CRISPR: Treatment and Management of Sickle Cell Disease

Abstract

Sickle cell disease (SCD) is a chronic genetic disorder causing severe pain, organ damage, and reduced life expectancy, with treatment historically focused on symptom management. Casgevy (exagamglogene autotemcel), an FDA-approved CRISPR-Cas9 gene-editing therapy, represents the first potential cure by addressing the genetic root of SCD. This evidence-based review evaluates the comparative effectiveness of Casgevy and other CRISPR-based treatments versus standard care (e.g., hydroxyurea, chronic transfusions) and other treatments on key patient outcomes, including quality of life, functional status, and the frequency of vaso-occlusive crises. Current evidence, primarily from clinical trials, indicates that Casgevy can lead to the complete resolution of severe vaso-occlusive events and significantly improve patient-reported quality of life, though with a complex and lengthy treatment process. For nursing practice, this necessitates a standard shift from chronic symptom management to specialized care coordination for patients undergoing this novel therapy. Nurses must be prepared to provide extensive patient education on the treatment process, monitor for unique side effects (e.g., myelosuppression during the interim period), and provide long-term supportive care for individuals transitioning to a life free from SCD symptoms.