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Updated: Jul 16, 2025

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Effect of Hydroxyurea Therapy on Growth Parameters in Older Children (6-15 Year-Old) with Sickle Cell Disease: Low
Doaa Khater1,2, Sharef Al-Mulaabed3, Anwar Alomairi2
1Department of Pediatrics, Faculty of Medicine, Alexandria University, Alexandria, Egypt.
Insights
Hydroxyurea (HU) therapy did not negatively impact height and weight in children with sickle cell disease (SCD). However, body mass index (BMI) Z-scores improved in patients on low-dose HU over 18 months.
Area of Science:
- Pediatric Hematology
- Growth and Development
- Pharmacological Effects
Background:
- Growth impairment is a recognized complication in children with sickle cell disease (SCD).
- Limited research exists on the specific impact of hydroxyurea (HU) dosage and response on growth parameters in pediatric SCD patients.
- Understanding HU's effect on growth is crucial for optimizing treatment strategies in this population.
Purpose of the Study:
- To investigate the effects of hydroxyurea (HU) on growth parameters (weight, height, BMI) in children with SCD.
- To correlate HU's impact on growth with clinical improvement and dosage.
- To compare growth outcomes against WHO standards.
Main Methods:
- Prospective study involving 91 children with SCD under 16 years old initiating HU therapy.
- Collection of anthropometric data (weight, height, BMI Z-scores) at baseline, 6, and 18 months.
- Comparison of anthropometric data with World Health Organization (WHO) standards and analysis of dose-dependent effects.
Main Results:
- Initial height and BMI Z-scores were below WHO norms but did not significantly change after 6 or 18 months of HU therapy.
- A significant improvement in BMI Z-scores was observed at 6 and 18 months (p<0.05).
- Low-dose HU showed a significant improvement in BMI Z-scores after 18 months, whereas high-dose HU did not yield significant changes in BMI, height, or weight Z-scores.
Conclusions:
- Hydroxyurea (HU) therapy is safe concerning weight and height growth in older children with SCD.
- BMI Z-scores demonstrated improvement at 18 months, particularly in the low-dose HU group, suggesting a potential benefit.
- Further research may elucidate optimal HU dosing strategies to maximize growth benefits in pediatric SCD patients.
Abstract:
Growth impairment is a known complication of sickle cell disease (SCD). Few studies explored the potential effects of hydroxyurea (HU) on growth in children with SCD in relation to HU dose and response. This is a prospective study conducted at Sultan Qaboos University Hospital, Oman, and included 91 SCD patients with age below 16 years when started on HU, aiming to explore the potential effect/s of HU on growth parameters of older children with SCD in relation to their clinical improvement and the dose required for this improvement. Weight, height, and body mass index (BMI) were collected at baseline, 6 and 18 months after initiation. Anthropometric data were compared to WHO standards. Initial height and BMI Z scores (HAZ and WAZ) were lower compared to WHO norms. HAZ and WAZ did not change significantly after 6 and 18 months on HU therapy. However, BMI Z-scores improved significantly after 6 and 18 months of follow-up (p value 0.044 and 0.028 respectively). No significant changes were observed in WAZ or HAZ among patients on low dose versus those on high dose. BMI Z score improved significantly after 18 months of low dose group (p = 0.014) but did not change in those on high dose HU. In conclusion, HU therapy did not adversely affect weight and height growth in older children with SCD. BMI Z scores improved at 18 months in patients on low dose but not in those on high dose (p = 0.014).
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