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.

Hemoglobin
|September 11, 2023
PubMed

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.

Related Concept Videos

Renal Failure: Dose Adjustments01:11

Renal Failure: Dose Adjustments

In patients with renal impairment, drugs undergo significant changes in their pharmacokinetics, which require dosage adjustments to ensure safe and effective therapy.
Reduced renal clearance and elimination rate are common outcomes of renal impairment. These alterations lead to a prolonged elimination half-life and an altered apparent volume of distribution for drugs. As a result, dosage adjustments are typically necessary to maintain optimal drug levels in the body.
However, dosage adjustments...
115
Role of Hematopoietic Growth Factors01:28

Role of Hematopoietic Growth Factors

Hematopoietic growth factors are molecules that regulate the differentiation rate of hematopoietic stem cells (HSCs). Erythropoietin (EPO), primarily produced by the kidneys, plays a crucial role in erythrocyte production. When oxygen levels in the blood are low, EPO is released into the bloodstream, reaching the bone marrow, where it stimulates HSCs to differentiate and mature into erythrocytes, which are vital for oxygen transport.
Thrombopoietin (TPO), mainly released by the liver,...
1.4K
Factors Affecting Erythropoiesis01:24

Factors Affecting Erythropoiesis

The cardiovascular system regulates the number of erythrocytes in the bloodstream to ensure optimal oxygen transport. It also prevents over-proliferation of these cells, which helps to maintain blood viscosity and flow rate.
Several factors influence the erythrocyte production rate, with tissue oxygen level being among the most critical. Intense exercise or high altitudes can cause tissue hypoxia, which triggers the kidneys to release more erythropoietin (EPO) into the bloodstream.
EPO then...
3.3K
Factors Affecting Drug Response: Overview01:21

Factors Affecting Drug Response: Overview

When it comes to infants and young children, they are typically administered smaller doses of medication in comparison to adults. This is primarily because their organ functions still need to fully develop, meaning their bodies are not as efficient at metabolizing or eliminating drugs. Additionally, their blood-brain barrier is more permeable than in adults. As a result, high concentrations of drugs can easily penetrate the central nervous system (CNS), potentially leading to neurological...
2.0K