Emily Niu

Osteochondritis dissecans: Deciding the best candidates for nonoperative treatment

Emily Niu

“When patients come to see me with this condition, they’re often at their lowest point. But then I get to watch them go through this transformation as they’re getting better,” says Emily Niu, M.D. “It’s really wonderful to see someone’s personality blossom over the course of treatments.”

The adage of “practice makes perfect” is true for young competitive athletes; however it also puts them at risk for overuse injuries. One of these injuries is a condition called osteochondritis dissecans (OCD), in which repeatedly overloading joints causes increased stress to certain areas of bone. This area of bone can lose its blood supply, become unhealthy and ultimately end up fragmented. In the late stages, this area of bone can break off from the surrounding healthy bone and as a result, the overlying cartilage (which relies on the bone for a foundation) can become prone to damage. This process can be likened to the formation of potholes in a road. Typically, individuals affected can have pain, limited range of motion or even arthritis down the line.

This condition can happen at different locations throughout the body, including the knees, ankles and elbows. Baseball players and gymnasts are particularly prone to getting OCD of the capitellum, or the outside of the elbow, from throwing or tumbling.

If this injury is unstable, with bone and cartilage already fragmenting, it’s typically treated with surgery, explains Emily Niu, M.D., a Children’s National orthopaedic surgeon. Stabilizing the fragment and drilling tunnels in the affected bone surface allows new bone to grow and repair the defect. But for stable OCD, the treatment path is unclear.

Sometimes nonoperative treatments, such as rest, physical therapy or bracing the joint, can allow it to fully heal over time; however, she says, some patients treated nonoperatively may not be able to heal and will still require surgery later.

What distinguishes these two groups has thus far been unclear. Dr. Niu adds that there are few studies that have looked at what characteristics might make patients better candidates for a surgical or nonoperative route. The studies that do exist are limited to very small groups of patients.

To help doctors and their patients make more informed decisions, Dr. Niu and her colleagues performed a retrospective review of 89 patients aged 18 years old and younger treated at Boston Children’s Hospital for stable OCD of the capitellum. The vast majority of these 49 male and 40 female patients were baseball players and gymnasts. Most had just a single elbow affected; four patients (all gymnasts) experienced this problem in both elbows.

Each of these patients was initially treated nonoperatively, with activity restriction, physical therapy and progressive return to activity at the discretion of the treating physician. During this time, all of the athletes had elbow radiographs, elbow MRIs or both to image the injury and follow its healing process.

The researchers report in the November 2018 that just over half of these 93 elbows healed successfully with nonoperative treatments, taking an average of about eight months for symptoms to subside and imaging to show that the bone had healed properly.

When Dr. Niu and her colleagues looked for characteristics that might have influenced whether nonoperative treatments worked or didn’t, they didn’t find any difference in the two groups with age, bone maturity, sex, hand dominance or sport. However, the healing group had symptoms for an average of four months shorter than the non-healing group before they sought treatment. Those patients with bone lesions without clear margins visible on MRI were more likely to heal than those with clear margins, as were those without cyst-like lesions on their bones – both signs of a more advanced process. In addition, those whose bone lesions were relatively small were more likely to heal than those with larger lesions compared to the size of their bones.

Dr. Niu notes that OCD can be a devastating injury for young athletes, interrupting their participation in sports on average for a minimum of six months and significantly longer if nonoperative treatments fail and surgery becomes necessary. Being able to shave some time off that schedule with better knowledge of which type of treatment is most likely to work, she says, can help her patients get back to doing what they love significantly faster.

“When patients come to see me with this condition, they’re often at their lowest point. But then I get to watch them go through this transformation as they’re getting better,” Dr. Niu says. “It’s really wonderful to see someone’s personality blossom over the course of treatments. It’s such a relief for both of us when they’re back where they want to be.”

Femoral fracture

Broken system? Pain relief for fractures differs by race/ethnicity

Femoral fracture

Data collected by a multi-institutional research team show that kids’ pain from long bone fractures may be managed differently in the emergency department depending on the child’s race and ethnicity.

Children who experience broken bones universally feel pain. However, a new multi-institutional study presented at the American Academy of Pediatrics (AAP) 2018 National Conference & Exhibition suggests that emergency treatment for this pain among U.S. children is far from equal. Data collected by the research team show that kids’ pain may be managed differently in the emergency department depending on the child’s race and ethnicity. In particular, while non-Latino black children and Latino children are more likely to receive any analgesia, non-white children with fractured bones are less likely to receive opioid pain medications, even when they arrive at the emergency department with similar pain levels.

“We know from previously published research that pain may be treated differentially based on a patient’s race or ethnicity in the emergency department setting. Our prior work has demonstrated that racial and ethnic minorities are less likely to receive opioid analgesia to treat abdominal pain, even when these patients are diagnosed with appendicitis,” says study leader Monika K. Goyal, M.D., MSCE, assistant division chief and director of Academic Affairs and Research in the Division of Emergency Medicine at Children’s National Health System. “Emergency departments delivering evidence-based care should treat all pediatric patients consistently. These findings extend our work by demonstrating that children presenting with long bone fractures also experience differential treatment of pain based on their race or ethnicity.”

