By Ahmar U. Zaidi, MD
“No conclusions can be drawn from this case. Not even a definite diagnosis can be made,” James Herrick wrote in 1910 about the case of the first known patient in America with sickle cell disease (SCD), Walter Clement Noel. More than one century later, we seem to be close to the apex of our scientific knowledge surrounding this once enigmatic disease. In fact, in the past month, two new drugs have been approved for SCD. Both voxelotor and crizanlizumab have been discussed at the ASH annual meeting within the past five years. And once again, it is this meeting that has become the pièce de résistance in the landscape of scientific meetings where SCD is deliberated and new therapies are considered. A dedication to the propagation and proliferation of knowledge and awareness is evident again in 2019, where throughout the course of four days, the ASH annual meeting will provide a schedule packed with various events that drive our understanding of this disease forward.
On the first day of the meeting, a session chaired by Kim Smith-Whitley, MD, of Children’s Hospital of Philadelphia, expanded on the discussion of complications in SCD beyond the most common offenders and addressed hepatic complications, avascular necrosis (AVN), and complications associated with pregnancy (this session will be offered again on Monday). Several oral abstracts highlight novel therapeutics (PP-14, recombinant heme oxygenase, and cannabidiol, along with three presentations from Kalpna Gupta, PhD, and colleagues), as well as basic and translational science research, looking at the gut microbiome, hemoglobin modulators, target gene transduction, and engraftment and KEAP1 inhibition. Updates on gene therapy were provided in the poster session by Julie Kanter, MD, and created a clear sense of optimism on our journey toward a cure. A session chaired by Swee Lay Thein, DSc, MBBS, of the National Institutes of Health addresses challenges faced by children, teens, and those who are making it well into adulthood — all key times of vulnerability and medical management lapses.
The growing concerns of access to care, particularly for adults with SCD were addressed in a session moderated by Biree Andemariam, MD, of University of Connecticut Health Center, on SCD Centers to address topics relevant to the formation of specialized centers, including standardizing approaches to pain management; securing funding from the C-suite and other sources; and engaging patients, the com- munity, and other stakeholders to achieve success. Throughout the next few days, we received updates on the ASH Clinical Practice Guidelines on Sickle Cell Disease led by Robert Liem, MD, of Lurie Children’s Hospital. In this Special Education Session, speakers highlighted particularly noteworthy recommendations (on cardiopulmonary and kidney disease, cerebrovascular disease, pain, transfusions, and stem cell transplantation) and discussed imperative considerations for the implementation of the recommendations in practice. Michael DeBaun, MD, MPH, of Vanderbilt University was honored with the ASH Mentor Award for his extraordinary footprint and influence on the SCD educators, clinicians, and researchers that he has mentored throughout his career. Finally, the ASH-FDA session will also highlight voxelotor and crizanlizumab, with clinical spotlights from Caterina Minniti, MD, of Albert Einstein College of Medicine.
These collaborative discussions and intellectual connections that occur every year at the ASH annual meeting are a reminder that although this population of patients has been largely ignored, the tide is turning, and a dedicated group of individuals committed to change exist. Their collective voice will be heard, and these trailblazers will ensure that an up and coming new generation of patients will be able to live well with SCD.
Dr. Zaidi indicated no relevant conflicts of interest.