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Hydroxyurea prescription, availability and use for children with sickle cell disease in Italy: Results of a National Multicenter survey
Author(s) -
Colombatti Raffaella,
Palazzi Giovanni,
Masera Nicoletta,
Notarangelo Lucia Dora,
Bonetti Elisa,
Samperi Piera,
Barone Angelica,
Perrotta Silverio,
Facchini Elena,
Miano Maurizio,
Del Vecchio Giovanni Carlo,
Guerzoni Maria Elena,
Corti Paola,
Menzato Federica,
Cesaro Simone,
Casale Maddalena,
Rigano Paolo,
Forni Gian Luca,
Russo Giovanna,
Sainati Laura
Publication year - 2018
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.26774
Subject(s) - medicine , acute chest syndrome , sickle cell anemia , medical prescription , pediatrics , cohort , anemia , retrospective cohort study , population , regimen , hematologist , disease , environmental health , pharmacology
Background The 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 methods The 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. Results Out of 504 children followed in 11 centers, 206 (40%) were on HU (194 SS/Sβ°, 12 SC/Sß+); 74% came from Sub‐Saharian Africa and 18% from Europe. HU therapy indications for SS/Sβ° 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 ≤ 8–8.5 g/dl, revealing scarce acceptance of low Hb values by pediatric hematologist). Mean starting dose was 15.5 mg/kg, and dose at full regimen was 17.1 mg/kg. Mean age at HU therapy was 7.68 years, although it was lower for SS/Sβ° patients. Only 10% started HU before 3 years. In 92%, 500 mg capsule was used; in 6%, the galenic was used; and in 2%, 100 mg tablet was used. Significant reduction of clinical events and inpatients admissions, with improvement in hematological parameters, was observed for SS/Sβ° patients and a trend toward improvement for SC/Sß+ patients was also observed. Conclusions HU 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.