The AAP calls appropriately controlling children’s pain and stress “a vital component of emergency medical care” that can affect the child’s overall emergency medical experience. Because fractures of long bones – clavicle, humerus, ulna, radius, femur, tibia, fibula – are commonly managed in the emergency department, the research team tested a hypothesis about disparities in bone fracture pain management.

They conducted a retrospective cohort study of children and adolescents 21 and younger who were diagnosed with a long bone fracture from July 1, 2014, through June 30, 2017. They analyzed deidentified electronic health records stored within the Pediatric Emergency Care Applied Research Network Registry, which collects data from all patient encounters at seven pediatric emergency departments.

During that time, 21,642 patients with long bone fractures met the study inclusion criteria and experienced moderate to severe pain, rating four or higher on a 10-point pain scale. Some 85.1 percent received analgesia of any type; 41.5 percent received opioid analgesia. Of note:

  • When compared with non-Hispanic white children, minority children were more likely to receive pain medication of any kind (i.e. non-Latino black patients were 58 percent more likely to receive any pain medication, and Latino patients were 23 percent more likely to receive any pain medication).
  • When compared with non-Latino white children, minority children were less likely to receive opioid analgesia (i.e., non-Latino black patients were 30 percent less likely to receive opioid analgesia, and Latino patients were 28 percent less likely to receive opioid analgesia).

“Even though minority children with bone fractures were more likely to receive any type of pain medication, it is striking that minority children were less likely to receive opioid analgesia, compared with white non-Latino children,” Dr. Goyal says. “While it’s reassuring that we found no racial or ethnic differences in reduction of patients’ pain scores, it is troubling to see marked differences in how that pain was managed.”

Dr. Goyal and colleagues are planning future research that will examine the factors that inform how and why emergency room physicians prescribe opioid analgesics.

American Academy of Pediatrics National Conference & Exhibition presentation

  • “Racial and ethnic differences in the management of pain among children diagnosed with long bone fractures in pediatric emergency departments.”

Monika K. Goyal, M.D., MSCE, and James M. Chamberlain, M.D., Children’s National; Tiffani J. Johnson, M.D., MSc, Scott Lorch, M.D., MSCE, and Robert Grundmeier, M.D., Children’s Hospital of Philadelphia; Lawrence Cook, Ph.D., Michael Webb, MS, and Cody Olsen, MS, University of Utah School of Medicine; Amy Drendel, DO, MS, Medical College of Wisconsin; Evaline Alessandrini, M.D., MSCE, Cincinnati Children’s Hospital; Lalit Bajaj, M.D., MPH, Denver Children’s Hospital; and Senior Author, Elizabeth Alpern, M.D., MSCE, Lurie Children’s Hospital.

Matthew Oetgen

Surgical home program for spinal fusion achieves long-term success

Matthew Oetgen

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

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

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

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

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

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

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

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

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

Making the grade: Children’s National is nation’s Top 5 children’s hospital

Children’s National rose in rankings to become the nation’s Top 5 children’s hospital according to the 2018-19 Best Children’s Hospitals Honor Roll released June 26, 2018, by U.S. News & World Report. Additionally, for the second straight year, Children’s Neonatology division led by Billie Lou Short, M.D., ranked No. 1 among 50 neonatal intensive care units ranked across the nation.

Children’s National also ranked in the Top 10 in six additional services:

For the eighth year running, Children’s National ranked in all 10 specialty services, which underscores its unwavering commitment to excellence, continuous quality improvement and unmatched pediatric expertise throughout the organization.

“It’s a distinct honor for Children’s physicians, nurses and employees to be recognized as the nation’s Top 5 pediatric hospital. Children’s National provides the nation’s best care for kids and our dedicated physicians, neonatologists, surgeons, neuroscientists and other specialists, nurses and other clinical support teams are the reason why,” says Kurt Newman, M.D., Children’s President and CEO. “All of the Children’s staff is committed to ensuring that our kids and families enjoy the very best health outcomes today and for the rest of their lives.”

The excellence of Children’s care is made possible by our research insights and clinical innovations. In addition to being named to the U.S. News Honor Roll, a distinction awarded to just 10 children’s centers around the nation, Children’s National is a two-time Magnet® designated hospital for excellence in nursing and is a Leapfrog Group Top Hospital. Children’s ranks seventh among pediatric hospitals in funding from the National Institutes of Health, with a combined $40 million in direct and indirect funding, and transfers the latest research insights from the bench to patients’ bedsides.

“The 10 pediatric centers on this year’s Best Children’s Hospitals Honor Roll deliver exceptional care across a range of specialties and deserve to be highlighted,” says Ben Harder, chief of health analysis at U.S. News. “Day after day, these hospitals provide state-of-the-art medical expertise to children with complex conditions. Their U.S. News’ rankings reflect their commitment to providing high-quality care.”

The 12th annual rankings recognize the top 50 pediatric facilities across the U.S. in 10 pediatric specialties: cancer, cardiology and heart surgery, diabetes and endocrinology, gastroenterology and gastrointestinal surgery, neonatology, nephrology, neurology and neurosurgery, orthopedics, pulmonology and urology. Hospitals received points for being ranked in a specialty, and higher-ranking hospitals receive more points. The Best Children’s Hospitals Honor Roll recognizes the 10 hospitals that received the most points overall.

This year’s rankings will be published in the U.S. News & World Report’s “Best Hospitals 2019” guidebook, available for purchase in late September.

Pedbot video game

New robotic therapies for cerebral palsy

Little girl on hippobot

The hippobot is a mechanical horseback riding simulator that provides hippotherapy for children.

Cerebral palsy is the most common type of movement disorder in children, affecting 1 in 500 babies born each year. For these infants, learning to sit up, stand and walk can be a big challenge which often requires years of physical therapy to stretch and strengthen their muscles. A team led by Kevin Cleary, Ph.D., technical director of the Bioengineering Initiative at Children’s National Health System, and Sally Evans, M.D., director of Pediatric Rehabilitation Medicine at Children’s National, has created two new types of robotic therapy that they hope will make physical therapy more enjoyable and accessible for children.

Hippobot equine therapy simulator

One of the most effective types of therapy for children with cerebral palsy is hippotherapy, which uses horseback riding to rehabilitate children with neurological and musculoskeletal disabilities. The movement of horses helps riders with cerebral palsy improve endurance, balance and core strength, which in turns helps them gain the ability to sit without support. If a child with cerebral palsy does not master independent sitting early in life, he or she may never gain the ability to stand or walk. Unfortunately, many children never have the chance to experience hippotherapy due to geographical constraints and cost issues.

To increase patient access to hippotherapy, the bioengineering team (Reza Monfaredi, Ph.D.; Hadi Fooladi Talari, M.S.; Pooneh Roshani Tabrizi, Ph.D.; and Tyler Salvador, B.S.) developed the hippobot — a mechanical horseback riding simulator that provides hippotherapy for children ages 4 to 10 in the office setting. To create the hippobot, the researchers mounted a carousel horse on a six-degree of freedom commercial motion platform (the platform moves in the x, y and z directions and rotates about roll, pitch and yaw axes). They then programmed the platform to simulate a horse walking, trotting and cantering.

“Several experienced horse riders have tried the motion platform and commented that it gives a realistic feel,” says Dr. Cleary.

The team then incorporated optical tracking of the hippobot rider’s spine and pelvis to monitor their posture and created a virtual reality video display that simulates a horse moving down a pier. As other animals come towards the horse, the rider must lean right or left to avoid them.  The trackers on their back show which way they are leaning and feed that information into the gaming system.

“We wanted to see how the patient’s spine reacts as the horse moves through different patterns, and if the patients get better at maintaining their posture over several sessions,” says Dr. Cleary.

To date the system has been used with several children with cerebral palsy under an IRB-approved study. All of the participants enjoyed riding the horse and came back for multiple sessions.

The hippobot system was developed in close collaboration with the Physical Medicine and Rehabilitation Division at Children’s National, including Olga Morozova, M.D., Justin Burton, M.D., and Justine Belschner, P.T.

Pedbot ankle rehabilitation system

Pedbot video game

Patients use pedbot as an input device to pilot an airplane through a series of hoops. The level of the difficulty of the game can be easily adjusted based on the patient’s capability and physical condition.

More than half of children with cerebral palsy also have gait impairment as a result of excessive plantar flexion and foot inversion/eversion, or equinovarus/equinovalgus at their ankle and foot. To help these patients, Dr. Cleary’s team developed the pedbot — a small robot platform that enables better strengthening, motor control and range of motion in the ankle joint.

“Children with cerebral palsy have difficulty walking in part because they have trouble controlling their feet,” explains Dr. Evans. “Use of pedbot as part of therapy can help to give them increased control of their feet.”

Most ankle rehabilitation robots are limited in their movements, and have only one or two degrees of freedom, focusing on ankle dorsiflexion/plantarflexion and sometimes inversion/eversion. Pedbot is unique in that it has three degrees of freedom with a remote center of motion in the ankle joint area that allows it to move in ways other devices can’t.

The pedbot platform can move in three translational directions (x, y and z) and also rotate about three axes (the x, y and z axes). As an analogy, this is similar to the movement of a flight simulator. The system also includes motors and encoders at each axis and can be used in passive and active modes.

In both modes, the patient sits on a therapy chair with their foot strapped to the robotic device. In the passive mode, the therapist assists the patient in training motions along each axis. The robot can then repeat the motion under therapist supervision while incrementally increasing the range of motion as desired by the therapist.

For the active mode or “gaming” mode, the team developed a video game based on an airplane motif. Patients use pedbot as an input device to pilot an airplane through a series of hoops. The level of the difficulty of the game can be easily adjusted based on the patient’s capability and physical condition.

To date, four patients have participated in an IRB-approved clinical trial for the pedbot. All of the patients enjoyed the game and they were willing to continue to participate as suggested by a physiotherapist.

