Hydroxyurea to Increase Hemoglobin Level in Thalassemia Intermedia.

Hydroxyurea (HU) has been shown to increase fetal hemoglobin (HbF) levels in patients with Sickle Cell Disease and Thalassemia Intermedia. Because there are no defined protocols for the use of this drug, we studied the effect of HU in a group of thalassemia intermedia (genetically defined) patients with at least one of the following features:

1. Low hemoglobin level ( hemoglobin < 7 gr/dl)

2. Bulky extramedullary erythroid masses

We started the enrollment in February 2003 and 8 patients with a minimum follow up of 8 months have been evaluated ( range 8–16 months ) ( mean 13,6 months; median 14 months ). Mean age of the patients was 39.8 years ( range 28–49 years); all were not transfused at the moment of the study entry. HU was administered orally at a mean daily dose of 8.8 mg/kg (range 4–12 mg/kg). The drug was well tolerated; only one patient discontinued the treament for 2 months for pleural effusion. Table summarizes the pre and post treatment characteristics of this group of patients. Data reported are median (range), the differences between pre and post treatment values were analyzed with the the Wilcoxon test for non parametric observations. In this group of patients a mean increase of hemoglobin level of 1.2 g/dl was registered. Extramedullary erythroid expansion, studied by computed tomography scan did not show any change in this follow-up period.

These results confirm that HU may improve Hb level in thalassemia intermedia patients even if other aspects must be studied and longer follow-up is necessary.

Treatment results