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Endocrinology

Addressing Endocrine Issues in Cancer Survivors

Dr. Nicole Barnes’ clinic tackles the many endocrine-related challenges cancer survivors can face.

When pediatric endocrinologist Nicole Barnes, M.D., started at Children’s of Alabama in December 2021, it made sense for her to begin a clinic designed specifically for childhood cancer survivors, who often experience endocrine issues from their cancer or treatment. Barnes came to Children’s from St. Jude Children’s Research Hospital in Memphis, where she specialized in survivorship care.

“About 50% of children who are cancer survivors have at least one endocrine disorder,” she said. These endocrinopathies include growth hormone deficiency, disorders of puberty, thyroid abnormalities, poor bone mineral density and metabolic disorders. Some survivors are also at risk for infertility. Barnes started the Endocrine Care for Children with Cancer Clinic to address all these issues.

“Primary care physicians may not be accustomed to identifying endocrinopathies associated with survivors’ treatment exposures,” Barnes said. Even general pediatric endocrinologists may not be familiar with the clinical guidelines published by the Children’s Oncology Group for monitoring childhood cancer survivors. “We’re taught in training about cancer-related endocrine disorders, but until you’ve treated multiple children with cancer you may not fully appreciate the impact endocrine disorders can have,” she said.

One reason is the subtlety of the signs and symptoms. “For instance, a premenarchal 13-year-old female may not be concerning in the general population,” Barnes said. “However, a premenarchal 13-year-old female childhood cancer survivor exposed to total body irradiation and alkylating chemotherapy agents is at high risk for ovarian failure and should be monitored and treated accordingly.” 

Barnes’ clinic currently meets twice monthly. So far, she has seen several patients during and after treatments. She hopes to coordinate more closely with oncology and the Children’s of Alabama Taking on Life after Cancer (TLC) Clinic—which provides a varied and multidisciplinary approach to managing the special needs of pediatric cancer survivors—in order to catch endocrine issues early.

She hopes to bring more attention to endocrine disorders in pediatric cancer survivors while educating clinicians and families that remission is not the end of a child’s cancer-related health journey. “Ongoing monitoring for growth, puberty, bone health and metabolism remains essential,” she said

“I enjoy treating these children and adolescents. They’re survivors,” Barnes said. “Although cancer may rob them of some of their potential, I have the opportunity to optimize their growth and development.”

August 22, 2023 by
Endocrinology

Creating Brighter Futures for Patients with Turner Syndrome

Dr. Giovanna Beauchamp leads the Turner Syndrome Clinic at Children’s of Alabama.

Turner syndrome is a genetic condition that affects only females. Caused by a missing or partially missing X chromosome, it can lead to a variety of health and developmental challenges. For years now, Children’s of Alabama has been addressing the diverse needs of this population with its quarterly Turner Syndrome Clinic.

Pediatric endocrinologist Giovanna Beauchamp, M.D., runs the multidisciplinary clinic, which includes specialists in genetics, pediatric and adolescent gynecology, audiology, child life, nephrology, cardiology and social work. Patients range from infants through teenagers and are typically referred just after diagnosis.

The clinic model is crucial, Beauchamp said, because Turner syndrome impacts numerous body systems, as well as girls’ emotional health. Common issues include short stature, puberty and fertility problems, hearing loss, heart defects, thyroid disorders, diabetes risk, learning difficulties, anxiety and social struggles.

Treatments offered through the clinic include growth hormone injections to increase height, estrogen therapy to induce puberty, medications for endocrine problems like thyroid issues, and referrals for neuropsychiatric testing to identify learning needs.

The clinic’s social worker helps secure any necessary school accommodations, such as more time for test taking. However, Beauchamp said, “these girls are extremely bright. They can sometimes have a few struggles with math, but they tend to be pretty gifted with their language skills.” They may also have issues with focusing and a higher risk of attention deficit disorder, all of which the clinic team can help with. “It’s important to know if they have any learning differences that may need to be tackled with a little bit of extra help and support,” she said.

