Sickle Cell Disease

People with SCD may experience symptoms such as anemia (fatigue, low energy), episodes of moderate to severe pain (called pain crises), swelling in the hands and feet, frequent infections, delayed growth, and vision problems. Serious complications can include stroke, acute chest syndrome, organ damage, and more.

Exciting new treatments are on the horizon! Outcomes of BMT with alternate donors continue to improve. Also, in December 2023, the FDA approved two groundbreaking gene therapies- Lyfgenia and Casgevy for children older than 12 years and adults. These advances offer new hope for people living with SCD.

SCD is a lifelong condition, but with proper care and treatment, individuals can lead fulfilling lives. Treatment plans are tailored to manage symptoms and reduce complications. This may include:

• Pain Management: Pain crises are managed with over-the-counter pain relievers, prescription medications, and sometimes stronger pain management strategies, including hospitalization in severe cases.
• Hydration: Adequate fluid intake helps reduce the risk of pain crises and complications.
• Blood Transfusions: Regular blood transfusions can help reduce the risk of stroke and treat severe anemia.
• Medications: Hydroxyurea is a medication that can reduce the frequency of pain crises and the need for blood transfusions. Antibiotics and vaccinations are also crucial to prevent infections. Other disease modifying medications such as voxelotor (oxbryta), crizanlizumab (Adakveo) and L-glutamine (Endari) have been used.
• Blood and Marrow Transplant (BMT): This is currently the only potential cure for SCD. It involves replacing the affected blood stem cells with healthy ones from a compatible donor. 
• Gene Therapy: Recent advances in gene therapy offer new treatment options. In December 2023, the FDA approved two new SCD therapies: one that adds a gene to the body and another that modifies an existing gene.