The pedbot team, in addition to the engineers mentioned above, includes Catherine Coley, P.T.; Stacey Kovelman, P.T.; and Sara Alyamani, B.A. In future work, they plan to expand the system to include electromyography measurements with Paola Pergami, M.D.,Ph.D. They also are planning to develop a low cost, 3D printed version for the home market so children can do Pedbot therapy every day.

Matthew Oetgen

3D printed implant used to repair knee cartilage

Matthew Oetgen

“Our preliminary study shows this novel 3D printed material is able to allow ingrowth from the bone, so the body started to grow into the material to help fix it in place,” says Matthew Oetgen, M.D., M.B.A. “These are the first step requirements for an implant like this to be acceptable for treating lesions.”

Every year, an estimated 1 million children tear the articulate cartilage that lines their knees. Unfortunately, these types of injuries are extremely hard to repair because of the cartilage’s poor healing qualities and unique physiochemical properties.

Now, a new study by Children’s National Health System researchers has found that a three dimensional (3D) printed synthetic implant can be successfully used as a scaffold to encourage the healing and repair of articulate cartilage lesions.

Three bones meet in the knee joint: the femur, the tibia and the patella. The surface of these bones is covered with articulate cartilage, which can be damaged by injury or by normal wear and tear. Because articulate cartilage has poor healing qualities, these injuries will rarely heal or regenerate on their own, especially in younger and more active patients.

“These are active 12 to 19 year olds, so it can really affect relatively normal kids,” says Matthew Oetgen, M.D., M.B.A., Division Chief of Orthopaedic Surgery and Sports Medicine at Children’s National. “While there are many ways to repair these lesions — from implanting autogenous cells to using grafts to fill the defect — none of these options are perfect, and they all have some down sides.”

To facilitate repair of these injuries, a team of researchers led by Dr. Oetgen received a grant from the Pediatric Orthopaedic Society of North America (POSNA) to design a 3D printed implant that promotes bone and cartilage growth.

To make the implant, the team used nanoporous thermoplastic polyurethane (TPU), a biodegradable material that is highly elastic and yet strong, very much like the native cartilage in the osteochondral region. TPU is also porous, which allows blood and nutrient flow through the implant.

“The implant is designed to allow native cells to repair the lesions with normal articular cartilage and not scar tissues like some repairs,” says Dr. Oetgen.

The implant itself has a stratified structure: an upper region that contains micro channels to allow for increased perfusion; a middle zone with a nanoporous structure that mimics porous cartilage and encourages stem cell recruitment, growth and differentiation; and a lower region, or articular surface, that allows for smooth transition from the articulating surface to the implant surface and minimizes adverse interactions between the articulate cartilage and the meniscus.

When tested in vitro, the implant was able to support the growth of stem cells and vascular cells, and structurally mature vascularized bone was formed around the implant after 10 days. In animal models with full thickness osteochondral lesions the implant did just as well: The scaffold was able to promote bone, soft tissue and vascular growth without eliciting an immune response.

“Our preliminary study shows this novel 3D printed material is able to allow ingrowth from the bone, so the body started to grow into the material to help fix it in place,” says Dr. Oetgen. “These are the first step requirements for an implant like this to be acceptable for treating lesions.”

Because of the ease with which 3D printing can be scaled up, Dr. Oetgen is hopeful that the implant will one day become a viable option for repairing articulate cartilage injuries. He plans on trying the implants in a larger animal model and on larger lesions, and is also looking at custom printing for the implants to match natural lesion shapes and sizes.

Femoral fracture

POSNA grant addresses variations in femoral fracture treatment

Femoral fracture

While there are plenty of options for treating pediatric femoral diaphyseal fractures, doctors don’t have a lot of specific guidance on the optimal regimen for each patient age, fracture location and fracture pattern.

Pediatric femoral diaphyseal fractures are some of the most common types of long bone fractures. There are many effective ways to treat these injuries, but unfortunately this assortment of options also leads to variations in cost and clinical outcome for patients and makes it difficult to develop clinical trials exploring the treatment of pediatric femur fractures.

To address this issue, a Children’s National research team led by Matthew Oetgen, M.D., M.B.A., Division Chief of Orthopaedic Surgery and Sports Medicine, received a $30,000 grant from the Pediatric Orthopaedic Society of North America (POSNA) to design a multi-centered, randomized, controlled clinical trial for the treatment of pediatric diaphyseal femur fractures. The team’s ultimate goal is to submit the resulting trial design to an extramural agency for study funding.

While there are plenty of options for treating pediatric femoral diaphyseal fractures, doctors don’t have a lot of specific guidance on the optimal regimen for each patient age, fracture location and fracture pattern. As a result, many treatment decisions are based on surgeon preference, regional variation in care and previous training or experience.

Another issue that arises in the treatment of diaphyseal femur fractures is the impact on the patient’s family. In general, femur fractures are caused by significant trauma that affects both the patient and the family members. On top of this, families are faced with issues such as extended hospitalization, the need for wheelchairs and walkers, pain control, missed school and secondary surgeries for removal of implants. Often, families are left to their own devices to resolve these issues, many of which are more impactful than the injury itself.

