Hydroxyurea and growth in young children with sickle cell disease

Sohail Rana, Patricia E. Houston, Winfred C. Wang, Rathi V. Iyer, Jonathan Goldsmith, James F. Casella, Caroline K. Reed, Zora R. Rogers, Myron A. Waclawiw, Bruce Thompson

Research output: Contribution to journalArticle

23 Citations (Scopus)

Abstract

BACKGROUND: Growth impairment is a known complication of sickle cell disease. Effects of hydroxyurea (HU) on growth in very young children are not known.

METHODS: Height, weight, BMI, and head circumference (HC) were compared with World Health Organization (WHO) standards in BABY HUG, a multicenter, randomized, double-blinded, placebo-controlled 2-year clinical trial of HU in 193 children 9 to 18 months of age. Anthropometric data were closely monitored and converted to z scores by using WHO standardized algorithms for descriptive analyses. The treatment and placebo groups were compared longitudinally by using a mixed model analysis.

RESULTS: At entry, the z scores of BABY HUG children were higher than WHO norms. After 2 years of HU or placebo treatment, there were no significant differences between the groups, except for the mean HC z scores at study exit (HU: +0.8 versus placebo: +1.0, P = .05). Baseline z scores were the best predictors of z scores at study exit. The absolute neutrophil count, absolute reticulocyte count, and total white blood cell count had significant negative correlations with growth measures.

CONCLUSIONS: Both groups had normal or near normal anthropometric measures during the study. The HC z scores at study entry and exit were slightly greater than WHO norms. Higher baseline white blood cell count, absolute reticulocyte count, and absolute neutrophil count were associated with poorer growth. The significance of the slightly lower HC in the treatment group at study exit is not clear. Trends toward normalization of weight and height and effects on HC will be monitored in ongoing BABY HUG follow-up studies.

Original languageEnglish (US)
Pages (from-to)465-472
Number of pages8
JournalPediatrics
Volume134
Issue number3
DOIs
StatePublished - Sep 1 2014

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Hydroxyurea
Sickle Cell Anemia
Head
Placebos
Reticulocyte Count
Growth
Leukocyte Count
Neutrophils
Weights and Measures
Therapeutics
Sickles
Cells
Young children
Clinical Trials
World Health Organization
Exit
Placebo

Keywords

  • BMI
  • Children
  • Growth
  • Head circumference
  • Height
  • Hydroxyurea
  • Sickle cell disease
  • Weight

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Arts and Humanities (miscellaneous)

Cite this

Rana, S., Houston, P. E., Wang, W. C., Iyer, R. V., Goldsmith, J., Casella, J. F., ... Thompson, B. (2014). Hydroxyurea and growth in young children with sickle cell disease. Pediatrics, 134(3), 465-472. https://doi.org/10.1542/peds.2014-0917

Hydroxyurea and growth in young children with sickle cell disease. / Rana, Sohail; Houston, Patricia E.; Wang, Winfred C.; Iyer, Rathi V.; Goldsmith, Jonathan; Casella, James F.; Reed, Caroline K.; Rogers, Zora R.; Waclawiw, Myron A.; Thompson, Bruce.

In: Pediatrics, Vol. 134, No. 3, 01.09.2014, p. 465-472.

