[Quality of Life and Experienced Distress of Patients Suspected of having a Rare (Chronic) Health Condition - Initial Findings from the ZSE-DUO Study]. / Lebensqualität und erlebte Belastungen von Patient*innen mit Verdacht auf eine Seltene Erkrankung ­ erste Erkenntnisse aus der ZSE-DUO Studie.

OBJECTIVE: Patients suspected of having a rare (chronic) health condition have often gone a long way within the healthcare system. To date, little is known about the health-related quality of life of this group of patients. The study aims to describe the health-related quality of life and the perceived distress of patients suspected of having a rare (chronic) health condition and compare the results with standard values of the German population. METHODS: Eighty patients suspected of having a rare (chronic) health condition were recruited in the nationwide intervention study "ZSE-DUO" and reported their health-related quality of life and perceived distress using the SF-8 and the Distress-Thermometer. RESULTS: The patients rated all eight dimensions of quality of life as well as the physical and mental component scores of the SF-8 significantly lower than the general population. On average, the perceived distress was rated significantly higher. More than 90% of the sample indicated distress in the clinical range. Exhaustion, pain, limited mobility as well as worries and fears were mentioned most frequently as concrete problems, with percentages ranging from 73% to 90% of the total sample. DISCUSSION: In comparison to German reference data, patients suspected of having a rare (chronic) health condition report a massive impairment of their quality of life and a high burden, which is especially characterized by physical and emotional problems. The lack of a diagnosis could explain the high proportion of emotional problems, as it can create a form of legitimation of one's own disease experience. CONCLUSION: The present results underline the need for research on the psychosocial impact of the possible presence of a rare (chronic) health condition. The high distress and the impact on the physical and psychological quality of life domains also highlight the need for care in this patient group.

Psychometric properties of the quality of life in short statured youth (QoLISSY) questionnaire within the course of growth hormone treatment.

BACKGROUND: The Quality of Life of Short Stature Youth (QoLISSY) questionnaire is a patient- and parent-reported outcome measure assessing health-related quality of life (HRQOL) in short stature youth. This study evaluates the psychometric properties of the QoLISSY questionnaire within a German prospective trial of short statured children treated with human growth hormone (hGH). METHOD: The instrument was administered to children with idiopathic growth hormone Deficiency (IGHD) and small for gestational age (SGA) before and after 12 month of hGH treatment. Children with idiopathic short stature (ISS) served as a reference group receiving no treatment. Psychometric testing included scale distribution characteristics, reliability (internal consistency), criterion-and convergent validity (correlations with the generic KIDSCREEN-Index, inter-correlations among QOLISSY subscales), known-group validity (treatment status, height SDS), and responsiveness analysis (ability to detect change). RESULTS: One hundred fifty-two parents and 66 children/adolescents completed both HRQOL assessments. The QoLISSY demonstrated good reliability with Cronbach's alpha > .70. Moderate significant correlations between QoLISSY domains and the KIDSCREEN-10 Index supported criterion validity. Statistically significant differences in HRQOL were observed between treatment groups at baseline with children who were about to start treatment reporting a significantly lower HRQOL compared to the children who will not receive treatment. No significant differences were found between the level of short stature based on height SDS scores (≤ - 2 SDS, > - 2 SDS). Furthermore, the instrument detected significant changes in HRQOL between the treated and the untreated group in patient-reports. CONCLUSIONS: In conclusion, the scales showed satisfactory reliability, adequate validity and ability to detect change in self-reported HRQOL within GH treatment. Findings support QoLISSY's further use in clinical trials, offering the opportunity to adequately assess HRQOL from the patients' and caregivers' perspective to improve patient-centered care.

Cross-cultural selection and validation of instruments to assess patient-reported outcomes in children and adolescents with achondroplasia.

PURPOSE: Achondroplasia, as the most common form of disproportionate short stature, potentially impacts the health-related quality of life (HRQOL) and functioning of people with this condition. Because there are no psychometrically validated patient-reported outcome (PRO) condition-specific instruments for achondroplasia, this study selected and tested available generic, disease-specific and under development questionnaires for possible use in multinational clinical research. METHODS: A three-step approach was applied. First, a literature review and clinician/expert opinions were used to select relevant PRO questionnaires. Second, focus group discussions, including a group cognitive debriefing for piloting of the questionnaires with children/adolescents with achondroplasia and their parents, were performed in Spain and Germany. Third, a field-test study was conducted to test the psychometric properties of these instruments. RESULTS: Six questionnaires were identified as potentially relevant in children with achondroplasia. In each country, five focus groups including a cognitive debriefing were conducted, and the results narrowed the possibilities to three instruments as most appropriate to assess HRQOL (the generic PedsQL, the height-specific QoLISSY, and the achondroplasia-specific APLES). Results of the field study indicate the QoLISSY and the PedsQL questionnaires to be most appropriate for use in clinical research at this time. CONCLUSION: This selection study is a step forward in assessing the impact of achondroplasia on HRQOL. Of the instruments examined, the QoLISSY and the PedsQL both capture items relevant to children with achondroplasia and have met the psychometric validation criteria needed for use in research. The APLES instrument is a promising tool that should be revisited upon psychometric validation.

Agreement between mothers', fathers', and children's' ratings on health-related quality of life in children born with esophageal atresia - a German cross-sectional study.

BACKGROUND: Esophageal atresia (EA) is a rare congenital malformation, which is characterized by the discontinuity of the esophagus. We investigated the agreement between mothers', fathers', and children's' ratings on health-related quality of life (HRQOL) in children born with EA. We aimed to broaden the understanding of subjective experiences of HRQOL from different perspectives. We hypothesized that the agreement between mother and father ratings would be high, whereas the agreement between child and mother ratings as well as child and father ratings would show more substantial differences. METHODS: We obtained data from 40 families (23 mother-father dyads of children aged 2-7 years and 17 mother-father-child triads of children and adolescents aged 8-18 years) with children born with EA, who were treated in two German hospitals. HRQOL was measured using the generic PedsQL™ questionnaires and the condition-specific EA-QOL© questionnaires. We calculated intraclass coefficients and performed one-way repeated measures ANOVAs to analyze differences for each domain as well as for the total scores. RESULTS: Intraclass correlation coefficients (ICCs) indicated a strong agreement (≥.80) between mother and father reports of children's HRQOL for both generic and condition-specific measurements. The ICCs for the generic HRQOL for mother/father-child-dyads revealed only fair to good agreement, whereas ICCs for condition-specific HRQOL showed high agreement for mother-child and father-child-agreement. Analyses of Covariance revealed differences in mother/father-child agreement in the generic domain School, both parents reporting lower HRQOL scores than the children themselves. Fathers reported significantly higher scores in the condition-specific domain Social than their children. CONCLUSIONS: Results showed that mothers' and fathers' reports corresponded to each other. Nonetheless, these reports might not be interchangeably used because mother-child and father-child agreement showed differences. Children might know the best on how they feel, and parent proxy-report is recommended when reasons such as young age, illness, or cognitive impairments do not allow to ask the child. But parent-report - no matter if reported by mother or father - should only be an additional source to broaden the view on the child's health status and well-being. The current study contributes to a better understanding of the complex family relationships involved when parenting a child born with EA.

Health-related quality of life experiences in children and adolescents born with esophageal atresia: A Swedish-German focus group study.

