Study: Partial stem-cell transplant reverses sickle cell disease
- 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.
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.