Department of Endocrine and Genetics and Metabolism, Beijing Children's Hospital, Capital Medical University, National Centre for Children's Health, No.56 Nanlishi Road, Xicheng District, 100045, Beijing, China.
Department of Child Health Care, Hunan Children's Hospital, Changsha, 410007, China.
BMC Endocr Disord. 2022 Aug 9;22(1):201. doi: 10.1186/s12902-022-01101-8.
To evaluate the effectiveness of individualized-dose polyethylene glycol recombinant human growth hormone (PEG-rhGH) for short stature.
This real-world study enrolled children with short stature in 19 hospitals throughout China. They were treated with PEG-rhGH for 6 months. The starting dosage ranged from 0.10 to 0.20 mg/kg/week. The primary outcome was the change in height standard deviation score (ΔHt SDS).
Five hundred and ten patients were included and grouped based on dosage as A (0.10-0.14 mg/kg/week), B (0.15-0.16 mg/kg/week), C (0.17-0.19 mg/kg/week), and D (0.20 mg/kg/week). The mean 6-month ΔHt SDS for the total cohort was 0.49 ± 0.27, and the means differed among the four dose groups (P = 0.002). The ΔHt SDS was lower in group A than in groups B (LSM difference [95%CI], -0.09 [-0.17, -0.01]), C (LSM difference [95%CI], -0.10 [-0.18, -0.02]), and D (LSM difference [95%CI], -0.13 [-0.21, -0.05]) after adjusting baseline covariates. There were no significant differences among groups B, C, and D. When the baseline IGF-1 was < -2 SDS or > 0 SDS, the △Ht SDS was not different among the four groups (P = 0.931 and P = 0.400). In children with baseline IGF-1 SDS of -2 ~ 0 SDS, a higher dosage was associated with a better treatment effect (P = 0.003), and the △Ht SDS was lower in older children than in younger ones (P < 0.001).
PEG-rhGH could effectively increase height in prepubertal short children. When the baseline IGF-1 was < -2 SDS, 0.10 mg/kg/week could be a starting dose. In other IGF-1 statuses, 0.15-0.20 mg/kg/week might be preferred.
ClinicalTrials.gov: NCT03249480 , retrospectively registered.
评估个体化剂量聚乙二醇重组人生长激素（PEG-rhGH）治疗矮小症的疗效。
本真实世界研究纳入中国 19 家医院的矮小症患儿，给予 PEG-rhGH 治疗 6 个月。起始剂量范围为 0.10 至 0.20mg/kg/周。主要结局为身高标准差评分变化（ΔHt SDS）。
共纳入 510 例患儿，根据剂量分为 A 组（0.10-0.14mg/kg/周）、B 组（0.15-0.16mg/kg/周）、C 组（0.17-0.19mg/kg/周）和 D 组（0.20mg/kg/周）。全队列 6 个月平均ΔHt SDS 为 0.49±0.27，四组间差异有统计学意义（P=0.002）。与 B、C 和 D 组相比，A 组ΔHt SDS 较低（LSM 差值[95%CI]，-0.09[-0.17，-0.01]）。调整基线协变量后，差异仍有统计学意义。B、C 和 D 组间差异无统计学意义。当 IGF-1 基线<-2 SDS 或>0 SDS 时，四组间ΔHt SDS 无差异（P=0.931 和 P=0.400）。在 IGF-1 基线 SDS 为-2~0 的患儿中，较高剂量与更好的治疗效果相关（P=0.003），且年长患儿的ΔHt SDS 低于年幼患儿（P<0.001）。
PEG-rhGH 可有效增加青春期前矮小儿童的身高。当 IGF-1 基线<-2 SDS 时，起始剂量为 0.10mg/kg/周；在其他 IGF-1 状态下，0.15-0.20mg/kg/周可能更优。
ClinicalTrials.gov：NCT03249480，回顾性注册。
