Inside Pediatrics Winter 2018

P E D I A T R I C S

Fall/Winter 2018

CLAIMING INDEPENDENCE Children’s at Lakeshore merges clinical, transitional care

A nother year is almost behind us, of Alabama and pediatric enterprise. September saw the grand opening of Children’s at Lakeshore, a partnership among Children’s, the UAB Division of Pediatric Rehabilitation Medicine and the Birmingham-based Lakeshore Foundation, the internationally known organization that promotes physical activity among disabled individuals. What began as a remote idea among colleagues has evolved into a brick-and- mortar building on the Lakeshore campus staffed by UAB physicians and Children’s physical therapy, occupational therapy and speech-language pathology staff. This unique collaboration is designed to help each child achieve his/her highest level of independence, and Children’s is proud to be part of this opportunity to provide seamless care to patients. June marked a significant milestone for Children’s with our first-ever patient to be weaned from a pediatric ventricular assist device (VAD) without the need for a heart transplant. Our Pediatric Advanced Heart Failure and Transplant Team was among the first in the nation to use VADs in children with complex congenital heart disease, and this initial success was published in a landmark study in the New England Journal of Medicine. With continued use and study of VADs, our hope is to safely bridge children to and the passing months speak of a time of growth for both Children’s

transplantation or, in our recent patient’s case, to long-term recovery without transplant. Also this year, Children’s joined a handful of pediatric hospitals nationwide

to offer CAR T-cell therapy to children diagnosed with a difficult-to-treat blood cancer. The UAB Division of Pediatric Hematology and Oncology and the Alabama Center for Childhood Cancer and Blood Disorders at Children’s study a range of antibody immunotherapies like CAR T-cell to activate the immune system to attack cancer cells, and the early results have been promising. Last but not least, Children’s continues to expand its global footprint through the establishment of the Cystic Fibrosis Training Network for Latin America, with Children’s and UAB colleagues traveling to Chile to lay the foundation for a center network to improve patient care. Yes, 2018 will go down as a year of growth and advancement at Children’s of Alabama, and we resolve to continue our efforts to provide excellent patient care and education in the year ahead.

On the Cover: Four-year-old Kelsey Beasley plays in a rehabilitation room at Children’s at Lakeshore during its grand opening ceremony on September 6, 2018. Read more about the latest collaboration among Children’s of Alabama, the University of Alabama at Birmingham (UAB) and the Lakeshore Foundation on page 10.

Enjoy,

Children’s of Alabama 1600 7th Avenue South Birmingham, Alabama 35233

(205) 638-9100 childrensal.org

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Network for Change

ADMINISTRATION Mike Warren, President and CEO Coke Matthews, Executive Vice President Garland Stansell, Chief Communications Officer EDITORIAL Cassandra Mickens, Editor Trent Graves, Design

Kylin’s Brave Heart

Independent Streak

Amy Dabbs, Digital Content Denise McGill, Photography Patrick Deavours, Photography CONTRIBUTORS

Andre Green Adam Kelley

John Tracy Tina Wilson Mary Stephens Pugh Mike Strawn L. Amanda Owens PHYSICIAN MARKETING Tiffany Kaczorowski MEDICAL LEADERSHIP Mitchell Cohen, M.D. Katherine Reynolds Ireland Chair of Pediatrics, University of Alabama at Birmingham Physician-in-Chief, Children’s of Alabama Mike Chen, M.D. Joseph M. Farley Chair in Pediatric Surgery, University of Alabama at Birmingham Chief of Pediatric Surgery and Surgeon-in-Chief, Children’s of Alabama Lee I. Ascherman, M.D., M.P.H. Chief of Service, Child and Adolescent Psychiatry, University of Alabama at Birmingham For questions or additional information or to share feedback, please contact us at insidepediatrics@childrensal.org. An online version of the magazine is available at childrensal.org/insidepediatrics.

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Novel Cancer Therapy Squares Up Against Leukemia

News, Honors and Awards

CONT E N T

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NETWORK FOR CHANGE

The Children’s of Alabama and University of Alabama at Birmingham Cystic Fibrosis Center plays a leading role in efforts to develop multidisciplinary cystic fibrosis centers in Latin American countries.

F rom his office on the south side of Birmingham, Alabama, pediatric pulmonologist Hector Gutierrez, M.D., shares his passion to help cystic fibrosis (CF) patients around the world. In addition to being director of the University of Alabama at Birmingham (UAB) Division of Pediatric Pulmonology and the Cystic Fibrosis Center at Children’s of Alabama, Gutierrez is also principal investigator of the Cystic Fibrosis Training Network for Latin America (CFTN-LA). He leads a specialized team whose goal is to establish CFTN-LA as a permanent presence in Latin America. “Unlike the United States, most Latin American countries lack a center network to provide shared information and collaboration to help improve the care of patients. Because of this, they have been at a great disadvantage,” Gutierrez said. “A sustainable training program for CF centers can help improve quality of care, clinical outcomes and life expectancy for Latin American CF patients.”

