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Objective: The aim of this study was to evaluate executive function (EF), such as inhibition and working memory, in children with isolated growth hormone deficiency (IGHD) using performance-based tests and parent-report scales., Methods: A total of seventy children between the ages of 7 and 12 years were included in the study. Half (n=35) had children with IGHD and half were healthy controls. To evaluate the EF performances of the participants, the Visual Aural Digit Span Test-B Form (VADS-B) and Stroop task were applied. EF was also evaluated using the Behavior Rating Inventory of Executive Function (BRIEF)., Results: Children with IGHD scored lower on the VADS-B form for short-term memory (p<0.05) compared to healthy controls. In addition, the completion time for the Stroop-color/word test was significantly longer in children with IGHD (p<0.05). For children with IGHD, their parents reported higher scores on all sub-scales of the BRIEF scale, with statistically significant differences for all sub-scales with the exception of “organization of materials” (p<0.05)., Conclusion: In this study, children with IGHD had poorer EF skills compared to unaffected peers. EF skills may influence academic success by affecting children’s language skills, mathematical comprehension, cognitive flexibility, and hypothetical thinking. We believe that psychiatric evaluation of children with IGHD before and during treatment may positively contribute to both their academic performance and social relationships., (©Copyright 2024 by Turkish Society for Pediatric Endocrinology and Diabetes / The Journal of Clinical Research in Pediatric Endocrinology published by Galenos Publishing House.)

Objective: Treatment of pediatric growth hormone deficiency (pGHD) with daily injection of recombinant human growth hormone (somatropin) aims to increase height velocity and improve health-related quality of life (HRQoL). The Quality of Life in Short Stature Youth (QoLISSY) questionnaire was administered in a phase 3 clinical trial that evaluated efficacy and safety of once-weekly somatrogon versus once-daily somatropin in children with pGHD (ClinicalTrials.gov no NCT02968004)., Methods: Treatment-naïve prepubertal children with pGHD received once-weekly somatrogon or once-daily somatropin for 12 months. The QoLISSY core module (physical/social/emotional subscales) was administered at baseline and 12 months after treatment initiation. QoLISSY-Parent was completed by parents/caregivers of children <7 years old and some parents/caregivers of children ≥7 years old; children ≥7 years old self-completed QoLISSY-Child., Results: Baseline characteristics were similar between treatment groups ( N  = 117). Among children <7 years old, QoLISSY-Parent total and subscale scores showed similarly improved HRQoL at 12 months relative to baseline in both treatment groups. Self-reported QoLISSY-Child total and subscale scores in children ≥7 years old indicated HRQoL improvements at 12 months that were numerically better with somatrogon than somatropin (similar results with QoLISSY-Parent in this age group). At both time points, children reported better HRQoL than perceived by their parents/caregivers., Conclusion: Treatment for 12 months with once-weekly somatrogon or once-daily somatropin resulted in comparable improvements in HRQoL among children with pGHD. Lower HRQoL perceived by parents/caregivers possibly reflect children's tendency to emphasize adaptation. These results suggest that evaluation of HRQoL could help support treatment decisions in children with pGHD treated with growth hormone.

Purpose: The aim of this study was to produce evidence on quality of life (QoL) among Italian growth hormone deficiency (GHD) children and adolescents treated with growth hormone (GH) and their parents., Methods: A survey was conducted among Italian children and adolescents aged 4-18 with a confirmed diagnosis of GHD and treated with GH therapy and their parents. The European Quality of Life 5 Dimensions 3 Level Version (EQ-5D-3L) and the Quality of Life in Short Stature Youth (QoLISSY) questionnaires were administered between May and October 2021 through the Computer-Assisted Personal Interview (CAPI) method. Results were compared with national and international reference values., Results: The survey included 142 GHD children/adolescents and their parents. The mean EQ-5D-3L score was 0.95 [standard deviation (SD) 0.09], while the mean visual analogue scale (VAS) score was 86.2 (SD 14.2); the scores are similar to those of a reference Italian population aged 18-24 of healthy subjects. As for the QoLISSY child-version, compared to the international reference values for GHD/ idiopathic short stature (ISS) patients, we found a significantly higher score for the physical domain, and lower scores for coping and treatment; compared to the specific reference values for GHD patients, our mean scores were significantly lower for all domains except the physical one. As for the parents, we found a significantly higher score for the physical domain, and a lower score for treatment; compared to reference values GHD-specific, we found lower score in the social, emotional, treatment, parental effects, and total score domains., Conclusions: Our results suggest that the generic health-related quality of life (HRQoL) in treated GHD patients is high, comparable to that of healthy people. The QoL elicited by a disease specific questionnaire is also good, and comparable with that of international reference values of GHD/ISS patients., (© 2023. The Author(s).)

