scholarly journals A Retrospective Analysis of Patients with Short Stature in Eastern China between 2013 and 2019

2021 ◽  
Vol 2021 ◽  
pp. 1-8
Author(s):  
Qianqian Zhao ◽  
Mei Zhang ◽  
Yuntian Chu ◽  
Hailing Sun ◽  
Hui Pan ◽  
...  

Objective. To identify the aetiology of growth and development diseases and assess the long-term effectiveness of recombinant human growth hormone (rhGH) therapy in a real-life clinical setting and provide better guidance in clinical strategy and decision making. Methods. This retrospective study included 1145 children and adolescents with short stature admitted to the Department of Endocrinology, Affiliated Hospital of Jining Medical University, from January 2013 to December 2019, of whom 484 received rhGH treatment. The related anthropometrics and laboratory examinations were assessed in all participants. Results. A total of 1145 children and adolescents with short stature aged 10.5 ± 3.3 years, including 740 boys and 405 girls, were analysed in this study. The number of children and adolescents with short stature gradually increased per year from 2013 to 2019. The mean pretreatment height standard deviation score (SDS) and insulin-like growth factor-1 SDS were − 2.93 ± 1.05 and -1.01 (-1.83--0.16), respectively. The majority of the children (658, 57.47%) were prepubescent. In total, 484 subjects aged 10.6 ± 3.2 years received rhGH and were followed up, and among them, 292 children were treated for more than one year. As the treatment time increased, the children’s height SDS gradually increased, and most of them attained a height SDS within the normal range. The mean height SDS in children who were treated for more than one year was − 3.0 ± 1.0 at baseline and gradually increased to − 0.8 ± 0.3 by year 6. The results were consistent across subgroups of different aetiologies of short stature. Conclusions. Increasing attention has been given to the height of children during the period of 2013–2019 in eastern China. The present findings indicate that children with short stature need to be referred to a specialist centre to diagnose the cause of growth failure and that short children receiving rhGH therapy show a significant increase in height over time.

2021 ◽  
Vol 20 (1) ◽  
Author(s):  
Yuntian Chu ◽  
Qianqian Zhao ◽  
Mei Zhang ◽  
Bo Ban ◽  
Hongbing Tao

Abstract Background Elevated triglyceride (TG) levels are a biomarker for cardiovascular disease (CVD) risk. The correlation between serum uric acid (SUA) and TG concentrations in adults or obese children is well established. However, studies on SUA and TG in children with short stature are limited. Aim To determine the relationship between SUA and TG levels in short children and adolescents. Method This was a cross-sectional evaluation of a cohort of 1095 patients with short stature (720 males and 375 females). The related clinical characteristics, including anthropometric and biochemical parameters, were determined. Results Smooth curve fitting, adjusted for potential confounders was performed, which indicated the existence of a non-linear relationship between these measures. Piecewise multivariate linear analysis revealed a significant positive relationship between SUA and TG at SUA concentrations over 7 mg/dL (β = 0.13, 95% CI: 0.05–0.22, P = 0.002) but no significant correlation at lower SUA levels (β = 0.01, 95% CI: 0.01–0.04, P = 0.799). Furthermore, a stratified analysis was performed to appraise changes in this relationship for different sexes and standard deviation levels of body mass index (BMI). The non-linear relationship remained consistent in males and females with BMI standard deviation scores (BMI SDS) ≥ 0, with inflection points of 6.71 mg/dL and 3.93 mg/dL, respectively. Within these two groups, SUA and TG levels showed a positive association when SUA levels were higher than the inflection point (β = 0.21, 95% CI: 0.11–0.31, P < 0.001 for males and β = 0.1, 95% CI: 0.03–0.17, P = 0.005 for females). However, a specific relationship was not observed at lower SUA levels. No significant relationships were found between SUA and TG levels in males and females with BMI SDS < 0. Conclusion The present study identified the non-linear association of SUA and TG levels with short children and adolescents. This relationship was based on BMI status. This finding suggests that health status should be considered for short stature children with high SUA levels, especially in children with a high BMI standard deviation score.


Author(s):  
Gajanthan Muthuvel ◽  
Andrew Dauber ◽  
Eirene Alexandrou ◽  
Leah Tyzinski ◽  
Melissa Andrew ◽  
...  

