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Loche S.,Servizio di Endocrinologia Pediatrica
Hormone Research in Paediatrics | Year: 2011

Children with idiopathic short stature (ISS) may not reach an adult height within their genetic target. In 2003, the United States Food and Drug Administration approved biosynthetic growth hormone (GH) for the treatment of children with ISS whose heights exceeded 2.25 standard deviation scores below the mean and who were considered unlikely to reach a normal adult height. Results of controlled studies have shown that, although GH treatment leads to a substantial increase in adult height, the individual response to therapy is difficult to predict. A number of auxological variables (i.e., age and height at start of treatment, bone age delay, mean predicted height, height velocity and first-year responsiveness) are used in multivariate analyses to predict outcomes. Estimation of target height, predicted adult height and pattern of growth should guide the decision to treat a child with ISS. Copyright © 2011 S. Karger AG, Basel.

Rapaport R.,Mount Sinai School of Medicine | Saenger P.,Winthrop University | Schmidt H.,Ludwig Maximilians University of Munich | Hasegawa Y.,Tokyo Metropolitan Childrens Medical Center | And 5 more authors.
Medical Devices: Evidence and Research | Year: 2013

Close adherence to the recommended treatment regimen is important for the success of recombinant human growth hormone therapy, although nonadherence can be common. Ease of use and safety during use/storage are among several important factors in the design of a growth hormone injection device intended for long-term use. This study was performed to validate the usability and assess the ease of use of a new pen device (SurePal™) that has been developed to support daily administration of the recombinant human growth hormone product, Omnitrope® (somatropin). The primary objectives of the study were to assess if study participants, representing intended users of the pen in clinical practice, were able to perform an injection procedure into an injection pad effectively and safely and disassemble the pen without receiving a needlestick injury. A total of 106 participants (61 adults and 45 children/adolescents) were enrolled at two study centers (one in the US, one in Germany). Results for both primary usability tasks met the predefined acceptance criteria, with >85% of participants successfully performing each task. All of the other tasks/handling steps assessed were also successfully performed by most participants, with high success rates reflected in the high proportion of participants who classified each task as "very easy" or "easy". After a second use of the device, 87%-97% of participants rated it as "very easy" or "easy" to use. In summary, the new pen device is safe and easy to use for both adults and children, and will help to support effective, long-term daily administration of the recombinant human growth hormone product, Omnitrope®. © 2013 Rapaport et al.

Di Iorgi N.,University of Genoa | Napoli F.,University of Genoa | Allegri A.,University of Genoa | Secco A.,University of Genoa | And 9 more authors.
Journal of Clinical Endocrinology and Metabolism | Year: 2010

Context: The accuracy of the glucagon test in the diagnosis of central adrenal insufficiency in young children has not yet been definitively established. Objective: The aim of this study was to investigate the diagnostic accuracy of the glucagon test as an alternative to the insulin tolerance test (ITT) in children with GH deficiency under 6 yr of age. Design and Setting: This was a prospective study conducted in two Pediatric Endocrinology Centers. Patients and Methods: Forty-eight children (median age, 4.2 yr) with GH deficiency confirmed by a peak GH to ITT and arginine less than 10 μg/liter were enrolled: 24 with normal hypothalamicpituitary anatomy, seven with isolated anterior pituitary hypoplasia, and 17 with structural hypothalamic-pituitary abnormalities at magnetic resonance imaging. Twelve subjects had central adrenal insufficiency defined by a peak cortisol response of less than 20 μg/dl to ITT. All children underwent a glucagon stimulation test with blood sampling for cortisol and glucose (time 0 to 180 min) after the im administration of 30 μg/kg of glucagon. Results: Themeanpeakcortisol after glucagonwasnot significantly differentfromthat obtained after ITT in the whole cohort (25.9 vs. 26.0 μg/dl; P=0.908), and it was significantly reduced in patients with structural hypothalamic-pituitary abnormalities (P < 0.001). Receiver operating characteristic curve analysis showed that the best diagnostic accuracy was obtained with a peak cortisol cutoff to glucagon of 14.6 μg/dl (sensitivity, 66.67%; specificity, 100%; area under the curve = 0.91; 95% confidence interval, 0.82-0.99). Using this cutoff, 91.67% of the patients were correctly classified. Conclusions: This study shows that glucagon is an accurate and safe diagnostic test for adrenal function in young children who are at risk for adrenal insufficiency. Copyright © 2010 by The Endocrine Society.

Zavattari P.,Servizio di Endocrinologia Pediatrica | Zavattari P.,University of Cagliari | Loche A.,Servizio di Endocrinologia Pediatrica | Loche A.,Friedrich Miescher Institute for Biomedical Research | And 6 more authors.
Annals of Human Genetics | Year: 2011

Several studies have reported an association of the intronic single nucleotide polymorphism (SNP) rs9939609 of the fat mass and obesity-associated (FTO) gene with obesity and with a number of obesity-related features. We studied the association of rs9939609 with obesity in 912 obese children and adolescents (426 males and 486 females, mean ± SD age 10.5 ± 3.3 years) and in 543 normal weight subjects. A number of biochemical and clinical parameters was also evaluated in 700 of these patients. In the obese group, mean body mass index standard deviation score (BMI-SDS) was similar between the three genotypes. The A allele was present in 55% of the patients' and in 43% of controls' chromosomes. The distribution of heterozygotes was similar between patients and controls (47%), while the distribution of AA homozygotes was significantly higher in patients (31% vs. 20%). Logistic regression analysis on the genotypes yielded a χ 2 of 35.5 with an odds ratio of 1.6 (CI = 1.3-1.8), P < 1 × 10 -5. None of the clinical and metabolic parameters tested was associated with the genotype. In conclusion, we have confirmed the strong association between FTO and obesity, and shown that only AA homozygotes are predisposed to develop obesity while TT homozygotes might be protected. Finally, we found no association between rs9939609 and a number of obesity-related abnormalities. © 2011 The Authors, Annals of Human Genetics © 2011 Blackwell Publishing Ltd/University College London.

Ibba A.,Servizio di Endocrinologia Pediatrica | Pilia S.,Servizio di Endocrinologia Pediatrica | Zavattari P.,Servizio di Endocrinologia Pediatrica | Zavattari P.,University of Cagliari | And 6 more authors.
Journal of Pediatric Endocrinology and Metabolism | Year: 2013

Aim: We aimed to study the influence of the fat mass and obesity-associated ( FTO ) gene on eating behavior in 412 obese Sardinian children and adolescents. Genome-wide association studies (GWAS) have identified several susceptibility loci for obesity. Among these, the polymorphisms in the intron 1 of the FTO gene has been found associated to weight gain and obesity in various populations. Methods : All obese patients were genotyped for the FTO single nucleotide polimorphysm (SNP) rs9939609. In all subjects we evaluated eating behavior using the Child Eating Behaviour Questionnaire (CEBQ). Results: We found no differences in eating behavior according to the genotype, either in the entire cohort, or when subjects were subdivided into four different age groups. Conclusions: FTO genotype is associated with body mass index but does not influence eating behavior in a selected cohort of obese children from the isolated genetic population of Sardinia.

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