Low-dose hydroxyurea shows promise in sickle cell disease
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A reduced hydroxyurea dose yielded similar efficacy and safety outcomes as the standard dose in a cohort of children with sickle cell disease, according to study findings.
The researchers investigated the efficacy of hydroxyurea administered at an initial dose of 10 mg/kg/day to 12 mg/kg/day, which is lower than the standard starting dose of 15 mg/kg/day to 20 mg/kg/day. They aimed to determine the success of a lower starting dose and cautious dose escalation in a cohort of 161 children with sickle cell disease.
Eligible participants were retrospectively examined from 1998 to 2008 and prospectively examined from 2009 to 2011 at Sultan Qaboos University Hospital in Muscat, Oman.
The study included 44 children in a low-dose cohort who received 10 mg/kg/day to 15.9 mg/kg/day, and 98 children in a high-dose cohort who received 16 mg/kg/day to 26 mg/kg/day.
Children in both groups experienced a significant reduction in the annual number of admissions due to vaso-occlusive crisis (P<.001). Researchers observed a statistically insignificant difference between the two groups for these admissions (P>.05). The researchers also reported a clinical improvement in acute chest syndrome.
Significant improvements in biomarkers such as absolute neutrophil count, hemoglobin and mean corpuscular volume were reported in the low- and high-dose groups.
The treatment was well tolerated by all patients in both cohorts, with reversible toxicities linked to the low and high dose.
“In sickle cell disease patients, [a] low-dose regimen of hydroxyurea is a feasible option that ensured safety and yet did not affect efficacy,” the researchers concluded.