Pulmonology

New study explains ETI’s effectiveness on Cystic Fibrosis

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

Stock photo of a mom helping a child in the bathroom

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—like 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. This surgery uses 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,” Williams said.

Though the 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.

Gastroenterology

Helping heart patients with swallowing and feeding problems

Stock photo

Within days of being born with hypoplastic left heart syndrome, or HLHS, in early 2025, “James” underwent life-saving surgery to reconfigure blood flow so the right ventricle of his underdeveloped heart could pump blood to his little body. Unsurprisingly, the newborn had trouble swallowing and building the endurance needed to feed by mouth. In swooped skilled clinicians from the Dysphagia Clinic at Children’s of Alabama, who worked with James and his parents to determine the safest, most optimal feeding strategy for him after discharge.

The baby—now heading toward his first birthday and doing well—exemplifies the value of the Dysphagia Clinic’s multidisciplinary approach. The clinic previously existed in other capacities at Children’s but coalesced in January 2025 after the arrival of physician assistant Alex Clifton, PA-C, who runs the once-weekly, full-day clinic, seeing about 10 patients each week. Clifton works closely with speech language pathologist Kristen Kirkland, MCD, CCC-SLP, along with several other speech clinicians who rotate to cover the clinic.

Leaders with the Children’s of Alabama Aerodigestive Program created the Dysphagia Clinic to address the feeding and nutrition needs of babies with congenital heart disease as well as babies followed within the Aerodigestive Program who need close support from the gastroenterology, nutrition and speech therapy teams. In the clinic, patients receive medical and speech therapy perspectives, along with nutrition support. Kelly Trumbull, a registered dietitian with the Children’s Clinical Nutrition team, is embedded in the clinic.

The cardiac portion of the program was designed to address the previous gap of multidisciplinary, outpatient feeding care for babies with congenital heart disease. The team cares for these patients using the pediatric feeding disorder framework, addressing medical, nutritional, skill and psychosocial needs. Cardiologists, pulmonologists, otolaryngologists and pediatric surgeons collaborate with the clinic on this effort.

“We long wanted to fill a local void in this type of care for these heart patients,” said Rachel Kassel, M.D., Ph.D., a pediatric gastroenterologist with the Aerodigestive Program. “Buy-in from the University of Alabama at Birmingham (UAB) Department of Pediatrics, the Children’s Hearing and Speech team and the Children’s Nutrition team—paired with having a team of passionate individuals in the Dysphagia Clinic—has improved outpatient feeding and nutrition care for babies with complex congenital heart disease.”

Congenital heart disease encompasses a set of conditions that greatly predispose a baby to feeding and swallowing difficulties. These problems occur in pediatric heart patients in several ways: either by compressing the esophagus or vessels; through nerve damage; or from rapid breathing and fatigue in infants that can disrupt the coordination of sucking, swallowing and breathing. Their cases can also be complicated by vocal cord paralysis, ECMO treatment or the need for intubation.

Clifton and Kirkland estimate that perhaps 70% of pediatric cardiac surgery patients deal with some component of feeding difficulty or aspiration. “Many pediatric GI programs in outlying communities refer to us, so we’re often the last step in the region for these complex kids,” Clifton said.

Clifton also sees patients in the Children’s GI clinic, addressing challenges with formula tolerance, advancing diet, and any other feeding issues. Additionally, she refers patients to the aerodigestive team, which treats children with overlapping problems involving the airway, lungs and upper digestive tract. In the Dysphagia Clinic, Clifton and Kirkland are often joined by nutrition specialists and other speech clinicians, and they also consult with social work and lactation professionals as needed.

“It’s helpful to have multiple disciplines looking at a child with complex medical problems,” Clifton said. “Every kiddo is different.”

