Inside Pediatrics Spring/Summer 2024

PEDIATRICS

Spring/Summer 2024

PLUS: The phone call that helped a family heal Treatment Hashizume brings a focus on intranasal delivery to Children’s The Future of DIPG

I n the world of medicine, research and of Alabama, we’re proud to have medical professionals who are committed to finding the most effective ways to treat our patients. In this issue of Inside Pediatrics , you’ll get a glimpse of our leading-edge ideas. In some cases, innovating means hiring the best people in their fields—for example, Rintaro Hashizume, M.D., Ph.D., who joined our Division of Pediatric Hematology, Oncology, and Blood and Marrow Transplantation in 2023. His research on a rare, deadly brain tumor has led him to a new approach that could save lives. For the patient, it may one day be as easy as using a nasal spray. We also push the envelope when it comes to more common conditions, such as type 1 diabetes. Endocrinologist Gail Mick, M.D., is leading a study that shows how an over-the counter supplement could help treat new-onset cases of type 1 diabetes. In nephrology, our researchers are proving that certain patients previously thought to have little chance at life have hope, after all. In a study recently published in Pediatrics , they show how ECMO support and kidney support therapy provide hope for neonates with congenital kidney disease and severe respiratory failure. innovation are two of the keys to improving patient outcomes. Here at Children’s

Tom Shufflebarger, President and CEO

our first deep brain stimulation procedures for patients with progressive dystonia. This was made possible because of the expertise of neurologist Emily Gantz, M.D., who, according to one of her colleagues, “opened the door for us to be able to perform the technical aspects of the surgical procedure.” At Children’s, our patients experience our commitment to excellence all the time. In this issue, you’ll find the story of Liam Blakely, who credits the Children’s Psychiatric Intake Response Center, or PIRC, with saving his life. Since opening in 2018—when it was the first program of its kind in the Southeast—the PIRC has connected tens of thousands of parents with the right mental health resources for their children. Patients like Liam already know about the great things we do at Children’s. In this issue, you can learn about a few yourself.

New surgical procedures also have the power to transform lives. In 2023, Children’s neurosurgeons and neurologists performed

INSIDE THIS ISSUE

ADMINISTRATION Tom Shufflebarger, President and CEO Garland Stansell, Chief Communication Officer EDITORIAL Conan Gasque, Editor Amy Dabbs Leslie Edmondson DESIGN Scott LeBlanc Dana Stuckey PHOTOGRAPHY Patrick Deavours Eric Gray Lance Holloway Denise McGill CONTRIBUTORS Corinn Cross, M.D. Debra L. Gordon, MS Sarah Handzel, BSN, RN Rhonda Lee Lother Heather Watts GLC This is Alabama DIGITAL CONTENT Andre Green MEDICAL LEADERSHIP Mitchell Cohen, M.D. Katharine Reynolds Ireland Chair of Pediatrics, University of Alabama at Birmingham Physician-in-Chief, Children’s of Alabama Mike Chen, M.D. Joseph M. Farley Chair in Pediatric Surgery, University of Alabama at Birmingham Chief of Pediatric Surgery and Surgeon-in-Chief, Children’s of Alabama James Cullinan, D.O. Associate Professor Director, Child & Adolescent Psychiatry Chief of Service, Child & Adolescent Psychiatry, Children’s of Alabama Trenton Graves Becca Hodges

Division Rounds Quick briefs and news from around the hospital 2

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Taking Aim at DIPG New researcher studying intranasal delivery for DIPG treatment

Exploring GABA and GAD for Type 1 Diabetes Study shows promising results for treating new-onset cases

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The Journey to Better Mental Health: Liam’s story

New Hope for Neonates with Severe Congenital Kidney Disease

Deep Brain Stimulation for Progressive Dystonia A groundbreaking procedure now offered at Children’s

News, Honors and Awards

For questions or additional information or to share feedback, please contact us at insidepediatrics@childrensal.org. An online version of the magazine is available at childrensal.org/insidepediatrics.

