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

Ling Hou; Ke Huang; Chunxiu Gong; Feihong Luo; Haiyan Wei; Liyang Liang; Hongwei Du; Jianping Zhang; Yan Zhong; Ruimin Chen; Xinran Chen; Jiayan Pan; Xianjiang Jin; Ting Zeng; Wei Liao; Deyun Liu; Dan Lan; Shunye Zhu; Zhiya Dong; Huamei Ma

Context: The evidence of long-term polyethylene glycol recombinant human GH (PEG-rhGH) in pediatric GH deficiency (GHD) is limited. Objective: This study aimed to examine the effectiveness and safety of long-term PEG-rhGH in children with GHD in the real world, as well as to examine the effects of dose on patient outcomes. Design: A prospective, observational, posttrial study (NCT03290235). Setting, participants and intervention: Children with GHD were enrolled from 81 centers in China in 4 individual clinical trials and received weekly 0.2 mg/kg/wk (high-dose) or 0.1 to <0.2 mg/kg/wk (low-dose) PEG-rhGH for 30 months. Main outcomes measures: Height SD score (Ht SDS) at 12, 24, and 36 months. Results: A total of 1170 children were enrolled in this posttrial study, with 642 patients in the high-dose subgroup and 528 in the low-dose subgroup. The Ht SDS improved significantly after treatment in the total population (P< 0.0001), with a mean change of 0.53±0.30, 0.89± 0.48, 1.35±0.63, 1.63±0.75 at 6 months, 12 months, 24 months, and 36 months, respectively. In addition, the changes in Ht SDS from baseline were significantly improved in the high-dose subgroup compared with the low-dose subgroup at 6, 12, 24, and 36 months after treatment (all P<0.05). A total of 12 (1.03%) patients developed serious adverse events. There was no serious adverse event related to the treatment, and no AEs leading to treatment discontinuation or death occurred. Conclusions: 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.

CLINICAL trials; POLYETHYLENE glycol; SOMATOTROPIN

Journal of Clinical Endocrinology & Metabolism, 2023, Vol 108, Issue 8, p2078

0021-972X

Academic Journal

10.1210/clinem/dgad039