Dr. Oetgen believes that a well-planned and well-structured randomized clinical trial guided by patient and family concerns as well as expert surgical opinion has the potential to improve both treatment and care of femoral diaphyseal fracture patients.

“It is no longer good enough to design studies that only look at healing time for femur fractures,” explains Dr. Oetgen. “These injuries have such significant secondary impacts for the families of these patients, we need to determine which treatment is optimal for both fracture healing and is easiest for the families to tolerate. This grant will allow us to consider all of these outcomes in designing a study to find the best treatment for these injuries.”

To aid in the design of their clinical trial, Dr. Oetgen and his team will:

  1. Conduct an extensive literature review on the impact and treatment of pediatric femur fractures.
  2. Survey a diverse group of pediatric orthopaedic surgeons to establish areas of agreement, opposition and equipoise on the surgical treatment of pediatric femur fractures, and use that information to form a consensus opinion on the optimal design of the clinical trial.
  3. Solicit input from non-physician stakeholders (families, parents, payers, state Medicaid representatives, patient advocacy groups, professional organizations) on the important aspects of care in pediatric femur fracture treatment.

The team expects to have the study design competed by February 2019.

Clubfoot

Assessing clubfoot recurrence rates and causes

Clubfoot

A Children’s National research team performed a structured literature review to determine the reported rates of clubfoot correction and recurrence after the Ponseti technique and to identify factors that contribute to these issues.

Clubfoot, or talipes equinovarus, is a congenital foot deformity that affects the bones, muscles, tendons and blood vessels in the feet. It occurs in approximately 1 to 3 of every 1,000 births and is traditionally treated with the nonsurgical Ponseti technique, which uses manipulation and casting to correct the condition. Unfortunately, recurrence of clubfoot after treatment is somewhat common.

A Children’s National research team led by Matthew Oetgen, M.D., Division Chief of Orthopaedic Surgery and Sports Medicine, recently performed a structured literature review to determine the reported rates of clubfoot correction and recurrence after the Ponseti technique and to identify factors that contribute to these issues.

Ponseti treatment is generally administered during the first few weeks of life in order to take advantage of the elasticity of tissues forming the ligaments, joint capsules and tendons. These structures are stretched with weekly, gentle manipulations, and a plaster cast is applied after each session to retain the degree of correction obtained and to soften the ligaments. Over the course of six to eight weeks, the displaced bones are brought into the correct alignment. In order to maintain this alignment, braces are then worn for 23 hours a day for up to three months, and then at night for two to four years.

Matthew Oetgen

The team, led by Matthew Oetgen, M.D., determined that the average rate for correction of clubfoot via the Ponseti technique is 95 percent, with a recurrence rate of 23 percent.

The team from Children’s National, which included Princeton intern Michelle Richardson and Allison Matthews, focused on 81 articles found in the PubMed, Cumulative Index to Nursing and Allied Health Literature (CINAHL), and Scopus databases. From this data, they determined that the average rate for correction of clubfoot via the Ponseti technique is 95 percent, with a recurrence rate of 23 percent.

The researchers also found that 78 percent of recurrence was due to five factors: brace non-compliance, lack of parent education, functional brace issues, casting issues and poor patient tolerance.

Looking further into non-compliance of bracing, the team discovered that the average non-compliance rate was 27 percent, and that factors affecting non-compliance in about half the cases included parent education, financial difficulties, practical brace issues, social difficulties and cultural issues.

The team’s findings should be helpful in establishing programs aimed at decreasing recurrence rates and improving compliance with bracing in children treated for clubfoot.

Karun Sharma

Osteoid osteoma successfully treated with MR-HIFU

Karun Sharma

Doctors from the Sheik Zayed Institute for Pediatric Surgical Innovation at Children’s National Health System have completed a clinical trial that demonstrates how osteoid osteoma, a benign but painful bone tumor that commonly occurs in children and young adults, can be safely and successfully treated using an incisionless surgery method called magnetic resonance-guided high-intensity focused ultrasound (MR-HIFU).

Published in The Journal of Pediatrics on Aug. 17, 2017, the study compares nine patients, ages 6 to 16 years old, who were treated for osteoid osteoma using MR-HIFU with a nine-patient historical control group, ages 6 to 10 years old, who were treated at Children’s National using radiofrequency ablation (RFA) surgery, the standard treatment at most U.S. hospitals. The study results show that treatment using MR-HIFU is feasible and safe for patients, eliminating the incisions or exposure to ionizing radiation that is associated with the RFA procedure. Children’s National is the first U.S. children’s hospital to successfully use MR-HIFU to treat osteoid osteoma.

CT-guided RFA, the most commonly used osteoid osteoma treatment, requires drilling through muscle and soft tissue into bone and also exposes the patient and operator to radiation from the imaging necessary to guide the probe that is inserted to heat and destroy tumor tissue.

