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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.

New app helps build daily routines for kids with autism spectrum disorder

A couple of years ago, Adam Gold, CHOC’s chief technology officer, started thinking about what kind of app could help parents of kids with autism – or kids with other cognitive challenges, for that matter.

Gold has a nephew who has autism spectrum disorder (ASD), and his wife, Lisa, is a special education teacher. 

Now, Gold’s creation, an app he calls uTine (a play off the word “routine”), is on the verge of being tested on patients at the Thompson Autism Center (TAC), with a pilot version set to be ready by July 2021.

“The idea was to develop an app for children with autism and other cognitive challenges that they could use to help them get into routines or complete daily tasks,” says Gold, who will detail the app at CHOC Innovation Day on June 25.

Adam Gold, chief technology officer at CHOC

Casey Clay, PhD, director of the Behavior Program at the TAC, will pilot the app with patients and their parents.  

“Kids on the autism disorder spectrum like predictability of events, and this app will provide them with visual reminders of their next activity or task,” Clay says.

“What’s interesting about this app,” he adds, “is there’s a rich literature base in the field of behavior analysis and a lot of research on the effects of picture activity schedules, or visual activity schedules to help kids follow a schedule or get through an activity.”

CHOC Innovation Day — “A celebration of innovation at CHOC — will be held Friday, June 25, from 1-3 p.m. The online session, held in honor of the late Dr. Nick Anas for his contributions to innovation and medical intelligence, is open to all CHOC physicians and associates.

The virtual event will feature remarks by CHOC CEO Kim Cripe and Dr. Anthony Chang and also will showcase groundbreaking artificial intelligence and virtual reality projects, innovation by nurses, and much more.

On May 20, Gold previewed the uTine app during Pediatric Innovation Start-up Demo Day, an online forum hosted by The Innovation Lab in Newport Beach.

Gold worked with the Innovation Lab, a for-profit organization owned by non-profit healthcare systems, in developing uTine.

“I’m excited about it for a couple of reasons,” says Suzy Engwall, national director at The Innovation Lab. “First, the incidence of autism continues to grow. Kids with autism grow up to be adults with autism, and many who are on the spectrum never learn some of the basic life skills they need to have in order to take care of themselves every day.

“Second, as those with ASD get older, they ‘age out’ of many services, and if they don’t have family members who can take care of them, they will often end up in institutions or other places that they really shouldn’t be in, instead of living on their own because they haven’t learned all of the necessary life skills to do so.

“It’s really important to start as young as we can to teach these life skills in a fun and memorable way. We’ve really got to help those of the autism spectrum become as self-sufficient as possible.”

Suzy is working on the uTine app along with Innovation Lab colleagues Matt Keller, client engagement executive; Ganesh Laxminarayan, executive director, Health IT; Hesham (Sam) Mahdawi, technical project manager; and Durai Ashok, senior solutions architect.

How the app works

The app, which helps kids complete routine tasks such as brushing their teeth, putting on their shoes, and eating lunch, is easy to use with a simple design starring several cartoon animals, including a dog, a cat and a porcupine.

A parent signs up and creates a profile for his or her child, including name, age and personal preferences.

The parent can then start creating “uTines” with step-by-step instructions on how to complete a task. 

Using their own phone or their parent’s phone, the child captures each attempted or completed task by snapping a picture. The parent receives a notification and reviews the picture. The parent then can reward the child for completing a task – such as 15 minutes of TV time, 10 minutes on YouTube, one hour of playing with the dog – whatever the child likes to do.

When they reward their child, a parent can click on a pre-written message to send, such as, “You’re on fire today!”

Gold says he’s working on creating a public library parents can use for very simple uTines so they don’t have to create them themselves.

A key feature of the app is that parents can invite members of the child’s care team into the experience – a doctor, a nurse, a teacher, a psychologist. They, in turn, can gain insights into how the child is doing and chat back on forth securely with the parent.

Gold says he hopes the app gains traction for children with any condition or kids in general who have a hard time establishing routines.

“This app builds on a rich body of evidence-based research, so it’s very likely to succeed,” adds Clay. “This is all about pushing evidence-based research further so we at CHOC can help parents who have kids with autism and other conditions.”

