Distress in parents of children with first-onset steroid-sensitive nephrotic syndrome

Background:Steroid-sensitive nephrotic syndrome (SSNS) is associated with a relapsing–remitting course that can be stressful for parents. As little is known of parental distress at the first onset of SSNS, this study aims to describe parental distress and everyday problems in mothers and fathers of a child with newly diagnosed SSNS participating in a randomized controlled trial of levamisole added to corticosteroids. 'Methods: To assess distress, the Distress Thermometer for Parents (DT-P) was used, which includes questions on distress (thermometer score 0–10, ≥ 4 “clinical distress”) and presence of everyday problems in six domains: practical, social, emotional, physical, cognitive, and parenting. The DT-P was completed 4 weeks after the onset of SSNS. Total sum and individual items of everyday problems were compared with reference data from mothers and fathers of the Dutch general population. Results: There was no difference in clinically elevated parental distress between SSNS mothers (n = 37) and fathers (n = 25) and reference parents. Compared to reference fathers, fathers of a child with SSNS scored significantly higher on emotional problems (P = 0.030), while mothers experienced more parenting problems (P = 0.002). Regression analyses showed that lower parental age and having a girl with SSNS were significantly associated with more practical problems and higher distress thermometer scores, respectively. Conclusions: Four weeks after onset, SSNS mothers and fathers experience equal distress as reference parents. However, both parents endorsed significantly more everyday problems. Therefore, monitoring parental distress, even in the first weeks of the disease, could contribute to timely interventions and prevent worsening of problems. Clinical trial registry: Dutch Trial Register (https://onderzoekmetmensen.nl/en/trial/27331 ). Graphical abstract: [Figure not available: see fulltext.]</p

Health-related quality of life of children with first onset steroid-sensitive nephrotic syndrome

This study assessed HRQoL and emotional and behavioral difficulties (EBD) and associated variables in children with first onset SSNS. While relapsing steroid-sensitive nephrotic syndrome (SSNS) in children is associated with lower health-related quality of life (HRQoL), little is known about first onset. Four weeks after onset, children (2–16 years) and/or their parents who participated in a randomized placebo-controlled trial, completed the Pediatric Quality of Life Inventory 4.0 (PedsQL) and Strengths and Difficulties Questionnaire (SDQ) to measure HRQoL and EBD, respectively. Total and subscale scores and the proportion of children with impaired HRQoL (&gt; 1 SD below the mean of the reference group) or SDQ clinical scores (&lt; 10th and &gt; 90th percentile) were compared to the Dutch general population (reference group). Regression analyses were used to identify associated variables. Compared to the reference group, children 8–18 years reported significantly lower total HRQoL, and physical and emotional functioning. A large proportion (&gt; 45%) of these children had impaired HRQoL. There were no differences in HRQoL between children 2–7 years and the reference group, except for higher scores on social functioning (5–7 years). Similar proportions of SSNS and reference children scored within the clinical range of SDQ subscales. Age, sex, and steroid side-effects were negatively associated with HRQol and/or EBD. Conclusion: This study showed that HRQoL and EBD are affected in children of different ages with first onset SSNS. This calls for more awareness from healthcare providers and routinely monitoring of HRQoL and EBD in daily clinical care to prevent worsening of symptoms. Clinical trial registry: Netherlands Trial Register (https://trialsearch.who.int/ ; NTR7013), date of registration: 02 June 2018. What is Known: • Health-related quality of life (HRQoL) is lower and emotional and behavioral difficulties (EBD) is more affected in children with frequently-relapsing and steroid-dependent nephrotic syndrome. What is New: • HRQoL and EBD are affected in children with first onset steroid-sensitive nephrotic syndrome compared to a reference group of the Dutch general population. • To what extent HRQoL and EBD are affected depends on the age of the patient.</p

Psychometrics of the patient-reported outcomes measurement information system measures in hemophilia:the applicability of the pediatric item banks

