Impact of hydroxyurea on clinical events in the BABY HUG trial

Blood. 2012 Nov 22;120(22):4304-10; quiz 4448. doi: 10.1182/blood-2012-03-419879. Epub 2012 Aug 22.


The Pediatric Hydroxyurea Phase 3 Clinical Trial (BABY HUG) was a phase 3 multicenter, randomized, double-blind, placebo-controlled clinical trial of hydroxyurea in infants (beginning at 9-18 months of age) with sickle cell anemia. An important secondary objective of this study was to compare clinical events between the hydroxyurea and placebo groups. One hundred and ninety-three subjects were randomized to hydroxyurea (20 mg/kg/d) or placebo; there were 374 patient-years of on-study observation. Hydroxyurea was associated with statistically significantly lower rates of initial and recurrent episodes of pain, dactylitis, acute chest syndrome, and hospitalization; even infants who were asymptomatic at enrollment had less dactylitis as well as fewer hospitalizations and transfusions if treated with hydroxyurea. Despite expected mild myelosuppression, hydroxyurea was not associated with an increased risk of bacteremia or serious infection. These data provide important safety and efficacy information for clinicians considering hydroxyurea therapy for very young children with sickle cell anemia. This clinical trial is registered with the National Institutes of Health (NCT00006400,

Publication types

  • Clinical Trial, Phase III
  • Multicenter Study
  • Randomized Controlled Trial
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Acute Chest Syndrome / chemically induced
  • Acute Chest Syndrome / diagnosis
  • Acute Chest Syndrome / epidemiology
  • Anemia, Sickle Cell / complications
  • Anemia, Sickle Cell / drug therapy*
  • Anemia, Sickle Cell / epidemiology
  • Antisickling Agents / adverse effects
  • Antisickling Agents / therapeutic use
  • Child, Preschool
  • Double-Blind Method
  • Drug-Related Side Effects and Adverse Reactions / epidemiology
  • Female
  • Hospitalization / statistics & numerical data
  • Humans
  • Hydroxyurea / adverse effects*
  • Hydroxyurea / therapeutic use*
  • Infant
  • Inflammation / chemically induced
  • Inflammation / diagnosis
  • Inflammation / epidemiology
  • Male
  • Pain / chemically induced
  • Pain / diagnosis
  • Pain / epidemiology
  • Placebos


  • Antisickling Agents
  • Placebos
  • Hydroxyurea

Associated data