Register here for CHOC Innovation Day.

Learn more about the Thompson Autism Center at CHOC.

As COVID-19 wanes, CHOC’s medical epidemiologist, Dr. Jasjit Singh, recalls 2016 dental clinic outbreak

As the grip of the COVID-19 pandemic continues to weaken, Dr. Jasjit Singh, CHOC’s medical epidemiologist and medical director of infection prevention and control, recalls a ghost of outbreaks past.

Nearly five years ago, a mysterious outbreak of oral infections that eventually was traced to a clinic in Anaheim alarmed parents and dominated the local news.

The health crisis spurred Dr. Singh and a multidisciplinary team at CHOC, working with several community partners, to search for answers – and to provide optimal care for the patients, whose median age was 6.

Over 100 children were admitted to CHOC for evaluation, of whom 70 were confirmed cases, hospitalized for an average of more than a week.

Some suffered permanent tooth loss – as many as six teeth.

The culprit: a Mycobacterial abscessus infection that was detected after each child underwent a pulpotomy procedure, or “baby root canal,” to remove or treat an infected tooth at the Anaheim clinic between Jan. 1 and Sept. 6, 2016.

The outbreak turned out to be the largest ever of invasive Mycobacterial abscessus infections associated with a dental practice. The commonly occurring M. abscessus bacteria is found in water, dust, and soil, but it’s an uncommon cause of healthcare-associated infection.

Between July and September 2016, three patients were admitted to CHOC with atypical infections. All had some combination of facial cellulitis, dental abscess, and/or cervical adenitis that had been present for weeks.

“We had our first child present with what appeared to be a really unusual infection,” Dr. Singh recalls. “Our first thought was, ‘What’s wrong with this child’s immune system?’

“When the second child came in, one of my very astute colleagues, Dr. Negar Ashouri, ascertained that the child had been treated at the same dental clinic. She alerted the OC Health Care Agency immediately, who soon found unexplained symptoms brewing in other kids.” 

A mobilized effort

The California Department of Public Health, the Centers for Disease Control and Prevention, and the Dental Board of California worked with the OC Health Care Agency (OCHCA) to investigate the infections.

The team at CHOC helped OCHCA with the epidemiologic and diagnostic probe. Of 1,081 at-risk patients, 71 case patients (22 confirmed; 49 probable) were identified.

Here at CHOC, 27 of the most severely affected children were treated with a complex regimen of antibiotics, including clofazimine, marking the largest number of children to ever receive that medication outside of treatment for leprosy.

Details of the work of Dr. Singh and many others recently was published by Oxford University Press on behalf of the Infectious Disease Society of America.

Publication of the paper, “Invasive Mycobacterium abscessus Outbreak at a Pediatric Dental Clinic,” was delayed a year and a half by the COVID-19 pandemic, Dr. Singh says.

The infections were caused by untreated municipal water the Anaheim clinic was using for drilling and irrigation during pulpotomy procedures. Because pulpotomies are not considered surgical procedure, sterile water is not required.

A change in state water standards

The work of Dr. Singh and an army of others led to a change in water standards for pediatric dental procedures in California.

In September 2018, the governor signed into law a bill that specified as unprofessional conduct the use of water that is not sterile or that does not contain recognized disinfecting or antibacterial properties when performing dental procedures on exposed dental pulp.

Dr. Singh is hopeful that the publication of the paper, whose listed authors include 11 CHOC physicians and three officials with the OC Health Care Agency, will lead to similar laws being enacted in other states.

As the paper puts it, “The authors believe the measure adopted in California for the use of sterile water for all pulpotomies is an appropriate standard which we would like to see embraced by the American Dental Association and state dental boards around the country.”

Dr. Singh credits the multi-disciplinary team for caring for the patients. The team included specialists in infectious disease, oral surgeons, ENT doctors, radiologists, dentists, pharmacists, and staff members of Providence Speech and Hearing, among others.

“These were normal, healthy children that were affected,” Dr. Singh says. “The multi-disciplinary coordination was a huge part of the success of this story. Still, many of the patients who lost permanent teeth will need dental rehabilitation in the future. It was a very difficult period for these families.

