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

Publication ,  Journal Article
Thornburg, CD; Dixon, N; Burgett, S; Mortier, NA; Schultz, WH; Zimmerman, SA; Bonner, M; Hardy, KK; Calatroni, A; Ware, RE
Published in: Pediatr Blood Cancer
May 2009

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.

Duke Scholars

Published In

Pediatr Blood Cancer

DOI

EISSN

1545-5017

Publication Date

May 2009

Volume

52

Issue

5

Start / End Page

609 / 615

Location

United States

Related Subject Headings

  • Ultrasonography, Doppler, Transcranial
  • Spleen
  • Quality of Life
  • Pilot Projects
  • Oncology & Carcinogenesis
  • Male
  • Kidney
  • Hydroxyurea
  • Humans
  • Female
 

Citation

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Thornburg, C. D., Dixon, N., Burgett, S., Mortier, N. A., Schultz, W. H., Zimmerman, S. A., … Ware, R. E. (2009). A pilot study of hydroxyurea to prevent chronic organ damage in young children with sickle cell anemia. Pediatr Blood Cancer, 52(5), 609–615. https://doi.org/10.1002/pbc.21738
Thornburg, Courtney D., Natalia Dixon, Shelly Burgett, Nicole A. Mortier, William H. Schultz, Sherri A. Zimmerman, Melanie Bonner, Kristina K. Hardy, Agustin Calatroni, and Russell E. Ware. “A pilot study of hydroxyurea to prevent chronic organ damage in young children with sickle cell anemia.Pediatr Blood Cancer 52, no. 5 (May 2009): 609–15. https://doi.org/10.1002/pbc.21738.
Thornburg CD, Dixon N, Burgett S, Mortier NA, Schultz WH, Zimmerman SA, et al. A pilot study of hydroxyurea to prevent chronic organ damage in young children with sickle cell anemia. Pediatr Blood Cancer. 2009 May;52(5):609–15.
Thornburg, Courtney D., et al. “A pilot study of hydroxyurea to prevent chronic organ damage in young children with sickle cell anemia.Pediatr Blood Cancer, vol. 52, no. 5, May 2009, pp. 609–15. Pubmed, doi:10.1002/pbc.21738.
Thornburg CD, Dixon N, Burgett S, Mortier NA, Schultz WH, Zimmerman SA, Bonner M, Hardy KK, Calatroni A, Ware RE. A pilot study of hydroxyurea to prevent chronic organ damage in young children with sickle cell anemia. Pediatr Blood Cancer. 2009 May;52(5):609–615.
Journal cover image

Published In

Pediatr Blood Cancer

DOI

EISSN

1545-5017

Publication Date

May 2009

Volume

52

Issue

5

Start / End Page

609 / 615

Location

United States

Related Subject Headings

  • Ultrasonography, Doppler, Transcranial
  • Spleen
  • Quality of Life
  • Pilot Projects
  • Oncology & Carcinogenesis
  • Male
  • Kidney
  • Hydroxyurea
  • Humans
  • Female