The child life specialist is particularly important, Beauchamp said. “A lot of our girls can have some anxiety and get nervous when it comes to their visits.” Plus, the visits typically last longer than routine endocrinology visits. But the child life specialist provides a variety of activities and games, such as drawing and arts and crafts, to keep them engaged and relaxed.

“We aim to be a one-stop shop to provide the best and most thorough care for girls with Turner syndrome,” Beauchamp said.

August 22, 2023 by
Cardiology

Code Committee Brings Innovation and Improvements

HC3 is led by Dr. Ahmed Asfari (top row, fourth from left) and Ashley Moellinger (top row, third from left).

Since the inception of the Heart Center Code Committee (HC3) in 2014, Children’s of Alabama’s cardiac arrest rate in the Cardiovascular ICU has fallen nearly 50%. The impact this committee has had on cardiac arrest reduction has come from numerous quality improvement initiatives and safety changes. In 2022, some of the initiatives Children’s implemented include the development of four guidelines, three communication enhancement tools, checklists and numerous safety changes.

Whenever a patient goes into cardiac arrest or a near miss is encountered, HC3 discusses the case. This multidisciplinary committee is composed of physicians, nurse practitioners, bedside nurses, respiratory therapists, cardiovascular operating room staff, chaplains and leadership from the heart center. HC3 meets every other week to evaluate each case, identify what was done well and areas for improvement.

Nurse practitioner Ashley Moellinger, CRNP, MSHQS, and cardiac intensivist Ahmed Asfari, M.D., who participate in quality improvement initiatives within the heart center, began leading the committee in 2021. They’ve focused on three key areas: education, inclusivity and innovation.

“We have unique patients with really complicated conditions that can be challenging to understand,” Moellinger said. “Whenever we review the event, we look for areas where there’s a knowledge gap or a need to enhance skills.” Then the team sends education briefs to the nurses to highlight committee findings.

Every nurse in the department is involved with the committee. “Really, the committee is owned by the nurses,” Moellinger said. An elite team of nurses reviews each case, interviews those involved and develops a presentation to tell the story of the event. Then, the committee decides together what they need to change. “And that’s where the education rollout comes into play,” Moellinger said.

The committee uses artificial intelligence and near-real-time analytic algorithms to analyze cardiac arrest and near-arrest events. “When you’re talking about a cardiac arrest, everyone’s recollection is going to be different,” Asfari said. With the platform, “we can use objective data to show the patient’s course.” It also allows the team to view vital signs in a continuous manner on one screen.

“One of the things we are most proud of is the ‘green’ epinephrine action plan,” Asfari said. When a patient goes into cardiac arrest, epinephrine is one of the first medications administered, but it takes time to prepare the drug. Shorter time to administer it is associated with better outcomes. The action plan calls for prepared epinephrine at the bedside and includes standing orders for the nurse to administer it once the patient’s vitals reach a certain threshold. “It’s made a huge impact on our patient care,” he said. “Cardiac arrest is a real problem for children with cardiac disease because they are so fragile,” Asfari said. “Improving resuscitation and, more importantly, preventing the arrest can improve the outcome.”

August 22, 2023 by
Cardiology

New Registry Studying Long-Term Outcomes of Heart Procedures

A new national registry is tracking patients’ progress over time following heart procedures as neonates.

Children’s of Alabama has joined a new national registry managed by the Congenital Cardiac Research Collaborative (CCRC), attempting to answer the question: What is the best way to treat ductal-dependent pulmonary blood flow congenital heart conditions?

Conditions such as Tetralogy of Fallot, critical pulmonary valve stenosis and complex single-ventricle arrangements can be treated with various interventions, both percutaneous and surgical. “We’re looking at all neonates (first 30 days of life) who require a procedure, so they get enough pulmonary blood flow to survive,” Children’s pediatric cardiologist Mark Law, M.D., said. “The goal is to take this broad grouping of patients and understand how they do, not just with their first intervention or even their second intervention if they need one, but how do they do as they grow up?” This question of long-term outcomes hasn’t been studied, he said.

“In our field, we tend to be very focused on the short-term, procedural outcome. We have a disease, we have a problem. We do an intervention, and we look at that intervention or outcome. But we haven’t looked at overall survival and longevity; some of the more nuanced outcomes, including quality of life,” Law said.

The idea came about as the result of the Comparison of Methods of Pulmonary Blood Flow Augmentation in Neonates: Shunt Versus Stent (COMPASS) trial, which is comparing overall survival and post-surgical complications, hospital length of stay and quality of life between a surgically inserted shunt and one threaded into the ductus arteriosus, the artery that connects the aorta and pulmonary artery in newborns. That trial, in which Children’s is also participating, will follow patients for two years. Part of the funding enabled the development of the registry. Participation requirements for the registry are also broader than for the clinical trial, opening it up to a larger population. It will also be part of Cardiac Networks United, which manages numerous other registries.

The CCRC registry, which currently has 14 participating institutions, started in 2022 and has been collecting data for less than a year. Children’s was among the first institutions to join. The registry will track how patients fare over time and provide valuable information about which treatment pathway is best for which condition and which child. Children’s has been enrolling patients since October 2022 and currently has about 11. The goal is 15 to 20 a year. Registry information on each participant will be updated annually, but Law said that’s one of the challenging parts of the study—for instance, if families move out of the area and are seen at other institutions. That challenge, Law said, could be overcome through the robust electronic health records children’s hospitals use to share information.

August 22, 2023 by
Cardiology

Society of Thoracic Surgeons National Database Reinforces Cardiothoracic Program’s Success

The Children’s of Alabama cardiothoracic surgery program is among the nation’s best in expected-to-observed mortality rate.

A pivotal way to measure a program’s success is by comparing it to others. And according to outcomes recorded in the Society of Thoracic Surgeons (STS) National Database, the Children’s of Alabama cardiothoracic surgery program continues to surpass national trends.

“In 2023, quality is the big catchphrase, but the only way we know we’re providing the best care is to look at results,” Robert Dabal, M.D., chief of pediatric cardiothoracic surgery at Children’s said. “The database allows us to analyze data over lots of different time frames—months or years. It gives us the ability to look at results over time to make sure we’re always improving.”

Established in 1989, the database has become the gold standard for clinical registries, containing data on more than 8.8 million patients and 4,300 surgeons. An important subset is the STS Congenital Heart Surgery Database, which has captured records from more than 600,000 congenital heart surgeries in North America with more than 1,000 physicians. It monitors metrics such as patient complications, reoperations and deaths.

Cardiothoracic surgeons at Children’s perform about 440 congenital heart surgeries each year. The database shows that patients who might not survive in other hospitals are surviving at Children’s, Dabal explained. “Our observed mortality is less than the expected mortality, which is right where we want to be.”

Additionally, the most recent analysis of all pediatric cardiothoracic surgery programs in the Southeast shows Children’s has the second-lowest rates in the region—and one of the lowest rates in the country—in that same expected-to-observed mortality category. Combining the STS data with information culled from several other databases helps provide both a big-picture and granular assessment of Children’s progress and where it may still fall short.

“Perfect is not a realistic goal in medicine, but continually improving your results is an attainable goal,” Dabal said. “That’s why we’re always looking at these results.”

Even the best databases, however, can’t capture all factors related to a program’s success or goals. Along those lines, Dabal hopes future iterations can track young congenital heart patients’ long-term outcomes, not just perioperative data points.

“Most of our patients survive their operation, so the larger pediatric cardiothoracic community is focused on what happens to them one year, five years or 10 years out,” he said. “We want patients to survive surgery, of course, but also to lead normal lives—to go to school, get married and have children of their own. Our bigger focus is looking at the long-term quality of the outcomes we’re providing.”

August 21, 2023 by
Urology

With New Surgical Robot, Urology Team Can Better Serve Patients

Dr. Carmen Tong and the Children’s of Alabama urology team are preparing to start using the new Da Vinci surgical robot.

