Sickle cell disease: Expanded surveillance, new treatments and the power of music therapy

World Sickle Cell Day is observed each year on June 19.

The observation is intended to raise awareness of sickle cell disease, as well as the challenges patients encounter.

Photo of sickle cells — *Source: Adobe Stock.*

In conjunction with World Sickle Cell Day, Healio presents the following updates in sickle cell disease treatment and research.

The CDC established the Sickle Cell Data Collection program to compile data on the complications, treatments, health outcomes and health access of individuals with sickle cell disease in 11 states. Healio spoke with Mary Hulihan, DrPH, health scientist in the epidemiology and surveillance branch of the division of blood disorders at CDC’s National Center on Birth Defects and Developmental Disabilities, about the potential value of data being collected through this program. Read more.
Exposure to systemic corticosteroids appeared to increase risk for hospitalization for vaso-occlusive episodes among patients with sickle cell disease. Read more.
An investigational CRISPR/Cas9 gene-edited cell therapy demonstrated significant, durable efficacy among patients with transfusion-dependent beta-thalassemia and sickle cell disease. Read more.
Prevalence of organ dysfunction appeared low among pediatric patients who underwent hematopoietic stem cell transplantation for sickle cell disease. Read more.
Sickle cell disease increased risk for transplant-associated thrombotic microangiopathy among children who underwent hematopoietic stem cell transplantation. Read more.
Most male adolescents and young adults with sickle cell disease reported a desire to have children later in life but few knew of disease-related fertility issues. Read more.
UCSF Benioff Children’s Hospital Oakland will initiate a 4-year trial to evaluate CRISPR/Cas9 gene editing as a potential cure for sickle cell disease. Read more.
Healio spoke with Samuel Rodgers-Melnick, MT-BC, about the potential of a music therapy intervention to address pain and functional deficits caused by sickle cell disease. Read more.
The gene therapy LentiGlobin (bb1111; bluebird bio) led to a reduction in hemolysis and complete resolution of severe vaso-occlusive events among a small cohort of patients with sickle cell disease. Read more.

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Sickle cell disease: Expanded surveillance, new treatments and the power of music therapy

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