Kirkland works closely with patients’ parents and caregivers to help them look for feeding stress cues in infants as well as signs of aspiration. She helps align the child’s individual challenges—which can also include aversion to oral feeding—with the needs of the family, often employing feeding tubes to fill the gap in nourishment. Other interventions can involve thickening formula, adjusting feeding utensils or bottles, and prescribing medications to help with reflux, nausea or appetite.

“Some babies associate eating with negative past experiences such as frequent emesis or overall GI discomfort or intolerance,” Kirkland explained. “And malnutrition alone makes them less likely to eat, because they tend to fatigue earlier.”     

All of these issues can affect a patient’s ability to lead a normal life, even down the road. But Kirkland and Clifton are doing their best to smooth the path forward.

“Feeding is a very emotional experience for a lot of families. It’s also something that families just expect to occur—that everyone can eat and swallow and enjoy those experiences,” Kirkland said. “While they’re inpatient, we’re trying to get them home, and in clinic, we’re focused on the long-term goals of how this will shape their future.”

The Dysphagia Clinic strongly complements and reinforces the care these cardiac patients are getting at Children’s, Clifton said. “We’re looking at the big picture,” she said, “and making sure they get all the resources they need—the individual parts that might otherwise get forgotten or overlooked.”

Neurology & Neurosurgery

McRae Addressing PTSD in Kids With Hydrocephalus

Elizabeth McRae, Ph.D., is a psychologist embedded within the Children’s of Alabama Neurosurgery team.

The young child was beyond terrified of the hospital. Born with hydrocephalus, he’d had numerous surgeries, and his anxiety was so high that just getting him to the car for doctors’ appointments was a struggle. It could take an hour to get from the parking garage to the hospital entrance given his tantrums and refusal to walk. The behavior continued at home every time someone opened the front door. “The parents really couldn’t live their life because it was so intense,” said Elizabeth McRae, Ph.D., a pediatric clinical child psychologist at Children’s of Alabama.

Here was a clear case of post-traumatic stress disorder (PTSD) related to the boy’s illness. Resolving it is exactly what McRae, who joined the neurosurgery team in January 2024, was hired to do.

For years, neurosurgeons and families caring for children with hydrocephalus understood the physical stakes: shunts that could fail without warning, repeated surgeries, emergency trips to the hospital. What was less visible—and often unaddressed—was the psychological toll of living in constant vigilance both for the patient and the family.

Earlier work at Children’s helped bring that reality into focus, documenting high rates of medical post-traumatic stress among families coping with hydrocephalus. But identifying the problem was only the beginning.

“Based on the results of that previous survey, we brought Dr. McRae on board and embedded her in our neurosurgery practice to provide psychological support for PTSD from screening and diagnosis through interventions to getting people plugged in to community resources,” pediatric neurosurgeon Brandon Rocque, M.D., said.

What’s emerged is an integrated, trauma-informed model of care that treats psychological health as part of standard neurosurgical practice.

From Measuring Stress to Building Resilience

Families of children with hydrocephalus face a unique kind of uncertainty, McRae said. Even when a child is medically stable, the possibility of sudden deterioration and a need for a new shunt never disappears. That’s why resilience, which she defines as strengthening the ability of families and patients to view difficulties as challenges rather than barriers, is so important.

Whether she’s meeting a family for the first time at diagnosis or after a child’s 10th surgery, she starts from the same place: helping them identify strengths they already have that can enable them to cope.

McRae also emphasizes connection. “One of the key predictors of potential traumatic stress is feeling like we’ve lost power and feeling isolated,” she said. “So if, right off the bat, we can empower them and encourage a connection, to me, those are two of the best things we can do up front.”

“There’s also a big piece of how do we prevent the trauma?” she continued. One approach, she said, is “taking a trauma-informed approach to our service so we mitigate the risk on the front end.” That includes explaining what’s going to happen to children; giving them options and a sense of control whenever possible; creating a sense of structure and predictability in the hospital setting as much as possible; and relying on other services such as Child Life to help children cope and adjust through play. 

