A Clinical Trial of High-Dose Growth Hormone in a Patient With a Dominant-Negative Growth Hormone Receptor Mutation
Authors' conclusion
A precision medicine approach of extremely high dose GH was able to overcome GH resistance in a patient with a dominant-negative variant in the GH receptor resulting in elevated GH binding protein levels.
Study context
- Population
- Patient with severe short stature and growth hormone (GH) resistance due to a heterozygous dominant-negative GH receptor variant with elevated GH binding protein
- Participants
- 1 participants
- Outcome
- Dose of GH required to achieve an IGF-1 level above the midpoint of the normal range
Methodological detailsascending-dose phase followed by dose-stable phase
- Ascending-dose phase followed by dose-stable phase
Main finding
As reported in the abstract
GH dose achieving target IGF-1
High-dose GH
250
µg/kg/day
Secondary outcomes
| Outcome | Values |
|---|---|
| Annualized height velocity · first year of treatment | High-dose GH 8.7 cm/year |
- Annualized height velocity — Increase from baseline
- 3.4 cm/year
Change in height SDS · first year of treatment
- Gain in height
- 0.81 SD
Original publication
A Clinical Trial of High-Dose Growth Hormone in a Patient With a Dominant-Negative Growth Hormone Receptor Mutation. J Clin Endocrinol Metab. Published 10 April 2024. doi:10.
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