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Inside Pediatrics, Neurology & Neurosurgery

Birmingham to Cape Town: Children’s Neurologist Consults Across Continents

Leon Dure, MD, neurologist trained in pediatric movement disorders at Children’s of Alabama.

It’s not easy to find a specialist trained in pediatric movement disorders. At Children’s of Alabama, there are two: Leon Dure, MD, and Emily Gantz, DO. They and their multidisciplinary team provide the bulk of care for children with movement disorders in Alabama and beyond. 

Considering the dearth of movement disorder specialists here in the U.S., Dure wondered what the numbers were like in Africa. His curiosity led him to become a consultant to a pediatric neurologist in Cape Town, South Africa. 

Dure’s interest in helping physicians in under-developed countries was piqued through his work with the International Child Neurology Association (ICNA), which seeks to foster education and resources for practitioners in low-resource environments, such as South Asia, Africa and South America. 

He recalls a meeting where a doctor from sub-Saharan Africa presented. “She said there are 80 million children under the age of 18 within that region and two or three neurologists,” Dure said. “So, I realized that the types of problems and issues they’re facing are very different from what we face here.”

In the U.S., Dure often receives requests from colleagues for his opinion on a child, typically via a video sent by text. Why, he wondered, couldn’t the same be done for clinicians in low- and moderate-income countries?

Turns out it wasn’t quite so simple, given the challenges of moving information from places as disparate as South Africa and Birmingham. Dure persevered and found vCreate, a tech company that works with the U.K.’s National Health System to provide secure video messaging. There is even a separate unit called vCreateNeuro, a cloud-based service that allows registered patients and clinicians to securely share smartphone-recorded videos. 

He and vCreate developed a proof-of-concept project with a neurologist who has an interest in movement disorders at the Red Cross Hospital in Cape Town. When he has something he wants Dure to review, the neurologist uploads it to the vCreate platform, and Dure gets an email alert. 

“Then we begin a back-and-forth regarding what to call it, what to do about it, how to work it up, et cetera,” he said. 

The pilot has been in place for about six months, and Dure has consulted on about eight videos. 

“These are relatively unusual conditions that are very difficult to characterize,” he said. “So just having someone else say ‘Yeah, you got it, you’re right,’ is quite helpful.”

Dure would like to grow the program to other countries with other U.S.-based pediatric neurologists providing their expertise, but that will require funding. For now, vCreate has been providing the technology for free. “I don’t know if that’s going to be a long-term possibility,” he said. “But so far, it works. And I’m able to provide my expertise to somebody in Cape Town without a whole lot of effort.”

Cardiology, Inside Pediatrics

Xenotransplantation Takes Steps Toward Clinical Trials

Children’s of Alabama cardiothoracic surgeon David Cleveland, MD, MBA, leader of the xenotransplantation program at Children’s of Alabama and the University of Alabama at Birmingham (UAB). 

News that surgeons at the University of Maryland Medical Center had implanted a genetically modified pig’s heart into a human rocked the medical world earlier this year. But it didn’t surprise Children’s of Alabama cardiothoracic surgeon David Cleveland, MD, MBA, who is leading a similar xenotransplantation program at Children’s and the University of Alabama at Birmingham (UAB). 

The program focuses on developing genetically modified solid organs from pig models for transplantation. To date, Cleveland’s team has successfully transplanted a genetically modified pig kidney into a brain-dead patient. The kidney produced urine.

Three years ago, Cleveland presented preliminary results from a study showing little reactivity in an infant’s blood to cells from a triple-knockout (TKO) pig. The pig had been genetically modified to delete the three major antigens that react with natural human anti-pig antibodies. Even those human cells that did react demonstrated a very mild reaction.

Back then, Cleveland said the next step was a transplant in a non-human primate, something required before the FDA would approve human trials. 