“Our objective is to provide a noninvasive treatment option for children with osteoid osteoma and we’re very pleased with the results of this clinical trial,” says Karun Sharma, M.D., Ph.D., director of Interventional Radiology at Children’s National and principal investigator for the osteoid osteoma trial. “We have now shown that MR-HIFU can be performed safely with clinical improvement that is comparable to RFA, but without any incisions or ionizing radiation exposure to children.”

High-intensity focused ultrasound therapy uses focused sound wave energy to heat and destroy the targeted tumor under MRI guidance. This precise and controlled method does not require a scalpel or needle, greatly reducing the risk of complications like infections and bone fractures. It is also a faster treatment option, with expected total procedure time of 90 minutes or less. In the U.S., MR-HIFU is used to treat uterine fibroids and painful bone metastases from several types of cancer in adults, but has not previously been used in children.

This breakthrough is the latest from the Image-Guided Non-Invasive Therapeutic Energy (IGNITE) program, a collaboration of the Sheikh Zayed Institute and the departments of RadiologyOncologySurgery, and Anesthesiology at Children’s National. The goal of the IGNITE program is to improve the quality of life and outcomes for pediatric patients through the development and clinical introduction of novel minimally invasive and noninvasive surgery technologies and combination therapy approaches. The team is led by Peter Kim, M.D., Ph.D., vice president of the Sheikh Zayed Institute.

“The use of MR-HIFU ablation of osteoid osteoma is a perfect example of our mission in the Sheikh Zayed Institute to make pediatric surgery more precise, less invasive and pain-free,” says Dr. Kim. “Our leading team of experts are also exploring the use of MR-HIFU as a noninvasive technique of ablating growth plates and pediatric solid tumors. We also have another clinical trial open for children and young adults with refractory soft tissue tumors, which is being performed in collaboration with Dr. Bradford Wood’s team at the National Institutes of Health, and if successful, it would be the first in the world.”

In addition to Drs. Sharma and Kim, the Children’s National team for the ablation of osteoid osteoma clinical trial included: AeRang Kim, M.D., Ph.D., pediatric oncologist; Matthew Oetgen, M.D., division chief of Orthopaedic Surgery and Sports Medicine; Anilawan Smitthimedhin, M.D., radiology research fellow; Pavel Yarmolenko, Ph.D., Haydar Celik, Ph.D., and Avinash Eranki, engineers; and Janish Patel, M.D., and Domiciano Santos, M.D., pediatric anesthesiologists. Ari Partanen, Ph.D., a senior clinical scientist from Philips, was also a member.

Laura Tosi

Giving voice to adult osteogenesis imperfecta patients

Laura Tosi

“I have a number of OI patients moving into adulthood who cannot get care in the adult world, because my colleagues who care for adults have less experience with the disease and because caring for OI adult patients is largely uncharted territory,” says Laura L. Tosi, M.D.

With the influx of increasingly effective technology at our fingertips, the landscape of patient care for complex diseases has changed for the better in recent years. Doctors and researchers can accelerate new discoveries and improvements in patient care by querying and utilizing patient data gathered from all over the world.

For Laura L. Tosi, M.D., director of the Bone Health Program at Children’s National Health System, these changes have galvanized years of research into patients with osteogenesis imperfecta (OI), a population that is particularly difficult to trace into adulthood.

OI is a rare genetic disorder characterized by excessively fragile bones with a high susceptibility to recurrent fractures. Commonly known as “brittle bone disease,” OI is mostly caused by mutations in type I collagen genes. The severity of the disease varies widely and a cure for OI still remains to be found. Currently, treatment methods include medications, physical and occupational therapy, as well as surgery – all of which aim to reduce risk of further fracture, help patients manage pain and promote a healthy lifestyle.

Two of the most critical challenges that accompany the treatment of rare diseases, however, are the paucity of data on the adult patient experience and the challenge of transitioning patients safely from the multispecialty clinics frequently available in childhood to adult care givers who may have never seen the disorder in their career.

Through her research, Dr. Tosi aims to fill these critical knowledge gaps, and has found the Patient-Reported Outcome Measurement Information System (PROMIS®), the patient-reported outcome platform funded by the National Institutes of Health, to be particularly advantageous.

PROMIS® harnesses a set of measurement tools that uses computer adaptive technology and person-centered measurements to evaluate and monitor physical, mental and social health in adults and children. These tools quickly tailor themselves to individual responses and, because of their user-friendly design, provide a level of convenience and easy accessibility that other platforms lack.

“I have a number of OI patients moving into adulthood who cannot get care in the adult world, because my colleagues who care for adults have less experience with the disease and because caring for OI adult patients is largely uncharted territory,” says Dr. Tosi. Realizing the importance of giving voice to adults with OI, Dr. Tosi has harnessed a diverse range of standardized PROMIS® tools to attempt to capture a more complete understanding of the patient experience, ranging from the quality of social participation and peer relationships to physical and emotional distress.

When her first PROMIS®-based mailing to patients received an overwhelming response of more than 1,100 respondents in just 90 days, Dr. Tosi knew that pushing this research forward and out into the community was imperative. The results from that first survey, published in 2015 in the Orphanet Journal of Rare Diseases, demonstrated that adults with OI generally reported lower physical health status and were more likely to struggle with auditory and musculoskeletal problems.

