Nephrology, Urology

A New Pathway for Prenatal Hydronephrosis

A model of the kidney (Stock photo)

A multidisciplinary team at Children’s of Alabama and the University of Alabama at Birmingham (UAB) has developed a standardized clinical pathway for newborns diagnosed prenatally with hydronephrosis, reducing variability in care, improving coordination among specialties, and helping families receive timely guidance without unnecessary testing or travel.

The pathway, developed by general pediatricians in the newborn nursery in collaboration with pediatric nephrology and pediatric urology, provides a risk-stratified approach to determining which infants require immediate evaluation, which can be safely monitored, and which need little to no intervention. The initiative has already prompted changes in workflow, increased access to telehealth services, and generated interest from providers outside Birmingham.

Prenatal hydronephrosis, or dilation of the kidney’s collecting system, is one of the most common abnormalities identified on prenatal ultrasound. While some cases resolve spontaneously, others may signal underlying conditions such as urinary tract obstruction, vesicoureteral reflux, urinary tract infection risk, or chronic kidney disease. Determining which infants need urgent intervention has historically been challenging.

“We found the existing guidelines confusing,” said Terry Wall, M.D., division director of the UAB Division of Academic General Pediatrics and attending physician in the newborn nursery. “Different specialties interpreted them differently, radiology reports varied in terminology, and there was a lot of inconsistency in how babies were evaluated and referred.”

Wall, who is also a medical informaticist, brought together leaders in pediatric nephrology and pediatric urology to create a single algorithm that could be used consistently by nursery physicians, specialists, radiologists, and obstetric providers.

The resulting pathway categorizes infants based on prenatal and postnatal imaging findings, including the degree of hydronephrosis, bladder abnormalities, and progression over time. Infants identified as high risk are evaluated by urology or nephrology before discharge, while lower-risk patients receive targeted education and follow-up recommendations.

David Askenazi, M.D., pediatric nephrologist and medical director of the Pediatric and Infant Center for Acute Nephrology at UAB, said the pathway was designed with two goals in mind: ensuring that babies with significant disease are identified early while avoiding unnecessary interventions for families whose infants are unlikely to require treatment.

“We don’t want to miss a child who needs to be seen right away, but we also don’t want to burden families when their baby has a mild finding that is likely to resolve,” Askenazi said.

For infants requiring specialty follow-up, the pathway introduced another innovation. Stacy Tanaka, M.D., chief of pediatric urology, established a telehealth model that allows families to meet with pediatric urologists approximately two weeks after discharge.

Previously, referrals often occurred through community pediatricians, and specialists frequently lacked access to postnatal imaging before the initial visit. Families would travel to Birmingham for an appointment, only to learn that additional studies were needed.

Now, newborn ultrasounds are obtained before discharge when indicated, and specialists can review imaging in advance of the telehealth visit. During these appointments, providers discuss results, explain whether additional testing is needed, determine if antibiotic prophylaxis is appropriate, and establish a plan for ongoing surveillance.

“It has helped our workflow tremendously,” Tanaka said. “It also allows us to set expectations for families much earlier. If they need another study, they know why. If the hydronephrosis is likely to resolve, they understand what we’re watching and what to expect.”

This approach is particularly beneficial for families traveling from rural communities or other regions of Alabama. Parents of newborns can participate in appointments from home rather than making repeated trips for visits that may not require in-person evaluation.

The pathway also has strengthened communication among departments. Discussions with radiology and obstetrics have focused on standardizing prenatal ultrasound reporting so nursery providers can more easily place patients into the appropriate risk category.

Although the initiative was implemented only recently, providers say it already has reduced confusion, improved consistency, and enhanced education for both families and primary care physicians.

The work ultimately may extend beyond Children’s. Tanaka has shared the algorithm with pediatric urologists in Mobile, Ala., and Askenazi plans to discuss the model with colleagues in the Neonatal Kidney Collaborative, an international network focused on improving kidney care for newborns. “We’ve worked out many of the details,” Wall said. “If other centers can use what we’ve learned to standardize care and improve outcomes for families, that’s a win for everyone.”

Pulmonology

Finding Early Warning Signs of CF-Related Kidney Disease

William “Tom” Harris, M.D., is a pulmonologist at Children’s of Alabama and UAB.

By Charles Buchanan

Cystic fibrosis (CF) is often defined as a disease of the lungs. But as advanced therapies help today’s young patients breathe easier and live longer—many will reach their 60s, according to the 2024 Cystic Fibrosis Foundation Registry—attention is shifting to the disease’s impact on other organs that may be vulnerable to the consequences of aging. This evolving landscape of CF clinical care has prompted Tom Harris, M.D., a pulmonologist at Children’s of Alabama and the University of Alabama at Birmingham (UAB), to investigate the kidneys.

