Painful symptoms of sickle cell disease begin within the first year of life. Hydroxyurea (hydroxycarbamide) has been shown to reduce pain in adults (N Engl J Med 1995 May 18;332(20):1317) and has been associated with reduced hospitalization in school-age children (Pediatrics 2008 Dec;122(6):1332), but it has not previously been investigated in toddlers. The BABY-HUG trial evaluated the effects of hydroxyurea in 193 children aged 9-18 months. Children with sickle cell disease were randomized to hydroxyurea 20 mg/kg/day vs. placebo for 2 years.
The hydroxyurea group had significantly lower rates of pain events (177 events in 62 patients vs. 375 events in 75 patients, p = 0.002) and dactylitis (24 events in 14 patients vs. 123 events in 42 patients, p< 0.0001) (level 1 [likely reliable] evidence). Hydroxyurea was also associated with reduced gastroenteritis (p = 0.001) and trends toward reductions in acute chest syndrome, transfusions, and hospitalizations. The risk of mild-moderate neutropenia was increased in the hydroxyurea group, but no other treatment-related adverse events were noted. There were no significant differences in the primary surrogate outcomes of splenic and renal function (Lancet 2011 May 14;377(9778):1663).
For more information, see the Sickle cell disease topic in DynaMed.