Improved final height with long-term growth hormone treatment in Noonan syndrome.
OBJECTIVE
To assess whether children with Noonan syndrome on long-term growth hormone (GH) therapy improve their final height to near mid-parental height.


METHODS
Twenty-five prepubertal children (13 girls) with Noonan syndrome (NS) were studied. A single clinician made the diagnosis based on clinical criteria. GH treatment started at an age ranging from 3.1 to 13.8 y and was continued for at least 2 y. Improvement or "gain" in final height (FH) was defined as either the difference between adult height SD scores (SDS) and pre-treatment height SDS (the childhood component of the Swedish reference) or height SDS compared to the Noonan reference.


RESULTS
Ten children received a GH dose of 33 microg/kg/d (mean age at start 7.7+/-2.1 y, mean age at stop 17.6+/-1.7 y) and 15 received a dose of 66 microg/kg/d (mean age at start 8.6+/-3.3 y, mean age at stop 18.4+/-2.1 y). Eighteen out of 25 patients reached FH. A substantial improvement in FH of 1.7 SDS, equivalent to 10.4 cm compared to pre-treatment height, was observed. No significant difference was seen between the two GH doses. Females gained a mean height of 9.8 cm and males 1-13 cm (FH 174.5+/-7.8 cm vs mean adult height of 162.5+/-5.4 cm for males with NS) at final height. Moreover, 60% reached a mid-parental height of+/-1 SD.


CONCLUSIONS
GH treatment improves final height in patients with Noonan syndrome, with a mean gain of 1.7 SDS. The prepubertal height gain is maintained to final height and the children achieve a height close to their mid-parental height.
Osio
Deborah
D
Göteborg Paediatric Growth Research Centre, Department of Paediatrics, Institute for the Health of Women and Children, The Sahlgrenska Academy at Göteborg University, Göteborg, Sweden.
Dahlgren
Jovanna
J
Wikland
Kerstin Albertsson
KA
Westphal
Otto
O
D000293:Adolescent; D001827:Body Height / Q000187:drug effects*; D002648:Child; D002675:Child, Preschool; D004305:Dose-Response Relationship, Drug; D005260:Female; D013006:Growth Hormone / Q000627:therapeutic use*; D006801:Humans; D007223:Infant; D008297:Male; D009634:Noonan Syndrome / Q000188:drug therapy*; D011627:Puberty / Q000187:drug effects; D013548:Sweden; D016896:Treatment Outcome
D013006:Growth Hormone