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Recombinant human growth hormone in infants and young children with chronic renal insufficiency. Genentech
R N Fine1, K M Attie, J Kuntze
1Department of Pediatrics, State University of New York at Stony Brook 11794-8111, USA.
Insights
Recombinant human growth hormone (rhGH) significantly improved growth rates in young children with chronic renal insufficiency (CRI). This treatment led to a substantial increase in height standard deviation scores over two years.
Area of Science:
- Pediatric Nephrology
- Endocrinology
- Growth Hormone Therapy
Background:
- Children with chronic renal insufficiency (CRI) often experience significant growth retardation early in life.
- Congenital structural abnormalities are a common cause of CRI in pediatric populations.
- Early intervention is crucial for managing growth deficits in children with CRI.
Purpose of the Study:
- To evaluate the efficacy and safety of recombinant human growth hormone (rhGH) in young children (< 2.5 years) with CRI.
- To assess the impact of rhGH on growth rate and height standard deviation scores in this specific pediatric cohort.
Main Methods:
- A multicenter, placebo-controlled study involving 30 pediatric participants with CRI aged < 2.5 years at enrollment.
- Participants were randomized to receive either rhGH (0.05 mg/kg/day subcutaneously) or placebo injections.
- Growth rates and height standard deviation scores were monitored over a two-year period.
Main Results:
- The rhGH-treated group showed significantly higher growth rates in both the first year (14.1 cm/year vs. 9.3 cm/year) and second year (8.6 cm/year vs. 6.9 cm/year) compared to placebo (P < 0.00005 and P = 0.025, respectively).
- The delta height standard deviation score over two years was significantly greater in the rhGH group (+2.0) versus the placebo group (-0.2) (P < 0.00005).
- Adverse events were infrequent and occurred with similar frequency in both treatment groups.
Conclusions:
- rhGH therapy is effective in improving growth rates and height attainment in very young children with chronic renal insufficiency.
- The safety profile of rhGH in this pediatric population appears favorable, with minor adverse events comparable to placebo.
- Early initiation of rhGH treatment may be a valuable strategy for mitigating growth retardation in children with CRI.
Abstract:
Children with chronic renal insufficiency (CRI) secondary to congenital structural abnormalities frequently have significant growth retardation by 2 years of age. In a multicenter placebo-controlled study of the use of recombinant human growth hormone (rhGH), 30 of 125 (24%) participants were < 2.5 years of age at enrollment. Since the treatment arms of the study were balanced for age at randomization, data for these patients were examined for efficacy and safety. During the first 2 years of the study, approximately two-thirds of the patients (n = 19) received rhGH 0.05 mg/kg per day subcutaneously and one-third (n = 11) received placebo injections. At entry into the study, the mean (+/- SD) calculated creatinine clearance was 29.2 +/- 14.3 (range 12.0-63.7) ml/min per 1.73 m2 in the rhGH-treated group and 23.3 +/- 15.1 (range 8.0-59.4) ml/min per 1.73 m2 in the placebo-treated group. The 1st year growth rate was 14.1 +/- 2.6 cm/year for the rhGH-treated group and 9.3 +/- 1.5 cm/year in the placebo-treated group (P < 0.00005). During the 2nd year of the study, the growth rate was 8.6 +/- 1.2 cm/year in the rhGH-treated group compared with 6.9 +/- 1.0 in the placebo group (P = 0.025). The delta height standard deviation score was +2.0 +/- 0.7 for the rhGH-treated group compared with -0.2 +/- 1.1 in the placebo-treated group (P < 0.00005) during the 2 years of the study. Minor adverse events occurred with similar frequency in both groups.(ABSTRACT TRUNCATED AT 250 WORDS)