McRae works closely with the surgeons, nurses and residents in both clinic and hospital settings, participating in the morning clinical discussions. Nurses refer families they see struggling, and surgeons seek her input about how to provide trauma-informed care in communication and interactions with patients.

The clinical model emphasizes brief, targeted interventions—an intentional choice in a population already burdened by multiple appointments. “I’m doing them a disservice if I can’t do something fairly efficiently,” McRae said.

These strategies were helpful with the aforementioned young child. McRae worked with him and his mother using developmentally appropriate coping strategies such as play-based breathing exercises, predictable reassurance and gradual exposure. She had him pretend to be a snake and breathe in slowly and deeply like a snake to quell his anxiety. A scavenger hunt throughout the hospital helped provide distraction so he could become comfortable in the medical setting. And Rocque met with him dressed in his blue scrubs for a meet-and-greet, no medicine involved, since the child was usually so frightened by anyone in blue scrubs. McRae also involved the boy’s mother in the interventions, providing a greater sense of control over the situation.

The result? The walk from the car to the hospital takes just a few minutes. The tantrums in the clinic are over. His parents have space to breathe. All this was achieved over the course of just six, one-hour sessions.

Research is also a big part of the program, McRae said. To that end, she and the team are collecting data on how the model functions, including who benefits most, how referrals happen, what interventions are feasible, and whether the approach is sustainable.

“This is the first time anybody has tried to integrate psychology into pediatric neurosurgery like this,” Rocque said. “So there are so many questions that we need to answer.”

That includes developing screening tools to identify which families need support most urgently and tracking service metrics to ensure the model can be replicated.

“We really want to show that this works,” Rocque said.

Early signs suggest it is. The model has already been adopted in other specialty clinics, including tuberous sclerosis.

“Ideally,” Rocque said, “I would love for this to become the standard of care in pediatric neurosurgery.”

Nephrology

NINJA: A decade of preventing acute kidney injury

Acute kidney injury (AKI) is a common but under-recognized complication in hospitalized children. In non-ICU patients, the most common cause is due to nephrotoxic medications such as aminoglycosides, NSAIDs and vancomycin. It’s hardly a benign condition; even a single episode of AKI can set a child on a trajectory toward hypertension and chronic kidney disease later in life.

Traditional care often detects AKI only after it occurs—when creatinine rises. That’s reactive, not preventive. The NINJA (Nephrotoxic Injury Negated by Just-in-Time Action) initiative flips the script by identifying children exposed to high nephrotoxic medication—defined as three or more in one day or prolonged aminoglycoside/vancomycin use—and then screening daily for signs of kidney harm to prevent AKI.

Children’s of Alabama has been one of more than 20 children’s hospitals that are part of the NINJA project. “NINJA is designed to help prevent AKI by educating providers that AKI is not a benign condition, helping them identify high-risk patients, and providing them with the knowledge they need to prevent and mitigate medication-associated AKI,” Children’s pediatric nephrologist David Askenazi, M.D., MSPH, said.

Multi-center studies on NINJA show that collectively, institutions can reduce the rate of AKI by as much as 62%. Those reductions are attributed to the core tenets of NINJA: screening and stewardship; early detection through daily creatine monitoring; and a cultural shift toward preventive renal care.

“What we do is simple. We systematically identify patients at high risk of AKI and put them on the NINJA list,” Askenazi said. “Then, the pharmacist lead from each team ensures the team monitors the child’s creatinine levels daily and discusses possible alternative drugs with them.”

“Our pharmacists are the real ninjas of this program, and they deserve the credit,” he added.

After 10 years participating in the NINJA program, Children’s continues to see the number of AKI events drop. “It’s a testament to the dedication from the top down at Children’s to provide the best quality care to our children,” Askenazi said. He and his team are currently parsing the data to determine how many AKI episodes have been prevented. The number appears to be close to 1,000.

