Gene Expression Signatures Predict First-Year Response to Somapacitan Treatment in Children With Growth Hormone Deficiency
Authors' conclusion
Pretreatment transcriptome predicts first-year growth response in somapacitan-treated children with GHD. A common set of genes can predict the treatment response to both once-weekly somapacitan and conventional daily GH. This approach could potentially be developed into a clinically applicable pretreatment test to improve clinical management.
Study context
- Population
- Treatment-naïve prepubertal children with GH deficiency (GHD) who consented to baseline blood transcriptome profiling121 passed QC
- Participants
- 128 children
- Comparison
- Pretreatment blood transcriptome prediction of growth response to somapacitan vs daily growth hormone (GH)
- Somapacitann = 76
- Daily GHn = 45reference
- Primary endpoint
- Prediction of HV (cm/year) by pretreatment transcriptome: fastest vs slowest growing quartiles
- Follow-up
- Week 52
Methodological detailsmultinational, open-label, active-controlled, parallel group, phase 3, Differential RNA-seq analysis and machine learning used to predict response
- Multinational
- Open-label
- Active-controlled
- Parallel group
- Phase 3
- Differential RNA-seq analysis and machine learning used to predict response
Primary result
As reported in the abstract
OOB AUC
0.98-0.99
Validation AUC
0.83-0.84
Further findings
Prediction of height velocity (HV) standard deviation score (SDS), both treatments · week 52
- OOB AUC
- 0.99-1.0
- Validation AUC
- 0.75-0.78
Prediction of ΔHSDS, both treatments · week 52
- OOB AUC
- 0.98-1.0
- Validation AUC
- 0.61-0.75
Prediction of ΔIGF-I SDS, both treatments · week 52
- OOB AUC
- 0.96-1.0
- Validation AUC
- 0.85-0.88
Previously identified GH-response genes, both treatments
- OOB AUC, fastest growers
- 0.93-0.97
- OOB AUC, slowest growers
- 0.67-0.85
Original publication
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