ESPE Abstracts

Introduction: Paediatric growth hormone deficiency (pGHD) is a rare disorder characterised by inadequate secretion of growth hormone (GH). Daily injections of somatropin, a recombinant human GH, is the standard of care. Once-weekly GH treatments have been recently developed, but no direct comparisons have been published. This study examined the clinical efficacy of the available once-weekly treatments vs. daily treatment for pGHD via a systematic literature review (SLR) and indirect treatment comparison (ITC).

Methods: An SLR aimed to identify phase three trials of weekly treatment for pGHD. Results were used to conduct an ITC of available treatments. The base case employed Bucher ITC methods to estimate, at 12 months, annualised height velocity (HV), change in height standard deviation score (SDS), and change in bone maturation (BM). Frequentist random-effects meta-analysis was used in the sensitivity analyses. An established and a prespecified non-inferiority margin of -1.8 cm/year was used in follow-up analyses for annualised HV.

Results: The SLR identified four relevant trials of three once weekly GH interventions: somatrogon, lonapegsomatropin, and somapacitan. In the base case analysis, somatrogon was non-inferior to lonapegsomatropin (mean difference [MD]: -0.57; 95% confidence interval (CI): -1.43, 0.29) and somapacitan (MD: 0.83, 95% CI: -0.03, 1.69) in annualised HV. No statistically significant differences were found for changes in BM or height SDS between somatrogon versus lonapegsomatropin or somapacitan. Results of sensitivity analyses were consistent with the base case.

Conclusion: This research demonstrated that once weekly somatrogon was as efficacious as once weekly lonapegsomatropin and somapacitan for pGHD in the evaluations HV, BM and height SDS. Further, weekly treatment is as efficacious as daily treatment as reported in primary trial

Results: Studies on patient preferences and adherence between weekly and daily treatment would be beneficial to further demonstrate the benefits of weekly treatment for pGHD.