DIVISION ROUNDS

For a closer look at these stories and more, visit InsidePeds.org

CARDIOLOGY

Heart team discovers new lung injury biomarker

Nearly all children who undergo cardiac surgery suffer lung injury afterward, and its treatment can determine whether the injury is short-lived or will follow a child for a lifetime. That’s why the discovery at Children’s of Alabama of a new biomarker to predict which patients are at higher risk of this complication—steering the use of respiratory support—is being heralded as a groundbreaking development that may reap benefits for children far and wide. Using samples from a Children’s biorepository, heart specialists revealed that blood levels of a protein called proteoglycan 4, commonly known as lubricin, significantly differ between children undergoing long- and short-term mechanical ventilation after cardiac surgery. Until now, physicians haven’t been able to predict which patients stood at higher risk of lung injury in this care setting. It’s the first time the finding has been reported in the United States, said Ahmed Asfari, M.D. , a cardiac intensivist in the Pediatric & Congenital Heart Center of Alabama at Children’s. Study suggests link between infant mortality and insurance type A new study published in JAMA Network Open suggests that the type of health insurance pregnant people have may impact infant mortality rates. The research, led by Children’s of Alabama neonatologist Colm P. Travers, M.D. , found that pregnant people with private health insurance had lower rates of infant deaths compared to those insured by Medicaid. The study analyzed data on more than 13 million births that occurred in the U.S. from 2017 to 2020. Overall, 46% of the pregnant people in the study had Medicaid insurance, while 54% had private insurance. The infant mortality rate—defined as the number of deaths in the first year of life per 1,000 live births—was 2.75 deaths per 1,000 live births for those with private insurance compared to 5.30 deaths per 1,000 live births for those covered by Medicaid. NEONATOLOGY

“There’s been nothing like this in regard to acute lung injury, especially for our patient population with congenital heart surgery,” said Asfari, who’s also an assistant professor in the Department of Pediatrics and Division of Cardiology at the University of Alabama at Birmingham (UAB). “When we tested the blood of patients with very long mechanical ventilation duration and compared them to patients with short duration, we found the level of this marker goes down, especially within two days of surgery.” Asfari said the breakthrough likely will change the landscape for children’s care in the future. “Having the ability to have a serum biomarker that we can use to grade the level of acute lung injury will be extremely helpful,” he said.

Those with private insurance also had a 43% lower risk of postneonatal mortality (from 28 days to one year after birth), a 10% lower risk of a low-birthweight infant, a 20% lower risk of vaginal breech delivery, and an 8% lower risk of preterm birth. They were 24% more likely to have received first-trimester prenatal care than those with Medicaid.

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GASTROENTEROLOGY

ORTHOPEDICS

Grant paves way for IBD research

New XR technology improving patient care Children’s of Alabama has new XR technology in its sports medicine clinic, and orthopedic specialist Kevin Williams, M.D. , says it’s a game-changer, enabling specialists to quickly determine the best treatments to get young patients back to their chosen game. The new equipment, which is useful in about 75% of pediatric cases, includes an easily positioned moveable arm with a 135-degree range. The equipment has larger plates that can capture wider images of patients’ bones and soft tissue while “taking into account features of the joints above and below, allowing more comprehensive images while using less radiation,” Williams said. “There’s also a dynamic mode that allows us to take live, fluoroscopic images or continuous X-ray to evaluate a bone or joint dynamically,” he explained. “This is crucial when we’re trying to inject a joint or aspirate it to get fluid out. The whole system is smaller and more modern, which improves ease of use dramatically.”

In an effort to confront the challenges faced by patients with inflammatory bowel disease (IBD), the Crohn’s and Colitis Foundation recently received a transformative grant from the Centers for Disease Control and Prevention (CDC). This comprehensive five-year project marks a pivotal collaboration with Children’s of Alabama and the University of Alabama at Birmingham (UAB), targeting the identification and resolution of barriers hindering the diagnosis and

care of individuals affected by IBD. Traci Jester, M.D. , associate professor in the Division of Pediatric Gastroenterology, Hepatology and Nutrition at UAB, is a project

co-investigator. The project holds promise in transforming the approach to IBD management. The study aims to comprehensively address barriers to timely diagnosis and care through data collection initiatives. As the study unfolds over its five-year span, the final

phase will focus on developing and testing strategic interventions that aim to bridge the gaps in disease awareness among the public and specific demographics while also focusing on educating primary care providers to ensure timely referrals to specialists.

UROLOGY

Using games to help with surgical robot training

The system’s longer plates also help Williams and his colleagues evaluate leg alignment in patients who seek care for rotational injuries, congenital knock knees or bowed legs. This assessment can dictate if a patient needs bracing, physical therapy or even surgery in which bones may need to be broken and put back into place to align them better for the future and prevent degenerative problems.

When the Children’s of Alabama urology department acquired its first Da Vinci surgical robot in January 2023, Carmen Tong, D.O. , pediatric urologist at Children’s of Alabama, developed a training curriculum that brings in classic children’s games to help residents develop their skills with the new technology. “There’s a movement in urology to ‘gamify’ the robot,” Tong said. “Such training is vital to the safety of our patients.” Surgeons are very competitive, she said. “Gamification taps into our competitive side and allows us to push and encourage each other to improve. It helps with camaraderie.” Indeed, studies show that gamification, whether with actual games like Tong is using or embedding competitions and rewards into skill development, enhances resident engagement. The residents complete online modules and practice surgeries via a

