Response to growth hormone treatment and final height in Noonan syndrome in a large cohort of patients in the KIGS database.
BACKGROUND
Noonan syndrome (NS) is an autosomal dominant inherited disease, characterized by a distinctive facial appearance, congenital heart defects, and short stature. Treatment with growth hormone (GH) is an option to enhance height, but long-term effects are still unclear.


METHODS
A cohort of 402 patients (269 males, 133 females), mean age 9.7 years at start with GH, was studied within the KIGS International growth database with respect to long-term response to GH therapy and final height after GH therapy.


RESULTS
At the start of GH therapy median height was -2.61 SDS (Tanner 1966 standards). Seventy-three patients who were followed longitudinally for 3 years had an increment in height SDS (Ht SDS) over the first 3 successive years of 0.54, 0.13 and 0.13, respectively. Twenty-four patients had reached their final height after 4-12 years of GH treatment. Their Ht SDS increased from a median of -3.28 to a median of -2.41 at final height.


CONCLUSIONS
This group of patients with NS showed an early response to GH treatment, with an attenuation of this effect thereafter. At final height the median increment of final height was 0.61 SDS according to Tanner standards and 0.97 SDS according to Noonan standards. No serious side effects were reported.
Radboud University Nijmegen Medical Centre, Metabolic and Endocrine Disorders, Nijmegen, The Netherlands.
R
R
Raaijmakers
C
C
Noordam
G
G
Karagiannis
J W
JW
Gregory
N T
NT
Hertel
I
I
Sipilä
B J
BJ
Otten
D001827:Body Height / Q000187:drug effects*; D002648:Child; D015331:Cohort Studies; D016208:Databases, Factual; D005260:Female; D005500:Follow-Up Studies; D019382:Human Growth Hormone / Q000627:therapeutic use*; D006801:Humans; D008137:Longitudinal Studies; D008297:Male; D009634:Noonan Syndrome / Q000188:drug therapy*; D011994:Recombinant Proteins / Q000627:therapeutic use*; D016896:Treatment Outcome
D011994:Recombinant Proteins; D019382:Human Growth Hormone