Long-Acting PEGylated Growth Hormone in Children with Idiopathic Short Stature: 2-Year Results
Authors' conclusion
Once-weekly PEG-rhGH in children with ISS showed sustained efficacy over 2 years in all assessed height-based outcomes. Treatment remained safe and well tolerated with no new safety signals.
Study context
- Population
- Children with idiopathic short stature (ISS)268 completed 52 weeks
- Participants
- 280 children
- Comparison
- PEGylated recombinant human growth hormone (PEG-rhGH) 0.2/0.2 vs 0.1/0.2 vs 0/0.2 mg/kg/week
- Outcome
- Change in height SDS (ΔHT SDS) from baseline
- Follow-up
- Up to week 104
Methodological detailsmulticenter, uncontrolled, extension phase following 52-week main phase
- Multicenter
- Uncontrolled
- Extension phase following 52-week main phase
Main finding
As reported in the abstract
ΔHT SDS from baseline
0.2/0.2 mg/kg/week
1.52
least squares mean
0.1/0.2 mg/kg/week
1.24
least squares mean
0/0.2 mg/kg/week
1.07
least squares mean
Secondary outcomes
- Height velocity (HV) — 0/0.2 mg/kg/week
- 9.16 (SD 1.33) cm/year
HV
- HV highest in 0/0.2 mg/kg/week group, reflecting typical first-year catch-up growth
IGF-1 standard deviation score (SDS) · 2 years
- Mean IGF-1 SDS remained within 2SDS
Original publication
Long-Acting PEGylated Growth Hormone in Children with Idiopathic Short Stature: 2-Year Results. Horm Res Paediatr. Published 3 February 2026. doi:10. 1159/ 000550510
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