Christian Marinaccio, Praveen K Suraneni, Hamza Celik, et al.
Conclusion: The study demonstrates that loss of LKB1/STK11 promotes transformation of cells with activated JAK/STAT signaling and that STK11 is a prominent candidate tumor suppressor gene in post-MPN AML.
Terry B. Gernsheimer, Siobhan P Brown, Darrell J Triulzi, et al.
Conclusion: Prophylactic TXA has no effect on the incidence of WHO Grade 2+ bleeding when given in addition to routine plt transfusions to severely thrombocytopenic patients undergoing therapy for hematologic malignancy. An increased incidence of line occlusion in the TXA arm was observed but no increase in other types of thrombotic events was detected.
Min Xia, Liron David, Matthew Teater, et al.
Conclusion: Collectively, we find that BCL10 mutations induce aberrant canonical and non-canonical NF-κB activity through novel and structurally distinct biochemical mechanisms that are at least partially dependent on MALT1. BCL10 mutation should be considered as a biomarker for ibrutinib resistance in ABC-DLBCL, so that alternative targeted therapies can be prioritized for these patients.
4 Safety and Efficacy of CTX001 in Patients with Transfusion-Dependent β-Thalassemia and Sickle Cell Disease: Early Results from the Climb THAL-111 and Climb SCD-121 Studies of Autologous CRISPR-CAS9–Modified CD34+ Hematopoietic Stem and Progenitor Cells
Haydar Frangoul, Yael Bobruff, Maria Domenica Cappellini, et al.
Conclusions: These data demonstrate that CTX001, a first-in-human, CRISPR-Cas9–modified autologous HSPC product, has resulted in increases in HbF and total Hb in the first 7 patients infused. All patients infused with CTX001 demonstrated hematopoietic engraftment with a post-infusion safety profile generally consistent with myeloablation. All 5 patients with TDT have been transfusion-free since ~2 months after CTX001 infusion and the 2 patients with severe SCD have had no VOCs during follow-up after CTX001 infusion. These early data demonstrate that CTX001 is a potential functional cure for the treatment of TDT and SCD. Data will be updated for the presentation.
Data from these ongoing studies were submitted on behalf of the CLIMB THAL-111 and CLIMB SCD-121 Investigators.
Kerstin B Kaufmann, Andy G.X. Zeng, Etienne Coyaud, et al
Conclusions: Collectively, our data decipher the molecular intricacies underlying HSC heterogeneity and self-renewal regulation and point to latency as an orchestrated physiological response that integrates quiescence control with HSC fate choices to preserve a stem cell reservoir.
Bhavana Bhatnagar, Qiuhong Zhao, James L. Fisher, et al
Conclusion: Self-reported AA race is the most important pt-associated factor associated with poor survival in AML pts < 60 y of age based on SEER. Survival analyses in Alliance pts identify AA race as independent poor survival prognosticator in AML pts besides established molecular markers. . This disparity must be urgently addressed to ensure improved outcomes for AA AML pts, and larger studies to establish molecular risk profiles are needed.