Reduced Effectiveness and Comparable Safety in Biweekly vs. Weekly PEGylated Recombinant Human Growth Hormone for Children With Growth Hormone Deficiency: A Phase IV Non-Inferiority Threshold Targeted Trial.

ABSTRACT:

Context

Long-acting recombinant human growth hormone (rhGH) has transformed growth hormone deficiency (GHD) treatment. However, the possibility and rationality for flexible time regimen are pending.

Objective

We studied the efficacy of biweekly versus weekly PEGylated rhGH (PEG-rhGH) therapy in GHD children.

Design setting and patients

This multicenter, phase IV trial with a non-inferiority threshold ≥20% enrolled 585 Tanner stage I GHD children.

Intervention

Subjects randomly received 0.20 mg/kg once-weekly or biweekly PEG-rhGH, or 0.25 mg/kg.w rhGH once daily for 26 weeks.

Main outcome measure

The primary outcome was height SD scores for chronological age (HtSDS_CA) at week 26 and safety measurements including adverse events (AEs), IGF

SUBMITTER: Sun C 

PROVIDER: S-EPMC8655095 | biostudies-literature | 2021

REPOSITORIES: biostudies-literature