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This Article Full Text Full Text (PDF) All Versions of this Article: EJE-08-0413v1 159/3/203    most recent Alert me when this article is cited Alert me if a correction is posted Services Similar articles in this journal Similar articles in PubMed Alert me to new issues of the journal Download to citation manager Google Scholar Articles by Noordam, C Articles by Otten, B J PubMed PubMed Citation Articles by Noordam, C Articles by Otten, B J

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

Department of Metabolic and Endocrine Diseases 833Radboud University Medical Centre, PO Box 9101, 6500 HB Nijmegen, The Netherlands¹ Department of Epidemiology and BiostatisticsRadboud University Medical Centre, Nijmegen, The Netherlands² Department of Paediatric EndocrinologyUniversity of Leuven, Leuven, Belgium³ Department of Paediatric EndocrinologyFree University of Brussels, Brussels, Belgium⁴ Department of Human GeneticsRadboud University Medical Centre, Nijmegen, The Netherlands

(Correspondence should be addressed to C Noordam; Email: c.noordam{at}cukz.umcn.nl)

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.