Hydroxyurea prescription, availability and use for children with sickle cell disease in Italy: Results of a National Multicenter survey.

BackgroundThe number of patients with sickle cell disease (SCD) has increased in Italy in the past decade due to immigration. In spite of the established efficacy of hydroxyurea (HU) in childhood, population-based data regarding its prescription and effectiveness come mainly from studies performed in adults or outside Europe. Population and methodsThe Hydroxyurea in SCD: A Large Nation-wide Cohort Study from Italy was a retrospective cohort study of adult and pediatric patients with SCD attending 32 centers. Pediatric data are analyzed separately. ResultsOut of 504 children followed in 11 centers, 206 (40%) were on HU (194 SS/S degrees, 12 SC/S ss+); 74% came from Sub-Saharian Africa and 18% from Europe. HU therapy indications for SS/S degrees patients were as follows: 57% painful vaso-occlusive crisis, acute chest syndrome or both, 24% anemia, 8% anemia, and other reasons (the majority had Hb 8-8.5g/dl, revealing scarce acceptance of low Hb values by pediatric hematologist). Mean starting dose was 15.5mg/kg, and dose at full regimen was 17.1mg/kg. Mean age at HU therapy was 7.68 years, although it was lower for SS/S degrees patients. Only 10% started HU before 3 years. In 92%, 500mg capsule was used; in 6%, the galenic was used; and in 2%, 100mg tablet was used. Significant reduction of clinical events and inpatients admissions, with improvement in hematological parameters, was observed for SS/S degrees patients and a trend toward improvement for SC/S ss+ patients was also observed. ConclusionsHU effectiveness is demonstrated in a national cohort of children with SCD living in Italy, even at a lower dose than recommended, revealing good adherence to a treatment program by a socially vulnerable group of patients such as immigrants.