Now, the department is working with the hospital’s IT experts to integrate the NINJA system into the EPIC electronic medical record (EMR). This will enhance the clinician’s ability to see what’s happening in real time since patients on the NINJA list will be flagged immediately and a dashboard with all their medications and labs listed, Askenazi said. “We’re building data sets and clinical decision support tools for providers so they can navigate through NINJA and care for their patients with up-to-date data and suggestions.”

Until now, because pharmacists are not there on nights and weekends, at-risk patients could have been missed. With an EMR-based system, clinicians have access to the data 24 hours a day, 365 days a year. The EMR integration should also significantly increase efficiency for pharmacists and quality improvement specialists who previously had to manage the data on Excel spreadsheets and create reports manually.

In addition, a new urine test that can detect AKI without the need to prick the child for blood is being implemented. “We’ll incorporate that within our NINJA initiative,” Askenazi said, “as we continue to optimize patient comfort and safety throughout the hospital.”

Overall, he said, “NINJA has been successful because we’ve been able to convince everyone that preventing harm from nephrotoxic drugs is important.”

Gastroenterology

Expanding to Improve Patient Care

To improve patient care, the Children’s Division of Gastroenterology, Hepatology and Nutrition has added six new faculty members.

When Sandeep Gupta, M.D., arrived at Children’s of Alabama and the University of Alabama at Birmingham (UAB) as chief of the Division of Pediatric Gastroenterology, Hepatology and Nutrition in 2023, he immediately recognized the need for expansion. So, not long after his arrival, he embarked on the mission to grow his team. Within the last year-and-a-half, he has welcomed six new faculty, and they’re already making a difference in the way the division serves its patients.

For Gupta, that was the objective—providing better patient care. It’s a large undertaking considering Children’s massive catchment area—the entire state of Alabama along with surrounding states. “Every disease state we have is in the hundreds,” he said. “And the doctors we had in those areas were just one or two.”

Intestinal rehabilitation, or ‘short gut,’ is a good example. It’s a complex issue affecting more than 160 of the team’s patients. But previously, only one doctor was available to serve them. With inflammatory bowel disease (IBD), the team had two doctors for more than 400 patients. The math was similar for patients with liver issues. “There was such a need to bring [new hires] in to just serve what we have,” Gupta said. “We were simply to trying keep our heads above water.”

To address these issues, Gupta began the process of expanding the division in September 2024, making all the new hires over the course of the next year. In the case of IBD, the expansion was transformative. That group now has two new clinicians, Rahmath Althaf, M.D., and Maggie Vickers, M.D., and a basic scientist, Babajide Ojo, Ph.D. “Now, what we have is basically a team that goes from bench to bedside,” Gupta said. “We are starting studies where we are collecting samples from patients in the clinic, and then [Ojo] is processing these in the lab to do the studies. And with the discoveries he will make, we can then bring the knowledge back to the bedside.”

It’s an “ecosystem” that Gupta believes has not existed in gastroenterology at Children’s. The team has study coordinators and is part of national consortia. “So basically, we are creating a self-dependent and interdependent team of itself that can grow on its own,” he said.

Gupta aspires to create the same setup for intestinal rehabilitation. In the meantime, care is already improving. With Sirine Belaid, M.D., joining the team, the division now has two doctors to treat these patients, allowing them to see inpatients twice each week instead of once. “So now we are able to better serve the people more intentionally, more mindfully,” Gupta said.

Gupta also added liver doctors in David Willcutts, M.D., and Stephanie Saaybi, M.D., who “will helps us grow the liver team further,” he said.

Perhaps the most salient sign of the team’s success so far is this: patients who once had no choice but to go hundreds of miles away for treatment are now able to stay in Alabama for their care. “They are now being sent back to us by the doctors, who are saying, ‘Hey, UAB has a great program—go back there, you don’t need to come see us anymore,” Gupta said. “So that has been very fulfilling that people are recognizing we are ace-ing our game here.”

With changes of this magnitude, Gupta believes the division can start to move from excellence to eminence, which was another of his goals when he first arrived.

