Transformative therapies for sickle cell disease

Sickle cell disease (SCD) is associated with major morbidity, reduced life expectancy, and impaired quality of life.

Transformative therapies for SCD include allogeneic hematopoietic stem cell transplant and gene therapy.

After successful transformative therapy, people with SCD no longer demonstrate an SCD phenotype and experience a reduction in SCD-related morbidities and improvement in quality of life.

Transformative therapies for SCD can be associated with major adverse events, including death.

The decision to proceed with a transformative therapy must take into careful consideration the severity of the SCD, the risk of future SCD complications, and the risks of available transformative therapies.

The expertise and infrastructure to deliver transformative therapies lies within existing Canadian cancer care programs.