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Neonatal Consortium Advances Care for Patients with Rare Diagnoses

The Children’s Hospitals Neonatal Consortium (CHNC) works to improve patient outcomes. 

If you’re trying to make quality improvements, a good place to start is in the past. Historical data can hold the key to understanding what works, what doesn’t and what holds promise. But searching for answers in a small sample can be like panning for gold in a puddle. You need a stream of data to draw out the nuggets.

Prior to 2006, Children’s of Alabama was similar to most freestanding Children’s Hospitals: treating some of the sickest patients with unique conditions that didn’t always have textbook answers. Children’s neonatologist Tim Coghill, MD, was in the same position as his peers across North America; they knew there could be knowledge in numbers. With colleagues at 16 other Children’s Hospitals, he co-founded the Children’s Hospitals Neonatal Consortium (CHNC), an international group of level IV neonatal intensive care units (NICUs) that work together to improve patient outcomes. 

Now in its 16th year, the group consists of more than 40 top-rated Children’s Hospitals in the U.S. and Canada. All contribute to the Children’s Hospitals Neonatal Database, which allows them to compare a larger number of outcomes for complex patients and rare diagnoses and find the “gold” in their shared data.

Children’s neonatologist Allison Black, MD observes that, “We receive patients with rare diagnoses that you may only take care of once every 10 years. When you can pool the data on these patients with other centers, you can see if certain treatments and characteristics are associated with better outcomes and help formulate the best practices.”

One of the primary ways the CHNC establishes those best practices is through Collaborative Initiatives for Quality Improvement (CIQI) projects, such as STEPP-IN, a program aimed at improving neonatal surgical outcomes. The STEPP-IN initiative developed a standardized handoff and workflow process for patients being transported to and from the operating room. Through this process, the stability of infants during the time surrounding surgical procedures was greatly improved. Another quality improvement initiative, Erase Post-Op Pain, included an algorithm for managing and preventing pain after surgery in neonates. After initiation of these algorithms, the frequence of uncontrolled post-operative pain episodes following procedures decreased to less than 6% at Children’s of Alabama, a best in class outcome.  The CIQI projects often include clinicians, caregivers and specialists from various departments and disciplines throughout the hospital. Through the collaborations in these initiatives, there have been other permanent interdisciplinary teams established, such as the neurodevelopment care team, palliative care and infant feeding teams at Children’s. “Our involvement with the CHNC has helped foster a culture of collaboration, and this has helped us strive for a more well-rounded, multidisciplinary model when caring for our patients and supporting their families,” Black said.

Children’s involvement in the CHNC has provided the opportunity to help others seeking to improve care and create a collaborative environment in their units, as well. Hannah Hightower, M.D., presented at a national seminar highlighting her success with improving communications during high-stakes situations through a project involving debriefing after code events. In the past year and a half, Coghill and Black participated in two national workshops providing education to pediatric providers on difficult discussions and end-of-life decision making with families in the NICU. All three of these physicians have contributed to published manuscripts in the past year and have more in production through their involvement and collaborations in the CHNC. 

Outside of the QI projects, CHNC members also participate in an ever-expanding list of focus groups. Focus groups Children’s of Alabama participates in include:

  • Resuscitation
  • Discharge planning
  • Gastroschisis
  • Palliative Care and Ethics (PACE)
  • Neurosurgery
  • Micrognathia
  • Kidney and Urology
  • Necrotizing Enterocolitis
  • Genomics

These focus groups, said Black, “allow us to work together to develop the best practices for specific diseases.”  The greatest benefit of the CHNC is that patients get access to care that is constantly improving. “Through our involvement with the CHNC, we are lucky to be on the forefront of deciding how to best care for complex patients,” said Black. “We are constantly striving to gain knowledge and improve the care of our unique patient population, and in turn, our patients all benefit from this collective knowledge.”

