Review Article
Efficacy and safety of growth hormone therapy in children with Noonan syndrome

https://doi.org/10.1016/j.ghir.2023.101532Get rights and content
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Highlights

  • Noonan syndrome is a multisystem autosomal dominantly inherited disorder caused by dysregulation of the RAS-MAPK signal pathway.

  • Patients typically have a target height <2 standard deviations compared to the general population, and half of affected adults remain permanently below the 3rd centile for height, though their short stature might display a multifactorial etiology.

  • The present review discusses on available data dealing with growth hormone efficacy and safety in children and adolescents with Noonan syndrome.

Abstract

Patients with Noonan syndrome typically have a target height <2 standard deviations compared to the general population, and half of the affected adults remain permanently below the 3rd centile for height, though their short stature might result from a multifactorial etiology, not-yet fully understood. The secretion of growth hormone (GH) following the classic GH stimulation tests is often normal, with baseline insulin-like growth factor-1 (IGF-1) levels at the lower normal limits, but patients with Noonan syndrome have also a possible moderate response to GH therapy, leading to a final increased height and substantial improvement in growth rate. Aim of this review was to evaluate both safety and efficacy of GH therapy in children and adolescents with Noonan syndrome, also evaluating as a secondary aim the possible correlations between the underlying genetic mutations and GH responses.

Keywords

Noonan syndrome
Growth hormone
Growth hormone deficiency
Children
Personalized medicine

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