Purpose: Little attention has been directed towards examining the impact of predictors on change in health-related quality of life (HRQOL) within the course of growth hormone (GH) treatment in pediatric short stature. We aimed to assess changes in HRQOL and its sociodemographic, clinical and psychosocial predictors in children and adolescents diagnosed with growth hormone deficiency (GHD), and born short for gestational age (SGA) before and 12-month after start of GH treatment from the parents' perspective. Results were compared with an untreated group with idiopathic short stature (ISS). In this prospective multicenter study, 152 parents of children/adolescents (aged 4-18 years) provided data on their children's HRQOL at baseline and at 12-month follow-up., Method: Repeated-measures multivariate analyses of covariance were performed to examine parent-reported HRQOL changes from baseline to 1-year after treatment and hierarchical linear regressions to identify the predictors of HRQOL changes., Results: Results showed that parents of children that were treated with GH report an increase in their children's HRQOL after 1 year. Changes in HRQOL were mostly explained by psychosocial predictors followed by sociodemographic and clinical variables. Specifically, the diagnosis SGA significantly predicted a greater increase in parent-reported HRQOL. Furthermore, a lower caregiving burden significantly predicted a decrease in parent-reported HRQOL., Conclusion: In conclusion, a substantial percentage of explained variance in HRQOL relates to psychosocial and sociodemographic predictors. However, there appears to be other important factors that are predictors of HRQOL, which need to be determined in large, population-based samples.

Background: Health-related quality of life (HRQOL) may improve as an additional benefit of the growth hormone treatment (GHT) in children with short stature, but this effect has not been conclusively proven., Objectives: To explore the direct effect of GHT on HRQOL in children starting GHT due to isolated or multiple GH deficiency (IGHD), acquired GH deficiency (AGHD) and Turner syndrome (TS), in comparison with untreated short stature controls in 18 UK centres., Methods: We used recognized measures of HRQOL, the PedsQL, the Strengths and Difficulties Questionnaire and Youth Life Optimism Test scales to investigate the effect of GHT at 0, 6 and 12 months in children and adolescents 6-16 years with IGHD (n = 73) and AGHD (n = 45), and 22 girls with TS. 49 children with non-GHD short stature served as the controls., Results: Children rated their HRQOL better than their parents. Those with IGHD and TS rated their overall HRQOL lower than the controls at baseline, psychosocial scores significantly lower in IGHD. After 12 months, the control and TS groups scored higher than UK norms. Those with AGHD had lowest HRQOL scores at all time points, due to poorer physical functioning. The controls showed the greatest improvement in the strength and difficulties scale. All measures evaluated, whether from child, parent or teacher showed an equal improvement over the year of GHT with no discernible direct treatment effect, despite reduced numbers in some patient groups., Conclusions: Children with short stature resulting from GHD have lower functioning than controls but HRQOL appears to improve with GHT, most likely on account of greater attention and as a result of the retest phenomenon. We were not able to demonstrate an absolute and independent effect of GHT in itself. HRQOL should not be used as a primary measure, as in adults, to determine whether children should receive GHT., (© 2019 John Wiley & Sons Ltd.)

Purpose: This study aimed to validate the disease-specific "quality of life in short stature youth (QoLISSY)-instrument" that assessesQuery the health-related quality of life (HrQoL) in German children and adolescents diagnosed as small for gestational age (SGA) in a patient and parent report., Methods: The psychometric performance of the German version of the QoLISSY questionnaire was examined in terms of reliability and validity in 65 SGA families (17 child reports/64 parent reports) and compared to the psychometric performance of the original European QoLISSY dataset of over 200 children with growth hormone deficiency and idiopathic short stature (ISS)., Results: The analysis yielded psychometrically favorable results with excellent reliability and acceptable discriminant validity. The instrument's operating characteristics were comparable to the results of the original European QoLISSY data. In the parent- as well as child report, children with SGA had lower HrQoL scores than children with ISS. Convergent validity was demonstrated by significant correlations between the QoLISSY scales and the generic KIDSCREEN-10 Index., Conclusion: Psychometric testing suggests that QoLISSY is a promising instrument to assess the HrQoL of young German people with SGA. Both versions (parent- and child report) appear to detect differences between SGA and other conditions (e.g. ISS). QoLISSY can be used in clinical studies, health service research, as well as in practice in children with SGA and their parents. For a cross-cultural application of the instrument in SGA, the tool needs be validated in sufficiently large SGA samples within respective countries.

Purpose: The Quality of Life in Short Stature Youth (QoLISSY) questionnaire is a disease-specific instrument developed to assess health-related quality of life (HrQoL) in children with short stature. While the original instrument was simultaneously developed in five European countries, this study describes the results of the Italian QoLISSY translation, cultural adaptation, and validation., Methods: Focus group discussions and a cognitive debriefing process with children (N = 12) diagnosed with growth hormone deficiency or idiopathic short stature and one parent each, as well as parents of younger children (N = 20) were conducted to examine the linguistic and content validity of the Italian version. Psychometric testing was performed using data from the subsequent field- and re-test (N = 32)., Results: The results of the qualitative testing of the Italian sample revealed comparability of content to data of the original five European countries. The following field- and re-test results were psychometrically satisfactory including good item and scale operating characteristics, sufficient evidence of reliability, and acceptable evidence of construct validity., Conclusion: In conclusion, the Italian QoLISSY HrQoL-dimensions are comparable to other European countries. The psychometric quality of the Italian QoLISSY version is satisfactory and the instrument is ready for use in Italian patients and their parents.