“Interestingly, there’s more CFTR [cystic fibrosis transmembrane conductance regulator], the protein that causes cystic fibrosis, in the kidneys than the lungs,” Harris said. That means that as people with CF grow older, they face a higher incidence of chronic kidney disease than the general population, where the risk is about one in seven, according to Harris. Acute kidney injury (AKI), is more prevalent among people living with CF due to frequent antibiotic exposure and previous hospitalizations, and recurrent AKI is a major risk factor for development of chronic kidney disease. Harris also suspects loss of CFTR function takes away some of the kidney’s reserve.

Protecting the kidneys and other organs that express CFTR is crucial as lung disease stabilizes and people with CF age to help prevent health complications that can consume the patients’ hard-earned longevity. “The priorities of CF are changing,” Harris said. “We are now very much prioritizing quality of life across the years.”

Not every person with cystic fibrosis will develop kidney disease, of course. The challenge for researchers like Harris is to find biomarkers that will identify the ones at risk—and sound the alarm early enough for therapeutic intervention to preserve kidney function. “Right now, we are trying to establish which biomarkers are important and how they might indicate the progression of chronic kidney disease over time,” Harris said. Joining him in this mission are a team of scientists across the country who are part of a landmark project funded by the Cystic Fibrosis Foundation (CFF). Together they will follow a group of inpatients and outpatients for three years to study promising new diagnostic tools for CF-related kidney disease. They also are looking at kidney disease in the CF animal model that was pioneered at UAB. “We are very grateful to the CFF for having such vision to allow us to lay such a firm foundation to study kidney disease, combining both human observational study with mechanistic analyses in gene-edited animal models,” Harris said. “We are hopeful this project will allow us to pioneer the field of CF kidney disease and health.”

The researchers are focusing on urine in their quest to find novel biomarkers because of its direct connection to kidney health. Urine is “like a liquid biopsy,” Harris said. “With every urinary output, you can measure how well the kidney is working.” Physicians traditionally have tested urine or blood for elevated levels of specific proteins, electrolytes, and waste products such as creatinine to find proof of disease. But organ damage has already occurred by the time those reveal a problem, Harris said. Instead, he and his colleagues are investigating more subtle, sensitive measures that could flash the earliest warning signs of trouble. Candidates include kidney injury molecule (KIM-1) and neutrophil gelatinase-associated lipocalin (NGAL), two urinary biomarkers indicating minute structural changes in the kidney that precede a deterioration in function.

“In addition to detecting which patients might have some evidence of disease, we also want to use those biomarkers to help us understand the pathways of disease,” Harris said. “Is it more fibrotic? Is it more inflammatory?” Exosomes, which are the molecular packages transmitted between cells, offer a prime example. Exosomes can be informative about the intercellular communication in health and disease, but they also often carry the mediators that affect change. For instance, neutrophil-derived exosomes may shed light on the inflammatory triggers of kidney disease.

Such detailed methods of diagnosis could hold promise for designing personalized treatments for patients. CF already is a precision medicine success story following breakthrough discoveries about the CFTR gene that causes the disease, including the distinct ways in which each CFTR mutation affects protein function. Some mutations impact how much CFTR is made; some affect how well CFTR gets to the cell surface; some impact how well CFTR transports electrolytes. Knowing each patient’s genetic mutation plays a role in the choice of CFTR-directed therapy.

Now, researchers are applying what they know about CFTR’s impact in the lungs to the kidneys, where they suspect it plays a different role. In the lungs, CFTR is needed to transport chloride ions, and a lack of it can cause airway secretions to thicken. But in the kidneys, CFTR is involved in acid-base metabolism, which may be disrupted in chronic kidney disease. “Part of the reason CF-related kidney disease has been overlooked previously is that, instead of being a disease of obstructed lumens from mucus plugging, the kidney disease is an organ of pH balance and fluid transport,” Harris said. Scientists want to pinpoint the specific cell types and regions of the kidney that are affected most by CFTR dysfunction. “CFTR expression is not the same across all cell types in either the lung or the kidney. In the lung, an ionocyte that is positioned near mucus glands is the highest CFTR-producing cell,” Harris said. “Investigators at UAB have recently discovered a similarly high CFTR-expressing cell in the kidney. Future steps will be to define how this high CFTR-expressing cell promotes renal health.” These findings could illuminate new targets for potential treatments to protect kidney function and help patients thrive as they age.

“The future’s bright,” Harris said. “CF has primarily been considered a pediatric disease. Now it’s very much an adult disease. And we as pediatricians have to think about the future . . . to transition our focus from disease to health—and to celebrate the wonderful, previously unimaginable question of how can we help our pediatric patients with CF age well into senior adulthood.”

For more information about Harris’ research, check out this episode of the Children’s of Alabama PedsCast.