The CFTN-LA strives to meet the same goals of the CF Foundation, which over the past 60 years has established itself as a global leader in rare disease research and advancement of new CF therapies in the U.S. The network aims to provide high-quality care for thousands of Latin American patients, to establish comprehensive, multidisciplinary CF centers and to address preventive barriers. Gutierrez said the network would open important avenues for future research and drug development. “Several factors limit the optimal CF delivery of care in these countries,” Gutierrez said. “Whereas diagnostic testing and medications are increasingly available here in the United States, there has been a paucity of expert manpower and the lack of a multidisciplinary team approach in Latin America.” Gutierrez said the CFTN-LA would lay the foundation for a well-integrated infrastructure for CF centers and their

teams, with future training efforts led in qualified Latin America centers, ensuring continued growth and sustainability of the network. Since 2014, Gutierrez has led eight benchmarking visits to Chile to help ensure access to high-quality care and establish the CFTN-LA. In turn, the Santiago, Chile-based Hospital Clínico San Borja Arriaran (HCSBA) CF team, among the partners in this endeavor, visited Birmingham for a two-week training to accelerate the project. Because the current Latin America healthcare delivery systems do not integrate or share information, patients may change from one system to another without proper follow-up and data acquisition. In addition, some health professionals essential to the CF team work as independent contractors to both public and private systems, resulting in higher costs to insurers and families. The CF Center at Children’s has proposed establishing a multidisciplinary

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CF Center in Santiago, Chile, providing care adhering to CF Foundation standards. Collaborative work has led to significant improvement in clinical outcomes and team expertise for a number of hospitals in Chile. In 2015, the HCSBA CF team was approached by five other Santiago-based hospitals that are now part of the project as affiliate hospitals. After the incorporation of the affiliate hospitals, the HCSBA CF Center renamed itself Regional CF Center Santiago. “This project started with one center, one hospital, and other hospitals in Santiago started to get interested,” Gutierrez said. “They joined by participating in our visits in Chile, and we provided similar information and feedback, but the first center

benefits, however, the structure of care deliveries, healthcare benefits, et cetera are different. Although we have started small, we feel we are making progress and hope to continue to do so in the years to come.” A mentorship by high-performing, U.S.-based CF teams, namely Children’s, UAB and the Baylor College of Medicine in Houston, plans to expand and train Latin America-based CF teams in Chile, Mexico and Uruguay. The mentorship would continue the development of an effective, adaptable and scalable training program for both mentors and trainees, and adapt transformative care management to local resources, culture, customers and healthcare delivery. ROSALIE’S STORY Rosalie Walker was only 2 weeks old when her parents, Rachel and Raven, received a call from her pediatrician telling them she may have cystic fibrosis (CF). Rosalie’s newborn screening test came back positive, indicating that Rosalie had inherited the disease. The preliminary results were confirmed by an immediate visit to Children’s of Alabama. “It was a shock for us to hear,” said Rachel, whose family lives in East Alabama. “Our baby was born healthy without any indication until the results of the screening test came back, so we went directly to Children’s in Birmingham for a follow-up sweat test.” A chloride sweat test helps diagnose CF, an inherited disorder that disrupts the normal function of epithelial cells that make up the sweat glands in the skin and also line passageways inside the lungs, liver, pancreas and digestive and reproductive systems. Rosalie’s hospital test results were confirmed. She inherited a double copy of the most common disease-causing gene mutation, delta F508, indicating that both of her parents were carriers. “When we learned that she had CF, the hospital staff was wonderful,” Rachel said. “It was very scary and an uncertain time for us, but they were very good about explaining everything about medications, breathing treatments and just the daily routine we needed to follow.” At 3 weeks old, Rosalie became ill with frequent vomiting and pneumonia. A bronchoscopy was performed to determine the type of bacteria in her lungs. “To facilitate her care during her hospital stay, Rosalie had an IV access placed [PICC line] and received antibiotics to treat the infection. She was discharged in good condition and has not been admitted since,” said Hector Gutierrez, M.D., director of the Cystic Fibrosis Center at Children’s. Now 3 years old, Rosalie visits Gutierrez and the Children’s CF team four times a year at its specialized CF outpatient clinic to maintain her health. She attends preschool and loves gymnastics, riding her bike, swimming and playing with her 7-year-old brother, Reed.

(HCSBA) that came to Birmingham became their leader and the point group for the others to learn from them.” Efforts thus far have shown a marked improvement in nutritional outcomes, lung function and microbiology among CF patients cared for by the participating hospitals in Chile, Gutierrez said. “We have been in discussion for two years with the government officials in Chile and other Latin American countries to work toward developing a solid CF network similar to what we have in the United States,” Gutierrez said. “We have explained the Pediatric pulmonologist Hector Gutierrez, M.D., is principal investigator of the Cystic Fibrosis Training Network for Latin America (CFTN-LA). For the past two years, Gutierrez and his team have worked with government officials in Chile and other Latin American countries to develop permanent CF networks there.

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KYLIN’S BRAVEHEART

An East Alabama toddler has made history at Children’s of Alabama, becoming the first patient to have a lifesaving ventricular assist device removed without needing a heart transplant.