Now he’s done it. So far, Cleveland and his team have implanted four infant baboons with the genetically modified pig hearts, with one of the animals living as long as eight months. In humans, the goal isn’t to have the heart last a lifetime but, rather, just long enough for a human heart to become available for transplant. 

“I think we have to consistently demonstrate a four-to-six-month survival in non-human primates before the FDA approves a clinical trial,” Cleveland said. The team plans to implant more animals with the hearts this fall and is working on several grants to continue funding the study. Cleveland hopes to be able to submit the design for a clinical trial to the FDA sometime in 2024. In the meantime, he and his team published the results of the first baboon study in The Annals of Thoracic Surgery.

It’s quite possible, however, that the first transplant might occur outside a clinical trial with a humanitarian device exemption from the FDA. The authorization allows a device—in this case, the pig heart—to be used without showing effectiveness in formal clinical trials. That’s how the patient at the University of Maryland Medical Center was able to receive his heart.

“But our goal, ultimately, is to participate in an NIH-funded clinical trial,” Cleveland said, adding that those trials are conducted in more patients with strict safety monitoring and comprehensive data collection.

Inside Pediatrics, Nephrology

With Parental Training, Children Undergo Home Dialysis While They Sleep         

The team at Children’s of Alabama’s Renal Care Center works hard to transition children and their families to home dialysis

Going to the hospital three to five times a week and being hooked up to a machine for hours at a time is no life for a child. Yet that’s exactly what children with end-stage kidney disease waiting for a transplant spend their time doing. Which is why the team at Children’s of Alabama’s Renal Care Center works so hard to transition children and their families to home dialysis. Currently, of the 30 children they have on dialysis, 17 are able to get their treatments at home.

“In general, home therapy is best for children,” said pediatric nephrologist and Renal Care Center director  Sahar Fathallah-Shaykh, MD. Since the dialysis is typically performed while the child sleeps, they miss less school and have more free time for friends and family. Plus, parents don’t miss work. Still, she said, “We understand that some parents don’t want to do it because it’s very stressful to be responsible for it.”

If parents do want to try home dialysis, the first step is a home visit from a social worker or dialysis nurse. They ensure there is enough space for the machines with the correct electrical outlets and an environment that doesn’t increase the risk of infection, which would require hospitalization. 

Next, the family meets with a dialysis coordinator to learn what’s expected of them. “We make sure we tell them everything ahead of time,” Fathallah-Shaykh said. Finally, the medical team holds a home dialysis selection meeting, where they decide if home dialysis is the right choice for the patient. If it is,  the next step is surgery to implant the catheter needed for home therapy.

Meanwhile,  caregivers undergo extensive training. “It’s a very standardized process to ensure they understand not only how we do things, but why,” Fathallah-Shaykh said. Before the child goes on home dialysis, they spend a couple days in the hospital, where the parents perform dialysis under the watchful eye of the dialysis nurses. “We make sure they’re doing it right, and they have no hesitancy or issues with doing it at home,” she said. When both parents and the Children’s team are comfortable, patients are sent home. “We’re here for them all the time,” Fathallah-Shaykh added.

There are two types of home dialysis: peritoneal dialysis, which uses the lining of the abdomen to filter blood inside the body through a catheter, and hemodialysis, a more complex procedure that requires the caregiver to insert two needles into the child’s fistula or graft, so blood can flow from the body to a machine where it is filtered and then sent back into the body. Children’s is one of only three pediatric centers in the country that train families to do hemodialysis at home. To date, the Children’s Renal Care Center has placed three children on home hemodialysis. 

Although hemodialysis has gotten easier in recent years with machines specifically developed for the home that are easier to set up, clean and disinfect, it’s still invasive. “And they have to deal with blood, which is a lot for some families,” Fathallah-Shayk said. “So we have to have motivated families to do that.” 

Hemodialysis training typically lasts several weeks, while peritoneal training may take only a few days or a week. Regardless of which process is used, she said, “We take our time to make sure families are comfortable doing it.”

