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Introduction One-stop surgery (OSS) allows for same-day outpatient clinic visit, preoperative assessment, and surgical repair. This study aims to determine the efficiency, (cost-)effectiveness, and family satisfaction of one-stop inguinal hernia surgery compared with usual care. Material and Methods Children (≥ 3 months) with inguinal hernia and American Society of Anesthesiologists (ASA) grades I–II, scheduled for OSS (intervention) or regular treatment (control) between March 1, 2017, and December 1, 2018, were eligible for inclusion. Exclusion criteria consisted of age less than 3 months and ASA grades III–IV. The primary outcome measure was treatment efficiency (i.e., total number of hospital visits and waiting time [days] between referral and surgery). Secondary outcome measures were the effectiveness in terms of complication and recurrence rate, and parent-reported satisfaction and cost-effectiveness using the Dutch Pediatric Quality of Life Healthcare Satisfaction and Institute for Medical Technology Assessment Productivity Cost Questionnaire. Results Ninety-one (intervention: 54; control: 37) patients (56% boys) were included. Median (interquartile range) number of hospital visits was lower in the intervention group (1 vs 3; p Conclusions Pediatric one-stop inguinal hernia repair seems to be an effective treatment strategy that limits the number of hospital visits and provides enhanced family satisfaction without compromising the quality of care.

Objectives: The aim of the study was to describe the longitudinal development of health-related quality of life (HRQOL) and fatigue in children with chronic intestinal failure (CIF) on home parenteral nutrition (PN) and compare these children to the general population. Methods: Prospective, observational study conducted over 7 years in patients suffering from CIF receiving home PN from 2 tertiary hospitals in the Netherlands. Every 6 months, parents (if child

Introduction The KLIK Patient Reported Outcome Measures (PROM) portal (www.hetklikt.nu) has been implemented since 2011 in clinical practice in over 20 Dutch hospitals. Patients and/or parents complete PROMs before the outpatient consultation and answers are subsequently discussed by clinicians during consultation. This study aims to provide insight into patients’ and parents’ perspective on the use of the KLIK PROM portal in order to optimize its implementation in pediatric clinical practice. Methods Patients (12–19 years) and parents (of children 0–19 years) from the Emma Children’s Hospital were invited to participate. A mixed-method design was used; (1) Focus groups were held and analyzed using thematic analysis in psychology, (2) a questionnaire was sent out and analyzed using descriptive statistics. Results (1) Eight patients and 17 parents participated. Patients mentioned that KLIK has an attractive layout. However, PROMs were sometimes considered irrelevant and repetitive. Parents valued that KLIK provides insight into their child’s functioning, but they were not satisfied with the extent to which PROMs were discussed by clinicians. (2) 31 patients and 130 parents completed the questionnaire. Overall, patients and parents reported a satisfaction score of 7.9/10 and 7.3/10, respectively. 81% of patients and 74% of parents indicated that KLIK is easy to use. Conclusion Patients and parents are generally satisfied with KLIK, however, points of improvement were mentioned. These are currently being addressed by e.g., upgrading the KLIK website, implementing PROMIS item banks in KLIK to reduce irrelevancy and repetitiveness of PROMs, and implementation strategies to improve the discussion-rate. In this way, implementation of the KLIK PROM portal can be further optimized, with the ultimate goal to improve quality of care.

Purpose Since 2011, the evidence-based KLIK Patient Reported Outcome Measure (PROM) portal has been implemented in clinical practice in > 20 Dutch hospitals. Patients and/or parents complete PROMs on Health Related Quality of Life, symptoms and psychosocial functioning before their outpatient consultation. Answers are converted into an ePROfile and discussed by clinicians during consultation to monitor well-being over time and detect problems early. This study aims to get insight into the KLIK implementation from the clinician’s perspective. Methods As part of the KLIK implementation process, annual meetings were held with multidisciplinary teams to evaluate the use of KLIK. An online questionnaire was sent regarding (1) overall satisfaction, (2) feeling competent to discuss PROMs, (3) use of KLIK during the consultation, (4) influence of KLIK on the consultation, (5) usability of the KLIK PROM portal, (6) satisfaction with PROMs and feedback, and (7) support of the KLIK expert team. Open questions about (dis)advantages were included. Descriptive analyses were used. Results One hundred and forty-eight clinicians (response-rate 61%) from 14 hospitals in the Netherlands participated. Results show that: (1) clinicians report an overall satisfaction of median = 69/100 (visual analogue scale), (2) 85.8% feel competent discussing the ePROfile, (3) 70.3% (almost) always discuss the ePROfile, (4) 70.3% think that KLIK improves consultation, (5) 71.6% think KLIK is easy to use, (6) 80.4% are satisfied with the feedback of the overall KLIK ePROfile, (7) 71.6% experience sufficient support of the KLIK team. Conclusion Participating clinicians are generally satisfied with KLIK. Improvements to the KLIK PROM portal are now realized based on the mentioned disadvantages (e.g., shorten PROM completion by use of PROMIS and integrating KLIK with Electronic Health Records).

Purpose During the COVID-19 pandemic in the Netherlands, governmental regulations resulted in a lockdown for adults as well as children/adolescents. Schools were closed and contact with other people was limited. In this cross-sectional, population-based study, we aimed to investigate the mental/social health of children/adolescents during COVID-19 lockdown. Methods Two representative samples of Dutch children/adolescents (8–18 years) before COVID-19 (2018, N = 2401) and during lockdown (April 2020, N = 844) were compared on the Patient-Reported Outcomes Measurement Information System (PROMIS) domains: global health, peer relationships, anxiety, depressive symptoms, anger, sleep-related impairment by linear mixed models and calculating relative risks (RR (95% CI)) for the proportion of severe scores. Variables associated with worse mental/social health during COVID-19 were explored through multivariable regression models. The impact of COVID-19 regulations on the daily life of children was qualitatively analyzed. Results Participants reported worse PROMIS T-scores on all domains during COVID-19 lockdown compared to before (absolute mean difference range 2.1–7.1 (95% CI 1.3–7.9). During lockdown, more children reported severe Anxiety (RR = 1.95 (1.55–2.46) and Sleep-Related Impairment (RR = 1.89 (1.29–2.78) and fewer children reported poor Global Health (RR = 0.36 (0.20–0.65)). Associated factors with worse mental/social health were single-parent family, ≥ three children in the family, negative change in work situation of parents due to COVID-19 regulations, and a relative/friend infected with COVID-19. A large majority (> 90%) reported a negative impact of the COVID-19 regulations on daily life. Conclusion This study showed that governmental regulations regarding lockdown pose a serious mental/social health threat on children/adolescents that should be brought to the forefront of political decision-making and mental healthcare policy, intervention, and prevention.

OBJECTIVES: Health-related quality of life (HRQOL) of children with avoidant restrictive food intake disorder (ARFID) is impaired. AIM: To measure HRQOL and distress of parents of children with ARFID. METHODS: Cross-sectional cohort study. Parents of children with ARFID, visiting our multidisciplinary feeding team, completed questionnaires on the online Quality of Life in Clinical Practice portal; the Questionnaire for Adult's Health Related Quality of Life to assess parental HRQOL and the Distress Thermometer for Parents. Reference groups of parents of healthy (HC) and chronically ill children (CIC) were used. RESULTS: Eighty-five mothers and 62 fathers of 89 children with ARFID (58% female, median age 1.9 years) were included (response rate 68%). No differences were found regarding HRQOL in 11 of 12 domains between parents of children with ARFID and HC. Mothers of children with ARFID reported significantly higher HRQOL regarding pain and fathers a significantly lower HRQOL on depressive emotions compared to HC. No differences were found in overall and clinical distress scores between parents of children with ARFID and HC/CIC. Mothers of children with ARFID had significantly higher distress scores regarding cognitive problems compared to HC and parenting problems in children

Young people perinatally infected with HIV (pHIV) are at risk of a lowered health-related quality of life (HRQOL). Previous evaluation of the NeurOlogical, VIsual and Cognitive performance in HIV-infected Children (NOVICE)-cohort showed no difference in HRQOL between pHIV and matched HIV-uninfected controls (HIV-), yet a higher percentage of pHIV had impaired HRQOL. The aim of this study is to compare the change over time in HRQOL of pHIV to HIV- over a 5-year period. We used the Pediatric Quality of Life Inventory (PedsQL)™ 4.0 to repeat HRQOL assessment. High PedsQL scores indicate good HRQOL. Fifteen/33 (45.5%) pHIV and 17/37 (45.9%) HIV- completed both assessments. At the first assessment, the mean age was 13.1 years (range 8.0–18.4). PHIV scored higher than HIV- on Emotional functioning and on Total scale score. After five years, the mean age was 17.6 years (range 12.1–22.8). PHIV scored higher than HIV- on all scales, except Social functioning. PHIV did not differ significantly from the Dutch norm on either time-point. LMEM showed no difference in change over time for any of the PedsQL scales. In this study, young people with pHIV receiving high-quality health care, including monitoring of HRQOL, remain to experience a good HRQOL.

Marfan syndrome (MFS) is a multisystemic, autosomal dominant connective tissue disorder that occurs de novo in 25%. In many families, parent and child(ren) are affected, which may increase distress in parents. To assess distress, 42 mothers (29% MFS) and 25 fathers (60% MFS) of 43 affected children, completed the validated screening‐questionnaire Distress thermometer for parents of a chronically ill child, including questions on overall distress (score 0–10; ≥4 denoting “clinical distress”) and everyday problems (score 0–36). Data were compared to 1,134 control‐group‐parents of healthy children. Mothers reported significantly less overall distress (2, 1–4 vs. 3, 1–6; p = .049; r = −.07) and total everyday problems (3, 0–6 vs. 4, 1–8; p = .03; r = −.08) compared to control‐group‐mothers. Mothers without MFS reported significantly less overall distress compared to mothers with MFS, both of a child with MFS (1, 0–4 vs. 3.5, 2–5; p = .039; r = −.17). No significant differences were found between the father‐groups, nor between the group of healthy parents of an affected child living together with an affected partner compared to control‐group‐parents. No differences in percentages of clinical distress were reported between mothers and control‐group‐mothers (33 vs. 42%); fathers and control‐group‐fathers (28 vs. 32%); nor between the other groups. Distress was not associated with the children's MFS characteristics. Concluding, parents of a child with MFS did not show more clinical distress compared to parents of healthy children. However, clinical distress was reported in approximately one‐third and may increase in case of acute medical complications. We advise monitoring distress in parents of a child with MFS to provide targeted support.

Introduction Multidisciplinary management of women‐specific bleeding is important to preserve quality of life, healthy reproduction and social participation of women and girls with bleeding disorders (WBD). Aim To support appropriate multidisciplinary care for WBD in haemophilia treatment centres. Methods Two case examples are presented and management issues discussed from different health care perspectives, including the nurse, patient, psychologist, gynaecologist, geneticist, psychosexual therapist and haematologist. Results Woman with bleeding disorders may experience heavy menstruation from menarche onwards. This has a physical and psychosocial impact requiring a multidisciplinary approach. If a woman with an inherited bleeding disorder desires to become pregnant, preconception counselling is essential, to discuss genetic diagnosis, state of the art treatment options for the bleeding disorder in question and possible choices to prevent having an affected child, as well as maternal bleeding risks during conception, delivery and the post‐partum period. Conclusion Adequate management and good education of WBD requires a patient‐centred multidisciplinary approach with experienced specialists in a haemophilia treatment centre.

Objective: To assess the psychometric properties of 8 pediatric Patient-Reported Outcomes Measurement Information System (PROMIS) item banks in a clinical sample of children with juvenile idiopathic arthritis (JIA). Methods: A total of 154 Dutch children (mean ± SD age 14.4 ± 3.0 years; range 8–18 years) with JIA completed 8 pediatric version 1.0 PROMIS item banks (anger, anxiety, depressive symptoms, fatigue, pain interference, peer relationships, physical function mobility, physical function upper extremity) twice and the Pediatric Quality of Life Inventory (PedsQL) and the Childhood Health Assessment Questionnaire (C-HAQ) once. Structural validity of the item banks was assessed by fitting a graded response model (GRM) and inspecting GRM fit (comparative fit index [CFI], Tucker-Lewis index [TLI], and root mean square error of approximation [RMSEA]) and item fit (S-X2 statistic). Convergent validity (with PedsQL/C-HAQ subdomains) and discriminative validity (active/inactive disease) were assessed. Reliability of the item banks, short forms, and computerized adaptive testing (CAT) was expressed as the SE of theta (SE[θ]). Test–retest reliability was assessed using intraclass correlation coefficients (ICCs) and smallest detectable change. Results: All item banks had sufficient overall GRM fit (CFI >0.95, TLI >0.95, RMSEA 2 P > 0.001). High correlations (>0.70) were found between most PROMIS T scores and hypothesized PedsQL/C-HAQ (sub)domains. Mobility, pain interference, and upper extremity item banks were able to discriminate between patients with active and inactive disease. Regarding reliability, PROMIS item banks outperformed legacy instruments. Post hoc CAT simulations outperformed short forms. Test–retest reliability was strong (ICC >0.70) for all full-length item banks and short forms, except for the peer relationships item bank. Conclusion: The pediatric PROMIS item banks displayed sufficient psychometric properties for Dutch children with JIA. PROMIS item banks are ready for use in clinical research and practice for children with JIA.

Preconception expanded carrier screening (ECS) aims to detect carrier couples of autosomal recessive (AR) disorders before pregnancy in order to increase reproductive autonomy of prospective parents. Genetic knowledge and knowledge gained from experience influence decision making on participation in genetic testing and understanding carrier test results. In this study we assessed whether parents and relatives of patients with the severe AR condition mucopolysaccharidosis type III (MPS III), who are expected to have genetic and experiential knowledge, have more positive attitudes toward ECS than the Dutch reference group. Parents of all MPS III patients known to the Dutch expert center were invited to participate and asked to invite first and second degree relatives. The online questionnaire started with an educational text, and assessed attitudes toward ECS, genetic knowledge and perceived MPS III severity. Results were compared with the Dutch population. Parents and relatives of MPS III patients (n = 159) scored higher on the genetic knowledge test and perceived MPS III as more severe compared with the general Dutch population (n = 781). Parents and relatives reported to be more likely to participate in ECS (84.3% and 62.5%, respectively) compared with the public (31%) (p ' 0.001). Being a relative of a MPS III patient was the strongest variable in the regression analyses for intended ECS participation. Our results show that genetic knowledge influences ECS decision making. Therefore, appropriate information on ECS and genetic counseling is needed to enable prospective parents from the general population, including relatives of patients with severe hereditary disorders, to make informed decisions.

Introduction and Aim Knowledge on patterns of beliefs about the illness (illness cognitions) can provide insight into individual differences in adjustment to haemophilia. The current study aimed to identify (a) which sociodemographic and disease characteristics were associated with illness cognitions and (b) which illness cognitions were associated with health-related quality of life (HRQOL) in young adult men with haemophilia, besides sociodemographic and disease characteristics. Methods Young adult men (18-30 years) with haemophilia in the Netherlands participated in an online multicentre cross-sectional study. Participants completed the Pediatric Quality of Life Inventory Young Adult version (PedsQL_YA). Potential sociodemographic determinants were assessed with the Course of Life Questionnaire (CoLQ) and illness cognitions with the Illness Cognition Questionnaire (ICQ). Multiple linear regression analyses were performed to assess potential determinants of illness cognitions and HRQOL. Results Seventy young adult men with haemophilia (mean age 24.7 years, SD 3.5) participated. Born outside the Netherlands (beta-0.24) and >1 bleed past 6 months (beta-0.32) were associated with less acceptance of the disease. More acceptance was associated with better HRQOL in all domains:beta 0.23-0.39. More helplessness was associated with worse total (beta-0.30) and physical (beta-0.42) HRQOL. Disease benefits, sociodemographic and disease characteristics were not associated with HRQOL. Conclusion Illness cognitions are associated with HRQOL in young adult men with haemophilia. Early recognition and identification of illness cognitions are important to facilitate support and psychosocial treatment to optimize young adults' well-being. Extra attention is needed for young adult men with frequent bleeds because they are at risk of lowered levels of acceptance of the disease.

Objectives Parents of children with a chronic illness (CI) are at risk for psychosocial problems. The aim of this study was to refine an existing face-to-face intervention into an online psychosocial group intervention for parents by (1) exploring which themes are important, (2) determine what type of intervention parents would like and (3) assess parents’ practical preferences. Methods Parents of children with a CI (0–18 years) were invited to complete an online questionnaire. To acquire more in-depth information, focus groups and telephone interviews were conducted. Descriptive statistics were used. Results 272 parents (mean age = 43.1 years, 85% female) participated. Three focus groups (15 parents) and seven telephone interviews were conducted. Most important themes were: the CI of the child, family functioning, taking care of yourself, relationships with others and practical support. Parents preferred a group with parents of children in the same age category. At first, parents preferred face-to-face contact. After an explanation and demonstration of an online intervention, parents became more positive about online support, mostly because they could participate from home. Conclusions for Practice Parents have a need for psychosocial support focusing on different themes. Professionals should explain and demonstrate an online intervention to parents. Based on these results, Op Koers Online for parents was developed. An RCT to assess feasibility and effectiveness of the intervention is currently running.

Background Parents of children with severe inborn errors of metabolism (IEMs) frequently face stressful events related to the disease of their child and are consequently at high risk for developing parental posttraumatic stress disorder (PTSD). Assessment and treatment of posttraumatic stress disorder (PTSD) in these parents is however not yet common in clinical practice. PTSD can be effectively treated by Eye Movement Desensitization and Reprocessing (EMDR), mostly offered in multiple weekly sessions which may preclude participation as parents are generally overburdened by the ongoing and intensive care for their child. We offered time-limited EMDR therapy with a maximum of four sessions over two subsequent days to two parents (from different families) of mucopolysaccharidosis type III (MPS III) patients to explore the potential effect of this approach in reducing post-traumatic stress symptoms and comorbid psychological distress. Methods Both qualitative and quantitative outcomes were used. The case conceptualisation, EMDR sessions and the effects reported by parents are described. The change in the severity of post-traumatic stress symptoms and comorbid psychological distress were evaluated with the Reliable Change Index (RCI). Results All traumatic memories and catastrophic fears of the future reported by parents were successfully processed and neutralized. Parents felt more competent to face future difficulties related to the disease of their child, and no adverse effects were reported. Quantitative outcomes showed a clinically significant decrease in post-traumatic stress symptoms and comorbid psychological distress from pre- to post treatment, and these beneficial effects were maintained at follow-up. Conclusion Time-limited EMDR might be a successful treatment for traumatized parents of children with mucopolysaccharidosis type III, and we suggest that this approach may have a wider application including parents of children with other severe IEMs. More awareness in clinical practice of the need for assessment and treatment of PTSD in parents of children with IEMs is essential to improve the psychosocial wellbeing of both parent and child.

Background: Legacy hemophilia-specific questionnaires are considered too long, show floor-/ceiling effects, and/or include irrelevant questions. Patient Reported Outcomes Measurement Information System (PROMIS) item banks, including Computer Adaptive Tests (CATs) and short forms, were designed for more efficient outcome assessment. Objectives: Evaluate the feasibility, measurement properties, and relevance of seven PROMIS CATs and two short forms in patients with hemophilia. Patients/Methods: In this cross-sectional study, Dutch adults with hemophilia completed nine PROMIS item banks electronically. Feasibility was assessed by number of items and floor/ceiling effects. Reliability was determined as the proportion of reliable scores (standard error ≤3.2). Construct validity was assessed by comparison with legacy instruments and expected differences between subgroups. Relevance of item banks was determined by proportions of limited scores. Results: Overall, 142 of 373 invited patients (mean age, 47 [range, 18-79], 49% severe hemophilia, 46% receiving prophylaxis) responded. Per CAT item bank, mean number of items answered varied from 5 (range, 3-12) to 9 (range, 5-12), with floor effects in pain interference (26% lowest scores) and depression (18% lowest scores). Construct validity and reliability were good for physical function, pain interference, satisfaction with social roles and activities, and fatigue. The CAT physical function showed the most limited scores (38%). The self-efficacy short forms showed ceiling effects (22%-28%) and no relation with the legacy instruments. Conclusions: The PROMIS CATs physical function, pain interference, satisfaction with social roles and activities, and fatigue are feasible, reliable, and valid alternatives to legacy instruments for patients with hemophilia, with few items and low floor-/ceiling effects.

Background The International Consortium for Health Outcomes Measurement (ICHOM) develops condition-specific Standard Sets of outcomes to be measured in clinical practice for value-based healthcare evaluation. Standard Sets are developed by different working groups, which is inefficient and may lead to inconsistencies in selected PROs and PROMs. We aimed to identify common PROs across ICHOM Standard Sets and examined to what extend these PROs can be measured with a generic set of PROMs: the Patient-Reported Outcomes Measurement Information System (PROMIS®). Methods We extracted all PROs and recommended PROMs from 39 ICHOM Standard Sets. Similar PROs were categorized into unique PRO concepts. We examined which of these PRO concepts can be measured with PROMIS. Results A total of 307 PROs were identified in 39 ICHOM Standard Sets and 114 unique PROMs are recommended for measuring these PROs. The 307 PROs could be categorized into 22 unique PRO concepts. More than half (17/22) of these PRO concepts (covering about 75% of the PROs and 75% of the PROMs) can be measured with a PROMIS measure. Conclusion Considerable overlap was found in PROs across ICHOM Standard Sets, and large differences in terminology used and PROMs recommended, even for the same PROs. We recommend a more universal and standardized approach to the selection of PROs and PROMs. Such an approach, focusing on a set of core PROs for all patients, measured with a system like PROMIS, may provide more opportunities for patient-centered care and facilitate the uptake of Standard Sets in clinical practice.

The aim of the study was to assess internalizing problems before and during the pandemic with data from Dutch consortium Child and adolescent mental health and wellbeing in times of the COVID-19 pandemic, consisting of two Dutch general population samples (GS) and two clinical samples (CS) referred to youth/psychiatric care. In each sample, measures of internalizing problems were obtained from ongoing data collections pre-pandemic (NGS= 35,357; NCS= 4,487) and twice during the pandemic, in Apr.–May 2020 (NGS= 3,938; clinical: NCS= 1,008) and in Nov.–Dec. 2020 (NGS= 1,489; NCS= 1,536), in children and adolescents (8-18 years) with parent- (Brief Problem Monitor) and/or child reports (Patient-Reported Outcomes Measurement Information System®). Results show significantly greater proportions of worrisome internalizing problems (based on validated cut-offs) and significantly higher internalizing problems mean levels from pre-pandemic to pandemic measurements in the general population. These levels stabilized or decreased over the course of the pandemic. In the clinical sample, we found an increase in child-reported internalizing problems measures over the course of the pandemic, but parents reported no differences in internalizing problems measures over the course of the pandemic, nor from pre-pandemic to during the pandemic. Overall, the findings indicate that children and adolescents of both the general and clinical population were affected negatively by the pandemic in terms of their internalizing problems. Attention is therefore warranted to investigate what long-term effects this may cause and to monitor if internalizing problems return back to pre-pandemic levels or if they remain elevated post-pandemic.

Introduction Haemophilia carriers (HCs) face considerable haemostatic and psychological challenges during reproduction. Aim To explore the perspectives of HCs on healthcare in the current standard of haemophilia treatment during all reproductive phases: preconception, pregnancy, childbirth and the postpartum period. In addition, we examined the psychological impact of haemophilia during these phases. Material and methods Focus group discussions (FGDs) and semi-structured interviews were conducted with HCs in January/February 2020 until data saturation was reached. All sessions were recorded, transcribed verbatim and analysed by two independent researchers through thematic content analysis using MAXQDA® software. The results were then discussed within the research team until consensus was reached. The constructed themes were shared with and reviewed by the HCs. Results Fifteen HCs were included in three FGDs and four interviews. Five central themes were constructed: (1) communication by healthcare professionals, (2) lack of knowledge, (3) feeling insecure, (4) autonomy and (5) family experiences with haemophilia. Desired improvements in care mainly concerned counselling during preconception and pregnancy. This included timely access to comprehensive information during each consecutive phase, acceptance of HCs' choices by healthcare providers and healthcare tailored to the HC's family experience with haemophilia. Conclusions In recent years, haemophilia treatment has seen major advances, which could impact general and reproductive care for HCs. HCs indicated that reproductive care would benefit from a more personal and informative approach. Healthcare professionals could use these insights to adapt their consultations to meet the needs of these women when they are preparing for having children.

Contains fulltext : 244120.pdf (Publisher’s version ) (Open Access) BACKGROUND: The Patient-Reported Outcomes Measurement Information System (PROMIS) Profile-29 questionnaire is widely used worldwide, but it has not yet been validated in the Netherlands, nor in persons with hemophilia. OBJECTIVE: To validate the Dutch-Flemish version of the PROMIS-29 Profile v2.01 in adults with hemophilia. METHODS: Dutch males with hemophilia (all severities) completed questionnaires that contained sociodemographic and clinical characteristics, the PROMIS-29, RAND-36, and the Hemophilia Activities List (HAL). Structural validity of each subscale was assessed with confirmatory factor analysis (CFA). Internal consistency was calculated for each subscale with sufficient model fit in CFA. Construct validity was assessed by testing hypotheses about (1) correlations of each PROMIS-29 subscale with corresponding scales of RAND-36 and domains of HAL, and (2) mean differences in T-scores between subgroups with different hemophilia severities, self-reported joint impairment, and HIV infection status. We considered ≥75% of data in accordance with the hypotheses evidence for construct validity. RESULTS: In total, 770 persons with hemophilia participated in this cross-sectional study. CFA revealed sufficient structural validity for five subscales: Physical Function, Depression, Sleep Disturbance, Ability to Participate in Social Roles and Activities, and Pain Interference. Internal consistency was high and Cronbach's alpha ranged from 0.79 for Sleep Disturbance to 0.96 for Pain Interference. Differences between clinical subgroups were in the expected direction. Construct validity was confirmed for Physical Function, Anxiety, Depression, Fatigue, Sleep Disturbance, and Pain Intensity. CONCLUSION: This study revealed sufficient evidence for structural validity, internal consistency, and construct validity for most PROMIS Profile-29 subscales among people with hemophilia in the Netherlands.

Objective The aim of this study was to compare health related quality of life (HRQOL) in infants and children with avoidant restrictive food intake disorder (ARFID) to healthy and chronically ill controls. Method A cross‐sectional study was conducted in children who meet ARFID criteria at our tertiary care pediatric feeding clinic (September 2014 to July 2016). Before consultation, parents of patients (n = 100) were asked to complete questionnaires to determine HRQOL: the TNO‐AZL Preschool Children Quality of Life (0–5 years), and “Pediatric Quality of Life Inventory” (6–7 and 8–10 years). HRQOL of ARFID patients was compared to both healthy (0–5 years n = 241; 6–7 years n = 61; 8–10 years n = 192) and chronically ill (0–5 years n = 79; 6–7 years n = 11; 8–10 years n = 26) controls. Results The prevalence of ARFID was 64%. HRQOL of ARFID patients aged 0–5 years (n = 37) was significantly lower on 6/12 scales (appetite, lungs, stomach, motor functioning, positive mood and liveliness) compared to healthy controls (P

Background A substantial number of severely debilitating and often ultimately fatal inborn errors of metabolism (IEMs) still lack an effective disease-modifying treatment. Informing couples before a pregnancy about an increased risk of having a child with an inherited disorder is now feasible by preconception expanded carrier screening (ECS). While knowledge about carrier status enhances reproductive autonomy, it may also result in ethical dilemmas. The purpose of this study was to assess the attitudes of the general Dutch population towards preconception ECS and to investigate which factors influence these attitudes. Methods Data collection was carried out in collaboration with a market research agency. In total, 1188 Dutch individuals of reproductive age (18–45 years) were invited by email to complete an online ECS questionnaire in 2016. Prior to the start of the questionnaire, a written explanation of the concepts of autosomal recessive (AR) inheritance, carrier status and ECS was presented. Results The questionnaire was completed by 781 individuals (65.7%), of whom 31% indicated they would take an ECS test themselves. In addition, 55% agreed that ECS should be offered to all prospective parents. The most frequently selected argument in favor of ECS (47.2%) was that participants want to spare a child from a life with a severe hereditary disorder. The reason most often mentioned not to participate in ECS (48%) was that participants reported not having a hereditary disorder in the family. The majority preferred receiving individual test results above a couple-based disclosure method in which participants receive the carrier status results only when they are a carrier couple of the same disorder. Participants with religious beliefs were less likely to participate in ECS, whereas participants who were considering a (future) pregnancy were more likely to participate. Conclusion Our study demonstrates an overall positive attitude among participants of reproductive age in the general Dutch population towards preconception ECS. A striking misconception is that many of the participants believe that ECS is of interest only for those with a positive family history of one of the hereditary disorders. This finding emphasizes the importance of providing understandable, balanced information and education to the general public regarding the concepts of inheritance when presenting the option of carrier screening. Our results provide valuable insights that can be used in the debate about the responsible implementation of preconception ECS for AR disorders, including IEMs.

Introduction This paper describes the different ways in which information is being fed back to health care providers (HCPs) using the Dutch evidence-based KLIK Patient-Reported Outcome Measures (PROMs) portal (www.hetklikt.nu). KLIK was initially developed for children with a chronic illness and their parents, and recently expanded for use in adult health care. Patients complete online PROMs at home about their health-related quality of life (HRQOL), symptoms and/or psychosocial functioning before an outpatient consultation with their HCP. PROMs are subsequently converted into an individual KLIK electronic (e)PROfile and discussed during the consultation to facilitate systematic attention for HRQOL. Methods for pro score interpretation KLIK includes a variety of different PROMs. The KLIK ePROfile initially consisted of literal representations of the individual PROM items in the European traffic light colors (red, orange, green) and only one graph. Over the years, the KLIK ePROfile evolved into a broader spectrum of feedback options; (1) literal representation of individual items, (2) summary scores, and (3) graphic representations (4 options). Developing recommendations for acting on pro results The primary goal of KLIK is to longitudinally monitor patient and parent self-reported or proxy-reported health outcomes, which means that the focus of KLIK is not mainly on recommending actions for PRO results. However, there are several aids that help interpretation of the KLIK ePROfile and recommendations are given for actions based on PROM scores. The main aid is the KLIK training, which includes a theoretical and a practical part. In the training, 2 tools-a decision tree and a summary of information about the KLIK ePROfile-are given to the HCPs to assist them in using KLIK. Discussion Customization of a PROM portal is needed for each different patient group and clinical setting. Because the KLIK website is flexible, every PROM (with good psychometric qualities and permission of the publisher) can be built in. However, implementing and feeding back PROMs in pediatric health care encompasses different challenges, because different versions of the same PROM are needed for children of different ages, as well as proxy and self-report versions.

Background & aims: Parents of children with intestinal failure, dependent on Home Parenteral Nutrition (HPN), may experience psychosocial problems due to the illness and intensive treatment of their child. Literature concerning psychosocial problems is scarce. Therefore, we aimed to investigate Health-Related Quality of Life (HRQOL), levels of anxiety, depression, distress and everyday problems of these mothers and fathers. Methods: A multicenter study was conducted among 37 mothers and 25 fathers of 37 children on HPN (response-rate 37/49 = 76%, mean age children = 5.1 years, SD = 4.6). Parents completed three questionnaires to measure different outcomes on the KLIK website (www.hetklikt.nu): the TNO-AZL QOL Questionnaire (TAAQOL) to measure HRQOL, the Hospital Anxiety and Depression Scale (HADS) to measure anxiety and depression, and the Distress Thermometer for Parents (DT-P) to measure distress. Scores were compared to Dutch reference mothers and fathers using Mann–Whitney U-tests. Results: No differences were found in HRQOL, measured by the TAAQOL, between HPN parents compared to the reference groups, except for the subscale ‘depressive emotions’ for mothers (p =.01) and ‘daily activities’ for fathers (p =.04). HPN mothers reported higher levels of depression compared to reference mothers (p =.001). In addition, HPN mothers and fathers reported higher levels of distress than reference mothers (p =.001) and fathers (p =.03). HPN mothers reported significantly more problems in the practical, emotional, cognitive and parenting domains, fathers in the social, emotional and parenting domains. Conclusions: On HRQOL, anxiety and depression, HPN parents generally did not show much differences compared to reference parents. However, when asked about parental distress and everyday problems, HPN treatment of their child seems highly stressful for some parents and influences daily functioning. Therefore, structural screening for parental psychosocial problems in clinical practice, e.g. using the DT-P, is necessary in order to improve the well-being of both these parents and their children dependent on HPN.

Purpose Patient-reported outcome measures (PROMs) are standardized instruments used to collect data about the subjective assessment of medical care from the patient perspective. Implementing PROMs within pediatric clinical settings has gained increasing importance as health services prioritize patient-centred pediatric care. This study explores the perspectives of pediatric solid organ transplant patients, caregivers, and healthcare practitioners (HCPs) on implementing PROMs into clinical practice. Methods Qualitative description methods were used to elicit stakeholder perspectives. Semi-structured interviews were conducted across five Canadian transplant centres. Purposive sampling was used to obtain maximum variation across age, gender, and transplant program for all participants, as well as discipline for HCPs. Results The study included a total of 63 participants [patients (n = 20), caregivers (n = 22) and HCPs (n = 21)]. Nearly all participants endorsed the implementation of PROMs to enhance pediatric transplant clinical care. Three primary roles for PROMs emerged: (1) to bring a transplant patient’s overall well-being into the clinical care conversation; (2) to improve patient communication and engagement; and, (3) to inform the practice of clinical pediatric transplant care. Insights for effective implementation included completing electronic PROMs remotely and prior to clinical appointments by patients who are eight to 10 years of age or older. Conclusions This study contributes to current research that supports the use of PROMs in clinical pediatric care and guides their effective implementation into practice. Future directions include the development, usability testing, and evaluation of a proposed electronic PROM platform that will inform future research initiatives.

ImportanceIt is unknown how a lockdown during the COVID-19 pandemic impacts children’s and adolescents’ mental and social health.ObjectiveTo compare mental and social health of children and adolescents during the COVID-19 lockdown versus before, identify associated factors, describe the change in atmosphere at home and qualitatively assess the impact of COVID-19 regulations on daily life.DesignCross-sectional study comparing two Dutch representative samples of children and adolescents (8-18 years); before COVID-19 (Dec2017-July2018) and during the COVID-19 lockdown (April/May 2020).SettingPopulation-basedParticipantsChildren and adolescents aged 8-18 years (M=13.4, 47.4% male), representative of the Dutch population on key demographics.Exposure(s)COVID-19 pandemic lockdownMain Outcome(s) and Measure(s)Patient-Reported Outcomes Measurement Information System (PROMIS®) domains: Global Health, Peer Relationships, Anxiety, Depressive Symptoms, Anger and Sleep-Related Impairment. Single item on atmosphere at home and open question regarding the impact of the regulations on the child/adolescent’s daily lifeResultsChildren and adolescents reported significantly worse PROMIS T-scores on all domains (absolute mean difference range, 2.1-7.1; absolute 95% CI range, 1.3-7.9) during the COVID-19 lockdown as compared to before COVID-19. More children reported severe Anxiety (during 16.7% vs. before 8.6%; relative risk 1.95; 95% CI 1.55-2.46) and Sleep-Related Impairment (during 11.5% vs. before 6.1%; relative risk 1.89; 95% CI 1.29-2.78). Fewer children reported poor Global Health (during 1.7 vs. before 4.6%; relative risk 0.36; 95% CI 0.20-0.65). More mental and social health complaints during the COVID-19 lockdown were found in children and adolescents growing up in a single-parent family, having ≥three children in the family, a negative change in work situation of parents due to COVID-19 regulations, and having a relative/friend infected with COVID-19. A small effect was found on atmosphere at home during the lockdown compared to before (mean difference, −3.1; 95% CI, −4.1 −−2.1). A large majority (>90%) reported a negative impact of the COVID-19 regulations on their daily life.Conclusions and RelevanceThis study showed that governmental regulations regarding lockdown pose a serious mental and social health threat on children and adolescents that should be brought to the forefront of political decision making and mental health care policy, intervention and prevention.Key pointsQuestionWhat is the impact of lockdown during the COVID-19 pandemic on mental and social health in children and adolescents compared to before COVID-19?FindingsThis population-based study shows that during the COVID-19 lockdown children and adolescents report lower mental and social health, especially on anxiety and depressive symptoms.MeaningIn proposing new COVID regulations (e.g., closing schools) governments should be mindful of the negative impact of a lockdown on mental and social health of children and adolescents.

Introduction: Integration of patient-reported outcome measures (PROMs) in routine clinical care is growing but lacks consolidated evidence around its impact on pediatric care. This systematic review aims to evaluate the impact of integrating PROMs in routine pediatric clinical care on various outcomes in pediatric clinical care. Data Sources: MEDLINE, Embase, CINAHL, PsycINFO, and Cochrane Library. Web of Science database was searched selectively to ensure extended coverage. Study Selection: We included longitudinal studies reporting on the integration of PROMs in routine pediatric clinical care of chronic diseases. Studies in languages other than English, published prior to the year 2000, and reporting on secondary data were excluded. Data Extraction: Two reviewers independently extracted data from included studies. Extracted data included citation of each study, type of healthcare setting, location of the study, characteristics of patient population, type of chronic disease, name and type of PROM, mode of administration, and reported outcomes. Results: Out of 6,869 articles, titles and abstracts of 5,416 articles and full text of 23 articles were screened in duplicate. Seven articles reporting results from six studies met eligibility criteria. Integration of PROMs increased the identification and discussion around health-related quality of life (HRQOL), especially in psychosocial and emotional domains, but showed mixed results with the impact on quality of care. No studies assessed the impact of integrating PROMs on healthcare utilization. Limitations: Due to significant heterogeneity in the studies, a meta-analysis was not conducted. Conclusions: Integrating PROMs could have a positive impact on HRQOL; however, further studies are required to determine the impact of PROMs in routine pediatric clinical care.

BACKGROUND This study was aimed at assessing fathers' and mothers' distress 6 months after a pediatric cancer diagnosis and at determining whether this is related to the level of family psychosocial risk 1 month after the diagnosis. METHODS A sample of 192 families completed the electronic Psychosocial Assessment Tool (ePAT) 1 month after the diagnosis. At 6 months after the diagnosis, 119 mothers and 98 fathers completed the Distress Thermometer for Parents (DT-P; of which n=132 had also completed the ePAT at baseline). The DT-P consists of a thermometer score ranging from 0 to 10 (with a score ≥ 4 indicating clinical distress), problem domains (total, practical, social, emotional, physical, cognitive, and parenting for children < 2 years old and for children ≥ 2 years old), and a desire for a referral. The DT-P scores of mothers and fathers were compared with the scores of a reference group of 671 mothers and 463 fathers with healthy children. Within the pediatric cancer group, the DT-P scores of families with elevated total ePAT-scores were compared with the DT-P scores of parents with universal ePAT scores. RESULTS Parents of children with cancer more often reported clinical distress on the DT-P than parents of healthy children (fathers, 59.2% vs 32.3%; P < .001; mothers, 63% vs 42.3%; P < .001) and reported more problems on all DT-P domains (P < .001 to P = .042) except for the parenting domain for children < 2 years old. Furthermore, the ePAT predicted parental distress 6 months after the diagnosis because parents with elevated ePAT scores reported more problems than parents with universal scores on the DT-P thermometer and most of the DT-P domains (P < .001 to P = 1.00). CONCLUSIONS Initial ePAT risk scores at diagnosis are predictive of future mean levels of parental distress. Cancer 2017. © 2017 American Cancer Society.

This patient-reported outcome (PRO) study reports on 102 children with Robin sequence (RS) and their parents. There has been differentiated between those with isolated RS and those with RS as part of a syndrome, and take various treatments into account. All RS families from an earlier described cohort were invited to participate. Parents and RS children completed online questionnaires regarding health-related quality of life (HRQoL), satisfaction with appearance, parental distress, and RS specific topics. Results were compared with the Dutch norm population if available. There was no major difference in HRQoL in RS children and the Dutch norm population, nor between children with isolated RS and those with RS as part of a syndrome. The latter is likely due to the large percentage of children with Stickler syndrome, and small number of RS children with intellectual disability. Parental distress was higher in RS children with syndromes compared to parents of isolated RS children. When comparing various treatments, the subgroup treated by mandibular distraction showed a tendency of lower HRQoL scores, less satisfaction with appearance, and more parental distress. Also in the NPA group parents showed a tendency of more parental distress. Subgroups for each treatment were very small, however, and firm conclusions cannot be drawn. In this study, HRQoL in RS children is demonstrated comparable to the norm population, despite variations in treatment, possibly with less favorable outcome for children who received mandibular distraction. Markedly larger studies are needed to allow more reliable comparison of PROs in various treatments, and to incorporate PROs in management guidelines to obtain optimal patient care. © 2016 Wiley Periodicals, Inc.

Introduction and Aim: Suboptimal health-related quality of life and lowered employment rates found in a previous study in young adults (YA) with congenital coagulation disorders (CCD) in the Netherlands underline the need for more insight into professional functioning of YA with CCD and into determinants of professional functioning. Methods: Young adults (18-30 years) with CCD participated in a cross-sectional study. Professional functioning was assessed with the Work Productivity and Activity Impairment questionnaire (WPAI). Potential determinants were assessed with the Course of Life Questionnaire (CoLQ), Pediatric Quality of Life Inventory Young Adult version (PedsQL_YA), Illness Cognition Questionnaire (ICQ) and Haemophilia Activities List (HAL). Logistic regression analyses were performed in the complete sample of YA with CCD, and in YA men with haemophilia separately, to examine determinants of WPAI outcomes. Results: Ninety-four YA (77 men; mean age 24.1 years, SD 3.5 and 17 women; mean age 24.5 years, SD 3.8) with CCD (74% haemophilia A/B) participated. 74.5% of YA were paid employed for on average 30 hours per week. Of these, more than a quarter reported work impairment. Older age and a non-severe type of haemophilia (in the sample of YA men with haemophilia) were associated with successful (paid) employment. No variables were associated with professional functioning (expressed as Presenteeism and Overall work impairment) in patients with CCD or haemophilia. Conclusion: Three-quarters of YA with CCD were successful in finding paid employment. Though absenteeism was low, YA with paid employment needs attention as a considerable part experienced work impairment.

IntroductionIdiopathic nephrotic syndrome (INS) is characterised by a high relapse rate up to 80% after initial response to standard therapy with corticosteroids. Steroid toxicity is common and causes a great burden of disease that negatively influences the health-related quality of life (HRQoL). Recently, studies have shown that levamisole, an anthelminthic drug, significantly improves relapse-free survival in children with frequent relapses or steroid dependency. Compared with other steroid-sparing drugs, levamisole has relatively few side effects. We hypothesise that adding levamisole to standard therapy with corticosteroids in children with a first episode of INS will prevent relapses, decrease cumulative dosage of steroids used and improve HRQoL. This paper presents the study protocol for the LEARNS study (LEvamisole as Adjuvant therapy to Reduce relapses of Nephrotic Syndrome).Methods and analysisAn international, double-blind, placebo-controlled randomised trial will be conducted in 20 participating hospitals in the Netherlands and Belgium. Participants (n=92) with a first episode of INS, aged 2–16 years, who achieve remission after 4 weeks of oral prednisolone will be randomly assigned (1:1) to receive either levamisole 2.5 mg/kg alternate day or placebo added to prednisolone (18-week tapering schedule) for a total of 24 weeks. Follow-up will be until 2 years after first presentation. Additionally, parents and/or children will fill out five HRQoL questionnaires. Primary outcome of the LEARNS study is occurrence of relapses within 12 months after first presentation. Secondary outcomes include time to first relapse, cumulative steroid dose after 2 years, safety parameters and quality of life scores.Ethics and disseminationThe trial was approved by the Medical Ethical Committee. Results of the study will be published in a peer-reviewed journal.Trial registration numberNL6826, 2017-001025-41

OBJECTIVE: The KLIK method is an online tool that monitors and discusses electronic patient-reported outcomes (ePROs), which has been shown to enhance outcomes. This study aimed (1) to determine the fidelity (ie, extent to which used as intended) of the KLIK method as implemented in outpatient pediatric cancer care and (2) to study health care professional (HCP)-reported barriers and facilitators for implementation. METHODS: Two hundred five children with newly diagnosed cancer (enrollment rate 85%) participated. At 1 (T1), 3 (T2), and 6 (T3) months after diagnosis, patients (8-18 years) or parents (of patients 0-7 years) completed health-related quality of life (HRQoL) questionnaires, which were transformed into an ePROfile and discussed by their HCP during consultations. Fidelity was determined by the following: percentage of website registrations, HRQoL questionnaires completed, and ePROfiles discussed. Implementation determinants were assessed with HCPs after the final T3 with the Measurement Instrument for Determinants of Innovations. RESULTS: Depending on the time point (T1-T3), fidelity was 86% to 89% for website registration, 66-85% for completed HRQoL questionnaires, and 56% to 62% for ePROfile discussion. Barriers were mainly related to organizational issues (eg, organizational change) and less frequently to users (eg, motivation to comply) or the intervention (compatibility). Facilitators were related to the user (eg, positive outcome expectations) and intervention (simplicity) but not to the organization. CONCLUSIONS: When implementing ePROs in outpatient pediatric oncology practice, HCPs report determinants that influence ePRO integration. To improve implementation and outcomes, tailored organizational (eg, formal ratification by management and time) and specific local (eg, individualized assessments) strategies should be developed to achieve optimal ePRO discussion.

Background Using patient-reported outcomes (PROs) in clinical practice has been shown to enhance detection of health-related quality of life problems and satisfaction with care in children with cancer. This study seeks to identify which PRO information healthcare professionals (HCPs) find useful and what the perceived barriers for routinely assessing PROs are. Procedure A total of 352 pediatric HCPs (43% male) from 52 countries completed a semistructured online 28-item questionnaire. Descriptive statistics (percentages) were used to identify highly important PRO information and perceived barriers. HCPs' perceived barriers were compared according to gender, years of work experience, and country using a Fishers exact test. Results The five highest ranked PRO topics relevant in routine assessment by HCPs were as follows: pain (98%), feeling sad or depressed (96%), overall physical symptoms (95%), problems with therapy adherence (94%), and overall emotional issues (93%). Five lowest ranked topics were as follows: difficulties praying (50%), other spiritual concerns (55, 56, and 60%), and feeling bored (60%). Barriers for assessing PROs included: time (58%), insufficient staff (49%), logistics (32%), and financial resources (26%). Providers from developing countries more often reported barriers concerning insufficient staff, logistics, and financial resources. Conclusions HCPs strongly value the use of physical and psychosocial PROs within pediatric oncology practice, but mainly perceive organizational barriers for routine assessment. To successfully integrate PROs, efforts should be made to address HCP-perceived barriers, such that patient-reported problems can be detected and timely referrals made.

Background: Mucopolysaccharidosis type III (MPS III or Sanfilippo syndrome) is a lysosomal storage disease resulting in progressive neurocognitive decline during childhood and early demise. Its diagnosis may have a great impact on parents, potentially leading to psychosocial problems such as anxiety, depression, parental distress, and posttraumatic stress. Methods: Twenty-six mothers and 19 fathers of 34 Dutch MPS III patients completed the “Hospital Anxiety and Depression Scale” (HADS), the “Distress Thermometer for Parents” (DT-P), and the “Self-Rating Scale for Posttraumatic Stress Disorders” (SRS-PTSD). Independent-sample T-tests and chi-square tests were used to assess differences between parents of MPS III patients and reference groups regarding anxiety and depression (HADS), distress (DT-P), and posttraumatic stress (SRS-PTSD). Results: Mothers met the criteria for clinically relevant anxiety (50%) and depression (34.6%) more frequently compared to reference mothers (p ¼ 0.001). Fathers more often met the criteria for clinically relevant depression (36.8%) compared to reference fathers (p ¼ 0.022). Clinically relevant distress was highly prevalent in mothers (84.6%) and fathers (68.4%) of MPS III patients compared to reference parents (p < 0.01). Finally, the prevalence of PTSD was strikingly higher in both mothers (26.9%) and fathers (15%) than reported in the general Dutch population (respectively, p < 0.001 and p < 0.05). Conclusions: We report a clinically relevant impact of parenting an MPS III patient on psychosocial functioning, which is demonstrated by high levels of anxiety, depression, distress, and a remarkably high prevalence of PTSD. Structural monitoring of the psychosocial functioning of MPS III parents is therefore essential and may be beneficial for the whole family.

Objectives: To assess health-related quality of life (HRQoL) across three renal replacement therapy modalities (preemptive transplant, non-preemptive transplant, and dialysis) in comparison with the healthy norm and other chronic health conditions, and to explore related patient factors. Study design: All prevalent end-stage renal disease (ESRD) patients aged 8–18 years who spent at least 6 months on their current treatment modality in the Netherlands, Belgium, and part of Germany were approached to complete the Pediatric Quality of Life Inventory 4.0 (PedsQL™) questionnaire. We determined the differences between groups on PedsQL™ mean scores, the proportion of children with an impaired HRQoL (≥ 1 SD lower than the healthy norm), the proportion of problems on individual items of the PedsQL™, and the effect of time on current treatment. Linear regression models were used to explore determinants of HRQoL. Results: 192 out of 278 patients (20% preemptive transplant, 58% non-preemptive transplant, 22% dialysis) filled in the PedsQL™ (response rate 69%). Independent of treatment modality, patients had significantly lower mean scores and consequently higher proportions of impaired HRQoL on almost all domains compared to the healthy norm and other chronic health conditions. Patients with a preemptive transplant only reported higher scores on physical health compared to the other treatment modalities. Having comorbidities was the most important determinant associated with lower HRQoL scores. Conclusion: Dialysis and renal transplantation both have a severe impact on the HRQoL of children with ESRD. Physicians should be aware of this continuous burden. Furthermore, to develop tailored interventions for children with ESRD, qualitative studies are needed to gain more insight in the determinants of HRQoL in the different treatment modalities., SCOPUS: ar.j, info:eu-repo/semantics/published

BACKGROUND This study assesses health-related quality of life (HRQOL), and variables associated with HRQOL, in children and adolescents with haemophilia and congenital bleeding disorders (CBD) in the Netherlands. METHODS Patients

OBJECTIVES The interrelation between the course of inflammatory bowel disease (IBD) in children and parent's distress, and the subsequent impact this may have on health-related quality of life (HRQOL) of the child is unclear. Therefore, we investigated patient's HRQOL and parental distress and the association between the course of IBD, parental distress, and HRQOL of pediatric patients with IBD. METHODS Pediatric patients with IBD (8-18 years) and parents were invited. Patients completed the Pediatric Quality of Life Inventory, and parents the Distress Thermometer for Parents, simultaneously. Disease course was expressed as current clinical disease activity or months since last IBD flare. Patient's HRQOL and parental distress were compared to healthy controls. RESULTS In total, 87 patients (71% response rate, 59% boys, median age 15.2 years) and parents were included. Patients had an impaired total HRQOL (β = 0.125, P = 0.010), driven by lower physical (0.196, P = 0.001) and school (β = 0.232, P

Background: The evaluation of health related quality of life (HRQOL) is essential for a full assessment of the influence of an illness on patients’ lives. The aim of this paper is to critically appraise and compare the measurement properties of HRQOL questionnaires studied in haemophilia. Methods: Bibliographic databases (Embase, Medline, Cinahl and PsycInfo) were searched for articles evaluating measurement properties of HRQOL questionnaires in haemophilia. Articles were excluded that did not report HRQOL measurement properties, or when

Introduction Children growing up with haemophilia are at greater risk for psychosocial problems than their healthy peers. Providing psychosocial care to children with haemophilia and their families is indispensable, since psychosocial factors can have a significant impact on health and health-related quality of life (HRQOL). Aims Our aim was to give a description of psychosocial care provided by the multidisciplinary team of the Hemophilia Comprehensive Care Centre (HCCC) at the Emma Children's Hospital in Amsterdam, the Netherlands. With this overview, other caregivers and hospitals can benefit in organizing their psychosocial care for children with haemophilia. Methods The focus of the psychosocial care provided by the multidisciplinary team is on preventing psychosocial problems and medical-related stress, and supporting and equipping the child with haemophilia and its parents with as many skills as possible to lead an independent life with a high HRQOL. Results Core elements of the psychosocial care are therefore monitoring and screening of HRQOL (e.g. in daily clinical practice via www.hetklikt.nu), psychoeducation (haemophilia camp, haemophilia school, disease-specific activities, meetings for girls, parent meetings), practical help (Emma at Work, an employment agency for adolescents and young adults; Educational Facility and school visits), psychosocial interventions (the On Track group intervention and the Haemophilia Coping and Perception Test) and individual care (psychological counselling and referrals). Conclusion By providing this overview of psychosocial support offered and by sharing this knowledge, psychosocial care can become more structured and consistent between HCCCs around the world. Potentially, processes and outcomes of care can be improved.

Contains fulltext : 174753.pdf (Publisher’s version ) (Open Access) BACKGROUND: The purpose of this study is to provide Dutch normative data and to assess internal consistency and known-groups validity for the TNO AZL Preschool Children Quality of Life (TAPQOL) and the acute version of the generic Pediatric Quality of Life Inventory (PedsQL 4.0) in Dutch young children aged 0-7 years. METHODS: Participants were selected from a panel of a large Dutch market research agency. A sample of 794 parents (response rate 61%, 39% fathers) of children (53% boys) from the general Dutch population, completed an electronic version of the TAPQOL (N = 227 infants aged 0-1 years) or PedsQL 4.0 (N = 293 toddlers aged 2-4 years and N = 274 young children aged 5-7 years). RESULTS: Except for the 'stomach' scale (alpha = .39), the TAPQOL showed acceptable to excellent internal consistency (alpha = .60-.88). The PedsQL 4.0 showed acceptable to excellent reliability in children aged 2-4 years (alpha = .60-.88) and in children aged 5-7 years (alpha = .76-.90). Children with a chronic health condition had lower scores than healthy children on 3 out of 12 domains of the TAPQOL (p = .001-.013) and on 2 out of 6 domains of the PedsQL 4.0 for children aged 2-4 years (p = .016-.04). The PedsQL 4.0 differentiated on all domains (p < .05) between children aged 5-7 years with and without a chronic health condition. CONCLUSION: In Dutch children aged 0-7 years old, HRQoL can be relialy measured with the TAPQOL and the PedsQL 4.0. However, it remains unclear whether these HRQoL instruments can distinguish between healthy children and children with a chronic health condition under the age of 5.

Several studies in adults have shown patient reported outcomes (PROs) to be effective in enhancing patient-physician communication and discussion of Health Related Quality of Life outcomes. Although less studied, positive results have been demonstrated in children. A PRO-intervention needs to be feasible in clinical practice to be successful. In the current study, 74 parents of children who successfully completed their cancer treatment and 21 pediatric oncologists (POs) evaluated a PRO-intervention and gave recommendations for future use in their practice. Most parents and POs suggested PROs to be an important part of standard care, starting during treatment, with an assessment frequency of every 3 months.

The use of patient reported outcomes (PROs) in pediatric practice is effective in increasing discussion about emotional and psychosocial functioning. This finding forms the basis for implementing KLIK: a web-based program using electronic PROs (ePROs). The aim of this article is to describe the KLIK implementation in line with the 8 methodological recommendations composed by the International Society for Quality of Life Research (ISOQOL). (1) Goal of KLIK: to monitor and screen children (aged 0–18) with chronic illnesses over extended periods of time. (2) Children aged 8–18 complete the questionnaires themselves. Parents complete the questionnaires for young children. (3) The basis is a generic HRQOL questionnaire. Disease-specific HRQOL and psychosocial questionnaires are also available. (4) A web-based mode was selected. (5) The questionnaires in KLIK are available prior to a consultation. Pediatricians retrieve the ePROfile from the website (www.hetklikt.nu) and discuss it with the patients. The ePROfile consists of a literal representation of the answers and a graphic presentation. (6) Various tools are used to aid in its interpretation. (7) All members of the multidisciplinary team receive training in how to use the website and how to adequately respond to the patient’s ePROfile. (8) Improvements to and evaluation of KLIK are ongoing. Since implementation began, 17 patient groups, 160 professionals and >1,450 patients have started using KLIK. The implementation of KLIK appears to be feasible and workable. Many pediatricians have shown an interest in using KLIK and it is therefore being expanded and adapted for different patient groups and hospitals. (PsycINFO Database Record (c) 2014 APA, all rights reserved)

The theme of ISOQOL’s 19th Annual Conference in Budapest, Hungary, was The Journey of Quality of Life Research: A Path Towards Personalized Medicine. Innovations in e-health was one of four plenary panels. E-health is changing the landscape of clinical practice and health care, but the best way to leverage the many promised benefits of emerging e-health technologies is still not clear. The Innovations in e-health panel presented emerging changes in technologies and applications that will facilitate clinical decision making, improve quality and efficiency of care, engage individuals in clinical decision making, and empower them to adopt healthy behaviors. The purpose of this paper was to present emerging trends in e-health and considerations for successful adoption of new technologies, and an overview of each of the presentations in the e-health plenary. The presentations included a personal perspective on the use of technology for self-monitoring in Parkinson’s disease, an overview of online social networks and emerging technologies, and the collection of patient-reported outcomes through web-based systems in clinical practice. The common thread across all the talks was the application of e-health tools to empower individuals with chronic disease to be actively engaged in the management of their health. Considerations regarding data ownership and privacy, universal access to e-health, interactivity between different types of e-health technologies, and tailoring applications to individual needs were explored.