One-year-old Kylin Harris is greeted with smiles during a celebration at Children’s of Alabama on June 5, 2018. 6

H earts are full at Children’s of Alabama because Kylin Harris’ heart is well. Inside a hospital conference room in June 2018, the banner was hung, the tablecloth was draped and the cake was cut in celebration of Kylin, the first-ever patient at Children’s to be successfully weaned from a pediatric ventricular assist device without the need for a heart transplant. The 1-year-old guest of honor arrived in what is by far Children’s most popular mode of transportation – a red Radio Flyer wagon – a stark contrast from her arrival just four months prior. Kylin was flown by helicopter to Children’s after her mother, Keianna Harris, came home to find her daughter uncharacteristically lethargic. Kylin hadn’t slept well the past two nights and a startled Harris, a nurse by trade, took Kylin to their local emergency room. While en route to the hospital, Kylin had a seizure and fell limp. Doctors at Children’s intubated Kylin and later diagnosed her with advanced heart failure due to myocarditis. Kylin experienced cardiac arrest while being transferred to the Cardiovascular Intensive Care Unit. Doctors performed chest compressions and CPR, and placed her on extracorporeal membrane oxygenation therapy (ECMO), which pumps and oxygenates a patient’s blood outside the body allowing the heart and lungs to rest for a limited time. The Pediatric Advanced Heart Failure and Transplant Team at Children’s discussed every detail of Kylin’s care with Harris. It was apparent ECMO would not provide long enough support for Kylin’s heart. First, doctors would convert Kylin from ECMO to the Berlin Heart® EXCOR, a long-term cardiac assist device that functions as a heart outside of the body. The Berlin Heart acts as a bridge to transplantation for children in heart failure, allowing the patient mobility and freedom to rehabilitate in preparation for transplant surgery. Available in several sizes, the Berlin Heart is not totally implanted inside the body. Doctors insert cannulas, or flexible tubes, into the heart, which extend through the skin and connect to a small pump located outside the body. That pump, along with its computerized drive unit, maintains blood flow.

Cardiothoracic Surgery. The research team, known as the Berlin Heart Study Investigators, evaluated ECMO and the Berlin Heart to see which offered children the best chance of survival until they could receive a new heart or recover enough heart function not to need a support device or transplant. Pearce says 46 percent of children diagnosed with heart failure die or receive a heart transplant within the first five years after diagnosis. A heart transplant is a child’s best hope of survival, with the survival rate after a transplant estimated at 83 percent at three years. However, with limited donor hearts available, the wait is often long. “Children on waiting lists for heart transplants experience the highest waiting-list mortality for any age or organ,” Pearce says. “This research demonstrates that the Berlin Heart, available in a variety of appropriate sizes for children, has the potential to effectively and safely bridge children from diagnosis to transplantation or in some cases recovery, for long periods of time.” Before the study and subsequent approval of the Berlin Heart by the U.S. Food and Drug Administration, ECMO was the mainstay for mechanical circulatory support as a bridge to transplantation. The effective period of support with ECMO is typically limited to 10 to 20 days before serious complications such as bleeding and major organ system failure occur, often prohibiting transplantation. The short duration of support afforded by ECMO is often inadequate, the study reads, citing only 40 to 60 percent of children requiring support with ECMO survive long enough to undergo heart transplantation. Researchers found that the longest duration of support for the younger, smaller children was 174 days for the Berlin Heart and 21 days for ECMO therapy. The longest duration of support for the larger, older children was 192 days for the Berlin Heart and 28 days for ECMO. “The Berlin Heart provided better survival over longer support periods than ECMO therapy,” Pearce says. “These longer support periods on the Berlin Heart allow for patient rehabilitation, improved nutrition and

“It was a lot to take in … It all happened so fast,” Harris said. “My biggest fear was losing her. It was scary not knowing whether she was going to make it through.” The Pediatric Advanced Heart Failure and Transplant Team, a partnership between Children’s and the University of Alabama at Birmingham (UAB), is among the first in the U.S. to use the Berlin Heart in children and reported the first successful Berlin Heart bridge to transplantation for a child with a single ventricle. Children’s and UAB first used the device in 2005, when UAB was home to the pediatric cardiac unit. The following year, the team weaned its first patient, now an adult, from the Berlin Heart without the need for transplant at UAB. In 2012, with the opening of the Benjamin Russell Hospital for Children and Bruno Pediatric Cardiac Unit, all pediatric cardiac care relocated to Children’s. The team’s success with the Berlin Heart led to a landmark study published in the New England Journal of Medicine in 2012 featuring two team members as co-authors – pediatric cardiologist F. Bennett Pearce, M.D., and David Naftel, Ph.D, professor in the UAB Division of Kylin was diagnosed with advanced heart failure with myocarditis. She was first placed on ECMO and then later the Berlin Heart once it was established ECMO would not provide long enough support. Photo submitted.

