GH treatment safe, effective in Silver-Russell syndrome
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Adolescents with Silver-Russell syndrome born small for gestational age and treated with growth hormone have a similar metabolic health and safety profile as adolescents born without the syndrome but small for gestational age, study findings indicate.
Carolina C. J. Smeets, MD, of the department of pediatrics, subdivision of endocrinology at Erasmus University Medical Centre, the Netherlands, and colleagues evaluated data from a large multicenter GH trial on 29 children with Silver-Russell syndrome and 171 children without Silver-Russell syndrome to assess metabolic health during and until 2 years after discontinuation of GH treatment.
Participants were all born small for gestational age and/or had a birth weight of –2 or less standard deviation score (SDS) for gestational age and received GH treatment until adult height achievement.
All participants had lean body mass, fat mass percentage, insulin sensitivity and beta-cell function evaluated.
Compared with participants without Silver-Russell syndrome, participants with Silver-Russell syndrome were younger at GH treatment start (5.4 years vs. 6.7 years; P = .003), had a lower height SDS (P < .001) and weight for height SDS (P < .001). Achievement of adult height was attained at a younger age by participants with the syndrome (15.7 years) compared with those without the syndrome (16.4 years; P = .01).
Lean body mass SDS was lower in participants with the syndrome compared with those without the syndrome at GH treatment start (P = .12); however, it was similar in both groups during treatment. Fat mass percentage was similar between the groups at the start of treatment and 2 years after treatment ended.
Insulin sensitivity and beta-cell function increased significantly in both groups in the 6 months after treatment stopped and it remained stable in the 18 months after.
Metabolic syndrome, type 2 diabetes and adverse events did not develop in any participant with Silver-Russell syndrome.
“We showed that there are no metabolic differences between [Silver-Russell syndrome] and non-[Silver-Russell syndrome] subjects born [small for gestational age], before, during and after GH treatment,” the researchers wrote. “However, a longer follow-up of [small for gestational age] born adults, and [Silver-Russell syndrome] patients specifically, is needed to see whether this will be maintained over the years when patients reach their 30s and 40s.” – by Amber Cox
Disclosure: Smeets reports no relevant financial disclosure. One researcher reports receiving an independent research grant from Novo Nordisk B.V., the Netherlands.