[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.

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.

DySMA - an Instrument to Monitor Swallowing Function in Children with Spinal Muscular Atrophy ages 0 to 24 Months: Development, Consensus, and Pilot Testing.

Background: The manifestation of bulbar symptoms, especially swallowing, is important for evaluating disease-modifying therapies for spinal muscular atrophy (SMA). Due to the lack of instruments, the topic is still underrepresented in research. Objective: This study aimed to develop a tool to monitor swallowing development in children aged 0 to 24 months with SMA. Methods: The method was guided by the COSMIN guidelines and followed a multi-stage Delphi process. The first step was a rapid review of swallowing outcomes in children with SMA younger than 24 months. In the second step, online group interviews with experts (n = 7) on dysphagia in infants were conducted, followed by an anonymous online survey among experts in infants with SMA (n = 19). A predefined consensus threshold for nominal scaled voting was set at≥75 % and for 5-point Likert scale voting at 1.25 of the interquartile range. The third step was the pilot test of the instrument, performed with three groups (healthy controls n = 8; pre-symptomatic n = 6, symptomatic n = 6). Results: Based on the multi-level interprofessional consensus, the DySMA comprises two parts (history and examination), ten categories, with 36 items. Implementation and scoring are clearly articulated and easy to implement. The pilot test showed that swallowing development could be recorded in all groups. Conclusion: The DySMA is well suited for monitoring swallowing development in pre-symptomatic and symptomatic treated infants with SMA. It can be performed in a time-efficient and interprofessional manner. The resulting score is comparable to results from other instruments measuring other domains, e.g., motor function.

Health-related quality of life and patient-reported outcome measurements in patients with cystinosis.

Nephropathic cystinosis is a rare autosomal recessive lysosomal storage disorder. With the availability of treatment and renal replacement therapy, nephropathic cystinosis has evolved from an early fatal disease to a chronic, progressive disorder with potentially high impairment. We aim to review the literature on the health-related quality of life and identify appropriate patient-reported outcome measurements to assess the health-related quality of life of patients with cystinosis. For this review, we conducted a literature search in PubMed and Web of Science in September 2021. Inclusion and exclusion criteria for the selection of articles were defined a priori. We identified 668 unique articles through the search and screened them based on title and abstract. The full texts of 27 articles were assessed. Finally, we included five articles (published between 2009 and 2020) describing the health-related quality of life in patients with cystinosis. All studies, apart from one, were conducted in the United States, and no condition-specific measurement was used. Patients with cystinosis reported a lower health-related quality of life (for certain dimensions) than healthy subjects. Few published studies address the health-related quality of life of patients with cystinosis. Such data must be collected standardized and follow the FAIR (Findable, Accessible, Interoperable, and Reusable) principles. To gain a comprehensive understanding of the impact of this disorder on health-related quality of life, it is necessary to use generic and condition-specific instruments to measure this, preferably in large samples from longitudinal studies. A cystinosis-specific instrument for measuring health-related quality of life has yet to be developed.

Health-Related Quality of Life, Stress, Caregiving Burden and Special Needs of Parents Caring for a Short-Statured Child-Review and Recommendations for Future Research.

Children with short stature can experience a range of burdens due to their chronic condition. However, little is known about parents' experiences dealing with their child's short stature and the potential caregiving burdens and concerns they may face. We aim to review the literature on health-related quality of life (HRQOL), caregiving burden, and special needs among parents caring for a child with isolated growth hormone deficiency (IGHD) or idiopathic short stature (ISS). Using pre-defined inclusion and exclusion criteria, we systematically searched for literature using PubMed and Web of Science from its inception to December 2022. We identified 15 articles assessing HRQOL, special needs, or caregiving burdens in parents of IGHD/ISS children. The main problems included concerns about the future, organizational issues, side effects from growth hormone treatment, and social stigmatization. Furthermore, two studies assessed parents' special needs to cope with caregiving stress, mainly the dialogue between them and their families or parent support groups. This review outlines parental burdens, needs, and resources when caring for an IGHD/ISS child. Furthermore, it provides information about previously used measures appraising parents' special needs and underlines the need for disease-specific measurements.

[Measures for optimal access to psychosocial care of children with rare diseases and their families]. / Maßnahmen für einen optimalen Zugang von Kindern mit seltenen Erkrankungen und ihrer Familien zur psychosozialen Versorgung.

