Impact of growth hormone preparation switching on treatment outcomes in children with growth hormone deficiency: a retrospective real-world cohort study.

BACKGROUND

Switching between recombinant human growth hormone (rhGH) preparations has become increasingly common in pediatric practice because of reimbursement policies, product availability, and patient preferences. However, concerns persist regarding potential effects on growth outcomes and biochemical responses in real-world settings. This study aimed to evaluate whether switching growth hormone (GH) preparations is associated with differences in growth and biochemical outcomes compared with continuing the same product in children with growth hormone deficiency (GHD).

METHODS

This single-center retrospective cohort study included 130 children with GHD receiving GH therapy between 2019 and 2024: 66 who underwent a single switch between preparations and 64 who continued the same product. Annualized changes in height standard deviation score (SDS), height velocity, insulin-like growth factor-1 (IGF-1) SDS, and insulin-like growth factor binding protein-3 (IGFBP-3) SDS were assessed over predefined 12-month observation windows. Patients were additionally stratified according to GHD severity, defined by a peak GH response < 7 ng/mL (severe/complete GHD) or 7-10 ng/mL (partial/borderline GHD). Within-group comparisons were performed using Wilcoxon signed-rank or Friedman tests, and between-group comparisons were performed using Mann-Whitney U tests or chi-square tests, as appropriate. Multivariate linear regression analyses adjusted for age, pubertal status, GH dose, baseline height SDS, and GHD severity.

RESULTS

Baseline demographic, auxological, and perinatal characteristics were similar between the groups. In both cohorts, height SDS, IGF-1 SDS, and IGFBP-3 SDS improved significantly during follow-up, whereas GH dose remained stable. When outcomes were compared during equivalent treatment intervals, no significant differences were observed between the switch and nonswitch groups in height velocity (8.72 ± 2.64 vs. 8.42 ± 1.62 cm/year; p = 0.108), ΔHeight SDS (0.53 ± 0.51 vs. 0.39 ± 0.37; p = 0.206), ΔIGF-1 SDS (0.82 ± 0.69 vs. 0.71 ± 0.54; p = 0.486), or ΔIGFBP-3 SDS (0.86 ± 0.90 vs. 0.64 ± 0.45; p = 0.283). Similar findings were observed in patients with severe GHD (peak GH < 7 ng/mL). Multivariate regression analysis identified baseline height SDS and GH dose as independent predictors of 12-month height SDS change, whereas switch status and GHD severity were not independently associated with growth response.

CONCLUSIONS

Switching between rhGH preparations was not associated with impaired growth or adverse biochemical outcomes in children with GHD. Growth and biochemical responses were comparable between switched and nonswitched patients during equivalent treatment intervals, including among patients with severe GHD. These findings support the clinical feasibility of switching between daily rhGH preparations when required because of reimbursement policies, product availability, or other practical clinical considerations.

TRIAL REGISTRATION: Not applicable.