Bone Marrow Transplants May Cure Sickle Cell in Adults

Dec. 10 -- WEDNESDAY, Dec. 9 (HealthDay News) -- Researchers say that a new method of bone marrow transplantation cured nine out of 10 adult patients with sickle cell disease, an inherited condition that causes bouts of severe pain, organ damage and sharply limits life expectancy.

Adults have typically not been candidates because they were thought to be too sick to handle the high doses of chemotherapy and radiation necessary to prep the body for the procedure, explained senior study author Dr. John Tisdale, a senior investigator in the molecular and clinical hematology branch at the U.S. National Institutes of Health.

Until now, transplantation was generally reserved for more resilient children, whose bodies had not yet suffered as much damage from sickle cell disease.

But the new method allows for a less grueling pre-transplantation routine, one that even adults with severe sickle cell can tolerate.

More than 70,000 Americans suffer from sickle cell disease, and it is especially common among blacks. People with the disease have abnormal, crescent-shaped hemoglobin. The abnormal cells have difficulty passing through small blood vessels, causing blockages and damaging tissues. Over time, the damage can lead to stroke and severe bouts of pain in the chest, arms, legs, chest and abdomen. Sickle cell disease also damages the kidneys, liver and spleen, leaving people, especially children, more susceptible to infection, said Dr. Lanetta Jordan, chief medical officer for the Sickle Cell Disease Association of America.

Treatments include prophylactic antibiotics to fight infections, blood transfusions and hydroxyurea, the only drug U.S. Food and Drug Administration-approved drug for treating sickle cell, Jordan said.

In the new study, Tisdale and his colleagues gave 10 patients ages 16 to 45 with severe sickle cell disease alemtuzumab, a drug used to suppress immune system T-cells; relatively low doses of radiation; and sirolimus, an immune suppressant to fight rejection. Marrow donors were siblings with matched HLA (human leukocyte antigen) markers in their blood.