Long-term Pegylated GH for Children With GH Deficiency: A Large, Prospective, Real-world Study

Area of Science:

• Pediatric Endocrinology
• Pharmacology
• Clinical Trials

Background:

• Limited evidence exists on the long-term efficacy and safety of polyethylene glycol recombinant human growth hormone (PEG-rhGH) for pediatric growth hormone deficiency (GHD).
• Real-world data is crucial for understanding treatment outcomes in diverse patient populations.

Purpose of the Study:

• To evaluate the long-term effectiveness and safety of PEG-rhGH in children with GHD.
• To investigate the impact of different PEG-rhGH dosages on patient outcomes.

Main Methods:

• A prospective, observational, posttrial study involving 1170 children with GHD across 81 centers in China.
• Participants received either a high-dose (0.2 mg/kg/wk) or low-dose (0.1 to <0.2 mg/kg/wk) of PEG-rhGH weekly for 30 months.
• Height standard deviation score (Ht SDS) was measured at 6, 12, 24, and 36 months.

Main Results:

• Significant improvement in Ht SDS was observed in the total population after 30 months of PEG-rhGH treatment (mean change: 1.63 ± 0.75 at 36 months).
• The high-dose subgroup demonstrated significantly greater Ht SDS improvements compared to the low-dose subgroup at all measured time points (P < 0.05).
• Serious adverse events were rare (1.03%), with none attributed to PEG-rhGH, and no treatment discontinuations or deaths occurred due to adverse events.

Conclusions:

• PEG-rhGH is effective and safe for the long-term treatment of pediatric GHD in a real-world setting.
• While both low and high doses yielded positive outcomes, the higher dose (0.2 mg/kg/wk) may offer additional benefits in terms of height gain.