Colombatti R, et al. - This multicenter survey focused on the effectiveness and availability of hydroxyurea (HU) in children with sickle cell disease (SCD). HU showed effectiveness in children with SCD in Italy, even at a lower dose than recommended.

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 were evaluated 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.
• 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.
• 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 .
• A trend toward improvement for SC/Sß+ patients was also reported.