E-News for Referring Offices

Department of Pediatrics Welcomes 3 New Faculty Members in August 2020

Jessica Schmitt, M.D.Assistant Professor in Pediatric Endocrinology and Diabetes, earned her medical degree from the University of Alabama at Birmingham. Dr. Schmitt completed her pediatric residency at Cincinnati Children’s Hospital in Cincinnati, Ohio. She started her pediatric endocrinology and diabetes fellowship at Massachusetts General Hospital in Boston and completed her fellowship at the University of Alabama at Birmingham. Her clinical and research interests include quality improvement, specifically assessing process changes to improve management of and outcomes for youth with diabetes.


Nora Switchenko, M.D., Assistant Professor in Neonatology, earned her medical degree at Oregon Health and Science University in Portland, Ore. Dr. Switchenko completed her pediatric residency and her neonatology fellowship at the University of Utah in Salt Lake City, Utah. Her clinical and research interests include improving care for sick neonates in resource limited areas of the world with a particular focus on respiratory support for small and premature infants.


Carmelle Wallace, M.D., MPH, DTMH, Assistant Professor in Pediatric Emergency Medicine, earned her medical degree from Baylor College of Medicine in Houston, Texas. Dr. Wallace completed her pediatric residency at the University of Texas-Southwestern in Dallas, Texas and completed her pediatric emergency medicine fellowship and global health fellowship at Children’s Hospital of Philadelphia in Philadelphia, Pa. Her clinical and research interests include international pediatric emergency medicine capacity building, child human rights and international and domestic child trafficking and abuse. She has worked in Zambia, Tanzania, Swaziland, South Africa, India and Laos.

E-News for Referring Offices

Department of Pediatrics Welcomes 10 New Faculty Members in July 2020

Guillermo Beltran Ale, M.D., Assistant Professor in Pediatric Pulmonary and Sleep Medicine, earned his medical degree at Universidad Peruana Cayetano Heredia, Facultad de Medicina Alberto Hurtado in Lima, Peru. Dr. Beltran Ale completed his pediatric residency and pediatric pulmonology fellowship at Cincinnati Children’s Hospital Medical Center in Cincinnati, Ohio. His major research interests include Pulmonary Alveolar Proteinosis with a focus on BAL based diagnosis and optimizing care for pulmonary patients who are technology dependent with a focus on infections diagnosis and treatment. His clinical interests are aerodigestive pathology, the integrative care of patients on long term ventilation and technology dependence, rare lung diseases and pulmonary transplantation.


Vidit Bhargava, M.D., Assistant Professor in Pediatric Critical Care, earned his medical degree at Byramjee Jeejeebhoy Medical College in Pune, India. Dr. Bhargava completed his pediatric residency at the University of Texas Medical Branch in Galveston, Texas. He did a pediatric critical care fellowship and emergency ultrasound fellowship at Stanford University in Palo Alto, Calif.


Jared Buchan, M.D., Instructor in Pediatric Emergency Medicine, earned his medical degree from Florida State University in Tallahassee, Fla. Dr. Buchan completed his pediatric residency at Memorial Health University Medical Center in Savannah, Ga.


Viral Jain, M.D.Assistant Professor in Neonatology, earned his medical degree from MGM Medical College, Mumbai, India. Dr. Jain completed his pediatric residency at Case Western Reserve University in Cleveland, Ohio. He did his neonatology fellowship and research fellowship at Cincinnati Children’s Hospital in Cincinnati, Ohio. His primary area of interest is in Developmental Origins of Health & Disorders (DOHaD) or how changes during early period of in utero development influences life-long outcomes, and neonatal evidence-based medicine. He is also interested in exploring family’s contribution to young child’s learning and is the founder of TinyVoices – a patient advocacy program through which is founded the nationally renowned NICU Bookworms, a NICU Infant Reading program. Dr. Jain is also a children’s storybook writer and a musician and has written storybooks for NICU infants as well as written songs with Grammy award winners for NICU families.


Jeremy Loberger, M.D., Assistant Professor in Pediatric Critical Care Medicine, earned his medical degree at the University of South Carolina in Colombia, S.C. Dr. Loberger completed his combined internal medicine and pediatric residency at the University of South Carolina in Greenville, S.C. He did a pediatric critical care fellowship at the University of Alabama at Birmingham. His major research/clinical interests include quality improvement and clinical research on the topics of extubation readiness assessment, mechanical ventilation and sepsis.


Brittany Marlin, M.D., Instructor in Pediatric Hospital Medicine, earned her medical degree at the Florida State University in Tallahassee, Fla. Dr. Marlin completed her pediatric residency at the University of Alabama at Birmingham.


