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      Quality of Life of Short-Statured Children Born Small for Gestational Age or Idiopathic Growth Hormone Deficiency Within 1 Year of Growth Hormone Treatment

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          Abstract

          Aside from clinical endpoints like height gain, health-related quality of life has also become an important outcome indicator in the medical field. However, the data on short stature and health-related quality of life is inconsistent. Therefore, we examined changes in health-related quality of life in German children with idiopathic growth hormone deficiency or children born small for gestational age before and after 12 months of human growth hormone treatment. Children with idiopathic short stature without treatment served as a comparison group. At baseline, health-related quality of life data of 154 patients with idiopathic growth hormone deficiency ( n = 65), born small for gestational age ( n = 58), and idiopathic short stature ( n = 31) and one parent each was collected. Of these, 130 completed health-related quality of life assessments after 1-year of human growth hormone treatment. Outcome measures included the Quality of Life in Short Stature Youth questionnaire, as well as clinical and sociodemographic data. Our results showed that the physical, social, and emotional health-related quality of life of children treated with human growth hormone significantly increased, while untreated patients with idiopathic short stature reported a decrease in these domains. Along with this, a statistically significant increase in height in the treated group can be observed, while the slight increase in the untreated group was not significant. In conclusion, the results showed that human growth hormone treatment may have a positive effect not only on height but also in improving patient-reported health-related quality of life of children with idiopathic growth hormone deficiency and children born small for gestational age.

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          Most cited references40

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          The LMS method for constructing normalized growth standards.

          T. J. Cole (1990)
          It is now common practice to express child growth status in the form of SD scores. The LMS method provides a way of obtaining normalized growth centile standards which simplifies this assessment, and which deals quite generally with skewness which may be present in the distribution of the measurement (eg height, weight, circumferences or skinfolds). It assumes that the data can be normalized by using a power transformation, which stretches one tail of the distribution and shrinks the other, removing the skewness. The optimal power to obtain normality is calculated for each of a series of age groups and the trend summarized by a smooth (L) curve. Trends in the mean (M) and coefficient of variation (S) are similarly smoothed. The resulting L, M and S curves contain the information to draw any centile curve, and to convert measurements (even extreme values) into exact SD scores. A table giving approximate standard errors for the smoothed centiles is provided. The method, which is illustrated with US girls' weight data, should prove useful both for the construction and application of growth standards.
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            Consensus statement on the diagnosis and treatment of children with idiopathic short stature: a summary of the Growth Hormone Research Society, the Lawson Wilkins Pediatric Endocrine Society, and the European Society for Paediatric Endocrinology Workshop.

            Our objective was to summarize important advances in the management of children with idiopathic short stature (ISS). Participants were 32 invited leaders in the field. Evidence was obtained by extensive literature review and from clinical experience. Participants reviewed discussion summaries, voted, and reached a majority decision on each document section. ISS is defined auxologically by a height below -2 sd score (SDS) without findings of disease as evident by a complete evaluation by a pediatric endocrinologist including stimulated GH levels. Magnetic resonance imaging is not necessary in patients with ISS. ISS may be a risk factor for psychosocial problems, but true psychopathology is rare. In the United States and seven other countries, the regulatory authorities approved GH treatment (at doses up to 53 microg/kg.d) for children shorter than -2.25 SDS, whereas in other countries, lower cutoffs are proposed. Aromatase inhibition increases predicted adult height in males with ISS, but adult-height data are not available. Psychological counseling is worthwhile to consider instead of or as an adjunct to hormone treatment. The predicted height may be inaccurate and is not an absolute criterion for GH treatment decisions. The shorter the child, the more consideration should be given to GH. Successful first-year response to GH treatment includes an increase in height SDS of more than 0.3-0.5. The mean increase in adult height in children with ISS attributable to GH therapy (average duration of 4-7 yr) is 3.5-7.5 cm. Responses are highly variable. IGF-I levels may be helpful in assessing compliance and GH sensitivity; levels that are consistently elevated (>2.5 SDS) should prompt consideration of GH dose reduction. GH therapy for children with ISS has a similar safety profile to other GH indications.
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              Guidelines for Growth Hormone and Insulin-Like Growth Factor-I Treatment in Children and Adolescents: Growth Hormone Deficiency, Idiopathic Short Stature, and Primary Insulin-Like Growth Factor-I Deficiency

