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.
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Dixit, A., Chatterjee, T.C., Mishra, P. et al. Hydroxyurea in thalassemia intermedia—a promising therapy. Ann Hematol 84, 441–446 (2005). https://doi.org/10.1007/s00277-005-1026-4
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DOI: https://doi.org/10.1007/s00277-005-1026-4