Recombinant growth hormone improves growth and adult height in patients with maternal inactivating GNAS mutations
Main finding · Adult height SDSResult — go to the resultDifference 1.9 SDS CI +1.1 to +2.7Authors' conclusion
Recombinant human growth hormone treatment of iPPSD2/PHP1A patients with short stature improves growth and adult height. More studies are needed to confirm long-term efficacy and safety.
Study context
- Population
- Patients with maternal inactivating GNAS mutations (iPPSD2/PHP1A)
- Participants
- 190 patients
- Exposure
- Recombinant human GH (rhGH) vs untreated controls
- rhGHn = 26
- Outcome
- Adult height (effect of rhGH, linear mixed models)
Methodological detailsmulticenter
- Multicenter
Main finding
Adult height SDS
Difference
1.9 SDS
CI +1.1 to +2.7
P < 0.001
rhGHvs Untreated controls
difference 1.9 SDS (CI +1.1 to +2.7); P < 0.001
As reported in the abstract
Observational data: associations, not effects.
Further findings
Gain in height standard deviation scores (SDS) with rhGH · 1 year
- Height gain, CI +0.5 to +0.8, P < .001
- 0.7 SDS
Gain in height SDS with rhGH · 3 years
- Height gain, CI +1.0 to +2.0, P < .001
- 1.5 SDS
BMI SDS
- BMI SDS did not vary significantly upon rhGH therapy
Original publication
Recombinant growth hormone improves growth and adult height in patients with maternal inactivating GNAS mutations. Eur J Endocrinol. Published 13 July 2023. doi:10.
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