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Ex Vivo Drug Sensitivity Profiling In Myelodysplastic Syndrome (MDS) Patients Defines Novel Drug Sensitivity Patterns For Predicting Clinical Therapeutic Outcomes

We performed drug sensitivity profiling on 60 patient samples in both newly diagnosed and treatment-refractory myeloid neoplasms (46 MDS, 4 CMML, 10 AML). Fresh bone marrow aspirates and/or peripheral blood specimens were RBC-lysed and re-suspended in serum-free media with cytokines.

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A Feasibility Study of Biologically Focused Therapy for Myelodysplastic Syndrome Patients Refractory to Hypomethylating Agents

We performed a prospective feasibility study in 21 patients with HMA-refractory MDS enrolled at Stanford University from April 2018 through March 2019. All patients had a baseline bone marrow (BM) biopsy with BM aspirate and peripheral blood (PB) samples sent for mutation testing (596-gene panel, Tempus, Chicago, IL) and ex vivo DSS (Notable Labs, Foster City, CA).

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Comprehensive Transcriptome Profiling of Cryptic CBFA2T3-GLIS2 Fusion-Positive AML Defines Novel Therapeutic Options – a COG and Target Pediatric AML Study

A cryptic inv(16)(p13.3q24.3) encoding the CBFA2T3-GLIS2 fusion is associated with poor outcome in infants with acute megakaryocytic leukemia. We aimed to broaden our understanding of the pathogenesis of this fusion through transcriptome profiling. Experimental Design: Available RNA from children and young adults with de novo AML (N=1,049) underwent transcriptome sequencing (mRNA and miRNA).