Pedro Anis Nourani, M.D., Assistant Professor in Pediatric Pulmonary and Sleep Medicine, earned his medical degree at the University of São Paulo in Ribeirão Preto, Brazil and completed his pediatric residency at Texas A&M’s Driscoll Children’s Hospital in Corpus Christi, Texas. Dr. Nourani completed his sleep medicine and pediatric pulmonology fellowships at the University of Alabama at Birmingham. His clinical activity has an emphasis on pediatric sleep disorders, Continuous Positive Airway Pressure (CPAP) therapy and general pediatric pulmonology disorders. His clinical research focuses on the identification of different sleep parameters related to clinical outcomes of sickle cell disease as potential targets for intervention.


Bhuvana Sunil, M.D., Assistant Professor in Pediatric Endocrinology and Diabetes, earned her medical degree from the Bangalore Medical College & Research Institute in Bengaluru, India. Dr. Sunil completed her pediatric residency at the Harlem Hospital Center in New York, N.Y. She completed her pediatric endocrinology and diabetes fellowship at the University of Alabama at Birmingham. Her clinical and research interests include obesity, type 2 diabetes, dyslipidemia and metabolic syndrome.


Kent Willis, M.D., Assistant Professor in Neonatology, earned his medical degree at the Ben-Gurion University of the Negev in Beer Sheva, Israel. Dr. Willis completed his pediatric residency at the Our Lady of the Lake Children’s Hospital in Baton Rouge, La., and his neonatology fellowship at the University of Tennessee Health Science Center in Memphis, Tenn. His research interests include understanding how commensal fungi, the mycobiome, influence newborn physiology and disease, principally via exploring the gut-lung axis in bronchopulmonary dysplasia. Willis Lung Lab is supported by the NIH, NHLBI K08HL151907, the UAB Microbiome Center and UAB Pediatrics.


Cynthia Wozow, D.O., Assistant Professor in Pediatric Rehabilitation Medicine, earned her medical from William Carey College of Osteopathic Medicine in Hattiesburg, Miss. Dr. Wozow completed her physical medicine and rehabilitation residency at the University of Texas-San Antonio in San Antonio, Texas and completed her pediatric physical medicine and rehabilitation medicine fellowship at Baylor College of Medicine and Texas Children’s Hospital in Houston, Texas. Her clinical interests include cerebral palsy, spasticity management, congenital neuromuscular disorders, brachial plexus injuries, transitional care, electrodiagnostic medicine and medical education.

Cardiology, E-News for Referring Offices, Inside Pediatrics

NEW PHYSICIANS JOIN PEDIATRIC CARDIOLOGY

Leslie Collins, M.D., Assistant Professor in Pediatric Cardiology, earned her medical degree at the East Carolina University – Brody School of Medicine. Dr. Collins completed her pediatric residency and pediatric cardiology fellowship at the University of Alabama at Birmingham. Her research/clinical interests include imaging, fetal ECHO, and heart failure and transplant.


Austin Kane, M.D.,AssistantProfessor in Pediatric Cardiologyearned his medical degree at Columbia University College of Physicians and Surgeons in New York, New York. Dr. Kane completed his pediatric residency at Northwestern University Feinberg School of Medicine in Chicago, Illinois. He completed a fellowship in pediatric cardiology at Emory University School of Medicine in Atlanta, Georgia and an additional fellowship in pediatric and congenital electrophysiology. Prior to joining the University of Alabama at Birmingham, he was a pediatric and congenital electrophysiology attending at Providence Sacred Heart Medical Center and Children’s Hospital in Spokane, Washington. 


Khalisa Syeda, D.O., AssistantProfessor in Pediatric Cardiologyearned her medical degree at the University of North Texas in Fort Worth, Texas. Dr. Syeda completed her pediatric residency at the University of Illinois at Chicago and her pediatric cardiology fellowship at the University of Texas Health Science Center in Houston, Texas. Her research/clinical interests include pediatric cardiology, imaging, fetal echocardiography and preventive cardiology. 

Inside Pediatrics, Neonatology

Generous Donation to NeuroNICU B.R.A.I.N. Program Helps Reduce Risk of Brain Injury in Premature Infants

NICU_WEB

Through a generous donation by Robert and Kathleen Israel, Children’s of Alabama is now home to new technology that helps dramatically prevent brain injury and improve brain development and function in its sickest patients.

Children’s of Alabama is thrilled to announce a very generous gift of a cutting-edge technology designed to help reduce the risk of brain injury in preterm infants. The gift was donated by Robert and Kathleen Israel in honor of the care their daughter, Ivy, received in the Neonatal Intensive Care Unit (NICU) in 2018. Ivy is home and doing very well. “The NICU team at Children’s of Alabama saved our daughter’s life,” said Robert Israel, “and we are forever grateful.”

“This new technology made possible by the Israel family is helping us  dramatically prevent brain injury and improve brain development and function in our sickest patients,” said Manimaran Ramani, M.D., director of the NeuroNICU program.

