Abstract
BACKGROUND: Recombinant human growth hormone (rhGH) is a standard treatment for idiopathic growth hormone deficiency (IGHD) patients to normalize growth. Prior studies on children mainly focused on short-term growth velocity effects, with limited long-term data on adult height outcomes. This study aims to assess adult height outcomes in patients with and without rhGH treatment, as well as evaluate the efficacy of rhGH treatment in individuals with IGHD. METHODS: A total of 169 individuals with IGHD who had attained their adult height were recruited. The related clinical and laboratory data, including anthropometric parameters, insulin-like growth factor-1 (IGF-1) levels, and the peak growth hormone (GH) levels, were collected. To assess the effectiveness of rhGH treatment, we evaluated the increase in final adult height and height standard deviation score (SDS). RESULTS: The final adult height SDS was -0.78 (interquartile range: -1.78 to 0.45) in the rhGH untreated group and -0.45 (interquartile range: -1.13 to 0.05) in the rhGH-treated group. The study results revealed that, in the IGHD population, the final adult height SDS and the increase in height SDS in the rhGH treatment group were significantly greater than those in the untreated group (P<0.05). Furthermore, the results of multiple regression analysis showed a significant increase in adult height SDS in patients treated with rhGH compared to those not treated with rhGH (β=0.41, 95% confidence interval: 0.14, 0.69; P=0.003) in the IGHD population. The baseline height SDS, peak GH, and rhGH treatment significantly affected the final adult height and height SDS gain in the IGHD population. CONCLUSIONS: Our findings demonstrate that rhGH treatment effectively improves the final height SDS and height SDS gain in children with IGHD.