Cardiology

Aortic Arch Surgery and Cognitive Development

A Children’s cardiac surgeon is researching the connecting between aortic arch surgery and brain development. (Stock image)

By Charles Buchanan

Parents naturally ask plenty of questions when their newborn needs aortic arch reconstruction, a life-saving surgery for congenital malformations restricting blood flow. Their major concerns involve the procedure’s complex logistics and potential complications, which Hampton Gray, M.D., a Children’s of Alabama cardiac surgeon, can explain in detail. But not every answer comes so easily. He says some of the simplest questions from parents are also the toughest: “How’s my kid going to be when he is 5 years old? Can he go to school, or is he going to be delayed? Is he going to be behind the other kids?”

Their worries arise from a critical component of aortic arch reconstruction—the temporary reduction or shutdown of blood circulation throughout the body and to the brain. And the reason those questions are difficult to answer is that little is known about the surgery’s long-term effects on brain development. “There is limited data currently for us to properly educate parents on how their kid will cognitively develop through infancy, toddler, and school-age years,” Gray said. Now he is spearheading a novel, multiyear research project to fill that knowledge gap. The findings could provide parents with the answers they need and help them understand what to expect as their children grow.

Cardiac Surgery Meets Psychology

The trial kicked off in July, funded by an $80,000 grant from the Kaul Pediatric Research Institute, a Children’s/University of Alabama at Birmingham (UAB) program supporting trailblazing work from rising investigators. During the next two years, Gray and his team will follow up with Children’s patients who had major open-heart surgery to repair a congenitally small native aorta in the last 15 years. That cohort includes up to 200 children, though Gray anticipates the study will attract approximately 80 participants ranging in age from infants to school-age children.

To measure the patients’ long-term progress, Gray has forged an innovative collaboration with the clinical psychologists at UAB and Children’s. “They are pros at testing kids about cognitive function, executive function, fine motor skills, and all the things that dictate how your brain is developing as a young child,” Gray explained. UAB already has an established Newborn Clinic, where the clinical psychologists gauge the growth and development of children who spent time in the neonatal intensive care unit (NICU). For the cardiac surgery study, they created a novel algorithm to tailor their tests for the varying age levels of patients following aortic arch surgery. They also will administer the tests to participants and interview parents about their child’s cognitive development.

“It will be very valuable to test these kids at different time points and different age groups,” Gray said. With the cognitive development data in hand, the researchers will evaluate the trajectories of aortic arch patients alongside children who have not experienced heart surgery.

A Tale of Two Strategies

The study also may shed new light on the two cerebral perfusion strategies used to manage blood circulation during aortic arch surgery. The researchers are eager to know if the techniques affect brain development differently, which leads to the inevitable question: Is one option better than the other?

The original cerebral perfusion strategy, deep hypothermic circulatory arrest (DHCA), involves cooling the body to a temperature between 18 and 20 degrees Celsius and turning off the heart-lung machine so that no blood circulates through the body. That gives cardiac surgeons approximately 40 minutes to complete the reconstruction without increasing the risk for brain injury, Gray said. Cardiac surgeons following the newer strategy, antegrade cerebral perfusion (ACP), cool the body to the same low temperatures but also sew a small graft to the innominate artery and keep the heart-lung machine running, selectively perfusing the brain. As a result, about 15 to 20% of the entire cardiac output continues flowing, according to Gray’s estimate. Today, at least 70 to 80% of cardiac surgery centers, including Children’s, have adopted ACP while the rest continue to rely on DHCA, he noted.

“It would make sense, theoretically, that giving the brain a little bit of blood flow would be better than giving it no blood flow,” Gray said. However, in a 2024 study comparing the two cerebral perfusion strategies, he and his research team found no significant differences in short-term hospital outcomes among 165 newborns and infants undergoing aortic arch surgery at Children’s between 2012 and 2023. Both types of patients also experienced a low rate of neurologic events such as stroke and seizure.

Gray’s current project, with its long-term, cognitive development focus, offers a logical next step in identifying any differences that emerge between DHCA and ACP patients as they grow up. The study’s participants will include the two groups, since both cerebral perfusion strategies were in use at Children’s as recently as 2022, giving the researchers ample opportunities to compare outcomes. The team also will gather information about nonsurgical factors that can influence brain development, including the children’s family structures and education, to round out the picture of each child’s growth.

Answers at Last

Gray hopes the research will serve as a stepping stone to a broader exploration of aortic arch patients and their progress. Ultimately, he would like for the findings to serve as pilot data for a larger initiative involving multiple cardiac surgery centers and extramural funding from sources such as the National Institutes of Health. The most useful information would come from a trial that pairs a DHCA-exclusive center with an ACP-exclusive center and follows patients from both over several years, he said.