Preterm infants born at 30 weeks or earlier are at higher risk for developing intraventricular hemorrhage (IVH), which is associated with long-term neurocognitive and motor deficits. The risk for neurocognitive and motor deficits is also higher for term infants with hypoxic-ischemic encephalopathy (HIE), seizures, metabolic disorders, or stroke, and those undergoing ECMO therapy.

However, a multidisciplinary initiative in the NICU at Children’s of Alabama and the University of Alabama at Birmingham (UAB) called NeuroNICU B.R.A.I.N. (Brain Rescue and Avoidance of Injury in Neonates) aims to prevent and reduce neurocognitive and motor deficits in high-risk neonates.  

The objective of the B.R.A.I.N. program is to identify and prevent brain injury early in high-risk neonates through state-of-the-art diagnostic techniques and neuroprotective care. An interdisciplinary team of medical professionals meets every week to strategize individualized comprehensive neuroprotective plans for infants enrolled in B.R.A.I.N.

Though standard vital monitoring techniques used in NICUs such as blood pressure, heart rate and pulse oximetry provide valuable information about the infant’s hemodynamic status, such standard monitoring techniques don’t provide real-time information regarding the brain’s oxygenation saturation, oxygenation extraction and perfusion status of a sick neonate.

This is where infrared spectroscopy (NIRS) monitoring comes in. “This technology allows us to monitor cerebral oxygenation in very sick infants,” Ramani said. It is a non-invasive method that can be used continually at the bedside as well as during surgery to monitor the health of the brain. It can also be combined with amplitude-integrated electroencephalography (aEEG) to monitor cerebral electrical activity and to diagnose seizures in sick neonates in real-time.

“With the two NIRS devices donated by the Israel family, we are now able to monitor the brain health and adjust our therapies and strategies in real-time on our patients,” Ramani said.

 

 

Cardiology, Inside Pediatrics

Children’s of Alabama Receives Prestigious Accreditation from Children’s Cardiomyopathy Foundation

Cardiomyopathy_WEB

Livie Wheeler of Pell City, Alabama was diagnosed with dilated cardiomyopathy and underwent a successful heart transplant at Children’s of Alabama. Livie was discharged home just in time for her second birthday.

Children’s of Alabama was recently named an accredited center of care by the Children’s Cardiomyopathy Foundation (CCF), a national nonprofit committed to improving the health outcomes and quality of life for children with cardiomyopathy.

“We’re glad to be able to participate,” said F. Bennett Pearce, M.D., outgoing medical director of the Pediatric Advanced Heart Failure and Transplant Program at Children’s of Alabama. “CCF promotes education and helps families connect and choose centers with experience in these kinds of patients.”

The Pediatric and Congenital Heart Center of Alabama at Children’s of Alabama is one of the largest pediatric cardiovascular programs in the Southeast. In 2018, its team of 250 dedicated professionals treated more than 230 patients with pediatric cardiomyopathy. The team includes surgeons, intensivists, cardiologists and many others, including nurses, social workers, child life specialists, genetic counselors, nutritionists, occupational and physical therapists, and chaplains.

“We have traditionally had very strong clinical abilities and success with treating a variety of these conditions,” Pearce said. “Over the 25 years that I’ve worked in the program, I feel we have been among the finest centers, but we have not been quite so active in getting the word out. That needs to change because we want to make families aware that there’s a good resource for them here.”

The CCF offers a plethora of educational and supportive programs for families, as well as a research grant program for basic, clinical, population/epidemiologic, or translational studies focused on primary pediatric cardiomyopathy.

Its accreditation program was established in 2017 to recognize excellence in diagnosing and treating pediatric cardiomyopathy and provide families with standardized center information to help them find an expert treatment center in their area. To date, 39 centers in the U.S. and Canada, including Children’s of Alabama, have been accredited.

The CCF has very strict criteria for accreditation. Centers must:

  • Manage 30 or more cardiomyopathy patients up to age 18 annually or 60 pediatric cardiomyopathy patient visits a year.
  • Have at least one pediatric cardiologist who treats patients with dilated, hypertrophic, restrictive arrhythmogenic and right ventricular or left ventricular non-compaction cardiomyopathy.
  • Be part of a teaching hospital affiliated with a medical school.
  • Offer the following:
    • Prenatal or fetal echocardiography
    • High quality imaging (echocardiogram, cardiac magnetic resonance imaging)
    • Interventional cardiology (catheterization, endomyocardial biopsy)
    • Pediatric cardiac electrophysiology
    • Cardiac surgical services (septal myectomy, cardiac device implantation)
    • Genetic testing and counseling
    • Pediatric neurology
    • Social work
    • Child life services
  • Centers must also have at least one of the following (Children’s of Alabama meets all four):
    • A specialized clinic or program focused on pediatric cardiomyopathy or familial hypertrophic cardiomyopathy with a dedicated team of professionals.
    • Involvement with the North American Pediatric Cardiomyopathy Registry, Pediatric Cardiomyopathy Repository or Pediatric Heart Transplant Study Group.
    • Engagement in pediatric cardiomyopathy research.
    • Advanced heart failure management and transplantation.