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weaning from ventilator support to use of the patient’s own respiratory system so that the children may be better candidates for successful transplantation or so that long- term cardiac recovery can occur.” Kylin lived with the Berlin Heart for 83 days. In that time span, doctors noted signs of cardiac recovery, and further testing gave doctors the green light to again, operate on Kylin to remove the device. Pearce credits fellow pediatric cardiologist Waldemar F. Carlo, M.D., who was the first to see hints of Kylin’s recovery. “He was the strongest advocate for us to explore whether her degree of recovery was enough for her to be off the device,” said Pearce, adding Kylin’s echocardiogram and catheterization data yielded positive results. “We followed

the protocol of the Berlin Heart Clinical Support Team. We were in daily communication with the team from the moment we considered weaning her off. “She has continued to show us normal cardiac function. We removed her from the transplant list because she had improved so much. We’re following her closely, but our hope is she won’t require a heart transplant,” Pearce said. And much to Harris’ delight and relief, her baby girl did make it through. Kylin is her old self again, she says, smiling from ear to ear. “When Kylin was in the hospital, she wouldn’t smile. She felt miserable,” Harris said. “But now her personality is back. She’s been grinning ever since we left.”

Kylin lived with the Berlin Heart for 83 days. Since being weaned off the device, “she has continued to show us normal cardiac function,” says her pediatric cardiologist F. Bennett Pearce, M.D.

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Recognized Among Nation’s Best by U.S. News and World Report

Children’s of Alabama is proud to be ranked among the Best Children’s Hospitals by U.S. News & World Report for the ninth consecutive year. Eight of our pediatric specialty services – Cancer, Cardiology & Heart Surgery, Diabetes & Endocrinology, Gastroenterology & GI Surgery, Neonatology, Nephrology, Neurology & Neurosurgery and Pulmonology – are ranked among the nation’s best. Our mission is clear – to provide health services to all children in an environment that fosters excellence in research and medical education. At Children’s of Alabama, we are committed to providing the very best in medicine to every child who comes to us for care. Yes, Nine Consecutive Years

Childrens AL .org

Erica Wilson, a wheelchair basketball player at the University of Illinois Urbana-Champaign, says she wouldn’t be where she is today without the support and care of Children’s of Alabama and the Lakeshore Foundation. “The two work hand-in-hand,” she says. Photo courtesy University of Illinois.

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INDEPENDENT STREAK

With the opening of Children’s at Lakeshore, Children’s of Alabama and the Lakeshore Foundation further solidify a partnership with a common goal of encouraging independence and healthy competition among disabled youth.

E rica Wilson was unable to attend the grand opening ceremony for Children’s at Lakeshore this past September, but her absence from the festivities captures the mission of the new partnership between Children’s of Alabama and the Lakeshore Foundation. Wilson, 20, is a college junior studying kinesiology at the University of Illinois Urbana-Champaign, where she earned a scholarship to play point guard and shooting guard for the Fighting Illini women’s wheelchair basketball team. She lives independently in an apartment that is about 600 miles away from her hometown of Childersburg, Alabama. She drives her own car. She lives a life she couldn’t have imagined eight years ago. On February 23, 2010, Wilson was a 12-year-old in love with dance. She had spent the last eight years honing her tap,

jazz and ballet skills, and she was set to take up pointe ballet. She was playing basketball in her middle school physical education class when she felt an intense pain in her lower right calf. “It felt like somebody was squeezing my muscles from the inside out,” Wilson said. The pain spread from Wilson’s right calf to her left leg and up to her waist. When she arrived at the door of Children’s Emergency Department, she couldn’t get out of the car. She couldn’t feel her legs. Wilson was diagnosed with transverse myelitis, a neurological disorder caused by inflammation of the spinal cord. She has since regained some sensation and movement in her legs. The news stunned Wilson and her family, but thanks to the quick diagnosis by pediatric neurologist Jayne Ness, M.D., Wilson received immediate treatment.

After evaluation by the physicians in the University of Alabama at Birmingham (UAB) Division of Pediatric Rehabilitation Medicine, Wilson started intensive inpatient rehabilitation physical and occupational therapy. She spent six weeks in inpatient therapy adjusting to her new normal, learning how to use a wheelchair and modifying daily life activities. She admits she wasn’t always the most pleasant patient. “I was 12, hormonal and upset. I’m sure I was a handful.” Wilson said. “But my experience at Children’s was the best it could have been. They helped me realize that because this quote-unquote bad thing happened to me, my life wasn’t over. There are ways to adapt.” During her time at Children’s, Wilson’s occupational therapist mentioned the Lakeshore Foundation, a Birmingham-

