CHOC’s virtual pediatric lecture series continues with a lesson on cardiology.
This online discussion will be held Wednesday, July 28 from 12:30 to 1:30 p.m. and is designed for general practitioners, family practitioners and other healthcare providers.
Dr. Sanjay Sinha, pediatric cardiologist at CHOC, will discuss several topics, including:
Accurately diagnosing and refering patients with cardiac symptoms related to the current coronavirus pandemic.
Identifying new therapy and guidelines as they pertain to your patient populations.
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.
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.”
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.
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.”
In the yard of his home just outside Boise, Idaho, Ely Bowman loves to toss balls and play with Bobo, the family Goldendoodle. He also loves the trampoline.
“If you were to come over and just watch him,” says his mother, Bekah, “you would not believe me if I told you he was blind.”
Ely, who turns 8 in July, lost his sight when he was 6 due to the rare neurological disorder CLN2 disease, one of the most common forms of a group of inherited disorders known as Batten disease.
Kids with CLN2 disease are missing an enzyme that chews up waste products in the brain. This lack of a cellular “Pac Man” to gobble up the bad stuff eventually leads to the destruction of neurons, resulting in blindness, loss of ability to speak or move, dementia, and death – usually by the teens.
There is no cure for CLN2 disease. But thanks to genetic scientists, neurosurgeons and nurses at CHOC, there is hope for delaying progression of the disease – one that claimed the life of Ely’s older brother, Titus, at age 6 in September 2016 before a cutting-edge therapy became available at CHOC six months later.
The therapy, Brineura, is a medication that treats the brain via a port under the scalp with a synthetic form of the missing enzyme. CLN2 patients come to CHOC every two weeks for the four-hour infusion to keep the drug working effectively.
Largest infusion center in country
CHOC since has grown into the largest Brineura infusion center in the country and the second largest in the world. Kids from all over the United States have come to CHOC for Brineura treatment since it first was offered in March 2017 following a three-year effort by Dr. Raymond Wang to get the green light for CHOC to become the second infusion site in the U.S.
“When a family has a child with a rare disease,” Dr. Wang says, “and if the South Pole were the only place that was offering treatment, the family would find a way to get there. Those are the lengths that a rare disease family would go to help their child.”
CHOC now has treated 13 Brineura patients, the latest being 3-year-old Max Burnham, whose parents having been making the trek to Orange every two weeks from their home in the Bay Area since Max’s first infusion on Feb. 8, 2021.
CHOC’s Brineura program underscores its growing reputation as a destination for kids with rare diseases.
Recently, CHOC specialists started treating a 3-month-old with Hurler syndrome, another serious and neurodegenerative condition. The family drove across the country because CHOC is the only site in the world that has a clinical trial of gene therapy for their son’s condition.
Because the family will be staying at CHOC for at least through April 2021, a team of three study coordinators — Nina Movsesyan, Harriet Chang, and Ingrid Channa – helped the family get settled in at an Airbnb in Irvine.
Dr. Wang says CHOC became an active site for the RGX-111 gene therapy after treating a child from a family in Indio in 2019. Another 14-year-old girl from West Virginia has received the same treatment.
“All of these cases wouldn’t be possible without the awesome teamwork from team members, who all are dedicated to the mission of CHOC,” says Dr. Wang. “I think it’s pretty remarkable that people from all over the country are coming here for clinical care and research studies because of our expertise and what we offer them: hope for their beloved children.”
Susan See is nurse manager of CHOC Hospital’s neuroscience unit, where the patients receive their infusion and stay for care afterward.
“We quickly put together a comprehensive program that really treats the patient and family not just medically, but also from an emotional support standpoint,” she says.
Batten disease especially is terribly cruel because its symptoms typically hit just as parents are starting to enjoy their child reaching several developmental and cognitive milestones such as walking and talking.
Untreated, the disease eventually takes all that away.
“What makes them who they are gets rapidly erased,” says Dr. Wang. “As a practitioner, it’s hard. I’m trying to imagine being in the shoes of a parent knowing this is going to happen to their child.”
For Bekah Bowman and her husband, Daniel, the diagnosis for Titus and, two months later, Ely, was like being on a high diving board and being shoved off and belly flopping into the water.