No matter where his investigation leads, Gray says the goal remains the same: to provide parents with meaningful answers to their simple, yet tough, questions about their child’s future. And if the cardiac surgeons and clinical psychologists make pioneering discoveries about the impact of aortic arch surgery along the way, that’s a bonus. “Children’s supports pushing the envelope to make children’s lives and parents’ lives better,” Gray said.

Neurology & Neurosurgery

Time is brain: A pediatric code stroke program

Code Stroke at UAB and Children’s of Alabama is designed to improve recognition, response and outcomes. (Stock photo)

By Heather Watts

A stroke is often thought of as a condition of adulthood, yet it remains a significant and underrecognized cause of morbidity and mortality in the pediatric population. Despite being cited among the leading causes of death in children, pediatric stroke is frequently missed or diagnosed late, in part due to its relative rarity, diverse clinical presentations and the unique risk factors that distinguish it from adult stroke. As a result, many children with stroke present outside the narrow window during which acute interventions may be considered, limiting opportunities for therapies that can meaningfully alter neurologic outcomes. In pediatric stroke, as in adult stroke, the principle remains the same: Time is brain.

Improving pediatric stroke education

During her training in child neurology at the University of Alabama at Birmingham (UAB), Sarah Novara, M.D., MSQHS, FCNS, associate professor in the Division of Pediatric Neurology, had several patient encounters that highlighted both the devastating potential of delayed stroke recognition in children and the extraordinary opportunity that exists when stroke is identified and treated promptly.

“When we were able to quickly evaluate and treat stroke symptoms in our pediatric patients, we saw amazing neurologic recovery,” Novara explained. “Seeing the importance of early stroke recognition firsthand inspired me to improve our pediatric stroke education and standardization processes at Children’s of Alabama and UAB.”

As a senior resident, Novara developed pediatric stroke education and streamlined order sets and processes for residents, fellows and faculty. Novara worked with Tony McGrath, M.D., associate professor in the Division of Pediatric Neurology and Nancy Tofil, M.D., M.Ed., professor in the Division of Pediatric Critical Care, to develop a pediatric stroke simulation as part of pediatric stroke education for trainees and faculty. “The simulation has been highly efficacious in advancing pediatric stroke evaluation and management knowledge in a variety of learner settings since its creation,” Novara explained.

From education to systems change: Creating a Pediatric Code Stroke

Once on faculty at UAB, Novara continued her work with pediatric stroke education and began working on improving early stroke recognition among providers at Children’s and UAB by establishing a Code Stroke for pediatrics.

“We decided to model a Code Stroke process after our adult neurology colleagues and other select children’s hospitals across the U.S.,” Novara said. The pediatric Code Stroke initiative aims to offer necessary, efficient evaluation and management of patients presenting with signs of possible stroke, and if inevitably they have not had a stroke, the Code Stroke process assists in evaluating and managing conditions that mimic stroke and deserve appropriate treatment. 

In the summer of 2024, the Code Stroke working group met to create the Children’s of Alabama Code Stroke protocol for patients greater than one month of age with acute neurologic deficits and who were last known at their neurologic baseline less than 24 hours prior to presentation at the hospital. This is the window in which options of acute stroke intervention may be offered, if there are no contraindications. In addition to the protocol, order sets within EPIC were streamlined for the evaluation of Code Stroke patients in the emergency department (ED) or on the hospital floors, and stroke patient admission order sets were provided as well.

This Code Stroke effort was spearheaded by Novara, Tofil and Jeffrey Blount, M.D., MPH, professor in the Division of Pediatric Neurosurgery. It also included colleagues from pediatric emergency medicine, neuro-interventionalists, radiology, nursing, and operators.

After the Code Stroke protocol was established and education was provided across the pediatric divisions, the Children’s of Alabama Code Stroke went live in September 2024. As of July 2026, a Code Stroke had been called 131 times (either in the ED or on a hospital floor). Approximately 12% of these Code Strokes have identified ischemic or hemorrhagic strokes, allowing for efficient evaluation and management. Several of the pediatric acute ischemic stroke patients have even benefited from an acute thrombectomy performed by the on-call neuro-interventionalists. Code Stroke patients with hemorrhagic stroke have benefited from the expedited care of the pediatric neurosurgery team. Many of the Code Stroke patients have made remarkable recoveries.

“We have been very excited about the success of multidisciplinary care within the Code Stroke process, as many patients—even those without strokes—have received expedited treatment for life-altering conditions,” Novara said.

“The Code Stroke working group meets quarterly to continuously discuss ways in which to improve our Code Stroke protocol and to collaborate together as we try to provide the best care for our patients,” Tofil added.

Expanding care beyond the acute phase: Pediatric Stroke Clinic

In early 2025, a monthly Stroke Clinic was established by Novara and McGrath to provide a unique pediatric stroke follow-up clinic that offers ongoing patient support, education and follow-up care, including best efforts at secondary stroke prevention. 