Continued research in this area will not only generate much-needed knowledge about long-term healthcare issues and needs for OI patients, but also help clinicians improve their current treatment methodologies to anticipate these concerns ahead of time, if possible.

“The number of responses to our first survey demonstrated that patients really want to be heard. When you give them tools and ask them to tell you about themselves in ways that they hope will change how you practice, they want to help,” says Dr. Tosi, “because everyone wants to grow old well.”

At the end of August, Dr. Tosi will present her research at the 13th International Conference on Osteogenesis Imperfecta in Oslo, Norway. She also presented her research at the 8th International Conference on Children’s Bone Health as well as at the 17th Annual OI Foundation Scientific Meeting.

Now taking part in designing and executing a national natural history study of patients with OI, Dr. Tosi plans to lead the charge for incorporating and implementing PROMIS® tools into the study. “Once we improve our tools, we will have the ability to query individuals from Alaska to Timbuktu, and provide a far more comprehensive understanding of this very complex and multi-faceted disorder. Harnessing the power of the internet and engaging the patient in delineating their disorder as well as their response to treatment offer a giant step forward in caring for individuals with rare diseases,” she says.

Karun Sharma, M.D., poses with two patients

Treating osteoid osteoma with MR-HIFU

Karun Sharma, M.D., poses with two patients

Karun Sharma, M.D., poses with two patients who participated in the MR-HIFU trial for pediatric osteoid osteoma.

Doctors from the Sheikh Zayed Institute for Pediatric Surgical Innovation and surgeons from Children’s National are the first in the U.S. to use Magnetic Resonance-Guided High-intensity Focused Ultrasound (MR-HIFU) to treat pediatric osteoid osteoma.

The trial, led by Principal Investigator Karun Sharma, M.D., Ph.D., Director of Interventional Radiology at Children’s National, began in 2015 and is demonstrating early success in establishing the safety and feasibility of noninvasive MR-HIFU as an alternative to the current, more invasive approaches to remove tumor tissue.

Osteoid osteoma is a painful, but benign, bone tumor that commonly occurs in children and young adults. Removal generally requires orthopaedic surgery to scrape the tumor from the bone or CT (computerized tomography) image-guided radiofrequency ablation (RFA), which is less invasive than surgery but is associated with ionizing radiation exposure and requires drilling through muscle and soft tissue into bone.

MR-HIFU, on the other hand, is a precise and controlled method that does not require a scalpel or needle, greatly reducing the risk of complications, including infections and bone fractures. Even better, it promises reduced procedure time, typically an hour or less.

“Our team set out to provide a noninvasive and radiation free treatment option for children with osteoid osteoma and our pilot feasibility and safety trial is almost completed. We have treated 9 patients and we’re very pleased with the success of the treatments so far. Although follow up will continue for another year, results to date that show that MR-HIFU may be a completely non-invasive and radiation free treatment for osteoid osteoma,” Dr. Sharma says. “Several of the children we treated were very active prior to the onset of their tumor, one a soccer player and the other a swimmer, but because of pain from the tumor, they were unable to enjoy their favorite activities, until now.”

“The use of MR-HIFU ablation of osteoid osteoma is a perfect example of our mission in the Sheikh Zayed Institute to make pediatric surgery more precise and less invasive,” adds Peter Kim, M.D., C.M., Ph.D., Vice President of the Sheikh Zayed Institute, who leads the Image Guided Non-Invasive Therapeutic Energy (IGNITE) program.

IGNITE is a joint clinical and research collaboration between the Sheikh Zayed Institute and the Divisions of Radiology, Oncology, Surgery, and Anesthesiology at Children’s National. MR-HIFU is also being used to treat pediatric refractory soft tissue tumors, a first-in-the-world clinical trial that is a collaboration between Children’s National and the NIH Center for Interventional Oncology directed by Bradford Wood, MD. Additionally, the IGNITE team has started preliminary work to explore applications of MR-HIFU for noninvasive ablation of growth plates and pediatric solid tumors.

In addition to Drs. Sharma and Kim, the team for the ablation of osteoid osteoma clinical trial includes: AeRang Kim, MD, PhD, pediatric oncologist; Matthew Oetgen, M.D., Division Chief of Orthopaedic Surgery and Sports Medicine; Kaleb Friend, M.D., pediatric orthopedic surgeon; Pavel Yarmolenko, Ph.D., Haydar Celik, Ph.D., and Avinash Eranki, biomedical engineers; Viktoriya Beskin, MR technologist; and Janish Patel, M.D., and Domiciano Santos, M.D., pediatric anesthesiologists.

MAGEC Rod Tool

MAGEC growing rod improves orthopaedics

MAGEC Spinal Growing Rod Inside Boy

After implanting a MAGEC Spinal Growing Rod, doctors use an external remote control to lengthen the magnetically controlled rod as the child grows.