When Stacy Tanaka, M.D., arrived at Children’s of Alabama as the chief of pediatric urology, she resolved to bring in more technology. “I don’t want families of patients who need our services to go elsewhere because they think we can’t provide it,” she said at the time. That was in January 2022. By the end of her first year, one big piece of the puzzle was in place.

The da Vinci surgical robot arrived in December 2022, and according to Carmen Tong, D.O., director of pediatric robotic surgery, staff should be ready to use it within the next several months. “I’m so excited to offer this service for our children,” she said.

The surgical robot provides an alternative to laparoscopic surgery, in which instruments are inserted through two or three small incisions. Laparoscopic surgery is minimally invasive, but the technique is not ideal. “The instruments don’t articulate at the wrist, so they don’t mimic actual hands in the body,” Tong said. This makes certain maneuvers, such as internal suturing, quite challenging. “It’s as though you’re using chopsticks,” she said. There is also a steep learning curve.

The robot, however, “completely changed the landscape of minimally invasive surgery,” according to Tong. It provides a three-dimensional view with improved depth perception. In addition, the instruments enable much more refined movements—mimicking hands and fingers—and are gentler, which is particularly important when operating on babies, who have very delicate tissue. Overall, robotic surgery is less invasive, less disfiguring and results in quicker recovery than the traditional open technique. One small study also found it resulted in shorter surgeries and less suturing than using a laparoscopic approach for the same procedure.[i]

One major advantage is the three-dimensional, magnified view of the surgical field through the console, which isn’t available with the tiny cameras used in laparoscopic procedures. “It’s fabulous,” Tong said. “You’re able to see exactly what you’re picking up and what you’re cutting and stopping the bleeding on. From a safety standpoint, it’s a superior product.” One study found that robotic-assisted surgery could complement the motor skills of the surgeon’s nondominant hand, eliminating the innate difference in dexterity between hands and conveying ambidexterity.[ii]

Having the da Vinci robot also means Children’s no longer has to rely on access to the surgical robot at the University of Alabama at Birmingham (UAB), which is an adult hospital.

[i] Silay MS, Danacioglu O, Ozel K, Karaman MI, Caskurlu T. Laparoscopy versus robotic-assisted pyeloplasty in children: preliminary results of a pilot prospective randomized controlled trial. World J Urol. 2020;38(8):1841-1848. doi:10.1007/s00345-019-02910-8

[ii] Choussein S, Srouji SS, Farland LV, et al. Robotic Assistance Confers Ambidexterity to Laparoscopic Surgeons. J Minim Invasive Gynecol. 2018;25(1):76-83. doi:10.1016/j.jmig.2017.07.010

January 17, 2023 by
Urology

Children’s of Alabama Leads Practice-Changing Spina Bifida Study

Dr. David Joseph is a pediatric urologist at Children’s of Alabama.

Spina bifida, a condition in which the neural tube doesn’t completely close, is one of the most common congenital malformations, affecting approximately one out of every 2,700 births. While neurosurgeons provide immediate care, it isn’t long before urologists and nephrologists get involved. That’s because damage to the spinal cord and nerves may keep brain signals from reaching the bladder. If that happens, urine can back up into the kidneys, possibly causing kidney damage. “Yet nearly all newborns with spina bifida show normal kidney function at birth,” Children’s of Alabama pediatric urologist David Joseph, M.D., said. “But over time, at least half will deteriorate to some degree.”

Which begs the question: How do you manage these children? A 10-year, nine-center initiative at Children’s of Alabama has been trying to answer that question. Called Urologic Management to Preserve Initial Renal Function Protocol for Young Children with Spina Bifida (UMPIRE), the initiative is designed to provide an evidence-based protocol for testing and monitoring kids with spina bifida to identify early kidney injury. The Centers for Disease Control and Prevention (CDC) funds the study, which is now following more than 500 children with the most severe form of spinal bifida, myelomeningocele, in which a sac of fluid containing part of the spinal cord and nerves protrudes through an opening in the baby’s back.

Unlike most clinical trials, where the outcomes are evaluated at the end, the UMPIRE investigators review the data every quarter and tweak the protocol accordingly. “Obviously, this is not as clean as a randomized control trial,” Joseph said. “But it’s an effective way to manage a small population without a control group.”