Inside Pediatrics, Nephrology

Lecture Series helps Pediatric Nephrology Fellows Learn and Gain Confidence

Michael Seifert, M.D., is a pediatric nephrologist at Children’s of Alabama and an associate professor in the Division of Nephrology in the University of Alabama at Birmingham Department of Pediatrics.
Michael Seifert, MD, is a pediatric nephrologist at Children’s of Alabama and an associate professor in the Division of Nephrology at the University of Alabama at Birmingham.

At any given time, the nephrology department at Children’s of Alabama is training three fellows, one for each year of its three-year program. The program is one of the best in the country, but one thing it lacked until recently, according to pediatric nephrologist Michael E. Seifert, MD, was a formalized educational curriculum. 

To fill that need, the department started a weekly lecture series in July 2021. It runs throughout the academic year, and lectures focus on topics that correspond to content themes in the American Board of Pediatrics Pediatric Nephrology Certification Exam. The goal is to educate the fellows about topics they will encounter frequently—such as hypertension—or topics most fellows would have had limited experience with as pediatric residents—such as dialysis and kidney transplantation. 

It’s not the first time the department has offered lectures for fellows; the difference now is that it’s a formalized program.

“We always had some faculty give lectures for our fellows but had never organized a formal didactic curriculum for our fellowship program,” said Seifert, who’s the associate program director for the Pediatric Nephrology Fellowship program at the University of Alabama at Birmingham (UAB). “We often sent our fellows to formal lectures delivered through the adult nephrology program.  While these are high quality, they were often not relevant for pediatric nephrology.” 

In the new program, Division of Pediatric Nephrology faculty give the majority of the lectures. Occasionally, speakers also include guests from other divisions who speak on topics that might relate to kidney issues. For example, a urologist might speak on bowel and bladder dysfunction; a rheumatologist might provide a broader view of lupus, which often affects the kidney.

The fellows are also encouraged to prepare and deliver their own talks to develop some of their presentation and public speaking skills in a friendly environment. “They really appreciate that opportunity to hone their skills in a relatively low-risk setting,” Seifert said.

The didactic lectures are interspersed with journal clubs and case discussions. “We had a fellow recently who did a great presentation of a complex case that he was taking care of and how it spurred a quality improvement project,” pediatric nephrologist Erica Bjornstad, MD, said.

“We’re really trying to help build their confidence in clinical pediatric nephrology but also in how we can take that further beyond just clinical education to spur their research or quality improvement ideas,” she said. 

Inside Pediatrics, Nephrology

Children’s Clinicians Track COVID’s Surprising Impact on the Kidney

Erica Bjornstad, MD, is a pediatric nephrologist at Children’s of Alabama.

What is the impact of COVID-19 on the kidney, both in adults and children? That’s the question Erica Bjornstad, MD, and others have been studying since the pandemic started. Their recent paper, published in BMC Nephrology, provides the first data demonstrating the risk of acute kidney disease among the entire age range, from newborns to 99-year-olds. The findings, Bjornstad said, were surprising.

“We all know that the severity of COVID is worse the older you are, and we’re not disputing that,” she said. “But what we found is that risk of acute kidney injury, or AKI, does not go in a straight line.” Instead, it has a bimodal distribution, with the risk peaking in children between ages 10 and 15, decreasing until about 30, then continuing to rise in a more linear fashion. The study was based on the records of 6,874 patients hospitalized with the virus.

“Why does a 10-to-15-year-old have the same risk of acute kidney injury as a 65-year-old, but a higher risk than a 35-year-old?” she asked. The higher risk in children held even after accounting for those with chronic medical conditions.

Bjornstad and her coauthors hypothesize that it may have to do with how the virus attacks the endothelium, the lining of the blood vessels, although that doesn’t explain the effect in adolescents. “Maybe there are hormonal changes happening during puberty that make them more susceptible to kidney injury risk. We really don’t know why. All we can say is that the epidemiological data shows it exists, and we need to study that further.”