Abstract Context Patients with aggrecan (ACAN) deficiency present with dominantly inherited short stature, often with advanced skeletal maturation and premature growth cessation. There is a paucity of information on the effects of growth-promoting interventions. Objective The aim of this study was to evaluate the efficacy and safety of recombinant human growth hormone (rhGH) therapy on linear growth in children with ACAN deficiency. Design and Setting Open-label, single-arm, prospective study at Cincinnati Children’s Hospital Medical Center. Patients Ten treatment-naïve patients were recruited. Inclusion criteria were: a confirmed heterozygous mutation in ACAN, age ≥ 2 years, pre-pubertal, bone age (BA) ≥ chronological age (CA), and normal IGF-I concentration. Intervention Treatment with rhGH (50 mcg/kg/day) over one year. Main Outcome Measure(s) Main outcomes measured were height velocity (HV) and change in (Δ) height SD (HtSDS). Results Ten patients (six females) were enrolled with median CA of 5.6 yrs (range 2.4 to 9.7). Baseline median HtSDS was -2.5 (range -4.3 to -1.1). Median baseline BA was 6.9 yrs (range 2.5 to 10.0), with median BA/CA of 1.2 (range 0.9 to 1.5). Median pre-treatment HV was 5.2 cm/y (range 3.8 to 7.1), increased to 8.3 cm/y (range 7.3 to 11.2) after one year of therapy (p=0.004). Median ΔHtSDS after one year was +0.62 (range +0.35 to +1.39) (p=0.002). Skeletal maturation did not advance inappropriately (median Δ BA/CA -0.1, p=0.09). No adverse events related to rhGH were observed. Conclusion Treatment with rhGH improved linear growth in a cohort of patients with short stature due to ACAN deficiency.


2011 ◽  
Vol 26 (S2) ◽  
pp. 280-280
Author(s):  
A. Erdogan ◽  
N. Yurteri ◽  
A.E. Tufan ◽  
H. Ankarali ◽  
E. Demirci

ObjectiveRisperidone is an atypical antipsychotic agent, despite its many advantages and widespread use, there is increasing attention to the adverse effects associated with long-term exposure to this drug.We aimed to investigate the changes in the liver function tests (LFTs) associated with one year risperidone treatment in children and adolescents.MethodsOne hundred youths who treated with risperidone more than one year were included in the study. For this study, patients’ baseline and follow-up weight and hepatobiliary function tests including alanine aminotransferases(ALT) and aspartat aminotransferases (AST), gamma gluatamyl transerase (GGT), alkaline phosphatase (ALP) and serum bilirubin levels were measured baseline, after the treatment period of six months and one year.ResultsAsymptomatic liver function test abnormalities mostly ALP elevation was found in subjects treated with risperidone. The mean levels of liver enzymes and billuribin of the patients were significantly higher after one year of treatment than the baseline. Also the mean levels of liver enzymes and billuribin of the patients were significantly higher after one year of treatment than the six months. There was significant association between changes in weight, risperidone dose and liver enzymes and billuribin levels.ConclusionsThese findings suggest that risperidone treatment in the long term commonly leads to liver function changes however it rarely may induce a serious hepatic toxicity at therapeutic doses in children and adolescents.


2001 ◽  
Vol 171 (2) ◽  
pp. 285-292 ◽  
Author(s):  
Y Khalfallah ◽  
G Sassolas ◽  
F Borson-Chazot ◽  
N Vega ◽  
H Vidal

Our aim was to investigate the effects of one year recombinant human growth hormone (rhGH) therapy on the regulation by insulin of gene expression in muscle and adipose tissue in adults with secondary GH deficiency (GHD). Six GHD subjects without upper-body obesity were submitted to a 3-h euglycemic hyperinsulinemic clamp before and after one year of rhGH therapy. Muscle and abdominal subcutaneous adipose tissue biopsies were taken before and at the end of each clamp. The mRNA levels of insulin receptor, p85 alpha-phosphatidylinositol-3 kinase (p85 alpha PI-3K), insulin dependent glucose transporter (Glut4), hexokinase II, glycogen synthase, lipoprotein lipase (LPL) in muscle and in adipose tissue, hormone sensitive lipase and peroxisome proliferator-activated receptor gamma (PPAR gamma) in adipose tissue were quantified by RT-competitive PCR. One year treatment with rhGH (1.25 IU/day) increased plasma IGF-I concentrations (54+/-7 vs 154+/-11 ng/ml, P<0.01) but did not affect insulin-stimulated glucose disposal rate measured during the hyperinsulinemic clamp (74+/-9 vs 85+/-5 micromol/kg free fat mass/min). Insulin significantly increased p85 alpha PI-3K, hexokinase II and Glut4 mRNA levels in muscle both before and after rhGH treatment. One year of GH therapy increased LPL mRNA levels in muscle (38+/-2 vs 70+/-7 amol/microg total RNA, P<0.05) and in adipose tissue (2490+/-260 vs 4860+/-880 amol/microg total RNA, P<0.05), but did not change the expression of the other mRNAs. We conclude from this study that GH therapy did not alter whole body insulin sensitivity and the response of gene expression to insulin in skeletal muscle of adult GHD patients, but it did increase LPL expression in muscle and adipose tissue. This result could be related to the documented beneficial effect of GH therapy on lipid metabolism.