In the monthly stroke clinic, the pediatric neurology team collaborates with pediatric hematology and neurosurgery colleagues in the care of unique pediatric patient populations seen in their clinics who are at risk for stroke. The stroke clinic also allows the pediatric neurology team to provide effective transition of care for patients from pediatric to adult stroke care at UAB as needed.  

The pediatric stroke clinic at Children’s of Alabama is a participating site within the International Pediatric Stroke Study, which is a research group working hard within the International Pediatric Stroke Organization to expand the knowledge about pediatric stroke and best practices for these unique patients.

The Code Stroke program has demonstrated that targeted educational initiatives, combined with the development of standardized, multidisciplinary response protocols, can meaningfully improve the evaluation and management of children presenting with possible pediatric strokes.

As awareness grows and standardized care pathways expand, there is an opportunity to reduce the burden of pediatric stroke and ensure that children receive timely, evidence-informed care when minutes matter most.

Neurology & Neurosurgery

Lalor appointed Director of Pediatric Neurology

Kathryn Lalor, M.D. was appointed director of the Division of Pediatric Neurology in May 2026.

By Angel Pine

As the new director of the Division of Pediatric Neurology, Kathryn Lalor, M.D., aims to build on the strong foundation already established within the division.

Lalor, who was appointed to the position in May, has been a valued member of the division as associate professor, contributing to its continued growth in both clinical care and academic excellence. In her new role, she will lead efforts to further strengthen the division’s mission across patient care, education, and research.

“Dr. Lalor joined the division in the midst of its recent growth,” said Leon Dure, M.D., professor and former division director of Pediatric Neurology. “She has the skills and temperament needed to ensure UAB Pediatric Neurology’s reputation of academic and clinical excellence.”

“This appointment represents an opportunity to grow into a new kind of leadership which I plan to approach with humility and intentionality,” Lalor said. “Leon Dure has left big shoes to fill, but I am thankful to continue growing the supportive culture he has fostered.”

Lalor also expressed enthusiasm about the division’s future, noting its significant growth over the past decade and the opportunity to build on that momentum. Lalor emphasized the importance of collaboration across the division, adding, “If you want to go fast, go alone. If you want to go far, go together,” as she looks forward to working alongside faculty, staff and trainees to continue advancing care for children across Alabama.

Her appointment reflects the department’s continued commitment to strong leadership and advancing pediatric neurological care.

Neonatology

Studying magnesium sulfate in very preterm babies

Research shows magnesium sulfate does not improve neurodevelopmental outcomes in very preterm babies. (Stock photo)

By Anna Jones

Research led by Children’s of Alabama and the University of Alabama at Birmingham (UAB) shows that magnesium sulfate does not improve neurodevelopmental outcomes in babies born very preterm between 22 and 24 weeks gestation.

Babies born during 22-24 weeks of gestation have a higher risk of neurodevelopmental impairment. While prior studies have found that magnesium sulfate helps prevent the development of cerebral palsy for babies born at later gestational ages between 24 and 32 weeks, this observational study led by Margaret Page, M.D., and Ashley Battarbee, M.D., in the UAB Department of Obstetrics and Gynecology, along with colleagues in the Department of Pediatrics, is one of the few studies of its kind to look at the use of magnesium sulfate for babies born at 22-24 weeks of gestation.

“This multicenter study led by Dr. Page and colleagues at UAB is important as it is the largest study designed to assess the two-year outcomes of infants born extremely preterm exposed to antenatal magnesium sulfate and/or steroids, two common treatments given before delivery,” said Wally Carlo, M.D., co-director of Neonatology at UAB and Children’s of Alabama and the Neonatal Research Network.

Battarbee says that OBGYNs cannot always stop a preterm birth, so they oftentimes need to shift their focus to how they can best prepare the baby to be born early.

“We consider interventions such as administering magnesium sulfate or antenatal corticosteroids to the pregnant patient to help improve outcomes for their baby after birth,” Battarbee said. “While the American College of Obstetricians and Gynecologists (ACOG) recently updated their guidance on corticosteroids for babies at 22 weeks, we did not have updated guidance on magnesium sulfate and wanted to explore this treatment further.”

The results of the recent study reinforce current national guidelines recommended by ACOG and the Society of Maternal-Fetal Medicine that do not recommend giving magnesium sulfate at 22 weeks and instead prioritizing antenatal corticosteroids to improve neurodevelopmental outcomes. Unlike magnesium, steroids have demonstrated benefit for infants born at very early gestational ages and remain the primary intervention when an early delivery is anticipated.

“There is not a lot of guidance on the use of magnesium sulfate in babies at this gestational age,” said Page, the lead investigator of the study. “Through this research, we were able to find that there was no benefit to using magnesium sulfate in addition to antenatal corticosteroids. This shows us that more treatment does not always equal better outcomes.”