New hires since September 2024

Rahmath Althaf, D.O., an assistant professor, earned her medical degree from the Touro College of Osteopathic Medicine. She completed her residency in general pediatrics at the Medical College of Georgia and her pediatric gastroenterology fellowship at UAB. Her research and clinical interests include IBD and intestinal ultrasound.

Sirine Belaid, M.D., an assistant professor, earned her medical degree from the University of Pittsburgh. She completed her residency in general pediatrics at the University of Iowa Stead Family’s Children’s Hospital and her pediatric gastroenterology fellowship at the University of Pittsburgh Medical Center.

Babajide Ojo, Ph.D., an assistant professor, earned his doctoral degree in nutritional sciences from Oklahoma State University. He completed his postdoctoral research at Stanford University School of Medicine. He received the NIH MOSAIC K99/R00 award in 2023. His research interests include using patient-derived intestinal organoids and murine models to determine how the environment (dietary components, therapies) shapes epithelial metabolism and differentiation in intestinal health and inflammatory bowel diseases.

Stephanie Saaybi, M.D., an assistant professor, earned her medical degree from the American University of Beirut in Lebanon. She completed her residency in general pediatrics at MedStar Georgetown University and her pediatric gastroenterology fellowship at UAB. She completed an additional fellowship in pediatric advanced hepatology and liver transplant at Northwestern University.

Maggie Vickers, M.D., an assistant professor, earned her medical degree from UAB. She completed her residency in general pediatrics and her pediatric gastroenterology fellowship at the Le Bonheur Children’s Hospital, St Jude Children’s Research Hospital and Regional One Health. Her clinical interests include general gastroenterology, nutrition and inflammatory bowel disease.

David Willcutts, M.D., an assistant professor, earned his medical degree from the University of Texas Southwestern Medical Center, where he also completed his residency in pediatrics and his pediatric gastroenterology fellowship. He completed an additional fellowship in pediatric advanced hepatology and liver transplant at the University of Colorado.

Neonatology

How Children’s Neonatologists Are Playing a Crucial Role in Complex Surgeries

File photo of a surgical procedure at Children’s of Alabama.

In the Level IV Neonatal Intensive Care Unit (NICU), Children’s of Alabama cares for some of the sickest neonates in Alabama and the surrounding region. The unit, which expanded in 2025, frequently receives referrals from other hospitals for surgical and subspecialty care. Often, the babies cared for on the unit have conditions that require complex surgeries. In these cases, neonatologists work with surgeons and other specialists to manage the patient’s care. Jaw distraction is one example of a procedure they may manage.

Jaw Distraction Surgery and the Role of Neonatology

For some infants, a small or recessed jaw creates serious problems with breathing and feeding. Jaw distraction surgery—also called mandibular distraction osteogenesis—is an option that may dramatically improve outcomes for these patients. While the surgery itself is performed by craniofacial surgeons, the care before and after is deeply collaborative. Neonatologists play a central role in guiding babies and families through every step of the process.

Physicians typically consider jaw distraction surgery for young infants, often within their first month or two of life. Babies with the condition often work harder to breathe normally. They also may have difficulty feeding by mouth because the small jaw narrows the airway. If growth alone doesn’t lead to improvement, then a multidisciplinary team, including neonatologists, craniofacial surgeons, and often ear, nose, and throat (ENT) surgeons, evaluates whether jaw distraction could improve their symptoms and quality of life.

Surgeons focus on the technical aspects of lengthening the jaw, but before and after the operating room, neonatologists manage the airway, ventilation, nutrition and pain control. After the procedure, the baby returns to the NICU sedated and temporarily paralyzed. This protects the infant’s airway and allows the surgical site to heal while the jaw is gradually lengthened over several days. During this time, infants require ventilator support, careful airway management, pain control and nutritional support.