Children’s National Health System was among the first in the country to offer a novel spinal growing rod for children with scoliosis after it was approved by the FDA just three years ago – and has now treated 30 patients with this innovative technique. The MAGEC™ (MAGnetic Expansion Control) Spinal Growing Rod is a non-invasive treatment for children with early onset scoliosis.

After the initial procedure to implant the rod, doctors use an external remote control outside of the body to lengthen the magnetically controlled rod as the child grows. The adjustments are non-invasive, reducing the number of surgeries required during the course of treatment.

Growing rods have become effective tools for children whose spinal curvature is too significant to control with bracing or casting. The rods—which are surgically attached to the spine above and below the curve and then traditionally lengthened during follow-up surgical procedures—allow the spine to continue growing while managing the curve until the child is old enough for spinal fusion.

The problem: Children must bear the physical and psychological burden of undergoing lengthening procedures every six to 12 months until they are skeletally mature enough to have spinal fusion—typically around age 10 for girls and age 12 or 13 for boys.

Now, instead of returning to the hospital for a major surgery to adjust growth rods twice a year, children with the MAGEC rod have adjustments in just a few minutes four times a year – minus invasive surgery and recovery time, says Matthew Oetgen, M.D., Division Chief of Orthopaedic Surgery and Sports Medicine and Director of Orthopaedic Research at Children’s National.

“Traditional growing rods work, but they require multiple surgeries that increase complication rates and time spent in the hospital,” he says. “We treat many children each year who have or are candidates for growing rods, so it’s important for us to embrace new technology to make the lengthening process easier and less painful for children while decreasing morbidity.”

Children’s National orthopaedic surgeons lengthen the MAGEC rod every three to four months in the office using the electronic remote control. They then monitor the scoliosis and treatment progress with radiographs. Like traditional growing rods, MAGEC is a means, not an end—the system provides a bridge treatment spanning the years between the initial lengthening surgery and spinal fusion.

Dr. Oetgen says this game-changing technology may not be the right solution for every patient, but is the preferred choice because the patients can avoid some additional surgeries down the line. Patients in the 5-7 year age range at the time the rod is in place would potentially face 10 years of surgeries every six months with traditional growing rods.

“We’ve eliminated these regularly scheduled procedures, which is great if you’re a healthy kid,” Oetgen says. “But if you are a kid with other health challenges, such as a neuromuscular disease – it’s really life changing not to have to go into surgery every six months. It saves them a tremendous amount of intervention.”

Following MAGEC’s approval by the U.S. Food and Drug Administration in February 2014, surgeons at Children’s National performed two of the first 15 MAGEC implantations in the country, and the first in the greater Washington, DC, area. MAGEC rods are approved for children with scoliosis greater than 50 degrees in magnitude and under 10 years of age.

On the horizon for this new technology are some improvements Oetgen says would allow physicians using MAGEC rods to improve the patient experience even more. “Smarter” devices could potentially tell doctors how much lengthening has actually occurred after they’ve pushed the buttons on the remote control – instead of having to follow up the procedure with an x-ray to see how the rod interacted with soft tissue around the spine, he says.

And the next generations of MAGEC rods may be smaller devices, allowing younger, smaller kids to reap the benefits.

“In the future these improvements will allow us to treat more patients, and allow us to know what we’re doing and what kind of feedback we’re getting,” Oetgen says.

Spinal fusion surgical home helps kids go home sooner

scoli-1

The first of its kind for pediatric patients, the Children’s National Spinal Fusion Surgical Home implements a newly developed model of care to streamline and optimize the spinal fusion process for adolescent idiopathic scoliosis patients.

Using frameworks of care used in adult models, along with best practices and literature reviews, a multidisciplinary team developed the first Spinal Fusion Surgical Home for pediatric patients. It standardizes the infection-control process, pain-management pathway, and physical-therapy program for patients undergoing spinal fusion.

“This model eliminates variability in the care process and increases the quality of care for pediatric patients,” said Matthew Oetgen, MD, MBA, Chief of Orthopaedic Surgery and Sports Medicine. “It’s just the start—by developing this model specifically for our young patients with adolescent idiopathic scoliosis, we are paving the way for a number of other kids that require different kinds of surgeries.”

Hallmarks of the spinal fusion surgical home
From pre-operative care through recovery, the Spinal Fusion Surgical Home streamlines care with an emphasis on increasing quality outcomes for patients. Children’s National provides an informational website and a single point of contact for scheduling procedures and pre-operative laboratory exams. Before surgery, patients and families attend an evening education class that features presentations from orthopaedic nurse practitioners, physical therapists, and anesthesiologists.

After surgery, a nurse follows up by phone to assess how the patient is handling pain and healing.

Increasing the quality of care
By implementing these standardized protocols, Children’s National has seen a decrease in the average length of stay for spinal fusion patients from about five days to three and a half days. The surgical home also has reduced the transfusion rate from 30 to 12 percent, and patient pain scores have decreased.  “Patients are getting better faster with less pain, and are getting to leave the hospital sooner,” says Karen Thomson, MD.

Children’s National also is creating surgical homes for sickle cell disease patients, who need a variety of different types of surgery, as well as for children who need Nissen fundoplication and heart surgery.