CHOC’s growing thyroid surgery program can compete with the busiest centers when it comes to favorable outcomes, research shows

When Dr. Kevin Huoh arrived at CHOC in September 2013, the hospital wasn’t doing a lot in the way of thyroid surgeries and instead was referring out most cases.

A highly regarded pediatric otolaryngologist, Dr. Huoh has a special interest in thyroid surgery, especially thyroid cancer surgeries. So, working with endocrinologists at CHOC, he spearheaded a multi-disciplinary thyroid surgery program that steadily has grown since then.

Now, in a recently published research paper, Dr. Huoh and co-author Dr. Himala Kashmiri, a CHOC endocrinologist, have shown that the growing program at CHOC enjoys favorable outcomes comparable with those found at the nation’s largest-volume pediatric thyroid surgery centers.

“Other research papers and guidelines say in order to have the best outcomes for thyroid surgery, you need to do 30 surgeries per year,” Dr. Huoh says. “Fortunately, pediatric thyroid surgery is fairly uncommon. This makes it difficult for many newer thyroid surgery programs to achieve these numbers. Our study shows outcomes similar to those at higher volume centers.”

In the paper, published in early February 2021 in the International Journal of Pediatric Otorhinolaryngology, Drs. Huoh and Kashmiri studied 31 patients who underwent thyroid surgery at CHOC between 2014 and 2020. The numbers have grown from two to three a year to nearly nine, and are expected to continue to increase, Dr. Huoh says.

Key finding in the research paper: The 31 CHOC thyroid surgery patients, who ranged in age from 8 months to 20 years, experienced a rate of complications comparable to larger-volume pediatric thyroid surgical programs.

“Recent publications have advocated that patients in need of thyroid surgery should be referred to high-volume surgical centers, asserting that high-volume centers experience fewer complications,” the paper states. “In contrast to recent publications, our study demonstrates that low-volume and intermediate-volume thyroid surgery centers can achieve comparable results.”

A key reason why, according to the paper, is having a multidisciplinary team of pediatric otolaryngologists and endocrinologists, such as the team at CHOC.

Kids at greater risk of cancer

The thyroid is a gland that makes and stores hormones that help regulate the heart rate, blood pressure, body temperature, and the rate at which food is converted into energy.

The prevalence of thyroid masses in children is much lower compared with adults. But such masses in children tend to carry a greater risk of harboring malignancy compared with their adult counterparts. And thyroid surgery in the pediatric population is associated with a higher rate of complications than adult thyroid surgery.

Thyroid cancer is on the rise around the world, including in adolescents, Dr. Huoh notes.

“We saw a definite need for this program at CHOC,” he says.

Dr. Huoh works very closely with Dr. Kashmiri, a pediatric endocrinologist, in CHOC’s thyroid cancer program.

“When I first started here in 2015, as director of the thyroid cancer clinic program here at CHOC, our typical workflow was to find ENT surgeons for our patients in the community of Orange County or even Los Angeles,” Dr. Kashmiri says. “However, rather quickly as a pediatric endocrinologist who puts the patient first, I gained accelerated confidence with Dr. Huoh’s expertise, interpersonal skills, and poise to handle our simple-to-complex neck surgeries.

“This has been a game-changing experience for us to have a surgeon who we trust and value to take care of patients with excellent outcomes whom we recommend fully without any hesitation. I would just like to say thanks to Dr. Huoh for bringing his passion and commitment to CHOC and the children we serve.”

Scary experience, good outcome

In November 2019, Molly Pearce noticed a lump on the left side of her throat.

Then 13, Molly ended up at CHOC after a friend of her mother, Jacqueline, recommended going there and after Jacqueline did a lot of research.

“Dr. Huoh’s name kept coming up,” Jacqueline says.

Molly Pearce had her thyroid removed by Dr. Huoh in 2020 after discovering a lump in her neck.

 The two met with Dr. Huoh in January 2020.

“From the second we met,” Jacqueline says, “we knew we were going to love him. He’s got a great bedside manner. He’s reassuring. He’s thorough. He answered all our questions. He spent a lot of time with us, which isn’t always the case with busy surgeons. We definitely got the impression that he cared a lot.” 

In February 2020, Dr. Huoh removed one half of Molly’s thyroid.