Introduction The most common neurological finding in children and adults with sickle cell disease (SCD) is the presence of silent cerebral infarcts (SCIs). SCIs are, by definition, undetectable by neurologic examination and are recognized on magnetic resonance imaging (MRI) as increased signal intensity in multiple T2-weighted images (1). SCIs are associated with a decline in full-scale IQ (2, 3) and increased risk for future strokes (4). In this paper, we provide an overview of studies that have used magnetic resonance imaging (MRI) to detect SCIs in patients with SCD. We systematically review evidence on SCI prevalence, incidence and associated risk factors. Methods A comprehensive systematic search was performed of Embase, MEDLINE, Cochrane, Web of Science and Google Scholar. Publications through June 1st, 2019 were included. Search terms included synonyms for 'SCIs' and 'SCD' in various combinations. All references listed in identified papers, or noted in footnotes of data tables, were reviewed for additional articles missed by computerized database search. After removal of duplicates, 651 studies were screened on potential eligibility. Studies were included if they concerned patients of all ages with either homozygous or compound heterozygous SCD and assessed for and specifically reported MRI-detected SCIs (Figure 1). We present a weighted prevalence by study size. When multiple articles reported on a single or overlapping population sample, prevalence estimates were obtained from the article with the largest sample size to prevent duplications. Results We included 61 original papers describing results in a total of 3740 individual SCD patients. Most studies were performed in HbSS or HbSβ0thal SCD patients (n=2122). However, five studies separately reported SCI prevalence in other SCD genotypes i.e. HbSC, HbSβ+thal (n=254) and healthy controls (n=52). The pooled prevalence of SCI among all included patients with SCD (n=3740) was 28.4% (95% CI: 4.0;32.9). Data from included studies showed a statistically significant correlation (P Conclusions SCIs are common in patients with SCD with a weighted prevalence of 28.4%. Despite advancing neuroimaging technologies and therefore potentially enhanced detection of SCIs, there has been no apparent rise in SCI prevalence over the years. SCIs occur much more frequently in individuals with HbSS or HbSβ0thal than in other SCD genotypes. Analyses of risk- and protective factors showing varying results. The absence of robust findings is probably due to a combination of small sample sizes and weak associations. We believe SCIs are a poorly understood entity in patients with SCD. Despite substantial research efforts into risk- and protective factors, too little attention has been given to underlying pathophysiological mechanisms. Further research should focus on both SCI etiology in SCD as well as on clinical management of patients with evidence of these MRI lesions. Disclosures Cnossen: Pfizer: Other: Travel Grants, Research Funding; Bayer: Other: Travel Grants, Research Funding; Novo Nordisk: Research Funding; Nordic Pharma: Research Funding; CSL Behring: Other: Travel Grants, Research Funding; Sobi: Research Funding; Baxter: Other: Travel Grants, Research Funding; Shire: Other: Travel Grants, Research Funding; Takeda: Other: Travel Grants, Research Funding; Roche: Other: Travel Grants; NWO: Other: Governmental grants , ZonMW, Innovation fund and Nationale Wetenschapsagenda 2018.

IntroductionChronic diseases among children are associated with lower health-related quality of life (HRQOL) and higher utilisation of healthcare services. Integrating Patient-Reported Outcomes Measures (PROMs) in routine clinical care has been shown to reduce utilisation of healthcare services while improving patient outcomes. The objectives of our study are to: (1) identify previously implemented and evaluated PROMs for chronic conditions in paediatric settings; (2) consolidate the evidence to evaluate the impact of using PROMs on HRQOL, healthcare utilisation, patient outcomes (eg, symptoms control) and quality of care among paediatric patients with chronic conditions. The findings from this review will inform the future integration of PROMs in paediatric clinical practice.Methods and analysisWe will systematically search the following electronic databases: MEDLINE, EMBASE, CINAHL, PsychINFO and Cochrane library. Reference lists of included studies will also be searched in Web of Science (Thomson Reuters) database to ensure more complete coverage. Two reviewers will independently screen the studies and abstract the data using standardised form. Extracted data will be analysed and synthesised. Finally, a narrative synthesis of summarised data will be presented.Ethics and disseminationEthical approval is not required, as the proposed systematic review will use data from published research articles. The results of this study will be disseminated through publication in peer-reviewed journals, scientific conferences and meetings, and the lead author’s doctoral dissertation.PROSPERO registration numberCRD42018109035.

Objective To develop and validate a Distress Thermometer for Parents (DT-P) for chronically ill children and to determine a cutoff score for clinical distress. Study design Parents of a chronically ill child (0-18 years) were recruited via announcements or were actively approached at the outpatient clinics of the Emma Children's Hospital/Academic Medical Center and Vrije Universiteit Medical Center. We modeled the development of the DT-P on the Distress Thermometer used in oncology medical care. The DT-P consists of a thermometer score from 0 (no distress) to 10 (extreme distress) and a problem list (practical, social, emotional, physical, cognitive, and parenting domains). The DT-P was validated with the Hospital Anxiety and Depression Scale (HADS) and the Parenting Stress Index. Results The mean thermometer score of the 706 participating parents was 3.7 (SD 3.0). The thermometer score and the scores in the practical, emotional, physical, and cognitive problem domains were strongly related to anxiety, depression, and the total score of the HADS (0.55 ≤ r ≤ 0.72). The thermometer score and all problem domain scores were moderately-to-strongly related to the Parenting Stress Index (0.38 ≤ r ≤ 0.63). A cutoff-score of 4 correctly identified 86% of "clinical HADS cases" (sensitivity) and 67% of "nonclinical HADS cases" (specificity). Conclusions We developed the DT-P and examined its diagnostic utility in a large sample. The DT-P appeared to be a valid and useful short screening-tool for identifying parental distress.

As a paediatrician, you follow a number of children with chronic health conditions in your practice. You provide them with a variety of therapies and would like to know whether your treatments are having an impact, in particular whether there has been a change in the patient's health-related quality of life (HRQOL). HRQOL measures have the potential to augment the information that clinicians have available, to enhance their clinical decisions and assess the impact of a chronic health condition on a child's life. How should you try to capture this information? ### What is health-related quality of life? The WHO defines quality of life (QOL) as ‘a child's perception of their position in life in the context of the culture and value systems in which they live and in relation to their goals, expectations, standards and concerns’1 and HRQOL) as ‘a child's goals, expectations, standards or concerns about their overall health and health-related domains’.1 ,2 That being said, many other definitions of HRQOL have been proposed over the years, and a variety of terms are currently used.3 ,4 Although the term QOL is sometimes used interchangeably with HRQOL, QOL is actually a broader construct that encompasses aspects of life that may not be amenable to healthcare services.5 Thus, spirituality and financial resources are, for example, often included in QOL, but are not necessarily included as part of HRQOL. In this paper, we regard QOL in children as a multidimensional subjective concept that includes social, emotional, cognitive and physical functioning as well as cultural aspects of the child and family, while HRQOL incorporates measures of physical symptoms, functional status and disease impact on psychological and social functioning.6 ,7 Children growing up with chronic health conditions (or suffered a severe acute illness and experience late effects) are at greater risk for …