September 5, 2022 by
Inside Pediatrics

Children’s of Alabama named top children’s hospital in the Southeast by U.S. News and World Report

Children’s of Alabama is ranked in 10 specialties in the latest U.S. News & World Report rankings.

Children’s of Alabama is part of a three-way tie as the top children’s hospital in the Southeast, according to U.S. News & World Report. The publication released its 2022-23 Best Children’s Hospitals Survey results in June. It’s the second year it has ranked hospitals by state and region. Children’s remains the No. 1 children’s hospital in Alabama; it was No. 3 in the Southeast in last year’s rankings.

U.S. News and World Report also ranks pediatric specialty services, and Children’s is ranked in the top 50 nationally in all 10 services. These include:

“We are proud of our people and programs being recognized on the national and regional levels, as we strive to provide the finest possible care to the children and families we serve,” Children’s CEO and President Tom Shufflebarger said. “This process is one of the important benchmarking activities we use at Children’s of Alabama to help us improve. I want to congratulate our employees, medical staff and many partners whose efforts made this possible in this challenging environment.”

This is the 13th consecutive year that Children’s has participated in the U.S. News and World Report rankings program and 13th consecutive year to be included in the rankings among the best children’s hospitals in the nation. Children’s and the University of Alabama at Birmingham (UAB) Departments of Pediatrics and Surgery collaborated to submit the requested information. Children’s is the primary site for pediatric clinical and educational programs for the UAB School of Medicine. Children’s has provided specialized medical care for ill and injured children since 1911, offering inpatient and outpatient services throughout Central Alabama. The complete listing and corresponding rankings for the magazine’s 2022-23 Best Children’s Hospitals is online at  www.usnews.com/childrenshospitals.

U.S. News & World Report introduced the Best Children’s Hospitals rankings in 2007 to help families of sick children find the best medical care available. Children’s and UAB began participating during the 2010-11 ranking cycle. The rankings offer families an exclusive look at quality-related information at the individual hospital level. To create the pediatric rankings, U.S. News & World Report gathers key clinical data from nearly 200 medical centers through a detailed survey that looks at measures such as patient safety, infection prevention and adequacy of nurse staffing. In addition, part of each hospital’s score is derived from surveys of more than 15,000 pediatric specialists who are asked where they would send the sickest children in their specialty. 

September 5, 2022 by
Endocrinology, Inside Pediatrics

High-Risk Diabetes Clinic Keeps Kids Out of the Hospital

Children’s of Alabama offers clinic to help high-risk diabetes patients.

The 2018 death of a teenage patient was a wake-up call for Children’s of Alabama pediatric endocrinologist Mary Lauren Scott, MD. The patient had type 1 diabetes and had been admitted numerous times over the previous few years with sky-high blood sugar. He died at home from diabetic ketoacidosis (DKA), a diabetic emergency in which the body breaks down fat for fuel because it doesn’t have enough insulin.

In response, Scott, who also has type 1 diabetes, took action. She and her team searched their data and found 10 patients in the previous year who had been admitted three or more times with DKA. Such admissions are a red flag that the patient isn’t taking their insulin doses at home and that they have a risk of dying or having a medical emergency they can’t recover from. 

“When you’re dealing with a chronic disease such as diabetes and patients have recurrent hospitalizations, we get burnt out too,” she said. “That makes it difficult to have the kind of positivity, motivation and drive to help them surmount the insurmountable and deal with what, for them, feels impossible. It’s hard, it’s complicated, and it’s not uplifting when you feel like you’re not able to help the patient.”

Scott, who was already directing a clinic for children and adolescents with type 2 diabetes, started another clinic in 2018 specifically for these high-risk patients. The primary goals of the clinic are to prevent hospitalizations and saving lives. The patients are all adolescents, at an age when they—instead of their parents—typically start handling their insulin injections and blood glucose tracking. 