The tumor was encapsulated, but pathology reports after surgery turned up concerning cancerous cells in the mass. Dr. Huoh went ahead with a second surgery, removing the second half of Molly’s thyroid in April 2020.

“It was a very scary time, especially during the pandemic,” Jacqueline recalls, “but CHOC offered us a broad range of resources should we want to reach out and get some support. COVID-19 added an extra layer of fear. I have to say we felt comfortable in terms of the level of cleanliness. Molly had to do COVID tests. I really feel it was a challenging time kicked up to a much more challenging time, and we still felt comfortable and safe and well cared for.”

Jacqueline praises Dr. Huoh’s skill as a surgeon.

“A plastic surgeon could not have done the incision as well as he did,” says Jacqueline, who explained that only a faint pink incision line remains on Molly’s neck.

“You wouldn’t even know she was worse for the wear,” Jacqueline says. “Everything about the care we got at CHOC, from pre-registration to when we got to the hospital, to the post-op follow-ups, was exceptional.”

With regular blood work and ultrasounds, Molly continues to be under the continuous care and careful watch of Dr. Kashmiri.

“He has a great way of communicating and empathizing with his patients in a comfortable setting,” Jacqueline says. “We know we are in good hands. He has truly been a blessing to us on our journey.”

Molly now is 15. She will need to be on a thyroid supplement the rest of her life, but says she feels great.

“I feel good but sometimes feel a phantom thing,” she says. “I feel like my thyroid is still there but it’s not. Overall, I feel amazing.” 

Molly, a freshman at Dana Hills High School who loves beach volleyball and yoga and is a member of the National Charity League, says she appreciates the personal touches CHOC provided went she went in for her surgeries.

Ties to nuclear medicine program

Dr. Huoh notes that some patients need radioactive iodine treatment after thyroid cancer surgery. Such treatment requires a nuclear medicine program. It’s fortuitous, he says, that construction is under way on space that will house CHOC’s first nuclear medicine program.

The new space, which totals some 4,000 square feet, is scheduled to open in fall 2021 in the Bill Holmes Tower at CHOC’s main hospital campus.

 Its opening will be especially important for patients undergoing thyroid surgery who now must go to other hospitals for radioactive iodine treatment, says Dr. Hollie Lai, a radiologist who will be in charge of CHOC’s nuclear medicine program.

“This will be a huge benefit to patients,” Dr. Lai says. “Many of our thyroid cancer patients now have to go to adult facilities.”

Such treatment involves giving patients medicine, usually orally, that has radiation in it that zaps away remnants of cancerous tissue following surgery. Thyroid surgery patients will be one part of CHOC’s nuclear medicine program, which will provide full-service therapies in addition to research.

She praises Dr. Huoh’s skills.

“He’s a great surgeon who relates very well to his patients,” Dr. Lai says.

Dr. Huoh has big hopes for CHOC’s thyroid surgery program.

“Our goal is to be one of those centers doing 30 or more surgeries a year,” he says. “It’s nice to be able to show great outcomes on our way to becoming one of the high-volume pediatric surgery thyroid programs.”

Learn more about CHOC’s otolaryngology program.

Read another recent example of research conducted by Dr. Kevin Huoh.

Surgeon, NICU nurse say their lifesaving professions have made them better fathers

The patient was 6, a boy – the same age as a pediatric general and thoracic surgeon Dr. Peter Yu’s son, “P.K.”

The patient’s kidney cancer had spread to his lungs.

When Dr. Yu recently performed surgery on the boy, he caught himself thinking of P.K., whose full name is Peter Kai Yu – a ball-sport-loving kid with grown-up tastes in food such as sushi.

“When I looked at him,” Dr. Yu recalls of the patient, “I saw P.K. I thought, ‘What would I do for my son?’ And I would do anything for him.”

With Father’s Day this Sunday, Dr. Yu and Gene Paredes, a neonatal intensive care unit (NICU) nurse at CHOC at Mission Hospital, reflected on the challenge of balancing their demanding and often emotionally exhausting work with fatherhood.

Both Gene and Dr. Yu are married with three children.

Both say their professions make them better fathers, and both say having kids makes them better at what they do.

Ample time with children

Gene has been a father almost as long as he’s been a nurse.

His son, Gabriel, is 20. Gene has been a nurse at CHOC for 21 years (23 years overall).

Gabriel is in college, as is his 18-year-old sister, Gillian. Gene’s other daughter, Eliotte, 14, just started high school.