Background: The European Quality of Life in Short Stature Youth (QoLISSY) is a novel condition-specific instrument developed to assess health related quality of life (HrQoL) in children/adolescents with short stature from patient and parent perspectives. Study objective was to linguistically validate and psychometrically test the American-English version of the QoLISSY instrument., Methods: Upon conversion of the British-English version to American-English, content validity and acceptance of the questionnaire were examined through focus group discussions with cognitive debriefing in 28 children/adolescents with growth hormone deficiency (GHD) or idiopathic short stature (ISS) and their parents. In the subsequent field test with 51 families and a re-test with 25 families the psychometric performance of the American-English version was examined and compared with the original European dataset., Results: Pilot test results supported the suitability of the American-English version. Good internal consistency with Cronbach's Alpha ranging from 0.84 to 0.97 and high test-re-test reliabilities were observed in the field test. The QoLISSY was able to detect significant differences according to the degree of short stature with higher HrQoL for taller children. Correlations with a generic HrQoL tool support the QoLISSY's concurrent validity. The scale's operating characteristics were comparable to the original European data., Conclusion: Results support that the QoLISSY American-English version is a psychometrically sound short stature-specific instrument to assess the patient- and parent- perceived impact of short stature. The QoLISSY instrument is fit for use in clinical studies and health services research in the American-English speaking population.

Objectives: The Quality of Life in Short Stature Youth (QoLISSY) questionnaire was recently developed in five European countries to assess health-related quality of life in children and adolescents with idiopathic short stature or growth hormone deficiency from child and parent perspectives. In addition to the existing French version, a Flemish version is needed for use of QoLISSY in the Flemish speaking part of Belgium., Methods: Children (8-18 years) and their parents recruited from two Belgian paediatric endocrinology clinics completed the QoLISSY in a cross-sectional study. Cronbach's Alpha and test-retest reliability was assessed. Validity was examined by correlation with the generic KIDSCREEN questionnaire as well as by group comparisons according to diagnostic and treatment status., Results: The QoLISSY scales had an acceptable internal consistency with Cronbach's Alpha ranging from 0·80 to 0·94 (child version) and from 0·77 to 0·92 (parent version). Test-retest reliability correlation coefficients ranged from r = 0·75 to 0·89 in the child version and from r = 0·58 to 0·85 in the parent version. Moderate correlations with the generic KIDSCREEN questionnaire suggested construct validity. Differences between child groups according to child age, underlying diagnosis, and degree of height deficit were found. Correlations with the European QoLISSY were significant for all scales., Discussion: The Flemish QoLISSY instrument is a psychometrically sound, reliable, and valid short stature specific questionnaire measuring health-related quality of life. It is expected to be of great use in upcoming clinical research on growth disorders and growth hormone treatment in Belgium and Europe.

Background: Transition is a term used to describe the period of adolescence after which the final adult height during growth hormone (GH) treatment is achieved. According to re-evaluation results in insulin tolerance test (ITT) patients with severe and partial growth hormone deficiency (GHD) may be distinguished., Objectives: The aim of the study was to assess QoL in patients with different degrees of GHD in transition phase., Methods: QoL was evaluated in 76 subjects aged 16-25 years with severe (SGHD, n=26), partial GHD (PGHD, n=22) and normal GH secretion (NGH, n=28) using SF-36 v.2™ Health Survey and the Quality of Life Assessment of Growth Hormone Deficiency in Adults (QoL-AGHDA) questionnaires., Results: Physical Component Score (PCS), Physical Functioning (PF) and General Health (GH) results were significantly lower in patients with SGHD than in NGH group. SF-36 v.2™ Health Survey scores in PGHD were similar as in NGH patients. There were no statistically significant differences in QoL-AGHDA scores between the examined groups. We found positive correlations between peak GH in ITT and PF (r=0.29; p=0.02) or Role Emotional (r=0.37; p=0.002) scores., Conclusions: We demonstrated that the QoL in adolescents and young adults with severe GHD in transition period is disturbed mainly in terms of physical health and emotions. These changes were detected only by generic SF-36, but not by disease-specific QoL-AGHDA questionnaire. Therefore AGHDA-QoL assessment may not be applicable in GHD patients in transition period. QoL in the patients with partial GHD is unchanged in comparison to growth hormone sufficient subjects.

Objective: To determine differences between parents and children in ratings of child health-related quality of life (HRQL) prior to growth hormone treatment., Method: HRQL measures were collected from 144 children and their caregivers. Inclusion criteria were aged between 10 and 16 years, diagnosed with Turner's syndrome, acquired or idiopathic growth hormone deficiency (AGHD or IGHD) and eligible to begin human GH treatment (GHT), or non-growth hormone deficient (GHD) short stature., Results: Parents rated children to have poorer physical and psychosocial HRQL than children rated themselves. Differences depended on the measure used. Parents rated children with IGHD and non-GHD short stature better than children rated themselves, but they rated children with AGHD or Turner's much worse than children rated themselves in terms of physical but not psychosocial functioning., Conclusions: Decisions to prescribe GHT should include children's perspectives of HRQL whenever possible. Differences between parents and children are most likely in conditions that involve more complex medical needs (AGHD and Turner's). Generic and disease-specific HRQL measures may vary in sensitivity to HRQL differences between groups. More work is required to evaluate HRQL among younger children., (© 2013 John Wiley & Sons Ltd.)

