A study sponsored by the National Institutes of Health and published in the New England Journal of Medicine has found that custom-crafted stem cell transplantation can reverse sickle cell disease in adults. Until now, only children have received stem cell transplants because the procedure—which has required that bone marrow be completely destroyed—was too toxic for adults damaged by long-term disease.
   In the study, which included 10 adults with severe disease, researchers administered alemtuzumab and low-dose, whole-body radiation insufficient to destroy the bone marrow. Then they transplanted healthy, donor-matched stem cells and gave sirolimus to reduce the risk of rejection. Researchers hoped the healthy cells would live alongside and eventually replace the defective cells.
   At the 30-month follow-up, 9 of the 10 patients had bone marrow filled with healthy cells. Serious adverse effects included sirolimus-related pneumonitis and arthralgia, but no patients developed acute or chronic graft-versus-host disease.
   The researchers concluded that partial stem cell transplantation plus treatment with total-body irradiation, alemtuzumab, and sirolimus can achieve stable, mixed donor-recipient chimerism and, because the donor cells eventually replace the sickled cells, can cure sickle cell disease. Eventually, scientists hope to try the treatment using half-matched cells from a sibling, parent, or child rather than the harder to find, fully matched cells.
   Sickle cell disease results from a genetic mutation that causes formation of crescent-shaped red blood cells that block blood flow and may cause anemia, severe pain, stroke, and organ damage. In the United States, about 80,000 people have the disease, most of African descent. (N Eng J Med, 361:2309-2317)

Home - Guest Registry - Customer Service

Brought to you by Lippincott Williams & Wilkins.
For help on how you should use this site, see our Disclaimer .

© Copyright 2010 by Lippincott Williams & Wilkins