E Papadopoulou1, A Teli1, S Theodoridou2, N Gompakis3, M Economou1. 1. 1 Department of Pediatrics, Aristotle University of Thessaloniki, Hippokratio General Hospital, Thessaloniki, Greece. 2. Hemoglobinopathy Prevention Unit, Hippokratio General Hospital, Thessaloniki, Greece. 3. 2 Department of Pediatrics, Aristotle University of Thessaloniki, AHEPA General Hospital, Thessaloniki, Greece.
Abstract
BACKGROUND: Hydroxyurea is a cytotoxic and myelosuppressive drug that has been used during recent years in the treatment of children with severe sickle cell disease. Nevertheless, questions remain regarding its role in young patients with no severe course, like sickle/beta-thalassemia (S/b-thal) patients often present. The aim of the present study was to evaluate the safety and efficacy of hydroxyurea in young patients with S/b-thal, which is the commonest form of the disease in Greece. PATIENTS- METHODS: Hydroxyurea was given in thirteen children with S/b-thal for 24 months and for that period clinical and laboratory evaluation of the children was performed. RESULTS: A reduction in pain crises and rate of hospitalization was noted. None of the patients presented with a severe clinical event, related to the disease during the study period. A significant increase in hemoglobin, hemoglobin F, mean corpuscular volume, and mean corpuscular hemoglobin and a decrease in reticulocyte count, white blood cell and platelet count, and total bilirubin level was noted. With regards to adverse events, these were transient, short-term and dose-dependable. CONCLUSIONS: To the best of our knowledge, this is the first study to specifically assess the effect of hydroxyurea therapy in young patients with S/b-thal and the results indicate is safe and efficacious in this patient cohort. Hippokratia 2015; 19 (2):172-175.
BACKGROUND:Hydroxyurea is a cytotoxic and myelosuppressive drug that has been used during recent years in the treatment of children with severe sickle cell disease. Nevertheless, questions remain regarding its role in young patients with no severe course, like sickle/beta-thalassemia (S/b-thal) patients often present. The aim of the present study was to evaluate the safety and efficacy of hydroxyurea in young patients with S/b-thal, which is the commonest form of the disease in Greece. PATIENTS- METHODS:Hydroxyurea was given in thirteen children with S/b-thal for 24 months and for that period clinical and laboratory evaluation of the children was performed. RESULTS: A reduction in pain crises and rate of hospitalization was noted. None of the patients presented with a severe clinical event, related to the disease during the study period. A significant increase in hemoglobin, hemoglobin F, mean corpuscular volume, and mean corpuscular hemoglobin and a decrease in reticulocyte count, white blood cell and platelet count, and total bilirubin level was noted. With regards to adverse events, these were transient, short-term and dose-dependable. CONCLUSIONS: To the best of our knowledge, this is the first study to specifically assess the effect of hydroxyurea therapy in young patients with S/b-thal and the results indicate is safe and efficacious in this patient cohort. Hippokratia 2015; 19 (2):172-175.
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