The clinic meets half a day each month. The focus is on communication. Parents, social workers, nurses, nurse practitioners, diabetes educators and counselors work as a team to build trust with the teenagers. “We speak to them differently to get them to open up to us more than they perhaps ever have with other providers,” Scott said.

That means ensuring them there is no shame attached to missing insulin doses; what’s important is that they talk about it. “We tell them, ‘We need to know how to help you, so we can help you find success,’” Scott said. “We try to make the focus more about what’s working for [them] with [their] diabetes, what’s not working, and what [they] think would make things easier.” 

Patients must go six months without hospitalization before they graduate from the program.

The clinic is not just for patients, but for their families, as well. The patients are often drawn from minority and low-income backgrounds and have single parent households, Scott said. “The parent works and is also trying to manage a teenager who’s depressed or burnt out,” she said. “They can’t do everything.”

Because many of the parents have a history of interaction with child protective services, they rarely opened up to the staff about their child’s difficulties. With the new approach, parents are now more likely to talk about what’s happening at home and what they need help with. “That’s where we typically find success for these patients,” she said. 

The results have been impressive. 

  • 14 of the original 15 patients graduated. The one remaining is the youngest patient with significant home life issues. 
  • 8 of the 14 had a lower A1C when discharged. 
  • 6 of the 14 had no DKA admissions while in the clinic; all had three or more before joining the clinic. 
  • 12 of the 14 had fewer missed visits even though they came to clinic monthly instead of every three to four months.
  • Half had lower rates of DKA.
  • 12 of the 14 started continuous glucose monitoring while in clinic, and four also received insulin pumps, leading to a dramatic improvement in their health.
  • No patients died.

There are currently eight more patients enrolled, and more will be invited in the coming months, Scott said.

September 5, 2022 by
Endocrinology, Inside Pediatrics

PCOS Clinic Provides Holistic Care with a Multidisciplinary Approach

Doctor speaking with patient.

Excessive weight gain, acne, hirsutism, thinning hair, irregular menstrual cycles and insulin insensitivity –  these are some of the symptoms of polycystic ovary sydrome (PCOS), the most common reproductive condition in women and a risk factor for a plethora of metabolic diseases, including diabetes and cardiovascular disease, as well as infertility. It affects between 3% and 15% of women, who often wait years for a diagnosis, visiting numerous clinicians in the process.[i]

That’s changed for the young women of Alabama since pediatric endocrinologist Christy A. Foster, MD, started a multidisciplinary clinic in 2020 for adolescents with PCOS.

“Our goal is to try to improve the care patients receive in a more holistic manner,” she said. The clinic’s foundation is a multidisciplinary team that includes Foster, a pediatric gynecologist, a dermatologist, a social worker and family counselor, and a nutritionist. 

Many of these young women have been seen multiple times by their primary care doctor before they’re referred. “Coming to the clinic enables them to put a voice to what’s wrong—a feeling that they’re understood—which can be helpful,” Foster said. The multidisciplinary approach also provides expertise from several different providers. “That certainly helps with their care because otherwise they might have to make several different appointments and take more time to travel.” And most are just glad to have a home with providers who have expertise with PCOS. 

The physical manifestations of PCOS create a challenge at a particularly difficult time for young women, Foster said. “Their peers notice they ‘look different,’” she said. “That’s certainly a challenge for them from a mental health perspective.” That’s why it’s so important that the team includes counseling and mental health.

The Children’s clinic is one of only a limited number in the country, which is one reason Foster felt strongly about starting it. “I wanted to improve access to care in our region and address the patient as a whole person,” she said.

The team tailors treatment to address the patient’s greatest concerns, be it pre-diabetes, facial hair, severe acne or other repercussions of PCOS. “My hope is that when we start them on treatment, they feel their concerns are being addressed and heard and understood,” Foster said. Treatment options include birth control pills to regulate their menstrual cycles, an insulin sensitizer such as metformin, laser hair treatment and androgen receptor blockers. 

