ESPE Abstracts (2016) 86 P-P1-806

Metabolic Health and Safety of GH-Treatment in Silver-Russell Syndrome

Carolina Smeetsa, Judith Renesa & Anita Hokken-Koelegaa,b


aErasmus Medical Centre, Rotterdam, The Netherlands; bDutch Growth Research Foundation, Rotterdam, The Netherlands


Background: Silver-Russell syndrome (SRS) is characterized by small for gestational age (SGA) birth, severe short stature and variable dysmorphic features. Children born SGA are at increased risk to develop adult-onset disease at a relatively young age. Growth hormone (GH)-treatment is a registered growth-promoting therapy for short children born SGA, including SRS. Data on metabolic health and long-term safety of GH-treatment in SRS are limited.

Objective and hypotheses: To compare metabolic health and long-term safety of GH-treatment between GH-treated SRS and non-SRS subjects born SGA.

Method: We measured Fat Mass percentage (FM%) and Lean Body Mass (LBM) by DEXA-scan, blood pressure, serum lipid levels and insulin sensitivity (Si) by frequent sampled intravenous glucose tolerance (FSIGT)-test in 30 SRS (n=15 hypomethylation 11p15, n=6 mUPD7, n=9 clinical SRS) and 151 non-SRS subjects, at start of GH-treatment, at attainment of adult height (AH), and 2 years thereafter.

Results: At start of GH-treatment, mean (SD) age was 5.3 (3.0) years in SRS and 6.9 (2.3) in non-SRS (P=0.01). SRS subjects had a lower weight for height SDS (P<0.001) and there was a trend towards lower FM% (7.0 (4.3) in SRS vs. 10.4 (5.0) in non-SRS (P=0.20)) and LBM SDS (−2.30 (2.9) in SRS vs. −0.83 (2.2) in non-SRS (P=0.13)). At AH, after a mean treatment duration of 10.4 years, FM% and LBM SDS had increased in both groups, but LBM was still lower in SRS (P=0.005). Blood pressure, serum lipid levels and Si were similar in SRS and non-SRS during and after cessation of GH-treatment. In both groups, no adverse events considered to be drug-related were observed.

Conclusion: Besides a lower LBM and FM%, GH-treated SRS subjects have a similar metabolic health profile as non-SRS subjects born SGA, and show no adverse events during long-term GH-treatment.

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