The study also found a high rate of AKI in non-critically ill patients—those who were not in the ICU—something that previously had not been reported. However, the children who do get AKI with COVID are generally very sick and hospitalized, she said. “I haven’t seen children come to my attention from the outpatient side who got COVID, were never hospitalized, and now have kidney problems,” although there have been very rare cases elsewhere.

The findings are important for clinicians who treat children, adolescents and younger adults, she said. “For the most part, COVID is benign in kids. But we have to have a higher index of suspicion for acute kidney injury in this population.”

“If something doesn’t feel right, or the child isn’t acting right, even after very mild cold-like symptoms, we should run some blood tests to check for the injury resulting from the endothelial attack,” she said. The concern about AKI comes because it increases the risk for high blood pressure and chronic kidney disease.

Bjornstad and other nephrologists at Children’s also published a review article in Pediatric Nephrology last year highlighting all that was known to date on the impact of COVID on the kidney in pediatric patients. The paper included several clinical pearls for clinicians. For instance, early in the pandemic there was concern that anyone with COVID taking an ACE inhibitor should stop. “But there’s really good data that if you have a child with COVID on an ACE inhibitor they should not stop it,” she said.

Their papers and others on the topic generally end with the same call for more research. “There are some immune dysregulation syndromes that affect the glomerular part of the kidney that is presumably triggered by COVID,” Bjornstad said.  “But we need to keep looking to see if there are other mechanisms.”

Inside Pediatrics, Nephrology

Children’s Clinicians Teach the Rest of the World How to Start a Neonatal Dialysis Program

Pediatric nephrologist David Askenazi, MD.

It’s always wonderful when something you created exceeds your expectations. That’s what’s happened with the Neonatal and Infant Course for Kidney Support (NICKS), a one-and-a-half-day educational program on infant dialysis that combines didactic teaching from a variety of specialists, an opportunity to hear a parental perspective, “hands-on” skills sessions, and virtual small group case simulations.

Pediatric nephrologist David Askenazi, MDco-founded the course in 2019 with acute dialysis nurse practitioner Kara Short, MSN, CRNP, after new technology enabled safer and more effective dialysis on neonates and small children. The two realized there was a huge need for clinicians to understand how dialysis is different for this population. 

The program was supposed to be held in person, but COVID drove it online, which worked to their benefit by making the course more accessible. They’ve held nine courses so far, all sold out with large waiting lists (they try to limit it to 50 a class). “We don’t just talk about the dialysis machines,” Askenazi said, “but also clinical scenarios, troubleshooting, medications, nutrition, educating the nursing staff and tracking quality improvement. Participants learn how to build a program, not just run a machine.”

To date, 359 clinicians have attended the training, including 120 nephrologists and 52 neonatologists, as well as nurses and nurse practitioners. Attendees have logged on from as far as Qatar, Israel and South Africa, and nearly every major children’s hospital in the country has had at least one attendee. 

“We find it so important to share our patient stories and our lessons learned the hard way in order to empower other programs to confidently treat their patients and hopefully help babies like we have,” Short said.

The course will begin live sessions this fall, with a limit of 20 participants. Askenazi and Short are also planning an international neonatal nephrology symposium for fall 2024.

For more information, contact David Askenazi, MD, MSPH, FASN, at daskenazi@peds.uab.edu or Kara Short, MSN, CRNP, at kara.short@childrensal.org.

Hematology and Oncology, Inside Pediatrics

Hope and Cope Program Helps Families Navigate Medical Journeys

With the diagnosis and treatment of childhood cancer or serious blood disorders like sickle cell disease comes an emotional rollercoaster for the child and the family. From practical issues including getting to appointments, caring for siblings, and managing the costs of care to psychosocial issues such as anxiety, fear, grief, and even anger, there are needs beyond the medical support physicians and nurses can provide. That’s where Children’s of Alabama’s Hope and Cope Psychosocial and Education Program comes in. The program provides emotional support and services using a family-centered approach in which the family and healthcare providers work together to best meet the needs of the entire family. 

