Published in:
01-07-2005 | Original Article
Hydroxyurea in thalassemia intermedia—a promising therapy
Authors:
Ashish Dixit, T. C. Chatterjee, Pravas Mishra, Dharma R. Choudhry, M. Mahapatra, S. Tyagi, Madhulika Kabra, Renu Saxena, V. P. Choudhry
Published in:
Annals of Hematology
|
Issue 7/2005
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Abstract
Pharmacological agents such as hydroxyurea (HU) have been known to cause induction of fetal hemoglobin and possibly may alleviate the symptoms in thalassemia intermedia patients. Thirty-seven patients with β-thalassemia intermedia were enrolled to assess response to HU therapy. Major response was defined as transfusion independence or hemoglobin rise of more than 20 g/l and minor response as rise in hemoglobin of 10–20 g/l or reduction in transfusion frequency by 50%. The median age was 10 years (range: 4–50 years) and median follow-up was 12 months (range: 4–36 months). Twenty-six patients (70.2%) showed response to HU therapy. Seventeen patients (45.9%) were major responders, and nine patients (24.3%) showed minor response. There was no correlation of response with β-thalassemia mutation or XmnI polymorphism; however, the presence of α3.7 deletion was associated with major response in three patients. Mean fetal hemoglobin (HbF) levels rose on HU therapy. Older age, low baseline F cell percent, and low baseline HbF levels (below 10%) were predictors of poor response. Response was evident within 1 month of starting HU therapy in the majority of responders. Thus, a short trial of HU therapy can predict durable response.