M. van der Steen

Metabolic Health and Long-Term Safety of Growth Hormone Treatment in Silver-Russell Syndrome

Context Children with Silver-Russell syndrome (SRS) are born small for gestational age (SGA) and remain short. Growth hormone (GH) treatment improves height in short SGA children, including those with SRS. Data on metabolic health and long-term safety of GH treatment in SRS are lacking. Objective To investigate metabolic health in SRS patients during and until 2 years after discontinuation of GH treatment. Design Metabolic health was assessed longitudinally at GH-start, GH-stop, 6 months, and 2 years thereafter. Patients Twenty-nine SRS patients vs 171 non-SRS subjects born SGA. Main Outcome Measures Lean body mass (LBM), fat mass percentage (FM%), insulin sensitivity (Si), β-cell function, blood pressure, and serum lipids. Results At GH-start [mean age (standard deviation) 5.4 (2.1) years in SRS and 6.7 (2.0) years in non-SRS (P = 0.003)], blood pressure, serum lipids, glucose, and insulin levels were similar and within normal ranges in SRS and non-SRS. LBM standard deviation score (SDS) and FM% SDS were lower than average in both groups. During treatment, LBM SDS remained stable whereas FM% SDS increased in both groups. During the 2 years after GH-stop, LBM decreased and FM% increased, whereas Si and β-cell function improved. At 2 years after GH-stop (mean age 18 years), all parameters were similar and within normal ranges in SRS and non-SRS. None of the SRS patients developed metabolic syndrome, diabetes mellitus type 2, or adverse events. Conclusion GH-treated SRS patients have a similar metabolic health and safety profile as non-SRS subjects born SGA, both during and until 2 years after GH-stop.

The Contested Autonomy of Policy Advisory Bodies: The Trade-off Between Autonomy and Control of Policy Advisory Bodies in the Netherlands, the United Kingdom, and Sweden

Autonomy and control are important dynamics in each advisory system. Autonomy is important because it ensures independent and critical advice and allows advisors to ‘speak truth to power’ (Wildavsky 1989; Pielke 2010). At the same time, government control is also an important aspect (Van Twist et al. 2015; Craft and Halligan 2015; Halligan 1995) to assure that advices are on-topic, on-time, in the right format, and relevant for government decision-making,- and policymaking processes. This paper describes the trade-off the Netherlands, Sweden, and the United Kingdom make with regard to the level of ‘autonomy’ and ‘control’ of their policy advisory bodies. We make a distinction between legal, economic, and operational means (Kunneke 1991) through which autonomy is stimulated or control is exercised on a managerial and policy (primary processes) level of policy advisory bodies.