2023 Department of Pediatrics Academic Annual Report

care to children with retinoblastoma in the state of Alabama. Dr. Alva runs the pediatric cancer predisposition clinic at Children’s of Alabama, which is the only clinic in the state providing evidence-based screening for patients with genetic cancer predispositions. On a national level, she is also involved in the development of the upcoming NCCN guidelines for neuroblastoma. Jamie Aye, M.D., is a young investigator within the Soft Tissue Sarcoma Committee of the Children’s Oncology Group (COG) and has served on the COG’s Intermediate-Risk Rhabdomyosarcoma Working Group. In addition to her COG involvement, Dr. Aye is a young investigator within the National Pediatric Cancer Foundation Sunshine Project. She received the 2022 Award for Research for her work involving the preclinical use of oncolytic herpes simplex virus in fusion-positive rhabdomyosarcoma. Dr. Aye is also the principal investigator for a pooled analysis of outcomes and descriptions of clinical features in children with biliary rhabdomyosarcoma through the International Soft Tissue Sarcoma Consortium. Moreover, she is a member of the NCCN’s Wilms Tumor Panel. Locally, Dr. Aye is the principal investigator of the UAB Pediatric Tumor Bank and Tumor Graft Development Program, which actively enrolls patients for the collection of tumor tissue for ongoing research at Children’s of Alabama. Kimberly Whelan, M.D., is an active member of the Children’s Oncology Group. She is a member of the Pediatric Normal Tissues in the Clinic (PENTEC) Ocular complications task force, where she is working on an analysis of the organs and tissues that are at risk from radiation injury in children. She played an integral role in the establishment of Children’s of Alabama as a member of the Sunshine Project/NPCF Consortium and served as the initial site PI for this group. She is the Medical Director of Taking on Life after Cancer. In addition, she is a co-investigator on an NIH-funded trial examining adaptive immunity and persistent SARS-CoV-2 replication in pediatric patients with cancer and blood disorders. BLOOD & MARROW TRANSPLANTATION PROGRAM The BMT team consists of four faculty members: Joseph Chewning, M.D, Hilary Haines, M.D., Sheetal Phadnis, M.D., and Loretta Parker, D.O. We are a Foundation for the Accreditation of Cellular Therapy (FACT)-accredited blood and marrow transplant (BMT) and cellular therapy program. We are a part of the Radiation Injury Treatment Network (RITN), a national network of medical centers with expertise in the management of bone marrow failure. Dr. Chewning serves as director of the BMT and Cellular Therapy Programs. He is a national leader in BMT for pediatric acute myeloid leukemia (AML) and the local expert on BMT for acute and chronic leukemia and graft versus host disease (GVHD). He is on the COG Myeloid Diseases Steering Committee and Cellular Therapy Committee, and co-chairs the AML-BMT Task Force of the Myeloid Diseases Committee. Dr. Chewning is program PI for the Pediatric Transplantation and Cellular Therapy Consortium and is a member of multiple steering committees, including the Oncology Committee. SICKLE CELL DISEASE PROGRAM The UAB Division of Pediatric Hematology and Oncology has a strong team dedicated to cutting-edge research in the field of sickle cell disease. The program currently cares for 850 patients from birth to high school and 50 patients that are older and in the process of transitioning. Hilary Haines, M.D., is participating in curative clinical trials for patients with sickle cell disease and developing patient-centered approaches to educate patients about curative therapies. The Alabama Center for Childhood Cancer and Blood Disorders has opened a collaborative haploidentical BMT protocol, CTN1507, for patients with sickle cell disease. Additionally, the center, in collaboration with the adult hematology colleagues, will be participating in an autologous gene therapy protocol for sickle cell disease. Lee Hilliard, M.D., is the longest-serving member of the sickle cell team. She was the site investigator for several landmark sickle cell studies, including Stroke with Transfusions Changing to Hydroxyurea (SWiTCH), Transcranial Doppler with Transfusions Changing to Hydroxyurea (TWiTCH) and Dissemination, and Implementation of Stroke Prevention Looking at the Care Environment (DISPLACE), as well as several other therapeutic studies. Chibuzo Ilonze, M.D., received a prestigious award as a scholar at the 2023–2024 American Society of Hematology, Clinical Research Training Institute to understand the impact of sickle cell on the development of heart disease. He is also the clinical lead for our satellite clinic program, ensuring that barriers to transportation to Birmingham do not impede clinical care. Malgorzata Kasztan, Ph.D., leads the basic science program for pediatric sickle cell disease. She is funded by NIH/NHLBI (K99/ R00) grant to study the role of endothelin-1 (ET-1) in tubular injury in sickle cell disease. She also is focused on studying the role of endothelin-1 signaling in renal iron metabolism in sickle cell disease. Dr. Kasztan is also the lead in the translational sickle cell program. She leads a UAB/St. Jude research collaboration evaluating the impact of ET-1 and APOL1 mutations on sickle cell kidney disease. Jeffrey Lebensburger, D.O., MSPH, section head of Hematology, continues to focus his research efforts on understanding the progression to chronic kidney disease that affects one-fourth of adults with sickle cell anemia. He is currently funded by the NIH to conduct a multicenter study that will develop a novel approach to defining kidney function in children and adults with sickle cell anemia. He is also part of a recent NIH grant that will test the effectiveness of SMYLS, an mHealth intervention designed to facilitate self-management behaviors in adolescents and young adults (AYA) with sickle cell disease (SCD). Dr. Lebensburger is recognized for excellence in clinical trial design and mentorship. He has served as the co-director for the American Society of Hematology, Clinical Research Training Institute and mentors several NIH-funded K23/K08 awardees. Brandi Pernell, DNP, MSN, has a research focus on pulmonary outcomes of sickle cell disease as well as psychosocial/socioeconomic predictors of outcomes. Dr. Pernell received a CCTS Deep South Career Development award to study the impact of adverse childhood experiences (ACE) in sickle cell disease. She is currently funded by the NIH/National Heart, Lung, and Blood Institutes to study ways to promote exclusive breastfeeding in children with sickle cell disease for improvement of health outcomes.

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