PULMONOLOGY

Children’s adds flexible bronchoscopic cryotherapy

In 2023, the Children’s pulmonology team began using flexible bronchoscopic cryotherapy, an interventional pulmonology procedure that, until recently, was offered only to adult patients. It involves performing a bronchoscopy and then using a special probe to freeze a foreign body, piece of tissue or blood clot and pull it out for examination. “Previously, it could take hours because we often wouldn’t get the whole thing at once,” pediatric pulmonologist Ryne Simpson, M.D. , said. “Now we’re able to complete these procedures in significantly less time, which has been pretty amazing for the patients.” It’s one example of interventional pulmonology becoming more prevalent in pediatrics.

video game-type simulator. Then, every two months, Tong brings in the actual games,

and the residents compete against each other using the robotic arm in place of their own hands. Even though the gaming sessions

aren’t mandatory, “almost all residents participate.” That, she said, “says a lot about the program’s success.”

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Inside Pediatrics | Children’s of Alabama

HEMATOLOGY & ONCOLOGY

TAKING AIM AT DIPG

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Rintaro Hashizume, M.D., Ph. D.

NEW RESEARCHER STUDYING INTRANASAL DELIVERY FOR DIPG TREATMENT

I n 2023, the University of Alabama at a scientist specializing in pediatric neuro oncology. Hashizume, an associate professor of hematology and oncology, brings a commitment to pediatric brain tumor research, focusing specifically on the daunting challenge presented by diffuse intrinsic pontine glioma (DIPG). Birmingham (UAB) Department of Pediatrics welcomed Rintaro Hashizume, M.D., Ph.D., DIPG is an aggressive brain tumor that primarily affects children and has a grim prognosis, with most patients succumbing to the disease within 12 months. What makes DIPG particularly challenging is its location in a vital area of the brain that controls breathing and heartbeat, rendering surgery impractical.

blood-brain barrier and the lack of genomic alterations in pediatric brain tumors. However, in 2012, genome-wide sequencing identified a specific mutation in DIPG, providing a crucial target. Hashizume’s lab has been developing therapies that specifically target this mutation, particularly through epigenetic targeting therapy. One groundbreaking aspect of Hashizume’s research is intranasal delivery for drug administration. This method bypasses the blood-brain barrier, offering a non-invasive and convenient alternative method of drug delivery to the brain tumor. Hashizume’s team is exploring liposome-encapsulated drugs delivered intranasally, aiming to enhance the effectiveness of radiation therapy for the treatment of DIPG.

Traditional chemotherapy faces significant hurdles in treating DIPG, primarily due to the

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Inside Pediatrics | Children’s of Alabama

Intranasal delivery enables the liposome-encapsulated drug to enter the brain through specific neural pathways that bypass the blood-brain barrier. The liposome carrier protects the therapeutic drug as it passes through nasal membranes; when it gets into the brain, the liposome opens to disperse the drug. “Using

this method, the drug can be delivered quickly to target areas,” Hashizume said. This research is generously supported by a grant from the Prayers From Maria Children’s Glioma Cancer Foundation and by Hyundai Hope on Wheels.

“Using this method, the drug can be delivered quickly to target areas.” RINTARO HASHIZUME, M.D., PH.D.

While emphasizing the importance of mechanistic research, Hashizume acknowledges the urgency of translating discoveries into clinical applications. He envisions intranasal delivery becoming a feasible option for clinical trials, stressing the need for a swift transition from preclinical studies to clinical trials, especially considering the critical nature of pediatric brain tumors. “I think we’ll need a combination treatment approach using radiation, since that’s a current standard therapy in DIPG that can help provide transient relief from the disease,” Hashizume said. “Clinical trials are needed to discover how we can enhance the radiation’s effect in combination with the intranasally delivered drug.”

Hashizume’s dedication to pediatric brain tumors spans more than 15 years. Originally a physician-scientist in Japan, he transitioned to the University of California San Francisco (UCSF) with a focus on adult brain tumors. However, a pivotal moment led him to shift his research focus exclusively to pediatric neuro-oncology. The catalyst for this change was his long-standing connection with Girish Dhall, M.D., director of the Division of Pediatric Hematology, Oncology, and Blood and Marrow Transplantation at Children’s of Alabama, who played a crucial role in recruiting him to the hospital. With the support of organizations such as the Pediatric Brain Tumor Consortium, Hashizume aims to accelerate the translation of research into clinical practice. As research progresses, the potential for new, effective treatments for these devastating tumors becomes increasingly tangible. ●

Any opinions, findings, and conclusions or recommendations expressed in this material are those of the author(s) and do not necessarily reflect the views of the Prayers From Maria Foundation.

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PEDSCAST Podcast

The Children’s of Alabama PedsCast is a pediatric sub-specialist peer-to-peer podcast focused on research, innovative programs and advances in pediatric health care at Children’s of Alabama. Featured speakers include physicians, surgeons, clinical specialists and other experts in the field of pediatrics.

