Dataset Information

Final adult height of children with idiopathic short stature: a multicenter study on GH therapy alone started during peri-puberty.

ABSTRACT:

Background

To evaluate the efficacy of GH in improving FAH in ISS children in a multicenter study.

Methods

A 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.

Results

There 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?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.

SUBMITTER: Wu D

PROVIDER: S-EPMC7102429 | biostudies-literature | 2020 Mar

REPOSITORIES: biostudies-literature

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Publications

Final adult height of children with idiopathic short stature: a multicenter study on GH therapy alone started during peri-puberty.

Wu Di D Chen Rui-Min RM Chen Shao-Ke SK Liu Ge-Li GL Chen Lin-Qi LQ Yang Yu Y Wang Xin-Li XL Peng Ya-Guang YG Gong Chun-Xiu CX

BMC pediatrics 20200328 1

<h4>Background</h4>To evaluate the efficacy of GH in improving FAH in ISS children in a multicenter study.<h4>Methods</h4>A 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.<h4>Results</h4>There 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, ...[more]

PMID: 32222149

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Project description:BACKGROUND:Children with Idiopathic Short Stature do not attain a normal adult height. The improvement of adult height with treatment with recombinant human growth hormone (rhGH), at doses of 0.16 to 0.28 mg/kg/week is modest, usually less that 4 cm, and they remain short as adults. The benefit obtained seems dose dependent and benefits of 7.0 to 8.0 cm have been reported with higher doses of 0.32 to 0.4 mg/kg/week, but the number of studies is limited. The topic has remained controversial. OBJECTIVE:The objective was to conduct a retrospective analysis of our experience with 123 children with ISS treated with 0.32?±?0.03 mg/kg/week of rhGH, with the aim of comparing the different subgroups of non-familial short stature, familial short stature, normal puberty, and delayed puberty and to assess the benefit by comparison with 305 untreated historical controls, from nine different randomized and nonrandomized controlled studies. RESULTS:Eighty eight of our children (68 males and 20 females) attained an adult height or near adult height of -0.71 SDS (0.74 SD) (95% CI, -0.87 to -0.55) with a benefit over untreated controls of 9.5 cm (7.4 to 11.6 cm) for males and 8.6 cm (6.7 to 10.5 cm) for females. In the analysis of the subgroups, the adult height and adult height gain of children with non-familial short stature were significantly higher than of familial short stature. No difference was found in the cohorts with normal or delayed puberty in any of the subgroups, except between the non-familial short stature and familial short stature puberty cohorts. This has implications for the interpretation of the benefit of treatment in studies where the number of children with familial short stature in the controls or treated subjects is not known. The treatment was safe. There were no significant adverse events. The IGF-1 values were essentially within the levels expected for the stages of puberty. CONCLUSION:Our experience was quite positive with normalization of the heights and growth of the children during childhood and the attainment of normal adult heights, the main two aims of treatment.

Project description:Alström syndrome (ALMS) is a rare autosomal recessive monogenic disease included in an emerging class of genetic disorders called 'ciliopathies' and is likely to impact the central nervous system as well as metabolic and endocrine function. Individuals with ALMS present clinical features resembling a growth hormone deficiency (GHD) condition, but thus far no study has specifically investigated this aspect in a large population.Twenty-three patients with ALMS (age, 1-52 years; 11 males, 12 females) were evaluated for anthropometric parameters (growth charts and standard deviation score (SDS) of height, weight, BMI), GH secretion by growth hormone-releasing hormone + arginine test (GHRH-arg), bone age, and hypothalamic-pituitary magnetic resonance imaging (MRI). A group of 17 healthy subjects served as controls in the GH secretion study. Longitudinal retrospective and prospective data were utilized.The length-for-age measurements from birth to 36 months showed normal growth with most values falling within -0·67 SDS to +1·28 SDS. A progressive decrease in stature-for-age was observed after 10 years of age, with a low final height in almost all ALMS subjects (>16-20 years; mean SDS, -2·22 ± 1·16). The subset of 12 patients with ALMS tested for GHRH-arg showed a significantly shorter stature than age-matched controls (154·7 ± 10·6 cm vs 162·9 ± 4·8 cm, P = 0·009) and a mild increase in BMI (Kg/m(2) ) (27·8 ± 4·8 vs 24·1 ± 2·5, P = 0·007). Peak GH after GHRH-arg was significantly lower in patients with ALMS in comparison with controls (11·9 ± 6·9 ?g/l vs 86·1 ± 33·2 ?g/l, P < 0·0001). Severe GHD was evident biochemically in 50% of patients with ALMS. The 10 adult ALMS patients with GHD showed a reduced height in comparison with those without GHD (149·7 ± 6·2 cm vs 161·9 ± 9·2 cm, P = 0·04). MRIs of the diencephalic and pituitary regions were normal in 11 of 12 patients. Bone age was advanced in 43% of cases.Our study shows that 50% of nonobese ALMS patients have an inadequate GH reserve to GHRH-arg and may be functionally GH deficient. The short stature reported in ALMS may be at least partially influenced by impairment of GH secretion.

Dataset Information

Final adult height of children with idiopathic short stature: a multicenter study on GH therapy alone started during peri-puberty.

Background

Methods

Results

Publications

Final adult height of children with idiopathic short stature: a multicenter study on GH therapy alone started during peri-puberty.

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