“This isn’t a procedure we recommend lightly,” Children’s of Alabama neonatologist Hannah Hightower, M.D., explained. “Protecting the airway is our number one concern for infants who undergo this surgery.” Because surgeons place hardware in the jaw, any movement could risk complications. This also means post-op babies cannot be held. This period can be especially hard on new parents who may have already begun holding and feeding their child.

As recovery progresses, collaboration becomes increasingly important. Neonatologists and surgeons work together to determine when it is safe to reduce sedation, remove the breathing tube and transition the baby off respiratory support. “Then, we begin working on the infant’s oral skills, because our ultimate goal is for the family to be able to feed their baby at home,” Hightower said. Many of these infants require tube feeding at first, but most eventually learn to feed by mouth.

Even in ideal cases, infants typically remain hospitalized for several weeks as they recover, learn to eat and prepare for discharge. Parents play a significant role as the baby’s recovery progresses, becoming actively involved in feeding and comforting their child. Success after jaw distraction is measured in practical, meaningful ways, such as improved breathing without respiratory support and the ability to take feeds by bottle.

Though the procedure can seem daunting, Hightower emphasizes its impact. Jaw distraction surgery can transform the course of an infant’s life—turning a situation marked by breathing struggles and feeding challenges into one where a baby can go home, breathe comfortably and be fed by their family. “It’s remarkable how a child who is working to breathe and unable to eat may improve and gain these skills post-surgery,” Hightower said. “Being able to send a child home doing those normal baby things is a big deal for the parents.”

Hematology and Oncology

Finding Novel Treatments for Cancer

The Developmental Therapeutics team at Children’s of Alabama is working to develop new treatments for children with cancer.

Having cancer is challenging enough for a child and their family, but when the standard treatments aren’t working, they find themselves searching for answers. As Director of Developmental Therapeutics at Children’s of Alabama and the University of Alabama at Birmingham (UAB), Jamie Aye, M.D., is responsible for finding those answers.

Aye was appointed to the position in August 2025 after serving as associate director since 2023. In her current role, she leads a team dedicated to developing more effective, less toxic therapies for children with cancer. In some cases, the cancers are rare; in other cases, the cancers are recurrent with no available curative treatment. Either way, developmental therapeutics provides hope for solutions.

“It provides the family with hope that we will discover newer options that their cancer hasn’t seen,” said Aye, who’s also an associate professor of Pediatrics at UAB.

The team achieves this through a two-pronged strategy. One prong involves working to develop new therapies for cases that are recurrent or difficult to treat. “It’s really hard to see a patient go through that,” Aye said. The other prong is developing less-toxic therapies that reduce immediate and long-term side effects—for example, “If we’re able to use a new immunotherapy in conjunction with radiation that allows us to give the radiation at a lower dose,” Aye said, “now that patient has a lower risk for a secondary cancer from radiation.”

The program has a history of success. A few years ago, Gregory Friedman, M.D., one of Aye’s predecessors, led a successful early phase trial that used a new type of immunotherapy to treat patients with recurrent or progressive brain tumors.  Friedman’s novel treatment was the result of an investigator-initiated trial, where the team takes the work its researchers are doing and turns it into a clinical trial. Developing these types of trials is the overarching goal of the program. And the research avenues are broad, Aye says, “from researchers within pharmacology, radiology, immunology or genetics,” to name a few. Aye works to bring these researchers together “so that hopefully we can see commonalities between the different projects that we’re working on, so that people aren’t siloed and can collaborate through either new technologies, new biomarkers to measure how much disease, new immunotherapies, or new ways of detecting disease by imaging,” she said.

Any type of research the team is doing has the potential to turn into an effective treatment. “It could even just be a pathway a researcher is investigating,” Aye said. “Hopefully through collaborations that we’re establishing, we can see how that pathway may relate to a particular cancer that we’re hoping to find a treatment for—and then from that, test a new therapy, and then if it works in the lab, seeing how we can get it to a patient.”