“We have everything needed to offer to this group of patients,” Pearce said. “And we want to make sure that when a family confronts this difficult diagnosis, they can easily find our program and be assured that it is a good resource.”

 

Inside Pediatrics, Neurology & Neurosurgery

Children’s of Alabama Clinicians Bring Expertise and Training to Vietnam

Global Surgery NEW_WEB

Children’s of Alabama physicians review a brain scan at a hospital in Vietnam. Neurosurgeons, neurologists and other medical staff travel to Vietnam at least once a year to provide lectures and hands-on training at hospitals in Hanoi and Ho Chi Minh City as part of Children’s of Alabama’s Global Surgery Program.

Surgical interventions to reduce the burden of drug-resistant epilepsy in children have become an integral part of the field in the past 20 years. In low- or middle-income countries like Vietnam, however, it typically remains a vision, not a reality. Vietnam, for instance, has just two adult and two pediatric neurosurgery training programs for a country of 95 million people, and just four pediatric neurosurgeons serving a population of more than 50 million in the northern part of the country.

Enter Children’s of Alabama’s Global Surgery Program, which is designed to form strong, collaborative relationships with large pediatric hospitals in low- and middle-income countries and provide subspecialty fellowship training at Children’s. The hospital’s relationship with Vietnam began in 2013, with an initial visit to neurosurgeons in Ho Chi Minh City. Since then, a team from Children’s, including pediatric neurosurgeon Brandon Rocque, M.D., MS, FAANS, Pediatric Epilepsy Surgery Director Pongkiat Kankirawatana, M.D., Clinical Neurophysiology and Pediatric Epilepsy Program Director Monisha Goyal, M.D., and Director of Neuromonitoring Trei King, R.EEG.T, C.N.I.M., among others, has traveled the nearly 9,000 miles to Vietnam at least once a year to provide lectures and hands-on training at hospitals in Hanoi and Ho Chi Minh City. Their efforts have helped create premier epilepsy programs that draw children from throughout southeast Asia.

“We started with the corpus callosotomy,” Rocque said, a procedure performed on children with generalized epilepsy prone to drop attacks. It involves splitting the main connection pathway between the two cerebral hemispheres to prevent the attacks. The Vietnamese team, led by a neurosurgeon who specialized in brain tumors, performed two such surgeries with the Children’s of Alabama neurosurgeons, then went on to complete 10 themselves over the next six months, all with good long-term results.

Epilepsy surgery is not possible without advanced EEG monitoring, which is where King came in. His job was to teach EEG technologists how to use an EEG in the operating room, including electrode placements, and the most appropriate test for the child’s condition. “We started with the basics and now we’re going much deeper,” he said.

“The people there are extremely hard working and very, very smart,” King said. “They just didn’t have the opportunity and education. The training with our staff allows them to see the entire gamut of what we do in the field and, hopefully, grow to do what we do.”

The Children’s of Alabama team usually spends a week in each city, giving lectures and assisting with the more complex surgeries. “We’re not trying to hammer out a bunch of cases,” Rocque stressed. “The model is not missionary surgery; it’s teaching and working on the patients they asked us to assist with in order to reach a goal of improving specific techniques for the neurologists and surgeons.”

In addition to building the team’s skills in epilepsy surgery, the team hopes to improve how pediatric neurosurgeons are trained in Vietnam, Rocque said. Currently, residents are trained in neurosurgery but don’t receive any formal pediatric training. “There is a really big opportunity to improve the way pediatric neurosurgery is taught in this region,” Rocque said. To help in that goal, Vietnamese physicians and EEG nurses now come to Alabama for several months for focused training on various procedures.

The partnership has continued to grow, with the Vietnamese doctors sending PowerPoint presentations on difficult cases for discussion at Children’s of Alabama’s weekly multidisciplinary meeting, during which the neurology team develops treatment plans. Now the team also discusses the Vietnamese patients. “When we started, we often had to ask for more information and make some changes to the treatment plan,” Rocque said. “But over the last year, their own recommendations have been spot on.”

The experience has been eye opening for the Children’s of Alabama clinicians. “I found a pediatric neurology and neurosurgery program making a valiant effort in diagnosing and treating one child after another with minimal resources,” said Goyal, who visited City Children’s Hospital in Ho Chi Minh City. The children slept on cots that spilled out into the adjacent roofed but open courtyards and hallways, she said. “There were no fans and in September temperatures in Ho Chi Minh were far from balmy.” She was also struck by the fact that the government does not allow the use of benzodiazepines, one of the most common medications used to control seizures in the U.S.

