Understanding Growth Impairment in Pediatric Sickle Cell Disease and the Importance of Hydroxyurea Adherence

Hydroxyurea adherence strongly influences growth trajectories in children with sickle cell disease, materially changing short‑stature risk and warranting prioritized pediatric follow‑up.

Growth impairment is common in pediatric sickle cell cohorts; risk concentrates among those with more severe disease—frequent vaso‑occlusive crises, more severe anemia, and certain genotypes—summarized in a recent study.

In a cross‑sectional cohort of 225 children aged 6 months to 12 years, short stature (height <3rd percentile) affected roughly one‑third of participants; frequent VOCs (>3/year) occurred in about 32%. The study captured hospitalizations, transfusion burden, acute chest events, and hydroxyurea use, with anthropometry referenced to WHO height‑for‑age standards—data that can be integrated into routine risk stratification to flag children for intensified monitoring.

Regular hydroxyurea use correlated with better growth outcomes: 69.3% of the cohort reported regular use, while irregular or absent therapy carried an adjusted odds ratio of approximately 2.7 for short stature versus regular therapy. Lower BMI‑for‑age Z‑score had an adjusted OR of about 2.1. Children on uninterrupted hydroxyurea regimens experienced fewer growth deficits, indicating adherence materially influences long‑term growth trajectories and follow‑up priorities in pediatric practice.

Expected management changes include earlier diagnosis, routine initiation or optimization of hydroxyurea for eligible children, standardized growth monitoring using height‑for‑age Z‑scores, and active management of chronic anemia and vaso‑occlusive events to support growth. Recommended monitoring cadence based on the study and common clinical standards includes baseline anthropometry at diagnosis and at least semiannual height measurements plotted against WHO or local growth standards, with more frequent checks when centile crossing or growth faltering is observed. Triggers for endocrinology referral include persistent height below the 3rd percentile, a fall of more than 1 Z‑score over a 12‑month interval, or unexplained deceleration despite optimized hematologic management—systematic monitoring and timely specialty referral can help prevent progressive growth failure.