This approach has led to some important revelations. For example, the team learned that bringing newborns in for imaging every three months for the first year as they’d been doing had no benefit at the nine-month visit. They also recognized that prophylactic antibiotics to prevent infection weren’t needed in newborns. Children’s chief of pediatric urology Stacy Tanaka, M.D., discovered that each center assessed urodynamics (lower urinary tract function) differently, which was a serious problem given the reliance on those tests to classify a patient’s level of damage and determine treatment.

Finding participants for the study hasn’t been a problem, Joseph said. “I don’t think we’ve had a family in the past seven years that has turned down the opportunity to be in the study,”—something he attributes to Betsy Hopson, MSHA, coordinator of the Children’s of Alabama Comprehensive Spina Bifida Program. The goal is to follow the children for at least 10 years or for as long as the CDC continues to fund the study.

“The urologic community looks to the UMPIRE program for the protocol in anticipation that it will help direct future management,” Joseph said.

January 17, 2023 by
Pulmonology

New Protocol Dramatically Improves Outcomes for Children with Acute Chest Syndrome

An initiative led by Dr. Ammar Saadoon Alishlash is helping children with acute chest syndrome.

Acute chest syndrome (ACS) is a respiratory disease that starts suddenly, progresses rapidly and is the leading cause of death in children with sickle cell disease in the U.S. and other countries. However, in the last three years, since Children’s of Alabama instituted a standardized ACS protocol, there have been no ACS-related deaths among the hospital’s patients included in the protocol.

Children’s admits as many as 100 children with ACS every year, and the new protocol has improved outcomes among these patients, decreasing the average number of inpatient days from seven to four (a 42% drop) and reducing ICU admissions and mortality.

“We did not expect such significant results,” pediatric pulmonologist Ammar Saadoon Alishlash, M.D., the leader of the initiative, said. When the initiative began, it was up to each child’s physician whether to follow the protocol or standard procedure because it wasn’t clear if the protocol would actually improve outcomes. “But now we’re pushing to have all patients diagnosed with acute chest syndrome be put on the protocol,” he said.

The protocol is based on the latest evidence on the management of ACS. It includes order sets for tests and medications that are embedded in the electronic medical record, which makes it possible for the patient to receive appropriate care in the emergency room rather than waiting until they’re admitted. It also provides three order sets depending on where the child is in the hospital: in the emergency department, the hematology floor or the ICU. “Each has a specific set of orders designed for that specific population to improve outcomes wherever they are in the hospital,” Alishlash said. The standardization also reduces variation in how physicians treat the patients, which is one reason for the improved outcomes, he said. The protocol involves initiating therapy early in the emergency department before the condition progresses. The main interventions include oxygen supplementation and respiratory support.

To further improve ACS outcomes, Alishlash, who leads the pediatric pulmonary sickle cell disease program at Children’s and the University of Alabama at Birmingham, is performing clinical and laboratory research on the condition. He received a National Institutes of Health grant to study the effects of environmental exposures on its development and to test novel therapies. He has also identified risk factors for ACS, including low oxygen levels during sleep and an association between where the child lives and the condition. “To prevent death in these small children is a great achievement,” he said. “Hopefully it will dramatically improve outcomes in our sickle cell patient community.”

January 17, 2023 by
Neurology & Neurosurgery

Children’s Neurologist Receives NIH Grant to Explore New Pathway in DMD

Dr. Michael Lopez received a nearly $1 million grant to study a new pathway in Duchenne muscular dystrophy.

What happens when you knock out a ubiquitous protein in muscle that appears to be involved in numerous neuromuscular diseases, including Duchenne muscular dystrophy (DMD)? That’s the question Children’s of Alabama pediatric neurologist Michael Lopez, M.D., Ph.D., and his mentors, University of Alabama at Birmingham (UAB) professor Peter King, M.D., and assistant professor Matthew Alexander, Ph.D., are trying to answer.