BACKGROUND: The diagnosis and treatment of rare diseases in childhood can affect the quality of life and mental health of the entire family. The psychosocial challenges are significant for those affected and are often compounded by a lack of appropriate support services. RESEARCH QUESTION: This article aims to summarize the barriers of current pathways to psychosocial care and identify potential solutions for a care strategy adapted to the needs of families. MATERIALS AND METHODS: In three focus group sessions with 14 professionals from the field of rare pediatric diseases, the results of a previous qualitative study on barriers in current pathways to psychosocial care were presented. In the focus groups, the experts discussed possible solutions. RESULTS AND DISCUSSION: Despite an increased need for psychosocial support services, it is difficult for children with rare diseases and their families to access these. To facilitate access, interventions should address multiple levels. On the one hand, care should be increasingly adapted to the needs of families. At the same time, long-term and reliable care structures need to be established to ensure sustainable care. Structural interdisciplinary collaboration should be promoted, while the training of health professionals should also be more strongly geared to rare diseases.

Caregiving burden and special needs of parents in the care of their short-statured children - a qualitative approach.

Purpose: To explore caregiving burden, health-related quality of life (HRQOL), stress, and individual resources of parents in the care of children with isolated growth hormone deficiency (IGHD) or idiopathic short stature (ISS). Methods: Focused interview analysis of previously, within the Quality of Life in Short Stature Youth (QoLISSY) project, conducted structured focus group discussions (n=7) with parents (n=33) of children with IGHD/ISS aged 4 to 18 years were performed. Results: 26 out of the 33 parents reported mental stress due to their child's growth disorder. Social pressure and stigmatization were also mentioned as being demanding. Some parents reported having trouble with human growth hormone (hGH) treatment. Several parents wished for parent support groups with other like-minded parents of short-statured children. Conclusion: For physicians, it is essential to understand the parents' caregiving burden, stress, and individual resources in caring for IGHD/ISS children. If an impaired HRQOL is detected, psychological intervention for these parents may be scheduled, and coping mechanisms may be discussed. Furthermore, it seems essential for parents to be educated by their healthcare provider about the possible side effects of hGH treatment or to know where to find evidence-based information about it.

Living with a rare disease - experiences and needs in pediatric patients and their parents.

BACKGROUND: A rare disease (RD) diagnosis and therapy can affect the family's quality of life and mental health. A lack of information and missing care options lead to helplessness and psychological stress within families. This work aims to identify patients' and parents' experiences in daily life and with the health care system as well as their needs and current pathways to psychosocial care to develop implementation strategies adapted to the families' needs. METHODS: The present analysis is part of the national multicenter study "Children Affected by Rare Disease and Their Families-Network (CARE-FAM-NET)." We conducted semi-structured telephone interviews with children, adolescents, and young adults with RD (aged 12 to 21 years) and parents of children with RD (aged 0 to 17 years). We analyzed the transcribed and anonymized interviews using the method of focused interview analyses to identify previous experiences with medical and psychosocial care and possible needs for improvement and support. RESULTS: Seventy-four parents of children with RD and 15 children, adolescents, and young adults with RD participated. Five main themes emerged. Daily life with an RD: RD affects the everyday and social life of the respondents, negatively impacting mental well-being. Experiences with the health care system: The long diagnostic path is stressful for families. Professionals' lack of information/education leads to inadequate care for those affected. Psychosocial support: Families do not know about psychosocial care services. In some cases, the families take advantage of psychosocial support services (such as support groups or advocacy groups), which are predominantly very helpful. Difficulties and barriers: Time, socio-legal and organizational problems burden families and lead to advantages in using psychosocial services. Improvements for patient-oriented support: Those affected wished for timely, preventive support (especially in administrative and socio-legal matters) and education regarding psychosocial care services. CONCLUSION: RD represent a great challenge for all family members - patients, parents, and siblings. The patients' and parents' previous experiences in daily life, medical and psychosocial care show a need for target-group specific support, including training of health care professionals and low-threshold access care services and practical help for all family members.

Parent Perceptions of a Pretend Play Intervention for Their Children With Cancer.