“We talked to national experts and really delved through whatever literature was out there,” Dr. Singh adds. “We all came together to get the kids and families through this with the least morbidity and the best long-term outcome possible.” 

Read about Dr. Jasjit Singh and Dr. Antonio Arrieta earning a global distinction for excellence.

Virtual Pediatric Lecture Series: High Blood Pressure in Children and Adolescents

CHOC’s virtual pediatric lecture series continues with “Pediatric Nephrology: High blood pressure in children and adolescents.”

This online discussion will be held Thursday, June 17 from 12:30 to 1:30 p.m. and is designed for general practitioners, family practitioners and other healthcare providers.

Dr. John D. Nguyen, pediatric nephrologist at CHOC, will discuss several topics, including:

  • Diagnostics and work up for pediatric hypertension
  • Identifying findings and history suggestive of secondary hypertension.
Dr. John D. Nguyen, pediatric nephrologist at CHOC

This virtual lecture is part of a series provided by CHOC that aims to bring the latest, most relevant news to community providers. You can register here.

CHOC is accredited by the California Medical Association (CMA) to provide continuing medical education for physicians and has designated this live activity for a maximum of one AMA PRA Category 1 Credit™. Continuing Medical Education is also acceptable for meeting RN continuing education requirements, as long as the course is Category 1, and has been taken within the appropriate time frames.

Please contact CHOC Business Development at 714-509-4291 or BDINFO@choc.org with any questions.

State-of-the-art CHOC Outpatient Heart Center now open

A new state-of-the-art clinical space on CHOC’s main Orange campus brings together multidisciplinary pediatric cardiology and cardiothoracic surgery outpatient services under one roof.

The 10,000-square-foot CHOC Outpatient Heart Center, which began seeing patients May 24, offers general and subspecialty pediatric cardiology, as well as cardiothoracic surgery outpatient services, multidisciplinary cardiology and cardiothoracic surgery services — including evaluation and management — and cardiodiagnostics to patients with congenital and acquired cardiac disease.

The new outpatient center is devoted to evaluating children as early as possible, engaging children with diagnoses, symptoms and behaviors that diminish their quality of life and life expectancy. The center creates a treatment plan for each patient, coordinating care and establishing a long-term support system for children with complex care needs.

With 23 clinical rooms for echocardiograms, stress testing and consultations, as well as rooms that can be used for exams or echocardiograms, the center establishes a single location for patients’ cardiology needs.

Sky and seascape imagery in a soothing color palette fill the serene environment of the CHOC Outpatient Heart Center. In the waiting area, patients can interact with a digital, touchless coloring display.

The Heart Center also features a physician reading room; a conference room; an exercise stress test room; a flex space; and family consultation/telehealth rooms.

Refer a patient to the CHOC Outpatient Heart Center.

Rapid Whole Genome Sequencing continues to provide answers and hope for parents of critically ill children with rare diseases

If a Major League Baseball player were to step up to the plate 150 times and get a hit 76 times, his batting average would be an unthinkably torrid .507. 

When it comes to identifying genetic causes for some of the rarest and serious diseases in children, CHOC has put up numbers that even Mike Trout couldn’t dream of achieving. 

Since July 2017, CHOC has ordered the comprehensive and cutting-edge test of rapid whole genome sequencing (rWGS) on 150 patients, with 76 of them getting a precise diagnosis that, in many cases, has resulted in life-changing care. 

“We took what could have been a diagnostic odyssey for these patients and families and cut it down from weeks, months, and sometimes years to, in some cases, only three days,” says CHOC pediatric intensive care unit medical director Dr. Jason Knight, part of an informal leadership team that oversees treatment of critically ill kids with rare diseases in the NICUPICU and CVICU. Other ICU physician team leaders include Dr. Adam SchwarzDr. Juliette Hunt and Dr. John Cleary

CHOC’s rWGS research program was championed by the late Dr. Nick Anas, CHOC’s former pediatrician-in-chief who was director of pediatric intensive care and a beloved figure at the hospital. Dr. Anas, who started at CHOC in 1984, died on April 3, 2018. 

