Emerging Treatment Options for the Management of Sickle Cell Disease

An overview on the pathophysiology of sickle cell disease and expert perspectives on the clinical (and psychosocial) burden it presents.

Shared insight on how a patient’s life may progress following a diagnosis of sickle cell disease, along with considerations for pediatric versus adult management.

Expert panelists reflect on the global prevalence of sickle cell disease, as well as general disease awareness.

Expert perspectives on the importance of newborn screening to impact mortality rates in sickle cell disease.

Wally Smith, MD, and Payal Desai, MD, spearhead a discussion on patient education surrounding sickle cell disease symptomology and lifestyle modifications.

A broad perspective on the value of multimodality care in the management of patients diagnosed with sickle cell disease.

A focused discussion on how comprehensive care is applied by multidisciplinary teams to improve the management of sickle cell disease.

Reflections on optimal approaches to patient follow-up, particularly regarding pain management and inherent risks in high-dose opioids.

Focusing on disease-modifying therapeutics, expert panelists discuss foundational treatment goals and guidelines for sickle cell disease.

Expert perspectives on hydroxyurea as a staple therapy in the treatment armamentarium for sickle cell disease.

Shared insight on L-glutamine therapy in sickle cell disease, with deference to observed efficacy and safety considerations.

Panelists share their perspectives on the selection and use of crizanlizumab in certain patients with sickle cell disease.

Considerations for voxelotor’s unique mechanism of action and prospective use in patients with sickle cell disease.

Panelists take a step back to consider when they might use certain disease-modifying therapy in the setting of sickle cell disease.

An overview of gene therapy in sickle cell disease with a focus on the risk-benefit ratio observed in clinical trials and practice.

Shared insight on patient education surrounding gene therapy in sickle cell disease and common misconceptions about this approach.

Concluding their discussion on sickle cell disease management, expert panelists share key unmet needs and hopes for the future.