New guidance on long-acting growth hormone emphasizes safety, efficacy, and patient care

Long-acting growth hormone (LAGH) formulations are transforming the management of pediatric growth hormone (GH) deficiency, offering new treatment options with reduced injection frequency. Recent findings and expert consensus recommendations highlight the efficacy, safety, and practical considerations for LAGH use in children.

Proven Efficacy and Comparable Safety
Three approved LAGH formulations—lonapegsomatropin, somapacitan, and somatrogon—have demonstrated noninferiority to daily GH in terms of annualized height velocity. These formulations show comparable safety profiles to daily GH, with no new safety signals identified to date.

Unique Dosing Considerations
Due to differences in the pharmacokinetics, pharmacodynamics, and molecular weights of the LAGH formulations, weight-based dosing requires unique per-milligram calculations for each molecule. Direct milligram-to-milligram dose comparisons between LAGH formulations or with daily GH are inappropriate, necessitating tailored approaches to treatment.

Key Insights on IGF-1 Monitoring
Insulin-like growth factor 1 (IGF-1) monitoring requires careful timing relative to the last LAGH injection. Sampling at specified time points—day 4 for somapacitan and somatrogon and day 4.5 for lonapegsomatropin—provides an average IGF-1 value that does not require adjustment. Sampling outside this window may necessitate correction using product-specific factors.

Expanding Applications and Future Research
Although LAGH shows promise in other growth disorders beyond GH deficiency, additional trials are needed to establish efficacy, safety, and optimal dosing in these populations.

Consensus Recommendations and Future Directions
Experts emphasize the importance of patient selection, dose adjustments, and practical considerations when prescribing LAGH. Long-term real-world data, including insights from patient registries, are critical to addressing current knowledge gaps.

LAGH can enhance quality of life for children and caregivers by reducing treatment burden and improving adherence. Continued research and real-world evidence will shape comprehensive guidelines, the expert panel concluded.

Reference
Maniatis A, Cutfield W, Dattani M, et al. Long-Acting Growth Hormone Therapy in Pediatric Growth Hormone Deficiency: A Consensus Statement. J Clin Endocrinol Metab. 2024;dgae834. doi: 10.1210/clinem/dgae834. Epub ahead of print. PMID: 39672599.