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Growth Response to Weekly Somapacitan Therapy in Children With GH Deficiency Is Related to GH Thresholds in GH
Philippe F Backeljauw1, Claudia Boettcher2, Michael Højby3
1Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH 45229, USA.
Insights
Children with growth hormone deficiency (GHD) and lower GH peak concentrations showed better treatment responses. This highlights the importance of GH peak levels in assessing GHD severity and predicting treatment outcomes.
Area of Science:
- Pediatric Endocrinology
- Growth Hormone Therapy
- Clinical Trial Analysis
Background:
- Current diagnosis of growth hormone deficiency (GHD) in children lacks standardized severity definitions and growth hormone (GH) cutoff levels.
- This ambiguity complicates the interpretation of GH stimulation tests and influences treatment recommendations for pediatric GHD.
Purpose of the Study:
- To analyze treatment response differences in children with GHD receiving daily GH (somatropin) or once-weekly somapacitan.
- To investigate these differences based on GH peak concentrations measured at the time of diagnosis.
Main Methods:
- Subgroup analysis of 200 pediatric patients (aged ≥2.5 years) from the REAL4 randomized, phase 3 trial.
- Comparison of height velocity (HV), height standard deviation score (SDS), and IGF-I SDS changes from baseline.
- Patients were categorized into three GH peak concentration groups: ≤3 μg/L, >3 to <7 μg/L, and ≥7 to ≤10 μg/L.
Main Results:
- The group with GH peak concentration ≤3 μg/L demonstrated the highest HV at both 52 and 104 weeks.
- Significant changes in height SDS and IGF-I SDS from baseline were observed across treatment groups and GH peak categories.
- The ≤3 μg/L GH peak group consistently showed greater increases in height SDS and IGF-I SDS compared to other groups.
Conclusions:
- Pediatric patients with GHD in the lowest GH peak concentration group (≤3 μg/L) exhibited superior treatment response.
- Greater HV and improved changes in height SDS and IGF-I SDS were associated with lower GH peak levels at diagnosis.
- GH peak concentration is a significant factor in predicting treatment outcomes for children with GHD.
Context:
The lack of a severity definition and standardized GH cutoff level for GH deficiency (GHD) diagnosis in children leads to ambiguity in the interpretation of GH stimulation tests and treatment recommendations.
Objective:
To investigate treatment response differences among children with GHD treated with daily GH (somatropin) (year 1) or once-weekly somapacitan (years 1 and 2) based on GH peak concentrations assessed at diagnosis.
Methods:
This was a subgroup analysis of 200 patients with GHD aged ≥2.5 years participating in the REAL4 randomized, phase 3 trial. Height velocity (HV; cm/year) and changes in height SD score (SDS) and IGF-I SDS from baseline were compared for 3 GH peak groups: ≤ 3, >3 to <7, and ≥7 to ≤10 μg/L.
Results:
The ≤3 μg/L GH peak concentration group had the greatest HV at weeks 52 and 104. Mean change in height SDS ranged from 1.89 to 1.59, 1.17 to 1.06, and 0.92 to 1.07 at week 52 and 2.79 to 2.30, 1.64 to 1.54, and 1.33 to 1.51 at week 104 for the 2 treatment groups across the 3 GH peak concentrations, respectively. Mean change in IGF-I SDS ranged from 3.13 to 3.01, 2.11 to 1.96, and 1.87 to 2.26 at week 52 and from 2.81 to 2.11, 1.85 to 1.62, and 1.28 to 1.71 at week 104 for the 3 GH peak concentrations.
Conclusion:
Patients with GHD in the ≤3 μg/L GH peak concentration group had greater HV and greater changes in height SDS and IGF-I SDS from baseline.