Another advantage of the clinic is that the team can follow the women longitudinally and, hopefully, prevent some of the metabolic conditions they’re at risk for.

After clinic, the team holds a debrief to discuss the patient as a whole, “There is a benefit to having everyone in the same place,” Foster said. “It improves communication.”

The clinic is held once a quarter, although patients may see individual providers at other times. Since it began, about 50 young women have been seen, with about six to eight seen during each half-day clinic. “But certainly we’re looking to grow,” Foster said.

[i] Gibson-Helm M, Teede H, Dunaif A, Dokras A. Delayed Diagnosis and a Lack of Information Associated With Dissatisfaction in Women With Polycystic Ovary Syndrome. J Clin Endocrinol Metab. 2017;102(2):604-612. doi:10.1210/jc.2016-2963

September 5, 2022 by
Inside Pediatrics, Neurology & Neurosurgery

Headache Checklist Supports Primary Care Providers and Helps Patients Find Relief Faster

The Children’s of Alabama’s PCP Headache Referral Checklist is helping doctors evaluate and treat headache patients.

Even kids get headaches. Lots of headaches. A U.S. survey found that 17 percent of children had “frequent or severe headaches” according to their parents, and that rate is thought to be as high as 30 percent among post-pubescent children.[i]

The UAB pediatric neurology group at Children’s of Alabama knows the numbers. Headache is the second-most common referral to their practice. In 2019, for instance, they received at least 40 referrals a month of children with headaches, and that number has skyrocketed since the start of the pandemic. And that, according to UAB Child Neurology Residency Program Director and pediatric neurologist Sarah Novara, MD, MSHQS, makes it difficult to effectively triage patients into available appointment spots. 

“Our goal is to best help these children be evaluated and managed, but we knew our referral process was not what it could be,” she said. At the same time, the neurology group also recognized community pediatric providers could help many of the patients they referred if they had the tools to succeed, freeing up neurologists to see those who required more complex care.

“It became apparent that we weren’t educating the community providers well enough so they would feel confident managing primary headache disorders in children and adolescents,” neurology faculty Scott Turner, DNP, said. That’s important because most headaches are not caused by a life-threatening condition like a brain tumor, which is exceedingly rare. 

Evaluating and treating headache patients as quickly as possible is critically important given that migraine, a very common headache type, is the second leading cause of disability in the world.[ii] Plus, recurrent headaches in children can significantly impair their quality of life, leading to missed school and missed opportunities for successful learning.

The pediatric neurology team embarked on a mission from 2019 to 2020 to better educate primary care providers about pediatric headaches and, in turn, create a better system of triage and communication to the pediatric neurology group.

The result is the PCP Headache Referral Checklist. With a large, red stop sign front and center, the message is clear: referring providers need to work through the brief checklist to check for headache red flags that should prompt a referral to the emergency department. These include:

  • Focal neurologic deficit 
  • Altered mental status, high fever and neck rigidity
  • Sudden severe headache (called a “thunderclap headache”)
  • Papilledema
  • Headaches that wake the child from sleep 

They also document special circumstances that may necessitate an earlier appointment with the neurology team, such as headaches that recur 15 times or more a month. 

The checklist also contains tools to help primary care providers manage their patients’ headaches while they wait for their visit with a specialist, including medication doses, a link to the Alabama State Department of Education school medication permission form, and a link to the Headache Relief Guide, an online portal specifically designed to help children learn about headaches and how to prevent and treat them. A QR code provides a link to the American Academy of Neurology and American Headache Society guideline for the acute treatment of migraine in children and adolescents. 

“The primary care provider has often done a very good workup of the child,” Novara said. “The checklist helps us see that and avoid redundancies.”

The team piloted the form with three Birmingham pediatric practices, where it proved highly successful for both the referring provider and neurology team triaging. “We didn’t know if they would go for it,” Novara said. “They’re busy, and they already have a lot of forms to fill out, but they loved it.”

