A Novel Stakeholder Engagement Approach for Patient-centered Outcomes Research.

INTRODUCTION/OBJECTIVES: The engagement of patients and other stakeholders is a critical element in the design of patient-centered outcomes research studies. However, methodology for scalable engagement in research management particularly activities such as operationalization of principles and setting of priorities is not well-developed. The objective of this study is to describe a novel approach for scalable stakeholder engagement in research aligned with the Patient-Centered Outcomes Research Institute (PCORI) engagement principles, which was evaluated in a national clinical data research network. MATERIALS AND METHODS: Patient, patient advocate, clinician, and researcher stakeholders were recruited from clinical sites, as well as social media sites related to the 3 conditions of focus, heart failure, obesity, and Kawasaki disease. The engagement strategy was designed, implemented, and mapped to the PCORI engagement principles. Evaluation included internal assessment and quantitative measures of online engagement. RESULTS: We operationalized the PCORI principles with 12 stakeholder engagement strategies and convened stakeholder advisory boards and online research prioritization panels to determine research priorities in a rigorous, deliberative process. A total of 46 advisors (20 patients) and 339 panelists (159 patients) actively participated. There were not significant differences between patients and clinicians in level of online engagement. Nonetheless, while patients reported a slightly greater challenge with following online discussion, they overall had a more favorable opinion about use of the online format. DISCUSSION/CONCLUSION: An efficient way to engage large numbers of representative stakeholders in research is a necessary first step to assure the public of trustworthy use of data networks for health research. This paper describes a comprehensive approach to engagement in patient-centered outcomes research management that informs ongoing development of rigorous methodologies in this area.

Cognitive Development After Kawasaki Disease　- Clinical Study and Validation Using a Nationwide Population-Based Cohort.

BACKGROUND: This purpose of this study was to investigate whether Kawasaki disease (KD) increases the risk of cognitive impairment. In this clinical study, cognitive profiles were compared between KD patients, control subjects, and a nationwide population-based cohort to determine the potential correlation between KD and a subsequent diagnosis of an intellectual disability.Methods and Results:The clinical study consisted of 168 KD patients (mean age 5.6 years, 62.5% male) and 81 healthy controls (mean age 6.4 years, 54.3% male). The nationwide cohort consisted of 4,286 KD patients and 50,038 controls retrieved from the Taiwan National Health Insurance Research Database between 1996 and 2000. The clinical study sample revealed no significant difference in any developmental index or cognitive function between KD patients and controls across various age groups (P>0.05). In the nationwide cohort, Cox regression analysis showed that a diagnosis of KD did not significantly affect the likelihood of developing an intellectual disability (adjusted hazard ratio 0.87, 95% confidence interval 0.68-1.11). CONCLUSIONS: Both the clinical data and the population-based cohort consistently demonstrated that KD does not increase a child's risk of future cognitive impairment. Although the outcome of the present study is negative, caregivers and patients with KD can be reassured that KD will have no effect on developmental milestones or cognitive function later in life.

Kawasaki Disease Substantially Impacts Health-Related Quality of Life.

OBJECTIVE: To prospectively evaluate the acute impact of Kawasaki disease (KD) on health-related quality of life (HRQoL) and to assess deterioration in the HRQoL experienced by children with KD compared with other childhood diseases. STUDY DESIGN: We merged the Outcomes Assessment Program database obtained prospectively with the existing KD database and queried for KD admissions between 1 month and 13 years of age. HRQoL was evaluated with the parent-proxy Pediatric Quality of Life Inventory (PedsQL) 4.0 Generic Core and Infant Scales. We compared the KD HRQoL results with those obtained from newly diagnosed patients with cancer and pneumonia, matched for age, sex and race. PedsQL total scores over time were assessed with ANCOVA models, adjusted for matching variables and PedsQL score prior to admission. RESULTS: We identified 89 patients with KD and compared 65 subjects with an equal number with pneumonia and with 67 subjects with newly diagnosed cancer. Patients with demonstrated lower PedsQL total score on admission and suffered a significantly greater HRQoL decline from baseline to admission than the other groups. KD diagnostic subtype (complete or incomplete) and coronary artery dilatation were not associated with HRQoL outcomes. However, non-intravenous immunoglobulin responders showed greater HRQoL decline than responders (P = .03). CONCLUSIONS: Children with KD suffer acute and significant HRQoL impairment exceeding that of children newly diagnosed with cancer. Lack of immediate treatment response may exert an additional HRQoL burden, whereas KD subtype and coronary artery dilatation do not.

Kawasaki Disease With Coronary Artery Aneurysms: Psychosocial Impact on Parents and Children.