Even though his parents both were nurses, Gene never grew up thinking he wanted to be one, too.

Gene Paredes, a nurse in the NICU at CHOC Mission, and his family

But the Mission Viejo native did just that, joining CHOC in 1999 after completing training for two years in a neonatal intensive care program in Berkeley.

Like his father, who worked three 12-hour shifts per week, Gene has been able to be involved in his kids’ lives because of his work schedule.

“Working three days a week,” Gene says, “I was one of the few dads who were able to be involved in mid-week classroom activities at my kids’ schools. That was kind of rare. You didn’t see a lot of dads there.”

Gene and his wife, Chantelle, who used to teach, decided that the benefits of her being a full-time mom outweighed the challenges of being a single-income family.  

And that decision has paid off.

Over the years, Gene and Chantelle have enjoyed travelling with their children.

They did an RV road trip up the coast to the Pacific Northwest and have been to various national parks and states throughout the U.S. Two years ago, they vacationed in Paris and London.

Gene and his family taking in the sites of England

At CHOC Mission, where for years he was the only male nurse, Gene works throughout the hospital because he has special training in placing PICC (peripherally inserted central catheter) lines, which are used to dispense medications and liquid nutrition. At CHOC Mission, he also performs ultrasound-guided IV placements.

For 2 ½ years, Gene also picked up shifts at CHOC’s campus in Orange in the main NICU and Small Baby Unit.

But he spends most of his time caring for sick babies in the NICU at CHOC Mission.

“I think being in healthcare, you realize there are a lot of things that can go wrong in childhood, such as illnesses and accidents,” Gene says. “I definitely had an appreciation for having healthy children. Knock on wood, I’ve never had to bring any of my kids to the hospital.”

Being a nurse has huge benefits when raising kids, Gene says.

“I approached fatherhood with a lot of confidence,” he says. “I taught my wife how to give our babies a bath. And she never worried about the kids getting sick. She was like, ‘Gene’s got this. He knows babies.’” 

Being a male and a father, Gene brings a unique presence to the NICU.

“A lot of the focus tends to be around the moms and the connection they have with their babies,” he says. “I think me being a male allows fathers to have someone to connect with. I change diapers, I feed the babies – I do all the hands-on things. I like to empower fathers to get in there and get very involved — to make them feel they can be as involved as much as the moms.”

Gene is known throughout the hospital for his calm demeanor in stressful situations.

“As a nurse and father, I hope that my calm energy and presence would bring comfort to parents experiencing the stress and unknowns of their child’s hospitalization,” he says.

On Father’s Day, Gene and his family will host a large afternoon feast with relatives at a favorite park in Dana Point.

“Then we’ll take a sunset walk on the beach,” he says.

Off cooking duty this Sunday

Dr. Yu usually relieves his wife, Jean, of cooking duties on weekends, when he’s off his hectic weekly work schedule that often totals 80 hours.

This Sunday will be different.

“I told him I would cook for him,” Jean says, adding: “He’s a very good cook.”

Being a former clinical nurse, Jean totally gets the demands of her husband’s profession.

“I get what the daily grind is like and things that may come up,” Jean says. “As a family, we try to cherish every moment, even just little things like watching a show together at the end of the day. He can’t make every event, but the kids are very understanding and very aware he’s probably helping out a sick baby or a sick kid, and they don’t hold that against them.” 

Dr. Peter Yu, a CHOC pediatric general surgeon and co-medical director of the Fetal Care of Southern California, and his family

The two met in the surgical ICU at the medical center at UC San Diego School of Medicine, where Dr. Yu completed his internship, residency, and research fellowship in general surgery. Jean was a surgical ICU and trauma nurse there, and they met while taking care of a very sick patient.

Married for 11 years, the Yus have three children: Max, 10; Sasha, 8; and P.K. They dated for two years before marrying. Dr. Yu proposed to Jean in Nigeria while both were on a surgical mission. 

Almost every day, Dr. Yu awakes at 4:30 a.m. to hit the pools. He’s an avid swimmer who will compete in the U.S. Masters Swimming National Championships in Greensboro, N.C., on July 26.

Max also loves to swim, and is a voracious reader.

“He’ll read a Harry Potter book in one day,” Dr. Yu says.

Sasha loves to dance and is a huge avocado fan.

