Early drug therapy may help sickle cell patients

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Treatment with hydroxyurea has been shown to increase survival in patients with sickle cell anemia and now new research suggests that further gains might be achieved if the drug is begun earlier in patients at risk for poor outcomes.

Sickle cell anemia is an inherited disease in which red blood cells assume a sickle shape rather than their normal disc-like shape. This can cause serious problems, including painful sickle cell crises.

"This study emphasizes the fact that hydroxyurea is not effective in all patients with sickle cell disease, and those who die while on hydroxyurea therapy may represent a subgroup of older patients, possible with more severe disease and organ damage,” Dr. Abdullah Kutlar and colleagues, from the Medical College of Georgia in Augusta, note.

The findings, which appear in the medical journal Blood, stem from a study of 226 patients who were treated with hydroxyurea at the researchers’ institution. Of these patients, 38 died—including 34 from sickle cell-related causes.

The most common cause of death, present in 12 patients, involved low blood flow the heart, the researchers point out. The next most common cause was multiorgan failure, followed by stroke, and advanced kidney disease. The cause of death could not be determined for 7 patients.

In addition to being older than surviving patients when hydroxyurea was started, deceased patients were more anemic, more likely to have certain disease subtypes, and had impaired kidney function, the authors note.

In a related editorial, Dr. Martin H. Steinberg, from Boston University School of Medicine, comments that “even treated with hydroxyurea, severe sickle cell anemia has higher mortality than milder disease. Understanding how early in the disease process this treatment can be safely started is now paramount.”

SOURCE: Blood, January 15, 2005.

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