However, Goyal said, “the small number of clinicians do an admirable job with limited resources. They learn from textbooks, not from mentors, even though they have much fewer technical and pharmacological resources.”

“As a physician, this has been a very rewarding experience,” Goyal said. She, Rocque and King are planning another trip this spring to Ho Chi Minh City to continue helping the hospital develop of its own pediatric surgical epilepsy program.

King echoed Goyal’s comments. “It’s been one of the most rewarding things I’ve experienced in my career,” he said. “You see the big difference you make not only in the training, but in the impact on the patients receiving care that they would not get unless we’re there.”

“The patients and families are so appreciative,” King said. “I’ve never experienced that level of appreciation before. I think a lot of it has to do with realizing that without the partnership and collaboration we wouldn’t be able to do the surgeries.”

For instance, he recalls one family whose child was operated on returning to the hospital a year later with a picture of the child to express their thanks. “Those are the things they make this so rewarding,” he said. “When we see the difference it makes in the life of the patients and families and the joy that continues long after the surgery.”

Inside Pediatrics, Neurology & Neurosurgery

At Children’s of Alabama, Transitioning Spina Bifida Patients to Adult Care Begins at Age 13

Spina Bifida_WEB

Professional heavyweight boxer Deontay Wilder greets a patient while touring Children’s of Alabama’s Comprehensive Spina Bifida Program space in this 2015 photo. The multidisciplinary program treats about 500 patients a year.

Before the 1970s, about one in four infants born with spina bifida died. Today, thanks to advances in neurosurgery, genitourinary surgery, gastroenterology and physical medicine and rehabilitation, between 75 and 80 percent now survive into adulthood.[1]

But surviving doesn’t mean thriving. That’s what clinicians from Children’s of Alabama’s multidisciplinary Comprehensive Spina Bifida Program, which treats about 500 patients a year, learned when they joined specialists at the University of Alabama at Birmingham (UAB) to develop an adult spina bifida clinic. While there is a greater awareness in the pediatric community about the overall need for transitional programs, said Jeffrey P. Blount, M.D., FAANS, chief of pediatric neurosurgery at Children’s of Alabama, more is needed than simply transferring a patient’s medical records.

“Our patients were arriving unprepared not just for adult health care, but for adult life in general,” said Spina Bifida Program Coordinator Betsy Hopson, MSHA, who assists patients in the transition from pediatric to adult care.  “We’d watch them grow up and hear about their dreams but every year it seemed like the light inside them was becoming dimmer and their opportunities fading away.” By the time they arrived at the adult clinic, she said, many patients were depressed and their childhood dreams seemed to have disappeared.

“We thought, ‘We’ve got to do better with this next generation,’” Hopson said.

They, along with Children’s of Alabama neurosurgeon Brandon Rocque M.D., MS, FAANS, started with data, surveying 188 patients in the adult clinic about their perceptions of their own disability. More than half (56.4 percent) identified as permanently disabled, with the most important predictors of disability being poor bowel continence and low education.[2]

They also found that 70 percent of those surveyed were not working and, again, poor bowel continence and low education were determining factors, as well as diagnosis (open versus closed spinal dysraphism). Rocque emphasized that patients with daily bowel accidents were six times more likely to be unemployed, while those with weekly accidents were three times more likely to be unemployed.

Thus was born the Individualized Transitional Plan (ITP), which starts at age 13. The objective is to ready the child for adulthood by developing and achieving five goals, including bowel management and educational achievement. Goals also include transitional readiness (ie, do they know their medical history, can they make their own appointments and see the doctor without a parent present); meeting a parent-initiated goal (ie, showering without being told or learning how to cook); and a personal goal, such as learning to eat healthier. Hopson individualizes care to transform each goal into a measurable task and the results become a contract and framework for the next few years.

They also receive a transition binder that includes a career interest survey. “They complete it before they see me and then I help guide them,” Hopson said. She also ensures their career  goals are realistic. For instance, one patient said he wanted to be the Marines, which she knew wasn’t possible. Instead, she suggested a meeting with a recruiter to discuss civilian jobs in the military.

About 45 percent of patients fall into the age group for the ITP, which is part of the clinic’s Lifetime Care Model (LCM). The LCM begins prenatally and continues throughout the adult years.[3] Hopson developed the ITP, which has generated great interest from other clinics in North America who are struggling with the challenges of implementing a meaningful transition program.

Since starting the program, Blount said, clinicians from throughout the country have contacted the team at Children’s to learn how to start their own program. “There’s such a need for a supportive multidisciplinary clinic to support these patients,” he said.

