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Growth and puberty in African children with sickle cell anemia treated with hydroxyurea

Philippe Backeljauw1,2, George Tomlinson3,4, Luke R Smart2,5,6

  • 1Division of Endocrinology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH.

Blood Advances
|April 9, 2026
PubMed

Insights

Hydroxyurea treatment in children with sickle cell anemia (SCA) improved growth and did not negatively affect pubertal development. This study assessed hydroxyurea

Area of Science:

  • Pediatric Hematology
  • Growth and Development
  • Sickle Cell Anemia Treatment

Background:

  • Children with sickle cell anemia (SCA) often experience impaired growth and delayed pubertal development.
  • Hydroxyurea is a potential therapeutic agent for managing SCA, but its long-term effects on growth and puberty in children require further investigation, especially in resource-limited settings.

Purpose of the Study:

  • To evaluate the feasibility, safety, and benefits of hydroxyurea at maximum tolerated dose (MTD) for children with SCA in sub-Saharan Africa.
  • To assess the impact of long-term hydroxyurea treatment on growth parameters (height, weight, BMI) and pubertal development (Tanner staging, hormone levels) in pediatric SCA patients.

Main Methods:

  • A prospective trial (REACH, NCT01966731) involving children aged 1-10 years with SCA across four sites in sub-Saharan Africa.
  • Open-label hydroxyurea administration at MTD with longitudinal follow-up for up to 7 years.
  • Collection of anthropometric data (height, weight, BMI) and hormonal biomarkers (IGF-I, IGFBP-3, LH, FSH, AMH) at baseline and sequentially.

Main Results:

  • Hydroxyurea treatment was associated with significant improvements in weight-for-age Z-scores, height Z-scores, and BMI Z-scores compared to SCA-specific reference curves.
  • While insulin-like growth factor-I (IGF-I) remained low in many participants, pubertal onset showed gradual progress despite initial delays in 25-30% of children.
  • Anti-Mullerian hormone (AMH) levels, indicative of ovarian reserve and testicular function, were low at baseline in a significant proportion of boys (52%), improving to 28% by follow-up.

Conclusions:

  • Long-term hydroxyurea treatment at MTD demonstrates beneficial effects on growth in children with SCA in sub-Saharan Africa.
  • The treatment does not appear to negatively impact pubertal development, with gradual progression observed.
  • Further research is warranted to address persistent low IGF-I and AMH levels in a subset of patients.

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