Long-Term Treatment for Laron Syndrome with IGF-1 Injection over 22 Years in Saudi Arabia: A Cohort Study
Main finding · Height velocityResult — go to the resultMean difference 3.1 cm/yearAuthors' conclusion
IGF-1 therapy significantly enhanced linear growth in children with Laron syndrome and was generally well tolerated. Although many patients did not reach normal adult height, the growth achieved with IGF-1 treatment was markedly better than expected without therapy. This study underscores the effectiveness of IGF-1 in improving growth outcomes and highlights the need for continued longitudinal studies to optimize treatment strategies and manage potential complications.
Study context
- Population
- Patients with Laron syndrome (growth hormone (GH) insensitivity syndrome), 12 males and 16 females, treated 1998–2020 at KFSH & RC, Riyadh, Saudi Arabia; age >2 years, height standard deviation score (SDS) ≤−2.8, GH >2.5 ng/mL, IGF-1 <50 ng/mL, insensitivity to exogenous GH12 males · 16 females
- Participants
- 28 patients
- Exposure
- IGF-1 therapy: on-treatment vs baseline
- IGF-1n = 28
- Outcome
- Height velocity
- Follow-up
- First year
Methodological detailssingle-centre, long-term follow-up
- Single-centre
- Long-term follow-up
Main finding
As reported in the abstract
Mean difference
3.1 cm/year
P < 0.0001
Observational data: associations, not effects.
Further findings
| Outcome | Mean difference (cm/year) | P value |
|---|---|---|
| Height velocity · second yearIGF-1 second year vs Baseline · HV 5 cm/year | 1.6 | P = 0.0015 |
Long-term height velocity · 10 years
- Sustained height velocity (HV) improvements vs baseline, largest gains in the initial 5 years
Weight SDS
- Weight SDSs also showed significant improvement
Growth by age at start and treatment duration
- Age at start did not notably affect growth outcomes; longer treatment associated with greater growth
GHR genetic variants
- Ten disease-causing GHR variants identified
- 24 of the 28 patients
Original publication
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