BACKGROUND: Esophageal atresia (EA) is a rare malformation of the esophagus, which needs surgical treatment. Survival rates have reached 95%, but esophageal and respiratory morbidity during childhood is frequent. Child and parent perspectives and cultural and age-specific approaches are fundamental in understanding children's health-related quality of life (HRQoL) and when developing a pediatric HRQoL questionnaire. We aimed to increase the conceptual and cross-cultural understanding of condition-specific HRQoL experiences among EA children from Sweden and Germany and investigate content validity for an EA-specific HRQoL questionnaire. METHODS: Eighteen standardized focus groups (FGs) with 51 families of EA children aged 2-17 years in Sweden (n = 30 families) and Germany (n = 21 families) were used to explore HRQoL experiences, which were content analyzed into HRQoL domains. The Swedish HRQoL domains were analyzed first and used as framework to evaluate HRQoL content reported in the German FGs. HRQoL experiences were then categorized as physical, social, and emotional HRQoL burden or resource. RESULTS: One thousand nine hundred eight HRQoL statements were recorded. All nine EA-specific HRQoL domains identified in the Swedish FGs (eating, social relationships, general life issues, communication, body issues, bothersome symptoms, confidence, impact of medical treatment, and additional difficulties due to concomitant anomalies) were recognized in the FGs held in Germany, and no additional EA-specific HRQoL domain was found. The HRQoL dimensions referenced physical burden (n = 655, 34.5%), social burden (n = 497, 26.0%), social resources (n = 303, 15.9%), emotional burden (n = 210, 11.0%), physical resources (n = 158, 8.3%), and emotional resources (n = 85, 4.5%). CONCLUSION: This first international FG study to obtain the EA child and his or her parents' perspective on HRQoL suggests Swedish-German qualitative comparability of the HRQoL domains and content validity for a cross-cultural EA-specific HRQoL questionnaire. EA children make positive and negative HRQoL experiences, but prominently related to physical and social burden, which underlines appropriate follow-up care and future research.

Exploring the Potential of a Pretend Play Intervention in Young Patients With Leukemia.

PURPOSE: The aims of the study are 1) to gain knowledge of parents' and professionals' perceptions about cancer stricken children's resources, burdens, and ability to pretend play, and 2) to prepare the initiation of a pretend play intervention based on children's needs and included parents' and professionals' feedback. DESIGN AND METHODS: Qualitative design using semi-structured interviews with 13 parents of children diagnosed with leukemia and 15 professionals in the field of pediatric oncology. Themes were derived with content analysis via deductive and inductive coding. RESULTS: Analysis resulted in five topics. (1) Ability to play in the context of leukemia (2) ways of coping with leukemia (3) difficulty in transition to normality (4) parental quality of life and parents' needs (5) perceptions of the potential of pretend play. CONCLUSION: Study results indicate the potential of pretend play interventions for young cancer patients and the need for additional professional support of parents. PRACTICE IMPLICATIONS: Pretend play is a tool children carry with them regardless of their circumstances. If we can enhance their ability to play, doing so should give them an advantage in creative problem solving and creative expression as they deal with a life threatening disease.

The Esophageal-Atresia-Quality-of-life Questionnaires: Feasibility, Validity and Reliability in Sweden and Germany.

OBJECTIVES: Esophageal atresia (EA) is a rare malformation characterized of discontinuity of the esophagus, concurrent with or without a tracheoesophageal fistula (TEF). We report the feasibility validity and reliability of a condition-specific quality-of-life (QOL) tool for EA/TEF children, the age-adapted EA-QOL-questionnaires, when used in Sweden and Germany. METHODS: A total of 124 families of children with EA/TEF participated in the study; 53 parents completed the EA-QOL-questionnaire for children aged 2 to 7 years; 62 children/71 parents the EA-QOL-questionnaire for children 8 to 17 years. Feasibility was determined from the percentage of missing item responses. Based on clinical data and previously validated generic QOL-instruments (PedsQL 4.0, DISABKIDS-12), the final EA-QOL scores were evaluated against hypotheses of validity (known-groups/concurrent/convergent) and reliability (internal consistency/retest reliability of scores for 3 weeks). Significant level was P < 0.05. RESULTS: In the questionnaire for EA/TEF children aged 2 to 7 years, 16/18 items were completed with missing values <6% (range 0%-7.5%), and in the questionnaire for 8 to 17-year-olds, 24/24 child-reported items (range 0%-4.8%) and 21/24 parent-reported items (range 0%-7.0%). In both age-specific EA-QOL-questionnaires, desirable standards for known-groups and concurrent validity were fulfilled; digestive symptoms and feeding difficulties negatively impacted EA-QOL-Total-scores (P < 0.001), and as hypothesized, in 2 to 7-year-olds, respiratory symptoms decreased EA-QOL-Total-scores (P = 0.002). Correlations between the EA-QOL and generic QOL questionnaires supported convergent validity. Internal consistency reliability was satisfactory. The level of agreements of EA-QOL-scores between the field- and retest study were good to excellent. CONCLUSIONS: The overall psychometric performance of the EA-QOL-questionnaires for EA/TEF children is satisfactory and can enhance outcome evaluations in future research and clinical practice.