Dr. Anas’ vision for the rWGS research program continues to be realized with successful patient outcomes, from the 2019 diagnosis of an infant girl with the extremely rare cardiac condition Timothy Syndrome to, more recently, a baby boy – Oliver Marley – with a genetic disorder that has been detected in only 10 children worldwide. 

“The CHOC team believed in Oliver – they loved him and took care of him and saw worth in him,” says Caroline Marley of her son, who turns 10 months old this May and was cared for by CHOC clinical teams during two stays, once in the NICU and the second time in the PICU. 

“They told me, ‘We want you to take your baby home,’” Caroline says. 

Testing began in 2017 

Each of us has some 22,000 genes in our bodies that dictate things ranging from the color of our hair to whether we are tall or short. Genes also produce the proteins that run everything in our bodies. Although individually rare, there are more than 6,200 single-gene diseases. RWGS is the technology that, with just a teaspoon of our blood, allows us to look at all the genes in our cells.  

At CHOC, rWGS testing became prominent with the launch of Project Baby Bear in fall 2018. CHOC was among five hospitals to participate in that program, led by Rady Children’s Institute for Genomic Medicine (RCIGM) in San Diego. RCIGM has a lab that runs sequencing. 

“To have (the RCIGM) close by and to be a close partner with them has been great,” Dr. Knight says. “We are way ahead of many other pediatric hospitals in this area. It’s a great success story, and something I’m really glad to be a part of.” 

A total of 45 CHOC patients got tested through Project Baby Bear, a $2-million state program for critically ill infants age 1 or younger who were enrolled in Medi-Cal. Of those 45 patients, 55.6 percent – 25 children – were able to have their rare diseases properly diagnosed, says Dr. Neda Zadeh, a CHOC medical geneticist who was involved with setting up CHOC’s rWGS program with Dr. Anas and who has seen most of the 150 kids tested thus far. 

CHOC actually began ordering rWGS testing on patients the year before in a partnership with RCIGM and Illumina, a leading developer and manufacturer of life science tools and integrated systems for large-scale analysis of genetic variation and function. In that 2017 program, 82 CHOC patients were tested with a 47.6 percent positive diagnosis rate, says Ofelia Vargas-Shiraishi, a senior clinical research coordinator in critical care/neonatology research at CHOC. 

CHOC has paid for an additional 23 children to undergo rWGS testing outside of the now-completed Ilumina and Project Baby Bear programs, and continues to have funding on a case-by-case basis, says Dr. Schwarz. 

“In the long run,” Dr. Schwarz says, “we’re saving money by avoiding expensive workups.” 

Adds Dr. Knight: “For a lot of these families, having an answer – even one they might not want to hear – is extremely important.” 

For parents like Caroline Marley, the results have been priceless. 

‘Wouldn’t place money on your son’ 

Oliver was born at 33 weeks after a complicated pregnancy for Caroline, who had a partial placental abruption when she was 14 weeks pregnant. Caroline and her husband, Ted, have another son, Charlie, 4, who is healthy. 

Oliver Marley with his older brother, 4-year-old Charlie

Born weighing 5 pounds and 4 ounces, Oliver had bruises over much of his body and had to be intubated a day after birth when he went into respiratory failure. Doctors detected a small brain bleed and noticed that, at 6 days old, both of his middle fingers were contracted. 

“I’ve never seen this before,” a neurologist at another hospital where Oliver was being treated told the Marleys. 

Oliver also had difficulty swallowing. He could move his arms and legs a bit, but he couldn’t open his eyes. 

Doctors suspected he might have muscular dystrophy. 

After other complications, doctors told the Marleys that Oliver’s outlook looked grim and that he may have to be sent to an acute-care facility. 

“We can’t help him,” one doctor told Caroline. “I don’t believe he will ever come home. If I were going to Vegas, I wouldn’t place money on your son.”  

It got to the point where the Marleys felt Oliver wasn’t getting the best care, so they decided to transfer him to CHOC. A nurse at another hospital whom the Marleys knew recommended CHOC.  

“We will absolutely take him,” a CHOC nurse told the Marleys. 