Background: When evaluating the outcomes of treatment in paediatric endocrinology, the health-related quality of life (HrQoL) of the child is to be taken into consideration. Since few self-reported HrQoL instruments exist for children with diagnosed short stature (dSS), the objective of this study was to develop and psychometrically test a targeted HrQoL instrument for use in multinational clinical research., Methods: The target population were short stature (height<-2 SDS) children and adolescents (age 8-12 and 13-18 years) with a diagnosis of growth hormone deficiency (GHD) or idiopathic short stature (ISS), differing in growth hormone treatment status. Focus group discussions for concept and item generation, piloting of the questionnaire with cognitive debriefing, and instrument field testing with a retest were conducted simultaneously in five countries. After qualitative and preliminary quantitative analyses, psychometric testing of field test data in terms of reliability and validity including confirmatory factor analyses (CFA) was performed., Results: Following item generation from focus group discussions, 124 items were included in a pilot test with a cognitive debriefing exercise providing preliminary feedback on item and domain operating characteristics. A field test with 268 participants showed high internal consistency reliabilities (alpha 0.82-0.95), good correlations with generic measures (up to r=.58), significant known group differences (e.g. in height: F=32, df 244, p<0.001) and an acceptable CFA model fit suggesting construct validity of the three-domain core structure with 22 items, supplemented by three mediator domains with 28 items., Conclusions: The QoLISSY questionnaire is a promising step forward in assessing the impact of dSS on HrQoL. It is based on items generated from the subjective experience of short stature children referred for endocrine investigation, is validated for use in five languages and it is easy to administer in clinical and research settings.

Objective: The aim of this study was to analyze the individual impact of short stature (SS) or untreated isolated growth hormone deficiency (IGHD) on voice quality and the influence of IGHD on voice aging., Methods: A cross-sectional study was carried out on 73 adults: 33 IGHD, 10 SS, and 30 normal controls (CO), by evaluating vocal perception using Voice-Related Quality-of-Life (V-RQOL) scores and fundamental frequency (ƒ0). Analysis of variance with Bonferroni post-test was used to compare groups, and the Student t test was used to verify the influence of aging., Results: Stature of the SS and IGHD groups was similarly reduced in comparison to CO. Cephalic perimeter (CP) in SS males was larger than CO (P<0.05), and this was larger than in IGHD (P<0.0001). CP was similar in SS and CO females, and both were larger than in IGHD (P<0.0001). V-RQOL scores were lower in IGHD than in SS and CO. ƒ0 (Hz) was similar in IGHD females and SS and higher than in CO (P<0.05). f0 of IGHD males was higher than in SS (P=0.01) and CO (P=0.001). IGHD abolished the effect of aging on ƒ0 exhibited by CO., Conclusions: Lower vocal perception and higher ƒ0 were found in IGHD in comparison to CO in both genders; in comparison to SS, higher ƒ0 was only found in IGHD males. Because SS males have higher CP than IGHD, this suggests that CP and craniofacial growth can influence voice in IGHD. Finally, IGHD seems to abolish the effects of aging on voice., (Copyright © 2012 The Voice Foundation. Published by Mosby, Inc. All rights reserved.)

The growth hormone-insulin-like growth factor-1 axis plays a role in normal brain growth but little is known of the effect of growth hormone deficiency on brain structure. Children with isolated growth hormone deficiency (peak growth hormone <6.7 µg/l) and idiopathic short stature (peak growth hormone >10 µg/l) underwent cognitive assessment, diffusion tensor imaging and volumetric magnetic resonance imaging prior to commencing growth hormone treatment. Total brain, corpus callosal, hippocampal, thalamic and basal ganglia volumes were determined using Freesurfer. Fractional anisotropy (a marker of white matter structural integrity) images were aligned and tract-based spatial statistics performed. Fifteen children (mean 8.8 years of age) with isolated growth hormone deficiency [peak growth hormone <6.7 µg/l (mean 3.5 µg/l)] and 14 controls (mean 8.4 years of age) with idiopathic short stature [peak growth hormone >10 µg/l (mean 15 µg/l) and normal growth rate] were recruited. Compared with controls, children with isolated growth hormone deficiency had lower Full-Scale IQ (P < 0.01), Verbal Comprehension Index (P < 0.01), Processing Speed Index (P < 0.05) and Movement-Assessment Battery for Children (P < 0.008) scores. Verbal Comprehension Index scores correlated significantly with insulin-like growth factor-1 (P < 0.03) and insulin-like growth factor binding protein-3 (P < 0.02) standard deviation scores in isolated growth hormone deficiency. The splenium of the corpus callosum, left globus pallidum, thalamus and hippocampus (P < 0.01) were significantly smaller; and corticospinal tract (bilaterally; P < 0.045, P < 0.05) and corpus callosum (P < 0.05) fractional anisotropy were significantly lower in the isolated growth hormone deficiency group. Basal ganglia volumes and bilateral corticospinal tract fractional anisotropy correlated significantly with Movement-Assessment Battery for Children scores, and corpus callosum fractional anisotropy with Full-Scale IQ and Processing Speed Index. In patients with isolated growth hormone deficiency, white matter abnormalities in the corpus callosum and corticospinal tract, and reduced thalamic and globus pallidum volumes relate to deficits in cognitive function and motor performance. Follow-up studies that investigate the course of the structural and cognitive deficits on growth hormone treatment are now required to confirm that growth hormone deficiency impacts significantly on brain structure, cognitive function and motor performance.

Background: Quality of life (QoL) as it is related with growth hormone deficiency (GHD) is a matter of controversy., Methods: We analyzed QoL in 95 children aged 8-18 years with isolated GHD (72% male) treated with growth hormone (GH). These children were compared to 190 age- and gender-matched healthy children with similar height [height <10th percentile; control group 1 (CG1)] and age- and gender-matched 285 healthy children of normal stature (control group 2: CG2). QoL was measured by the KINDL® questionnaire referring to six domains (physical well-being, emotional well-being, self-esteem, family, friends, and school)., Results: QoL was significantly reduced in CG1 (effect-size 0.21) compared to CG2, while QoL was not significantly altered in children with GHD. In multiple linear regression analyses adjusted to age, gender, BMI, migration background, and socioeconomic status, decreasing height-SDS was associated with poorer QoL (especially emotional well-being), and treatment with GH was related significantly to better self-esteem. Increase of height-SDS in children treated with GH was associated positively with QoL and all its subscales except family and school., Conclusions: These findings suggest psychological consequences of short stature in children and an improvement of QoL in children treated with GH with the focus on self-esteem and emotional well-being., (Copyright © 2012 S. Karger AG, Basel.)

Background and Objective: Deficiency of growth hormone (GH) in adult is accompanied by disorders that can resolve with its administration. The aim of the study is to assess clinical, biochemical and HR-QoL (Nottingham Health Profile and Qol AGHDA test) response to GH replacement at long term; and to difference between childhood onset (Group A) and adult onset (Group B)., Patients and Methods: This was a retrospective observational study of 17 patients with hypopituitarism and GH deficiency, treated in our center for at least 60 months with GH. We assessed changes in biochemical parameters, anthropometric and bone mineral density (BMD). HR-QoL was assessed using Nottingham Health Profile and Qol-AGHDA questionnaires, and they were analyzed separately in two groups, based on whether the diagnosis was made during childhood (Group A) or it was adult-onset (Group B)., Results: We observed a significant increase in fasting blood glucose at the first year, which was maintained at 5 years. BMD remained unmodified at the first year but increased after 5 years of treatment with GH. In the cluster analysis, results were similar. Regarding the assessment of HRQL, Group A presented no improvement in the Qol-AGHDA or evaluated areas of the NHP, but in group B a significant improvement at first year in the Qol-AGHDA and in area of energy and emotion in the NHP test that persisted at 5 years (p<0.05) were observed., Conclusions: Replacement therapy with GH produces long-term beneficial effects on BMD and HRQL, with good long-term tolerance without remarkable side effects., (Copyright © 2010 Elsevier España, S.L. All rights reserved.)

Psychosocial dwarfism is a rare condition, but can still be observed. We report the case of a 5-year-old girl seen in our clinic for severe growth retardation that had been evolving for several years. A growth arrest was observed beginning at the age of 3.5years. Initial clinical examination and biological investigations were negative. As the child was hospitalized, serious disturbances in mother-child relations were observed. A significant modification of the child's behavior was observed when the mother was absent. Following questioning, the mother admitted social problems; the pregnancy had not been desired and this contributed to a lack of attachment between the mother and the child. Psychosocial dwarfism seems to originate from serious disturbances in the mother-child relationship. It seems to be caused by a partial growth hormone deficiency and a frequent context of malnutrition due to food behavior disorders. Family problems need to be taken into account and intensive medical and psychological follow-up is required. Prognosis is good if an early diagnosis is made and followed with a close and prolonged follow-up., (Copyright 2010 Elsevier Masson SAS. All rights reserved.)

