Long-term Pegylated GH for Children With GH Deficiency: A Large, Prospective, Real-world Study
Authors' conclusion
PEG-rhGH showed long-term effectiveness and safety in treating children with GHD. Both dose subgroups showed promising outcomes, whereas PEG-rhGH 0.2 mg/kg/wk might show additional benefit.
Study context
- Population
- Children with GH deficiency (GHD) enrolled from 4 clinical trials at 81 centers in China; Polyethylene glycol recombinant human GH (PEG-rhGH) weekly for 30 months
- Participants
- 1,170 children
- Exposure
- PEG-rhGH high-dose vs low-dose
- High-dosen = 642
- Low-dosen = 528reference
- Outcome
- Height SDS (Ht SDS)
- Follow-up
- 12, 24, and 36 months
Methodological detailsmulticentre, real-world
- Multicentre
- Real-world
Main finding
As reported in the abstract
Change in Ht SDS from baseline, total population · 6, 12, 24 and 36 months
- Mean change in Ht SDS at 6 months
- 0.53 ± 0.30
- Mean change in Ht SDS at 12 months
- 0.89 ± 0.48
- Mean change in Ht SDS at 24 months
- 1.35 ± 0.63
- Mean change in Ht SDS at 36 months
- 1.63 ± 0.75
- Ht SDS improved significantly after treatment
- P < 0.0001
Observational data: associations, not effects.
Further findings
| Outcome | P value |
|---|---|
| Change in Ht SDS from baseline, by dose · 6, 12, 24 and 36 monthsSignificantly greater improvement with high-dose | all P < 0.05 |
Safety
Serious adverse events
- Patients with serious AEs
- 12 (1.03%)
- Treatment-related serious AEs
- 0
- AEs leading to treatment discontinuation or death
- 0
Original publication
Long-term Pegylated GH for Children With GH Deficiency: A Large, Prospective, Real-world Study. J Clin Endocrinol Metab. Published 21 January 2023. doi:10.
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