Dr. Yu hits the sack around his kids’ bedtime.

“Usually 8:30 – 9 p.m. is really pushing it,” Jean says.

Dr. Yu says once he’s at home, he strives to be present with his children. Things have been even more hectic than usual at work recently, with the just-opened Fetal Care Center of Southern California, of which Dr. Yu is co-medical director.

“Our family works very well,” Dr. Yu says. “The credit really goes to Jean. She’s the chief operating officer of our family. I am so blessed to have her. She really allows me to work. Being a nurse, she knows how important it is for me to take care of these kids (at CHOC). She never gives me grief when I have to work, and that’s huge.”

Dr. Yu has been at CHOC for six years. Jean worked at CHOC for two years in the post anesthesia care unit (PACU).

“Jean was an amazing nurse,” Dr. Yu says. “I think she could have been a high-level nursing leader, but she sacrificed her career to follow me.”

Hospital work lends perspective to mishaps at home, such as a scraped knees, Jean says.

“Things that happen at hospitals can be completely life-changing for families,” she says. “So, when things happen at home, we don’t get too alarmed.” 

Dr. Yu and his son take it to the hoop with authority

Dr. Yu, whose parents emigrated to the United States in the 1960s, was born in America, and spent most of his early years in St. Louis, Mo. He has an older brother, David, also a physician, who adopted a boy from China who now is 10.

Dr. Yu says he became sold on California after attending Stanford University as an undergraduate, majoring in psychology.

It’s a good thing Dr. Yu has a ton of energy. He will need it to continue his balancing act of caring for sick and injured kids at CHOC and tending to his three young kids at home.

Says Dr. Yu: “You have to be present in the operating room, and you have to be present for your family.”

New clinical evidence shifts approach to vesicoureteral reflux (VUR) care

One of the most common conditions managed by pediatric urologists is vesicoureteral reflux (VUR), which occurs when urine in the bladder flows back into the ureters and/or kidneys. For years, the accepted practice involved diagnosing and treating the condition in all patients, regardless of symptoms, which also included routine follow-up testing. Recently, this approach has been questioned, and CHOC urologists have determined that repeated follow-up testing for some patient groups is often unnecessary.

“There is a wide spectrum of severity with VUR, from asymptomatic disease that is incidentally found to severe disease leading to subsequent kidney infections, renal scarring and deterioration of renal function,” says Dr. Heidi Stephany, a pediatric urologist at CHOC. “Our goal is to stratify patients by risk factors and severity to diagnose and treat at the appropriate level.”

Dr. Heidi Stephany, pediatric urologist at CHOC

Evaluating patient data from 2014 to present day, CHOC urologists have prospectively reviewed the diagnosis, treatment and outcome data to stratify VUR patients into three risk categories, including:

  • Low Risk: Female, VUR grade 1–3, without bladder and bowel dysfunction (BBD); circumcised males, any VUR grade, without BBD; and uncircumcised males, over 1 year of age, any VUR grade, no BBD
  • Intermediate Risk: Female, VUR grade 1–3, with BBD; female, VUR grade 4–5, presents without UTI, any BBD status; circumcised male, any grade VUR, with BBD; uncircumcised male, over 1 year of age, any grade VUR, with BBD; uncircumcised male, under 1 year of age, any VUR grade, any BBD status
  • High Risk: Female, VUR grade 4 or 5, present with UTI, any BBD status

These classifications now drive patient care at CHOC. Historically, many children with low-risk VUR presented no symptoms and often over time, those with asymptomatic VUR and lower grades outgrew the condition, typically by age 5. Despite this, when VUR was diagnosed, even asymptomatic VUR often entailed annual testing. At CHOC, repeat testing is reserved for those with persistent symptoms such as urinary tract infections with fever or those in the high-risk category.

A variety of tests help diagnose VUR, including abdominal ultrasound and the gold standard, voiding cystourethrogram (VCUG). While diagnosing patients with high-risk disease is important, it’s equally as important to minimize over-diagnosis of patients with low-risk disease who will likely remain asymptomatic with no long-term sequelae. “VCUG is not pleasant for kids, so we limit its use,” Dr. Stephany says. “We want to focus on finding the patients at the highest risk for long-term sequelae who truly require treatment to prevent further upper tract damage.”

