A Study of the Genetic Basis of Response to Growth Hormone Treatment in Children With Idiopathic Short Stature

Exclusion Criteria

• •Previous participation in this study. Participation is defined as having given informed consent in this study.
• •Mental incapacity, unwillingness or language barriers precluding adequate understanding, cooperation or informed consent.
• •Receipt of any investigational medicinal product within 3 months before or during the first year of GH therapy that could influence response to GH therapy.
• •Concomitant illness within 3 months before or during the first year of GH therapy that could (positively or negatively) influence the first year of GH therapy. Exception: Attention Deficit Hyperactive Disorder and its treatment can be included but should be recorded.
• •Children with suspected or confirmed growth hormone deficiency according to local practice.
• •Concomitant use of medication including gonadotropin-releasing hormone (GnRH) analogues, aromatase inhibitors, sex steroids, glucocorticoids or any other medication that can influence response to GH therapy. Exception: Attention Deficit Hyperactive Disorder and its treatment can be included but should be recorded.
• •Any known or suspected clinically significant abnormality likely to affect growth or the ability to evaluate growth with standing height measurements, such as but not limited to:
• •* Significant spinal abnormalities including but not limited to scoliosis, kyphosis and spina bifida variants.
• •* Any other disorder that can cause short stature such as, but not limited to, psychiatric disorders, nutritional disorders, chronic systemic illness, chronic respiratory conditions (e.g. asthma), and chronic renal disease.
• •* Turner Syndrome (including mosaicism).
• •* Noonan Syndrome.
• •* Born small for gestational age (defined as birth length below -2 SDS OR birth weight below -2 SDS OR both) (according to national standards).
• •* Extreme prematurity, defined as gestational age less than 32 weeks.
• •* Syndromic short stature defined by the presence of significant dysmorphic features and/OR major malformations, Laron syndrome, Prader-Willi syndrome, Russell-Silver syndrome.
• •* Significant developmental delays, autism spectrum or intellectual deficit of any degree.
• •* Skeletal dysplasia.
• •* Magnetic resonance imaging (MRI) result confirming pituitary structural abnormalities.
• •* Poor adherence to GH therapy or interruption of it for any time during the first year of therapy, as judged by the treating physician.