Editor's Note: The 66th Annual Meeting of the American Society of Hematology (ASH) in 2024 concluded successfully, gathering over 30,000 experts and scholars from around the world. Through meticulously planned sessions, the conference provided invaluable insights and breakthroughs in basic research, innovative therapies, and disease management across the hematology field. Hematology Frontier invited ASH President Dr. Mohandas Narla of the New York Blood Center to reflect on the conference’s highlights and takeaways. Additionally, Hematology Frontier presents key updates from ASH’s official reports, focusing on breakthroughs in hemoglobinopathies to provide readers with the latest developments in this important field.Reflections from ASH President Mohandas Narla
Dr. Mohandas Narla: I have been involved in ASH activities for 49 years, and ASH has consistently prioritized presenting novel and innovative content at its meetings. Each year’s conference evolves to reflect the most noteworthy developments from the prior year and the cutting-edge trends of the present. These innovations not only inspire but, more importantly, translate into tangible benefits for patient care. ASH’s core mission remains conquering all forms of blood diseases.
One of our primary focuses this year has been addressing accessibility and cost control. Despite the availability of highly effective therapies, their high costs often limit access. Ensuring broader availability is crucial—our mission is not only to benefit Americans but to extend these advancements to patients worldwide.
ASH has increasingly embraced global hematology, gaining a deeper understanding of developments in regions outside North America. The agenda for this year’s meeting reflects my personal interest in broadening ASH’s reach, which is why there was a particular focus on regions like India. To further amplify this commitment, we launched the journal Blood Global Hematology, dedicated to highlighting academic contributions from Asia and beyond, showcasing global excellence in hematology research.
ASH has also shifted its focus to better serve its global members. In earlier years, we concentrated mainly on U.S.-specific hematology issues. However, as demonstrated by this year’s attendance of over 30,000 participants—many from outside the U.S.—we must address their unique needs and expectations. This year’s program was carefully curated to include speakers from diverse regions and encourage international collaboration. Additionally, initiatives like the Blood Global Hematology journal aim to attract emerging researchers and scientists worldwide, furthering hematology as a truly global endeavor.
Looking ahead to next year’s conference, I am confident it will be as impactful as this year’s, with highlights shaped by the groundbreaking developments of the next several months.
Groundbreaking Achievements in Hemoglobinopathies at ASH 2024
By Jason N. Payne
The ASH 2024 Annual Meeting featured numerous outstanding sessions and reports. Below are key highlights in hemoglobinopathy research:
“Most Memorable” Award
With many new gene therapies emerging, the accessibility of these treatments for most sickle cell disease (SCD) patients remains a significant challenge. A session titled Optimizing Non-Curative Therapies for Sickle Cell Disease, led by Dr. Susan Elizabeth Creary from The Ohio State University, offered practical guidance on maximizing the benefits of current disease-modifying therapies, acute management strategies, and real-world care models.
The session sparked vibrant discussions about the current era of SCD treatment. Dr. Creary emphasized the importance of collaboration between healthcare providers and patients to balance therapeutic efficacy and treatment failure evaluations.
Another impactful session, Challenges in End-of-Life Hematology Care, tackled end-of-life management of hematological diseases. Multidisciplinary perspectives addressed prognosis, symptom burden, and palliative care considerations for various blood disorders.
“Family Legacy” Award
In the Spectrum of Determinants of Health Equity oral presentation, speakers explored socioeconomic and other determinants of health across areas such as neonatal, maternal, and mental health care.
One standout presentation featured Dr. Jeffrey D. Lebensburger and his teenage daughter, Hannah, who discussed SCD patients’ access to mental health care. Hannah’s insights highlighted the urgent need for equitable access to psychological support, underscoring the importance of fostering the next generation of hematology leaders.
“Superhero” Award
A session titled Gene Editing and Alternative Therapies in Hemoglobinopathies: From Bench to Bedside showcased “superhero” advancements in gene therapy.
- Dr. Stacey Rifkin-Zenenberg shared groundbreaking results on Lovotibeglogene Autotemcel (Lovo-cel) for SCD, which showed a remarkable 94% resolution of vaso-occlusive events (VOEs) and sustained improvement in hemoglobin levels (median 12.3 g/dL).
- Dr. Franco Locatelli presented data on Exagamglogene Autotemcel (Exa-cel) for transfusion-dependent β-thalassemia, where over 94% of patients achieved durable transfusion independence, with significant improvements in hemoglobin levels and quality of life.
- Dr. Matthew W. Heeney introduced BEAM-101, a base-editing therapy for severe SCD. Early results demonstrated rapid induction of fetal hemoglobin (HbF), reduced hemolysis, and elimination of VOEs, positioning BEAM-101 as a potential game-changer.
This session underscored the transformative power of gene editing, particularly CRISPR, in reshaping hemoglobinopathy treatment.
“Breaking Barriers” Award
Some therapies remain underutilized due to barriers in implementation. A plenary session on the PIVOT Study—a double-blind, placebo-controlled trial of hydroxyurea for HbSC—aimed to overcome these challenges. The results revealed that hydroxyurea is well-tolerated, with mild and reversible side effects. Patients in the hydroxyurea arm showed significant reductions in VOEs and hospitalizations, along with improved biomarkers, including increased HbF and mean corpuscular volume (MCV).
Another highlight was the development of a novel HbSC mouse model introduced by Dr. Tahereh Setayesh. This CRISPR-edited model replicates the genetic and clinical characteristics of HbSC, including anemia, increased red cell fragility, and retinal damage. The model will bridge critical gaps in research and enable the development of targeted therapies.
Conclusion
The 2024 ASH Annual Meeting reaffirmed that hematology is at a transformative juncture. Through groundbreaking discoveries, innovative research, and a shared commitment to improving global health, the field continues to redefine what is possible in medicine.
Whether you are at the forefront of CRISPR advancements or celebrating novel breakthroughs in SCD management, one message resounds clearly: hematology is not only advancing science but also leading the medical community toward a new era of innovation and collaboration.