Stratified treatment for VUR begins with the least-invasive option: expectant management with behavioral modifications to ensure healthy bowel and bladder habits. Often, lower grades of VUR resolve as the child grows. In children at intermediate or high risk, a low-dose daily antibiotic may be prescribed along with an intent focus on bowel and bladder management in the toilet-trained child. Surgical intervention, such as an open ureteral reimplant or endoscopic treatment with injection of Deflux® (a bulking agent to prevent urinary reflux) is also available. In general, surgical intervention is offered to those with high-grade VUR who have recurrent kidney infections and potential for further kidney damage.

Regardless of the grade or risk group, CHOC urologists have a singular purpose. “Our goal is to protect the kidneys and bladder,” Dr. Stephany says. “There are many ways to approach VUR, and there is no standardized treatment. By constantly evaluating our diagnostic and treatment best practices, we force ourselves to consider whether a change in care would mean better outcomes for our patients. When supported by clinical evidence, we make the appropriate modification and VUR patients reap the benefit.”

Our Care and Commitment to Children Has Been Recognized

CHOC Children’s Hospital was named one of the nation’s best children’s hospitals by U.S. News & World Report in its 2020-21 Best Children’s Hospitals rankings and ranked in the urology specialty.

Learn how CHOC’s urology care, ongoing treatment and surgical interventions preserve childhood for children in Orange County, Calif., and beyond.

Study of COVID-19 infection rates among CHOC’s Emergency Department personnel suggests most got virus through community exposure

A team from CHOC has published original research on the prevalence of COVID-19 infection among its Emergency Department workers during the early stages of the pandemic.

A key finding of the study, called PASSOVER (Provider Antibody Serology Study of Virus in the Emergency Room), suggests that most infections were transmitted through community exposure rather than co-workers, although the study stopped short of drawing a definitive conclusion based on the relatively small sample size of workers who agreed to be tested for SARS-CoV-2.

Researchers observed a seroconversion rate of about one new positive case every two days during the period from April 14-May 13, 2020, during which 143 CHOC ED personnel were repeatedly tested for the virus. They included doctors, physician assistants, nurse practitioners, nurses, medical technicians, secretaries, monitor technicians, and additional administrative staff.

“The acquisition of seropositivity in our study group appeared to follow a linear trend, which is not consistent with the exponential rate of growth that would be expected for transmission within a closely interacting group of people,” the study concludes.

The research project, the results of which were electronically published on April 9, 2021 in the Western Journal of Emergency Medicine, was led by Dr. Theodore Heyming, chair of emergency medicine at CHOC, and Dr. Terence Sanger, a physician, engineer, and computational neuroscientist and vice president, chief scientific officer at CHOC, and vice chair of research for pediatrics at the UCI School of Medicine. The other co-authors of the study are John Schomberg, PhD, CHOC’s Department of Nursing; and Aprille Tongol, Kellie Bacon, and Bryan Lara, all of CHOC’s Research Institute.

The study noted that there is limited data that is publicly available on the seroprevalence of SARS-CoV-2 among healthcare workers. Another of the report’s key findings was that rapid antibody testing may be useful for screening for SARS-CoV-2 seropositivity in high-risk populations such as healthcare workers in the ED.

In the CHOC study, blood samples were obtained from asymptomatic ED workers by fingerstick at the start of each shift from April 14-May 13, 2020. Each worker’s blood sample was obtained every four days until the end of the study period. In addition, a nasopharyngeal swab (NPS) was collected from each participant on the date of study entry.

At the time of the study, 35 percent of the participants had known exposure to a COVID-19-positive individuals within the preceding five days.

Depending on the method used for analysis, the seroprevalence of SARS-CoV-2 among CHOC’s pediatric ED workers ranged from 2 percent to 10.5 percent – levels that were slightly higher than those reported for the local general population, the study found.

“This study would benefit from replication at additional sites that draw from larger samples of ED staff,” the report says.

Learn more about CHOC’s COVID-19 vaccine clinic.

Neuroscience leaders discuss innovations and their successes, and the need for much more work to be done

Two of CHOC’s leading pediatric neurosurgeons recently shared their insights on how innovation is helping to close the gap between clinical needs and the availability of pediatric devices, but how there is much more work to be done to get critically ill kids the treatments they need.

The webinar, “From Clinical Insight to Commercialization: Innovations That Can Transform Pediatric Healthcare,” featured Dr. Suresh N. Magge, CHOC CS Neurosurgery Division Chief, and co-director of CHOC’s Neuroscience Institute, and Dr. Michael G. Muhonen, the institute’s previous co-director.

