Growth hormone treatment in Noonan syndrome: the National Cooperative Growth Study experience

Area of Science:

• Pediatrics
• Endocrinology
• Genetics

Background:

• Noonan syndrome (NS) is a genetic disorder associated with short stature.
• Growth hormone (GH) deficiency is a common endocrine disorder affecting children's growth.
• Turner syndrome (TS) and idiopathic GH deficiency (IGHD) are conditions with established responses to GH therapy.

Purpose of the Study:

• To evaluate the efficacy of growth hormone (GH) therapy in children with Noonan syndrome (NS).
• To compare the growth response of children with NS to GH therapy with that of children with IGHD and TS.

Main Methods:

• Analysis of growth data from 150 children with NS from the National Cooperative Growth Study.
• Comparison of NS patient data with growth data from children with IGHD and TS within the same study.
• Assessment of annualized growth rates and height standard deviation (SD) scores during GH therapy.

Main Results:

• Children with NS were significantly shorter at baseline compared to those with IGHD and TS.
• GH therapy led to significant increases in growth rates for children with NS, which were intermediate between IGHD and TS groups.
• Significant improvement in height SD scores was observed in children with NS after at least 4 years of GH therapy.
• Three out of six boys with NS reached or exceeded their predicted adult height.

Conclusions:

• Growth hormone (GH) therapy is effective in improving growth in children with Noonan syndrome (NS).
• While GH therapy benefits children with NS, their growth response is distinct from that seen in IGHD and TS.
• GH therapy offers potential for improved adult height outcomes in some individuals with NS.