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Idiopathic short stature: results of a one-year controlled study of human growth hormone treatment. Genentech

    The Journal of Pediatrics
    |November 1, 1989
    PubMed

    Insights

    Children with short stature not meeting growth hormone deficiency criteria showed improved growth rates with recombinant somatropin (human growth hormone). This treatment significantly increased growth velocity and predicted height without adverse effects.

    Area of Science:

    • Pediatrics
    • Endocrinology
    • Growth Disorders

    Background:

    • Short stature in children can stem from various causes, including familial short stature and constitutional delay of growth and development.
    • Traditional criteria for growth hormone deficiency (GHD) exclude some children with significant short stature and slow growth.
    • Recombinant somatropin is a synthetic form of human growth hormone used to treat GHD.

    Purpose of the Study:

    • To evaluate the efficacy and safety of recombinant somatropin in children with short stature who do not meet classic GHD criteria.
    • To assess the impact of growth hormone treatment on growth rate, bone age, and predicted adult height.
    • To identify potential predictors of growth response to somatropin therapy.

    Main Methods:

    • A multicenter randomized trial involving 121 children with short stature.
    • Treatment group received recombinant somatropin (0.1 mg/kg three times weekly) for 1 year.
    • Control group received no treatment. Growth rate, bone age, predicted height, and plasma insulin-like growth factor I were measured.

    Main Results:

    • Treated children experienced a significant increase in mean growth rate (4.6 to 7.5 cm/yr) compared to controls (4.2 to 5.0 cm/yr).
    • A 1-year advance in bone age was observed in both groups, but only the treated group showed a significant increase in predicted height.
    • Baseline insulin-like growth factor I levels inversely correlated with growth response (r = -0.50, p = 0.0003); growth hormone levels did not predict response.

    Conclusions:

    • Recombinant somatropin therapy significantly improves growth rate and predicted height in children with short stature not meeting classic GHD criteria.
    • The treatment is well-tolerated, with no observed side effects.
    • Insulin-like growth factor I levels may serve as a predictive biomarker for treatment response.

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