2018 ◽  
Vol 90 (1) ◽  
pp. 54-63 ◽  
Author(s):  
Jin Soon Hwang ◽  
Hae Sang Lee ◽  
Kee-Hyoung Lee ◽  
Han-Wook Yoo ◽  
Dae-Yeol Lee ◽  
...  

Background/Aims: To determine the optimal dose of LB03002, a sustained-release, once-weekly formulation of recombinant human growth hormone (rhGH), and to compare its efficacy and safety with daily rhGH in children with idiopathic short stature (ISS). Methods: This multicenter, randomized, open-label, phase II study included GH-naïve, prepubertal children with ISS, randomized to receive daily rhGH 0.37 mg/kg/week (control, n = 16), LB03002 0.5 mg/kg/week (n = 14), or LB03002 0.7 mg/kg/week (n = 16). The primary endpoint was height velocity (HV) change at week 26. Results: At week 26, the least square (LS) means for HV change (cm/year) with control, LB03002 0.5 mg/kg/week, and LB03002 0.7 mg/kg/week were 5.08, 3.65, and 4.38, and the LS means for the change in height standard deviation score were 0.65, 0.49, and 0.58, respectively. The lower bound of the 90% confidence interval for the difference between LB03002 0.7 mg/kg/week and the control in the LS mean for HV change (–1.72) satisfied the noninferiority margin (–1.75). Adverse events were generally mild and short-lived. Conclusion: A once-weekly regimen of LB03002 0.7 mg/kg demonstrated noninferiority to the daily regimen of rhGH 0.37 mg/kg/week in terms of HV increments. LB03002 was well tolerated and its safety profile was comparable with that of daily rhGH.


2021 ◽  
Vol 5 (Supplement_1) ◽  
pp. A679-A679
Author(s):  
Benjamin Udoka Nwosu ◽  
Sadichchha Parajuli ◽  
Gabrielle Jasmin ◽  
Austin F Lee

Abstract Context: There is no consensus on the effect of recombinant human growth hormone (rhGH) therapy on skeletal maturation in children despite the current practice of annual monitoring of skeletal maturation with bone age in children on rhGH therapy. Aims: To investigate the effects of long-term rhGH therapy on skeletal age in children and explore the accuracy of bone age predicted adult height (BAPAH) at different ages based on 13 years of longitudinal data. Methods: A retrospective longitudinal study of 71 subjects aged 2-18 years, mean 9.9 ± 3.8y, treated with rhGH for non-syndromic short stature for a duration of 2-14y, mean, 5.5 ± 2.6y. Subjects with syndromic short stature and systemic illnesses such as renal failure were excluded. Results: Bone age minus chronological age (BA-CA) did not differ significantly between baseline and the end of rhGH therapy (-1.05 ± 1.42 vs -0.69 ± 1.63, p=0.09). Piece-wise regression however showed a quantifiable catch-up phenomenon in BA of 1.6 months per year of rhGH therapy in the first 6.5y, 95%CI 0.023 - 0.229, p=0.017, that plateaued thereafter, β=0.015, 95% CI -0.191-0.221, p=0.88. There was no relationship between BAPAH z score – height z score and the duration of rhGH therapy, p=0.68. BAPAH overestimated final adult height in younger subjects but became more precise in older subjects (p&lt;0.0001). Conclusion: Long-term rhGH therapy demonstrated an initial catch-up phenomenon in skeletal maturation in the first 6.5y that plateaued thereafter with no overall significant advancement in bone age. These findings are reassuring and do not support the practice of yearly monitoring of skeletal maturation with bone age in children on rhGH therapy, especially in younger subjects where BAPAH is imprecise.