Multicenter Anticoagulation Study Jonathan Byrnes, M.D.

Quality Improvement Initiative to Improve Kidney Stone Surgical Aftercare Carmen Tong, D.O.

CDC/Crohns and Colitis Foundation Grant Focusing on Disparities in IBD Traci Jester, M.D.

Elbow Fracture Studies Kevin A. Williams, M.D.

To tune in, visit ChildrensAL.org/PedsCast or subscribe through your favorite podcast app.

ENDOCRINOLOGY

GABA + GAD COMBINATION SHOWS PROMISE FOR TREATING NEW-ONSET TYPE 1 DIABETES C ould an over-the-counter supplement help save insulin production in new-onset type 1 diabetes? That’s the question pediatric

“It took a lot of luck, hope and a dream we were going to cure diabetes with something safe and oral” to get FDA approval for the trial, she said. The study randomized 97 children (average age of 11) within five weeks of their diagnosis to oral GABA with or without a GAD-alum injection. However, the researchers were constrained to low doses of the compounds, given the FDA’s concern about potential side effects. Although the study didn’t meet its primary goal of preserving beta cell insulin production, the GABA/GAD combination significantly reduced glucagon levels, improving blood sugar levels. The study also found lower levels of the inflammatory cytokine expression implicated in the pathogenesis of type 1 diabetes. There were no adverse effects.

endocrinologists Gail J. Mick, M.D., Kenneth McCormick, M.D. (now retired) and colleagues at Children’s of Alabama set out to explore. The answer, according to their recently published article in the journal Nature Communications , is, quite possibly. The study explored the potential of the amino acid gamma-aminobutyric acid (GABA), found in health food stores, and glutamic acid decarboxylase (GAD), an enzyme that acts on glutamate to form GABA, to preserve pancreatic islet function. GABA is an important neurotransmitter in the brain. However, it is also critical to insulin production, with GABA receptors found within the islet beta cells responsible for insulin production. Meanwhile, GAD converts abundant circulating glutamate from dietary protein and intestinal microbiota into GABA. Animal studies showed their potential to stimulate insulin secretion, inhibit glucagon overproduction, dampen inflammation and promote beta cell regeneration. Still, getting a clinical trial approved in a pediatric population took years. “Nobody does first-line studies in children,” Mick said. “For safety reasons, studies are typically conducted in adults, but in type 1 diabetes, adults differ from children. In children, the autoimmune destruction of insulin secretion is faster.

It’s fascinating, just fascinating. GAIL J. MICK, M.D. “ ”

“Not only did we see reduced glucagon, but there were also beneficial immunologic effects,” Mick said. “We’re delighted by that.” The immunology results were recently published in the journal Biomedicines .

The positive outcomes show enough promise that further studies with higher GABA doses are warranted, Mick said, perhaps in combination with other agents, such as GLP-1 receptor agonists, which also have beta-cell regeneration effects. ●

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BEHAVIORAL HEALTH

LIAM’S JOURNEY HOW A CALL TO THE PSYCHIATRIC INTAKE RESPONSE CENTER CHANGED A BOY’S LIFE

S tefanie Blakely was at a work event one night in October 2022, when she received a text message that would leave almost any parent in shock. It was from her 12-year-old son, Liam. He was considering suicide.

wondering how to help Liam, Stefanie remembered it. She pulled into a parking lot, looked up the number and called. The PIRC is a phone response center for adults seeking mental health resources for their children or adolescents. Callers can have free, confidential conversations with licensed mental health clinicians who assess a child or teen’s mental, emotional and behavioral needs and recommend the best treatment options, including the contact information of multiple mental health professionals in their community. “For our PIRC therapists, it’s been a very rewarding experience for them to be able to talk to folks who need that help and who are looking to do the right thing for their child. They are getting an opportunity to be there in that moment,” PIRC director Cindy Jones said. “And answering callers’ questions, we do notice a lot of times that if we are there for them, that it decreases the anxiety and the stress level and the frustration that a lot of parents or caregivers or whomever have while trying to find those right resources.”

“I just felt like the world stopped completely,” Stefanie said.

Scared and nervous, Stefanie felt she needed to get to Liam immediately. But he was on the other side of town, and she worried she might not be able to get to him quickly enough to keep him safe. She left the event and drove to pick him up, calling and texting with him along the way. When she arrived, Liam shared the emotions that led to his text. He was still upset. “I just was really lost as a parent trying to figure out what the best resource was for my son at that point,” Stefanie said. Stefanie works at Children’s of Alabama and was familiar with the hospital’s Psychiatric Intake Response Center, or PIRC. Liam’s therapist also had mentioned it. While driving down the road

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“

I think that may have saved my life that night.

