Benefits of hidroxyurea in children with sickle cell disease

Abstract

Objective: To assess the toxicity and benefits of hydroxyurea (HU) in the management of children with sickle cell disease (SCD).

Materials and methods: a descriptive observational-retrospective study of patients with SCD treated with HU was carried out at Hospital Pablo Tobón Uribe in Medellin, Colombia, from May 2004 to September 2009. Sixteen patients aged under 15 years were treated with this drug; out of them, 11 (68.8%) were male. All patients had sickle cell anemia (Hb SS). The variables were studied before and after initiation of HU.

Results: Average number of painful crises was 3.31 before and 1.13 after HU (p = 0.006); average number of red blood cell transfusions was 2.69 before and 0.75 after HU (p = 0.112); average number of acute chest syndrome episodes was 0.19 before and 0.13 after HU (p = 0.705); average number of hospitalizations was 1.94 before and 1.06 after HU (p = 0.155). One patient (6.3%) had hematologic toxicity, two patients (12.5%) had liver toxicity, and three patients (18.6%) had strokes. Renal toxicity was not found. There were no malignancies.

Conclusion: HU reduced the frequency of painful crisis in our patients with SCD. Toxicity was generally acceptable. Prospective multicenter, double-blind, placebo-controlled studies are required in order to define the role of HU in pediatric patients with SCD.