Children's psychosocial functioning and parents' quality of life in paediatric short stature: The mediating role of caregiving stress.

OBJECTIVES: On the basis of the multidimensional model of the caregiving process, this study aimed (a) to compare the levels of quality of life (QoL) and psychological problems of children with short stature and the levels of caregiving stress and QoL of their parents, between diagnostic, treatment, and current height deviation groups, and (b) to examine the direct and indirect links, via caregiving stress, between children's psychosocial functioning and their parents' QoL. METHOD: The sample was collected in 5 European countries and comprised 238 dyads of 8- to 18-year-old children and adolescents with a clinical diagnosis of growth hormone deficiency or idiopathic short stature and one of their parents. The children completed self-report measures of height-related QoL (Quality of Life in Short Stature Youth Core Module) and psychological problems (Strengths and Difficulties Questionnaire); the parents reported on their own QoL (EUROHIS-QOL-8 Index) and caregiving stress (Quality of Life in Short Stature Youth Effects on Parents subscale). RESULTS: Children who were treated and who achieved normal height reported better QoL compared to those untreated and with current short stature. Parents of children with idiopathic short stature and current short stature presented greater caregiving stress than parents of children with growth hormone deficiency and achieved normal height. Children's better psychosocial functioning was indirectly associated with parents' better QoL, via less caregiving stress, and these links were invariant across diagnoses, treatment status, and current height deviation. CONCLUSIONS: These results suggest that, along with growth hormone treatments, multidisciplinary interventions in paediatric endocrinology should be family-centred, by targeting both the children's psychosocial functioning and the parents' stress, in order to improve individual and family adaptation.

Understanding, Assessing and Improving Health-Related Quality of Life of Young People with Achondroplasia- A Collaboration between a Patient Organization and Academic Medicine.

Achondroplasia (ACH) is a rare, genetically determined health condition. Patients suffer from disproportional short stature and multiple physical and functional impairments as well as socioemotional problems. Despite the burden of disease, only few studies focus on health-related quality of life (HrQoL) of young ACH patients. In a series of studies, the BKMF e.V. in cooperation with the UKE studied ACH patients' and parents' experience of HrQoL, their responses to HrQoL questionnaires and their evaluation of a HrQoL based intervention. Both qualitative and quantitative approaches were used. Psychometrically appropriate instruments were identified and the wellbeing of young patients with ACH was analyzed showing no difference from a healthy norm sample using generic instruments. However, disease-specific instruments showed discrepancies between patients with proportional and disproportional short stature. Still, results show a significant effect by age and the evaluation of the counselling concept reveals that young ACH-patients especially benefit from such intervention.

Experiencing health-related quality of life in paediatric short stature - a cross-cultural analysis of statements from patients and parents.

OBJECTIVES: Direct assessment of the patient perspective is necessary to thoroughly understand patients' experiences of disease. We aimed to examine information from children with short stature on their perceived HrQoL within 5 European countries. METHODS: Patients, identified through clinical databases, were approached by their clinicians according to the inclusion criteria regarding a diagnosis of growth hormone deficiency or idiopathic short stature and age requirements. A focus group methodology was applied in 84 children and 112 parents. Based on a category system, individual statements were allocated to domains. To evaluate the emerging topics, qualitative content analysis was conducted. Domains and respective coding frequencies per category were compared across countries and respondents. RESULTS: The highest number of statements produced by the children and parents were related to social (29%) and emotional needs and concerns (28%). In particular, children stressed their experience of social exclusion but also their perception of social support. Regarding emotional needs, they stated mainly the desire to be taller in order to be less teased by peers. National differences were identified, for example, Swedish (22%) and British (16%) children and their parents (Sweden 26%; Britain 23%) stressed physical HrQoL aspects, whereas German children (21%) strongly focused on treatment aspects, mainly the benefit of treatment. CONCLUSION: Comprehensive knowledge of the impact of a chronic condition such as short stature on wellbeing is an important precondition of effective treatment. Because socioemotional topics were rated in all the countries to be most important, interventions aimed at improving HrQoL should target social and emotional responses to short stature.

