2023 Department of Pediatrics Academic Annual Report

THROMBOSIS AND BLEEDING PROGRAMS Christy Bemrich-Stolz, M.D., MSPH, is the research lead for bleeding disorder research programs. She oversees the ATHN (American Thrombosis and Hemostasis Network) database, Community Counts and other therapeutic/observational bleeding studies. Drs. Bemrich-Stolz and Wilson, in collaboration with the UAB Division of Adolescent Medicine, have developed a women’s and children bleeding disorders clinic. Dr. Bemrich-Stolz also co-leads an idiopathic thrombocytopenia purport (ITP) study to evaluate a novel therapy for the upfront management of ITP. Hope Wilson, M.D., conducts research to characterize children who have a history of thrombosis and persistent thrombotic risk factors to inform the precise use of secondary anticoagulation to prevent recurrent venous thromboembolism. She received a trainee award from the ASH Clinical Research Training Institute (CRTI) for this research. She was voted to serve as the trainee representative to the Executive Committee for ASH CRTI. Recently, she received four years of funding ($440,000) from the NIH to study the association between thrombotic events and renal disease in children and adults with sickle cell disease. This multicenter study is being conducted at UAB, St. Jude Children’s Research Hospital/University of Tennessee Health Science Center and the University of Illinois Chicago. BONE MARROW FAILURE PROGRAM Chibuzo Ilonze, M.D., developed a bone marrow failure program at Children’s of Alabama/UAB. He serves as the site lead for the North American Aplastic Anemia Consortium. He recently opened a national study for patients with newly diagnosed aplastic anemia to compare immunosuppressive therapy versus bone marrow transplant. INSTITUTE FOR CANCER OUTCOMES AND SURVIVORSHIP (ICOS) In order to meet the needs of cancer survivors, UAB Heersink School of Medicine founded the Institute for Cancer Outcomes and Survivorship (ICOS) in 2015, with a mission to improve the understanding of the long-term effects of cancer treatment on the overall health and well-being of survivors, and to mitigate the burden of morbidity through research, health promotion and education. Currently, the institute has 17 faculty members with a primary research focus on cancer outcomes, with the total annual funding of $5.6 million. The key areas of excellence include the following: burden of morbidity in cancer populations, molecular pathogenesis of treatment-related complications, adherence to therapy, end of life/palliative care, health services research, prevention/mitigation of morbidity and patient/family education. ICOS members published 143 peer-reviewed publications in 2022 and 109 publications between January and June 2023. Smita Bhatia, M.D., MPH, is the founding director of ICOS. Dr. Bhatia was awarded the National Cancer Institute Outstanding Investigator Award in 2018, providing her with funding for the next seven years to identify childhood cancer survivors at highest risk for late-occurring cardiomyopathy and second cancers. As part of this initiative, she has a multi-institutional study at more than 100 institutions to understand the molecular pathogenesis of treatment-related complications. Using this resource of nearly 5,000 DNA samples, she has identified genomic variants that modify radiation-related subsequent neoplasm and anthracycline-related cardiac dysfunction. This has led to improved models to identify survivors most at risk for these complications. She also received funding from the NCI to understand the pathogenesis of therapy-related leukemia in patients with lymphoma receiving autologous stem cell transplantation. In addition, Dr. Bhatia has been funded by the Leukemia Lymphoma Society and the NCI (through a U01 mechanism) to construct a cohort of 10,000 BMT survivors to understand the burden of morbidity borne by the survivors. Findings have informed the need for lifelong follow-up of BMT survivors. She is developing FDA-approved and NIH-funded strategies to reduce the risk of radiation-related breast cancer in survivors of Hodgkin lymphoma. She has demonstrated that non-adherence to oral mercaptopurine in children with acute lymphoblastic leukemia is prevalent and is associated with high risk of relapse. She and Dr. Wendy Landier conducted a randomized clinical trial to improve adherence using personalized text messaging and directly supervised therapy (funded by the National Cancer Institute though an R01 mechanism). She has developed the infrastructure and leads the effort in maintaining lifelong follow-up of all patients treated on COG therapeutic trial across the US (funded by the NCI Childhood Cancer Data Initiative). She chairs four national cooperative group studies. Emily Johnston, M.D., MPH, has received funding from the St. Baldrick’s Foundation, the Leukemia Lymphoma Society, Alex’s Lemonade Stand, Kaul Pediatric Research Institute (KPRI), the Conquer Cancer Foundation and the American Cancer Society to improve the quality of end-of-life care of children dying of cancer and other life-threatening illnesses. She has published extensively on the prevalence and patterns of high-intensity care received at end-of-life in children dying of cancer and is currently developing strategies to ensure that children receive goal-concurrent care at end-of-life. Drs. Johnston and Wolfson have led a large multi-site effort to develop a registry of children with cancer who developed SARS-CoV-2 infection. Wendy Landier, Ph.D., deputy director of ICOS, received funding from the NIH to understand the facilitators and barriers to HPV vaccination in childhood cancer survivors, and to test the immunogenicity and safety of using this vaccine in childhood cancer survivors. Findings from this study informed the next large initiative funded by the NCI through a U01 mechanism to improve HPV vaccination rates in childhood cancer survivors by educating the healthcare providers at six participating institutions. She has also received funding from the Alex’s Lemonade Stand Foundation to develop a patient-family education intervention for children with newly diagnosed cancer and from Kaul Pediatric Research Institute (KPRI) to develop an educational smartphone app for parents of children with a new diagnosis of cancer. Within COG, she is the past chair of the Nursing Discipline and serves on the Scientific Council. Donna Murdaugh, Ph.D., is a board-certified neuropsychologist, funded by the K23 career development award to develop cognitive remediation programs for children with sickle cell disease. She is also conducting a cognitive remediation intervention trial in survivors of childhood cancer to facilitate transition of healthcare from a pediatric to an adult facility. Dr. Murdaugh is the primary neuropsychologist taking care of all childhood cancer survivors with cognitive issues at the Children’s of Alabama.

2023 Academic Annual Report

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