What question did this study set out to answer?

To evaluate the long-term efficacy and safety of GH treatment in pediatric patients with SHOX deficiency.

Efficacy and safety of GH treatment in Japanese pediatric patients with SHOX deficiency: an open-label extension study

View Full Paper

Ask AI

Bookmark

Authors

An open-label extension study reports GH treatment effects on height in pediatric patients, suggesting benefits for SHOX deficiency.

To evaluate the long-term efficacy and safety of GH treatment in pediatric patients with SHOX deficiency.
Conducted an open-label extension study for 19 Japanese pediatric patients.
Administered GH treatment at a dosage of 0.35 mg/kg/wk, later adjusted to 0.23 mg/kg/wk for one patient.
Measured height SDS for chronological age and serum IGF-1 levels.
Assessed height velocity and bone age progression over 48 months.
Height SDS increased in 18 of the 19 patients, with 17 exceeding -2.0 at the latest visit.
Height velocity significantly increased during the first year of treatment.
Bone age progressed faster than chronological age, maintaining ΔBA/ΔCA above 1.0.
Serum IGF-1 SDS showed an initial increase, stabilizing thereafter.
GH treatment was well-tolerated with minimal adverse events.

Ogata et al. (2026) studied this question.

View Full Paper

Ask AI

Bookmark