“This model could be replicated for other children with chronic conditions,” Hopson said. “If we’re doing our jobs well, our children are going to be adults. So we have some obligation to get them there in a way that provides a good quality of life.”

Bowel Management Program

Children with spina bifida are born with neurogenic bladder and bowel issues. While urination can be managed through catheterization, with children as young as 3 years old learning to catheterize themselves, “bowel management is much trickier,” Hopson said. Gastrointestinal motility slows and constipation becomes a chronic issue. Unfortunately, she said, many families prefer constipation to regular movements because it’s easier to handle. But “once you set up that pattern it’s hard to break,” she said.  And bowel management, she said, “becomes the most important issue in whether they are able to get a job.”

Today, the children in the spina bifida clinic benefit from working with pediatric gastroenterologist Mitchell B. Cohen, M.D., and members of the Division of Physical Medicine and Rehabilitation. Cohen chairs the UAB Department of Pediatrics and is physician-in-chief at Children’s of Alabama. He became interested in the program after hearing Hopson speak. He developed a protocol beginning at age 4 to help families understand the importance of a bowel management program and try to end their reliance on constipation.

Nonetheless, it remains an “extraordinarily difficult problem,” Roque said. “There is no magic bullet.” Still, “with Cohen’s help we’re making inroads on this extraordinarily difficult problem that has profound impact that echoes through rest of a child’s care, including issues of self-image and self-worth.”


[1] Talamonti G, D’Aliberti G, Collice M. Myelomeningocele: Long-term neurosurgical treatment and follow-up in 202 patients. J Neurosurg 2007; 107: 368-86.

[2] Davis MC, Hopson BD, Blount J, et al. Predictors of permanent disability among adults with spinal dysraphism. J Neurosurg Spine. 2017 August ; 27(2): 169–177.

[3] Hopson B, Rocque BG, Joseph DB, et al. The development of a lifetime care model in comprehensive spina bifida care. J Ped Rehab Med. 2018; 11:323–334

 

Cardiology, Inside Pediatrics, Nephrology

Children’s of Alabama Leads Consortium Dedicated to Improving Outcomes in Cardiac Surgery-Acute Kidney Injury

NEPHRON_WEB

Children’s of Alabama is one of 22 hospitals in the U.S. that is a member of the Neonatal and Pediatric Heart and Renal Outcomes Network (NEPHRON).

Neonatal acute kidney injury (AKI) occurs in 52 to 64 percent of patients undergoing cardiac surgery (CS) and is associated with increased morbidity and mortality.

However, because CS-AKI rates vary widely between centers, it appears that interventions to prevent or mitigate the condition could reduce the overall rate.

Yet, noted Santiago Borasino, M.D., medical director of Children’s of Alabama’s Cardiovascular Intensive Care Unit (CVICU), “there are critical gaps in our understanding as to how to best define CS-AKI, who is at risk, and which patients could best benefit from interventions to prevent or  mitigate the effects of CS-AKI.”

To improve understanding of CS-AKI in this population, Borasino is one of the leaders of the Neonatal and Pediatric Heart and Renal Outcomes Network (NEPHRON), composed of 22 children’s hospitals around the country. The consortium’s goals are to describe neonatal kidney injury epidemiology, evaluate variability in diagnosis and management, identify risk factors, investigate the impact of fluid overload and explore associations with outcomes. It involves multidisciplinary teams including clinicians from cardiac critical care, cardiology, nephrology, and cardiac surgery.

“NEPHRON is providing multicenter data on CS-AKI for the first time,” Borasino said. “The large size of the cohort will enable us to look at details that are not possible with single-center studies.”

NEPHRON published its preliminary results in April 2019, reporting an overall incidence of 54 percent among 2,240 patients in its database.[1] In November 2019, NEPHRON presented additional results during the American Heart Association’s annual meeting, showing a threefold variation in rates among centers, from 27 percent to 86 percent, with significant variations in KDIGO stage (adult AKI definition) to identify AKI (65 percent by oligo oligo-anuria versus 35 percent by creatinine).

The results also showed that the use of cardiopulmonary bypass, but not time spent on bypass, increased the odds of CS-AKI, and that only KDIGO Stage 3 was associated with mortality. There was no impact of CS-AKI on the duration of mechanical ventilation or hospital length of stay.[2]

“NEPHRON preliminary results highlight the limitations of the KDIGO definition and the need to better understand CS-AKI as it occurs with incredible variability among centers, opening the door for future quality improvement intervention,” Borasino said.