“(Nurse practitioners) don’t get a lot of guidance or training in headache,” Turner said. And the time primary care providers can spend with patients is limited. The checklist, he said, “gives them the support to make decisions for themselves.”

Today, the checklist is required with the referral. A pediatric neurologist or nurse practitioner reviews it and triages the patient for appointments. Even before the child is seen by the pediatric neurology group, however, the primary care provider can start some of the interventions listed on the form. “This is empowering and also helps the family see that the referring provider is on the same wavelength as the pediatric neurology team at Children’s of Alabama,” Novara said. 

One important outcome is a better-quality referral, she said. “We have more information and are better able to care for the patient.”

In the meantime, the number of kids with headaches is increasing due to COVID. For some, recurring headaches are a lingering symptom of a COVID infection. In other cases, Turner said, parents held off seeking care during the height of the pandemic. Plus, the pandemic itself took a toll on children’s mental health. “When you completely turn a child’s life upside down, you’re going to end up with a lot of stress, which we know is a common comorbidity with chronic pain.”

In addition to the universal use of the headache referral checklist, the team is also reaching out to community providers through social media and hosting webinars and other educational activities about pediatric headache. “Primary care providers really seem to want this information,” Turner said. 

[i] Lateef TM, Merikangas KR, He J, Kalaydjian A, Khoromi S, Knight E, Nelson KB. Headache in a national sample of American children: prevalence and comorbidity. J Child Neurol. 2009 May;24(5):536-43. doi: 10.1177/0883073808327831. PMID: 19406755; PMCID: PMC2794247.

[ii] Powers, Scott W., et al. “Prevalence of Headache Days and Disability 3 Years After Participation in the Childhood and Adolescent Migraine Prevention Medication Trial.” JAMA Network Open, vol. 4, no. 7, American Medical Association, 2021, pp. e2114712–e2114712,

September 5, 2022 by
Hematology and Oncology, Inside Pediatrics

New BMT Program Director Focused on Expanding Immunotherapy

Joseph Chewning, MD, is the clinical director of the Pediatric Blood and Marrow Transplantation program at Children’s of Alabama.

As Joseph Chewning, MD, takes over as the clinical director of the Pediatric Blood and Marrow Transplantation (BMT) Program at Children’s of Alabama, he has an eye toward the program’s future, especially when it comes to immunotherapy.

It’s a field that Chewning says is growing very quickly. CAR-T cell therapy, in particular, is changing the paradigm for blood cancer treatment. With CAR-T, the child’s own immune cells are programmed to recognize and destroy a patient’s cancerous cells. Children’s became certified in 2018 to provide the therapy to children and young adults with recurrent acute lymphoblastic leukemia (ALL). As the new clinical director of the pediatric BMT program, Chewning wants to make sure Children’s can use CAR-T and other immunotherapies to help as many patients as possible.

“That’s really the goal for the program going forward—to continue to expand the novel treatments that we can provide for the children of Alabama, including cellular therapies,” he said.

One of the major advantages of immunotherapies is that, while they do have side effects, they’re typically less toxic than bone marrow transplant. For that reason, Chewning believes they’ll continue to become more common.

“At some point I think these cellular therapies will eclipse bone marrow transplant in usefulness,” he said. 

Chewning’s focus on immunotherapies is one part of his overall goal of providing the best quality care for patients in the safest way possible. 

“It’s really important to me that we fulfill the responsibility we have to the children of our state,” he said. Chewning wants to bring cutting-edge therapies to Children’s so families in Alabama won’t have to travel to get them.

“I’ve got four kids of my own,” he said. “I can’t imagine having a sick child who needs life-saving therapies and then having to separate from the rest of my family and go four or five states away.” 

In addition to serving as director of the BMT program, Chewning is the medical director for patient safety.