Explaining parent-child (dis)agreement in generic and short stature-specific health-related quality of life reports: do family and social relationships matter?

BACKGROUND: In the context of health-related quality of life (HrQoL) assessment in pediatric short stature, the present study aimed to examine the levels of agreement/disagreement between parents' and children's reports of generic and condition-specific HrQoL, and to identify socio-demographic, clinical and psychosocial variables associated with the extent and direction of parent-child discrepancies. METHODS: This study was part of the retest phase of the QoLISSY project, which was a multicenter study conducted simultaneously in France, Germany, Spain, Sweden and UK. The sample comprised 137 dyads of children/adolescents between 8 and 18 years of age, diagnosed with growth hormone deficiency (GHD) or idiopathic short stature (ISS), and one of their parents. The participants completed child- and parent-reported questionnaires on generic (KIDSCREEN-10 Index) and condition-specific HrQoL (QoLISSY Core Module). Children/adolescents also reported on social support (Oslo 3-items Social Support Scale) and parents assessed the parent-child relationships (Parental Role subscale of the Social Adjustment Scale) and burden of short stature on parents (QoLISSY- additional module). RESULTS: The parent-child agreement on reported HrQoL was strong (intraclass correlation coefficients between .59 and .80). The rates of parent-child discrepancies were 61.5 % for generic and 35.2 % for condition-specific HrQoL, with the parents being more prone to report lower generic (42.3 %) and condition-specific HrQoL (23.7 %) than their children. The extent of discrepancies was better explained by family and social relationships than by clinical and socio-demographic variables: poorer parent-child relationships and better children's social support were associated with larger discrepancies in generic HrQoL, while more parental burden was associated with larger discrepancies in condition-specific HrQoL reports. Regarding the direction of discrepancies, higher parental burden was significantly associated with parents' underrating, and better children's social support was significantly associated with parents' overrating of condition-specific HrQoL. CONCLUSIONS: Routine assessment of pediatric HrQoL in healthcare and research contexts should include child- and parent-reported data as complementary sources of information, and also consider the family and social context.

Psychometric performance of the Quality of Life in Short Stature Youth (QoLISSY) questionnaire in the Netherlands.

UNLABELLED: The European Quality of Life in Short Stature Youth (QoLISSY) questionnaire is a disease-specific instrument assessing quality of life (QoL) in children with short stature from the child and parent perspectives. In order to use the QoLISSY in Dutch samples, a translation process and psychometric testing is needed. Children diagnosed with short stature (8 to 18 years) and their parents were recruited from a Dutch growth clinic. Reliability was assessed using Cronbach's α and intraclass correlation coefficients (ICCs). Pearsons' correlations with the generic KIDSCREEN and a confirmatory factor analysis (CFA) were performed to test validity. Scales showed good internal consistency with α ranging from 0.80 to 0.94 (child report) and from 0.85 to 0.95 (parent report). Test-retest reliability (ICC) ranged from 0.15 to 0.91 (child report) and from 0.14 to 0.83 (parent report). Correlations with the KIDSCREEN in the mean range indicated criterion validity. The models' goodness of fit was confirmed by CFA results in the Dutch and in comparison with the European sample. CONCLUSION: The Dutch QoLISSY is a psychometrically reliable and valid short stature-specific QoL measure. It is now available for use in clinical research and practice to evaluate well-being and possible effects of growth hormone treatment and psychological interventions in the Netherlands.

Stunted Growth. Proceedings of the 23rd Aschauer Soiree, Held at Aschauhof, Germany, November 7th 2015.

