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Long-term results of GH therapy in GH-deficient children treated before 1 year of age
F Huet1, J C Carel, J L Nivelon
1Service de Pédiatrie 1, Hôpital d'Enfants, Dijon, France.
Insights
Growth hormone (GH) therapy effectively normalizes height in children with early-onset GH deficiency. Most patients achieved significant height gains, demonstrating the long-term benefits of timely GH treatment.
Area of Science:
- Pediatric Endocrinology
- Growth Hormone Therapy
- Hormone Deficiency
Background:
- Early diagnosis of growth hormone (GH) deficiency is crucial for effective intervention.
- Neonatal manifestations like hypoglycemia and jaundice are common in infants with GH deficiency.
- Severe growth retardation is often present at the time of diagnosis in these patients.
Purpose of the Study:
- To assess the long-term efficacy of GH therapy in patients diagnosed with GH deficiency before one year of age.
- To evaluate growth outcomes and final height in a cohort of early-treated GH-deficient individuals.
Main Methods:
- A retrospective study of 59 patients treated with GH before age one.
- Analysis of clinical presentation, neonatal manifestations, and growth parameters during GH therapy.
- Assessment of height standard deviation scores (SDS) before and after treatment.
Main Results:
- Patients experienced severe growth retardation at diagnosis (mean -3.5 SDS).
- GH therapy over a mean of 8.0 years resulted in a significant height SDS increase (+3.11 SDS).
- 19 patients exceeded 4 SDS gain, and only 15% did not reach -2 SDS height.
Conclusions:
- Standard doses of GH therapy are effective in normalizing height for patients with early-onset GH deficiency.
- Pre-treatment height SDS was a significant predictor of overall growth response.
- Early GH treatment leads to substantial and sustained height normalization.
Objectives:
To evaluate the long-term effects of GH therapy in early diagnosed GH-deficient patients treated before 1 year of age.
Study Design:
We studied all 59 patients (33 males) recorded by Association France-Hypophyse and treated with GH (0.50+/-0.15 IU/kg (S.D.) per week) before 1 year of age. Clinical presentation and growth parameters under GH treatment were analyzed.
Results:
Neonatal manifestations of hypopituitarism were frequent: hypoglycemia (n=50), jaundice (n=25) and micropenis (n=17/33). Although birth length was moderately reduced (-0.9+/-1.4), growth retardation at diagnosis (5.8+/-3.8 months) was severe (-3.5+/-1.9 standard deviation scores (SDS)). Fifty patients (85%) had thyrotropin and/or corticotropin deficiency. After a mean duration of GH therapy of 8.0+/-3.6 years, change in height SDS was +3.11+/-2.06 S.D., exceeding 4 SDS in 19 patients. Only 9 patients (15%) did not reach a height of -2 S.D. for chronological age and 20 patients (34%) exceeded their target height. Pretreatment height SDS was independently associated with total catch-up growth.
Conclusion:
Conventional doses of GH allow normalization of height in patients with early GH deficiency and treatment.