Long-Term Growth Hormone Therapy Improves Adult Height in Children with Indian Hedgehog-Related Short Stature
Authors' conclusion
rhGH therapy was associated with clinically meaningful height gains, including adult height improvement, in children with IHH-related short stature; variant class may modify response.
Study context
- Population
- Children with short stature carrying heterozygous deleterious IHH variants10 attained adult height
- Participants
- 19 children
- Outcome
- Height SDS, height velocity and Δheight SDS after 1 year of rhGH
- Follow-up
- 1 year
Main finding
As reported in the abstract
- Height SD score (SDS) at treatment start, median (IQR)
- −2.6 (−3.0; −2.2)
- Height velocity at treatment start, median (IQR)
- 5.1 cm/year (4.3; 6.2)
- Height velocity after 1 year (IQR)
- 9.2 cm/year (7.9; 10.3)
- Height SDS after 1 year (IQR)
- −1.9 (−2.3; −1.6)
- Δheight SDS after 1 year (IQR)
- 0.7 (0.5; 1.0)
Observational data: associations, not effects.
Further findings
Total Δheight SDS and height SDS ≥ −2.0 at last follow-up · median 4.0 years of therapy (IQR: 3.4; 5.7)
- Total Δheight SDS (IQR)
- 1.2 (0.8; 1.5)
- Height SDS ≥ −2.0 at last follow-up
- 16/19 (84%)
Height SDS and Δheight SDS by variant class at last follow-up
- Height SDS: predicted loss-of-function (n = 6) vs missense (n = 13), higher with loss-of-function
- −0.8 [IQR: −0.9; −0.7] vs. −1.7 [IQR: −1.9; −1.6]
- Δheight SDS: predicted loss-of-function vs missense, greater with loss-of-function
- 1.4 [IQR: 1.2; 1.9] vs. 0.8 [IQR: 0.7; 1.3]
Adult height SDS among those reaching adult height · median 5.8 years of therapy (IQR: 4.0; 7.3)
- Adult height SDS (IQR)
- −1.7 (−1.9; −1.6)
- Δheight SDS (IQR)
- 0.8 (0.7; 1.4)
Original publication
Long-Term Growth Hormone Therapy Improves Adult Height in Children with Indian Hedgehog-Related Short Stature. Horm Res Paediatr. Published 16 February 2026. doi:10.
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