Twenty-four scientists met at Aschauhof, Altenhof, Germany, to discuss the associations between child growth and development, and nutrition, health, environment and psychology. Meta-analyses of body height, height variability and household inequality, in historic and modern growth studies published since 1794, highlighting the enormously flexible patterns of child and adolescent height and weight increments throughout history which do not only depend on genetics, prenatal development, nutrition, health, and economic circumstances, but reflect social interactions. A Quality of Life in Short Stature Youth Questionnaire was presented to cross-culturally assess health-related quality of life in children. Changes of child body proportions in recent history, the relation between height and longevity in historic Dutch samples and also measures of body height in skeletal remains belonged to the topics of this meeting. Bayesian approaches and Monte Carlo simulations offer new statistical tools for the study of human growth.

Evaluation of the American-English Quality of Life in Short Stature Youth (QoLISSY) questionnaire in the United States.

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.

Understanding the impact of statural height on health-related quality of life in German adolescents: a population-based analysis.

UNLABELLED: Improving health-related quality of life (HrQoL) is one aim of growth hormone treatment for short-statured patients. Yet, evidence of the relationship between height and HrQoL is inconclusive, especially for young people. This study investigates the association of height and HrQoL in the general German adolescent population. A total of 6646 adolescents and 6388 parents from the German Health Interview and Examination Survey for Children and Adolescents (KIGGS) were included in the analysis. An analysis of covariance was used to evaluate the effect of height deviation on HrQoL as assessed with the KINDL-R in adolescent self-report and parent-report, taking into account sociodemographic and health-related variables. Height was found to be a weak predictor of HrQoL in both adolescent-report and parent-report. Short and tall adolescents did not substantially differ from the normal statured. Sociodemographic and health-related variables failed to significantly interact with height. In contrast, psychosocial variables contributed to the explained HrQoL variance. CONCLUSION: Height does not appear to be a strong determinant of HrQoL on the population level. These results suggest that the height gain in short stature treatment may not be the most important objective. Contrary to prevalent stereotypes, short adolescents can experience a high HrQoL.

[Living with achondroplasia- how do young persons with disproportional short stature rate their quality of life and which factors are associated with quality of life?]. / Leben mit Achondroplasie.

OBJECTIVE: Presently, little is known aqout the quality of life (QoL) as well as the strengths and difficulties of young people with achondroplasia. This study describes these patient-reported indicators and identifies possible correlates. METHOD: At the invitation of a patient organization, a total of 89 short-statured patients aged 8 to 28 years and their parents participated in this study. QoL was assessed cross-sectionally with both generic and disease-specific instruments and the Strengths and Difficulties Questionnaire (SDQ) as a brief behavioral screening. In addition to descriptive analyses, patient data were compared with a reference population. Hierarchical regression analyses reflecting sociodemographic, clinical, and psychological variables were conducted to identify correlates of QoL. RESULTS: QoL and the strengths and difficulties of young patients with achondroplasia did not differ substantially from a healthy norm sample. However, the participants reported more behavioral problems and limitations in their physical and social QoL compared to patients with another short stature diagnosis. Strengths and difficulties, height-related beliefs, and social support correlated significantly with QoL. Adding psychological variables to the regression model increased the proportion of variance explained in QoL. CONCLUSIONS: Young persons with achondroplasia did not differ in their QoL and strengths and difficulties from healthy controls. Characteristics such as height appear less important for the self-perceived QoL than are strengths and difficulties and protective psychosocia~factors.

Insightfulness and later infant attachment in clinically depressed and nonclinical mothers.

The aim of this pilot study was to investigate the relationship between maternal insightfulness and sensitivity and subsequent infant attachment security and disorganization in clinically depressed and nonclinical mother-infant groups. Nineteen depressed mothers with infants ages 3 to 11 months participated in this study. Twenty nonclinical mother-infant dyads were matched to the clinical sample according to infant sex and age. Maternal depression was assessed using the Diagnostic and Statistical Manual of Mental Disorders, fourth edition (American Psychiatric Association, 1994), insightfulness using the Insightfulness Assessment (IA), and sensitivity using the Maternal Sensitivity Scales (M.D.S. Ainsworth, 1969). IA classifications and subscales were considered separately. Later infant attachment was assessed by the Strange Situation Procedure (M.D.S. Ainsworth, M.C. Blehar, E. Waters, & S. Wall, 1978). Depressed mothers tended to have less securely attached children than did nonclinical mothers. Within the clinical sample, the insightfulness categories correlated slightly moderately with attachment security, but were not related to attachment disorganization. Within the nonclinical sample, the IA categories were slightly moderately associated with attachment security and with disorganization. On IA subscales, relationship patterns differed in clinically depressed and nonclinical mother-infant dyads. These findings provide the first evidence of the predictive power of the IA categorization and subscales on subsequent infant attachment. They also may allow the development of different foci of intervention for enhancing insightful caregiving.

