Long-term hydroxyurea reduced mortality, morbidity in adults with sickle cell disease

50th ASH Annual Meeting

Long-term treatment with hydroxyurea reduced painful crises, the incidence of hospital admissions and transfusion requirements among adults with sickle cell disease, according to recently presented data.

"Whether hydroxyurea can prevent the severe chronic complications or quantify the morbidity of these patients [with sickle cell disease] remains an interesting and unresolved question," Ersi Voskaridou, MD, Thalassemia Center, Laikon General Hospital in Athens, Greece, said during a press briefing.

Voskaridou and colleagues examined the efficacy of prolonged administration of hydroxyurea on quality of life, frequency and length of hospital stays, complications and mortality in adult patients with sickle cell disease. The study included data for 330 patients with sickle cell disease; median age 42 years. Data from 20 years were prospectively analyzed.

Patients data was divided into two groups: patients treated with hydroxyurea 20 mg/kg/day (n=131) or patients treated with conventional therapy (n=199). Median follow-up was eight years for hydroxyurea and five years for conventional therapy.

Patients in the hydroxyurea group had a 91% probability of 10-year survival compared with 70% for patients in the conventional treatment group.

According to the researchers, patients in the hydroxyurea group experienced a decrease in painful crises (7.4 episodes per year pretreatment to 0.2 episodes post-treatment), reduction of transfusion requirements (mean number of administered packed red cell units 1.5 per year pretreatment to almost zero during treatment). Hospital admissions decreased (2.1 per year pretreatment to 0.06 per year post-treatment) and the researchers reported a drop in the frequency of chest syndrome (6.1% pretreatment to 0.8% during treatment).

There was no difference in aspectic avascular necrosis or cerebrovascular episodes among patients treated or those not treated, according to the researchers. However, the researchers reported fewer deaths in the hydroxyurea group (12 vs. 47; P=.001).

This is a nice article that shows that hydroxyurea not only alleviates symptoms, which we knew, but also improves survival. The population is not that similar to the American population, as it contains more patients with sickle-thalassemia than we have. Nevertheless, the message is clear.

– Wendell Rosse, MD

HemOnc Today Editorial Board member

For more information:

Voskaridou. #1445. Presented at: 50th Annual Meeting of the American Society of Hematology; December 5-9, 2008; San Francisco.