LIAM BLAKELY

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Inside Pediatrics | Children’s of Alabama

The Psychiatric Intake Response Center (PIRC) at Children’s of Alabama

When Stefanie called, she “got the most reassuring voice on the other end of the line,” she said. Ashley Loftis, the therapist she spoke with, advised her to take Liam to the Children’s Emergency Department. “I think that may have saved my life that night,” Liam said. The call led to a positive turn in what had been a long process for Liam. Stefanie says he started struggling with depression around 3rd grade and was diagnosed that year with attention deficit hyperactivity disorder (ADHD). School closures resulting from the COVID-19 pandemic led to more challenges. “The isolation of being stuck at home was very difficult for him,” Stefanie said. Even when schools reopened, Liam was still struggling and met with school counselors. His pediatrician recommended increasing his ADHD medications, but by early 2022, it was obvious to Stefanie that

The PIRC began as a shared vision between Children’s of Alabama and generous partners in the community. It opened in March 2018 as a free, confidential phone response center linking adult callers to child and adolescent mental health providers. It was the first program of its kind in the Southeast and one of only three nationally at the time.

Since opening, it has served almost 14,000 callers along with 22,000 emergency

more resources were needed. He started seeing a licensed therapist,

department patients. PIRC leaders have also identified more than 1,700 metal health providers and services in Alabama and added them to a database created by PIRC team members. “As the youth mental health crisis has worsened, the PIRC has been positioned and able to educate, support, guide and provide direct mental

PIRC therapist Ashley Loftis (top) with Stefanie and Liam Blakely

and in the fall of that year, his pediatrician put him on a low dose of Zoloft. But he was still dealing with depression and having trouble sleeping. Then came the night of the text message. Initially, it seemed like a low point. “I felt that feeling when

health care statewide,” PIRC director Cindy Jones, MA, LPC-S, NCC, said. “The PIRC has prevented unnecessary visits to the emergency department and provided callers better understanding and access to the right mental health provider and/or treatment.”

you can feel the color just draining from your face into your body,” Stefanie said. Ultimately, however, it became a turning point, leading to changes in Liam’s mental health services. “They gave us the guidance that we needed,” he said. “And I think that may be what started my journey to better mental health.”

For more information about the PIRC, go to ChildrensAL.org/PIRC

One of the next steps in that journey was the Children’s Partial Hospitalization Program, or PHP—

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a day treatment program that helps patients who, at the time, are not candidates for an inpatient stay but need more intense treatment than the typical outpatient structure provides. There, Liam worked with a therapist who was able to identify his depression, anxiety, ADHD and obsessive compulsive disorder (OCD) as symptoms of a larger issue. She suggested having him evaluated for autism spectrum disorder (ASD), with which he was diagnosed a few months later. “[The ASD diagnosis] has been huge for us in navigating his mental health as well as his school experience,” Stefanie said. Now, Liam visits the Children’s outpatient clinic every six months, and his doctor has a full picture of who he really is.

Watch this video to learn more about the PIRC at Children’s of Alabama.

But the process of understanding Liam and getting him the help he needs began with the PIRC.

Stefanie says she’s incredibly thankful for it and for the calming therapist on the other end of the line. “To have someone be the definitive voice of reason to say, ‘This is what you need to do,’ in a moment when you feel just completely lost—not knowing what’s going on or what your next steps are supposed to be—is so nice,” she said. “And I was so grateful in that moment.” ●

Please visit us at booth #422

at the annual PAS Conference May 2-6, 2024 Toronto, Canada

For more information, contact Heather Watts at hwatts@uabmc.edu

NEW HOPE FOR NEONATES WITH CONGENITAL KIDNEY DISEASE

T here was a time when conventional wisdom held that babies with congenital kidney disease (CKD) and severe respiratory failure couldn’t survive. Thanks to a study led by Children’s of Alabama, that’s changing. Four children are thriving today due to the efforts of the Children’s nephrology team, who, almost a decade ago, began the journey toward improving outcomes in neonates with CKD by adapting the Aquadex machine, a small extracorporeal circuit used for adults with heart failure. Now, in a recently published study, the team shows its latest findings, which reveal that there is hope for these neonates.

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NEPHROLOGY + NEONATOLOGY

The nephrology team at Children’s used the Aquadex FlexFlow System to provide continuous venovenous hemofiltration for patients in the study.

valves, bilateral renal dysplasia and autosomal recessive polycystic kidney disease. Despite gestational age ranging from 35.6 to 37.1 weeks and birth weight from 2,740 grams to 3,140 grams, all five patients received KST by postnatal day seven. Additionally, they were all placed on ECMO “What we learned in this very small cohort is that these lungs can develop and grow if given a chance.” KARA SHORT, MSN, CRNP, CPNP-PC

within the first nine postnatal days due to severe respiratory distress after being unresponsive to conventional interventions.

