James Griffin, sickle cell anemia patient advocate, gives advice to patients with sickle cell anemia and healthcare providers.
Sickle cell anemia is a rare genetic disease in which the body produces red blood cells that are sickle-shaped. The sickle shaped red blood cells lead to reduced blood flow as the cells get stuck in blood vessels. Signs and symptoms of sickle cell disease usually begin in early childhood and may include anemia, repeated infections, and periodic episodes of pain. This condition is caused by genetic changes in the HBB gene.
Although several drugs are approved for this indication, recent advancements have led to the approval of two gene therapies for patients with sickle cell anemia, Casgevy (exagamglogene autotemcel) and Lyfgenia (ovotibeglogene autotemcel). Mr. Griffin describes the feeling of hope these advancements have provided for patients and how encouraging it is to have momentum behind the disease with other therapies currently in development.
Sickle cell anemia greatly impacts the patient’s quality of life, so Mr. Griffin emphasizes the importance of destigmatizing the disease. He wants healthcare providers to understand what patients are going through and make them the focal point, providing adequate care and access to treatment.
His advice to patients highlights the necessity in patient organizations and support groups, noting that a rare disease diagnosis can feel isolating. Through these groups, patients can find strength in community and gain valuable insights in management and treatment options.
For a list of sickle cell anemia patient organizations, click here.
To learn more about sickle cell anemia and other rare hematologic conditions, visit https://checkrare.com/diseases/hematologic-disorders/