Context: Silver-Russell syndrome (SRS) is an imprinting disorder characterized by severe intrauterine and postnatal growth retardation, feeding difficulties, and risk of hypoglycaemia. Recombinant growth hormone (rGH) therapy has shown its positive effect on adult height in SRS.
Objective: We aimed to assess the short-term effects of rGH therapy on the growth and nutritional status of these children.
Design, children and settings: Retrospective monocentric data of growth and nutritional characteristics of 77 prepubertal children with molecularly proven SRS during the first two years of rGH therapy were analysed.
Results: The mean age at the initiation of rGH therapy was 3.7 (1.4-10.3) years. The mean height gain was 0.8 standard deviation score (SDS) after one year of treatment and 1.3 SDS after two years. The ideal weight for height (WfH), reflecting the nutritional status, increased from a mean of 81% at rGH initiation, to 84% after one year of treatment (p < 0.001) and 86% after two years (p < 0.001). The proportion of SRS children below a WfH of 75% decreased from 22.1% at rGH therapy initiation to 7.8% after two years of treatment. Starting rGH therapy before the age of four years was associated with a greater increase in height after two years, 1.5 vs 1.1 SDS, p = 0.012.
Conclusions: In prepubertal SRS children, the first two years of standard-dose rGH therapy significantly enhance both height and nutritional status. Early initiation of treatment, before the age of four years, further optimizes height gain.
Keywords: Silver-Russell syndrome; growth; growth hormone; imprinting disorders; nutrition; prepubertal children.
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