title: 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.
authors.affiliation: Department of Haematology, All India Institute of Medical Sciences, New Delhi, India.
authors.forename: Ashish
authors.initials: A
authors.lastname: Dixit
authors.forename: T C
authors.initials: TC
authors.lastname: Chatterjee
authors.forename: Pravas
authors.initials: P
authors.lastname: Mishra
authors.forename: Dharma R
authors.initials: DR
authors.lastname: Choudhry
authors.forename: M
authors.initials: M
authors.lastname: Mahapatra
authors.forename: S
authors.initials: S
authors.lastname: Tyagi
authors.forename: Madhulika
authors.initials: M
authors.lastname: Kabra
authors.forename: Renu
authors.initials: R
authors.lastname: Saxena
authors.forename: V P
authors.initials: VP
authors.lastname: Choudhry
mesh_terms: D000284:Administration, Oral; D000293:Adolescent; D000328:Adult; D000970:Antineoplastic Agents / Q000008:administration & dosage*; D002648:Child; D002675:Child, Preschool; D015672:Erythroid Precursor Cells / Q000378:metabolism; D005260:Female; D005786:Gene Expression Regulation / Q000187:drug effects; D006410:Hematopoiesis / Q000187:drug effects; D006454:Hemoglobins / Q000032:analysis / Q000235:genetics; D006801:Humans; D006918:Hydroxyurea / Q000008:administration & dosage*; D008297:Male; D008875:Middle Aged; D012150:Polymorphism, Restriction Fragment Length; D017086:beta-Thalassemia / Q000097:blood / Q000188:drug therapy* / Q000235:genetics
chemical_list: D000970:Antineoplastic Agents; D006454:Hemoglobins; D006918:Hydroxyurea