Impact of hydroxyurea on clinical events in the BABY HUG trial.
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, www.clinicaltrials.gov).
Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA. courtney.thornburg@dm.duke.edu
Courtney D
CD
Thornburg
Beatrice A
BA
Files
Zhaoyu
Z
Luo
Scott T
ST
Miller
Ram
R
Kalpatthi
Rathi
R
Iyer
Phillip
P
Seaman
Jeffrey
J
Lebensburger
Ofelia
O
Alvarez
Bruce
B
Thompson
Russell E
RE
Ware
Winfred C
WC
Wang
D056586:Acute Chest Syndrome / Q000139:chemically induced / Q000175:diagnosis / Q000453:epidemiology; D000755:Anemia, Sickle Cell / Q000150:complications / Q000188:drug therapy* / Q000453:epidemiology; D000986:Antisickling Agents / Q000009:adverse effects / Q000627:therapeutic use; D002675:Child, Preschool; D004311:Double-Blind Method; D064420:Drug-Related Side Effects and Adverse Reactions / Q000453:epidemiology; D005260:Female; D006760:Hospitalization / Q000706:statistics & numerical data; D006801:Humans; D006918:Hydroxyurea / Q000009:adverse effects* / Q000627:therapeutic use*; D007223:Infant; D007249:Inflammation / Q000139:chemically induced / Q000175:diagnosis / Q000453:epidemiology; D008297:Male; D010146:Pain / Q000139:chemically induced / Q000175:diagnosis / Q000453:epidemiology; D010919:Placebos
D000986:Antisickling Agents; D010919:Placebos; D006918:Hydroxyurea