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based nonprofit rehabilitation center that promotes active, independent lifestyles for people with physical disabilities or chronic health conditions, and serves as a training site for the U.S. Olympic and Paralympic teams. Unwilling to let go of her passion for dance, Wilson brushed the suggestion off, but two years later, her mother nudged her to attend a summer camp at Lakeshore. Wilson promised she would stay one day. That was the day she fell in love with wheelchair basketball. She became a starting player for the Lakeshore Lakers wheelchair basketball team and was selected as one of five inspirational athletes nationwide to be featured in Gatorade’s Win From Within video series campaign. “Being in an environment with people who didn’t judge my disability was pretty awesome,” Wilson said. “Lakeshore helped me learn that even though I’m in a wheelchair, there are people going through the same experience and doing amazing things.” Heather Baty, Divisional Director of Ambulatory Operations at Children’s, said Wilson’s journey is the crux of Children’s at Lakeshore – to help children achieve their highest level of independence. With physician care and therapy services provided by the UAB Division of Pediatric Rehabilitation Medicine and Children’s Physical Therapy, Occupational Therapy and Speech-Language Pathology staff, respectively, Children’s at Lakeshore merges a medically based model of care with community-based healthy living and transitional services. “Bringing the Children’s at Lakeshore facility to the Foundation campus provides an opportunity for a more seamless transition to support our patients in pursuing an active lifestyle,” Baty said. Lakeshore President Jeff Underwood said Children’s and Lakeshore have enjoyed a successful partnership for many years. The addition of Children’s at Lakeshore stands to broaden the impact of each organization’s mission. “We are looking forward to expanding our collaboration and strengthening our relationship with Children’s so that more parents and their children with physical disabilities can learn about the opportunities available to them at Lakeshore,” Underwood said. Medical professionals evaluate and provide patients with a customized and comprehensive rehabilitation, physical, occupational and/or speech-language therapy treatment program specifically designed for their needs in an outpatient program. According to Drew Davis, M.D., division director of the UAB Division of Pediatric Rehabilitation Medicine who treated Wilson at the time of her initial illness and still follows her progress today, “Erica really exemplifies what is possible when children are able to take advantage of access to both high-quality healthcare and world- class community-level sports, fitness and recreation opportunities. With the opening of Children’s at Lakeshore, we are building a bridge between the medical rehabilitation model and the community-based model of healthy living with the goal of helping all of our patients achieve the highest level of independent function.”

Wilson is confident that Children’s at Lakeshore will be a source of healing and hope for patients and their families. This past summer, she worked as an aide at Children’s on 3rd Outpatient Center, which houses specialty clinics for physical and occupational therapy, and speech-language pathology. Inspired by her experience as both a patient and employee, she plans to enroll in graduate school and pursue a career in occupational therapy. “Children’s and Lakeshore connectedly got me to where I’m at today. The two work hand-in-hand.” Wilson said. “I can do all of these things I thought I wouldn’t be able to do, and that’s pretty awesome.”

Wilson visits with Drew Davis, M.D., medical director of the UAB Division of Pediatric Rehabilitation Medicine, at Children’s of Alabama in 2016. “Erica really exemplifies what is possible when children are able to take advantage of access to both high-quality healthcare and world-class community-level sports, fitness and recreation opportunities,” Davis says.

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ABOUT LAKESHORE FOUNDATION

Children’s helped me realize that because this quote-unquote bad thing happened to me, my life wasn’t over. There are ways to adapt.

Lakeshore Foundation is a nonprofit organization located in Birmingham, Alabama, that provides opportunities for people with a physical disabilities or chronic health conditions to be active and independent through physical activity, research and advocacy and policy.

ACTIVITY

Lakeshore’s aquatics, fitness, recreation and athletic programs get everyone moving. It is distinct as a U.S. Olympic and Paralympic Training Site that brings elite athletes from across the globe to train and compete.

RESEARCH

The UAB/Lakeshore Research Collaborative develops and evaluates new programs and technology in exercise, sport science, health promotion and rehabilitation engineering.

ADVOCACY

The Lakeshore advocacy and policy team invites people with and without disabilities to be engaged, informed and empowered to create partnerships that promote inclusion, access and equal representation in local, national and global policy initiatives.

NCHPAD

The National Center on Health, Physical Activity and Disability (NCHPAD), part of the UAB/Lakeshore Research Collaborative housed at Lakeshore, works to improve the health and wellness of people with disabilities by supporting accessible and inclusive public health promotion programs. NCHPAD also provides individualized information through web-based materials and health communication.

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NOVEL CANCER THERAPY SQUARES UP AGAINST LEUKEMIA

Children’s of Alabama is among a handful of healthcare facilities nationwide to offer chimeric antigen receptor T-cell therapy, which has shown promise in fighting hard-to-treat blood cancers.

W restling is 12-year-old Hayden Parker’s favorite sport to watch, but in his day-to-day life, he takes on an opponent far more challenging than his favorite televised wrestling competitor. For the past four years, Hayden has personally wrestled with pre-B cell acute lymphoblastic leukemia (ALL), a cancer of the white blood cells. When Hayden was diagnosed, he and his father, Bryan Qualls, who live just outside Birmingham, Alabama, were faced with the unexpected. Following a visit to the pediatrician for Hayden’s fever and bruising on his legs, Qualls received a call he never expected. “The pediatrician told us to go immediately to Children’s of Alabama because Hayden’s lab work results looked like he had leukemia,” said Qualls. That day, Hayden became one of the approximately 3,000 children and adolescents who are diagnosed with the disease in the U.S. each year. “Typically, about 90 percent of ALL patients are cured with intensive chemotherapy treatment,” said Matthew Kutny M.D., director of the Leukemia, Lymphoma and Histiocytosis Program in the University of Alabama at Birmingham (UAB) Division of Pediatric Hematology-Oncology and the Alabama Center for Childhood Cancer and Blood Disorders at Children’s of Alabama. “Unfortunately, Hayden’s leukemia has been harder to cure,” Kutny said. “He has been through a number of different cycles of chemotherapy treatments and has not stayed in remission.” Over the years, Hayden has experienced several relapses and life-threatening health issues due to the harsh effects of chemotherapy. He has lived with a compromised immune system and some of his organs have been adversely affected. “He would take all of the medications, the treatments, and then relapse, and then he would have to do it all over again,” Qualls said. “You don’t want to have to see your kid go through this – it’s just heartbreaking. As a parent you just want to protect them, and you can’t take this away from them. They just have to fight it, and you are their support staff. You have to cheer them on to just keep going.”