September 5, 2022 by
Hematology and Oncology, Inside Pediatrics

Solid Tumor Program Features Many Experts With One Goal

Elizabeth Alva, MD, directs the solid tumor program at Children’s of Alabama and the University of Alabama at Birmingham.

The pediatric solid tumor program at Children’s of Alabama and the University of Alabama at Birmingham (UAB) is made up of experts from several specialties who work together to achieve one goal: provide the best care possible for patients with solid tumors.

“There are so many advances happening today in pediatric oncology,” Elizabeth Alva, MD, director of the program, said. “It’s helpful to create a niche in which you have a team that’s knowledgeable with good expertise in that area.” 

The program is part of the Children’s hematology/oncology department. Alva is joined by fellow oncologists Jamie Aye, MD; Emily Johnston, MD; and Kimberly Whelan, MD. The team also includes advanced practice nurse practitioners and specialists from surgical oncology, orthopedic oncology, radiation oncology, palliative care and developmental therapeutics, as well as emotional support and psychosocial services.

One of the team’s objectives is to standardize care while still individualizing it. A key component of that is identifying the best clinical trials for their patients, something they do during their monthly “protocol” meetings. They also treat children with high-risk disease, such as neuroblastoma, with a comprehensive search of the literature to ensure everyone is up to date on the latest therapies and scientific findings, and then develop practice standards. “We’re making sure that we’re all focused on getting those patients the best care they can have,” Alva said.

The team also aims to increase participation in national consortiums of pediatric cancer centers to bring additional state-of-the-art care and innovative research to patients at Children’s. “Children’s and the University of Alabama at Birmingham are well recognized as a good center, but we want to continue to improve our national presence and our recognition as a top-notch program,” Alva said.

One area where that’s already happening is with the tumor xenograft project directed by Aye and pediatric surgeon Elizabeth A. Beierle, MD, in conjunction with others at UAB. Since the project’s inception in 2013, 175 patients have agreed to let the researchers implant their tumors into an animal model—a much more accurate way of studying these diseases. The goal is to better understand how the tumors behave and identify treatments that not only halt the cancer growth but have fewer side effects than current therapies.

September 5, 2022 by
Inside Pediatrics, Urology

New Chief of Urology Aims to Make Cutting-Edge Tech More Accessible to Alabama Families

Stacy Tanaka, MD, Chief of Pediatric Urology at Children’s of Alabama.

For pediatric urologist Stacy Tanaka, MD, it was an easy decision to accept the role of chief of pediatric urology at Children’s of Alabama. She already had relationships with several physicians here, including working on a spina bifida project with some of the pediatric neurosurgeons. 

She was also close with David Joseph, MD, who had been chief since 1986 but had decided to step down from the administrative role. “He’s so well respected in the pediatric urology community and urology community,” Tanaka said.

Coming to Children’s felt like a new but comfortable challenge. She started in January after 12 years at Vanderbilt Children’s Hospital in Nashville.

Tanaka’s vision for the department is to ensure that the children of Alabama rarely have to leave the state for pediatric urology services. And that means bringing in more technology. “I don’t want families of kids who need our services to go elsewhere because they think we can’t provide it.”

One area is robotic surgery. Laparoscopic surgery has revolutionized the field, but it’s challenging. “When you’re doing conventional laparoscopy, it’s a little bit like putting a fork through one hole and a knife through the other and trying to cut your steak,” she said. “With the surgical robot, there is more freedom of movement, making it closer to using your hands,” she said. 

Currently, the Children’s team can access a surgical robot at the University of Alabama at Birmingham, but UAB is not a children’s hospital. So, with Dr. Tanaka’s arrival, Children’s will soon have its own robot.  Having robotic surgery at Children’s would help keep families here, Tanaka said. “Your care is going to be as good or better than anywhere else you go, and you don’t have to travel.”

September 2, 2022 by
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.”

September 2, 2022 by
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.

September 2, 2022 by