Treatment is often centered around the use of narcotics for pain control, along with frequent blood transfusions and hospitalizations.
The good news: bone marrow transplants involving fully matched tissue donors have shown the potential to effect a cure in some patients.
The bad news: the vast majority of patients are black (with about one in every 400 blacks struck by the disease) and national donor registries are limited, leaving many to wait in vain for a donor match.
Brodsky and his team performed bone marrow transplants involving both fully matched donors (three cases) and half-matched donors (14 cases), among patients between 15 and 46 years of age.
Both full and partial donor matches were drawn from family members such as parents, children and/or siblings. And all patients underwent a comparatively "gentle" pre-procedure prep regimen of immuno-suppression, chemotherapy and radiation treatment, the study authors said.
The result: 11 of the 17 transplants were deemed "successful." And of these 11 patients, eight had undergone half-matched operations.
The study authors said there were no fatalities. They concluded that the half-matched approach appeared to produce a success rate greater than 50 percent.
Dr. Zora Rogers, clinical director of the General Hematology Program at the Children's Medical Center in Dallas, said that while the findings are "encouraging," they should be interpreted with caution.
"First of all," she said, "it's important to recognize that this half-matched procedure performed predominantly among adults was not as good as a fully matched transplant performed among children would be, where the cure rate exceeds 90 percent. In fact, when you look at the 11 patients they say got better, six of them, if you will, were really 'cured' of their disease
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