Abstract | BACKGROUND: Packed red blood cell (PRC) transfusion with iron chelation is the mainstay of treatment for β- 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 β- thalassemia major. METHODS: One hundred fifty-two patients with β- 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 β- thalassemia major, and resulted in the reduction of transfusion requirement and in an increase in the interval between transfusions.
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Authors | Saqib H Ansari, Tahir S Shamsi, Mushtaq Ashraf, Kousar Perveen, Tasneem Farzana, Muneera Bohray, Sajida Erum, Tabassum Mehboob |
Journal | Journal of pediatric hematology/oncology
(J Pediatr Hematol Oncol)
Vol. 33
Issue 5
Pg. 339-43
(Jul 2011)
ISSN: 1536-3678 [Electronic] United States |
PMID | 21602718
(Publication Type: Controlled Clinical Trial, Journal Article)
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Chemical References |
- Nucleic Acid Synthesis Inhibitors
- Hydroxyurea
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Topics |
- Adolescent
- Child
- Child, Preschool
- Erythrocyte Transfusion
(statistics & numerical data)
- Female
- Follow-Up Studies
- Humans
- Hydroxyurea
(administration & dosage, adverse effects)
- Infant
- Male
- Nucleic Acid Synthesis Inhibitors
(administration & dosage, adverse effects)
- Treatment Outcome
- Young Adult
- beta-Thalassemia
(drug therapy)
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