“It is becoming more and more important to provide these services to families dealing with cancer and serious blood disorders,” program director Avi Madan-Swain, PhD, said. “Even if we cure the disease medically, for some the cost of cure is high and results in neurocognitive difficulties as well as psychological difficulties, such as post-traumatic stress disorder.” 

To reduce stress during hospitalizations or clinic visits, the Hope and Cope Psychosocial and Education Program offers inpatient schooling (either group or bedside); art, music, and animal-assisted therapy; parental consult service; a weekly inpatient caregiver dinner support group; and expressive activities such as drumming, drama, and journaling. 

The program’s education/school liaisons help patients transition back to school, maintaining communication between the medical team, the child’s school, and the family and providing education to schools and families about special services the child may need. This includes classroom presentations to help classmates understand the child’s diagnosis and treatment. 

Once a child is diagnosed with cancer or a serious blood disorder, families complete a screening questionnaire to help the team identify immediate needs. The team shares the results with the family and initiates evidence-based interventions to address identified needs. The overall goal is to prevent crises by intervening early rather than being reactive. The team then meets weekly to discuss the psychosocial needs of newly diagnosed patients and those who have relapsed and need close monitoring. They also talk about any difficulties the patient/family may be experiencing in the inpatient unit or outpatient clinic.

“We’re building wellness and resiliency,” Madan-Swain said. “We’re not just looking at family risk factors, but also their strengths, and developing plans to support young people and their families to manage the challenges of their medical journey, make the most of their strengths, and ultimately celebrate life.”

A new initiative is strengthening psychosocial service delivery for parents of children undergoing bone marrow transplant, Madan-Swain said. “I’ve been working to systematically standardize parent assessment and education and providing psychosocial resources because of the intensity of the process. It’s like being in a pressure cooker.”      

One thing that sets Hope and Cope apart from similar initiatives at other children’s hospitals, according to Madan-Swain, is its bereavement component. Services begin when the disease is no longer treatable and continue for two years after a child’s death. All family members work with art and music therapists to create a “legacy piece,” such as a painting, sculpture, or music. For instance, the music therapist may record the child’s heartbeat and place it in a bear for the family. “We found that families who work on meaning-making activities cope much better after the loss of the child,“ Madan-Swain said. “This has really pushed us in the direction of making sure that we’re not waiting until the very end to create a legacy piece.”

Despite the success of the program, Madan-Swain is not resting. “I always want to move on to developing psychosocial care pathways based on family risk level and providing evidence-based interventions and expressive activities to improve adjustment and quality of life for both our oncology and hematology patients and their families,” she said. 

Endocrinology, Inside Pediatrics

Managing Congenital Adrenal Hyperplasia With Precision

Nurse practitioner Leslie Pitts (left) and Dr. Gail Mick, director of the Children’s of Alabama endocrine newborn screening program.

Congenital Adrenal Hyperplasia (CAH)—a group of rare genetic disorders affecting the adrenal gland—occurs in approximately 1 in 16,000 children each year, and these rare cases present significant challenges for patients, parents and doctors. Without proper treatment, it can be a life-threatening condition, but treating it can be difficult for doctors. That’s why the endocrine team at Children’s of Alabama is participating in an international pediatric, phase 2 trial called CAHtalyst to investigate a new nonsteroidal treatment.

The treatment, Crinecerfont, may improve adrenal hormone balance by tamping down excess androgen production. The drug has already shown efficacy and safety in adult CAH trials, and Gail J. Mick, MD, director of the Children’s endocrine newborn screening program, says she’s delighted that Children’s can participate in the study. “The families are excited about new CAH research because they want new therapeutic options,” she said. 

