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Hydroxyurea in thalassemia intermedia--a promising therapy.

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 beta-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 beta-thalassemia mutation or XmnI polymorphism; however, the presence of alpha(3.7) 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.

journal_name

Ann Hematol

journal_title

Annals of hematology

authors

Dixit A,Chatterjee TC,Mishra P,Choudhry DR,Mahapatra M,Tyagi S,Kabra M,Saxena R,Choudhry VP

doi

10.1007/s00277-005-1026-4

subject

Has Abstract

pub_date

2005-07-01 00:00:00

pages

441-6

issue

7

eissn

0939-5555

issn

1432-0584

journal_volume

84

pub_type

临床试验,杂志文章

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