“We had to learn what little control we have in life,” Bekah says.
The Bowmans worked closely with Dr. Wang to get the Brineura clinical trial launched at CHOC.
“When we met Dr. Wang,” Bekah says, “he told us: ‘We don’t have the answers for you right now, but I want you to know we’re going to keep fighting and we’re not going to give up.’”
Brineura families form tight bonds with their team at CHOC, which includes eight nurses who have been trained to care for them: Allison Cubacub, Genevieve Romano-Valera, Anh Nguyen, Melissa Rodriguez, Kendall Galbraith, Annsue Truong, Monica Hernandez and Trisha Stockton.
Some families, including the Bowmans, have moved on from the program at CHOC when Brineura infusions became available near their hometowns. The Bowmans returned to their native Idaho outside Boise in October 2018. Leaving CHOC was difficult.
“That was one of the hardest goodbyes we had to say,” Bekah says.
All Brineura patients receive the transfusions on the same day – something unique to CHOC, See says.
“We learn what is unique about each patient and we become very close to them,” she adds. “It really reminds us why we said yes to nursing. What we thrive on is being able to care for families.”
Quick to action
Laura Millener, the mother of Max, CHOC’s latest Brineura patient, says she selected CHOC for Max’s condition, diagnosed in January 2021, because he needed to be treated right away. She first spoke to Dr. Wang on Jan. 11, and Max got his first infusion less than a month later.
“You could just tell how much he cares about his patients,” Laura says of Dr. Wang.
Says Dr. Wang, who has three children ages 10 to 18: “I count [my patients and my families] as my extended family, and I want the best for all of them.”
Laura and her husband, Matthew, a C-5 pilot in the U.S. Air Force, will be relocating to Quantico Marine Base in Virginia this summer from Pleasantville, Calif. Max, who has a 6-year-old sister, Ella, will continue his Brineura infusions at Children’s National Hospital in Washington, D.C.
“I don’t want to leave CHOC,” Laura says. “CHOC has done such an amazing job of making this easier on us. I am so grateful for the team.”
Dr. Wang says the Brineura infusions have made it possible for the patients to maintain meaningful interactions with their parents and siblings – despite having such conditions as, in Ely’s case, blindness.
Ultimately, the goal is for CHOC to be considered for a gene therapy clinical trial aimed at giving brain cells the ability to produce the missing enzyme by itself so Batten disease patients wouldn’t have to receive infusions every two weeks. Dr. Wang says such a trial could happen this fall.
“If there’s anything in my power I can do to help these families,” says Dr. Wang, “I’m going to try to make it happen.”
While there is less data about pediatric patients, emerging evidence shows that people infected with COVID-19 are at increased risk for myocarditis. Because of this, it is important that pediatricians appropriately evaluate patients before they are cleared to return to play as sports resume after a prolonged COVID-prompted off season.
Patients should be seen in the provider’s office for an in-person, formal evaluation and physical exam to determine clearance, recommend Drs. Kornswiet and Koutures. The following decision tree can aid in triaging patients, as well as providing consistent patient care. This decision tree is applicable to middle and high school athletes, as well as to those who compete in high-exertion activities and to other patients on an individual basis.
The California Interscholastic Federation recommends that if a patient’s infection was over three months ago, they had an asymptomatic, mild or moderate illness, and the patient has regained fitness or is back to full activity without symptoms, then they can return to sports as long as they have an active/recent preparticipation physical exam.
Once an athlete is cleared for a return to sports, Drs. Kornswiet and Koutures recommended that they go through a gradual and step-wise return to play. This is similar to the return-to-play protocol for concussions, and should be performed under the supervision of a physician and athletic trainer, if possible.
Each phase should last at least 24 to 48 hours and should not cause return of symptoms. If the athlete/student experiences a return of symptoms or develops unexpected fatigue, dizziness, difficulty breathing, chest pain/pressure, decreased exercise tolerance, or fainting, then they should stop their return progression and return to their physician for further evaluation.
These protocols are not substitutes for medical judgment, and additional queries should be directed to pediatric cardiologists or sports medicine specialists.
Following are more general return-to-sports guidance for parents and coaches: