Feasibility and acceptance of KIDSCREEN-52 as a screening tool for unmet needs in children with rare inflammatory diseases

Feasibility and acceptance of KIDSCREEN-52 as a screening tool for unmet needs in children with rare inflammatory diseases | Research Square window.SnipcartSettings = { analytics: { enabled: false } }; (function() { var accessVector = localStorage.getItem('access_vector') || ''; window.dataLayer = window.dataLayer || []; if (accessVector) { window.dataLayer.push({ user: { profile: { profileInfo: { snid: accessVector } } } }); } })(); (function(w,d,s,l,i){w[l]=w[l]||[];w[l].push({'gtm.start':new Date().getTime(),event:'gtm.js'});var f=d.getElementsByTagName(s)[0],j=d.createElement(s),dl=l!='dataLayer'?'&l='+l:'';j.async=true;j.src='https://www.googletagmanager.com/gtm.js?id='+i+dl;f.parentNode.insertBefore(j,f);})(window,document,'script','dataLayer','GTM-K279D39R'); Browse Preprints In Review Journals COVID-19 Preprints AJE Video Bytes Research Tools Research Promotion AJE Professional Editing AJE Rubriq About Preprint Platform In Review Editorial Policies Our Team Advisory Board Help Center Sign In Submit a Preprint Cite Share Download PDF Research Article Feasibility and acceptance of KIDSCREEN-52 as a screening tool for unmet needs in children with rare inflammatory diseases Özlem Satirer, Gabi Erbis, Verena Heck, Tatjana Welzel, Christiane Reiser, and 3 more This is a preprint; it has not been peer reviewed by a journal. https://doi.org/ 10.21203/rs.3.rs-4616034/v1 This work is licensed under a CC BY 4.0 License Status: Posted Version 1 posted You are reading this latest preprint version Abstract Background: Children living with rare diseases often face significant psychosocial challenges; recognizing and addressing these effectively is crucial. However, there is a paucity of comprehensive screening tools. This study aimed to assess the feasibility and acceptance of the comprehensive KIDSCREEN-52 tool in identifying unmet needs of children with rare inflammatory diseases and their caregivers and identifying factors associated with low health-related quality of life (HRQoL). Methods A prospective single-center study of consecutive pediatric patients aged 8-18 with inflammatory diseases and their caregivers was performed to assess HRQoL utilizing the multidimensional KIDSCREEN-52 self-report and proxy tool. The validated KIDSCREEN-52 tool is available in 13 languages with corresponding Norm Data. It captures HRQoL across 10 domains including 52 inquiries. HRQoL of children with rare inflammatory diseases was described utilizing the multidimensional KIDSCREEN-52 self-report and proxy tool. The feasibility and acceptability of KIDSCREEN-52 was determined using a simple, dichotomous three item acceptance tool. Factors associated with low self-reported HRQoL were explored. Results: A total of 104 participants, comprising 51 pediatric patients and their 53 caregivers, were included. The patients were 35 females and 16 males, with a median age of 16 years (range: 9-18). Among them, 25 (49%) had autoinflammatory diseases, 26 (51%) had rheumatic diseases. Mean values from self-reports and proxies were consistent with the Norm Data across all domains. Self-report and proxy assessments showed high-degree agreement. Patients reported lower HRQoL levels compared to the control population in nearly all domains. Both caregivers and children expressed strong acceptance of the KIDSCREEN-52 questionnaire's clarity, relevance, and adequacy. The overall completion rate was 75%, the mean completion time 17 minutes (range: 10-25). Factors associated with low HRQoL included female gender, adolescent age and evidence of a rheumatic disease. Conclusion: The KIDSCREEN-52 demonstrated promise as a feasible and accepted tool for capturing the HRQoL and identifying unmet needs in children with rare inflammatory diseases. Its comprehensiveness and the availability in multiple languages with corresponding Norm Data, offers a unique opportunity to implement strategies to identify and address HRQoL challenges of children with rare diseases in routine clinical care. Rare diseases Inflammation children health-related quality of life (HRQoL) KIDSCREEN-52 tool feasibility acceptance Figures Figure 1 Figure 2 Background Rare diseases pose a significant health challenge globally, affecting an estimated 6–8% of Europeans during their lifetime, as highlighted by The European Union's Council of Ministers [ 1 ]. These conditions encompass a diverse range of illnesses, varying in number, type, and distribution worldwide. While some rare diseases are preventable or treatable, many are chronic, presenting complex clinical manifestations often emerging in childhood. The rarity and complexity of these disorders often result in prolonged diagnostic journeys for patients, delaying timely treatment initiation. Despite efforts to provide appropriate care, rare diseases frequently lead to morbidity, complications, and reduced life expectancy [ 2 – 4 ]. Inflammatory diseases are among the rare childhood diseases with a prevalence of less than 5 cases per 10,000 inhabitants in Europe [ 1 ]. These include autoinflammatory diseases such as Familial Mediterranean Fever (FMF), Cryopyrin-associated periodic syndromes (CAPS), and Tumor Necrosis Factor Receptor-associated periodic syndrome (TRAPS), as well as rheumatic diseases such as Juvenile Idiopathic Arthritis (JIA), Juvenile Dermatomyositis (JDM), and Juvenile Systemic Lupus Erythematosus (JSLE) [ 5 – 10 ]. Recent insights from the World Health Organization emphasize the profound impact of rare inflammatory diseases on health-related quality of life (HRQoL), extending beyond physical impairment to affect psychological well-being and social functioning. Children, adolescents, and families grappling with these conditions often face heightened vulnerability to mental health disorders such as depression and anxiety, underscoring the need for comprehensive psychosocial support [ 11 ]. Unfortunately, such support is frequently lacking in routine healthcare settings [ 12 ]. Addressing this gap requires a nuanced understanding of patient needs, enabling targeted support and resource allocation to improve the efficiency of patient care [ 13 ]. However, there is currently a dearth of established tools capable of effectively assessing these needs. Existing measurement instruments for evaluating well-being and HRQoL are often complex, potentially sacrificing precision in condensed versions. Additionally, the comparability between different measurement methods is limited due to variations in instrument usage across clinics, impeding widespread documentation of quality of life. The comprehensive, multidimensional and multi-lingual KIDSCREEN tool, funded by the European Commission, aims to capture children's perspectives on their physical and mental health and HRQoL [ 14 – 16 ]. Therefore, the aims of the pilot study were to 1) describe the HRQoL of children with rare inflammatory diseases using the multidimensional KIDSCREEN-52 self-report and proxy tool, 2) evaluate the feasibility and acceptability of the KIDSCREEN-52 as a method to rapidly and accurately determine areas of needs and 3) identify high-risk groups for low HRQoL. Methods A prospective, single-center study of consecutive pediatric patients diagnosed with autoinflammatory and rheumatologic diseases, along with their caregivers, who attended routine consultations at the Department of Pediatric Rheumatology and Autoinflammation (arcT) at the University Hospital Tuebingen between April 1, 2023, and October 1, 2023, was performed. Patients were included if they 1) were aged between eight and 18 years, and 2) were longitudinally followed and had at least three visits at the center. Data were prospectively captured in a designated electronic database, the institutional Arthritis and Rheumatism Database and Information System (ARDIS). Approval of the ethics committee of the medical faculty at the Eberhard-Karls-University and at the University Hospital Tuebingen was obtained (No 185/2023BO2). Clinical Data and Measurement Tools Patient demographics, including age, dichotomized into 8–12 years for children and 13–18 years for adolescents, gender, family background, diagnosis, disease history, and duration of care at the center, dichotomized into less than two years and two years or more, were assessed during the study. Caregiver demographics including family background and gender were also recorded. KIDSCREEN-52 The KIDSCREEN-52 consists of 52 inquiries distributed across ten distinct dimensions: Physical Well-Being (body movement and activity), Psychological Well-Being (contentment with life and optimistic outlook), Mood & Emotions (feelings of helplessness, isolation, or melancholy), Self-Perception (inner identity, and contentment with appearance), Autonomy (freedom of choice and self-reliance), Parent Relations & Home Life (interaction with caregivers, sense of parental care, and household ambiance), Financial Resources (adequate resources for social engagement), Social Support & Peer Relationships (interaction with peers, quality of social bonds), School Environment (contentment with school environment, educators, and academic achievements), and Social Acceptance (experiences of bullying, peer rejection, and social anxiety). The evaluated timeframe corresponds to the week preceding the completion of the questionnaire. Respondents are directed to evaluate either the frequency of behaviors/emotions or the intensity of attitudes using a five-point Likert scale (1–5). Questions phrased negatively underwent recoding as directed. Scores for individual items are summed and converted into T-scores for each dimension, standardized with a mean of 50 and a standard deviation of 10, based on a representative sample of the broader European populace [ 14 ]. Elevated T-scores signified a higher quality of life from a health perspective. In principle, values below the average (mean ± standard deviation) suggested a relatively lower HRQoL, while values above the average indicated a comparatively higher quality of life. The average values (mean ± standard deviation) were calculated using gender-separated German Norm Data from KIDSCREEN for children and adolescents aged 8–18. For each domain, the number and percentage of individuals below the average were calculated separately for self-report, proxy, and the entire cohort. Missing data refers to a situation where more than one item per scale is absent thereby rendering evaluation impossible. Language selection in KIDSCREEN-52 The majority of patients in the study were of German family backgrounds, however some were born and raised in Germany, but had family backgrounds from other countries (most commonly Turkey). As KIDSCREEN-52 is a validated tool in 13 languages, all language options were provided to the patients. All participants chose the German language option, indicating a strong identification with German culture and language. Correspondingly, German Norm Data were used for the scoring of KIDSCREEN-52. Outcomes The primary outcome was the acceptance of KIDSCREEN-52 using a simple, dichotomous three item acceptance instrument capturing clarity, relevance, and adequacy. Secondary outcomes were 1) KIDSCREEN-52 completion rates overall, 2) KIDSCREEN-52 completion rates for each domain, 3) time to completion of KIDSCREEN-52, 4) HRQoL as captured by KIDSCREEN-52 self-report and proxy by domains, and 5) factors associated with low HRQoL. Analysis Descriptive statistics, including means, standard deviations, medians, ranges, and frequencies, were computed as appropriate. Subsequently, the normality of the data distribution was assessed using Kolmogorov-Smirnov and Shapiro-Wilk tests. Comparative analyses were conducted using parametric and nonparametric tests as appropriate. Cronbach's alpha was utilized as a measure of internal consistency. The database was compiled by optically scanning the questionnaire responses with SPHYNX software (Sphynx®, Software Solution Incorporation, USA). Any potential erasures or errors introduced by respondents underwent manual verification by trained team members proficient in equipment operation. All statistical analyses were performed using SPSS statistical software (version21) (IBM, New York, United States). Results A total of 104 participants, consisting of 51 patients including 35 females and 16 males and 53 caregivers were included. Two patients were excluded due to incomplete data. Among the patients, 49% (25) had an autoinflammatory disease, while 51% (26) a rheumatological condition. At time of study the median age of the patient cohort was 16 years (range: 9–18). The median age at disease onset was 7 years (range: 0–16) and at diagnosis 8 years (range: 1–17). A total of 78% (39) of patients were of German, while 11% (6) were of Turkish background. The duration of care at the center was less than two years for 29% of all patients and two years or more for 71% (Table 1 ). Table 1 Baseline characteristics of the pediatric cohort with rare inflammatory diseases Children with autoinflammatory diseases (n = 25) Children with rheumatic disease (n = 26) Total cohort (n = 51) Gender, male/female 9/16 7/19 16/35 Median age at time of study in years (range) 14 (9–18) 17 (11–18) 16 (9–18) Median age at diagnosis in years (range) 6 (3–11) 9.5 (1–17) 8 (1–17) Duration of care, n (%) • < 2 years • ≥2 years 6 (24) 19 (76) 9 (35) 17 (65) 15 (29) 36 (71) Family background, n (%) • German • Turkish • other 17 (68) 5 (20) 3 (12) 22 (85) 1 (3) 3 (12) 39 (78) 6 (11) 6 (11) For patients with autoinflammatory diseases, at time of study the median age was 14 years (range: 9–18), at disease onset 3 years (range: 0–15) and at diagnosis 6 years (range: 3–11). In patients with rheumatic diseases, at time of study the median age was 17 years (range: 11–18), at disease onset 8.5 years (range: 1–16) and at diagnosis 9.5 years (range: 1–17) (Table 1 ). Among caregivers, there were 41 mothers (77%) and 12 fathers (23%). Among them, 42 (78%) were of German family background, 6 (11%) were Turkish, and 6 (11%) had different ethnicities. KIDSCREEN 52-Health-Related Quality of Life Among patients with rare inflammatory diseases and their caregivers the mean values obtained from the KIDSCREEN Proxy and Self-Report Questionnaires consistently aligned with the predefined ranges as outlined by the German Norm Data across all domains. The study cohort tended to report lower levels of well-being compared to the average German population of children and adolescents (Supplementary Tables 1 and 2). HRQoL as captured by KIDSCREEN-52 self-report by domains : A total of 29.7% of patients exhibited below-average values in the Physical Well-being domain, 25.5% in the Mood and Emotions domain, and 23.5% in both Psychological Well-being and Autonomy domains. HRQoL as captured by KIDSCREEN-52 proxy-report by domains : 31.5% of the caregivers reported below-average values in the Physical Well-being domain, 24.1% in the Autonomy domain, and 22.2% in both Moods & Emotions and Parent Relation & Home Life domains (Table 2 ). Table 2 Health-Related Quality of Life (HRQoL) documented by patient self-reports and caregiver proxy reports of the KIDSCREEN-52 instrument Subjects with low values in KIDSCREEN-52 domains Self-reported HRQoL n = 51 Proxy-reported HRQoL n = 53 Combined self-report and proxy HRQoL n = 104 Physical Well-being, n (%) 15 (29.7) 17 (31.5) 32 (32) Psychological Well-being, n (%) 12 (23.5) 9 (16.7) 21 (20) Moods & Emotions, n (%) 13 (25.5) 12 (22.2) 25 (25) Self-Perception, n (%) 5 (9.8) 5 (9.3) 10 (9) Autonomy, n (%) 12 (23.5) 13 (24.1) 25 (25) Parent Relation & Home Life, n (%) 5 (9.8) 12 (22.2) 17 (17) Social Support & Peers, n (%) 6 (11.8) 8 (14.8) 14 (15) School Environment, n (%) 5 (9.8) 5 (9.3) 10 (9) Bullying, n (%) 9 (17.6) 6 (11.1) 10 (10) Financial Resources, n (%) 8 (15.7) 2 (3.7) 10 (9) Legend : Percentage of patients with below average values in each domain according to self-report and proxy results. Average values: (mean ± standard deviation) calculated using gender-separated German Norm Data from KIDSCREEN for children & adolescents aged 8–18. Comparing self-reported HRQoL with proxy-reports : A high level of internal consistency among the items within the measurement instrument was document with a Cronbach's alpha coefficient of 0.881. In all domains, except for 'Bullying' (proxy mean: 52.7 vs. self-report mean: 52.5) and 'Financial Resources' (proxy mean: 59.4 vs. self-report mean: 57.8), the results of self-report and proxy assessments demonstrated a high level of agreement, indicating consistency (Table 3 ). Table 3 Comparative analysis of KIDSCREEN-52 self-report and proxy Health-Related Quality of Life (HRQoL) results by domains KIDSCREEN-52 domains self-report HRQoL mean (SD) proxy report HRQoL mean (SD) comparison p-value Physical Well-being 47.8 (12.1) 46.9 (13.1) 0.717 a Psychological Well-being 50.5 (10.7) 53.9 (12.6) 0.141 a Moods & Emotions 47.7 (11.2) 48.2 (13.7) 0.827 a Self-Perception 52.6 (11.1) 52.2 (10.3) 0.746 b Autonomy 50.4 (10.9) 52.1 (10.6) 0.546 b Parent Relation & Home Life 54.7 (9.97) 54.1 (9.75) 0.759 b Social Support & Peers 52.2 (13.3) 51.6 (12.2) 0.811 b School Environment 54.0 (10.7) 54.1 (12.1) 0.951 a Bullying 52.4 (9.95) 52.7 (9.32) < 0.01 b Financial Resources 56.05 (8.85) 59.4 (6.95) 0.01 b Legend : In most domains, self-report and proxy assessments demonstrated substantial agreement and consistency. The results for the domains 'Bullying' and 'Financial Resources' differed significantly between patients and caregivers. Analysis: t-test (a), Mann-Whitney U-test (b) p values < 0.05 are considered significant. Acceptance of KIDSCREEN-52 Both patients and caregivers demonstrated strong acceptance of KIDSCREEN-52 as per the instrument assessing clarity, relevance, and adequacy. The majority of both children (94%) and caregivers (94%) responded positively regarding clarity, indicating a clear understanding of the questions. Similarly, patients (96%) and caregivers (93%) highlighted the relevance of the KIDSCREEN-52 questions, suggesting those are sufficiently covering the spectrum of needs. Furthermore, both groups (96% vs. 93%) demonstrated high acceptance rates regarding the adequacy of the questions (Fig. 1 ). Feasibility of KIDSCREEN-52 Completion rates : 80/106 participants (75% of the total sample) completed the entire KIDSCREEN-52 questionnaire. Completion rates were generally high across most domains for both self-report and proxy-report with an average of 97.6% (range: 96–100%) for self-reports and 96.6% (range: 88–100%) for proxy reports. (Supplementary Table 3). Time to completion : The average time to complete the KIDSCREEN-52 questionnaire, for both self-report and proxy assessments, was 17 minutes (range: 10–25). Self-reports required an average of 19 minutes (range: 13–25), while proxy assessments took an average of 15 minutes (range: 10–19). Factors associated with low HRQoL There was a significantly higher percentage of patients with low HRQoL in the age group of 13–18 years compared to the younger age group across nearly all domains. Significant differences were seen for domains of Physical Well-being (80% vs. 20%), Psychological Well-being (83% vs. 17%), Mood and Emotions (77% vs. 27%), Self-Perception (60% vs. 40%), Autonomy (75% vs. 25%), Parental Relationships and Home Life (80% vs. 20%), Social Support and Peers (66% vs. 34%), and School Environment (100% vs. 0%) (Fig. 2 a). Gender Female patients more commonly demonstrated below average HRQoL values across nearly all domains compared to males. Significant differences were seen for domains of Physical Well-Being (86% vs. 14%), Psychological Well-Being (83% vs. 17%), Mood and Emotions (69% vs. 31%), Autonomy (75% vs. 25%), Parental Relationships and Home Life (60% vs. 40%), Social Support and Peers (66% vs. 34%), School Environment (60% vs. 40%), Financial Resources (62% vs. 38%), and Bullying (66% vs. 34%) (Fig. 2 b). Disease subgroup Patients with rheumatic diseases were more likely to have below average HRQoL scores compared to those with autoinflammatory diseases Significant differences were seen for the domains Physical Well-Being (66% vs. 34%), Psychological Well-Being (75% vs. 25%), Mood and Emotions (69% vs. 31%), Autonomy (75% vs. 25%), Parental Relationships and Home Life (80% vs. 20%), Social Support and Peers (66% vs. 34%), School Environment (80% vs. 20%), and Financial Resources (75% vs. 25%) (Fig. 2 c). Duration of care Patients cared for at our center for more than 2 years demonstrated lower HRQoL compared to those with shorter durations, this affected particularly domains related to Self-Perception (80% vs. 20%), Autonomy (58% vs. 42%), Social Support & Peers (66% vs. 34%), and Financial Resources (88% vs. 12%) (Fig. 2 d). Discussion This study is the first to evaluate the utility of KIDSCREEN-52 as a screening tool for comprehensively capturing HRQoL and identifying the unmet needs of pediatric patients with rare inflammatory diseases. The findings indicate robust acceptance of the KIDSCREEN-52 by both caregivers and children, supported by high completion rates, and overwhelmingly positive evaluations of clarity, relevance, and adequacy. The high level of agreement between self-report and proxy ratings further underscores the reliability of the KIDSCREEN-52. These results suggest that the KIDSCREEN-52 is a practical and effective tool for this patient population. KIDSCREEN-52 is a reliable, well-accepted comprehensive instrument for HRQoL characterized by high internal consistency and compliance, facilitating the rapid identification of areas of unmet needs. Our study aligned with previous research, as mean values derived from both KIDSCREEN-52 Proxy and Self-Report within our patient cohort were consistent with the predefined ranges set by the German Norm Data in all domains [ 15 ]. However, despite this alignment, our results revealed lower levels of well-being of children with inflammatory diseases. Notably, domains such as Physical Well-being, Moods & Emotions, and Autonomy exhibit the highest percentages of patients reporting values below the German Norm Data averages. These findings resonate with a study conducted in Germany in 2017, focusing on children and adolescent vertigo patients, which also indicated lower mean scores in Physical Well-being, Psychological Well-being, Autonomy domains, and the general HRQoL index compared to German Norm Data averages [ 17 ]. In nearly all domains, the findings from both self-report and proxy aligned closely with each other, indicating a high level of consistency. Research investigating the agreement between caregivers and children on KIDSCREEN has yielded varied findings [ 18 , 19 ]. Generally, there exists a moderate to good level of agreement between self-report and proxy in populations without clinical conditions. This agreement tends to be stronger in areas related to physical activity, functioning, and symptoms, compared to aspects like emotional and social HRQoL that are not easily observable [ 20 ]. Cognitive science research demonstrated that even young children are capable of effectively responding to questionnaires regarding their own health and well-being [ 22 ]. Consequently, it is recommended to interpret self- and proxy-reports in conjunction to provide a more comprehensive understanding [ 21 ]. Moreover, the importance of ensuring evaluation by caregivers if the patient is unable to fill out the assessment independently should be emphasized. Identification of high-risk factors impacting HRQoL is crucial for the selection of effective interventions. In this regard, the utilization of KIDSCREEN-52 is significant as it has the capacity to reveal disparities in quality of life among patient groups, thereby opening avenues for personalized interventions. This study showed that patients aged 13–18 years and female patients had lower HRQoL scores on multiple dimensions assessed by KIDSCREEN-52 self-report. Age is a well-established factor known to significantly influence one's HRQoL. A multicentre study conducted in Europe in 2009 using the KIDSCREEN questionnaire showed that increasing age was associated with lower HRQoL, especially in terms of physical symptoms and perceptions of health. Children generally exhibited better HRQoL compared to adolescents. While boys and girls exhibited similar HRQoL at a young age, girls‘ HRQoL decreased more than boys’ with increasing age [ 23 ]. In our study, we also observed a significant disparity in the percentage of rheumatology patients scoring below average according to German Norm Data compared to patients with autoinflammatory diseases. The number and percentage of rheumatology patients with below-average scores was higher in almost all areas than in autoinflammatory patients. This discrepancy suggested a greater need for support among rheumatology patients. Previous research in a single-center study aimed at identifying unmet needs among children with autoinflammatory diseases highlighted their need for support, particularly in the realms of school and the healthcare system [ 13 ]. When directly comparing the psychosocial burden between pediatric rheumatology patients and those with autoinflammatory diseases, a greater need for support among the former group became evident. These findings underscore the importance of considering individual factors in clinical decision-making, particularly when assessing the need for psychosocial support. The study has several limitations. Firstly, the sample size, while adequate for statistical analysis within the scope of rare diseases, is nonetheless limited. Additionally, the KIDSCREEN-52 questionnaire focuses solely on the past week, precluding inquiries about a broader timeframe. Furthermore, since the questionnaire was administered only once in our study, the consistency of responses across repeated measurements remains unverified. Lastly, the study did not explore the relationship between patients' psychosocial needs and socioeconomic factors within families of comparable socioeconomic status, representing another limitation. Conclusions The KIDSCREEN-52 tool demonstrated promise as a feasible and accepted screening tool for identifying unmet needs in children with rare inflammatory diseases. Its brevity and comprehensiveness, available in multiple languages, offers a unique opportunity to implement strategies for improving HRQoL in routine care. Declarations Funding Infrastructural funding was provided by the University Hospital Tübingen. No other funding was received for this study. Acknowledgements The authors want to thank Christine Michler and Lisa Fauser for supporting data collection. Conflicts of interest JKD has received research grants and speaker’s fees from Novartis and SOBI. CR has received speaker’s fees from Novartis, Pfizer, Galapagos and sobi. TW has given invited talks by Novartis (no personal honorarium). The other authors declare no conflicts of interest. Availability of data and materials Data is available upon request. Contributorship Statement Conceptualization: Ö.S., J.B.K.-D., S.M.B.,TW,GE Methodology: Ö.S., J.B.K.-D., S.M.B., TW, Formal analysis: Ö.S. Writing—original draft preparation: Ö.S., Review and Editing: Ö.S., J.B.K.-D., S.M.B.,GE, VH,TW., CR, A-K.G M.D. Each author made substantial contributions to the manuscript and has approved the submitted version. All authors have read and agreed to the published version of the manuscript. Ethics approval Approval of the ethics committee of the medical faculty at the Eberhard-Karls-University and at the University Hospital Tuebingen was obtained (Research Ethics Board, Project Number: No 185/2023BO2). Consent for publication Not applicable. References Council of the European Union. Council recommendation on action in the field of rare diseases— 2947th employment, social policy, health and consumer affairs—council meeting. 2009 . 2009. Valdez R, Ouyang L, Bolen J. Public Health and Rare Diseases: Oxymoron No More. Prev Chronic Dis. 2016;13:E05. Boettcher J et al. 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Children's Quality of Life Based on the KIDSCREEN-27: Child Self-Report, Parent Ratings and Child-Parent Agreement in a Swedish Random Population Sample. PLoS ONE. 2016;11(3):e0150545. Helseth S, Haraldstad K, Christophersen KA. A cross-sectional study of Health Related Quality of Life and body mass index in a Norwegian school sample (8–18 years): a comparison of child and parent perspectives. Health Qual Life Outcomes. 2015;13:47. Eiser C, Morse R. Can parents rate their child's health-related quality of life? Results of a systematic review. Qual Life Res. 2001;10(4):347–57. Meyer M, et al. Health-related quality of life in children and adolescents: Current normative data, determinants and reliability on proxy-report. J Paediatr Child Health. 2016;52(6):628–31. Riley AW. Evidence that school-age children can self-report on their health. Ambul Pediatr. 2004;4(4 Suppl):371–6. Michel G, et al. Age and gender differences in health-related quality of life of children and adolescents in Europe: a multilevel analysis. Qual Life Res. 2009;18(9):1147–57. Additional Declarations No competing interests reported. Supplementary Files supp.docx Cite Share Download PDF Status: Posted Version 1 posted You are reading this latest preprint version Research Square lets you share your work early, gain feedback from the community, and start making changes to your manuscript prior to peer review in a journal. As a division of Research Square Company, we’re committed to making research communication faster, fairer, and more useful. We do this by developing innovative software and high quality services for the global research community. Our growing team is made up of researchers and industry professionals working together to solve the most critical problems facing scientific publishing. Also discoverable on Platform About Our Team In Review Editorial Policies Advisory Board Help Center Resources Author Services Accessibility API Access RSS feed Manage Cookie Preferences © Research Square 2026 | ISSN 2693-5015 (online) Privacy Policy Terms of Service Do Not Sell My Personal Information {"props":{"pageProps":{"initialData":{"identity":"rs-4616034","acceptedTermsAndConditions":true,"allowDirectSubmit":true,"archivedVersions":[],"articleType":"Research Article","associatedPublications":[],"authors":[{"id":324506140,"identity":"e2829065-4749-45cc-bf45-91d09a79ee8b","order_by":0,"name":"Özlem Satirer","email":"","orcid":"","institution":"University of Tübingen","correspondingAuthor":false,"prefix":"","firstName":"Özlem","middleName":"","lastName":"Satirer","suffix":""},{"id":324506141,"identity":"84267c09-1baa-4baf-8b1b-9226cfa0c68d","order_by":1,"name":"Gabi Erbis","email":"","orcid":"","institution":"University of Tübingen","correspondingAuthor":false,"prefix":"","firstName":"Gabi","middleName":"","lastName":"Erbis","suffix":""},{"id":324506142,"identity":"9a6afd08-e177-4c5e-bdcb-3af172219a12","order_by":2,"name":"Verena Heck","email":"","orcid":"","institution":"University of Tübingen","correspondingAuthor":false,"prefix":"","firstName":"Verena","middleName":"","lastName":"Heck","suffix":""},{"id":324506143,"identity":"301a4e63-d5cc-406b-a8b4-49222d9a7c35","order_by":3,"name":"Tatjana Welzel","email":"","orcid":"","institution":"University of Basel","correspondingAuthor":false,"prefix":"","firstName":"Tatjana","middleName":"","lastName":"Welzel","suffix":""},{"id":324506145,"identity":"dcb30798-63c2-4d50-a5a4-7653f6ec0c7f","order_by":4,"name":"Christiane Reiser","email":"","orcid":"","institution":"University of Tübingen","correspondingAuthor":false,"prefix":"","firstName":"Christiane","middleName":"","lastName":"Reiser","suffix":""},{"id":324506148,"identity":"e71e6083-8660-43a4-94ce-84c5523c68e7","order_by":5,"name":"Anne-Kathrin Gellner","email":"","orcid":"","institution":"University of Bonn","correspondingAuthor":false,"prefix":"","firstName":"Anne-Kathrin","middleName":"","lastName":"Gellner","suffix":""},{"id":324506149,"identity":"74da645e-6b39-4015-b864-d35f42a9c882","order_by":6,"name":"Susanne M. Benseler","email":"","orcid":"","institution":"Alberta Children's Hospital","correspondingAuthor":false,"prefix":"","firstName":"Susanne","middleName":"M.","lastName":"Benseler","suffix":""},{"id":324506151,"identity":"3e71e2b0-a9e0-4780-b6b8-fddd22288696","order_by":7,"name":"Jasmin Kümmerle-Deschner","email":"data:image/png;base64,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","orcid":"","institution":"University of Tübingen","correspondingAuthor":true,"prefix":"","firstName":"Jasmin","middleName":"","lastName":"Kümmerle-Deschner","suffix":""}],"badges":[],"createdAt":"2024-06-21 08:36:27","currentVersionCode":1,"declarations":"","doi":"10.21203/rs.3.rs-4616034/v1","doiUrl":"https://doi.org/10.21203/rs.3.rs-4616034/v1","draftVersion":[],"editorialEvents":[],"editorialNote":"","failedWorkflow":false,"files":[{"id":60633728,"identity":"9478077e-1d5f-448d-ad73-4db74d33954e","added_by":"auto","created_at":"2024-07-19 01:42:11","extension":"png","order_by":1,"title":"Figure 1","display":"","copyAsset":false,"role":"figure","size":23685,"visible":true,"origin":"","legend":"\u003cp\u003e\u003cstrong\u003eAcceptance assessment of KIDSCREEN-52: Evaluating clarity, relevance, and adequacy\u003c/strong\u003e\u003c/p\u003e\n\u003cp\u003e\u003cbr\u003e\u003c/p\u003e\n\u003cp\u003e\u003cstrong\u003eLegend:\u003c/strong\u003e Both, children and caregivers documented strong acceptance of the KIDSCREEN-52 as per a dichotomous three-item acceptance instrument assessing clarity, relevance, and adequacy.\u003c/p\u003e","description":"","filename":"1.png","url":"https://assets-eu.researchsquare.com/files/rs-4616034/v1/1da759e77b14bf332f646ff9.png"},{"id":60633727,"identity":"7904feb1-231f-4c44-bf53-0a8acd4eabc1","added_by":"auto","created_at":"2024-07-19 01:42:11","extension":"png","order_by":2,"title":"Figure 2","display":"","copyAsset":false,"role":"figure","size":74540,"visible":true,"origin":"","legend":"\u003cp\u003e\u003cstrong\u003eThe impact of high-risk factors on below-average values in each self-reported KIDSCREEN-52 domain for low health-related quality of life (HRQoL)\u003c/strong\u003e\u003c/p\u003e\n\u003cp\u003e\u003cstrong\u003eFigure 2a: Impact of age\u003c/strong\u003e\u003c/p\u003e\n\u003cp\u003e\u003cstrong\u003eLegend: \u003c/strong\u003eIn nearly all domains, patients aged 13-18 years reported lower HRQoL than those aged 8-12. Average values (mean ± standard deviation) were calculated using German Norm Data for children and adolescents aged 8-18.\u003c/p\u003e\n\u003cp\u003e\u003cstrong\u003eFigure 2b: Impact of gender\u003c/strong\u003e\u003c/p\u003e\n\u003cp\u003e\u003cbr\u003e\u003c/p\u003e\n\u003cp\u003e\u003cstrong\u003eLegend:\u003c/strong\u003e In nearly all domains, females reported lower HRQoL than males. Average values (mean ± standard deviation) were calculated using German Norm Data for children and adolescents aged 8-18.\u003c/p\u003e\n\u003cp\u003e\u003cstrong\u003eFigure 2c: Impact of disease type\u003c/strong\u003e\u003c/p\u003e\n\u003cp\u003e\u003cbr\u003e\u003c/p\u003e\n\u003cp\u003e\u003cstrong\u003eLegend:\u003c/strong\u003e In nearly all domains, patients with rheumatic diseases reported lower HRQoL than those with autoinflammatory diseases. Average values (mean ± standard deviation) were calculated using German Norm Data for children and adolescents aged 8-18.\u003c/p\u003e\n\u003cp\u003e\u003cstrong\u003eFigure 2d: Impact of care duration\u003c/strong\u003e\u003c/p\u003e\n\u003cp\u003e\u003cbr\u003e\u003c/p\u003e\n\u003cp\u003e\u003cstrong\u003eLegend: \u003c/strong\u003eIn the domains of Self-Perception, Autonomy, Social Support \u0026amp; Peers, and Financial Resources, patients with a care duration of more than 2 years reported lower HRQoL. Average values (mean ± standard deviation) were calculated using German Norm Data for children and adolescents aged 8-18.\u003c/p\u003e","description":"","filename":"2.png","url":"https://assets-eu.researchsquare.com/files/rs-4616034/v1/0d9e89a39594ebae2eaa166a.png"},{"id":66100989,"identity":"23fc88e0-b725-4252-a155-fea305f1c9f7","added_by":"auto","created_at":"2024-10-07 17:02:07","extension":"pdf","order_by":0,"title":"","display":"","copyAsset":false,"role":"manuscript-pdf","size":796394,"visible":true,"origin":"","legend":"","description":"","filename":"manuscript.pdf","url":"https://assets-eu.researchsquare.com/files/rs-4616034/v1/4adf5e06-da65-48b4-b0c5-6491bbbefe55.pdf"},{"id":60633729,"identity":"bee8ae95-ba97-46da-bee6-e1e98351a913","added_by":"auto","created_at":"2024-07-19 01:42:11","extension":"docx","order_by":1,"title":"","display":"","copyAsset":false,"role":"supplement","size":26224,"visible":true,"origin":"","legend":"","description":"","filename":"supp.docx","url":"https://assets-eu.researchsquare.com/files/rs-4616034/v1/ed9fd9f3a881ba45bbd550c3.docx"}],"financialInterests":"No competing interests reported.","formattedTitle":"Feasibility and acceptance of KIDSCREEN-52 as a screening tool for unmet needs in children with rare inflammatory diseases","fulltext":[{"header":"Background","content":"\u003cp\u003eRare diseases pose a significant health challenge globally, affecting an estimated 6\u0026ndash;8% of Europeans during their lifetime, as highlighted by The European Union's Council of Ministers [\u003cspan citationid=\"CR1\" class=\"CitationRef\"\u003e1\u003c/span\u003e]. These conditions encompass a diverse range of illnesses, varying in number, type, and distribution worldwide. While some rare diseases are preventable or treatable, many are chronic, presenting complex clinical manifestations often emerging in childhood. The rarity and complexity of these disorders often result in prolonged diagnostic journeys for patients, delaying timely treatment initiation. Despite efforts to provide appropriate care, rare diseases frequently lead to morbidity, complications, and reduced life expectancy [\u003cspan additionalcitationids=\"CR3\" citationid=\"CR2\" class=\"CitationRef\"\u003e2\u003c/span\u003e\u0026ndash;\u003cspan citationid=\"CR4\" class=\"CitationRef\"\u003e4\u003c/span\u003e].\u003c/p\u003e \u003cp\u003eInflammatory diseases are among the rare childhood diseases with a prevalence of less than 5 cases per 10,000 inhabitants in Europe [\u003cspan citationid=\"CR1\" class=\"CitationRef\"\u003e1\u003c/span\u003e]. These include autoinflammatory diseases such as Familial Mediterranean Fever (FMF), Cryopyrin-associated periodic syndromes (CAPS), and Tumor Necrosis Factor Receptor-associated periodic syndrome (TRAPS), as well as rheumatic diseases such as Juvenile Idiopathic Arthritis (JIA), Juvenile Dermatomyositis (JDM), and Juvenile Systemic Lupus Erythematosus (JSLE) [\u003cspan additionalcitationids=\"CR6 CR7 CR8 CR9\" citationid=\"CR5\" class=\"CitationRef\"\u003e5\u003c/span\u003e\u0026ndash;\u003cspan citationid=\"CR10\" class=\"CitationRef\"\u003e10\u003c/span\u003e].\u003c/p\u003e \u003cp\u003eRecent insights from the World Health Organization emphasize the profound impact of rare inflammatory diseases on health-related quality of life (HRQoL), extending beyond physical impairment to affect psychological well-being and social functioning. Children, adolescents, and families grappling with these conditions often face heightened vulnerability to mental health disorders such as depression and anxiety, underscoring the need for comprehensive psychosocial support [\u003cspan citationid=\"CR11\" class=\"CitationRef\"\u003e11\u003c/span\u003e]. Unfortunately, such support is frequently lacking in routine healthcare settings [\u003cspan citationid=\"CR12\" class=\"CitationRef\"\u003e12\u003c/span\u003e]. Addressing this gap requires a nuanced understanding of patient needs, enabling targeted support and resource allocation to improve the efficiency of patient care [\u003cspan citationid=\"CR13\" class=\"CitationRef\"\u003e13\u003c/span\u003e].\u003c/p\u003e \u003cp\u003eHowever, there is currently a dearth of established tools capable of effectively assessing these needs. Existing measurement instruments for evaluating well-being and HRQoL are often complex, potentially sacrificing precision in condensed versions. Additionally, the comparability between different measurement methods is limited due to variations in instrument usage across clinics, impeding widespread documentation of quality of life. The comprehensive, multidimensional and multi-lingual KIDSCREEN tool, funded by the European Commission, aims to capture children's perspectives on their physical and mental health and HRQoL [\u003cspan additionalcitationids=\"CR15\" citationid=\"CR14\" class=\"CitationRef\"\u003e14\u003c/span\u003e\u0026ndash;\u003cspan citationid=\"CR16\" class=\"CitationRef\"\u003e16\u003c/span\u003e].\u003c/p\u003e \u003cp\u003eTherefore, the aims of the pilot study were to 1) describe the HRQoL of children with rare inflammatory diseases using the multidimensional KIDSCREEN-52 self-report and proxy tool, 2) evaluate the feasibility and acceptability of the KIDSCREEN-52 as a method to rapidly and accurately determine areas of needs and 3) identify high-risk groups for low HRQoL.\u003c/p\u003e"},{"header":"Methods","content":"\u003cp\u003e A prospective, single-center study of consecutive pediatric patients diagnosed with autoinflammatory and rheumatologic diseases, along with their caregivers, who attended routine consultations at the Department of Pediatric Rheumatology and Autoinflammation (arcT) at the University Hospital Tuebingen between April 1, 2023, and October 1, 2023, was performed. Patients were included if they 1) were aged between eight and 18 years, and 2) were longitudinally followed and had at least three visits at the center. Data were prospectively captured in a designated electronic database, the institutional Arthritis and Rheumatism Database and Information System (ARDIS). Approval of the ethics committee of the medical faculty at the Eberhard-Karls-University and at the University Hospital Tuebingen was obtained (No 185/2023BO2).\u003c/p\u003e \u003cdiv id=\"Sec3\" class=\"Section2\"\u003e \u003ch2\u003eClinical Data and Measurement Tools\u003c/h2\u003e \u003cp\u003ePatient demographics, including age, dichotomized into 8\u0026ndash;12 years for children and 13\u0026ndash;18 years for adolescents, gender, family background, diagnosis, disease history, and duration of care at the center, dichotomized into less than two years and two years or more, were assessed during the study. Caregiver demographics including family background and gender were also recorded.\u003c/p\u003e \u003c/div\u003e \u003cdiv id=\"Sec4\" class=\"Section2\"\u003e \u003ch2\u003eKIDSCREEN-52\u003c/h2\u003e \u003cp\u003eThe KIDSCREEN-52 consists of 52 inquiries distributed across ten distinct dimensions: Physical Well-Being (body movement and activity), Psychological Well-Being (contentment with life and optimistic outlook), Mood \u0026amp; Emotions (feelings of helplessness, isolation, or melancholy), Self-Perception (inner identity, and contentment with appearance), Autonomy (freedom of choice and self-reliance), Parent Relations \u0026amp; Home Life (interaction with caregivers, sense of parental care, and household ambiance), Financial Resources (adequate resources for social engagement), Social Support \u0026amp; Peer Relationships (interaction with peers, quality of social bonds), School Environment (contentment with school environment, educators, and academic achievements), and Social Acceptance (experiences of bullying, peer rejection, and social anxiety). The evaluated timeframe corresponds to the week preceding the completion of the questionnaire. Respondents are directed to evaluate either the frequency of behaviors/emotions or the intensity of attitudes using a five-point Likert scale (1\u0026ndash;5). Questions phrased negatively underwent recoding as directed. Scores for individual items are summed and converted into T-scores for each dimension, standardized with a mean of 50 and a standard deviation of 10, based on a representative sample of the broader European populace [\u003cspan citationid=\"CR14\" class=\"CitationRef\"\u003e14\u003c/span\u003e]. Elevated T-scores signified a higher quality of life from a health perspective. In principle, values below the average (mean\u0026thinsp;\u0026plusmn;\u0026thinsp;standard deviation) suggested a relatively lower HRQoL, while values above the average indicated a comparatively higher quality of life. The average values (mean\u0026thinsp;\u0026plusmn;\u0026thinsp;standard deviation) were calculated using gender-separated German Norm Data from KIDSCREEN for children and adolescents aged 8\u0026ndash;18. For each domain, the number and percentage of individuals below the average were calculated separately for self-report, proxy, and the entire cohort. Missing data refers to a situation where more than one item per scale is absent thereby rendering evaluation impossible.\u003c/p\u003e \u003c/div\u003e \u003cdiv id=\"Sec5\" class=\"Section2\"\u003e \u003ch2\u003eLanguage selection in KIDSCREEN-52\u003c/h2\u003e \u003cp\u003eThe majority of patients in the study were of German family backgrounds, however some were born and raised in Germany, but had family backgrounds from other countries (most commonly Turkey). As KIDSCREEN-52 is a validated tool in 13 languages, all language options were provided to the patients. All participants chose the German language option, indicating a strong identification with German culture and language. Correspondingly, German Norm Data were used for the scoring of KIDSCREEN-52.\u003c/p\u003e \u003c/div\u003e \u003cdiv id=\"Sec6\" class=\"Section2\"\u003e \u003ch2\u003eOutcomes\u003c/h2\u003e \u003cp\u003eThe primary outcome was the acceptance of KIDSCREEN-52 using a simple, dichotomous three item acceptance instrument capturing clarity, relevance, and adequacy. Secondary outcomes were 1) KIDSCREEN-52 completion rates overall, 2) KIDSCREEN-52 completion rates for each domain, 3) time to completion of KIDSCREEN-52, 4) HRQoL as captured by KIDSCREEN-52 self-report and proxy by domains, and 5) factors associated with low HRQoL.\u003c/p\u003e \u003c/div\u003e \u003cdiv id=\"Sec7\" class=\"Section2\"\u003e \u003ch2\u003eAnalysis\u003c/h2\u003e \u003cp\u003eDescriptive statistics, including means, standard deviations, medians, ranges, and frequencies, were computed as appropriate. Subsequently, the normality of the data distribution was assessed using Kolmogorov-Smirnov and Shapiro-Wilk tests. Comparative analyses were conducted using parametric and nonparametric tests as appropriate. Cronbach's alpha was utilized as a measure of internal consistency. The database was compiled by optically scanning the questionnaire responses with SPHYNX software (Sphynx\u0026reg;, Software Solution Incorporation, USA). Any potential erasures or errors introduced by respondents underwent manual verification by trained team members proficient in equipment operation. All statistical analyses were performed using SPSS statistical software (version21) (IBM, New York, United States).\u003c/p\u003e \u003c/div\u003e"},{"header":"Results","content":"\u003cp\u003eA total of 104 participants, consisting of 51 patients including 35 females and 16 males and 53 caregivers were included. Two patients were excluded due to incomplete data. Among the patients, 49% (25) had an autoinflammatory disease, while 51% (26) a rheumatological condition. At time of study the median age of the patient cohort was 16 years (range: 9\u0026ndash;18). The median age at disease onset was 7 years (range: 0\u0026ndash;16) and at diagnosis 8 years (range: 1\u0026ndash;17). A total of 78% (39) of patients were of German, while 11% (6) were of Turkish background. The duration of care at the center was less than two years for 29% of all patients and two years or more for 71% (Table\u0026nbsp;\u003cspan refid=\"Tab1\" class=\"InternalRef\"\u003e1\u003c/span\u003e).\u003c/p\u003e \u003cp\u003e \u003cdiv class=\"gridtable\"\u003e\u003ctable float=\"Yes\" id=\"Tab1\" border=\"1\"\u003e \u003ccaption language=\"En\"\u003e \u003cdiv class=\"CaptionNumber\"\u003eTable 1\u003c/div\u003e \u003cdiv class=\"CaptionContent\"\u003e \u003cp\u003eBaseline characteristics of the pediatric cohort with rare inflammatory diseases\u003c/p\u003e \u003c/div\u003e \u003c/caption\u003e \u003ccolgroup cols=\"4\"\u003e \u003cdiv align=\"left\" class=\"colspec\" colname=\"c1\" colnum=\"1\"\u003e\u003c/div\u003e \u003cdiv align=\"left\" class=\"colspec\" colname=\"c2\" colnum=\"2\"\u003e\u003c/div\u003e \u003cdiv align=\"left\" class=\"colspec\" colname=\"c3\" colnum=\"3\"\u003e\u003c/div\u003e \u003cdiv align=\"left\" class=\"colspec\" colname=\"c4\" colnum=\"4\"\u003e\u003c/div\u003e \u003cthead\u003e \u003ctr\u003e \u003cth align=\"left\" colname=\"c1\"\u003e\u0026nbsp;\u003c/th\u003e \u003cth align=\"left\" colname=\"c2\"\u003e \u003cp\u003eChildren with autoinflammatory diseases\u003c/p\u003e \u003cp\u003e(n\u0026thinsp;=\u0026thinsp;25)\u003c/p\u003e \u003c/th\u003e \u003cth align=\"left\" colname=\"c3\"\u003e \u003cp\u003eChildren with rheumatic disease\u003c/p\u003e \u003cp\u003e(n\u0026thinsp;=\u0026thinsp;26)\u003c/p\u003e \u003c/th\u003e \u003cth align=\"left\" colname=\"c4\"\u003e \u003cp\u003eTotal cohort\u003c/p\u003e \u003cp\u003e(n\u0026thinsp;=\u0026thinsp;51)\u003c/p\u003e \u003c/th\u003e \u003c/tr\u003e \u003c/thead\u003e \u003ctbody\u003e \u003ctr\u003e \u003ctd align=\"left\" colname=\"c1\"\u003e \u003cp\u003eGender, male/female\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c2\"\u003e \u003cp\u003e9/16\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c3\"\u003e \u003cp\u003e7/19\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c4\"\u003e \u003cp\u003e16/35\u003c/p\u003e \u003c/td\u003e \u003c/tr\u003e \u003ctr\u003e \u003ctd align=\"left\" colname=\"c1\"\u003e \u003cp\u003eMedian age at time of study in years (range)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c2\"\u003e \u003cp\u003e14 (9\u0026ndash;18)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c3\"\u003e \u003cp\u003e17 (11\u0026ndash;18)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c4\"\u003e \u003cp\u003e16 (9\u0026ndash;18)\u003c/p\u003e \u003c/td\u003e \u003c/tr\u003e \u003ctr\u003e \u003ctd align=\"left\" colname=\"c1\"\u003e \u003cp\u003eMedian age at diagnosis in years (range)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c2\"\u003e \u003cp\u003e6 (3\u0026ndash;11)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c3\"\u003e \u003cp\u003e9.5 (1\u0026ndash;17)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c4\"\u003e \u003cp\u003e8 (1\u0026ndash;17)\u003c/p\u003e \u003c/td\u003e \u003c/tr\u003e \u003ctr\u003e \u003ctd align=\"left\" colname=\"c1\"\u003e \u003cp\u003eDuration of care, n (%)\u003c/p\u003e \u003cp\u003e\u0026bull; \u0026lt;\u0026thinsp;2 years\u003c/p\u003e \u003cp\u003e\u0026bull; \u0026ge;2 years\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c2\"\u003e \u003cp\u003e6 (24)\u003c/p\u003e \u003cp\u003e19 (76)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c3\"\u003e \u003cp\u003e9 (35)\u003c/p\u003e \u003cp\u003e17 (65)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c4\"\u003e \u003cp\u003e15 (29)\u003c/p\u003e \u003cp\u003e36 (71)\u003c/p\u003e \u003c/td\u003e \u003c/tr\u003e \u003ctr\u003e \u003ctd align=\"left\" colname=\"c1\"\u003e \u003cp\u003eFamily background, n (%)\u003c/p\u003e \u003cp\u003e\u0026bull; German\u003c/p\u003e \u003cp\u003e\u0026bull; Turkish\u003c/p\u003e \u003cp\u003e\u0026bull; other\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c2\"\u003e \u003cp\u003e17 (68)\u003c/p\u003e \u003cp\u003e5 (20)\u003c/p\u003e \u003cp\u003e3 (12)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c3\"\u003e \u003cp\u003e22 (85)\u003c/p\u003e \u003cp\u003e1 (3)\u003c/p\u003e \u003cp\u003e3 (12)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c4\"\u003e \u003cp\u003e39 (78)\u003c/p\u003e \u003cp\u003e6 (11)\u003c/p\u003e \u003cp\u003e6 (11)\u003c/p\u003e \u003c/td\u003e \u003c/tr\u003e \u003c/tbody\u003e \u003c/colgroup\u003e \u003c/table\u003e\u003c/div\u003e \u003c/p\u003e \u003cp\u003eFor patients with autoinflammatory diseases, at time of study the median age was 14 years (range: 9\u0026ndash;18), at disease onset 3 years (range: 0\u0026ndash;15) and at diagnosis 6 years (range: 3\u0026ndash;11). In patients with rheumatic diseases, at time of study the median age was 17 years (range: 11\u0026ndash;18), at disease onset 8.5 years (range: 1\u0026ndash;16) and at diagnosis 9.5 years (range: 1\u0026ndash;17) (Table\u0026nbsp;\u003cspan refid=\"Tab1\" class=\"InternalRef\"\u003e1\u003c/span\u003e). Among caregivers, there were 41 mothers (77%) and 12 fathers (23%). Among them, 42 (78%) were of German family background, 6 (11%) were Turkish, and 6 (11%) had different ethnicities.\u003c/p\u003e \u003cdiv id=\"Sec9\" class=\"Section2\"\u003e \u003ch2\u003eKIDSCREEN 52-Health-Related Quality of Life\u003c/h2\u003e \u003cp\u003eAmong patients with rare inflammatory diseases and their caregivers the mean values obtained from the KIDSCREEN Proxy and Self-Report Questionnaires consistently aligned with the predefined ranges as outlined by the German Norm Data across all domains. The study cohort tended to report lower levels of well-being compared to the average German population of children and adolescents (Supplementary Tables\u0026nbsp;1 and 2).\u003c/p\u003e \u003cp\u003e \u003cb\u003eHRQoL as captured by KIDSCREEN-52 self-report by domains\u003c/b\u003e: A total of 29.7% of patients exhibited below-average values in the Physical Well-being domain, 25.5% in the Mood and Emotions domain, and 23.5% in both Psychological Well-being and Autonomy domains. \u003cb\u003eHRQoL as captured by KIDSCREEN-52 proxy-report by domains\u003c/b\u003e: 31.5% of the caregivers reported below-average values in the Physical Well-being domain, 24.1% in the Autonomy domain, and 22.2% in both Moods \u0026amp; Emotions and Parent Relation \u0026amp; Home Life domains (Table\u0026nbsp;\u003cspan refid=\"Tab2\" class=\"InternalRef\"\u003e2\u003c/span\u003e).\u003c/p\u003e \u003cp\u003e \u003cdiv class=\"gridtable\"\u003e\u003ctable float=\"Yes\" id=\"Tab2\" border=\"1\"\u003e \u003ccaption language=\"En\"\u003e \u003cdiv class=\"CaptionNumber\"\u003eTable 2\u003c/div\u003e \u003cdiv class=\"CaptionContent\"\u003e \u003cp\u003eHealth-Related Quality of Life (HRQoL) documented by patient self-reports and caregiver proxy reports of the KIDSCREEN-52 instrument\u003c/p\u003e \u003c/div\u003e \u003c/caption\u003e \u003ccolgroup cols=\"4\"\u003e \u003cdiv align=\"left\" class=\"colspec\" colname=\"c1\" colnum=\"1\"\u003e\u003c/div\u003e \u003cdiv align=\"char\" char=\".\" class=\"colspec\" colname=\"c2\" colnum=\"2\"\u003e\u003c/div\u003e \u003cdiv align=\"char\" char=\".\" class=\"colspec\" colname=\"c3\" colnum=\"3\"\u003e\u003c/div\u003e \u003cdiv align=\"left\" class=\"colspec\" colname=\"c4\" colnum=\"4\"\u003e\u003c/div\u003e \u003cthead\u003e \u003ctr\u003e \u003cth align=\"left\" colname=\"c1\"\u003e \u003cp\u003eSubjects with low values in\u003c/p\u003e \u003cp\u003eKIDSCREEN-52 domains\u003c/p\u003e \u003c/th\u003e \u003cth align=\"left\" colname=\"c2\"\u003e \u003cp\u003eSelf-reported HRQoL\u003c/p\u003e \u003cp\u003en\u0026thinsp;=\u0026thinsp;51\u003c/p\u003e \u003c/th\u003e \u003cth align=\"left\" colname=\"c3\"\u003e \u003cp\u003eProxy-reported\u003c/p\u003e \u003cp\u003eHRQoL\u003c/p\u003e \u003cp\u003en\u0026thinsp;=\u0026thinsp;53\u003c/p\u003e \u003c/th\u003e \u003cth align=\"left\" colname=\"c4\"\u003e \u003cp\u003eCombined\u003c/p\u003e \u003cp\u003eself-report and proxy HRQoL\u003c/p\u003e \u003cp\u003en\u0026thinsp;=\u0026thinsp;104\u003c/p\u003e \u003c/th\u003e \u003c/tr\u003e \u003c/thead\u003e \u003ctbody\u003e \u003ctr\u003e \u003ctd align=\"left\" colname=\"c1\"\u003e \u003cp\u003ePhysical Well-being, n (%)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c2\"\u003e \u003cp\u003e15 (29.7)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c3\"\u003e \u003cp\u003e17 (31.5)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c4\"\u003e \u003cp\u003e32 (32)\u003c/p\u003e \u003c/td\u003e \u003c/tr\u003e \u003ctr\u003e \u003ctd align=\"left\" colname=\"c1\"\u003e \u003cp\u003ePsychological Well-being, n (%)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c2\"\u003e \u003cp\u003e12 (23.5)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c3\"\u003e \u003cp\u003e9 (16.7)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c4\"\u003e \u003cp\u003e21 (20)\u003c/p\u003e \u003c/td\u003e \u003c/tr\u003e \u003ctr\u003e \u003ctd align=\"left\" colname=\"c1\"\u003e \u003cp\u003eMoods \u0026amp; Emotions, n (%)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c2\"\u003e \u003cp\u003e13 (25.5)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c3\"\u003e \u003cp\u003e12 (22.2)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c4\"\u003e \u003cp\u003e25 (25)\u003c/p\u003e \u003c/td\u003e \u003c/tr\u003e \u003ctr\u003e \u003ctd align=\"left\" colname=\"c1\"\u003e \u003cp\u003eSelf-Perception, n (%)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c2\"\u003e \u003cp\u003e5 (9.8)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c3\"\u003e \u003cp\u003e5 (9.3)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c4\"\u003e \u003cp\u003e10 (9)\u003c/p\u003e \u003c/td\u003e \u003c/tr\u003e \u003ctr\u003e \u003ctd align=\"left\" colname=\"c1\"\u003e \u003cp\u003eAutonomy, n (%)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c2\"\u003e \u003cp\u003e12 (23.5)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c3\"\u003e \u003cp\u003e13 (24.1)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c4\"\u003e \u003cp\u003e25 (25)\u003c/p\u003e \u003c/td\u003e \u003c/tr\u003e \u003ctr\u003e \u003ctd align=\"left\" colname=\"c1\"\u003e \u003cp\u003eParent Relation \u0026amp; Home Life, n (%)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c2\"\u003e \u003cp\u003e5 (9.8)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c3\"\u003e \u003cp\u003e12 (22.2)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c4\"\u003e \u003cp\u003e17 (17)\u003c/p\u003e \u003c/td\u003e \u003c/tr\u003e \u003ctr\u003e \u003ctd align=\"left\" colname=\"c1\"\u003e \u003cp\u003eSocial Support \u0026amp; Peers, n (%)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c2\"\u003e \u003cp\u003e6 (11.8)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c3\"\u003e \u003cp\u003e8 (14.8)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c4\"\u003e \u003cp\u003e14 (15)\u003c/p\u003e \u003c/td\u003e \u003c/tr\u003e \u003ctr\u003e \u003ctd align=\"left\" colname=\"c1\"\u003e \u003cp\u003eSchool Environment, n (%)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c2\"\u003e \u003cp\u003e5 (9.8)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c3\"\u003e \u003cp\u003e5 (9.3)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c4\"\u003e \u003cp\u003e10 (9)\u003c/p\u003e \u003c/td\u003e \u003c/tr\u003e \u003ctr\u003e \u003ctd align=\"left\" colname=\"c1\"\u003e \u003cp\u003eBullying, n (%)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c2\"\u003e \u003cp\u003e9 (17.6)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c3\"\u003e \u003cp\u003e6 (11.1)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c4\"\u003e \u003cp\u003e10 (10)\u003c/p\u003e \u003c/td\u003e \u003c/tr\u003e \u003ctr\u003e \u003ctd align=\"left\" colname=\"c1\"\u003e \u003cp\u003eFinancial Resources, n (%)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c2\"\u003e \u003cp\u003e8 (15.7)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c3\"\u003e \u003cp\u003e2 (3.7)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c4\"\u003e \u003cp\u003e10 (9)\u003c/p\u003e \u003c/td\u003e \u003c/tr\u003e \u003c/tbody\u003e \u003c/colgroup\u003e \u003ctfoot\u003e \u003ctr\u003e\u003ctd colspan=\"4\"\u003e\u003cb\u003eLegend\u003c/b\u003e: Percentage of patients with below average values in each domain according to self-report and proxy results. Average values: (mean\u0026thinsp;\u0026plusmn;\u0026thinsp;standard deviation) calculated using gender-separated German Norm Data from KIDSCREEN for children \u0026amp; adolescents aged 8\u0026ndash;18.\u003c/td\u003e\u003c/tr\u003e \u003c/tfoot\u003e \u003c/table\u003e\u003c/div\u003e \u003c/p\u003e \u003cp\u003e \u003cb\u003eComparing self-reported HRQoL with proxy-reports\u003c/b\u003e: A high level of internal consistency among the items within the measurement instrument was document with a Cronbach's alpha coefficient of 0.881. In all domains, except for 'Bullying' (proxy mean: 52.7 vs. self-report mean: 52.5) and 'Financial Resources' (proxy mean: 59.4 vs. self-report mean: 57.8), the results of self-report and proxy assessments demonstrated a high level of agreement, indicating consistency (Table\u0026nbsp;\u003cspan refid=\"Tab3\" class=\"InternalRef\"\u003e3\u003c/span\u003e).\u003c/p\u003e \u003cp\u003e \u003cdiv class=\"gridtable\"\u003e\u003ctable float=\"Yes\" id=\"Tab3\" border=\"1\"\u003e \u003ccaption language=\"En\"\u003e \u003cdiv class=\"CaptionNumber\"\u003eTable 3\u003c/div\u003e \u003cdiv class=\"CaptionContent\"\u003e \u003cp\u003eComparative analysis of KIDSCREEN-52 self-report and proxy Health-Related Quality of Life (HRQoL) results by domains\u003c/p\u003e \u003c/div\u003e \u003c/caption\u003e \u003ccolgroup cols=\"4\"\u003e \u003cdiv align=\"left\" class=\"colspec\" colname=\"c1\" colnum=\"1\"\u003e\u003c/div\u003e \u003cdiv align=\"char\" char=\".\" class=\"colspec\" colname=\"c2\" colnum=\"2\"\u003e\u003c/div\u003e \u003cdiv align=\"char\" char=\".\" class=\"colspec\" colname=\"c3\" colnum=\"3\"\u003e\u003c/div\u003e \u003cdiv align=\"left\" class=\"colspec\" colname=\"c4\" colnum=\"4\"\u003e\u003c/div\u003e \u003cthead\u003e \u003ctr\u003e \u003cth align=\"left\" colname=\"c1\"\u003e \u003cp\u003eKIDSCREEN-52 domains\u003c/p\u003e \u003c/th\u003e \u003cth align=\"left\" colname=\"c2\"\u003e \u003cp\u003eself-report HRQoL\u003c/p\u003e \u003cp\u003emean (SD)\u003c/p\u003e \u003c/th\u003e \u003cth align=\"left\" colname=\"c3\"\u003e \u003cp\u003eproxy report\u003c/p\u003e \u003cp\u003eHRQoL\u003c/p\u003e \u003cp\u003emean (SD)\u003c/p\u003e \u003c/th\u003e \u003cth align=\"left\" colname=\"c4\"\u003e \u003cp\u003ecomparison\u003c/p\u003e \u003cp\u003ep-value\u003c/p\u003e \u003c/th\u003e \u003c/tr\u003e \u003c/thead\u003e \u003ctbody\u003e \u003ctr\u003e \u003ctd align=\"left\" colname=\"c1\"\u003e \u003cp\u003ePhysical Well-being\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c2\"\u003e \u003cp\u003e47.8 (12.1)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c3\"\u003e \u003cp\u003e46.9 (13.1)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c4\"\u003e \u003cp\u003e0.717\u003csup\u003ea\u003c/sup\u003e\u003c/p\u003e \u003c/td\u003e \u003c/tr\u003e \u003ctr\u003e \u003ctd align=\"left\" colname=\"c1\"\u003e \u003cp\u003ePsychological Well-being\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c2\"\u003e \u003cp\u003e50.5 (10.7)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c3\"\u003e \u003cp\u003e53.9 (12.6)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c4\"\u003e \u003cp\u003e0.141\u003csup\u003ea\u003c/sup\u003e\u003c/p\u003e \u003c/td\u003e \u003c/tr\u003e \u003ctr\u003e \u003ctd align=\"left\" colname=\"c1\"\u003e \u003cp\u003eMoods \u0026amp; Emotions\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c2\"\u003e \u003cp\u003e47.7 (11.2)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c3\"\u003e \u003cp\u003e48.2 (13.7)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c4\"\u003e \u003cp\u003e0.827\u003csup\u003ea\u003c/sup\u003e\u003c/p\u003e \u003c/td\u003e \u003c/tr\u003e \u003ctr\u003e \u003ctd align=\"left\" colname=\"c1\"\u003e \u003cp\u003eSelf-Perception\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c2\"\u003e \u003cp\u003e52.6 (11.1)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c3\"\u003e \u003cp\u003e52.2 (10.3)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c4\"\u003e \u003cp\u003e0.746\u003csup\u003eb\u003c/sup\u003e\u003c/p\u003e \u003c/td\u003e \u003c/tr\u003e \u003ctr\u003e \u003ctd align=\"left\" colname=\"c1\"\u003e \u003cp\u003eAutonomy\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c2\"\u003e \u003cp\u003e50.4 (10.9)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c3\"\u003e \u003cp\u003e52.1 (10.6)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c4\"\u003e \u003cp\u003e0.546\u003csup\u003eb\u003c/sup\u003e\u003c/p\u003e \u003c/td\u003e \u003c/tr\u003e \u003ctr\u003e \u003ctd align=\"left\" colname=\"c1\"\u003e \u003cp\u003eParent Relation \u0026amp; Home Life\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c2\"\u003e \u003cp\u003e54.7 (9.97)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c3\"\u003e \u003cp\u003e54.1 (9.75)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c4\"\u003e \u003cp\u003e0.759\u003csup\u003eb\u003c/sup\u003e\u003c/p\u003e \u003c/td\u003e \u003c/tr\u003e \u003ctr\u003e \u003ctd align=\"left\" colname=\"c1\"\u003e \u003cp\u003eSocial Support \u0026amp; Peers\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c2\"\u003e \u003cp\u003e52.2 (13.3)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c3\"\u003e \u003cp\u003e51.6 (12.2)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c4\"\u003e \u003cp\u003e0.811\u003csup\u003eb\u003c/sup\u003e\u003c/p\u003e \u003c/td\u003e \u003c/tr\u003e \u003ctr\u003e \u003ctd align=\"left\" colname=\"c1\"\u003e \u003cp\u003eSchool Environment\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c2\"\u003e \u003cp\u003e54.0 (10.7)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c3\"\u003e \u003cp\u003e54.1 (12.1)\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c4\"\u003e \u003cp\u003e0.951\u003csup\u003ea\u003c/sup\u003e\u003c/p\u003e \u003c/td\u003e \u003c/tr\u003e \u003ctr\u003e \u003ctd align=\"left\" colname=\"c1\"\u003e \u003cp\u003eBullying\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c2\"\u003e \u003cp\u003e\u003cb\u003e52.4 (9.95)\u003c/b\u003e\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c3\"\u003e \u003cp\u003e\u003cb\u003e52.7 (9.32)\u003c/b\u003e\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c4\"\u003e \u003cp\u003e\u003cb\u003e\u0026lt;\u0026thinsp;0.01\u003c/b\u003e\u003csup\u003e\u003cb\u003eb\u003c/b\u003e\u003c/sup\u003e\u003c/p\u003e \u003c/td\u003e \u003c/tr\u003e \u003ctr\u003e \u003ctd align=\"left\" colname=\"c1\"\u003e \u003cp\u003eFinancial Resources\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c2\"\u003e \u003cp\u003e\u003cb\u003e56.05 (8.85)\u003c/b\u003e\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"char\" char=\".\" colname=\"c3\"\u003e \u003cp\u003e\u003cb\u003e59.4 (6.95)\u003c/b\u003e\u003c/p\u003e \u003c/td\u003e \u003ctd align=\"left\" colname=\"c4\"\u003e \u003cp\u003e\u003cb\u003e0.01\u003c/b\u003e\u003csup\u003e\u003cb\u003eb\u003c/b\u003e\u003c/sup\u003e\u003c/p\u003e \u003c/td\u003e \u003c/tr\u003e \u003c/tbody\u003e \u003c/colgroup\u003e \u003ctfoot\u003e \u003ctr\u003e\u003ctd colspan=\"4\"\u003e\u003cb\u003eLegend\u003c/b\u003e: In most domains, self-report and proxy assessments demonstrated substantial agreement and consistency. The results for the domains 'Bullying' and 'Financial Resources' differed significantly between patients and caregivers. Analysis: t-test (a), Mann-Whitney U-test (b) p values\u0026thinsp;\u0026lt;\u0026thinsp;0.05 are considered significant.\u003c/td\u003e\u003c/tr\u003e \u003c/tfoot\u003e \u003c/table\u003e\u003c/div\u003e \u003c/p\u003e \u003c/div\u003e \u003cdiv id=\"Sec10\" class=\"Section2\"\u003e \u003ch2\u003eAcceptance of KIDSCREEN-52\u003c/h2\u003e \u003cp\u003eBoth patients and caregivers demonstrated strong acceptance of KIDSCREEN-52 as per the instrument assessing clarity, relevance, and adequacy. The majority of both children (94%) and caregivers (94%) responded positively regarding clarity, indicating a clear understanding of the questions. Similarly, patients (96%) and caregivers (93%) highlighted the relevance of the KIDSCREEN-52 questions, suggesting those are sufficiently covering the spectrum of needs. Furthermore, both groups (96% vs. 93%) demonstrated high acceptance rates regarding the adequacy of the questions (Fig.\u0026nbsp;\u003cspan refid=\"Fig1\" class=\"InternalRef\"\u003e1\u003c/span\u003e).\u003c/p\u003e \u003c/div\u003e \u003cdiv id=\"Sec11\" class=\"Section2\"\u003e \u003ch2\u003eFeasibility of KIDSCREEN-52\u003c/h2\u003e \u003cp\u003e \u003cb\u003eCompletion rates\u003c/b\u003e: 80/106 participants (75% of the total sample) completed the entire KIDSCREEN-52 questionnaire. Completion rates were generally high across most domains for both self-report and proxy-report with an average of 97.6% (range: 96\u0026ndash;100%) for self-reports and 96.6% (range: 88\u0026ndash;100%) for proxy reports. (Supplementary Table\u0026nbsp;3).\u003c/p\u003e \u003cp\u003e \u003cb\u003eTime to completion\u003c/b\u003e: The average time to complete the KIDSCREEN-52 questionnaire, for both self-report and proxy assessments, was 17 minutes (range: 10\u0026ndash;25). Self-reports required an average of 19 minutes (range: 13\u0026ndash;25), while proxy assessments took an average of 15 minutes (range: 10\u0026ndash;19).\u003c/p\u003e \u003c/div\u003e \u003cdiv id=\"Sec12\" class=\"Section2\"\u003e \u003ch2\u003eFactors associated with low HRQoL\u003c/h2\u003e \u003cp\u003eThere was a significantly higher percentage of patients with low HRQoL in the age group of 13\u0026ndash;18 years compared to the younger age group across nearly all domains. Significant differences were seen for domains of Physical Well-being (80% vs. 20%), Psychological Well-being (83% vs. 17%), Mood and Emotions (77% vs. 27%), Self-Perception (60% vs. 40%), Autonomy (75% vs. 25%), Parental Relationships and Home Life (80% vs. 20%), Social Support and Peers (66% vs. 34%), and School Environment (100% vs. 0%) (Fig.\u0026nbsp;\u003cspan refid=\"Fig6\" class=\"InternalRef\"\u003e2\u003c/span\u003ea).\u003c/p\u003e \u003cp\u003e \u003cstrong\u003eGender\u003c/strong\u003e \u003cp\u003eFemale patients more commonly demonstrated below average HRQoL values across nearly all domains compared to males. Significant differences were seen for domains of Physical Well-Being (86% vs. 14%), Psychological Well-Being (83% vs. 17%), Mood and Emotions (69% vs. 31%), Autonomy (75% vs. 25%), Parental Relationships and Home Life (60% vs. 40%), Social Support and Peers (66% vs. 34%), School Environment (60% vs. 40%), Financial Resources (62% vs. 38%), and Bullying (66% vs. 34%) (Fig.\u0026nbsp;\u003cspan refid=\"Fig6\" class=\"InternalRef\"\u003e2\u003c/span\u003eb).\u003c/p\u003e \u003c/p\u003e \u003cp\u003e \u003cstrong\u003eDisease subgroup\u003c/strong\u003e \u003cp\u003ePatients with rheumatic diseases were more likely to have below average HRQoL scores compared to those with autoinflammatory diseases Significant differences were seen for the domains Physical Well-Being (66% vs. 34%), Psychological Well-Being (75% vs. 25%), Mood and Emotions (69% vs. 31%), Autonomy (75% vs. 25%), Parental Relationships and Home Life (80% vs. 20%), Social Support and Peers (66% vs. 34%), School Environment (80% vs. 20%), and Financial Resources (75% vs. 25%) (Fig.\u0026nbsp;\u003cspan refid=\"Fig6\" class=\"InternalRef\"\u003e2\u003c/span\u003ec).\u003c/p\u003e \u003c/p\u003e \u003cp\u003e \u003cstrong\u003eDuration of care\u003c/strong\u003e \u003cp\u003ePatients cared for at our center for more than 2 years demonstrated lower HRQoL compared to those with shorter durations, this affected particularly domains related to Self-Perception (80% vs. 20%), Autonomy (58% vs. 42%), Social Support \u0026amp; Peers (66% vs. 34%), and Financial Resources (88% vs. 12%) (Fig.\u0026nbsp;\u003cspan refid=\"Fig6\" class=\"InternalRef\"\u003e2\u003c/span\u003ed).\u003c/p\u003e \u003c/p\u003e \u003c/div\u003e"},{"header":"Discussion","content":"\u003cp\u003eThis study is the first to evaluate the utility of KIDSCREEN-52 as a screening tool for comprehensively capturing HRQoL and identifying the unmet needs of pediatric patients with rare inflammatory diseases. The findings indicate robust acceptance of the KIDSCREEN-52 by both caregivers and children, supported by high completion rates, and overwhelmingly positive evaluations of clarity, relevance, and adequacy. The high level of agreement between self-report and proxy ratings further underscores the reliability of the KIDSCREEN-52. These results suggest that the KIDSCREEN-52 is a practical and effective tool for this patient population.\u003c/p\u003e \u003cp\u003eKIDSCREEN-52 is a reliable, well-accepted comprehensive instrument for HRQoL characterized by high internal consistency and compliance, facilitating the rapid identification of areas of unmet needs. Our study aligned with previous research, as mean values derived from both KIDSCREEN-52 Proxy and Self-Report within our patient cohort were consistent with the predefined ranges set by the German Norm Data in all domains [\u003cspan citationid=\"CR15\" class=\"CitationRef\"\u003e15\u003c/span\u003e]. However, despite this alignment, our results revealed lower levels of well-being of children with inflammatory diseases. Notably, domains such as Physical Well-being, Moods \u0026amp; Emotions, and Autonomy exhibit the highest percentages of patients reporting values below the German Norm Data averages. These findings resonate with a study conducted in Germany in 2017, focusing on children and adolescent vertigo patients, which also indicated lower mean scores in Physical Well-being, Psychological Well-being, Autonomy domains, and the general HRQoL index compared to German Norm Data averages [\u003cspan citationid=\"CR17\" class=\"CitationRef\"\u003e17\u003c/span\u003e]. In nearly all domains, the findings from both self-report and proxy aligned closely with each other, indicating a high level of consistency. Research investigating the agreement between caregivers and children on KIDSCREEN has yielded varied findings [\u003cspan citationid=\"CR18\" class=\"CitationRef\"\u003e18\u003c/span\u003e, \u003cspan citationid=\"CR19\" class=\"CitationRef\"\u003e19\u003c/span\u003e]. Generally, there exists a moderate to good level of agreement between self-report and proxy in populations without clinical conditions. This agreement tends to be stronger in areas related to physical activity, functioning, and symptoms, compared to aspects like emotional and social HRQoL that are not easily observable [\u003cspan citationid=\"CR20\" class=\"CitationRef\"\u003e20\u003c/span\u003e]. Cognitive science research demonstrated that even young children are capable of effectively responding to questionnaires regarding their own health and well-being [\u003cspan citationid=\"CR22\" class=\"CitationRef\"\u003e22\u003c/span\u003e]. Consequently, it is recommended to interpret self- and proxy-reports in conjunction to provide a more comprehensive understanding [\u003cspan citationid=\"CR21\" class=\"CitationRef\"\u003e21\u003c/span\u003e]. Moreover, the importance of ensuring evaluation by caregivers if the patient is unable to fill out the assessment independently should be emphasized.\u003c/p\u003e \u003cp\u003eIdentification of high-risk factors impacting HRQoL is crucial for the selection of effective interventions. In this regard, the utilization of KIDSCREEN-52 is significant as it has the capacity to reveal disparities in quality of life among patient groups, thereby opening avenues for personalized interventions. This study showed that patients aged 13\u0026ndash;18 years and female patients had lower HRQoL scores on multiple dimensions assessed by KIDSCREEN-52 self-report. Age is a well-established factor known to significantly influence one's HRQoL. A multicentre study conducted in Europe in 2009 using the KIDSCREEN questionnaire showed that increasing age was associated with lower HRQoL, especially in terms of physical symptoms and perceptions of health. Children generally exhibited better HRQoL compared to adolescents. While boys and girls exhibited similar HRQoL at a young age, girls\u0026lsquo; HRQoL decreased more than boys\u0026rsquo; with increasing age [\u003cspan citationid=\"CR23\" class=\"CitationRef\"\u003e23\u003c/span\u003e]. In our study, we also observed a significant disparity in the percentage of rheumatology patients scoring below average according to German Norm Data compared to patients with autoinflammatory diseases. The number and percentage of rheumatology patients with below-average scores was higher in almost all areas than in autoinflammatory patients. This discrepancy suggested a greater need for support among rheumatology patients. Previous research in a single-center study aimed at identifying unmet needs among children with autoinflammatory diseases highlighted their need for support, particularly in the realms of school and the healthcare system [\u003cspan citationid=\"CR13\" class=\"CitationRef\"\u003e13\u003c/span\u003e]. When directly comparing the psychosocial burden between pediatric rheumatology patients and those with autoinflammatory diseases, a greater need for support among the former group became evident. These findings underscore the importance of considering individual factors in clinical decision-making, particularly when assessing the need for psychosocial support.\u003c/p\u003e \u003cp\u003eThe study has several limitations. Firstly, the sample size, while adequate for statistical analysis within the scope of rare diseases, is nonetheless limited. Additionally, the KIDSCREEN-52 questionnaire focuses solely on the past week, precluding inquiries about a broader timeframe. Furthermore, since the questionnaire was administered only once in our study, the consistency of responses across repeated measurements remains unverified. Lastly, the study did not explore the relationship between patients' psychosocial needs and socioeconomic factors within families of comparable socioeconomic status, representing another limitation.\u003c/p\u003e"},{"header":"Conclusions","content":"\u003cp\u003eThe KIDSCREEN-52 tool demonstrated promise as a feasible and accepted screening tool for identifying unmet needs in children with rare inflammatory diseases. Its brevity and comprehensiveness, available in multiple languages, offers a unique opportunity to implement strategies for improving HRQoL in routine care.\u003c/p\u003e"},{"header":"Declarations","content":"\u003cp\u003e\u003cstrong\u003eFunding\u0026nbsp;\u003c/strong\u003e\u003c/p\u003e\n\u003cp\u003eInfrastructural funding was provided by the University Hospital T\u0026uuml;bingen. No other funding was received for this study.\u0026nbsp;\u003c/p\u003e\n\u003cp\u003e\u003cstrong\u003eAcknowledgements\u003c/strong\u003e\u0026nbsp;\u003c/p\u003e\n\u003cp\u003eThe authors want to thank Christine Michler and Lisa Fauser for supporting data collection.\u003c/p\u003e\n\u003cp\u003e\u003cstrong\u003eConflicts of interest\u003c/strong\u003e\u003c/p\u003e\n\u003cp\u003eJKD has received research grants and speaker\u0026rsquo;s fees from Novartis and SOBI. CR has received speaker\u0026rsquo;s fees from Novartis, Pfizer, Galapagos and sobi. TW has given invited talks by Novartis (no personal honorarium). The other authors declare no conflicts of interest.\u003c/p\u003e\n\u003cp\u003e\u003cstrong\u003eAvailability of data and materials\u0026nbsp;\u003c/strong\u003e\u003c/p\u003e\n\u003cp\u003eData is available upon request.\u003c/p\u003e\n\u003cp\u003e\u003ca href=\"http://journals.bmj.com/site/authors/editorial-policies.xhtml?_gl=1*vo2nzw*_ga*MTMyOTQ5Nzk1Ny4xNzAwNzM2MjY2*_ga_EXTSVLH45V*MTcxNjM5Njg3OC4yMi4xLjE3MTYzOTc1OTUuMzguMC44ODg5NTk3MQ..#authorship\u0026_gl=1*1v90kj9*_ga*MTMyOTQ5Nzk1Ny4xNzAwNzM2MjY2*_gid*MTU0ODQ0MTg5NS4xNzE2Mzk3NTcz\"\u003e\u003cstrong\u003eContributorship Statement\u003c/strong\u003e\u003c/a\u003e\u003c/p\u003e\n\u003cp\u003eConceptualization: \u0026Ouml;.S., J.B.K.-D., S.M.B.,TW,GE Methodology: \u0026Ouml;.S., J.B.K.-D., S.M.B., TW, Formal analysis: \u0026Ouml;.S. Writing\u0026mdash;original draft preparation: \u0026Ouml;.S., Review and Editing: \u0026Ouml;.S., J.B.K.-D., S.M.B.,GE, VH,TW., CR, A-K.G M.D. Each author made substantial contributions to the manuscript and has approved the submitted version. All authors have read and agreed to the published version of the manuscript.\u003c/p\u003e\n\u003cp\u003e\u003cstrong\u003eEthics approval\u003c/strong\u003e\u003c/p\u003e\n\u003cp\u003eApproval of the ethics committee of the medical faculty at the Eberhard-Karls-University and at the University Hospital Tuebingen was obtained\u0026nbsp;(Research Ethics Board, Project Number:\u0026nbsp;No 185/2023BO2).