Background: The use of patient-reported outcomes measures (PROMs) is important in hemophilia care, as it facilitates communication between patients and clinicians and promotes patient-centered care. Currently, a variety of PROMs with insufficient psychometric properties are used. Patient-reported outcomes measurement information system (PROMIS) measures, including Computer Adaptive Tests, were designed to measure generically and more efficiently and, therefore, are an alternative for the existing PROMs. Objectives: To assess the feasibility, measurement properties, and outcomes of 8 PROMIS pediatric measures for boys with hemophilia. Methods: In this multicenter study, boys with hemophilia completed 8 PROMIS measures and 2 legacy instruments. Feasibility was determined by the number of completed items and floor or ceiling effects (percentage of participants that achieved the lowest or highest possible score). Reliability was assessed as the percentage of scores with a SE ≤ 4.5. Construct validity was evaluated by comparing the PROMIS measures with the legacy instruments. Mean PROMIS T-scores were calculated and compared with the Dutch general population. Results: In total, 77 boys with hemophilia participated. Reliability was good for almost all PROMIS measures and legacy instruments. The total number of completed items varied from 49 to 90 for the PROMIS pediatric measures, while the legacy instruments contained 117 to 130 items. Floor and ceiling effects were observed in both the PROMIS measures (0-39.5%) and legacy instruments (0-66.7%), but were higher for the legacy instruments. Conclusions: The PROMIS pediatric measures are feasible to use for boys with hemophilia. With the use of the PROMIS measures in clinical care and research, a step toward worldwide standardization of PROM administration can be taken.</p

Patient-Reported Outcome Measures in Routine Pediatric Clinical Care: A Systematic Review

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

Including the voice of paediatric patients: Cocreation of an engagement game

Background: Engaging patients in health care, research and policy is essential to improving patient-important health outcomes and the quality of care. Although the importance of patient engagement is increasingly acknowledged, clinicians and researchers still find it difficult to engage patients, especially paediatric patients. To facilitate the engagement of children and adolescents in health care, the aim of this project is to develop an engagement game. Methods: A user-centred design was used to develop a patient engagement game in three steps: (1) identification of important themes for adolescents regarding their illness, treatment and hospital care, (2) evaluation of the draft version of the game and (3) testing usability in clinical practice. Adolescents (12–18 years) were engaged in all steps of the development process through focus groups, interviews or a workshop. These were audio-recorded, transcribed verbatim and analysed in MAXQDA. Results: (1) The important themes for adolescents (N = 15) were included: visiting the hospital, participating, disease and treatment, social environment, feelings, dealing with staff, acceptation, autonomy, disclosure and chronically ill peers. (2) Then, based on these themes, the engagement game was developed and the draft version was evaluated by 13 adolescents. Based on their feedback, changes were made to the game (e.g., adjusting the images and changing the game rules). (3) Regarding usability, the pilot version was evaluated positively. The game helped adolescents to give their opinion. Based on the feedback of adolescents, some last adjustments (e.g., changing colours and adding a game board) were made, which led to the final version of the game, All Voices Count. Conclusions: Working together with adolescents, All Voices Count, a patient engagement game was developed. This game provides clinicians with a tool that supports shared decision-making to address adolescents' wishes and needs. Patient or Public Contribution: Paediatric patients, clinicians, researchers, youth panel of Fonds NutsOhra and patient associations (Patient Alliance for Rare and Genetic Diseases, Dutch Childhood Cancer Organization) were involved in all phases of the development of the patient engagement game—from writing the project plan to the final version of the game