Both Page and Battarbee emphasize that administering magnesium sulfate at that gestational age did not lead to any measurable increase in maternal or fetal harm in this study, but magnesium does carry known maternal risks, making it important not to use it without proven benefit. Additionally, using magnesium for neuroprotection in babies at these very early gestational ages when it is not effective may hinder future research in identifying interventions that truly make a difference.

This analysis was conducted using data from and in collaboration with the Neonatal Research Network, a large research network encompassing academic institutions across the United States funded by the Eunice Kennedy Shriver National Institute of Child Health and Human Development.

Endocrinology

Ashraf co-edits textbook on pediatric diabetes

Ambika Ashraf, M.D. is the director of the Division of Pediatric Endocrinology and Diabetes at Children’s of Alabama and UAB.

By Amy Richardson

A new textbook, co-edited by Children’s of Alabama pediatric endocrinologist Ambika Ashraf, M.D., offers a comprehensive look at pediatric diabetes, highlighting the latest research findings and clinical guidance for managing diabetes in children and adolescents.

Written by international experts from leading diabetes care centers, “Pediatric Diabetes” covers major forms of the disease, including Type 1 diabetes (T1D), Type 2 diabetes (T2D), Wolfram syndrome and cystic fibrosis-related diabetes. It also provides up-to-date management strategies for evolving diabetes phenotypes and current clinical approaches to care.

Ashraf, who also serves as director of the UAB Division of Pediatric Endocrinology and Diabetes, says the book addresses a critical gap in clinical resources focused specifically on pediatric diabetes. “Most available information on pediatric and adolescent diabetes has historically been limited to chapters within larger pediatric endocrinology books, without the space to cover specific topics in depth,” she said. “That’s what makes this book valuable. It fills a genuine gap in the literature.”

Ashraf co-edited the 15-chapter volume with Benjamin Udoka Nwosu, M.D., professor of Pediatrics and Endocrinology at the Zucker School of Medicine at Hofstra/Northwell in New York. She also co-authored two of the book’s chapters: “Precision Medicine in Diabetes Phenotypes” and “Neonatal Diabetes.” Mary Margaret Barr, M.D., a fellow in the Joseph S. Bruno Pediatric Endocrinology Training Program at UAB, co-authored Neonatal Diabetes with Ashraf. Barr will be joining the Division of Pediatric Endocrinology and Diabetes as a faculty member in July 2026.

Pediatric diabetes one of the most common chronic diseases among individuals under age 20 in the United States. In 2022, an estimated 21,732 youth under 18 were newly diagnosed with T1D, and 14,490 were diagnosed with T2D. Between 2001 and 2017, the prevalence of T1D in U.S. youth increased by 45%, with the largest increases among non-Hispanic white and non-Hispanic Black youth. Over the same period, T2D prevalence increased by 95%, with the largest increases among non-Hispanic Black and Hispanic youth. Based on increasing rates, by 2060, the number of youth with T1D is projected to reach 335,000, and the number of youth with T2D is expected to reach 220,000—increases of 65% and 673%, respectively.

Pediatrics Diabetes is published by Springer as part of the Contemporary Endocrinology series and is available in print (ISBN: 978-3-032-17035-4) and eBook (ISBN: 978-3-032-17036-1) formats at Springer.com and Amazon.

Ashraf also holds the Ralph Frohsin Endowed Chair in Pediatric Endocrinology at UAB and serves as associate director of the UAB Comprehensive Diabetes Center. Ashraf is also co-editor of “Pediatric Dyslipidemia,” published by Springer in 2023.

Pulmonology

New study explains ETI’s effectiveness on Cystic Fibrosis

A new study’s findings “moved the needle” on researchers’ understanding of how ETI works. (Stock illustration)

The 2019 approval of the transmembrane conductance regulator (CFTR) modulator elexacaftor, ivacaftor, and tezacaftor (ETI) for cystic fibrosis (CF) dramatically changed the landscape of the disease. For the first time, nearly 90% of those with CF had access to a disease-modifying drug. In the pivotal clinical trial, ETI significantly improved lung function and reduced pulmonary exacerbations while improving patient quality of life.

But exactly how the drug worked still needed explaining.

Now, a major new study, called PROMISE, involving pediatric pulmonologist Spencer Poore, M.D., and University of Alabama at Birmingham (UAB) pulmonologist George M. Solomon, M.D., clearly demonstrates that some of ETI’s effects are due to its ability to tamp down inflammation.

Inflammation is the background noise of CF: always present, always active, contributing to lung damage, infections, fatigue, weight loss and poor outcomes. Even when symptoms improve, some degree of inflammation continues unchecked.