Traditionally, extracorporeal membrane oxygenation (ECMO) was deemed unsuitable for this population due to perceived nonviability. In 2016, at the family’s request, a baby with severe congenital kidney failure and severe respiratory failure was placed on ECMO to be given a chance at life. The baby also required kidney support therapy (KST) to survive. After receiving a kidney transplant at age 2, the child now goes to school, plays sports, sings and dances. Since 2016, of the 31 neonates with congenital kidney failure who were admitted to the Children’s neonatal intensive care unit (NICU), five required ECMO support and KST in the first two weeks of life. In February 2024, Kara Short, MSN, CRNP, CPNP-PC, David Askenazi, M.D., MSPH, and others published a case report in Pediatrics , highlighting the complex treatment of the five babies and the journey to NICU discharge for the four survivors. This study challenges the previous norms and conventions that these babies had no chance at life. Congenital kidney failure poses unique challenges to neonates, affecting not only renal homeostasis but also respiratory integrity. Diagnoses among the five patients in the study included posterior urethral

An ECMO machine in use on a patient at Children’s of Alabama. Traditionally, ECMO has not been used on neonates with congenital kidney disease, but that’s changing thanks to a study done at Children’s of Alabama.

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Inside Pediatrics | Children’s of Alabama

and potential extended NICU stay, all five families opted for full medical support. Clear communication, counseling and informed decision making were instrumental when families made medical decisions about their baby’s care. The successful outcomes of neonates with congenital kidney failure undergoing ECMO challenge previous assumptions of nonviability. Meticulous ECMO, respiratory, nutritional and kidney support therapies are essential to favorable long-term results. Further investigation is needed to define the optimal strategies to improve outcomes

Four of the five patients survived and are thriving today. Pulmonary hypertension resolved in each survivor, with three requiring no oxygen support and one needing only nocturnal oxygen. Three survivors underwent successful kidney transplants, while one awaits transplant evaluation. This challenges the traditional notion of reflexively assigning nonviability to neonates with congenital kidney failure and severe pulmonary complications. This research highlights the significance of ECMO and kidney support therapy in mitigating the adverse effects of pulmonary edema, uremia and electrolyte complications. The use of a filter through the ECMO circuit—to perform continuous venovenous hemodialysis (CVVHD), continuous venovenous hemofiltration (CVVH) with the Aquadex machine, peritoneal dialysis and intermittent hemodialysis— showcases the synergism and need for different approaches to manage these complex cases. Managing congenital kidney failure requires a multidisciplinary approach involving neonatology, nephrology, surgery and multiple ancillary divisions. “As healthcare providers, we all bring something to

in severe congenital kidney disease cases. “We want to share this information with other programs to let them know that these kids have a chance at life; what we learned in this very small cohort is that these lungs can develop

Kara Short, MSN, CRNP, CPNP-PC

and grow if given a chance,” Short said.

the table,” Askenazi said. “The families need clear, concise information so they can understand their options when making treatment decisions. Our job is to develop programs, systems and plans to help those kids have the best chance at life.”

“When thinking about the future, we’re asking ourselves: How can we get the very best technology to care for these babies? How can we help other programs improve so they can better care for their kids? How do we ensure that families are counseled with all viable options in an honest and comprehensive way?” Askenazi said. “We will continue to make progress. We have recently received a grant from the National Institutes of Health (NIH) to partner with industry to make a better device for neonates. We recognize that we must continue to educate our colleagues across the U.S. and the world about what we have learned from these miracle babies.” ●

David Askenazi, M.D., MSPH

The families of the neonates faced challenging decisions, including the choice of full medical support or palliative care. Despite the complexity

Register now for the 2024 International Neonatal Nephrology Symposium September 11-15, 2024 | Birmingham, Alabama

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THE SECOND PATIENT IN THE COHORT STUDIED BY KARA SHORT AND DR. DAVID ASKENAZI WAS CHARLOTTE “CHARLIE BELLE” JACKS.

Born with failing kidneys, she spent the first seven months of her life in the hospital. Even after she was discharged, she required dialysis to stay alive. In 2020, just six days before her second birthday, she received a kidney transplant. Since then, she has thrived. She’s now running, jumping and talking more. She’s also enrolled in a preschool program three days a week. Her mom, Megg, calls her “a little miracle.”

Read more of Charlie Belle’s story online at Child of Children’s

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Inside Pediatrics | Children’s of Alabama

FOR PROGRESSIVE DYSTONIA Deep Brain Stimulation

The impact of a groundbreaking procedure now offered at Children’s of Alabama

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NEUROLOGY

P rogressive dystonia disorders, characterized by changes in movement patterns, can profoundly impact a child’s quality of life. In adults, such disorders are routinely treated with a procedure called deep brain stimulation (DBS). However, this intervention is less commonly used in pediatric populations.