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In 2018, Hayden was faced with very few options to continue fighting his cancer. Kutny and his team recommended he try an FDA-approved immunotherapy called chimeric antigen receptor (CAR) T-cell therapy. Children’s is one of about 35 healthcare institutions in the U.S. and the only pediatric provider in Alabama chosen to offer the treatment intended for children and young adults with ALL. “CAR T-cell therapy is truly a breakthrough therapy in cancer treatment,” Kutny said. “The CAR T-cell therapy takes a patient’s own immune cells, white blood cells that normally fight infection, and programs the cells to recognize and destroy the patient’s leukemia cells.” The U.S. Food and Drug Administration requires all treatment sites providing CAR T-cell therapy have certification and training to handle potential risks and complications involved with this type of treatment such as high fevers, low blood pressure and difficulty breathing that may require a stay in the intensive care unit. Investigators at Children’s and UAB are working to create a new strategy in CAR T-cells that can better control this immune response or shut it off once it is no longer needed. “We are excited about this therapy approach because it generally results in less toxicity and side effects compared to

other chemotherapy used to treat leukemia and lymphoma,” Kutny said. “Hayden and his father have always been troupers through his journey, and they have kept a positive attitude. Hayden is really into wrestling and that really encapsulates Hayden’s approach to taking on his cancer. He is always ready for the next fight to take it on.” Kutny says Hayden has responded very well to the CAR T-cell therapy so far. “We are watching closely how he responds to the treatment, and are hopeful this will be a cure for him,” Kutny said. Children’s is also studying a range of antibody immunotherapies. These therapies can activate the patient’s immune system to attack the cancer or the antibodies can be linked to chemotherapy medicines in order to more precisely deliver the medicine to the cancer cells. “This can result in fewer side effects compared to our traditional systemic chemotherapies,” Kutny said. “We have used these antibody therapies with great success in patients with relapsed leukemia and lymphoma. Now we are studying how to replace traditional chemotherapy with these immunotherapies in order to decrease the side effects of cancer treatment and further improve cure rates.”

Twelve-year-old Hayden Parker and his father, Bryan Qualls, check in with pediatric hematologist/oncologist Matthew Kutny, M.D., at Children’s of Alabama in August 2018. Hayden, diagnosed with acute lymphoblastic leukemia, is currently undergoing chimeric antigen receptor T-cell therapy, “truly a breakthrough therapy in cancer treatment,” Kutny says.

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News, Honors and Awards

FEIG ELECTED TO AAP SECTION ON NEPHROLOGY EXECUTIVE COUNCIL Daniel Feig, M.D. , Pediatric Nephrology, has been elected to the American Academy of Pediatrics Section on Nephrology Executive Council. Feig will serve on the council beginning November 1, 2018, and the term will extend through October 31, 2021. LADINSKY WINS INCLUSIVE RESEARCH & INITIATIVE AWARD Morissa Ladinsky, M.D. , Academic General Pediatrics, was awarded the Inclusive Research & Initiative Award at the Lavender Celebration on April 11. The Lavender Celebration is an annual event in the spring of each academic year wherein LGBTQ undergraduate, graduate and professional students receive recognition for both their contribution to campus and their success in graduating. During the celebration, awards are given out to students, faculty and staff. Ladinsky was honored for her work on a project to make UAB Medicine and our trained physicians and healthcare providers better at serving transgender patients. McKINNEY APPOINTED TO ADMISSIONS SELECTION COMMITTEE FOR UAB SCHOOL OF MEDICINE Jaime McKinney, M.D. , Academic General Pediatrics, has been appointed to the Admissions Selection Committee for the UAB School of Medicine. The Admissions Selection Committee reviews candidates and determines who will be accepted to join the School of Medicine. STALVEY ELECTED TO UAB FACULTY SENTATE Michael Stalvey, M.D. , Pediatric Endocrinology, was elected to the UAB Faculty Senate as a representative for the School of Medicine. Stalvey will serve as a Senator from September 1, 2018, to August 31, 2020. PEDIATRIC FACULTY AMONG 2018 DEAN’S EXCELLENCE AWARD RECIPIENTS Fifteen outstanding faculty members were named recipients of the 2018 Dean’s Excellence Awards, an honor recognizing exceptional contributions made by School of Medicine faculty in service, teaching, research, diversity enhancement and mentorship. Among the recipients are Stephen Rowe M.D., MSPH , Pulmonary Medicine, for Excellence in Research; Stephanie Berger M.D. , Hospital Medicine; Michael Barnett M.D., MS , Palliative Care, for Excellence in Teaching; and Jaime McKinney M.D. , Academic General Pediatrics, for Excellence in Diversity Enhancement. PEARCE RECEIVES CERTIFICATE Bennett Pearce, M.D. , Pediatric Cardiology, completed the Graduate Certificate in CERT Health Care Quality Safety in April. The Graduate Certificate is developed for healthcare leaders who desire to move their organizations forward in this value-driven healthcare system by spearheading quality and patient safety improvement initiatives.

