Clinical Advances and Research Initiatives from Duke Pediatrics

The Duke University Department of Pediatrics' commitment to excellence, innovation, and leadership is evident in our world-class patient care with outstanding outcomes, esteemed medical education, groundbreaking research, and dynamic advocacy.

Building on Duke’s long tradition of excellence in pediatrics, below is a collection of recent clinical advances and research initiatives in our subspecialty departments.

Our Pediatric Heart Program delivers expert care for a range of delicate and demanding heart surgeries, from structural defect repair to heart transplants. For the most complex congenital and pediatric heart surgeries, the last 3 years’ mortality rates are below 1% compared to a national average of 2.5-3%.

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• We established a single ventricle program to treat a range of defects, including hypoplastic left heart syndrome, unbalanced complete atrioventricular canal defect, pulmonary atresia with intact ventricular septum, and complex transposition of the great arteries.
• Our heart surgeons developed a new approach for the Norwood operation that reduces the risk for organ malfunction and increases the chances of survival for newborns. The Sustained Total All-Region (STAR) perfusion method perfuses the head, lower body, and heart continuously to minimize ischemic injury.
• We pioneered a minimally invasive technique to replace pulmonary valves in children born with pulmonary valve stenosis, which allows faster healing.
• Breakthroughs in transcatheter interventions include transcatheter pulmonary valve replacement, patent ductus arteriosus (PDA) stent placement, and closure of PDA occlusions in extremely premature infants.

Duke’s pediatric neurologists and neurosurgeons use the latest technology and approaches, including minimally invasive techniques, to treat pediatric brain and spinal tumors, cysts, and trauma, as well as epilepsy, spasticity, and other abnormalities.

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• Duke is a Level 4 Epilepsy Center as designated by the National Association of Epilepsy Centers.  We also have the region’s largest multidisciplinary pediatric autoimmune brain disease and neuromuscular programs.
• We offer advanced surgical techniques including robotic-assisted stereo EEG, source localization, laser ablation, deep brain stimulation, intraoperative MRI, virtual reality, and 3D printing technologies.
• Duke is a national leader in Duchenne Muscular Dystrophy and spinal muscle atrophy gene therapy trials and translational science efforts, including CRISPR-based gene editing and viral vector engineering.
• Our neurologists are developing a novel technology to address infant behavioral insomnia, with a clinical trial planned for summer 2021.
• We are members of the COllaborative Network for NEuro-oncology Clinical Trials (CONNECT) Consortium and the Pacific Pediatric Neuro-Oncology Consortium (PNOC), enabling us to enroll patients for a host of exciting surgical and medical trials to treat pediatric brain tumors.
• Our neurosurgery lab, funded by both the NIH and DoD, studies the mechanisms of oncolytic viral therapy to treat medulloblastoma and the role of ABL1 and ABL2 as drivers of leptomeningeal dissemination of medulloblastoma.

Duke’s Pediatric Oncology and Cell Therapy department offers leading-edge care from a highly skilled team that uses the latest techniques to diagnose and treat cancer.

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• Duke is at the forefront of research into gene therapy for treating sickle cell disease.
• We have an internationally renowned transplant center for inherited metabolic diseases.
• We are leaders in intrathecal cell therapy for inherited metabolic disorders, including DUOC-01—a cord-blood derived, macrophage-based cell therapy product.

Duke Pediatric Orthopaedics continues to be at the forefront of delivering high-quality, multidisciplinary patient care, research, and education for scoliosis, limb disorders, and complex neuromuscular disorders for children from infancy to adolescence.

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• We were the first center in NC to perform the anterior vertebral body tethering procedure for adolescent idiopathic scoliosis, and the only center in NC, SC, VA, or MD with faculty participating in the International Hip Dysplasia Institute.
• Our multidisciplinary scoliosis center provides nonoperative care from Barcelona Schroth–certified physical therapists and dedicated orthotists who specialize in spinal orthoses.
• Our faculty co-authored the guidelines for the orthopaedic care of children with Duchenne Muscular Dystrophy.
• We hold leadership positions on educational boards for international societies such as the Scoliosis Research Society.

Duke’s pediatric urologists treat children across the spectrum of complexity; from common conditions such as bed wetting, urinary tract infections, and incontinence to complex care. Complex procedures include repairing multi-system birth defects, treating kidney and/or bladder cancers, and performing reconstructive surgery for complex urologic conditions, including revisions of previous surgeries.

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• Our urology group leads the nation in management of the care of children and adults with spina bifida; we are active participants and leaders in CDC- and Spina Bifida Association-funded efforts to improve the quality of spina bifida care (National Spina Bifida Patient Registry (NSBPR) and Urologic Management to Preserve Initial Renal Function Protocol for Young Children with Spina Bifida (UMPIRE)).
• Our urologic surgeons are national leaders and active researchers in pediatric cancer care, particularly Wilms tumor and rhabdomyosarcoma.
• The Duke Center for Child and Adolescent Gender Care was the first multidisciplinary clinic in the Carolinas to provide high-quality, comprehensive, and compassionate family-centered care to transgender or gender-expansive youth, and children with differences of sex development.