What are your top takeaways in Classical Hematology from ASH 2024?

In the category of continuing what I already do, ASH 2024 provided an important confirmatory study of how to approach the treatment of patients with high-risk venous thromboembolism (VTE).

In the EINSTEIN CHOICE (Rivaroxaban) and the AMPLIFY-EXT (Apixaban) studies, extended reduced-dose anti-coagulat...

Based on what I was able to hear/see, three things that stood out to me were the papers showing progress on hematopoietic stem cell mobilization and marrow ablation without the use of genotoxic agents, thus reducing two of the risks associated with ex vivo gene therapy; progress listed in several ab...

1. LBA-3: "In patients with venous thromboembolism who need extended anticoagulation, the noninferiority of a reduced dose versus a full dose of direct anticoagulants to prevent recurrent venous thromboembolism could not be proven. In the reduced-dose ...

1. The reduced dose apixaban RENOVE trial – Paper LBA-3
2. Sickle cell transplant 10-year results – LBA-5
3. The scientific session on Complement and the Prothrombotic State

1. Efficacy, durability, and safety of genome editing and gene addition therapies for sickle cell disease and beta thalassemia.
2. Usefulness of hydroxyurea in young patients with HbSC disease.
3. Helpfulness of pyruvate kinase activators on sickle cell disease and beta thalassemia.

The two most impactful presentations for me were:

1. 490 Allotransplantation and Gene Therapy Equity for Children with Sickle Cell Disease: Distributional Cost-Effectiveness of Allotransplantation Vs Gene Therapy Vs Standard-of-Care in Pediatric Patients with Sickle Cell Disease in the United States –...

1. Efficacy Findings in a Phase 3, Randomized Trial of Eltrombopag Vs. Standard First-Line Treatment for Newly Diagnosed Immune Thrombocytopenia in Children

This study has the potential to really change practice in pediatric ITP. The study was closed early because the eltrombopag arm was superior to ...

1. 179 Etavopivat Reduces Incidence of Vaso-Occlusive Crises in Patients with Sickle Cell Disease: HIBISCUS Trial Phase 2 Results through 52 Weeks
2. 409 ENERGIZE-T: A Global, Phase 3, Double-Blind, Randomized, Placebo-Controlled Study of Mitapivat in Adults with Transfusion-Dependent Alpha- or Beta-Thal...

1. 2024 Ham-Wasserman lecture by Dipty Lalit Jain, MD, MBBS
2. Oral presentation by Matthew Heeney, MD: Initial Results from the BEACON Clinical Study: A Phase ½ Study Evaluating the Safety and Efficay of a Single Dose of Autologous CD34+ Stem Cells (BEAM-101) in Patients with Sickle Cell Disease with Se...