The next step is to develop an algorithm to predict which patients are more likely to develop AKI so physicians can intervene earlier. “Early recognition and proper management of AKI are at the forefront of critical care medicine,” said Children’s of Alabama pediatric nephrologist Tennille Webb, M.D. “However, most pediatric hospitals that perform cardiac surgeries do not have protocols in place for managing severe AKI post-operatively.” Webb is now working on developing a clinical pathway to identify patients at increased risk of AKI based on specific patient characteristics. “An advantage to developing this algorithm in the CVICU is that we are able to determine the exact timing and etiology of AKI development in individuals undergoing cardiopulmonary bypass,” she said. “If we can proactively identify risk factors that place these individuals at increased risk for AKI, we can provide earlier intervention, such as early initiation of renal replacement therapy, in an effort to mitigate some of the known severe consequences of AKI.”

“The work that we are doing is very important because we know that AKI post-cardiac surgery leads to worse outcomes and is associated with chronic kidney disease,” Webb said. “It’s great, and yet rare in other institutions, that we have been able to develop a strong relationship between the CVICU and nephrology to work as a cohesive team early AKI detection and prevention.”


[1] Gist KM, Blinder JJ, Bailly D, Neonatal and Paediatric Heart and Renal Outcomes Network: design of a multi-centre retrospective cohort study. Cardiol Young. 2019;29(4):511-518.

[2] Alten J, Cooper DS, Gist KM, et al. , Abstract 13177: Epidemiology of Neonatal Cardiac Surgery Induced Acute Kidney Injury From the Neonatal and Pediatric Heart and Renal Outcomes Network. Circulation. 2019;140(Suppl1).

 

Inside Pediatrics, Pulmonology

Children’s of Alabama Muscular Dystrophy Care Center Offers Life-Saving Treatment for Rare, Fatal Disease

MDA_WEB

Brad Troxler, M.D., director of the Muscular Dystrophy Care Center (MDA) at Children’s of Alabama, chats with a patient. The MDA Care Center is the only MDA clinic in Alabama and one of only a few in the region.

The Muscular Dystrophy Care Center (MDA) at Children’s of Alabama sees about 280 patients a year with a wide variety of neuromuscular conditions. They require multidisciplinary care from neuromuscular specialists, neurologists, pulmonologists, genetic counselors and cardiologists, among others. They also need respiratory, physical and occupational therapies, nutritional counseling and social work support.

One of the most devastating conditions the clinic sees is spinal muscular atrophy (SMA), a rare autosomal recessive genetic disorder that is the leading cause genetic cause of death in infants. It is characterized by degeneration and loss of lower motor neurons, leading to muscle atrophy. Infants with the most serious form of the disease, Type 0, can be diagnosed prenatally and typically die before 6 months, while those with Type 1 begin showing symptoms around 6 months, require ventilation and typically die before age 2. Other types of SMA may not manifest until childhood or even adulthood and are less severe, although all of those affected experience some neuromuscular effects.

Until recently, SMA was primarily treated with supportive care. Today, however, there are two groundbreaking treatments and Children’s is the only MDA clinic in the state, and one of only few in the region, to offer both: nusinersen (Spinraza), approved for children and adults, which is administered in several doses the first year and annually after that; and onasemnogene abeparvovec-xioi (Zolgensma), a single-dose treatment approved for use in children with SMA Type 1 under age 2.

Both are gene therapies designed to address genetic mutations in the SMN1 and/or SMN2 genes, which make the protein required to control muscle movement. Both are also extremely expensive, with the first 10 years of treatment with Spinraza estimated at $4 million and a single dose of Zolgensma costing $2.1 million, making it the most expensive drug ever marketed.[1]

The cost could be worth it, though, given the benefits of the drugs. “Clinical trials showed huge improvement,” said Brad Troxler, M.D., who MDA Care Center director. Indeed, after four years, infants treated with Spinraza before they turned 6 weeks were still achieving and maintaining motor milestones such as walking and sitting without assistance, things SMA babies would not normally be able to do.[2]

Participants in the Zolgensma trials were demonstrating similar results, with all children who received the drug still alive at 2 years and none requiring ventilation.[3]

Don’t call the treatments cures, however, says Troxler. “‘Cure’ is a hard word to define,” he said. While improved, patients still don’t have normal neuromuscular function after receiving the drug, with some muscle weakness remaining and an increased risk of scoliosis and other issues related to the disease. “But if we give it early enough, they are able to walk and talk and eat so it’s pretty close to a cure,” he said.

The earlier the drugs are administered, the greater the benefit. “Time is neurons,” said Shelley Coskery, MSN, CRNP-AC, MDA Care Center coordinator. However, Alabama is one of the few states that doesn’t screen newborns for SMA, which could delay treatment until symptoms appear. “We’re working on it,” Coskery said. “We need to start treating these patients before they develop symptoms and then, hopefully, we will see fewer and fewer affected with the disease.”

Becoming one of the first centers to offer SMA treatments, she said, “puts us at the forefront of the gene replacement movement.” Indeed, the center is also beginning clinical trials for genetic therapies for children with Duchenne, one of the most common neuromuscular diseases.