[How do Affected Children and Adolescents Experience their Short Stature, and what is the Point of View of their Parents?]. / Wie erleben betroffene Kinder und Jugendliche ihren Kleinwuchs, und wie ist die Sicht ihrer Eltern?

How do Affected Children and Adolescents Experience their Short Stature, and what is the Point of View of their Parents? Despite a large number of publications on the psychosocial situation of short statured children and their parents only a few qualitative studies focus on the perspective of the affected families. Within the European QoLISSY study ("Quality of Life in Short Stature Youth") an instrument to assess the health related quality of life of short statured children was developed. The aim of this project was to examine the self-perceived quality of life of the children themselves in comparison to their parents' perspective. During the development of the QoLISSY instrument, focus groups were conducted as a first step of this study. A total of 23 short statured children and 31 parents participated and discussed their experiences in separate groups with trained moderators. The discussions were analyzed qualitatively und results were used to generate a first list of items for the questionnaire to be developed. While parents focused on socio-emotional problems, children talked much more about their growth hormone treatment and problems in their social environment. In comparison to other studies children rated their quality of life worse than their parents. Not only medical treatment but also a psychological and socio-emotional intervention seems to be indicated.

Health-related quality of life in pre-pubertal children with pediatric growth hormone deficiency: 12-month results from a phase 3 clinical trial of once-weekly somatrogon versus once-daily somatropin.

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.

Pediatric growth hormone deficiency is a condition that causes slow growth. Children with this condition have height that is lower than normal unless the condition is treated. The slow growth and short height may have bad effects on the emotional and social well-being of these children. Treatment usually consists of a growth hormone that is administered by daily injection under the skin over a period of years. However, children and their parents may not like these daily injections and often stop treatment. A newer treatment is available that can be injected once weekly. This newer treatment increases growth the same as daily injections. We looked at whether 12 months of treatment given once a week has the same positive effects on the physical, social, and emotional health of children as the daily treatment. Children and their parents answered questions that asked how being short affects the physical, social, and emotional parts of their life. These questions were asked before starting treatment and 12 months after starting treatment. In children younger than 7 years old, improvements at 12 months in their physical, social, and emotional health were similar between the treatments. In children 7 years old or older, those who received the once-weekly injections had slightly better improvements than those who received the daily injections. These results can help parents and doctors make decisions about treating children with pediatric growth hormone deficiency.

Growth hormone treatment in children with short stature: impact of the diagnosis on parents.

OBJECTIVES: This prospective multicenter study aimed (1) to examine changes in parent-reported health-related quality of life (HRQOL) of children with short stature and the effects of the children's condition on parents themselves within the first year of human growth hormone (hGH) treatment and (2) to predict effects on parents based on main and interaction effects of children's HRQOL and increase in height. METHODS: A total of 110 parents of children aged 4-18 years, diagnosed with idiopathic growth hormone deficiency, small for gestational age, or idiopathic short stature, were recruited from 11 participating German pediatric endocrinologists and asked to fill out the short stature-specific Quality of Life in Short Stature Youth (QoLISSY) Questionnaire before hGH treatment was initiated and one year later. RESULTS: Negative effects of the children's short stature on the parents decrease over time, independent of diagnosis and treatment status. Furthermore, treatment status and height increase moderated the links between children's improved HRQOL as perceived by their parents and decreased caregiving burden. CONCLUSIONS: Based on the children's improved HRQOL and the parent's decrease in caregiving burden, patient-reported outcomes that consider parental and child's perspectives should be considered when deciding on hGH treatment for children.