In 2023, Curtis Rozzelle, M.D., and Emily Gantz, M.D., performed Children’s of Alabama’s first DBS procedures for progressive dystonia patients. This innovative therapy has shown promising results in several pediatric patients with limited treatment options. “We’re excited that this innovative procedure is now transferring over to our pediatric patients.” CURTIS ROZZELLE, M.D. “Kids with progressive dystonia seem to do particularly well, while those with other types of movement disorders may have mixed results,” Rozelle said.

Curtis Rozzelle, M.D.

Studies suggest that pediatric patients with progressive dystonia respond well to deep brain stimulation, especially after failing conventional medications. The decision to apply DBS in pediatric cases stems from the specific needs of young patients and advancements in the field.

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“Until Dr. Gantz arrived at the University of Alabama at Birmingham, we didn’t have a movement disorder neurologist here who also had training and experience with deep brain stimulation,” Rozzelle said. “When Dr. Gantz arrived, she opened the door for us to be able to perform the technical aspects of the surgical procedure. Now, we’ve done several.” CURTIS ROZZELLE, M.D.

Emily Gantz, M.D.

Not every child is an ideal candidate for DBS. The decision to initiate DBS is patient-specific, based on the severity of symptoms and the inadequacy of other treatments. A careful evaluation of each patient’s unique situation, including factors such as genetic mutations and the progression of the disease, must be completed before offering DBS surgery. Each deep brain stimulation procedure at Children’s uses stereotactic surgical techniques and the ClearPoint targeting system. This system, employed in an MRI scanner, ensures safe, precise electrode placement in the globus pallidus interna (GPI), a key target for treating progressive dystonia.

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“When we implant the electrode into a specific region of the brain, we can either edit the input throughout the stimulation, or we can take it completely away,” Gantz said. “Think of it as a series of relay circuits in the brain. If someone has dystonia, one of those relay circuits isn’t working properly. By putting in the stimulator and applying an electrical current intermittently, we can suppress the abnormal brain activity. “The stimulator stays in for life, so the procedure doesn’t need to be repeated,” she continued. “Occasionally, we’ll have to change the device’s battery, but they’re rechargeable and designed to last for up to 20 years.” After the initial procedure, patients return to see Gantz to have the device programmed. “I set the programming on their stimulator so they can make slight adjustments at home. It can take a little while for the device to be effective; we usually leave it alone for a few months and then reevaluate,” Gantz said. “We have guidelines for which settings will most likely help, and we start there. We’re looking to ensure we don’t get side effects, such as visual disturbances or muscle pulling, more than anything.” The pediatric patients who have begun DBS for progressive dystonia at Children’s are responding well to the new treatment. “I’m really excited about DBS and its future as a treatment in pediatric neurology, specifically movement disorders,” Gantz said. “It may eventually come into play in other treatment areas, and I’m glad the door is open to us here. I think there will be many more patients who will benefit from it.” ●

Drs. Paige Lundy (left), Sage Rahm (center) and Curtis Rozzelle perform part of a deep brain stimulation procedure at Children’s of Alabama.

A surgeon performs a system function test after placing a generator.

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Inside Pediatrics | Children’s of Alabama

NEWS, HONORS AND AWARDS

Tofil selected for Pediatric Leadership Development Program cohort Nancy Tofil, M.D., M.Ed., professor in the Division of Pediatric Critical Care, has been selected for the 13th Pediatric Leadership Development Program (PLDP) cohort. The PLDP was created by the Association of Medical School Pediatric Department Chairs (AMSPDC) to develop and mentor the next generation of pediatric chairs. The program lasts a year and consists of mentoring programs and project development among many other opportunities. Broadnax receives American Society of Hematology award for inclusion Alexandria Broadnax, M.D., fellow in the Division of Pediatric Hematology, Oncology, and Blood and Marrow Transplantation, was selected for the 2023 American Society of Hematology (ASH) Minority Hematology Fellow Award (MHFA). The goal of the ASH award is to encourage early-career researchers from historically underrepresented minority groups in the United States and Canada to pursue a career in academic hematology, and it provides salary support as well as funds to support hematology focused research projects. Broadnax will receive more than $70,000 in salary support. Ilonze selected to participate in the ASH Clinical Research Training Institute Chibuzo Ilonze, M.D., MPH, assistant professor in the Division of Pediatric Hematology, Oncology, and Bone Marrow Transplantation was selected to participate in the 2023 class of the ASH Clinical Research Training Institute (CRTI). The institute is a yearlong education and mentoring program for hematology fellows and junior faculty at academic medical centers. CRTI offers a broad education on clinical research methods, research collaborations, statistical analysis and managing the demands of family and career. The goal of the program is to produce leaders armed with ideas for clinical hematology research and the tools and resources to make their ideas a reality. Fowler receives the 2023 National CMV Foundation Professional Impact Award Karen Fowler, Ph.D., professor in the Division of Pediatric Infectious Diseases, received the 2023 National CMV Foundation Professional Impact Award at the Congenital Cytomegalovirus Public Health and Policy Conference in Salt Lake City, Utah, in October 2023. The National CMV Foundation’s Professional Impact Award is given to individuals who have utilized their clinical, scientific or professional expertise to increase the awareness of cytomegalovirus and its impact through research, awareness and/or educational efforts. The recipients may be (but are not limited to) physicians, researchers or public health/allied health professionals. James elected a fellow of the Infectious Diseases Society of America Scott James, M.D., associate professor in the Division of Pediatric Infectious Diseases, was elected a fellow of the Infectious Diseases Society of America (IDSA). Selection as a fellow of IDSA is an honor for those who have achieved professional excellence in the field of infectious diseases.