Objective: Reduction in health-related quality of life is common in children born small for gestational age (SGA) or children with growth hormone deficiency (GHD). Growth hormone treatment with somatropin in these children leads to normalisation of height. The aim of this study was to determine whether somatropin is a cost-effective treatment option for short children born SGA and GHD children in Sweden., Methods: A Markov decision-tree model was used to calculate the relative costs and health benefits associated with somatropin treatment over the lifetime of SGA and GHD children, compared with no treatment. The analysis was undertaken from a Swedish Health Service perspective. As quality-adjusted life-year (QALY) data were not obtained directly in the clinical studies, a degree of uncertainty is related to these results. Sensitivity analyses assessed the degree of uncertainty surrounding central parameters., Results: For short children born SGA, somatropin treatment was associated with an additional 3.29 QALYs at an incremental cost of 792,489 SEK (Swedish Krona), compared with no treatment. For GHD, somatropin treatment resulted in 3.25 additional QALYs at an incremental cost of 391,291 SEK. This equates to an incremental cost per QALY of 240,831 SEK and 120,494 SEK for SGA and GHD, respectively, below a cost-effectiveness threshold of 500,000-600,000 SEK/QALY., Conclusions: Somatropin is a cost-effective treatment strategy in Sweden for children with GHD and SGA. To overcome present study limitations future clinical research should incorporate appropriate quality of life questionnaires.

Objectives: A great similarity exists between growth hormone (GH) deficiency and obesity in terms of disturbances of organ morphology and function. The aim of the study was to compare health-related quality of life (HR-QoL) as well as exercise capacity and its subjective assessment in adult patients with GH deficiency and in adult patients with obesity., Methods: Ten (10) GH-deficient, thirty (30) obese, and thirty (30) healthy subjects participated in the study. HR-QoL comprised two parameters: QoL measured by using the Quality of Life Assessment of Growth Hormone Deficiency in Adults (QoL-AGHDA) questionnaire, and subjective evaluation of general health state by using the Visual Analogue Scale. The exercise capacity was determined in Six Minute Walking Test and it was subjectively assessed by Borg Scale for Rating Perceived Exertion and the modified Medical Research Council scale., Results: Decreased HR-QoL (both parameters) was observed in both GH-deficient and obese patients, with that effect being much more pronounced in the former group. Both, GH-deficient and obese patients, revealed decreased exercise capacity, which was also subjectively assessed as decreased, especially by GH-deficient patients. Positive relationships between HR-QoL and exercise capacity or its subjective assessment, observed in healthy subjects, partially lost their significance in obese, whereas they completely disappeared in GH-deficient subjects., Conclusion: A decrease in HR-QoL is more pronounced in GH-deficient than in obese patients, whereas exercise capacity is unfavourably affected by both disorder to a similar extent, with the lack of clear relationship between these two parameters especially in GH-deficient patients.

Impaired quality of life (QoL) is commonly described as being associated with growth hormone (GH) deficiency (GHD), and beneficial effects of GH replacement therapy on QoL have been reported. However, most studies examined heterogeneous cohorts of patients GHD of varying etiologies, severities and age of onset. Most of these patients miss other pituitary hormones, whose replacement can also influence QoL. We studied the QoL of a homogeneous cohort of 20 adults with isolated GH deficiency (IGHD) due to the same mutation in the GH-releasing hormone receptor gene (IGHD, 10 men) using the Life Satisfaction Hypopituitarism Module (QLS-H), and compared them with 20 matched controls residing in the same community (CO, 10 men). Additionally, the IGHD group was evaluated after 6 months of treatment with bi-monthly depot GH, and after 12 months from its interruption. There was no difference in the total score of QoL (TSQoL) or in any of the nine categories that composes the questionnaire between IGHD and CO. Similar results were obtained when data were analyzed by sex. GH treatment only increased satisfaction with physical endurance, but did not cause an increase in the TSQoL. We conclude that in this unique population congenital, untreated, lifetime IGHD does not reduce QoL, and treatment with GH for 6 months only causes improvement in satisfaction with physical resistance.

Objective: The impact of the aetiology of childhood-onset GH deficiency (CO-GHD) on the clinical presentation during adulthood and the response to GH replacement has been poorly defined. Our study aims to characterize CO-GHD in adults due to different aetiologies and evaluate the effect of 2 years of GH replacement therapy., Design and Methods: Data from 353 adults with CO-GHD from Pfizer International Metabolic Database KIMS were retrospectively grouped according to GHD aetiology: non-organic disorder (n=147), organic pituitary disease (n=159), and brain tumour (n=47). Extent of pituitary dysfunction, IGF-I concentration, lipid concentrations and quality-of-life (QoL) were assessed at baseline and after 2 years of GH replacement., Results: GHD was diagnosed at a later age in the organic pituitary group than in the other groups, resulting in a shorter duration of GH treatment during childhood. However, the final height was greater in the organic pituitary group. Panhypopituitarism was most common in the non-organic disorder and in the organic pituitary groups, while isolated GHD was more prominent in the brain tumour group. Serum IGF-I levels were the lowest in the non-organic group. QoL was the poorest in the brain tumour group. Lipid profile and QoL improved significantly during GH replacement., Conclusion: The adverse consequences of CO-GHD in adulthood vary between aetiologies, but improve similarly with GH treatment. It is, therefore, important to consider retesting all patients with CO-GHD in early adulthood and, if persistent severe GHD is confirmed, recommence GH replacement.

