2023 Department of Pediatrics Academic Annual Report

Dr. Lowell is the pediatric principal investigator for the American Lung Association’s Airways Clinic Research Center (ACRC) at UAB. The ACRC is a network of 39 clinical research centers around the nation dedicated to asthma and chronic obstructive pulmonary disease (COPD) research. Dr. Lowell works with the other network members to help design and implement asthma clinical trials to advance our understanding and management of this prevalent airway disease. She is currently the principal investigator on the ACRC study, Improving Medication Adherence with Telehealthcare Medication Therapy Management to Change Health Outcomes in Adolescents and Young Adults with Asthma (MATCH) that examines whether providers can best improve adherence to medications in asthma patients through the use of electronic monitoring tools and apps or the combination of these tools with telehealth-based medication therapy management. Drs. Lowell and Magruder have also helped design a comprehensive database to track the division’s pediatric asthma patients. They use this database to track individual and collective patient outcomes, provide data for quality-improvement projects and identify The Bronchopulmonary Dysplasia Program provides the highest quality evidence-based, interdisciplinary, family-centered care for infants and children with bronchopulmonary dysplasia (BPD) and other chronic lung diseases of infancy to improve health and developmental outcomes. Miles Fowler, M.D., and Brett Turner, M.D., along with Kathryn Petricevic, CRNP, provide care through an interdisciplinary model with respiratory therapy, clinical nutrition, social work and speech-language pathology to facilitate comprehensive management plans. The program continues to expand, serving more than 200 unique patients annually, referred locally from the neonatal intensive care units at UAB and Children’s of Alabama and community nurseries around the state. The BPD team continues to work closely with these NICU teams to facilitate a successful transition to the outpatient setting for these families. Emerging quality-improvement (QI) priorities include establishing nutritional and feeding safety guidelines, which can be implemented in the NICU and adapted to ongoing outpatient needs and tobacco/vaping smoke exposure and counseling. As the pediatric community welcomes newly approved therapies for respiratory syncytial virus (RSV) prophylaxis, our team has served as ongoing consultants for the implementation of these therapies in our high-risk infants with neonatal lung disease. CYSTIC FIBROSIS potential subjects for clinical research trials. BRONCHOPULMONARY DYSPLASIA

Jennifer Guimbellot, M.D., Ph.D., studies personalized medicine for cystic fibrosis (CF). Her Cystic Fibrosis Foundation (CFF) and NIH K23-supported research involves individualized pharmacokinetic analysis and pharmacogenomics approaches to gain insights into tailoring modulator therapy to provide maximal therapeutic benefit for every CF patient. She incorporates genetic variant analysis with plasma drug concentrations to better understand drug distribution and metabolism in special populations, including pediatrics, lung transplant recipients and pregnancy. She also utilizes this expertise to understand the intersection between diet and drug absorption with funding from the Nutrition and Obesity Research Center. Her team has also developed multiple personalized models for predicting the effectiveness of cystic fibrosis transmembrane conductance regulator (CFTR) modulators in CF patients and those with acquired CFTR dysfunction. Using cells from the nose, lower airway and sweat glands, her laboratory continues to develop minimally invasive, personalized models for individuals with pulmonary disease. She has support from the CFF and NIH for studies in pharmacokinetics and pharmacogenetics of epithelial-targeted therapeutics.

Wyn Hoover, M.D., leads the CF clinical center. His research interests include the implementation of quality improvement, investigation of difficult-to-treat infections and outcomes projects as they apply to improving and sustaining outcomes of the patients receiving care at our UAB/Children’s of Alabama Pediatric CF Care Center. Hector Gutierrez, M.D., works on implementing quality improvement, outcomes measurement, and management of clinical and non-clinical processes using CF as a model to improve the quality and value of clinical care, ultimately resulting in better survival. His current work involves training clinical CF teams in low- and middle-income countries (LMICs) by transferring the know-how to become centers of excellence. In addition, he is working on establishing a functional database to serve as a registry for CF in LMICs. Dr. Gutierrez has contributed to developing the CF Foundation’s CF minimal dataset that could be deployed in areas lacking registries. The CFF invited Dr. Gutierrez to submit his registry and data management solution for consideration by the Foundation to adopt it as the Universal Minimal CF Data Registry. Lastly, he is working on implementing mobile health (mHealth) solutions for better patient

self-management and patient-centered care. Grants from the CF Foundation support his work. Dr. Harris investigates targetable mediators for therapeutic intervention in cystic fibrosis (CF). His laboratory program focuses on limiting pathology secondary to TGF-beta, a leading genetic modifier of disease progression. He currently receives extramural support from the CFF and NIH to study the impact of transforming growth factor (TGF)-beta depending microRNA (miRNA) to alter therapeutic response to CFTR modulating drug. miRNA are small (~22 base pair) non-coding nucleotide sequences that destabilize messenger RNA transcripts and inhibit protein translation. Dr. Harris discovered that miR-145 impedes CFTR expression and response to CFTR directed therapeutics. Dr. Harris is studying a selective approach of utilizing antisense oligonucleotides (ASOs) to block the only the miR-145 target on CFTR while leaving other downstream miRNA or TGF-beta targets unperturbed. In addition to these projects, Dr. Harris is also investigating the direct role the second-hand smoke exposure has on CFTR-directed therapy. His laboratory utilizes preclinical cell culture and animal models to study these interactions. He also has led several pharmaceutical trials of CFTR correction at UAB that have led to FDA approval in the pediatric population.

2023 Academic Annual Report

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