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  • Title: [Effectiveness and acceptance of hydroxyurea in the treatment of severe forms of sickle cell disease: a prospective study of 65 cases].
    Author: Mellouli F, Chouaibi S, Dhouib N, Ouederni M, Ben Khaled M, Abbes S, Bejaoui M.
    Journal: Tunis Med; 2013; 91(8-9):544-50. PubMed ID: 24227514.
    Abstract:
    BACKGROUND: Sickle cell disease is a serious illness by its complications. For the severe forms, three therapeutic options are actually allowed: transfusion therapy, hydroxyurea and bone marrow transplantation. aim: To evaluate the contribution of hydroxyurea in the management of severe forms of sickle cell disease. methods: It is a prospective study carried out a period of 11 years in "Centre National de Greffe de Moelle Osseuse" of Tunis. They were 65 patients divided into 38 homozygote forms and 27 double heterozygous composite S/β thalassemics. The mean age was 130 months. The failure criterion of the treatment was hospitalization duration more than 15 days/ patient / year or the occurrence of a severe complication of the disease. results: The main indications of hydroxyurea were the prevention of the recidivism of an acute chest syndrome in 8 cases, iterative painful crises, more than 3 events per year, in 53 cases and anemic forms of the disease in 4 cases. We have observed, a rapid and durable improvement in the clinical manifestations and a significant fall of the number of hospitalization days / patient/year from 25.2 days to 2.6 days (p<0.001). The treatment was well tolerated. The rates of foetal hemoglobin have significantly increased from 6.4 to 27.45 % (p<0.001), of hemoglobin from 7.6 to 9.4 g/dl(p<0.001), of the mean corpuscular volume from 80.3 to 99.1 fl (p<0.001), and a significant fall of the white blood cell rate from 15077 to 8170/mm3 (p<0.001), of polynuclear neutrophils from 8015 to 3509/mm3 (p<0.001), and reticulocytes from 693736 to 209837 /mm3(p<0.001) was observed.Ten patients were considered as treatment failure with a failure rate of 15.3%. The main failure etiology was represented with bad observance. CONCLUSION: Hydroxyurea has a favored place in management of severe forms of sickle cell diseases of the child. Carefully used, with frequent monitoring does not have problems in short range but acceptance studies on the long term mast be undertaken.
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