Collaborations are one of the keys to making these trials happen. As director of the program, Aye works to build relationships both near and far. Locally, she leads an annual research retreat to bring together researchers—both basic science and clinical researchers—to work on developing investigator-initiated trials using novel therapies. She developed this retreat during her time as associate director of the program. Nationally, she works with industry and pharmaceutical companies—to investigate new therapeutic options—and with consortia to add to the program’s portfolio of trials available to patients at Children’s and UAB.

Aye is already working toward more investigator initiated trials, “which, hopefully in the coming years, will come to fruition,” she said, to provide more options and more hope for children with cancer. “It’s why we do what we do,” she added. “Because we want to improve their outcomes and also find treatments that don’t leave them with lifelong side effects.”

Hematology and Oncology

King providing psychological help for patients with sickle cell

Kathryn King, Ph.D., leads a new program designed to help patients with sickle cell disease manage psychological challenges.

Pain is as much a part of sickle cell disease as white lights are a part of Christmas. It’s what lands kids in the emergency room and hospital; severely affects their quality of life; and leaves them constantly on guard lest it return.

Less visible—but just as real—are the emotional, cognitive, and psychological burdens that accumulate over years of living with the condition.

Now, Children’s of Alabama has a new program designed to address the non-physical aspects of the disease. Funded by a generous donor and developed by pediatric psychologist Kathryn “Kate” King, Ph.D., the program’s aim is to help kids with mental health issues related not only to their disease but to the stress of growing up in today’s world.  

“Our kids and teens with sickle cell experience a lot of different psychosocial issues,” King said. “But first and foremost, they’re kids and teens. There’s so much that comes up that’s not even related to their sickle cell. But then it ends up impacting their sickle cell.”

Like their peers, they experience anxiety, depressed mood, stress and the challenges of growing up. But layered on top is a disease marked by chronic pain, complex treatment regimens, frequent medical visits and, for some, cognitive effects related to the disease itself.

When Pain Becomes Chronic

Sickle cell pain is often thought of as acute flares that are treated and subside. But for many children and adolescents, King said, the pain becomes chronic. “It starts to become more frequent and the pain signaling becomes more like a faulty fire alarm,” she said, continuously going off even when there’s no smoke.

At that point, medication alone is often not enough.

So an important focus is boosting patients’ ability to cope with pain. That includes evidence-based strategies such as diaphragmatic breathing and guided imagery to calm the autonomic nervous system and reduce the intensity of pain signals.

And it works. Screening tools find patients’ pain scores dropping several points after using such strategies even before receiving pain medication, King said.

Equally important is helping kids and their families reframe the pain. Understanding when pain is severe enough to require escalation to the hospital and when it can be managed while continuing daily activities can reduce fear and help maintain normalcy.

Another focus is helping adolescents transition to self-management. This period during adolescence is known for high rates of treatment lapses and worsening outcomes as kids try to become more independent with this complex disease.

“They’re managing so many different treatments and medications” so adherence drops, King said. “It could be forgetfulness, because they can experience cognitive issues related to sickle cell, but sometimes it’s more about not wanting to feel different.”

King teaches her patients to use alarms and visual reminders to improve adherence, and she works with parents to help them understand when to hand over responsibility to their child. Some parents struggle to let go, she said, while others step back too quickly.

The goal is a gradual, scaffolded approach. “The parent can still function as the safety net, but the patient is progressively learning the needed skills to become their own safety net.”

King tries to see patients in conjunction with their medical appointments. She also follows them when they’re hospitalized and offers telehealth visits. With nearly 1,000 patients in Children’s sickle cell program and just one dedicated psychologist, however, demand far exceeds capacity. Currently, she sees about 20 patients a week, with plans to grow gradually.

“My goal is to extend to as many of those as I can,” she said. “But there’s just one of me.”

She’s also focused on tracking data for the program, including barriers and facilitators for care, as well as outcomes. She’d eventually like to publish on the program’s experience to help other hospitals start their own.