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This Article Accepted manuscript (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, K. Articles by Otten, B. PubMed PubMed Citation Articles by Noordam, K. Articles by Otten, B.

Long term growth hormone treatment improves adult height in children with Noonan syndrome with and without mutations in PTPN11

K Noordam, Pediatrics, Radboud University Nijmegen Medical Centre, nijmegen, 6500 HB, Netherlands
N Peer, epidemiology and biostatistics, Radboud University Nijmegen, nijmegen, Netherlands
I Francois, paediatric endocrinology, university of leuven, leuven, Belgium
J De Schepper, Dept. of Pediatric Endocrinology, Academic Hospital VUB, brussels, Belgium
I Burgt, human genetics, radboud university nijmegen medical centre, nijmegen, Netherlands
B Otten, Paediatric Endocrinology, Radboud University Nijmegen Medical Centre, Nijmegen, Netherlands

Correspondence: Kees Noordam, Email: c.noordam{at}cukz.umcn.nl

Abstract

Objective: Noonan syndrome (NS) is characterized by short stature, typical facial dysmorphology and congenital heart defects. Short-term effect of growth hormone therapy in NS is beneficial, reports on the effect on adult height are scarce. This study was started to determine the effect of long-term growth hormone (GH) therapy in children with NS.

Design: 29 children with NS were treated with GH until final height was reached.

Methods: Children with the clinical diagnosis of Noonan syndrome, mean age at start of therapy was 11.0 years, 22 out of 27 tested children had a mutation in the PTPN11 gene. GH was administered subcutaneously at 0.05 mg/kg/day until growth velocity was 1 cm/6 months. 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 standard deviation scores (H-SDS) were - 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 5 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.