Partial Stem Cell Transplant Reverses Sickle Cell Disease

A modified partial stem cell transplant procedure effectively reversed sickle cell disease in almost all the participants of a study conducted by the National Health Institute (NIH).

Sickle cell disease (SCD) is a genetic red blood cell disorder. People with this condition are at higher risk of infection and other health problems. The only cure is a bone marrow or stem cell transplant, but these options can both be very risky because of side effects known as graft-versus-host disease (GVHD). GVHD is a complication experienced by the patient after receiving cells from a donor due to genetic differences. Complications usually surface within three months after the transplant and could last a lifetime.

Dr Matthew Hsieh, lead author of the study and staff clinician at the molecular and clinical hematology branch of NIH, and his colleagues performed the modified stem cell transplants on patients diagnosed with sickle cell disease.

The researchers combined the stem cells of healthy donors with the cells taken from the patients' own blood. They also reduced the doses of chemotherapy used to kill bone marrow before the actual blood-stem cell transplant. The procedure was called a "partial stem cell transplant."

After the operations, researchers asked the participants to stop their medications and observed whether they experienced complications. The team was surprised that half of the patients did not experience reverse of the sickle disease in an average of 3.4 years.

"Typically, stem-cell recipients must take immunosuppressants all their lives," said Dr. Hsieh. "That the patients who discontinued this medication were able to do so safely points to the stability of the partial transplant regimen."

The researchers believed that the results of their study could significantly improve the quality of life of SCD patients by providing an alternative treatment for those who could not endure a full stem cell transplant or lifetime use of immunosuppressants.

Further details of the study were published in the July 1 issue of the Journal of the American Medical Association.

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