Dive Brief:
- In a study of 30 people with sickle cell disease who received a stem-cell transplant between July 2004 and October 2013, 87% of patients experienced a full reversal of the disease.
- Half of patients were able to stop taking immunosuppressants within one year of the procedure, suggesting post-transplant stability.
- Among transplanted patients, use of narcotics declined. The rate of hospitalization also decreased.
Dive Insight:
According to the CDC, sickle cell disease affects up to 100,000 Americans, including one in 500 African Americans and one in 36,000 Hispanic Americans. Although SCD-related mortality has decreased significantly since the late 1990s (a vaccine was introduced in 2000 to protect against invasive pneumococcal disease), a diagnosis of the disease still means a lifetime of treatment and pain.
Patients in the stem-cell transplant study received a transplant consisting of peripheral blood stem cells from human leukocyte antigen-matched siblings. Because the stem cells came from siblings, this could limit the ability to treat every person with SCD with stem-cell transplantation. Only 20% of individuals with SCD have a sibling who is a full match at the white blood cell level. Nonetheless, this is definitely a game-changer and will shift treatment options for many patients.