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Sasser selected for educational leadership cohort Will Sasser, M.D., associate professor in the Division of Pediatric Critical Care, has been selected for the Association of Pediatric Program Directors (APPD) Leadership in Educational Academic Development (LEAD) cohort. APPD LEAD provides an opportunity for pediatric leaders in medical education to learn from experienced pediatric program directors, educators and leaders and grow their leadership and organizational skills.

Two faculty elected to American Pediatric Society Ambika Ashraf, M.D., professor in the Division of Pediatric Endocrinology and Diabetes, and Steven Rowe, M.D. , professor in the Division of Pulmonology, have been elected to the American Pediatric Society (APS). The mission of the APS is to advance academic pediatrics. This is accomplished through promoting pediatric research and scholarship, serving as a strong and effective advocate for academic pediatrics, recognizing and honoring achievement, and cultivating excellence, diversity and equity in the field of pediatrics through advocacy, scholarship, education and leadership development.

Sorrentino presents at FemInPEM Conference Annalise Sorrentino, M.D., professor in the Division of Pediatric Emergency Medicine, gave a TED Talk at the FemInPEM conference in Chicago on September 22, 2023. The conference was started three years ago as a networking opportunity for advocates of women working in pediatric emergency medicine (PEM). Sorrentino’s topic was “Leadership is SO 2020!” She talked about the importance of followership in addition to leadership. Kane receives a 2023 Inspiration Award from the AMA Women Physicians Section Andrea Kane, M.D., assistant professor in the Division of Neonatology, received a 2023 Inspiration Award from the American Medical Association (AMA) Women Physicians Section. The Inspiration Award is given to physicians who have mentored and supported fellow physicians throughout their career.

Two faculty receive award from the Clinical Research Forum Alan Tita, M.D., Ph.D., associate dean for Global and Women’s Health at UAB, and Waldemar Carlo, M.D., professor in the Division of Neonatology, received an award from the Clinical Research Forum. Their study, “Azithromycin to Prevent Sepsis or Death in Women Planning a Vaginal Birth,” was chosen for the 2024 Top 10 Clinical Research Award. This study was chosen from an impressive field of applications and extremely qualified research studies.

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Inside Pediatrics | Children’s of Alabama

NEWS, HONORS AND AWARDS

The Children’s of Alabama/UAB Pediatric Transplant and Heart Failure team receives PHTS Vanguard Award The Pediatric Heart Transplant Study (PHTS) has awarded the Children’s of Alabama/UAB Pediatric Transplant and Heart Failure team its PHTS Vanguard Award. This award is given to centers that have met criteria signaling a program’s reliability, timeliness and adequate volume. The PHTS is the world’s largest and oldest registry for pediatric patients who require listing for heart transplantation. PHTS was started in the mid 1990s at UAB as a collaborative effort between UAB Pediatric Cardiac Surgery and UAB Pediatric Cardiology.

Three faculty elected to Society for Pediatric Research Vivek Shukla, M.D., Samuel Gentle, M.D., and Jegen Kandasamy, M.D., all assistant professors in the Division of Neonatology, have been elected to the Society for Pediatric Research (SPR). The mission of the Society for Pediatric Research is to create a multidisciplinary network of diverse researchers to improve child health.

Goncalves wins Society for Pediatric Radiology’s Berdon Award Fabricio Goncalves, M.D., distinguished professor of pediatric imaging at UAB, was presented with the Walter E. Berdon Award for Best Clinical Research Paper by the Society for Pediatric Radiology (SPR). Goncalves’ paper, “The Role of Apparent Diffusion Coefficient Histogram Metrics for Differentiating pediatric Medulloblastoma Histological Variants and Molecular Groups,” was published in Pediatric Radiology . The Bedon Award recognizes the best clinical research paper submitted to Pediatric Radiology in the previous year.

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and lead to cures. And it benefits not only the patients and families who come to Children’s of Alabama, but people

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