Growth hormone therapy in a patient with short stature due to fetal alcohol syndrome: seven-year follow-up.

Fetal alcohol syndrome (FAS) is associated with persistent growth retardation, but the long-term efficacy of GH therapy for FAS-related short stature remains unclear. A Japanese girl born at 37 wk with birth weight 2,064 g (-2.1 SD) and birth height 41.5 cm (-2.7 SD) was diagnosed with FAS based on characteristic facial features, growth failure, developmental delay, and documented maternal alcohol consumption (120 g/d during pregnancy). She scored 4434 on the FASD 4-Digit Diagnostic Code. At 3 yr of age, her height was 78 cm (-4.18 SD) with poor growth velocity. GH therapy was initiated at 0.19 mg/kg/wk, increased to 0.23 mg/kg/wk, and gradually increased to 0.43 mg/kg/wk. After 7 yr of treatment, serum IGF-1 levels increased significantly from 59 ng/mL (-2.5 SD) at baseline to 306 ng/mL (+2.0 SD) at 8 yr, but height SD scores remained around -4 SD with growth velocity fluctuating between -3 and 0 SD. No adverse effects were observed. While GH therapy appears safe in FAS patients, its efficacy for improving linear growth is limited, suggesting that growth impairment in FAS involves mechanisms beyond GH deficiency, including growth plate dysfunction and peripheral GH resistance.