# Hydroxyurea Reduces Pain Events in Very Young Children with Sickle Cell Disease

 

 

      DynaMed Weekly Update - Volume 6, Issue 20 

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](http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?db=pubmed&cmd=Retrieve&dopt=Abstract&list%5Fuids=7715639&)) and has been associated with reduced hospitalization in school-age children ([Pediatrics 2008 Dec;122(6):1332](http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?db=pubmed&cmd=Retrieve&dopt=Abstract&list%5Fuids=19047254&)), 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****](http://www.epnet.com/dynamed/levels.php)**). 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](http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=pubmed&dopt=Abstract&list%5Fuids=21571150&)).

For more information, see the [Sickle cell disease](http://search.ebscohost.com/login.aspx?direct=true&site=dynamed&id=AN+115522) topic in DynaMed.