Quality of life (QoL) has emerged as an end point in the evaluation of adults with growth hormone deficiency and acromegaly. QoL is measured with questionnaires designed to be used in general population or any kind of disease (generic) or aimed at the specific dimensions affected in a determined condition; these latter ones are more likely to identify the impairments caused by the underlying disease and the benefits of treatment. QoL, which is severely impaired in adults with growth hormone deficiency, improves and normalizes after growth hormone replacement therapy and this effect is maintained over several years. Acromegalic patients also exhibit severe impairment of QoL, which despite improvement after successful therapy still remains below the reference values of normal population. QoL in these chronic endocrine diseases can be used as an measure for clinical and therapeutic evaluation.

Background: We report the health-related quality of life (QOL) of survivors of childhood cancer (acute lymphoblastic leukaemia, ALL, or central nervous system, CNS, tumour), and whether or not they had growth hormone deficiency (GHD) requiring growth hormone treatment (GHT)., Method: We assessed 77 survivors of childhood ALL (n = 51) or CNS tumours (n = 26), aged between 8-18 years, and free from disease for > or = 4 years. Survivors and their mothers independently rated survivors' QOL, and mothers completed semi-structured interviews to determine their views of the benefits and disadvantages of GHT., Results: Survivors, especially those treated for a CNS tumour, reported poorer QOL compared with UK population norms. Although survivors of ALL reported better QOL than survivors of CNS tumours, there were no differences depending on whether or not they were prescribed GHT. However, mothers reported that those prescribed GHT had worse QOL than those not. All but 2 survivors were responsible for their own injections. A minority of mothers were disappointed with the child's rate of growth, and reported that children experienced pain with injections., Conclusion: We conclude that QOL in survivors of childhood cancer is compromised compared with the normal population, especially following CNS tumours. Longitudinal studies are vital to determine whether GHT can contribute to improved QOL for cancer survivors, especially those who experience more intensive initial therapy regimes., (Copyright 2005 S. Karger AG, Basel.)

The present study evaluates the effects of one year of discontinuation and one year of growth hormone (GH) treatment on quality of life (QoL) in young adults with childhood-onset growth hormone deficiency (CO-GHD). Twenty-two subjects (14 males, 8 females; 11 isolated growth hormone deficient [IGHD], 11 multiple pituitary hormone deficient [MPHD]), aged between 15 and 22 years, on ongoing GH treatment were assessed during one year of discontinuation. Thereafter, 9 of these patients, who were found to be still GH deficient (GHD), added by 11 newly recruited GHD patients who also were not treated in the preceding year (in total 10 males and 10 females, aged between 17 and 27, 5 IGHD, 15 MPHD), restarted GH treatment for one year. During discontinuation and restart of GH treatment somatic and psychological assessments took place every 6 months. In the first 6 months of the GH discontinuation period insulin-like growth factor I (IGF-I) level significantly declined whereas no further decrease in IGF-I was seen after month 6. The number of psychological complaints and depression increased only during the first 6 months of discontinuation. Across the 12-month of discontinuation tension increased in MPHD and decreased in IGHD patients. Only in the first 6 months of GH treatment IGF-I level increased, anxiety decreased and QoL improved. Depression scores tended to decrease across the 12 month treatment period. During the 2-year discontinuation and treatment period intra-subject IGF-I level was negatively correlated with depression, fatigue, tension and anxiety and positively with vigor and memory. At the end of the treatment period all psychometric parameters were similar or even improved compared to those at the start of the discontinuation period. It is concluded that one year discontinuation of GH treatment leads to a decrease in QoL within 6 months which effect is counteracted within 6 months after restart of GH treatment.

This study was done to evaluate the perception of body image and psychosocial attitudes in a sample of 8 male adult subjects with growth-hormone deficit before and after six months of therapy. Before treatment the subjects tended to underestimate body size and were characterized by low self-esteem, a closed attitude towards social relationships, a pessimistic attitude with a tendency towards depression, and a strong sense of detachment from the outside world. After the treatment, even if the subjects continued to underestimate body size, we observed an improvement in terms of emotional control and well-being, accompanied by lower scores on anxiety. An integration of a psychological support in the GH therapy could help the patients to overcome problems of bodily and social acceptance.

Ninety-six adolescents referred to a pediatric endocrinology clinic were divided into eight groups according to degree of sexual maturity and height. Each adolescent was assessed by a psychiatric interview, a self-concept questionnaire, a human figure drawing test and a cognitive screening battery. The results showed a definite deleterious effect of growth retardation, but not sexual maturity, on self-concept. Sex of the adolescent did not affect the results. Cognitively there was no difference between groups. The psychological impact of short stature should be taken into consideration in the decision to utilize pharmacological or delay of puberty.

The syndrome of reversible somatotropin deficiency (psychosocial dwarfism) is characterized by environment-related impairment of stature and mental growth and growth-hormone secretion. The syndrome is well illustrated by this case report of an eight-year-old boy with psychosocial dwarfism since the third year of life. It was undetected for four years, despite his having been in regular contact with a number of health workers. After he was removed from the deleterious environment, both his growth and behaviour improved markedly, and at follow-up he had maintained this progress.