2015 ◽  
Vol 29 (4) ◽  
pp. 198-202 ◽  
Author(s):  
Himanish Panda ◽  
Philip Mitchell ◽  
Michael Curley ◽  
Michelle Buresi ◽  
Lynn Wilsack ◽  
...  

BACKGROUND: The efficacy of gastric neurostimulation therapy for diabetic gastroparesis (GP) in a ‘real-life’ Canadian setting has not been assessed.AIMS: To assess changes in health-related quality of life (QoL), weekly vomiting frequency (WVF), total symptom score (TSS) and health care utilization 12 months before and after gastric neurostimulator implantation in a diabetic GP cohort.METHODS: Medication-refractory diabetic GP patients (n=7, four female, mean age 42 years) were prospectively recruited from 2008 to 2012. QoL scores were self-administered and obtained at baseline, 24 and 48 weeks postimplantion. WVF and TSS were assessed similarly. Health care usage, measured as hospitalization frequency and medication cost, was obtained six and 12 months before and after implant. Changes from baseline to six and 12 months for all outcomes were compared.RESULTS: The mean (± SD) QoL according to EuroQol was significantly better at 24 weeks after the baseline measurement (baseline 29±5, 24 weeks 52±7; P=0.03). The mean improvement in TSS was significantly better at one year postintervention (baseline score 35±5 versus 12 months 27±3; P=0.03). Changes in Short-Form 36 Health Survey and WVF were not significant. Days of GP-related hospitalization were highly variable but decreased from a median of 71 days (range 0 to 227 days) to 29 days (range two to 334 days) one year before and after surgery, respectively (P=0.735). Outpatient medication costs did not decrease to a significant extent.CONCLUSION: Gastric neurostimulation for diabetic GP appeared to show some beneficial palliative effects overall in the present small open-label series, but the effect is highly variable among patients, and placebo effect cannot be ruled out.


1990 ◽  
Vol 10 (3) ◽  
pp. 209-214 ◽  
Author(s):  
Richard N. Fine ◽  
Vera H. Koch ◽  
M. Ines Boechat ◽  
Barbara H. Lippe ◽  
Pauline A. Nelson ◽  
...  

The authors studied the effect of recombinant growth hormone (rhGH) treatment on 5 growth retarded children, age 21/12 to 178/12 years, who had end-stage renal disease (ESRD) and were undergoing continuous cycling peritoneal dialysis (CCPD). Patients received 0.125 mg/kg of subcutaneous rhGH 3 times weekly. Accelerated height velocity compared to the previous year of CCPD was noted in 2 patients and improvement in the standard deviation score (SDS) as a parameter of improved growth velocity was noted in a third patient. This was associated with an increase in weight and improvement in the midarm muscle circumference (MAMC) suggesting an anabolic effect of rhGH treatment. Bone age advancement was consistent with the period of observation; no advancement greater than that expected for the increase in chronological age was observed. No significant side effects were attributable to rhGH therapy. These preliminary results indicate some growth retarded children without growth hormone deficiency with ESRD undergoing CCPD may respond to exogenous rhGH therapy with an acceleration in growth velocity: However, the failure to achieve uniform acceleration of height velocity indicates the need for controlled studies before rhGH can be recommended for all growth retarded children with ESRD undergoing peritoneal dialysis.


2012 ◽  
Vol 2012 ◽  
pp. 1-4 ◽  
Author(s):  
Mohammad I. El Mouzan ◽  
Abdullah S. Al Herbish ◽  
Abdullah A. Al Salloum ◽  
Ahmad A. Al Omer ◽  
Mansour M. Qurachi

Objective. To assess the magnitude of regional difference in prevalence of short stature in Saudi children and adolescents.Subjects and Methods. A representative sample from three different regions of the Kingdom of Saudi Arabia (KSA) (North, Southwest, and Center) was used to calculate the prevalence of short stature (standard deviation score less than-2) in children 5 to 17 years of age.Results. There were 9018 children and adolescents from 5 to 17 years of age (3366, 2825, and 2827 in the Northern, Southwestern and Central regions, resp.) and 51% were boys. In both school-age children and adolescents, there was a significantly higher prevalence of short stature in the Southwestern than in the Northern or the Central region (P< 0.0001).Conclusion. The finding of significant regional variation between regions helps in planning priorities for research and preventive measures.


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