Research output: Contribution to journalArticle

Rana, S, Houston, PE, Wang, WC, Iyer, RV, Goldsmith, J, Casella, JF, Reed, CK, Rogers, ZR, Waclawiw, MA & Thompson, B 2014, 'Hydroxyurea and growth in young children with sickle cell disease', Pediatrics, vol. 134, no. 3, pp. 465-472. https://doi.org/10.1542/peds.2014-0917
Rana S, Houston PE, Wang WC, Iyer RV, Goldsmith J, Casella JF et al. Hydroxyurea and growth in young children with sickle cell disease. Pediatrics. 2014 Sep 1;134(3):465-472. https://doi.org/10.1542/peds.2014-0917
Rana, Sohail ; Houston, Patricia E. ; Wang, Winfred C. ; Iyer, Rathi V. ; Goldsmith, Jonathan ; Casella, James F. ; Reed, Caroline K. ; Rogers, Zora R. ; Waclawiw, Myron A. ; Thompson, Bruce. / Hydroxyurea and growth in young children with sickle cell disease. In: Pediatrics. 2014 ; Vol. 134, No. 3. pp. 465-472.
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abstract = "BACKGROUND: Growth impairment is a known complication of sickle cell disease. Effects of hydroxyurea (HU) on growth in very young children are not known.METHODS: Height, weight, BMI, and head circumference (HC) were compared with World Health Organization (WHO) standards in BABY HUG, a multicenter, randomized, double-blinded, placebo-controlled 2-year clinical trial of HU in 193 children 9 to 18 months of age. Anthropometric data were closely monitored and converted to z scores by using WHO standardized algorithms for descriptive analyses. The treatment and placebo groups were compared longitudinally by using a mixed model analysis.RESULTS: At entry, the z scores of BABY HUG children were higher than WHO norms. After 2 years of HU or placebo treatment, there were no significant differences between the groups, except for the mean HC z scores at study exit (HU: +0.8 versus placebo: +1.0, P = .05). Baseline z scores were the best predictors of z scores at study exit. The absolute neutrophil count, absolute reticulocyte count, and total white blood cell count had significant negative correlations with growth measures.CONCLUSIONS: Both groups had normal or near normal anthropometric measures during the study. The HC z scores at study entry and exit were slightly greater than WHO norms. Higher baseline white blood cell count, absolute reticulocyte count, and absolute neutrophil count were associated with poorer growth. The significance of the slightly lower HC in the treatment group at study exit is not clear. Trends toward normalization of weight and height and effects on HC will be monitored in ongoing BABY HUG follow-up studies.",
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AU - Goldsmith, Jonathan

AU - Casella, James F.

AU - Reed, Caroline K.

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AU - Waclawiw, Myron A.

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N2 - BACKGROUND: Growth impairment is a known complication of sickle cell disease. Effects of hydroxyurea (HU) on growth in very young children are not known.METHODS: Height, weight, BMI, and head circumference (HC) were compared with World Health Organization (WHO) standards in BABY HUG, a multicenter, randomized, double-blinded, placebo-controlled 2-year clinical trial of HU in 193 children 9 to 18 months of age. Anthropometric data were closely monitored and converted to z scores by using WHO standardized algorithms for descriptive analyses. The treatment and placebo groups were compared longitudinally by using a mixed model analysis.RESULTS: At entry, the z scores of BABY HUG children were higher than WHO norms. After 2 years of HU or placebo treatment, there were no significant differences between the groups, except for the mean HC z scores at study exit (HU: +0.8 versus placebo: +1.0, P = .05). Baseline z scores were the best predictors of z scores at study exit. The absolute neutrophil count, absolute reticulocyte count, and total white blood cell count had significant negative correlations with growth measures.CONCLUSIONS: Both groups had normal or near normal anthropometric measures during the study. The HC z scores at study entry and exit were slightly greater than WHO norms. Higher baseline white blood cell count, absolute reticulocyte count, and absolute neutrophil count were associated with poorer growth. The significance of the slightly lower HC in the treatment group at study exit is not clear. Trends toward normalization of weight and height and effects on HC will be monitored in ongoing BABY HUG follow-up studies.

AB - BACKGROUND: Growth impairment is a known complication of sickle cell disease. Effects of hydroxyurea (HU) on growth in very young children are not known.METHODS: Height, weight, BMI, and head circumference (HC) were compared with World Health Organization (WHO) standards in BABY HUG, a multicenter, randomized, double-blinded, placebo-controlled 2-year clinical trial of HU in 193 children 9 to 18 months of age. Anthropometric data were closely monitored and converted to z scores by using WHO standardized algorithms for descriptive analyses. The treatment and placebo groups were compared longitudinally by using a mixed model analysis.RESULTS: At entry, the z scores of BABY HUG children were higher than WHO norms. After 2 years of HU or placebo treatment, there were no significant differences between the groups, except for the mean HC z scores at study exit (HU: +0.8 versus placebo: +1.0, P = .05). Baseline z scores were the best predictors of z scores at study exit. The absolute neutrophil count, absolute reticulocyte count, and total white blood cell count had significant negative correlations with growth measures.CONCLUSIONS: Both groups had normal or near normal anthropometric measures during the study. The HC z scores at study entry and exit were slightly greater than WHO norms. Higher baseline white blood cell count, absolute reticulocyte count, and absolute neutrophil count were associated with poorer growth. The significance of the slightly lower HC in the treatment group at study exit is not clear. Trends toward normalization of weight and height and effects on HC will be monitored in ongoing BABY HUG follow-up studies.

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