BACKGROUND: Although the rate of survival in childhood cancer today is close to 85%, a cancer diagnosis can still turn the world upside down for both children and parents. Often, children in oncology care are frustrated about their inability to control events and activities around them. Therapeutic pretend play has been suggested as a means to encourage children to express and handle emotions in a safe environment. PURPOSE: This study was developed to describe and explore parents' experiences of a pretend play intervention that consisted of six to eight play sessions with a play facilitator administered to their children undergoing cancer treatment. METHODS: A descriptive qualitative method was used, including individual interviews with 15 parents. RESULTS: Three main categories were developed, including (a) experiences of joining the project, (b) perceptions of the play intervention, and (c) reflections on effects and implications, with subcategories evolved for each category. The parents experienced that the play sessions helped improve their children's communication skills and made them more capable of participating in their care. They appreciated that the intervention focused on the child's well-being and saw it as a positive break in their child's cancer treatment. It also helped them better reflect on their own situation. CONCLUSIONS/IMPLICATIONS FOR PRACTICE: According to the parents' experiences, pretend play can be a helpful tool for improving children's participation in their cancer care that strengthens their autonomy, emotional repertoire, and communication skills. However, the results also highlighted that some of the children did not fully understand the information provided about this study, which weakened the validity of their consent to participate. Thus, more work is needed on developing age-appropriate information to obtain participation consent from children. In addition, more knowledge is needed regarding how to appropriately include children with cancer in research in an ethically acceptable way.

Effects of a Pretend Play Intervention on Health-Related Quality of Life in Children With Cancer: A Swedish-German Study.

Background: Cancer diagnosis can lead to massive physical, emotional, and social burdens on children and their families. Although children have the right to be informed and participate in their care, research shows that children's views are often not considered in care situations. Thus, it is essential to strengthen children's communication and self-efficacy (SE) to convey desires and needs. The present study explores whether a play intervention is associated with improved health-related quality of life (HrQoL) and SE for communication in care situations. We hypothesize that HrQoL and SE for communication will increase from the beginning to after the pretend play intervention. Methods: Children with cancer from Germany and Sweden were enrolled. The pretend play intervention consisted of six to 10 play sessions. A heterogenic selection of questionnaires was used to measure children's HrQoL and SE before the first pretend play session and after the last play intervention. Results: Nineteen families were included in the presented analyses, including 14 self-reports of children and 19 proxy reports of parents. We found improvements in child-reported communication, and emotional and psychosocial well-being using generic and cancer-specific HrQoL measurements. Further, children's SE in care situations improved during the play intervention. Parents also reported minor improvements in the physical dimensions in both generic and chronic-generic HrQoL, along with improvements in independence. Discussion: Overall, the cancer-specific pretend play intervention offers young children with cancer a secure environment and can contribute to their well-being, and communication skills, during or after cancer treatment.

Caring for a Child with Congenital Adrenal Hyperplasia Diagnosed by Newborn Screening: Parental Health-Related Quality of Life, Coping Patterns, and Needs.

Diagnosing a child by newborn screening with classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency (CAH) causes multiple challenges for the affected parents and the whole family. We aimed to examine the health-related Quality of Life (HrQoL), coping, and needs of parents caring for a child with CAH to develop demand-responsive interventions for improving the psychosocial situation of affected families. In a retrospective cross-sectional design, we assessed HrQoL, coping patterns, and the needs of parents caring for a CAH-diagnosed child using specific questionnaires. Data of 59 families with at least one child diagnosed with CAH were analyzed. The results show that mothers and fathers in this study reached significantly higher HrQoL scores compared to reference cohorts. Decisive for the above-average parental HrQoL were effective coping behaviors and the parental needs being met. These findings verify the importance of helpful coping patterns and rapid fulfillment of parental needs for maintaining a good and stable HrQoL of parents with a child diagnosed with CAH. It is crucial to strengthen the parental HrQoL to build a reasonable basis for a healthy upbringing and improve the medical care of CAH-diagnosed children.

Increased Digital Media Use in Preschool Children: Exploring the Links with Parental Stress and Their Problematic Media Use.

BACKGROUND: Intense or problematic media use behavior of parents could serve as a role model for their children's media use behavior. So far, knowledge is scarce about increased children's media use (ICMU) and its association with parental stress (PS) and problematic parental media use (PPMU). METHODS: ICMU was examined using a modified set of the DSM-5 criteria for Internet Gaming Disorder. PS was assessed via the widely used Parenting Stress Index, and PPMU was assessed using the Short Compulsive Internet Use Scale. A multiple linear regression analysis was conducted to evaluate the links between ICMU, PS, and PPMU. A mediation analysis was performed to examine if PPMU mediated the relationship between PS and ICMU. RESULTS: In sum, 809 parents (M = 36.89 years; SD = 4.87; 81.4% female) of preschool children (average age: 44.75 months, SD = 13.68) participated in the study. ICMU was statistically significantly related to higher age of the parent, greater PPMU, and higher PS. Furthermore, we found that the association between PS and ICMU was partially mediated by PPMU. CONCLUSIONS: The results indicate that more pronounced PS and PPMU are associated with ICMU, highlighting the necessity of considering these parental variables when developing early prevention strategies for preschool-age children.