Knowing someone with sickle cell disease (SCD) is witnessing firsthand the profound impact this disorder has on their daily life. As physicians working to help people with SCD, balancing the benefits of each treatment against significant side effects challenges us and the patients who strive to maintain their health while living with this arduous disease. Each episode of pain crisis, acute chest syndrome, thromboembolism, avascular necrosis, or cardiovascular event deals a heavy blow, hindering our patients' aspirations for an optimal life. The past decade has seen considerable growth in therapeutic options for the management of SCD, yet a considerable journey remains before patients can attain longer, healthier lives.
The ASH Research Collaborative (ASH RC) has made remarkable strides in advancing research, enhancing provider training, advocating for expanded care access, and improving data collection on many fronts. At this year's meeting, the Special Interest Session ASH and the ASH Research Collaborative: Advancing Progress in Sickle Cell Disease (Monday, 6:15 p.m. – 8:00 p.m., Convention Center, Room 28 A-D and Plaza Terrace) will feature an overview of ASH and the ASH RC’s advancements in driving change in SCD over the past year, presented by ASH President-Elect, Mohandas Narla, DSc. Attendees will also have ample opportunities to network with prominent contributors and investigators in the field.
As we continue to contend with the heightened vulnerability of patients with SCD against the steady backdrop of COVID-19, we invite you to arm yourself with the latest data courtesy of the ASH RC Clinical Trial Network (ASH RC CTN). This group undertook a pivotal mRNA vaccine trial to assess whether antibody responses following COVID vaccination were adequate to induce immunity in those with SCD, and whether mRNA vaccination precipitated any unique toxicities in this population. The “COVID mRNA Vaccination Responses in Individuals with Sickle Cell Disease: An ASH Research Collaborative Clinical Trial Network Study” will be presented by Charles S. Abrams, MD. (today, 6:00 p.m. – 8:00 p.m., Convention Center, Halls G-H). This study — the largest mRNA vaccine efficacy and safety trial in the SCD population, involving 59 patients from eight ASH RC sites — addressed common patient queries regarding vaccine efficacy and potential disease-related complications. Vaccine efficacy and side-effects were similar in individuals with SCD and the general population. Despite 17% of patients experiencing pain crises within 14 days of the first dose and one requiring inpatient admission, this study provides much-needed confidence for patients with SCD facing higher COVID-related risks.
Saturday’s Special Session on Sickle Cell Disease Centers focused on funding for such centers. The complex and multidimensional nature of SCD desperately calls for specialized centers to support quality care in this area, prompting ASH's efforts to develop workshops and blueprints for their creation. Despite a coordinated, multidisciplinary approach providing the optimal framework to approach the delivery of health care to people with SCD, start-up logistics and funding remain challenges, making this session invaluable for those seeking to bring specialized centers to life.
Missed the oral or poster sessions on SCD and looking to catch up on the latest and greatest clinical research in the field? Look no further. The ASH poster walk What’s Hot in Sickle Cell Disease Clinical Research in 2023 (Wednesday, 5:00 p.m. – 6:00 p.m., Virtual Program) promises a virtual tour through cutting-edge SCD research, often, refreshingly, through the eyes of more junior investigators from varied backgrounds. This session highlights novel therapeutics, potential curative therapies, and multi-system vasculopathy insights — a treasure trove for hematologists.
Overall, this year's ASH meeting unveils a wealth of insights into SCD, a testament not only to investigators' dedication but also to ASH and the ASH RC's support for SCD research. With such progress, we can confidently foresee a brighter future for our patients: a future in which the debilitating complications of SCD slowly take second place to the ability to access the care and tools that enable the pursuit of happy and healthy lives, healing not only hemoglobin, but, more importantly, humans.
Drs. Rafae and Al Hadidi indicated no relevant conflicts of interest.