Lopez recently received a Career Development Award worth nearly $1 million from the National Institute of Neurologic Disorders and Stroke to better understand a novel pathway involved in the development and progression of DMD.

The disease, which primarily affects males, is caused by a mutation in the gene that encodes for the dystrophin protein, which is critical for musculoskeletal health. Without this protein, muscles degrade over time, resulting in a severe paralysis that affects breathing and eventually causes the heart to fail. Patients typically die in their early 20s or 30s.

There is no satisfactory treatment for DMD. A multidisciplinary approach involving neurology, cardiology, pulmonary care and rehabilitation—among other specialties—helps patients manage the disease. Immune-dampening corticosteroids are the primary medical therapy.

Lopez and his team identified a new pathway involved in the sustained inflammation that underlies the disease. While chronic inflammation is driven, in part, by elevated levels of the cytokine transforming growth factor β (TGFβ1), clinical studies using drugs to inhibit TGFβ1 have been, by and large, unsuccessful. Lopez thinks that’s because the TGF signaling is more complicated, so any attempt to reduce levels must account for downstream signaling via transcription factors, called Smads, that receive instructions from TGFβ.

While it’s been known for some time that the Smad2 and Smad3 factors are important players in the TGFβ pathway, Lopez’s research identified another Smad called Smad8 that is not only turned on in a cellular model of DMD but is 48 times higher than other Smad factors. His findings were published in the International Journal of Molecular Science in July. “It appears to be a previously unrecognized pathway that could cause larger dysregulation of gene expression within the muscle,” he said.

When the researchers silenced Smad8 in cultured muscle cells, they found the cells differentiated into muscle fibers more successfully. “That’s a key experiment because it shows that too much of Smad8 was likely doing the opposite: preventing the muscle cells from differentiating into myofibers,” Lopez said.

The grant provides the funds to breed transgenic mouse lines in which the gene that encodes for Smad8 is deleted in cells destined to become muscle cells. “That way, we can answer the question, ‘Is it necessary for the normal function of muscle, and does it make DMD less severe in the mouse?’” Lopez said. “The premise is that we can intervene on this pathway and reverse these impairments.”

January 17, 2023 by
Neurology & Neurosurgery

LITT Device Makes Epilepsy Surgery More Precise, Less Invasive

Surgeons perform a laser interstitial thermal therapy (LITT) procedure at Children’s of Alabama.

A new procedure called laser interstitial thermal therapy (LITT) allows Children’s of Alabama surgeons to take a minimally invasive approach to brain surgery and target tissue for ablation with greater precision.

Usually, patients with drug-resistant epilepsy who experience intractable seizures undergo resective surgery, in which a surgeon removes part of the brain. The procedure is very invasive, however, entailing a craniotomy, or removing part of the skull and cutting through the dura, which covers the brain. Some areas of the brain are difficult to navigate, and removing certain sections, such as the eloquent cortex, can lead to a loss of important functions, such as sensory processing or speech. Resective surgery also requires several days in the hospital and carries a risk of infection and bleeding.

“The small LITT device enables us to get into a deep region of the brain easily and safely,” pediatric neurologist Kathryn Lalor, M.D., said. “We can find the seizure onset with the electrode and then target the same area with LITT.”

The robotic system inserts a 2-to-3-millimeter probe (about the size of the tip on a new crayon) through a hole drilled into the skull. MRI guidance precisely locates the target area responsible for seizures. Once the probe is in place, a burst of laser energy destroys the tissue.

The device was initially FDA approved for temporal and medial structures in the brain, where much of adult epilepsy surgery occurs. Now, Children’s and other pediatric centers are demonstrating its effectiveness at treating epilepsy in other areas of the brain. “There’s a lot of research on how to make the energy delivery even more specific, so no unintended areas are affected,” Lalor said.

Using the device also reduces brain swelling thanks to its less invasive nature. “So, the recovery time is much quicker, and many of these patients go home the next day,” she said. In fact, studies find few complications and a good safety record.

In 2022, the team completed six surgeries using the LITT system.

January 17, 2023 by