The ability to provide such effective treatments to children who otherwise would have died before age 2 “is very profound,” Troxler said. “You can see the difference it makes on individual families in a really powerful way. Conversations historically been about how ‘this is a terrible disease that will likely end in your child’s death.’” Now, Troxler said, the conversation has changed to, “‘Your child has a horrible disease, but we have some therapies that are going to change what this looks like.’ It’s completely different.”

“There is a lot of excitement throughout the SMA community,” Coskery said. “We are very grateful when the parents send emails of the child standing and achieving milestones we didn’t think they would.”


[1] Thomas K. his New Treatment Could Save the Lives of Babies. But It Costs $2.1 Million. The New York Times. May 24, 2019.

[2] Biogen Announces New Data Further Establishing SPINRAZA® (nusinersen) as a Foundation of Care in Spinal Muscular Atrophy for a Broad Range of Patients. Available at: http://investors.biogen.com/news-releases/news-release-details/biogen-announces-new-data-further-establishing-spinrazar

[3] New Data Shows Long-Term Benefits of Zolgensma in SMA Patients. Available at: https://www.biospace.com/article/in-wake-of-data-manipulation-scandal-avexis-releases-long-term-efficacy-of-zolgensma-at-conference/

Hematology and Oncology, Inside Pediatrics, Neurology & Neurosurgery

Big Changes Coming to Children’s of Alabama’s Neuro-Oncology Division

Children's Cancer Hospital_WEB

The Pediatric Neuro-Oncology Program at Children’s of Alabama will launch new initiatives this year, including forming a relationship with Children’s Cancer Hospital in Cairo, Egypt, pictured above.

Girish Dhall, M.D., can quickly tick off the attributes of Children’s of Alabama’s Pediatric Neuro-Oncology Program that lured him from Children’s Hospital Los Angeles to become division director for the Hematology, Oncology, and Blood and Marrow Transplantation Program as well head of the Neuro-Oncology program here. “It’s a very robust and academically active program in the country,” he said. “One of the strong points is that we have four neuro-oncologists, four pediatric neurosurgeons, two pediatric neuroradiologists and one pediatric neuropathologist. This is not available in most hospitals in the country.”

Indeed, the Pediatric Neuro-Oncology Program at Children’s of Alabama is one of the strongest in the country, treating between 50 and 70 newly diagnosed patients a year and participating in cutting-edge clinical trials for children with brain tumors.

In 2020, Dhall and his team will welcome their first neuro-oncology fellow. “So we will train the next generation of pediatric neuro-oncologists,” he said.

“There is such a need to have more people trained,” Dhall said. “Pediatric brain tumors are the most common cancer in children and we don’t have enough people trained to treat them.”

“Neuro-oncology is very specialized,” said pediatric neuro-oncologist Laura “Katie” Metrock, M.D., who will run the fellowship program.  “The general hematology/oncology fellowship is more broad. This opportunity provides a full year of exposure to all aspects of neuro-oncology that are not available in the general fellowship.”

Metrock, who completed her own neuro-oncology fellowship at Emory University in Atlanta before coming to Children’s of Alabama, noted how much she appreciated her year of training. “I’m very excited to help others in that phase of their career,” she said. “If they choose to stay here then they will be helping kids in our community and if they go to other centers it provides more opportunity for collaboration.”

Metrock is also leading initiatives in neurofibromatosis (NF), which predisposes patients to nervous system tumors. “I took the position at Children’s of Alabama because I saw such a huge opportunity here,” she said. She is excited about working with the Neurofibromatosis Clinical Trials Consortium, which is based at the University of Alabama-Birmingham. The Consortium, directed by Bruce Korf, M.D., Ph.D., is a national leader in clinical and genetic studies on NF. “The ability to work directly in the front line of these trials coming through the Consortium was a huge opportunity,” Metrock said. “We have the opportunity to build something really special here for families and patients with NF.”

That includes a multidisciplinary clinic where children with NF-related cancers can obtain the variety of care they need from not only oncologists and hematologists, but geneticists, neurologists, endocrinologists and others. “We try to streamline their care and make sure they have access to other specialists, available therapies, and clinical trials,” Metrock said. That includes a partnership with the Pediatric Cancer Genetics Clinic. “We want to help the kids as they go through each phase and then ensure a smooth transition to the adult world,” Metrock said.

Among the other initiatives Dhall has begun is a global outreach in neuro-oncology. An oncologist from Vietnam recently trained in Alabama for two months and Dhall is now formalizing a relationship with the Children’s Cancer Hospital in Cairo, Egypt, which treats about 60 percent of the country’s entire pediatric cancer population. “We see about 150 pediatric cancer patients a year,” he said. “They see 4,000 a year. So the impact that we could have in helping train their doctors here is huge.”