Sickle Cell Anemia Patients Get a Partial Reprieve

摘要

In sickle cell anemia, a single genetic mutation leads to abnormal, crescent-shaped red blood cells that clot in tiny blood vessels throughout the body, causing severe pain and eventual organ damage. The most common treatment is almost as debilitating: high-dose radiation therapy to remove the bone marrow that produces the abnormal cells, then a full bone-marrow transplant and a daily regimen of immunosuppressant drugs to keep the body from rejecting the transplanted marrow. But in July, John Tisdale of the National Institutes of Health and his team reported that 26 of 30 adult sickle cell patients recovered after a far less grueling treatment: low-dose radiation, a partial bone-marrow transplant and an antibody to prevent immune attacks on the new marrow. After a few patients serendipitously stopped taking their meds, they remained healthy. The researchers then replicated those results in 15 patients. If larger studies pan out, adult patients will be one step closer to living a pain- and drug-free life.