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Hydroxyurea Effective With Few Side Effects in the Sickest Sickle Cell Patients


"Perhaps the reason that hydroxyurea is reducing mortality is that it's preventing these vascular complications that are the chief acute problem of patients with sickle cell disease," says Steinberg. However, he notes that this study was not designed to assess why the drug works. Steinberg recommends that patients treated with hydroxyurea be followed closely.

"We selected the sickest patients [to study]," says Steinberg. "So I think it's very good news that, in the sickest group of patients, we appear to be extending their life. ... If it works this well in the very sickest people, perhaps it will work equally well, or possibly better, in people who are not so sick to begin with." Currently, the investigators are attempting to corroborate their findings by continuing the study with more patients over a longer period of time.

Vital Information:

  • Sickle cell anemia is an inherited blood disorder that causes red blood cells to form a sickle shape, leaving them fragile, unable to carry oxygen, and more prone to clogging blood vessels.
  • The drug hydroxyurea improves the ability of red blood cells to carry oxygen and decreases breakage of the cells among patients with moderate to severe sickle cell anemia.
  • Patients taking hydroxyurea experienced lower death rates, decreased pain attacks and fewer pneumonialike episodes.
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