Oliver transferred to CHOC on Aug. 11, 2020. 

At 8 weeks old, Oliver underwent a tracheotomy and was attached to a ventilator. 

“He literally started thriving,” Caroline recalls. “He started growing because he was not working so hard to breathe. You could just see he was doing better.” 

Still without a diagnosis, Oliver went home on Oct. 19, 2020 with a tracheostomy tube and a ventilator.  

He returned to CHOC after he contracted a viral infection. 

Not convinced Oliver had muscular dystrophy, Dr. Schwarz suggested him as a candidate for rWGS.  

Three days later, in mid-November 2020, the Marleys received an answer: Oliver had two extremely rare genetic changes in his AHCY gene that potentially resulted in S-AdenosylHomocysteine Hydrolase (SAHH) deficiency.  

It is an extremely rare condition with less than 30 patients reported in the world and CHOC’s Dr. Richard Chang, a metabolic disorders specialist and  biochemical geneticist, was consulted to confirm the diagnosis. The disease, which affects brain, muscle and liver development, is associated with high blood levels of methionine and extremely high levels of toxic S-AdenosylHomocysteine (SAH) that interferes with vital cellular growth. 

Oliver was put on a delicate protein-restricted diet to limit the production of SAH without causing protein malnutrition, and his condition immediately improved. Other medications were added subsequently to provide nutrients that deficient due to the toxicity of SAH. He has a condition that is identical to a girl in Pennsylvania who was diagnosed at age 3 and later underwent a liver transplant. That girl is now 9. 

Oliver is scheduled to receive a liver transplant soon, Caroline says. 

Expanding access to rWGS testing 

A lawmaker in San Diego, in partnership with Rady Children’s Hospital and Health Center, is pushing for a new law that would expand access to rWGS testing by qualifying it as a Medi-Cal covered benefit for babies hospitalized in intensive care. 

Assembly Bill 114, The Rare Disease Sequencing for Critically Ill Infants Act, not only would expand availability of such testing to more families, but also would reduce state spending by eliminating many unneeded procedures, treatments and longer hospital stays, State Assemblyman Brian Maienschein wrote in a recent op-ed piece.  

“For critically ill infants hospitalized with unexplained rare diseases,” Maienschein wrote, “the opportunity to benefit from a medical miracle has arrived.” 

Caroline Marley sees that miracle daily with Oliver, who now is up to 20 pounds and moving around more. 

“We at CHOC are slowly building a case for early introduction of rWGS into the clinical management of these difficult cases in high-acuity settings to improve lifelong clinical outcomes and quality of life,” says Brent Dethlefs, executive director of the CHOC Research Institute. 

“There’s growing evidence that early introduction of this technology results in overall cost savings,” Brent adds. “It’s important to get more insurance carriers to cover the cost of this testing over time, which will make rapid whole genome sequencing more available to vulnerable and underserved populations. CHOC always has been an advocate for social justice in health care, which includes greater access to genomic testing.” 

Caroline Marley praises the entire collaborative team at CHOC and the entire CHOC Specialists Metabolic Disorders division, including Dr. Chang, who is in charge of maintaining Oliver’s health until transplant; Erum Naeem, clinical research coordinator, NICU; and Cathy Flores, clinical research nurse coordinator, critical care. 

“It was a team effort involving the critical care, neonatology, metabolic and genetics teams, just to name a few, and a very strong partnership with RCIGM,” says Ofelia Vargas-Shiraishi, a clinical research coordinator at CHOC. 

“We had everyone by our side every step of the way,” Caroline adds. “Child life was amazing, and so is the spiritual care team. If you’re willing to learn, they’re willing to teach you.” 

Dr. Zadeh says the success of CHOC’s rWGS program – with its whopping .507 batting average – is a result of “a very unique blend of the right people coming together at the right time and the right institution with the right set-up.” 

She adds, “I don’t think it would have worked necessarily at every hospital. I think CHOC is unique. We have the right group of kids we are testing. And we have the right group of specialists involved. 

“We love our families. We get to have really great relationships with them. This program just shows that CHOC is all about the whole care of the child and the family.”