              Background/Aims: On behalf of the Drug and Therapeutics, and Ethics Committees of the Pediatric Endocrine Society, we sought to update the guidelines published in 2003 on the use of growth hormone (GH). Because idiopathic short stature (ISS) remains a controversial indication, and diagnostic challenges often blur the distinction between ISS, GH deficiency (GHD), and primary IGF-I deficiency (PIGFD), we focused on these three diagnoses, thereby adding recombinant IGF-I therapy to the GH guidelines for the first time. Methods: This guideline was developed following the GRADE approach (Grading of Recommendations, Assessment, Development, and Evaluation). Results: This guideline provides recommendations for the clinical management of children and adolescents with growth failure from GHD, ISS, or PIGFD using the best available evidence. Conclusion: The taskforce suggests that the recommendations be applied in clinical practice with consideration of the evolving literature and the risks and benefits to each individual patient. In many instances, careful review highlights areas that need further research.
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                Author and article information

                Contributors
                Journal
                Front Pediatr
                Front Pediatr
                Front. Pediatr.
                Frontiers in Pediatrics
                Frontiers Media S.A.
                2296-2360
                29 April 2019
                2019
                : 7
                : 164
                Affiliations
                [1] 1Center for Psychosocial Medicine, Institute for Medical Psychology, University Medical Center Hamburg-Eppendorf , Hamburg, Germany
                [2] 2Faculty of Psychology and Education Sciences, Center for Research in Neuropsychology and Cognitive Behavioral Intervention, University of Coimbra , Coimbra, Portugal
                [3] 3Children and Adolescent Endocrinology, MVZ am AKK GmbH , Hamburg, Germany
                [4] 4Clinic for Children and Adolescents, Augsburg Hospital , Augsburg, Germany
                [5] 5Clinic for Children and Adolescent Medicine, Chemnitz Hospital , Chemnitz, Germany
                [6] 6Endocrinology Clinic , Frankfurt am Main, Germany
                [7] 7Diabetes Center , Gauting, Germany
                [8] 8Center for Children and Adolescent Medicine, University Clinic of Heidelberg , Heidelberg, Germany
                [9] 9Pediatric Endocrinology, University Clinic of Cologne , Cologne, Germany
                [10] 10Center for Children and Adolescent Medicine, HELIOS Hospital , Krefeld, Germany
                [11] 11Kinderzentrum am Johannisplatz , Leipzig, Germany
                [12] 12University Children's Clinic, Otto von Geuricke University , Magdeburg, Germany
                [13] 13Clinic for Children and Adolescents, Erlangen-Nürnberg Universtiy , Erlangen, Germany
                Author notes

                Edited by: Mohamad Maghnie, University of Genoa, Italy

                Reviewed by: Lorenzo Iughetti, University of Modena and Reggio Emilia, Italy; George Paltoglou, National and Kapodistrian University of Athens Medical School, Greece

                *Correspondence: Julia Quitmann j.quitmann@ 123456uke.de

                This article was submitted to Pediatric Endocrinology, a section of the journal Frontiers in Pediatrics

                †These authors have contributed equally to this work and share joint-first authorship

                Article
                10.3389/fped.2019.00164
                6501464
                31111024
                bc50de8e-a9b3-4e83-a926-604e3cf82fcb
                Copyright © 2019 Quitmann, Bloemeke, Silva, Bullinger, Witt, Akkurt, Dunstheimer, Vogel, Böttcher, Kuhnle Krahl, Bettendorf, Schönau, Fricke-Otto, Keller, Mohnike and Dörr.

                This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.

                History
                : 13 February 2019
                : 09 April 2019
                Page count
                Figures: 3, Tables: 3, Equations: 0, References: 49, Pages: 11, Words: 7730
                Categories
                Pediatrics
                Original Research

                short stature,health-related quality of life,growth hormone treatment,iss,ighd,qolissy

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