The Division of Pediatric Neurosurgery maintains a robust clinical research commitment that addresses each major domain in Pediatric Neurosurgery. We have a long-standing history of active participation in prospective clinical registries, including the Hydrocephalus Research Network (HCRN), the CDC-sponsored National Spina Bifida Patient Registry (NSBPR) and the Park-Reeves Consortium for Chiari Malformation Research. We have continued to lead most registries in enrollment and have used these databases to conduct studies about important clinical questions in Pediatric Neurosurgery.
During the last year, researchers in the Division of Pediatric Neurosurgery have published more than 39 papers, presented more than 40 papers, and mentored more than 30 medical and public health students. Dr. Andrew Hale, a fourth-year Neurosurgery resident physician, won the prestigious Shulman Prize for quality research by a trainee in Neurosurgery at the annual Pediatric Section meeting of the AANS/CNS. Selected topics of active investigation include the following studies:
Endoscopic versus Shunt Treatment of Hydrocephalus in Infants (ESTHI)
PI: Dr. Rozzelle in collaboration with the HCRN
Objective: This is a randomized, multi-center, prospective clinical trial that seeks to determine in infants with hydrocephalus requiring treatment if endoscopic treatment of hydrocephalus (ETV+CPC) compared to shunt results in non-inferior cognitive outcome at 12 months from surgery.
Bioengineering Strategies to Investigate Shunt Revisions
PI: Dr. Rocque in collaboration with Dr. Caroline Harris, Wayne State University, Detroit, Michigan.
Objective: To understand how shunts fail in the treatment of hydrocephalus by characterizing the cellular pathology in and around shunt systems to identify what has blocked the catheter with a hypothesis that the number of past revisions increases the number of inflammatory cells present and blocking the shunt system. This work is done collaboratively with colleagues at Wayne State University in Michigan. Shunts are collected at surgery and undergo microscopic histologic and immunohistochemical analysis to discern the materials comprising the plug that leads to catheter obstruction.
Oncolytic Herpes Virus (HSV) for Medulloblastoma:
PI: Dr. Johnston in collaboration with Dr. Friedman from UAB/COA Hematology/ Oncology
Objective: Dr. Johnston carried out a phase 1 study using modified HSV to treat supratentorial high-grade glioma in children. This work is the pediatric extension of work developed within the Department of Neurosurgery at UAB in the laboratories of Dr. James Markert and Dr. Yancey Gillespie. Genetically modified herpes viruses (G207) were stereotactically injected into the resection cavities where tumors are removed. The initial 12 patients treated with G207 demonstrated safety of the technique, and the results were published in the New England Journal of Medicine. The current study is ongoing for treatment of cerebellar tumors with three patients treated to date. Multi-center phase II and NIH grant submission are underway.
PI: Dr. Johnston in collaboration with Dr. Ryan Miller/Dr. Anita Hjelmeland, UAB
Objective: To create a diverse biorepository of pediatric brain tumor tissue. After tumor tissue excised at surgery has been allocated for pathologic review, specimens are collected, compiled and stored to enable current and future molecular evaluation.
Craniopharyngioma and Biological Targets for Medical Therapy
PI: Dr. Johnston in collaboration with colleagues at University of Colorado.
Purpose: A prospective database of craniopharyngioma patient characteristics and tissue is being compiled to determine other therapeutic targets for craniopharyngioma and clinical characteristics of this patient population. Craniopharyngioma is an uncommon but well recognized brain tumor that is associated with very high morbidity, despite non-malignant histologic characteristics. Only modest success has been realized in effective treatment, and it is anticipated that collaborative, multi-centered investigation will contribute to improving this.
National Spina Bifida Patient Registry (NSBPR)
Almost 650 pediatric (and 145 adult/transition) patients have been enrolled from Children’s of Alabama/UAB into the NSBPR. This represents the largest enrollment of any center in the United States. Within the registry, several topics are under active investigation, including the impact of body composition upon SB morbidities, sleep disturbances in MMC and multiple studies pertaining to transitional care.
Chiari Surgical Success Score
PI: Dr. Rocque
Objective: To develop and internally validate the Chiari Surgical Success Scale (CSSS), a prognostic model capable of identifying the probability that clinical and radiologic variables will predict a favorable response to surgery in patients with Chiari Malformations.
CPRN Clinical Registry: Cerebral Palsy Research Network
PI: Dr. Roque
Purpose: The CPRN is a clinical registry that strives to characterize patient populations, treatments and outcomes in patients with cerebral palsy. By better understanding the variability, progression and current treatment practices for CP, therapeutic interventions for care can be optimized. Surgical treatments under study include selective dorsal rhizotomy and intrathecal Baclofen pump placement.
Global Surgery: The Lancet Commission on Global Surgery, published in 2015, showed that the global burden of disease that was treatable by surgery was large and growing. The Division of Pediatric Neurosurgery has prioritized outreach in Global Surgery during 2021, despite the pronounced limitations imposed by the COVID-19 pandemic.
Intersurgeon: Dr. James Johnston is the co-founder and current director of Intersurgeon. Intersurgeon is an interactive website designed to bring individuals and organizations together in partnerships to improve surgical care globally.
Development of Smart Glasses Technology: Low- and middle-income countries (LMIC) are plagued by a severe shortage of qualified and available surgeons. Surgical missions can assist local surgeons temporarily, especially for advanced procedures, but longer-term solutions are required. Surgical Sight Smart Glass Technology enables real-time projection of surgical fields around the world to enable real-time support, teaching and guidance. Drs. James Johnston and Brandon Rocque are actively working to pioneer this promising technology and have several studies evaluating effectiveness underway.
The Global Alliance for the Prevention of Spina Bifida-F (GAPSB-f): Even though it has been proven for 30 years that much of the world burden of Spina Bifida (SB) can be eliminated with fortification, only 56 countries fortify food sources with folic acid. Less than one quarter of preventable SB is currently being prevented, and the human and fiscal cost of this is extreme. We have developed and coordinated an international consortium of neurosurgeons, epidemiologists, nutritionists and other key stakeholders to embrace and facilitate science-based advocacy for folic acid fortification of foods and salt.
Dr. Ariana Barkley is the fellow in Pediatric Neurosurgery for 2021–2022. She is an honors graduate of the University of Pennsylvania School of Medicine and the University of Washington Neurosurgery Residency program. Dr. James Johnston is the Pediatric Neurosurgery fellowship director.
Jeffrey P. Blount M.D., MPH
James M. Johnston, M.D.
Brandon G. Rocque, M.D., MS
Curtis J. Rozzelle, M.D.