Early Growth Hormone Treatment Enhances Growth and Nutritional Status in Silver-Russell Syndrome.

Abstract

Silver-Russell syndrome (SRS) is an imprinting disorder characterized by severe intrauterine and postnatal growth retardation, feeding difficulties, and risk of hypoglycemia. Recombinant growth hormone (rGH) therapy has shown its positive effect on adult height in SRS. We aimed to assess the short-term effects of rGH therapy on the growth and nutritional status of these children. Retrospective monocentric data of growth and nutritional characteristics of 77 prepubertal children with molecularly proven SRS during the first 2 years of rGH therapy were analyzed. 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 1 year of treatment and 1.3 SDS after 2 years. The ideal weight for height (WfH), reflecting the nutritional status, increased from a mean of 81% at rGH initiation, to 84% after 1 year of treatment (P < .001) and 86% after 2 years (P < .001). The proportion of SRS children below a WfH of 75% decreased from 22.1% at rGH therapy initiation to 7.8% after 2 years of treatment. Starting rGH therapy before the age of 4 years was associated with a greater increase in height after 2 years, 1.5 vs 1.1 SDS, P = .012. In prepubertal SRS children, the first 2 years of standard-dose rGH therapy significantly enhance both height and nutritional status. Early initiation of treatment, before the age of 4 years, further optimizes height gain.