Hosting the “OC LIFe (Lifesciences Innovators Forum)” on April 28, 2021 was Dr. Terence Sanger, a physician, engineer, and computational neuroscientist and vice president, chief scientific officer at CHOC, and vice chair of research for pediatrics at the UCI School of Medicine.

“As innovators, we should never be satisfied,” said Dr. Sanger, who specializes in movement disorders and who helped pioneer deep brain stimulation, which has yielded positive outcomes. “An innovative and collaborative approach is required so that pediatric patients can have access to the fit-for-purpose devices they need.”

Brain tumor treatments

Drs. Magge and Muhonen took turns discussing new neurosurgical technologies and opportunities for interventions.

Dr. Magge focused on new technology that has been used to treat brain tumors, which are a different breed compared to adult brain tumors. More often, Dr. Magge said, pediatric brain tumors are of a lower grade and can be treated.

“Many kids have gone on to live good lives thanks to innovation, research, and applying the technologies we have,” Dr. Magge said.

In one example, he detailed how microsurgical techniques have greatly aided in the removal of a craniopharyngioma, a benign tumor that usually arises in the base of the brain near the pituitary gland that can be dangerous or life threatening if not treated.

“If you can get the tumor out,” Dr. Magge said, “you can cure the patient. But it’s challenging because it’s in a deep part of the brain.” 

During the procedure, the neurosurgeon must locate some of the natural divides of the brain and separate them out to get to the tumor. Microsurgery allows the neurosurgeon to work between very narrow areas.

With a technology known as surgical navigation, neurosurgeons can pinpoint exactly where they are in the brain and get to very specific areas. Another technology is a powerful microscope that magnifies small areas of the brain. In addition, ultrasound and MRI within the operating room can tell you in surgery if there is any tumor left. 

“This is all thanks to innovation and technology that we are incorporating in surgery,” Dr. Magge said.

Dr. Magge then discussed medulloblastomas, one of the most common types of tumors neurosurgeons see in kids. Such large tumors grow in the lower back part of the brain — the cerebellum, which is involved in muscle coordination, balance, and movement.

Thirty years ago, Dr. Magge said, kids with medulloblastomas received high doses of radiation that left a lot of them with severe cognitive and hormonal deficits.

The treatment for medulloblastomas had evolved so that less radiation is used in the treatment. In addition, in the last decade, researchers have discovered that these tumors differ significantly based on their genetic makeup.

“These tumors have multiple genetic subtypes, and we can target them genetically with different types of treatments,” Dr. Magge explained.

He said innovation also has led to advances in the treatment of diffuse intrinsic pontine gliomas (DIGP), highly aggressive and difficult-to-treat brain tumors that grow in an area of the brainstem that controls many of the body’s most vital functions such as breathing, blood pressure, and heart rate.

The prognosis for DIPGs remains very poor because they are considered non-resectable tumors – ones that cannot be removed with surgery. Life expectancy is eight to 12 months after diagnosis.

“This is one of the toughest diagnoses we have to give to families because of the lack of good treatment options,” Dr. Magge said.

For years, biopsies were ruled out because they could cause significant side effects, and neurosurgeons saw no point in performing them since there were no treatments. Without biopsies, the tumor tissue could not be studied in a lab for potentially effective treatments. 

Technology has changed this is the last 10 years, Dr. Magge said, thanks to stereotactically guided needles that allow neurosurgeons to perform DIPG biopsies safely.

“We at CHOC and other pediatric hospitals have shown we can do this safely with minimum morbidity,” said Dr. Magge, who has participated in a large clinical trial regarding DIPG biopsies.

“With this technology, we can get tissue and genetically sequence these tumors and find out if there are certain mutations that are particularly amenable to certain treatments,” Dr. Magge said of this precision-medicine approach. 

“These are small steps along the path,” he added. “We have by no means found all the answers. We have so much farther to go, but I think we’re on the right track.” 

Closing the gap

Dr. Muhonen recalled one of the first patients he saw when he came to Orange County in 1995: a young girl with severe spasms in her legs. She couldn’t walk without assistance.

“We had to do something innovative,” Dr. Muhonen said.

