A pilot study of hydroxyurea to prevent chronic organ damage in young children with sickle cell anemia.

Journal Article (Clinical Trial;Journal Article)

BACKGROUND: Hydroxyurea improves laboratory parameters and prevents acute clinical complications of sickle cell anemia (SCA) in children and adults, but its effects on organ function remain incompletely defined. METHODS: To assess the safety and efficacy of hydroxyurea in young children with SCA and to prospectively assess kidney and brain function, 14 young children (mean age 35 months) received hydroxyurea at a mean maximum tolerated dose (MTD) of 28 mg/kg/day. RESULTS: After a mean of 25 months, expected laboratory effects included significant increases in hemoglobin, MCV and %HbF along with significant decreases in reticulocytes, absolute neutrophil count, and bilirubin. There was no significant increase in glomerular filtration rate by DTPA clearance or Schwartz estimate. Mean transcranial Doppler (TCD) velocity changes were -25.6 cm/sec (P < 0.01) and -26.8 cm/sec (P < 0.05) in the right and left MCA vessels, respectively. At study exit, no child had conditional or abnormal TCD values, and none developed brain ischemic lesions or vasculopathy progression by MRI/MRA. Growth and neurocognitive scores were preserved and Impact-on-Family scores improved. CONCLUSIONS: These pilot data indicate hydroxyurea at MTD is well-tolerated by both children and families, and may prevent chronic organ damage in young children with SCA.

Full Text

Duke Authors

Cited Authors

  • Thornburg, CD; Dixon, N; Burgett, S; Mortier, NA; Schultz, WH; Zimmerman, SA; Bonner, M; Hardy, KK; Calatroni, A; Ware, RE

Published Date

  • May 2009

Published In

Volume / Issue

  • 52 / 5

Start / End Page

  • 609 - 615

PubMed ID

  • 19061213

Pubmed Central ID

  • PMC5600482

Electronic International Standard Serial Number (EISSN)

  • 1545-5017

Digital Object Identifier (DOI)

  • 10.1002/pbc.21738


  • eng

Conference Location

  • United States