CAH, which can occur in multiple forms, affects the adrenal gland’s ability to produce cortisol, a hormone critical for life; and aldosterone, a hormone that regulates salt and water balance. The most common form of CAH occurs due to a deficiency of the adrenal enzyme 21-hydroxylase. Without this enzyme, affected individuals are at risk of a life-threatening adrenal crisis as well as adrenal androgen overproduction. 

Doctors in the Children’s of Alabama’s endocrine newborn screening program specialize in helping newborns with CAH, which is one of more than 30 disorders screened for at birth by Alabama’s newborn screening program. At Children’s, newborns who screen positive for CAH are immediately evaluated for emergency care and management. To help parents and children with CAH, the Children’s Division of Endocrinology and Diabetes provides comprehensive care to nearly 100 CAH patients per year.

Treatment involves infusions of steroids and fluids, followed by carefully titrated doses of cortisol and mineralocorticoids. It’s not always easy. “It’s a very delicate balance to mimic normal adrenal function with oral medications,” Mick said. Give too much glucocorticoid, and you can suppress growth and expose patients to adverse steroid side effects. Give too little, and the adrenal gland overproduces androgens, which can spur early puberty and excess growth as well as other complications.

The adrenal imbalance is even more complex when children are sick and can’t take their medications. In those cases, parents must administer injectable glucocorticoids and seek emergency medical care. For this reason, routine childhood illnesses put a tremendous strain on parents. 

Extra support is essential, Mick says. CAH is challenging to manage, given individual nuances in adrenal insufficiency and the impact of growth and puberty on medical management.  “We offer special teaching and support for the parents in those early years as far as understanding how and when to give steroids and hormones to prevent adrenal crisis,” said nurse practitioner Leslie Pitts MSN, AC-PNP, CDE. 

The CAH and endocrine newborn screening clinic also serves as an important resource for pediatric providers throughout the region. “Providers consult with us when they have abnormal newborn screens on their patients,” Pitts said. “They know we’re available to answer questions and concerns regarding CAH diagnosis and management.”

Pitts and Mick stress the key role that parents play in managing their child’s CAH. “Hats off to the families of children with CAH,” Mick said. “They are a motivated community who support one another, advocate for CAH research and treatment, and have even put together a highly popular ‘Camp Cortisol’ at Children’s Harbor at Lake Martin.” 

“These are really quite extraordinary families,” Pitts said. And they may have a brighter future thanks to clinical trials like CAHtalyst. Mick predicts that in the next couple of years, they’ll have access to many more treatment options that will make the condition easier to live with.

Cardiology, Inside Pediatrics

Pea-Sized Device Proves Lifesaving for the Tiniest Babies

Mark Law, MD, and William McMahon, MD, are pediatric cardiologists at Children’s of Alabama.

Imagine a baby who weighs less than two pounds. Now imagine that baby has a life-threatening, congenital heart condition called patent ductus arteriosus (PDA), in which the opening between the two blood vessels leading from the heart hasn’t closed. Now imagine performing open-chest surgery on that tiny neonate. 

“Patent ductus arteriosus is a significant problem for many premature babies,” pediatric cardiologist William S. McMahon, MD, said. Usually, however, it closes on its own. That’s not the case in micropreemies, defined as babies born before the 26th week of pregnancy or weighing less than 28 ounces (700-800 grams). 

PDA affects up to 80% of micropreemies, keeping them on ventilators longer and increasing the risk of lung disease. It can also lead to gastrointestinal problems and affect neurological development. That’s why closing the opening is critical. 

Yet the risks of operating on such a tiny preemie are nearly as great as the risks of PDA, said pediatric cardiologist Mark Law, MD, so few surgeons perform the surgery. 

“It’s a hard decision to make because many babies will survive with the PDA, or it may get smaller,” he said. While there is medication available to treat PDA, it’s often unsuccessful and has its own risk of kidney complications. Still, given the risk of open-chest surgery in such a small baby, “many surgeons would choose to continue to manage the baby medically,” Law said.