Including the voice of paediatric patients: Cocreation of an engagement game

Abstract Background Engaging patients in health care, research and policy is essential to improving patient‐important health outcomes and the quality of care. Although the importance of patient engagement is increasingly acknowledged, clinicians and researchers still find it difficult to engage patients, especially paediatric patients. To facilitate the engagement of children and adolescents in health care, the aim of this project is to develop an engagement game. Methods A user‐centred design was used to develop a patient engagement game in three steps: (1) identification of important themes for adolescents regarding their illness, treatment and hospital care, (2) evaluation of the draft version of the game and (3) testing usability in clinical practice. Adolescents (12–18 years) were engaged in all steps of the development process through focus groups, interviews or a workshop. These were audio‐recorded, transcribed verbatim and analysed in MAXQDA. Results (1) The important themes for adolescents (N = 15) were included: visiting the hospital, participating, disease and treatment, social environment, feelings, dealing with staff, acceptation, autonomy, disclosure and chronically ill peers. (2) Then, based on these themes, the engagement game was developed and the draft version was evaluated by 13 adolescents. Based on their feedback, changes were made to the game (e.g., adjusting the images and changing the game rules). (3) Regarding usability, the pilot version was evaluated positively. The game helped adolescents to give their opinion. Based on the feedback of adolescents, some last adjustments (e.g., changing colours and adding a game board) were made, which led to the final version of the game, All Voices Count. Conclusions Working together with adolescents, All Voices Count, a patient engagement game was developed. This game provides clinicians with a tool that supports shared decision‐making to address adolescents' wishes and needs. Patient or Public Contribution Paediatric patients, clinicians, researchers, youth panel of Fonds NutsOhra and patient associations (Patient Alliance for Rare and Genetic Diseases, Dutch Childhood Cancer Organization) were involved in all phases of the development of the patient engagement game—from writing the project plan to the final version of the game

A retrospective assessment of the KLIK PROM portal implementation using the Consolidated Framework for Implementation Research (CFIR)

Purpose: The KLIK Patient-Reported Outcome Measure (PROM) portal is an evidence-based intervention implemented in clinical practice in > 25 Dutch hospitals for patients (children and adults) who regularly visit the outpatient clinic. Implementation science frameworks can be used to understand why implementation succeeded or failed, to structure barriers and enablers, and to develop implementation strategies to overcome barriers. This paper aimed to (A) retrospectively describe determinants of successful KLIK PROM implementation using the Consolidated Framework for Implementation Research (CFIR), and (B) identify current barriers and match implementation strategies. Methods: (A) The KLIK implementation process was described retrospectively based on literature and experience, using the 39 CFIR constructs organized in five general domains: intervention characteristics, outer setting, inner setting, characteristics of individuals, and implementation process. (B) The CFIR-Expert Recommendations for Implementing Change (ERIC) Implementation Strategy Matching tool identified current barriers in the KLIK implementation and matched implementation strategies that addressed the identified barriers. Results: (A) The most prominent determinants of successful KLIK PROM implementation lie in the following CFIR domains: intervention characteristics (e.g., easy to use), characteristics of individuals (e.g., motivation), and process of implementation (e.g., support). (B) 13 CFIR constructs were identified as current barriers for implementing the KLIK PROM portal. The highest overall advised ERIC strategy for the specific KLIK barriers was to identify and prepare champions. Conclusion: Using an implementation science framework, e.g., CFIR, is recommended for groups starting to use PROMs in clinical care as it offers a structured approach and provides insight into possible enablers and barriers

The Use of Patient-Reported Outcome Measures in Daily Clinical Practice of a Pediatric Nephrology Department

(1) Background: Health-related quality of life (HRQoL) is lower in patients with chronic kidney disease (CKD) compared to the general population. In 2011, the KLIK PROM portal was implemented in the Emma Children’s Hospital to monitor and discuss HRQoL in daily care. This study describes and assesses the implementation and use of the KLIK PROM portal in the pediatric nephrology department. (2) Methods: CKD patients (self-report, if 8–18 years of age) and their parents (proxy-report, if 1–8 years) were invited to complete HRQoL patient-reported outcome measures (PROMs): TNO-AZL Preschool children Quality Of Life (TAPQOL) or Pediatric Quality of Life Inventory for Children (PedsQL). The PROMs were completed before and discussed during outpatient consultations. The adaptation rate—the proportion of patients/parents who were invited and completed at least one PROM—was calculated. Reported HRQoL scores of CKD patients were compared to the general population. (3) Results: In total, 142 patients (proxy-and self-report) were invited, 112 patients completed at least one PROM (adaptation rate 79%). Patients (n = 84 with informed consent for scientific use) with CKD reported lower HRQoL and HRQoL was more often impaired compared to the general Dutch population. (4) Conclusions: The implementation of KLIK was successful and its use is feasible for daily care. Using KLIK, HRQoL problems can be easily identified and monitored