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Browse through all publications from the Institute of Global Health Innovation, which our Patient Safety Research Collaboration is part of. This feed includes reports and research papers from our Centre.
@article{Backeljauw:2026:10.1182/bloodadvances.2025019511,
author = {Backeljauw, P and Tomlinson, G and Smart, LR and Tshilolo, LMM and Williams, TN and Santos, B and Olupot-Olupot, P and Stuber, SE and Lane, A and Latham, TS and Ware, RE},
doi = {10.1182/bloodadvances.2025019511},
journal = {Blood Adv},
title = {Growth and puberty in African children with sickle cell anemia treated with hydroxyurea.},
url = {http://dx.doi.org/10.1182/bloodadvances.2025019511},
year = {2026}
}
TY - JOUR
AB - Children with sickle cell anemia (SCA) have poor growth and pubertal development. REACH (Realizing Effectiveness Across Continents with Hydroxyurea, NCT01966731) is a prospective trial evaluating the feasibility, safety, and benefits of hydroxyurea at maximum tolerated dose (MTD) for children with SCA in sub-Saharan Africa. Children 1-10 years old at 4 sites received open-label hydroxyurea with longitudinal follow-up. Height, weight, and pubertal (Tanner) staging were collected at enrollment and sequentially over 7 years of treatment. Biomarkers included insulin-like growth factor-I (IGF-I), insulin-like growth factor binding protein-3 (IGFBP-3), luteinizing hormone (LH), follicle stimulating hormone (FSH), and anti-Mullerian hormone (AMH). Hydroxyurea commenced at an average (mean±1SD) age of 5.9±2.4 years (range 1.6-10.2) for girls (n=296) and 5.4±2.4 years (range 1.3-10.1) for boys (n=310). Using natural history SCA-specific reference curves, the mean weight-for-age Z-score improved from 0.47±0.90 at enrollment to 0.69±1.00 on hydroxyurea treatment. Height increased from 0.26±0.90 to 0.42±1.00 on treatment, and BMI from 0.46±1.00 to 0.85±1.20. IGF-I remained low in many participants: mean IGF-I was -1.5 SD at baseline and -1.4 SD at follow-up in girls, and -2.3 at baseline and -2.2 at follow-up in boys. Pubertal onset was delayed in 25-30% of children, with gradual progress on treatment. AMH was low (<2.5th percentile) in 4% of girls, while 52% of boys had low AMH at baseline and 28% at follow-up. Long-term hydroxyurea treatment at MTD is associated with beneficial effects on growth with improved weight and height, and does not negatively impact pubertal development in children with SCA in sub-Saharan Africa.
AU - Backeljauw,P
AU - Tomlinson,G
AU - Smart,LR
AU - Tshilolo,LMM
AU - Williams,TN
AU - Santos,B
AU - Olupot-Olupot,P
AU - Stuber,SE
AU - Lane,A
AU - Latham,TS
AU - Ware,RE
DO - 10.1182/bloodadvances.2025019511
PY - 2026///
TI - Growth and puberty in African children with sickle cell anemia treated with hydroxyurea.
T2 - Blood Adv
UR - http://dx.doi.org/10.1182/bloodadvances.2025019511
UR - https://www.ncbi.nlm.nih.gov/pubmed/41954645
ER -