That changed in 2019 with the approval of the Amplatzer Piccolo Occluder, a pea-sized device that a cardiologist can snake through the femoral vein and into the heart via a catheter. “With this device, we now have the opportunity to intervene in a minimally invasive fashion with much less insult to the baby and a much lower risk of serious complications and still get the PDA closed,” McMahon said. 

The occluder also allows them to avoid the artery, which they use to close PDAs in full-term or larger premature babies. In such tiny babies, however, putting a catheter through the artery could cause significant damage. “It’s part of the miracle of this device that we don’t have to access the artery,” McMahon said. “It’s much safer going in through the vein.”

Unlike most catheterization procedures, implanting the occlusion device requires a team of specialists, including neonatology, the cardiac catheterization team and anesthesiology, among others. “They’re fragile babies at risk just for the exposure and movement,” Law said. To date, the team has implanted the device in more than 20 babies.

“Not only will the device keep more of these tiny babies alive,” he said, “but, hopefully, it will result in more micropreemies who get to the end of the first year of life with fewer difficulties like chronic lung disease, GI tract disease and neurodevelopmental problems.”

“Any little thing that you can help these babies do better in the first year of life, so they are less dependent on medical technology and even less damaged from the care required to keep them alive,” McMahon said, “ends up in a better baby in the future.”

Cardiology, Inside Pediatrics

Which Device is Most Effective at Treating Cyanotic Congenital Heart Defects?

Mark Law, MD, is a pediatric cardiologist at Children’s of Alabama.

Doctors at Children’s of Alabama are working to answer a question that could help save the lives of infants with cyanotic congenital heart defects. In a new study, they’re asking which device is better: the Blalock-Taussig-Thomas (BTT) shunt or the ductus arteriosus (DA) stent. 

The study is called the COMPASS Trial, short for Comparison of Methods of Pulmonary Blood Flow Augmentation in Neonates: Shunt Versus Stent. Children’s pediatric cardiologist Mark Law, MD, is the leading institutional investigator for Children’s.

“This is a very interesting study for our field,” Law said. “We have very few randomized controlled trials to show us which treatment strategy is best. There’s some thinking that DA stents may require additional, non-emergent interventions after they are implanted, and some data suggests the shunts may require a higher number of emergent interventions early on. We just don’t know.”

Of the two devices, the BTT shunt has been around longer. Developed in the 1950s, the shunt was the subject of a made-for-TV movie called “Something the Lord Made.” At the time, the shunt was lifesaving for babies with Tetralogy of Fallot. Today, it is used to manage babies with various heart defects that result in cyanosis, which causes insufficient blood flow from the heart to the lungs, resulting in low oxygen levels. These infants are often referred to as “blue babies.” 

“It was field-changing when it was developed,” Law said of the shunt. However, it also requires open-chest surgery, which can lead to more complications and a higher risk of death.

DA stents, developed more recently, a much less invasive. A stent can be threaded into the ductus arteriosus, the artery that connects the aorta and pulmonary artery in newborns. Once in place, the stent is expanded to prevent the DA from closing.

Both the BTT shunt and the DA stent provide a stable source of blood flow to the lungs until the baby is large enough for an operation to repair the heart defect. Both options are relatively safe and effective, but both also present the risk of complications and death. 

No one knows which is better because, until now, there’s never been a large study directly comparing the two procedures. The COMPASS Trial is a multi-institutional, NIH-funded study with the Pediatric Heart Network to see if one is safer and more efficacious than the other or if they are equally effective. 

Investigators hope to enroll 300 newborns over the next two years and follow each through the first year of life. Most centers, including Children’s, should start enrolling patients this fall.

Overall survival and post-surgical complications are key endpoints for the study, as well as hospital length of stay and quality of life. Investigators also hope to learn which approach is best based on the child’s anatomy.

“We hope to come out the other end smarter and knowing which is the best therapy,” pediatric cardiologist William McMahon, MD, said. “Because right now, we just don’t know.”