Post-ASH 2024 Highlights

Explore the breakthrough research, key sessions, and late-breaking discoveries from ASH 2024.

Abstract Highlights

Date: December 7th, 2024

Title: Genetic Determinants of Hypercoagulability in Complement and Hemostatic Systems Shape the Thrombotic Risk of Paroxysmal Nocturnal Hemoglobinuria:

Summary: Genetic variants in complement and hemostatic systems increase thrombotic risk and drive hypercoagulability in PNH.

Title: Dynamics of Oligoclonal Hematopoiesis in Severe Aplastic Anemia Patients Undergoing Immunosuppressive Treatment: Longitudinal Somatic Mutation Analysis from the EBMT RACE Trial

Summary: The EBMT RACE trial links clonal hematopoiesis to treatment outcomes in SAA, highlighting Eltrombopag's superiority.

Title: Recombinant ADAMTS13 Prophylaxis in Pediatric Patients with Congenital Thrombotic Thrombocytopenic Purpura: Results from the Phase 3 Randomized, Crossover Study and the Phase 3b Continuation Study

Summary: Phase 3 studies confirm the safety and efficacy of recombinant ADAMTS13 prophylaxis in managing congenital thrombotic thrombocytopenic purpura (cTTP) in pediatric patients.

Title: Treatment of Idiopathic Aplastic Anemia (AA) with Expanded Autologous Regulatory T Cells: The Tiara Phase One Trial

Summary: Phase 1 Tiara trial explores expanded autologous Tregs as a novel treatment for AA, showing promising stability, suppression, and polyclonality.

Title: Eltrombopag Added to Standard Immunosuppressive Treatment Improves Long-Term Outcomes As Front-Line Therapy for Severe Aplastic Anemia: Final 2-Year Analysis of EBMT-SAAWP RACE Study

Summary: The RACE study confirms eltrombopag with standard IST improves long-term outcomes, enhancing response rates and hematologic recovery in SAA.

Title: Efficacy and Safety of Avatrombopag in Combination with Immunosuppressive Therapy in Treatment-Naïve Severe Aplastic Anemia: Results of the DIAAMOND-AVA-First Trial

Summary: The DIAAMOND-AVA-First trial evaluates avatrombopag as a promising second-generation TPO-RA in combination with IST for treatment-naïve SAA.

Title: Improved Outcomes in Paroxysmal Nocturnal Hemoglobinuria Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplantation in 2011-2020: A Retrospective EBMT-SAAWP Study

Summary: A retrospective SAAWP study shows improved survival rates in PNH patients undergoing HSCT during the complement inhibitor era.

Date: December 9th, 2024

Title: Annualized Bleeding Rates in Patients with Hemophilia A or B and Inhibitors with and without Target Joints at Baseline: Results from the Concizumab Phase 3 Explorer7 Study

Summary: Phase 3 explorer7 results show daily concizumab prophylaxis reduces bleeding rates and resolves 92% of target joints in hemophilia A/B patients.

Late Breaking Abstract Highlights

Date: December 10th, 2024

Title: Extended Treatment of Venous Thromboembolism with Reduced- Vs Full-Dose Direct Oral Anticoagulants in Patients at High Risk of Recurrence

Summary: Extended anticoagulation with reduced-dose DOACs balances recurrence prevention and bleeding risk in high-risk VTE patients, offering an alternative to full-dose treatment.

Plenary Scientific Session Highlights

Date: December 8th, 2024

Title: Platelet Factor 4 (PF4) Regulates Hematopoietic Stem Cell Aging

Summary: PF4, secreted by bone marrow niches, maintains HSC quiescence and function; declining PF4 levels with age contribute to HSC aging and hematologic malignancy risks.

Title: Double-Blind, Placebo-Controlled Randomized Controlled Trial of Hydroxyurea for HbSC: Results of the Prospective Identification of Variables As Outcomes for Treatment (PIVOT) Trial

Summary: The PIVOT trial identifies clinical and laboratory outcomes of hydroxyurea treatment in HbSC disease, advancing its potential as a therapeutic option.

Title: Efficacy and Safety of Oral Bruton Tyrosine Kinase Inhibitor (BTKi) Rilzabrutinib in Adults with Previously Treated Immune Thrombocytopenia (ITP): A Phase 3, Placebo-Controlled, Parallel-Group, Multicenter Study (LUNA 3)

Summary: Phase 3 LUNA 3 study confirms rilzabrutinib achieves rapid, durable platelet responses and improves quality of life in ITP patients unresponsive to standard therapies.