Hydroxyurea as treatment for sickle cell anemia.

Hydroxyurea may be the most promising drug suggested thus far as a treatment for patients with sickle cell anemia, but its safety and efficacy remain unproved, and it probably will not be evaluable for several years. It is not "the answer" for the disease, it seems likely that crises will not be eliminated by treatment, and at this time its use should be reserved for seriously affected adult patients who can participate in a controlled clinical trial. Every hematologist, internist, or pediatrician sees a few patients who are so severely afflicted by sickle cell anemia that their lives seem totally blighted. It is very tempting to consider treating them with hydroxyurea, because the drug is available in any pharmacy, but it is equally important to consider whether the treatment would accomplish any more than treating the physician's own sense of futility, or making the patient feel that "something was being done." If hydroxyurea were as safe as folic acid, treatment for these purposes would be reasonable. It is not, but its use may still be appropriate (although illegal unless used under an Investigational New Drug Agreement, because its use for this purpose is not approved by the FDA). Whether or not it is legal, prescription of hydroxyurea for patients with sickle cell anemia would be ethical or proper only if the potential risks, the variability of the Hb F response, and the lack of proof of clinical efficacy were clearly explained to potential recipients.