Reflecting on the recent drug recalls within the sickle cell treatment landscape, I am reminded of the immense challenges our patients faced during the COVID-19 pandemic when the virus posed unprecedented threats to vulnerable populations. As hematologists, we were particularly concerned about our patients with sickle cell disease (SCD), individuals who already confront significant health challenges daily. The potential impact of a new virus on this vulnerable population was a source of deep anxiety for everyone.
My profound respect for members of the sickle cell community has only grown as I’ve witnessed their remarkable resilience throughout the years. Parents often become meticulous observers, noting every detail that might affect their children’s health. At the same time, young adults must learn to manage their disease and navigate life's complexities with grace. Despite limited treatment options available, they continue to adapt and thrive, embodying a spirit of perseverance that is truly inspiring.
There was a moment when the introduction of two new medications — voxelotor and crizanlizumab — offered a beacon of hope for those living with SCD. Beginning my career during such a pivotal time felt invigorating, as it seemed we were on the cusp of significant advancements. However, the abrupt discontinuation of these therapies over the last year was a sobering reminder of the challenges we continue to face. It left the community once again reliant on age-old yet effective treatments, underscoring the pressing need to optimize patient care within the constraints of limited disease-modifying options.
During last year’s conference, the U.S. Food and Drug Administration approved two gene therapies (lovotibeglogene autotemcel and exagamglogene autotemcel) for individuals aged 12 and above with SCD. While this development represents a significant milestone, the exorbitant costs associated with these treatments may restrict access for many who stand to benefit the most. This reality highlights the critical importance of also prioritizing non-curative therapies that can substantially improve the quality of life for patients in the interim.
This morning, we have the opportunity to address these challenges head-on during the Education Program session Optimizing Non-Curative Therapies for Sickle Cell Disease (9:30 a.m. - 10:45 a.m., CC, Room 33). This discussion, which will offer practical guidance and innovative strategies amid the landscape of evolving curative therapies, promises to be a highlight for any clinician, researcher, or advocate for the SCD community.
Session chair Susan Elizabeth Creary, MD, MSc, of the Ohio State University College of Medicine, will share her expertise on effectively using disease-modifying medications. I had the pleasure of speaking with Dr. Creary in preparation for this session, and she emphasized the need for doctors and providers to be “diligent about having shared decision-making, setting up expectations up front, and then reassessing as you go along … giving you [as providers] an opportunity to intervene.” Ultimately “patients want their disease to be better managed and optimized, and to live a normal life,” she said. Dr. Creary will also discuss overcoming common barriers to treatment and balancing medication characteristics with the outcomes desired by patients, offering practical approaches to optimize care. Through thoughtful shared decision-making, clinicians can help optimize current disease-modifying therapies before moving toward riskier, transformative treatments.
Amanda M. Brandow, DO, MS, of the Medical College of Wisconsin, will explore alternative methods for managing acute vaso-occlusive pain. She will review recent research supporting non-opioid and non-pharmacologic interventions, as well as care delivery processes that have demonstrated improvements in acute SCD pain outcomes. Her insights will help expand pain management strategies, which are essential for providing patient comfort and fostering well-being.
Lastly, Pablo Bartolucci, MD, PhD, of the Henri-Mondor University Hospital in France, will discuss how we can modernize clinical care models to make them more personalized, adaptive, and collaborative. He will address the unique challenges faced in countries with varying income levels, emphasizing the need for appropriate biomarkers and predictive algorithms. His global viewpoint will add needed perspective and encourage the adoption of best practices in diverse health care settings.
This session promises to provide practical solutions and management strategies to discuss with patients. It will highlight successful, real-world adaptive care models for patients with SCD in the context of potential curative therapies. Continuing this conversation tomorrow, be sure to check out the health services and quality symposia, Navigating Shared Decision-Making for Patient-Centric Care (12:00 p.m. - 1:30 p.m., CC, Room 25), which will highlight additional shared-decision making models, as well as e-health pain assessments and exercise programs for older adults with SCD.
I encourage attendees from all career stages to join this important discussion on optimizing non-curative therapies for SCD. Awareness of the current treatment landscape for patients with SCD, coupled with an appreciation of the significant challenges surrounding cost and access to care, is essential for bridging existing gaps and developing future solutions.
