Long-term GH treatment improves adult height in children with Noonan syndrome with and without mutations in protein tyrosine phosphatase, non-receptor-type 11.

Abstract

CONTEXT:

Noonan syndrome (NS) is characterized by short stature, typical facial dysmorphology and congenital heart defects. Short-term effect of GH therapy in NS is beneficial, reports on the effect on adult height are scarce.

OBJECTIVE:

To determine the effect of long-term GH therapy in children with NS.

DESIGN:

Twenty-nine children with NS were treated with GH until final height was reached.

SETTING:

Hospital endocrinology departments.

PATIENTS:

Children with the clinical diagnosis of NS, with mean age at the start of therapy of 11.0 years, 22 out of 27 tested children had a mutation in the protein tyrosine phosphatase, non-receptor-type 11 gene (PTPN11 gene). Interventions GH was administered subcutaneously at 0.05 mg/kg per day until growth velocity was 1 cm/6 months.

MAIN OUTCOME MEASURE:

Linear growth (height) was measured at 3-month intervals in the first year and at 6-month intervals thereafter until final height.

RESULTS:

At the start of treatment, median height SDS (H-SDS) was -2.8 (-4.1 to -1.8) and 0.0 (-1.4 to +1.2), based on national and Noonan standards respectively. GH therapy lasted for 3.0-10.3 years (median, 6.4), producing mean gains in H-SDS of +1.3 (+0.2 to +2.7) and +1.3 (-0.6 to +2.4), based on national and Noonan standards respectively. In 22 children with a mutation in PTPN11 mean gain in H-SDS for National standards was +1.3, not different from the mean gain in the five children without a mutation in PTPN11+1.3 (P=0.98).

CONCLUSION:

Long-term GH treatment in NS leads to attainment of adult height within the normal range in most patients.

• Authors: C Noordam, P G M Peer, I Francois, J De Schepper, I van den Burgt, B J Otten
• Journal: European journal of endocrinology (Eur J Endocrinol) Vol. 159 Issue 3 Pg. 203-8 (Sep 2008) ISSN: 1479-683X [Electronic] England
• PMID: 18562489 (Publication Type: Controlled Clinical Trial, Journal Article, Multicenter Study)

Chemical References

• Human Growth Hormone
• PTPN11 protein, human
• Protein Tyrosine Phosphatase, Non-Receptor Type 11

Topics

• Adolescent
• Age Determination by Skeleton
• Body Height (drug effects)
• Child
• Child, Preschool
• Female
• Follow-Up Studies
• Heart Defects, Congenital (drug therapy, genetics, physiopathology)
• Human Growth Hormone (pharmacology, therapeutic use)
• Humans
• Male
• Mutation (physiology)
• Noonan Syndrome (drug therapy, genetics)
• Protein Tyrosine Phosphatase, Non-Receptor Type 11 (genetics)
• Time Factors