Abstract
BackgroundTo evaluate the efficacy of GH in improving FAH in ISS children in a multicenter study.MethodsA real-world observation was carried out. Children with ISS in seven hospitals in China were enrolled. The height gains standard deviation score and the height gain over the target height were evaluated.ResultsThere were 344 ISS patients (217 boys and 127 girls). The baseline average age of boys and girls was 12.7 and 11.7 years, with bone age of 11.7 and 10.1 years, respectively. The baseline height SDS of boys and girls was − 3.07 and − 2.74, and the FAH SDS was − 1.91 and − 1.38, respectively. Compared with the baseline height SDS, the FAH SDS was significantly increased in both boys and girls (both P = 0.0000). The FAH SDS was the highest (gain by 1.54 SD) in the ≥2y treatment course group. Two hundred eighteen patients (218/344, 63.4%) had a FAH SDS > − 2 SD. Among these patients, girls in the 1-2y treatment course group and ≥ 2y group had a FAH SDS higher than TH SDS. Even in the control group, a spontaneous catch-up growth of 1.16 SD was observed. A multivariate linear regression model was used to analyze the results, with FAH SDS as the dependent variable. It was found that the treatment course and baseline height SDS in the boys’ model were statistically significant (P < 0.05), whereas the baseline height SDS and baseline bone age significantly affected the girls’ FAH SDS (P < 0.05).ConclusionsBoth girls and boys of ISS improved FAH by GH therapy even if treatments begin over 10 years old and majority of them reached TH. Some peri-puberty ISS will have a spontaneous height gain. We recommend the course of GH treatment more than 2 years for girls, and longer courses for boys.
Highlights
To evaluate the efficacy of growth hormone (GH) in improving final adult height (FAH) in Idiopathic short status (ISS) children in a multicenter study
Compared with the baseline height Standard deviation score (SDS), the FAH SDS was significantly increased in both boys and girls (Table 1)
Comparisons between FAH SDS and baseline height SDS According to the treatment course, patients who had been treated for less than 3 months were designated the control group, and the remaining patients were divided into the 3-6 m, 6-12 m, 1-2y, and ≥ 2y groups
Summary
To evaluate the efficacy of GH in improving FAH in ISS children in a multicenter study. The main purpose of GH therapy for ISS is to attain normal adult height and avoid daily life inconvenience and psychological problems caused by extreme or unacceptable short stature. Few clinical studies have explored whether the final adult height (FAH) can reach the normal range after GH therapy. Owing to the heterogeneity of the treated ISS populations and the individualization of treatment, only a few randomized trials with small sample sizes have observed ISS until FAH [6,7,8,9,10]. More studies are needed to confirm the efficacy of GH in the treatment of ISS
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