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TURNER COMPLETES MOMENTUM LEADERSHIP PROGRAM Cathy Turner, CPA, CGMA, Executive Administrator of UAB Pediatrics, has completed the MOMENTUM leadership program (Class 15). MOMENTUM, launched in 2002, accepts approximately 25 women annually to participate in its prestigious program. The curriculum is designed to provide tools and resources to inspire and educate women to serve in leadership roles, network these leaders to learn and work on problems together, enhance the image of executive women in business and community, and attract and retain the nation’s brightest women to help solve business and community challenges. SALAS RECEIVES RESEARCH GRANT, YOUNG PHYSICIAN AWARD Ariel Salas, M.D. , Neonatology, has received a pilot research award from the Obesity Health Disparities Research Center. He was awarded $50,000 for his project “Serial Assessments of Body Fat Accrual in Very Preterm Infants.” Salas was also awarded the Samuel J. Fomon Young Physician Award endowed by the Nestlé Nutrition Institute. This award is one of the National Scientific Achievement Awards given out by the American Society of Nutrition Foundation. These awards are given in honor of scientists, clinicians and scholars for significant achievements in nutrition research and practice. BHATIA NAMED FELLOW OF AMERICAN SOCIETY OF CLINICAL ONCOLOGY Smita Bhatia, M.D. , Pediatric Hematology/Oncology, has been named a Fellow of the American Society of Clinical Oncology. As mentioned on the ASCO website, “the Fellow of the American Society of Clinical Oncology (FASCO) distinction recognizes ASCO members for their extraordinary volunteer service, dedication, and commitment to ASCO. Their efforts benefit ASCO, the specialty of oncology, and, most importantly, the patients at risk for or with cancer.” SASSER NAMED AS NEW ASSISTANT PROGRAM DIRECTOR FOR PEDIATRIC RESIDENCY The Pediatric Residency Program announced Will Sasser, M.D. , Pediatric Critical Care, joined the program as an Assistant Program Director as of July 1, 2018. When asked about his new role, Sasser said, “I’m thrilled about the opportunity to work more closely with our residents alongside faculty and staff who have meant so much to me as a trainee and junior faculty member.” FRIEDMAN TO LEAD DEVELOPMENTAL THERAPEUTICS Gregory Friedman, M.D. , Pediatric Hematology/Oncology, has been appointed the new Phase 1 PI at Children’s of Alabama and approved by the DVL Executive Committee of the Children’s Oncology Group. In addition, Friedman will lead Developmental Therapeutics in the Division of Hematology-Oncology and the Department of Pediatrics. NEW APTC MEMBERS SELECTED Pallavi Iyer, M.D. , Pediatric Endocrinology, has been elected by the faculty to serve a three-year term on the Pediatric Appointment, Promotion, and Tenure Committee (APTC). Gregory Friedman, M.D. , Pediatric Hematology/Oncology, and Susan Walley, M.D. , Pediatric Hospital Medicine, have been selected to fill the two vacancies for appointed faculty on the APTC.

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KANDASAMY SELECTED FOR PRS YOUNG INVESTIGATOR AWARD Jegen Kandasamy, M.D. , Neonatology, has been selected for an award under the Perinatal Research Society Young Investigator program to attend the upcoming Perinatal Research Society Annual Meeting and the NIH - Abbott Nutrition Pre- meeting Workshop September 5-9, 2018, in Englewood, Colorado. Selection into this program is highly competitive. This is the third year in a row that one of our neonatal junior faculty has been selected to attend the pre-meeting workshop. LADINSKY AWARDED HOPE FOR TOMORROW AWARD Morissa Ladinsky, M.D. , Academic General Pediatrics, received the Hope for Tomorrow Award at the inaugural LGBTQ+ Community Service Awards and Gala. She was honored for her work in caring for and advocating for all youth. ASHWORTH APPOINTED AS PROFESSOR EMERITA Carolyn Ashworth, M.D. , Academic General Pediatrics, has been appointed as Professor Emerita in the UAB School of Medicine. She was appointed to this role in recognition of her outstanding service and years of dedication to the Department of Pediatrics. CRON APPOINTED TO ACR LEADERSHIP ROLE Randy Cron, M.D. , Pediatric Rheumatology, has been selected to chair the American College of Rheumatology (ACR) Rheumatology Research Foundation (RRF) Pediatric Rheumatology Visiting Professorship Selection Committee. The commitee matches up selected pediatric rheumatologists with pediatric programs across the U.S. that do not have a pediatric rheumatology presence for a three to four-day educational visit. Cron will serve a three-year term. WHITLEY SELECTED FOR ALEXANDER FLEMING AWARD Richard Whitley, M.D. , Pediatric Infectious Disease, has been awarded the 2018 Alexander Fleming Award for Lifetime Achievement by the Infectious Disease Society of America (IDSA). The Alexander Fleming Award for Lifetime Achievement is granted to an IDSA member or fellow in recognition of a career that reflects major contributions to the field of infectious diseases. STOOPS AWARDED P&F GRANT FOR BABY NINJA PROJECT Christine Stoops, D.O. , Neonatology, was recently awarded a P&F Grant to continue the Baby NINJA (Nephrotoxic Injury Negated by Just-in-Time Action) project at Children’s of Alabama and incorporate other NICUs in the NINJA Collaborative. The award is from the pilot and feasibility program of the O’Brien Center of Excellence in Pediatric Nephrology at Cincinnati Children’s Hospital Medical Center. BRITT, CARLO APPOINTED DISTINGUISHED PROFESSORS William Britt, M.D. , Pediatric Infectious Disease, and Waldemar Carlo, M.D. , Neonatology, have been appointed as Distinguished Professors in the UAB School of Medicine in recognition of their outstanding academic accomplishments and peer recognition.

