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Efficacy of hydroxyurea in providing transfusion independence in ß-thalassemia.
Ansari, Saqib H; Shamsi, Tahir S; Ashraf, Mushtaq; Perveen, Kousar; Farzana, Tasneem; Bohray, Muneera; Erum, Sajida; Mehboob, Tabassum.
Afiliação
  • Ansari SH; Department of Pediatric Hematology, National Institute of Blood Diseases, Karachi, Pakistan. ansarisaqib@hotmail.com
J Pediatr Hematol Oncol ; 33(5): 339-43, 2011 Jul.
Article em En | MEDLINE | ID: mdl-21602718
BACKGROUND: Packed red blood cell (PRC) transfusion with iron chelation is the mainstay of treatment for ß-thalassemia major. This prospective interventional trial serves as a follow up to our similar earlier study that evaluated the efficacy and safety of hydroxyurea (HU) in minimizing PRC transfusions in patients with ß-thalassemia major. METHODS: One hundred fifty-two patients with ß-thalassemia major received HU at a mean dose of 16 mg/kg/d. The results were analyzed at the end of 24 months. Transfusion requirement during the 6 months preceding the study was considered as the control. RESULTS: One hundred forty-six of 152 patients were evaluated after 24 months of follow up; 6 patients were either lost to follow-up or withdrew consent. Grade 1 myelosuppression was observed in 4 patients and diarrhea in 2 patients. Sixty children (41%) did not require any transfusion after using HU; 57 patients (39%) showed partial response with greater than 50% reduction in PRC transfusion; and 29 patients (20%) were nonresponders with less than 50% reduction in PRC transfusion. The mean volume of PRC transfused was reduced for all patients. CONCLUSIONS: HU was found to be safe in patients with ß-thalassemia major, and resulted in the reduction of transfusion requirement and in an increase in the interval between transfusions.
Assuntos

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Talassemia beta / Transfusão de Eritrócitos / Inibidores da Síntese de Ácido Nucleico / Hidroxiureia Idioma: En Ano de publicação: 2011 Tipo de documento: Article

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Talassemia beta / Transfusão de Eritrócitos / Inibidores da Síntese de Ácido Nucleico / Hidroxiureia Idioma: En Ano de publicação: 2011 Tipo de documento: Article