Medicine and Health Sciences Commons^™

A Novel Therapy For Refractory Cbfa2t3::Glis2-Associated Amkl Using Stro-002 And Plerixafor, Amy Johnson Md, Mba, Alan S. Gamis, Arturo Molina, Soheil Meshinchi, Karen Lewing

Research Days

Background: CBFA2T3::GLIS2-associated AML (CBF/GLIS AML) is an aggressive form of AML often associated with AMKL and recognized by its unique RAM phenotype by flow. It has a very poor prognosis, frequently refractory to standard of care regimens, with extreme marrow localization of leukemic blasts likely mediated by high CXCR4 (CD184) expression. The CBFA2T3::GLIS2 fusion gene is the most common oncogenic transcript in pediatric AMKL and universally results in high expression of FOLR1 surface antigen. STRO-002, an antibody drug conjugate targeting FOLR1, is currently in Phase I clinical trials for adults with refractory ovarian and endometrial malignancies A summary of 17 …

Bone Marrow Transplant For Macrophage Activation Syndrome In Systemic Juvenile Idiopathic Arthritis, Shailly Gaur, Michael J. Holland, J Allyson Hays, Ibrahim A. Ahmed

Research Days

Background

The recognition of macrophage-activation-syndrome (MAS) in the setting of systemic-juvenileidiopathic-arthritis (sJIA) is complex, but a necessary distinction given emerging treatment options. With overlapping clinical manifestations including fever, end-organ involvement and lymphadenopathy, there is a focus on laboratory values to differentiate MAS from a sJIA flare. Once parameters are met, multiple treatment modalities can be considered, as monotherapy or in combination, including steroids, immunosuppression, chemotherapy, intravenous immunoglobulin, and cytokine-directed biologics. Given the long-term morbidity and mortality associated with progressive inflammation and immune dysregulation due to MAS with sJIA, it is imperative to regain control over this complication. However, these treatments …