Pediatric endocrinology

Long-Acting PEGylated Growth Hormone in Children with Idiopathic Short Stature: 2-Year Results

Horm Res PaediatrPublished Open-label extension study

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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