Near Adult Height and Body Mass Index Changes in Growth Hormone Treated Short Children with Noonan Syndrome: The Belgian Experience
Authors' conclusion
Long-term GH therapy at a dose of 0.05 mg/kg/day in short NS patients is effective in improving adult height and BMI, irrespective of the genotype and presence or absence of cardiac and or thoracic anomalies.
Study context
- Population
- Growth hormone (GH)-treated short children with Noonan syndrome born before 2001, genotyped with a 9-gene panel, Belgian Belgrow registry35 male
- Participants
- 54 children
- Exposure
- PTPN11 vs SOS1 vs No-mutation
- PTPN11n = 32
- SOS1n = 5
- No-mutationn = 17reference
- Outcome
- NAH and BMI evolution
Methodological detailsnational registry (Belgrow), single-arm
- National registry (Belgrow)
- Single-arm
Main finding
As reported in the abstract
median NAH
- Median near adult height (NAH) SDS (P10; P90 −3.1; −0.8)
- −1.7
- Median total height gain SDS (P10; P90 0.1; 2.3)
- 1.1
- Median body mass index (BMI) SDS at start (P10; P90 −2.5; 0.0)
- −1.0
- Median BMI SDS at NAH (P10; P90 −1.5; 0.9); significant increase
- −0.2
- P for BMI SDS increase
- p < 0.001
Observational data: associations, not effects.
Secondary outcomes
Sex, genotype, thoracic or cardiac malformation vs NAH and total height gain
- Sex, genotype and thoracic or cardiac malformation did not correlate with NAH or total height gain
Independent predictors of NAH SDS (linear regression)
- Height SDS at start, β (p < 0.001)
- 0.90
- Mid-parental height SDS, β (p = 0.005)
- 0.27
- Birth weight SDS, β (p = 0.051)
- 0.15
- Age at start, β (p = 0.032)
- 0.07
Observed vs Ranke-predicted NAH (subgroup >3 years GH)
- Observed NAH not statistically different from predicted NAH by Ranke Noonan model
- Subgroup of 44 patients
Original publication
Near Adult Height and Body Mass Index Changes in Growth Hormone Treated Short Children with Noonan Syndrome: The Belgian Experience. Horm Res Paediatr. Published 1 March 2024. doi:10.
Composed by AI from the published abstract and checked against it. How Briefs are made →