He had injected baclofen, a muscle relaxer and antispasmodic agent, into the spinal column of an adult the year before, but never in a child. After receiving approval to do so, he implanted a device that allowed long-term injection of baclofen in the girl’s spinal cord. Six months later, she was able to walk and even run on her own.

In another example of innovation, Dr. Muhonen worked for five years on helping to develop a wireless sensor to measure pressure in the brain. The FDA approved the device for adults, but has yet to for children.

Most companies get medical devices approved for adults because it’s easier, because there’s a larger patient population, and there’s more money to be made. 

“The bulk of challenges associated with developing and accelerating pediatric medical devices is market-driven,” Dr. Muhonen said. “We want children to get the best possible care available, but the relative market size is small compared to adults, which is one reason some device makers avoid it.”

One of Dr. Muhonen’s chief interests is treating hydrocephalus, the buildup of fluid in the ventricles deep within the brain.

Innovation in this area has been a long time coming, he said, since the invention in the early 1950s of a shunt that drained fluid from the brain into the abdominal cavity. Many problems can occur with the shunt, such as spontaneously twisting up into a knot due to a child’s movement or calcifying and breaking apart after being in the body for a long time. Kids who received a shunt typically face more than 10 surgeries, Dr. Muhonen said.

“The holy grail for pediatric neurosurgeons is, can we create a ‘smart shunt?’” Dr. Muhonen said.

An ideal shunt, he said, could be programmed to drain a specific amount of water and measure pressure.

Dr. Muhonen said a derivative from cone snails is inspiring research into a new generation of painkillers for adults, but has yet to be approved for testing on kids.

Impediments to innovation

Dr. Sanger asked Drs. Magge and Muhonen about impediments to pediatric innovation. Ethically, he posited, shouldn’t new devices and other innovations be tested in adults first? 

“I don’t think there are any easy answers to this,” Dr. Magge said. “It’s difficult. You don’t just do a biopsy on a tumor that might help kids in the future. If you perform surgery on a child, there has to be some potential benefit to that child.”

Dr. Muhonen said children are the most vulnerable of society and thus are the worthiest of innovations in healthcare. 

Dr. Magge said he and others at CHOC have been looking at ways to inject dyes to paint brain tumors to more easily distinguish them from healthy brain tissue.

“Sometimes the tumor is obvious, sometimes it’s more challenging,” he said.  While dye injections have been used in adults, it is less commonly used in children.

Dr. Sanger mentioned “big effect sizes” resulting from innovation in pediatric medicine. 

“We’re used to the idea of statistical research involving a lot of patients,” he said. “But this is a different type of research. You take someone who has never walked before and now they’re running. You take someone who is going to die of a brain tumor and now they’re not. These are very big effect sizes.” 

“There are good reasons for the regulations we have,” Dr. Magge said. “That being said, that doesn’t mean we can’t innovate. And there are mechanisms for us to do that, and to do it safely.

“Our first motto is, ‘Do no harm,’” Dr. Magge continued. “I always tell residents to do the right thing and treat each patient as if they were your own child. Doing the right thing means asking the right questions. ‘How can we do this better?’ You can always learn from everything you do. At the end of every procedure, you critique it. You’re constantly learning. That’s what I always encourage.” 

Dr. Sanger closed the session by noting that clinical evidence should ideally be reflective of the spectrum of pediatric patients and the developmental differences that can impact the use and effectiveness of medical devices.

“This is a collaborative effort,” he added. “CHOC is working closely with the FDA’s new System of Hospitals for Innovation in Pediatrics – Medical Devices (SHIP-MD) Program, our academic partners, industry, entrepreneurs and the investor community to close the gaps. Also, we are now practicing medicine in a world immersed with data. Advances in computing and health information technology have given rise to new sources and types of biomedical data. Clinicians know real-world data will continue to emerge as a source of clinical evidence.”

The Presenting Sponsor of the webinar, “From Clinical Insight to Commercialization: Innovations That Can Transform Pediatric Healthcare,”  was Biocom California, which connects life science organizations to each other so they can collaborate and work smarter together. The CHOC Research Institute co-sponsored the hour-plus session.

The webinar was presented in partnership with SBDC @ UCI Beall Applied Innovation, a resource for any high-technology, high-growth, scalable venture from the community or the UCI ecosystem that needs help with business planning, business development and funding-readiness.

Learn more about CHOC’s Neuroscience Institute.