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Long-term effectiveness and safety of childhood growth hormone treatment in Noonan syndrome

Hormone Research in Paediatrics Jan 18, 2021

Rohrer TR, Abuzzahab J, Backeljauw P, et al. - In the present study, the researchers sought to assess the effectiveness and safety of growth hormone (GH) treatment in Noonan syndrome (NS) in clinical practice. Height gain, near-adult height (NAH), and safety have been evaluated in 2 complementary non-interventional studies: NordiNet IOS and ANSWER. The safety analysis involved 412 patients, and the effectiveness analysis involved 84 GH-treated patients (male, n = 67) with ≥ 4 years’ height standard deviation score data. During the first 3 years of follow-up, GH-treated children with NS achieved substantial height gain. Overall, 24 individuals reached NAH, with 70.8% having NAH SDS ≥ –2. No evidence has been given to suggest a higher incidence of neoplasm, cardiac or other comorbidities.

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