Hydroxyurea in the Treatment

In: Disease

31 May 2010

HydroxyureaApproximately 90,000 Americans have sickle cell disease, making it the most common genetic disease in the U.S. Its occurrence is most common in people who are descendants of residents in regions of the world where the parasite-borne disease malaria is prevalent. The ethnic groups most affected are African-Americans, Arabs, Turks, Greeks, Italians, Iranians, and Asiatic Indians.

Sickle cell anemia is an inherited, chronic disease in which the red blood cells assume a crescent (sickled) shape instead of the normal disc shape. As a result, the cells function abnormally and cause small blood clots. These clots give rise to recurrent painful episodes called “sickle cell pain crises.” Patients with sickle cell disease need treatment and follow-up even when they are not experiencing a painful crisis. Leg ulcers, blindness, kidney damage, lung damage, strokes, and acute chest syndrome are a few complications that sometimes result secondary to sickle cell anemia.

In this article, we attempt to create an awareness of sickle cell anemia and its symptoms and to help readers identify treatment options, with an emphasis on the use of hydroxyurea (Bristol-Myers Squibb). Therapy is the first effective pharmacological intervention that provides clinically significant prevention of complications in sickle cell disease and decreases the need for blood transfusions; it also reduces pain events and hospital admissions by 50%.


Although many ethnic groups are affected by sickle cell anemia, the African-American population seems to have the greatest predilection for the disease; one of every 500 African-Americans have sickle cell disease, and 8% of them have sickle cell trait. As a result of the ability to identify the disease through neonatal screening, the early initiation of penicillin therapy, close medical monitoring, and early intervention to relieve symptoms, the life expectancy for patients with sickle cell anemia has improved despite the absence of effective treatment modalities.

Some patients have one episode every few years; others may have multiple episodes each year. Approximately 10% to 15% of patients have three or more painful crises per year. Rates of early mortality are highest among those with severe disease. The more crises experienced, the greater the probability of premature death.

Half of all patients with sickle cell anemia survive into their 40s. Patients with a more severe form of the disease sometimes die 10 to 15 years earlier than patients with milder cases. Because of a lack of blood oxygen transport, their organs become more damaged.
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Studies have shown that hydroxyurea improves the survival of the most severely affected patients with sickle cell disease. Among sickle cell anemia patients who took hydroxyurea over nine years, there was a 40% reduction in mortality. Hydroxyurea reduced the number of painful crises and episodes of acute chest syndrome by 50%, and patients taking hydroxyurea also required almost 50% fewer transfusions and hospitalizations.

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