Effectiveness of Recombinant Human Growth Hormone Therapy in Small‐for‐Gestational‐Age Children With Short Stature: A Stratified Analysis Based on Genetic Variant Status
Authors' conclusion
As one of the first longitudinal analyses utilizing repeated-measures modelling in a genetically characterized patient population, this study demonstrates that while rhGH therapy effectively promoted linear growth in SGA-SS children, patients with a molecular diagnosis presented with lower baseline stature and an attenuated incremental growth response over time
Study context
- Population
- Children born SGA with persistent short stature (SGA-SS) with clinical and genetic evaluation, treated with recombinant human growth hormone (rhGH) for 3 years142 evaluated
- Participants
- 179 children
- Exposure
- Variant-positive vs variant-negative SGA-SS children on rhGH
- Variant-positiven = 30
- Variant-negativen = 149reference
- Outcome
- Growth outcomes (height SDS) on rhGH therapy
- Follow-up
- 3 years
Methodological detailsstratified by genetic variant status, linear mixed model
- Stratified by genetic variant status
- Linear mixed model
Main finding
As reported in the abstract
Interaction estimate (incremental height standard deviation score (SDS) gain), Year 1
−0.18
all p < 0.05
Variant-positive
Interaction estimate (incremental height SDS gain), Year 2
−0.31
all p < 0.05
Variant-positive
Interaction estimate (incremental height SDS gain), Year 3
−0.36
all p < 0.05
Variant-positive
Variant-positive
−1.30
estimated marginal mean · ± 0.10
Variant-negative
−0.93
estimated marginal mean · ± 0.04
Observational data: associations, not effects.
Further findings
Intellectual disability at baseline
P < 0.001
Congenital anomalies at baseline
P < 0.001
| Outcome | Variant-positive | Variant-negative | P value |
|---|---|---|---|
| Baseline height SDS | −2.83 | −2.28 | P < 0.001 |
Original publication
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