But as PROMISE showed, ETI dramatically reduces that inflammation. The PROMISE trial is a prospective, multi-center, observational study following 487 people ages 12 and older with CF. A group of 223 participants agreed to participate in the inflammation substudy, in which their blood and sputum were collected prior to starting ETI and then five times over the next 30 months.

The team measured markers of inflammation in the lungs, including neutrophil elastase (NE), a powerful enzyme linked to tissue damage; calprotectin, a marker of neutrophilic inflammation; and pro-inflammatory cytokines such as IL-1β and IL-8. In the blood, they tracked levels of the inflammatory markers high-sensitivity C-reactive protein (hsCRP); calprotectin; and HMGB-1, another inflammatory mediator. All are tied to lung destruction, bronchiectasis, exacerbations and outcomes.

Within one month of starting ETI, airway inflammation markers fell sharply and remained low throughout the 30 months. At the same time, markers of system inflammation (hsCRP and calprotectin), also significantly declined.

As the authors wrote, “These changes represent a disease-modifying benefit of this transformative therapy.”

What made the findings even more powerful was how closely inflammation tracked with clinical outcomes. So, lower neutrophil elastase levels meant better lung function, while lower hsCRP led to improved respiratory symptoms. Interestingly, an increase in airway IL-6 also correlated with improved lung function, a puzzle since IL-6 is often thought of as inflammatory. However, the authors noted, it also plays a role in regulating inflammation. This suggests its increase may reflect a shift toward a more normal immune response rather than chronic destructive inflammation.

Although ETI quelled much of the inflammation, it was still there, especially in older patients and those with more advanced lung disease.

“We have not seen complete resolution,” Poore said. But, he noted, the set point has shifted. And this represents a shift in the disease itself, he said. “What I was taught versus what I see now is different.”

This includes fewer patient admissions; less dependence on feeding supplementation, advanced feeding support and feeding tubes; improved growth; and more stable disease.

One of the biggest questions lies with children who start therapy very early given that ETI is now approved for kids as young as 2.

If they never experience that chronic inflammatory engine, “what does their health and outcomes look like when they’re 25?” Poore asked. Does early treatment prevent the damage entirely? Or does it simply delay it? “We’ve moved the needle,” he said. “But how far?”

That uncertainty is fueling ongoing research. “This isn’t done,” he said. “This is a living, breathing assessment.”

Urology

Treating Bathroom Issues Virtually

Children’s is using telehealth to answer parents’ questions about bathroom habits. (Stock photo)

For families of children with bladder and bowel dysfunction, the journey to care at Children’s of Alabama can involve a long drive, a crowded waiting room, and the worry that the problem was serious—only to be told that what their child needs most is better bathroom habits.

“We have a really wide catchment area,” said Children’s chief of pediatric urology Stacy Tanaka, M.D. “Sometimes we were seeing families coming in from the coast. They had driven three-and-a-half, four hours.” By the time they arrived, parked and waited, an entire day was lost and the advice they received was, well, less than earth-shattering.

“They get here, and you tell them, ‘Hey, you just need to urinate and poop a little bit better,’” she said. “It doesn’t go over that well sometimes.” In fact, it could have been handled by a phone call.

Today, that’s essentially how they handle it. Tanaka and nurse practitioner Kelsey Boswell Moore, CRNP, see more than 20 patients each week via telehealth.

The program launched in early 2025, partly out of necessity. “We were transitioning and were a bit understaffed,” Tanaka said. “We started it just as a ‘let’s try to get as many patients seen as possible.’”

What began as a staffing solution quickly became a new model of care.

One reason it works so well is that most children with bladder and bowel dysfunction improve just from counseling and conservative management on better bladder habits and better bowel habits. They don’t even require prescription medication. If any red flags pop up, such as a child who had back surgery or is having urinary tract infections with fever, the team brings them in for a face-to-face consultation. “Those are signals we need to see you sooner,” Tanaka said.

For most families, all it takes is talking and instructions.

“A lot of times they’ve never really paid attention to how often they’re going to the bathroom,” said Moore, who conducts most of the telehealth visits. “They’ll say they have urgency, or that they can’t hold it, but then you realize they’re waiting until the last minute every time.”

Telehealth makes it easier to explore those details.

“They’re sitting in their living room,” Tanaka said. “It’s a more relaxed environment. All the other distractions really go away. In that relaxed environment, it’s easier to talk about how often they’re going, when they’re going and what’s really happening,” she said.

“Sometimes they say they’ve done everything,” she said. “But the child is still drinking fluids late at night or didn’t actually go to the bathroom before bed.”

And for those who do need to be seen in person, the telehealth visit allows Moore and Tanaka to prepare for the appointment by ordering any necessary tests, which increases efficiency.

If families still want to be seen in the office or have the child undergo imaging, “We can absolutely do that,” Tanaka said. “We can rule out the scary things, and then it becomes easier to continue with telehealth knowing everything looks okay.”

