from the OI community.
  • Extended the reach of these activities to support other rate bone disease communities.

Osteogenesis imperfecta is a group of genetic disorders causing connective tissue dysfunction and bone fragility. It is the most common of nearly 450 rare skeletal disorders and affects an estimated 25,000 to 50,000 people in the U.S. Collecting the patient’s perspective about natural history, clinical best practices, quality of life and research priorities is challenging because, like so many rare diseases, the affected population is relatively small and  geographically dispersed.

“We hope this project will give us the ability to develop a set of best practices for care and research based on research that incorporates the patient’s point-of-view,” says Dr. Tosi. “I’m excited to work with this team and begin to change how we think about and care for OI patients and their families.”

Pediatric device competition

Premier annual pediatric medical device competition now accepting submissions

Pediatric device competition

Pediatric innovators pitch for grant awards and participation in a special accelerator program.

The official call for submissions is underway for the premiere annual pediatric medical device competition, sponsored by National Capital Consortium for Pediatric Device Innovation (NCC-PDI). The competition is led by Sheikh Zayed Institute for Pediatric Surgical Innovation at Children’s National Hospital, the A. James Clark School of Engineering at the University of Maryland and non-profit accelerator MedTech Innovator. The three organizations are all an integral part of the FDA-funded NCC-PDI, which aims to facilitate the development, production and distribution of pediatric medical devices. Additional NCC-PDI members include accelerator BioHealth Innovation and design firm Archimedic.

The competition focuses on pediatric devices in three areas of critical need: cardiovascular, orthopedic and spine, and neonatal intensive care (NICU) and is now accepting applications. Contestants will pitch for a share of up to $250K in grant awards and the opportunity to participate in the MedTech Innovator 2020 Accelerator – Pediatric Track.

The first stage of competition will be held on March 23 at the University of Maryland and will include up to 30 companies selected from all submissions received. Up to 10 finalists selected from that event will move on to the “Make Your Medical Device Pitch for Kids!” finals on October 4, 2020 in Toronto, Canada. Finalists from the March qualifying round will be notified in May, 2020.

“While there is a great need for pediatric devices in many specialty areas, the development and commercialization process is very challenging because of the small market size and dynamic characteristics of the patient population,” says Kolaleh Eskandanian, Ph.D., MBA, PMP, vice president and chief innovation officer at Children’s National Hospital and principal investigator of NCC-PDI. “To provide pediatric innovators with greater support in meeting these unique challenges, we must go beyond grant funding, which is why we are collaborating with MedTech Innovator to offer an accelerator program with a pediatric track.”

To date, NCC-PDI has mentored over 100 medical device sponsors to help advance their pediatric innovations, notes Eskandanian, with six devices having received either their FDA market clearance or CE marking. She says the success of NCC-PDI’s portfolio companies is attributed to funding, mentorship, support from partners, facilitated interactions between device innovators and potential investors, and being discovered during their presentations at the signature “Make Your Medical Device Pitch for Kids!” competitions.

While advancements have been made in some pediatric specialties, there is still a critical need for novel devices in cardiovascular, orthopedic and spine, and NICU areas. On average over the past decade, only 24 percent of life-saving medical devices approved by FDA – those that go through PMA and HDE regulatory pathways – have an indication for pediatric use. Of those, most are designated for children age 12 or older. “Devices designed specifically for the younger pediatric population are vitally needed and, at this early stage of the intervention, can significantly improve developmental outcomes for a child,” Eskandanian said.

For more information and to apply for the upcoming NCC-PDI pitch competition, visit https://medtechinnovator.org/pediatricapply/.

Enhancing access to resources for pediatric innovators is also one of the aims of the Children’s National Research and Innovation Campus, a first-of-its-kind focused on pediatric healthcare innovation, currently under development on the former Walter Reed Army Medical Center campus in Washington, D.C. and opening in December, 2020. With its proximity to federal research institutions and agencies, universities, academic research centers, as well as on site accelerator Johnson and Johnson Innovation – JLABS, the campus will create a rich ecosystem of public and private partners which, like the NCC-PDI network, will help bolster pediatric innovation and commercialization.

NOTE: The deadline for submissions has been extended to February 22 at midnight EST.

Joseph Letzelter

Offering more options for children with hand and upper extremity ailments

Joseph Letzelter

The Children’s National Hand and Upper Extremities Program is one of few dedicated pediatric programs in the country, and it just added another hand surgeon to the team of extensively trained and experienced specialists.

Joseph Letzelter III, M.D., joined Children’s National Hospital and Pediatric Specialists of Virginia in September 2019 following comprehensive medical training in orthopaedic surgery with a focus on pediatric hand and upper extremity surgery. Dr. Letzelter received his undergraduate and medical degrees from Georgetown University, and also completed his internship and orthopaedic surgery residency there. He then completed a fellowship in hand and upper extremity surgery at New York University before continuing his studies with Dr. Michael Boland at the prestigious Hand Institute of New Zealand in Auckland. To round out his fellowship training, Dr. Letzelter focused purely on pediatric hand and upper extremity surgery at Shriner’s Hospital in Sacramento.

With the addition of Dr. Letzelter, the combined Hand and Upper Extremities Program expands its capacity to treat children with pediatric hand disorders. The joint orthopaedic surgery and plastic surgery program, led by Emily Hattwick, M.D., and Gary Rogers, M.D., uses pediatric-specific surgical methods to treat children without affecting their growth. Partnering with the world-class researchers at the Sheikh Zayed Institute for Surgical Innovation at Children’s National, the team is part of new discoveries that change the way we care for rare conditions. Learn more about the Hand and Upper Extremities Program here.