Hormone Therapy

Long-term effectiveness and safety of long-acting growth hormone preparation in children with growth hormone deficiency.

TL;DR

Weekly long-acting growth hormone therapy appears to be an effective and safe alternative to daily GH treatment in children with GH deficiency over a 4-year period, with similar height SDS outcomes at 24 and 48 months despite lower initial height velocity in the first 12 months.

Key Findings

The weekly GH group and daily GH group had significantly different baseline characteristics, with the weekly group being older and having more pubertal boys.

  • Study included 996 children: 773 receiving daily GH and 193 receiving weekly GH
  • Weekly GH group was older than daily GH group (age: 8.46 ± 3.44 vs. 7.46 ± 2.89 years, p<0.001)
  • Weekly GH group had shorter mid-parental height SDS (-0.88 ± 0.73 SD vs. -1.02 ± 0.84 SD, p=0.044)
  • Weekly GH group had more pubertal boys (34.0 vs. 16.9%, p=0.006)

Height velocity and height SDS change during the first 12 months were significantly higher in the daily GH group compared to the weekly GH group.

  • Height velocity at 12 months: 9.06 ± 1.72 cm/year (daily) vs. 8.67 ± 1.98 cm/year (weekly), p=0.028
  • Height SDS change at 12 months: 0.78 ± 0.39 (daily) vs. 0.61 ± 0.41 (weekly), p<0.001
  • The daily GH group showed superior early growth response compared to weekly administration

Height SDS outcomes were similar between the daily and weekly GH groups at 24 and 48 months of treatment.

  • Despite differences at 12 months, height SDS at 24 months was similar between groups
  • Height SDS at 48 months was also similar between daily and weekly GH groups
  • No significant differences in overall height velocity were observed over 48 months
  • These findings suggest the weekly group catches up in height SDS gains after the initial treatment period

No significant differences in annualized treatment continuation rate or safety profile were observed between the weekly and daily GH groups over 48 months.

  • Annualized treatment continuation rates were similar between the two groups over the 4-year follow-up period
  • Safety profiles showed no significant differences between weekly and daily GH administration
  • Clinical and laboratory data were collected at baseline and throughout the study to monitor safety
  • The study was part of the 'LG Growth Study' design which included monitoring of anthropometric, clinical, and laboratory data

Weekly long-acting growth hormone therapy is an effective and safe alternative to daily GH treatment in children with GH deficiency over a 4-year period.

  • Study followed children for up to 48 months with anthropometric data collected at baseline and every 12 months
  • The authors conclude that further research with larger sample sizes and longer follow-up is needed to confirm these findings
  • Assessment of extended safety and effectiveness of long-acting GH (LAGH) was identified as a priority for future research
  • The study included a total of 996 children with GH deficiency across both treatment arms

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Citation

Kang E, Chung L, Rhie Y, Lee K, Nam H. (2024). Long-term effectiveness and safety of long-acting growth hormone preparation in children with growth hormone deficiency.. Journal of pediatric endocrinology &amp; metabolism : JPEM. https://doi.org/10.1515/jpem-2024-0351