TOPLINE:
A standard-dose recombinant growth hormone therapy administered for 2 years demonstrated positive effects on both growth and nutritional status in prepubertal children with Silver-Russell syndrome, a condition characterized by severe growth retardation and feeding difficulties. Initiating treatment before the age of 4 years optimized height gains in these patients.
METHODOLOGY:
- About 90% of children with Silver-Russell syndrome have severe feeding difficulties, primarily before the age of 6 years, and nearly 70% are malnourished. Data on the effects of recombinant growth hormone, especially during the initial years of therapy, are limited.
- To address this gap, researchers conducted a retrospective single-center analysis of 77 children (including 36 girls) with no sign of puberty and molecularly confirmed Silver-Russell syndrome who initiated growth hormone therapy at a mean age of 3.7 years.
- A clinical file, including extensive clinical, radiological, and biological data; growth charts; and a detailed phenotypic description, was completed for all children; these data were retrospectively collected at the initiation of treatment, 1 year before treatment, and 1-2 years after treatment onset.
- Changes in height standard deviation score (SDS) and ideal weight-for-height (as a proxy for nutritional status) were tracked.
TAKEAWAY:
- Children showed a significant improvement in height, gaining 0.8 SDS after 1 year and 1.3 SDS after 2 years of treatment, with most (71.4%) achieving normal height (above -2 SDS); girls had greater gains in both height and BMI than boys (P < .05 for both).
- After 2 years of treatment, children who started growth hormone therapy before the age of 4 years (n = 46) achieved greater height gains than those who started therapy later (1.5 vs 1.1 SDS; P = .012).
- The ideal weight-for-height increased from a median of 80% at the initiation of therapy to 84% after 1 year and 86% after 2 years (P < .001 for both).
- The proportion of children with weight-for-height below 75% declined after 2 years of therapy, whereas those surpassing the 85% target increased.
IN PRACTICE:
“The nutritional status is crucial for these children, especially during the early years of life, as there is a high risk of hypoglycemia; therefore, nutritional interventions, such as early oral feeding therapy, nutritional enrichment, and cyproheptadine for those who are severely undernourished is essential,” the authors of the study wrote.
“We show here that rGH [recombinant growth hormone] also plays a part in the nutritional intervention,” they added.
SOURCE:
This study was led by Eloïse Giabicani and Raphaëlle Billette de Villemeur of the Hôpital Armand Trousseau in Paris, France. It was published online on August 19, 2025, in The Journal of Clinical Endocrinology & Metabolism.
LIMITATIONS:
This study did not discuss any limitations.
DISCLOSURES:
This study received funding from the Rare Disorders Reference Centre (CRESCENDO). The authors reported having no conflicts of interest.
This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication.