title: Growth hormone therapy in noonan syndrome.
abstract: Noonan syndrome (NS) is an autosomal dominant disorder that can be difficult to diagnose. Growth retardation is a consistent feature, however, and although children are not typically growth hormone (GH) deficient, a minority may have suboptimal GH levels. An ongoing multicentre study examining the safety and efficacy of GH therapy in NS showed increases in height standard deviation scores (SDS; p < 0. 0001) and height velocity (p < 0.0001) after 12 months of GH at pharmacological doses. There was no increase in mean maximal cardiac left ventricular wall thickness during the 12-month treatment period. Long-term follow-up data covering 3 years of GH therapy showed sustained increases in height SDS and height velocity compared with baseline (p < 0.02). Further large and appropriately controlled studies of high-quality design are essential to further our understanding of NS and to establish the long-term safety and efficacy of GH.
authors.affiliation: Department of Child Life and Health, University of Edinburgh, UK. dkcantavark@dial.pipex.com
authors.forename: C J
authors.initials: CJ
authors.lastname: Kelnar
mesh_terms: D000293:Adolescent; D000328:Adult; D001827:Body Height; D002648:Child; D002675:Child, Preschool; D006130:Growth Disorders / Q000188:drug therapy* / Q000209:etiology; D019382:Human Growth Hormone / Q000009:adverse effects / Q000627:therapeutic use*; D006801:Humans; D009634:Noonan Syndrome / Q000150:complications*; D011627:Puberty
chemical_list: D019382:Human Growth Hormone