2 case reports regarding patients with a psychosocial dwarfism. Both children showed clinical and endocrinological signs of "idiopathic" hypopituitarism. Both patients came from economically well situated families of the upper middle-class. Disturbances in eating and sleeping behaviour are important hints that there might be a psychosocial etiology.

The authors report the results of treatment with human growth hormone (hGH) (for 3 1/2 years to 11 3/4 years, mean: 5 years 4 months) in 34 children presenting with complete growth hormone deficiency and an average growth failure reaching 3.9 standard deviations (SD). Treatment was discontinued, after spontaneous or induced puberty, when bone age reached or was beyond 15 years in boys, and 13 years in girls. A partial correction was obtained in almost all cases. However, average height remained at -2.4 SD with respect to age and -1.75 with respect to bone age, reaching normal limits in only half of the patients. School and professional performances were not appreciably different from those which are observed in normal adolescents. Although the acceptance of the diagnosis and therapy was not always good, the medical support associated with the supervision of the treatment seems to have had a favourable psychological effect.

A recent study by Holmes et al. (1982) revealed a high degree (23%) of grade retention in a group of children with significant short stature secondary to growth hormone deficiency (GHD), constitutional delay (CD); or Turner's Syndrome (TS). Parents indicated in a free response format that 'immaturity' and 'small size' were the reasons for retention. The present follow-up study obtained academic achievement data on 47 of the 56 short children from the Holmes et al. (1982) study to assess what role academic factors, not spontaneously mentioned by parents, may have had in the retention decision. Results indicated that despite repeating a year in the primary grades, the group of retained children were still functioning 6 months below grade expectation, compared to grade appropriate achievement in the never-retained group. Parents and teachers were both accurate in their perceptions of children's academic achievement. Behavioural ratings by teachers indicated more adjustment difficulties for retained children, while parent ratings of behaviour showed a similar high level of problems for short children regardless of retention status. Although both groups of children possessed average intellectual abilities, the retained children obtained lower IQ scores than the never retained children. In summary, a majority of short children were achieving appropriate grade levels, but a substantial minority were experiencing under-achievement, behaviour problems, and grade retention, despite average intelligence.

Fifteen patients (4 females and 11 males) with hypothalamo-hypopituitary dwarfism underwent extensive psychiatric investigation in 1962-1965. A follow-up study of the personality development and social conditions was made in 1977. The age of the patients ranged from 31 to 56 years (the average being 40). As before, the main finding was an infantile personality with a defective psychosexual maturity. Only a few patients had reached a somehat adult, independent personality in spite of hormonal deficiencies. Several patients had continued to grow and attained a stature slightly below average. However, this subsequent growth scarcely influenced personality development. Depressive moods are now more frequent than before and among the more differentiated patients, the neurotic symptoms are mainly phobic fears. Almost all of the patients have discontinued treatment with hormonal substitutes (androgenes, cortison, thyrotropic hormone) inspite of persistent deficiency symptoms, because the outcome did not match their high expectations. The symptoms of the endocrine psychosyndrome seem of minor importance compared with the psychic infantilism and the reactions to the experience of dwarfism and missing puberty. One female has suffered several psychotic episodes which were understood as being partially of endocrine origin.

IQ change in association with change of environment occurred in a sample of 34 patients with a diagnosis of abuse dwarfism. Low and persistent impairment of IQ was associated with abuse. By contrast, IQ elevation was associated with rescue. Multiple regression analysis revealed that duration of rescue was the primary variable associated with IQ elevation; age and IQ level at rescue were secondary. IQ elevation was gradual and progressive over the years, as rescue was maintained. In abuse, the mean IQ was 66 (range, 36-101), and after rescue it was 90 (range 48-133). The greatest magnitude of change, from IQ 36 to 120, was in a girl between the ages of 3 yr 8 months and 13 yr 11 months.

The research on the psychosocial development of children and adolescents with growth-retardation due to idiopathic hypopituitarism is reviewed. The results of this--scarce and not very recent--research appear to be far from consistent. There is disagreement, both as to the nature and to the seriousness of the psychosocial implications of the growth-retardation. Some research-workers stress the existence of psychological and social problems of a very serious nature and are convinced of the necessity of intensive psychotherapeutic help. The presence of such problems is denied by others and only occasionally some support should be needed. All authors agree, however, about adolescence being a psychosocially critical period. Furthermore in this review, the methodology of previous research is criticized.

Eleven children with documented growth hormone deficiency were studied to assess their cognitive and emotional functioning and their academic achievement before and after 1 year of human growth hormone replacement therapy. Standardized personality and intelligence measures were used, and records of school achievement, as well as developmental and family history from parents, were obtained. Although some subjects in this group were coping with problems other than their short stature (e.g., other medical and psychosocial problems), their intelligence and academic achievement were found to be positively correlated with socioeconomic status and independent of the condition of hypopituitarism. This study supports others that have found these children vulnerable to problems of emotional adjustment in middle and late childhood. Because of the heterogeneity of the group, findings must be interpreted cautiously. However, the consistent finding of disturbance in visual-motor integration is thought to be significant and demonstrates the need for further research that will examine possible neurological sequelae of long-term growth hormone deficiency.