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McGUINN NAMED DDBP DIVISION DIRECTOR Laura McGuinn, M.D. , has been named UAB Division Director for Developmental and Behavioral Pediatrics (DDBP) beginning January 1, 2019. McGuinn is currently the fellowship program director at the University of Oklahoma and is nationally recognized for her leadership positions in the American Academy of Pediatrics relating to developmental and behavioral pediatrics. McGuinn completed medical school and residency at University of Texas Medical Branch in Galveston, Texas, and a fellowship in developmental and behavioral pediatrics at Johns Hopkins Hospital, Baltimore, Maryland. McGuinn will work closely with her division; colleagues in the Sparks Clinics and Children’s Behavioral Health; child psychiatry; Tony Fargason, M.D., director of neurodevelopmental initiatives; and Craig Powell, M.D., incoming chair of Neurobiology and The Civitan International Research Center. McGuinn will be a terrific addition to our growing commitment to diagnosis and treatment of children with autism spectrum disorders, developmental delay, cognitive/ intellectual disabilities and attention deficit disorders/learning concerns. KONG NAMED WOMAN OF DISTINCTION BY GIRL SCOUTS Michele Kong, M.D. , Pediatric Critical Care, has been chosen as a 2018 Woman of Distinction for the Girl Scouts of North-Central Alabama. The Girl Scouts have been honoring Women of Distinction in Alabama since 1993. The Women of Distinction Award honors women who have made special contributions to their communities through civic, academic or professional efforts and who are exemplary role models for today’s girls. BARNES ACCEPTED INTO CLINICAL INVESTIGATOR TRAINING PROGRAM Margaux Barnes, Ph.D. , Gastroenterology, Hepatology and Nutrition, was accepted into the Clinical Investigator Training Program at UAB. This program is a first of its kind at UAB, a collaborative effort of UAB’s School of Medicine, Center for Clinical and Translational Science (CCTS), Comprehensive Cancer Center and Department of Medicine, and supported by funding from the Health Services Foundation General Endowment Fund/CCTS.Fifteen candidates are chosen from across campus. The program is a pragmatic, hands-on program focused on preparing new investigators to plan and implement human subject investigation in a Good Clinical Practice- compliant manner. SIMPSON RECEIVES INVISIBLE WARRIOR AWARD FROM BHAM BLACK PRIDE Bham Black Pride held their inaugural banquet on August 17, during which Tina Simpson,. M.D. , Adolescent Medicine, was named the first recipient of the “Dr. Tina Simpson Invisible Warrior Award.” The award, named in her honor, recognizes Simpson as the longest-serving African-American physician caring for children, adolescents and young adults with HIV in the Birmingham area. In the future, this award will recognize those who have made contributions to advance equality and create strong connections in the lives of LGBTQ Alabamians. SCHWARTZ TO JOIN INNOVATION ACADEMY Justin Schwartz, M.D. , Developmental and Behavioral Pediatrics, will join the Innovation Academy’s “Train the Trainer” program. This program is a unique, inter- professional approach to analyze and develop solutions for current challenges in healthcare delivery. The program hopes that participants will return to their respective units armed with new knowledge and a thorough understanding of the process of innovating at UAB Medicine. A key goal of this Academy is the creation of a cohort of innovation coaches dispersed throughout the organization.

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1600 7th Avenue South Birmingham, Alabama 35233

CMV 2019 Birmingham, Alabama April 7 - 11, 2019

7TH INTERNATIONAL CONGENITAL CMV CONFERENCE & 17TH INTERNATIONAL CMV WORKSHOP

IMPORTANT DATES Monday, 01 October 2018: Registration & Abstract Submission Sites Open Friday, 30 November 2018: Early Registration & Abstract Submission Deadline Friday, 11 January 2019: Abstract Disposition Notices Sent Friday, 22 February 2019: Standard Registration & Cancellation Deadline Sunday, 07 April 2019: See you in Birmingham!

To contact the CMV 2019 Conference Planner, please email Conference Solutions at CMV@ConferenceSolutionsInc.com. Visit cmv2019.org to learn more or register.