\u003cstrong\u003e\u0026nbsp;\u003c/strong\u003e\u003c/p\u003e\n\u003cp\u003e\u003cstrong\u003eConsent for publication\u003c/strong\u003e\u003c/p\u003e\n\u003cp\u003eNot applicable.\u003c/p\u003e"},{"header":"References","content":"\u003col\u003e\u003cli\u003e\u003cspan\u003e\u003cem\u003eCouncil of the European Union. Council recommendation on action in the field of rare diseases\u0026mdash; 2947th employment, social policy, health and consumer affairs\u0026mdash;council meeting. 2009\u003c/em\u003e. 2009.\u003c/span\u003e\u003c/li\u003e \u003cli\u003e\u003cspan\u003eValdez R, Ouyang L, Bolen J. Public Health and Rare Diseases: Oxymoron No More. Prev Chronic Dis. 2016;13:E05.\u003c/span\u003e\u003c/li\u003e \u003cli\u003e\u003cspan\u003eBoettcher J et al. \u003cem\u003eEvaluation of two family-based intervention programs for children affected by rare disease and their families - research network (CARE-FAM-NET): study protocol for a rater-blinded, randomized, controlled, multicenter trial in a 2x2 factorial design.\u003c/em\u003e BMC Fam Pract, 2020. 21(1): p. 239.\u003c/span\u003e\u003c/li\u003e \u003cli\u003e\u003cspan\u003eNguengang Wakap S, et al. Estimating cumulative point prevalence of rare diseases: analysis of the Orphanet database. Eur J Hum Genet. 2020;28(2):165\u0026ndash;73.\u003c/span\u003e\u003c/li\u003e \u003cli\u003e\u003cspan\u003eLainka E, et al. Analysis of cryopyrin-associated periodic syndromes (CAPS) in German children: epidemiological, clinical and genetic characteristics. Klin Padiatr. 2010;222(6):356\u0026ndash;61.\u003c/span\u003e\u003c/li\u003e \u003cli\u003e\u003cspan\u003eHiraki LT, et al. Prevalence, incidence, and demographics of systemic lupus erythematosus and lupus nephritis from 2000 to 2004 among children in the US Medicaid beneficiary population. Arthritis Rheum. 2012;64(8):2669\u0026ndash;76.\u003c/span\u003e\u003c/li\u003e \u003cli\u003e\u003cspan\u003eLachmann HJ, et al. The phenotype of TNF receptor-associated autoinflammatory syndrome (TRAPS) at presentation: a series of 158 cases from the Eurofever/EUROTRAPS international registry. Ann Rheum Dis. 2014;73(12):2160\u0026ndash;7.\u003c/span\u003e\u003c/li\u003e \u003cli\u003e\u003cspan\u003eThierry S, et al. Prevalence and incidence of juvenile idiopathic arthritis: a systematic review. Joint Bone Spine. 2014;81(2):112\u0026ndash;7.\u003c/span\u003e\u003c/li\u003e \u003cli\u003e\u003cspan\u003eMoegle C, Severac F, Lipsker D. Epidemiology of juvenile dermatomyositis in Alsace. Br J Dermatol. 2020;182(5):1307\u0026ndash;8.\u003c/span\u003e\u003c/li\u003e \u003cli\u003e\u003cspan\u003eGallego E et al. Familial Mediterranean Fever in Spain: Time Trend and Spatial Distribution of the Hospitalizations. Int J Environ Res Public Health, 2023. 20(5).\u003c/span\u003e\u003c/li\u003e \u003cli\u003e\u003cspan\u003eBaumbusch J, Mayer S, Sloan-Yip I. Alone in a Crowd? Parents of Children with Rare Diseases' Experiences of Navigating the Healthcare System. J Genet Couns; 2018.\u003c/span\u003e\u003c/li\u003e \u003cli\u003e\u003cspan\u003eWitt S, et al. Rare pediatric diseases and pathways to psychosocial care: a qualitative interview study with professional experts working with affected families in Germany. Orphanet J Rare Dis. 2021;16(1):497.\u003c/span\u003e\u003c/li\u003e \u003cli\u003e\u003cspan\u003eErbis G, et al. Living with autoinflammatory diseases: identifying unmet needs of children, adolescents and adults. Pediatr Rheumatol Online J. 2018;16(1):81.\u003c/span\u003e\u003c/li\u003e \u003cli\u003e\u003cspan\u003eRavens-Sieberer U, et al. KIDSCREEN-52 quality-of-life measure for children and adolescents. Expert Rev Pharmacoecon Outcomes Res. 2005;5(3):353\u0026ndash;64.\u003c/span\u003e\u003c/li\u003e \u003cli\u003e\u003cspan\u003eRavens-Sieberer U, et al. The KIDSCREEN-52 quality of life measure for children and adolescents: psychometric results from a cross-cultural survey in 13 European countries. Value Health. 2008;11(4):645\u0026ndash;58.\u003c/span\u003e\u003c/li\u003e \u003cli\u003e\u003cspan\u003eRavens-Sieberer U, et al. The European KIDSCREEN approach to measure quality of life and well-being in children: development, current application, and future advances. Qual Life Res. 2014;23(3):791\u0026ndash;803.\u003c/span\u003e\u003c/li\u003e \u003cli\u003e\u003cspan\u003eDeissler A, et al. Health-Related Quality of Life of Children/Adolescents with Vertigo: Retrospective Study from the German Center of Vertigo and Balance Disorders. Neuropediatrics. 2017;48(2):91\u0026ndash;7.\u003c/span\u003e\u003c/li\u003e \u003cli\u003e\u003cspan\u003eBerman AH, et al. Children's Quality of Life Based on the KIDSCREEN-27: Child Self-Report, Parent Ratings and Child-Parent Agreement in a Swedish Random Population Sample. PLoS ONE. 2016;11(3):e0150545.\u003c/span\u003e\u003c/li\u003e \u003cli\u003e\u003cspan\u003eHelseth S, Haraldstad K, Christophersen KA. A cross-sectional study of Health Related Quality of Life and body mass index in a Norwegian school sample (8\u0026ndash;18 years): a comparison of child and parent perspectives. Health Qual Life Outcomes. 2015;13:47.\u003c/span\u003e\u003c/li\u003e \u003cli\u003e\u003cspan\u003eEiser C, Morse R. Can parents rate their child's health-related quality of life? Results of a systematic review. Qual Life Res. 2001;10(4):347\u0026ndash;57.\u003c/span\u003e\u003c/li\u003e \u003cli\u003e\u003cspan\u003eMeyer M, et al. Health-related quality of life in children and adolescents: Current normative data, determinants and reliability on proxy-report. J Paediatr Child Health. 2016;52(6):628\u0026ndash;31.\u003c/span\u003e\u003c/li\u003e \u003cli\u003e\u003cspan\u003eRiley AW. Evidence that school-age children can self-report on their health. Ambul Pediatr. 2004;4(4 Suppl):371\u0026ndash;6.\u003c/span\u003e\u003c/li\u003e \u003cli\u003e\u003cspan\u003eMichel G, et al. Age and gender differences in health-related quality of life of children and adolescents in Europe: a multilevel analysis. Qual Life Res. 2009;18(9):1147\u0026ndash;57.\u003c/span\u003e\u003c/li\u003e\u003c/ol\u003e"}],"fulltextSource":"","fullText":"","funders":[],"hasAdminPriorityOnWorkflow":false,"hasManuscriptDocX":true,"hasOptedInToPreprint":true,"hasPassedJournalQc":"","hasAnyPriority":false,"hideJournal":true,"highlight":"","institution":"","isAcceptedByJournal":false,"isAuthorSuppliedPdf":false,"isDeskRejected":"","isHiddenFromSearch":false,"isInQc":false,"isInWorkflow":false,"isPdf":false,"isPdfUpToDate":true,"isWithdrawnOrRetracted":false,"journal":{"display":true,"email":"[email protected]","identity":"researchsquare","isNatureJournal":false,"hasQc":true,"allowDirectSubmit":true,"externalIdentity":"","sideBox":"","snPcode":"","submissionUrl":"/submission","title":"Research Square","twitterHandle":"researchsquare","acdcEnabled":true,"dfaEnabled":false,"editorialSystem":"","reportingPortfolio":"","inReviewEnabled":false,"inReviewRevisionsEnabled":true},"keywords":"Rare diseases, Inflammation, children, health-related quality of life (HRQoL), KIDSCREEN-52 tool, feasibility, acceptance","lastPublishedDoi":"10.21203/rs.3.rs-4616034/v1","lastPublishedDoiUrl":"https://doi.org/10.21203/rs.3.rs-4616034/v1","license":{"name":"CC BY 4.0","url":"https://creativecommons.org/licenses/by/4.0/"},"manuscriptAbstract":"\u003cp\u003e\u003cstrong\u003eBackground:\u003c/strong\u003e Children living with rare diseases often face significant psychosocial challenges; recognizing and addressing these effectively is crucial. However, there is a paucity of comprehensive screening tools. This study aimed to assess the feasibility and acceptance of the comprehensive KIDSCREEN-52 tool in identifying unmet needs of children with rare inflammatory diseases and their caregivers and identifying factors associated with low health-related quality of life (HRQoL).\u003c/p\u003e\n\u003cp\u003e\u003cstrong\u003eMethods\u003c/strong\u003e A prospective single-center study of consecutive pediatric patients aged 8-18 with inflammatory diseases and their caregivers was performed to assess HRQoL utilizing the multidimensional KIDSCREEN-52 self-report and proxy tool. The validated KIDSCREEN-52 tool is available in 13 languages with corresponding Norm Data. It captures HRQoL across 10 domains including 52 inquiries. HRQoL of children with rare inflammatory diseases was described utilizing the multidimensional KIDSCREEN-52 self-report and proxy tool. The feasibility and acceptability of KIDSCREEN-52 was determined using a simple, dichotomous three item acceptance tool. Factors associated with low self-reported HRQoL were explored.\u003c/p\u003e\n\u003cp\u003e\u003cstrong\u003eResults:\u003c/strong\u003e A total of 104 participants, comprising 51 pediatric patients and their 53 caregivers, were included. The patients were 35 females and 16 males, with a median age of 16 years (range: 9-18). Among them, 25 (49%) had autoinflammatory diseases, 26 (51%) had rheumatic diseases. Mean values from self-reports and proxies were consistent with the Norm Data across all domains. Self-report and proxy assessments showed high-degree agreement. Patients reported lower HRQoL levels compared to the control population in nearly all domains. Both caregivers and children expressed strong acceptance of the KIDSCREEN-52 questionnaire's clarity, relevance, and adequacy. The overall completion rate was 75%, the mean completion time 17 minutes (range: 10-25). Factors associated with low HRQoL included female gender, adolescent age and evidence of a rheumatic disease.\u003c/p\u003e\n\u003cp\u003e\u003cstrong\u003eConclusion:\u003c/strong\u003e The KIDSCREEN-52 demonstrated promise as a feasible and accepted tool for capturing the HRQoL and identifying unmet needs in children with rare inflammatory diseases. Its comprehensiveness and the availability in multiple languages with corresponding Norm Data, offers a unique opportunity to implement strategies to identify and address HRQoL challenges of children with rare diseases in routine clinical care.\u003c/p\u003e","manuscriptTitle":"Feasibility and acceptance of KIDSCREEN-52 as a screening tool for unmet needs in children with rare inflammatory diseases","msid":"","msnumber":"","nonDraftVersions":[{"code":1,"date":"2024-07-19 01:42:06","doi":"10.21203/rs.3.rs-4616034/v1","editorialEvents":[{"type":"communityComments","content":0}],"status":"published","journal":{"display":true,"email":"[email protected]","identity":"researchsquare","isNatureJournal":false,"hasQc":true,"allowDirectSubmit":true,"externalIdentity":"","sideBox":"","snPcode":"","submissionUrl":"/submission","title":"Research Square","twitterHandle":"researchsquare","acdcEnabled":true,"dfaEnabled":false,"editorialSystem":"","reportingPortfolio":"","inReviewEnabled":false,"inReviewRevisionsEnabled":true}}],"origin":"","ownerIdentity":"72d63b89-abb6-4a48-bb9c-f5bcb1b26736","owner":[],"postedDate":"July 19th, 2024","published":true,"recentEditorialEvents":[],"rejectedJournal":[],"revision":"","amendment":"","status":"posted","subjectAreas":[],"tags":[],"updatedAt":"2025-05-05T13:08:19+00:00","versionOfRecord":[],"versionCreatedAt":"2024-07-19 01:42:06","video":"","vorDoi":"","vorDoiUrl":"","workflowStages":[]},"version":"v1","identity":"rs-4616034","journalConfig":"researchsquare"},"__N_SSP":true},"page":"/article/[identity]/[[...version]]","query":{"redirect":"/article/rs-4616034","identity":"rs-4616034","version":["v1"]},"buildId":"qtupq5eGEP_6zYnWcrvyt","isFallback":false,"isExperimentalCompile":false,"dynamicIds":[84888],"gssp":true,"scriptLoader":[]}