“This only works if the patient and parent are engaged,” she added. “If they don’t feel right about the plan, it’s not going to work.”

And it does work. For instance, consider the 8-year-old boy with enuresis. The problem became obvious after a brief telehealth visit.

“When he woke up in the morning, he didn’t go to the restroom,” Tanaka said. “He would eat breakfast, get dressed, and the first time he went was at school.” The child had trained himself to ignore bladder signals.

The solution? A schedule. Go first thing in the morning and use the restroom at planned times during the day. Also, alert the teacher. Four weeks later, the problem was resolved.

“That family never had to step foot in the hospital,” Tanaka said.

Which, of course, is the goal.

Orthopedics

A New Approach for Patellar Instability in Children

An X-ray showing a displaced left kneecap

For Kevin Williams, M.D., and the Children’s of Alabama orthopedic team, patellar instability is a commonly seen problem. The condition—in which the kneecap repeatedly slips out of place—and all of its inherent challenges have been the subject of much discussion worldwide recently, Williams says. But he and his team have developed a solution—a modified procedure that combines existing, well-established methods and is already showing promise.

The procedure is called medial patellofemoral ligament (MPFL) reconstruction. Williams and his team began developing a modified version of it about two years ago and refined it in 2025. They’ve used it on approximately 15-25 patients, and the results have been encouraging. “Children and adolescents that are still growing and developing have been able to get back to doing activities they enjoy—such as dancing and gymnastics—faster compared with our previous, more invasive procedures,” he said. “Complications have been scarce so far in the early stages of this modified procedure.”

The Challenges of Patellar Instability

The procedure treats a problem that is challenging for several reasons, perhaps most notably the various ways patellar instability can present. In some cases, a child might have  been born with a kneecap problem or developed it early in childhood, which means the body is used to the anomaly. When treating children, orthopedic surgeons also have to manage challenges such as small bones and growth plates, “which we don’t want to impede or create any problems with, because it could be detrimental toward a kid’s growth,” Williams explained.

Williams and his team developed the modified procedure to address all of these challenges. It allows them to use small implants that are stitch-based or suture-based and don’t require a lot of drilling. It also allows surgeons to spare the growth plate and balance the soft tissues.

How it Works

MPFL reconstruction in young adolescents is performed in a limited fashion by surgeons in North America and abroad, Williams says. To perform the procedure, the surgery team creates holes in the bones to attach the kneecap to the inside of the femur bone via either an allograft or autograft tissue source. Williams and his team use smaller instrumentation and grafts for this procedure to spare the growth plate and account for the smaller anatomy. “The surgical procedure is designed to grow with the patient,” he said.

Though the team’s procedure is different from others that are available, it’s not experimental, Williams says, because it’s a combination of techniques that are already standard practice. The implants used in this procedure are approved by the U.S. Food and Drug Administration. 

Recovery

One of the biggest benefits of the new technique is recovery time. “Compared with the bigger anatomy-changing surgeries, it’s much improved,” Williams said. With modified MPFL reconstruction, the patient is typically on crutches for only a week or two before they’re able to walk around mostly normally. After that, they wear a brace for up to two months. In many cases, they’re back to playing sports within three or four months, although some may need six months depending on factors such as age and underlying conditions.

Risks Compared to Alternatives

Another benefit is decreased risk. Though every surgery comes with risks, Williams says the modified MPFL procedure seems to carry fewer risks compared to conventional techniques, especially when it comes to issues with the growth plate.

Wide Range of Uses

Part of what makes the procedure successful is its ability to treat patients whose kneecap issues stem from a variety of causes. For example, many children with Ehlers-Danlos syndrome, Down syndrome, Klinefelter syndrome and other congenital conditions are often predisposed to having kneecap problems. Williams and his team have successfully treated them with the modified MPFL procedure. It also works for patients with Nail-Patella syndrome and Genitopatellar syndrome. The Children’s orthopedic team sees many of these types of cases because they serve the entire state of Alabama.

Looking Forward

Despite the success of the procedure, it’s not something the team pushes. Williams says they typically try conservative approaches first, such as braces—which don’t typically work well in these cases—or rehab. But he says it’s a good option if surgery is needed.

Williams anticipates that they’ll learn more as they perform more of these procedures. With the hospital’s move to a new electronic health record system, the team is working on strategies for better tracking outcomes. “That’s one of the goals for this year—we’re  planning on tracking patient-reported outcomes more effectively and becoming more involved in national registries to track procedural success and contribute to research more vastly in the U.S. and abroad,” he said.

So far, the team and their patients have been pleased with the impact of the modified MPFL procedure. “We’ve been happy with the breadth of underlying conditions our modified procedure treats, with decreased complications, and with increasing capacity for returning to sport,” Williams said.