INTRODUCTION: For those living with Kawasaki disease and coronary artery aneurysms, little is known about the psychosocial burden faced by parents and their children. METHODS: Exploratory, descriptive, mixed-methods design examining survey and interview data about health-related uncertainty, intrusiveness, and self-efficacy. RESULTS: Parents' uncertainty was associated with missed diagnosis, higher income, and maternal education. Higher uncertainty scores among children were associated with absence of chest pain and lower number of echocardiograms. High intrusiveness scores among parents were associated with previous cardiac catheterization, use of anticoagulants, lower parent education and income, and missed diagnosis. High intrusiveness scores among children were associated with high paternal education. Children's total self-efficacy scores increased with chest pain and larger aneurysm size. Qualitative analysis showed two central themes: Psychosocial Struggle and Cautious Optimism. DISCUSSION: Negative illness impact is associated with a more intense medical experience and psychosocial limitations. Timely assessment and support are warranted to meet parents' and children's needs.

Factors associated with low moderate-to-vigorous physical activity levels in pediatric patients with Kawasaki disease.

BACKGROUND/METHODS: We sought to determine functional health status and physical activity determinants in 27 patients with Kawasaki disease (KD; 20 males, 11 ± 3 years old). Patient physical activity data were compared with a population-based study of healthy children (Canadian Health Measures Survey). RESULTS: KD patients performed less moderate-to-vigorous physical activity (MVPA) than healthy children (males, 27 vs 61 min/d, P < .001; females, 10 vs 47 min/d, P < .001). Male KD patients performed more MVPA than female KD patients (median = 27; quartiles [Q1 15, Q3 26] min/d vs 10 [Q1 7, Q3 11] min/day, P = .009). Lower MVPA in KD patients was significantly associated with female gender; lower child self-efficacy score; lower Child Health Questionnaire (CHQ-PF50) scores for role functioning behavioral issues, physical functioning, and family cohesion; and higher CHQ-PF50 scores for self-esteem and family activity limitations. CONCLUSION: Physical activity counseling should be a focus of management for children with a history of KD.

Quality of life and behavioral functioning in Dutch children with a history of Kawasaki disease.

OBJECTIVE: The authors evaluated health-related quality of life (HRQOL) and behavioral functioning in patients with a history of Kawasaki disease (KD). STUDY DESIGN: A cross-sectional study was conducted at a tertiary referral center for KD follow-up in 280 patients (mean age 8.6 years, 60.0% male). Patients were eligible when they were aged 0-18 years and had a history of KD. HRQOL was assessed using the TNO-AZL Preschool Children Quality of Life questionnaire for children 0-5 years old and the Pediatric Inventory of Quality of Life Core Scales 4.0 for those 6-18 years old. Behavioral functioning was evaluated using the Strength and Difficulties Questionnaire (8-16 years proxy report and 11-16 years self-report). KD results were compared with Dutch norm data, and patients with and without coronary artery aneurysms were compared. RESULTS: HRQOL was significantly worse for male patients aged 0-5 years on 4 of the 12 TNO-AZL Preschool Children Quality of Life questionnaire scales and for female patients on the motor functioning scale. At an older age, the HRQOL of patients was comparable with the norm population. Coronary artery status did not influence HRQOL. Parents reported more behavioral problems on the hyperactivity and emotional subscale in patients compared with the norm population. CONCLUSIONS: Although at an older age the HRQOL of patients with KD is comparable with the Dutch norm, HRQOL seems to be particularly impaired at younger age. Parents reported more hyperactivity and emotional problems in patients with KD.

Parental anxiety associated with Kawasaki disease in previously healthy children.

OBJECTIVE: The objective of this study was to explore the lived experience of parents of children diagnosed with Kawasaki disease (KD) and to identify factors associated with increased levels of parental anxiety. STUDY DESIGN: Three focus groups were conducted including 25 parents of 17 patients with KD, seven (41%) of whom had coronary artery complications. A conceptual model was developed to depict parental experiences and illustrate the key issues related to heightened anxiety. RESULTS: Themes identified included anxiety related to the child's sudden illness and delay in obtaining a correct diagnosis because of the lack of health care providers' awareness and knowledge regarding KD. Parents were frustrated by the lack of information available in lay language and the limited scientific knowledge regarding the long-term consequences of the disease. Parents also reported positive transformations and different perspective toward challenges in life. However, the parents of children with coronary artery complications expressed persistent anxiety even years after the acute phase of the illness due to the uncertainty of the long-term prognosis. CONCLUSIONS: There remains a critical need for richly textured research data on the perspective and experience of families of children with KD.

Behaviour sequelae following acute Kawasaki disease.

BACKGROUND: Kawasaki disease is a systemic vasculitis and may affect cerebral function acutely. The aim of the present study was to measure a number of behaviour and social parameters within a cohort of Kawasaki disease patients. METHODS: Parents of children with past diagnosis of Kawasaki disease were recruited to complete several behaviour screening questionnaires. Sixty five sets of questionnaires relating to the patient cohort received were eligible for inclusion. Two control groups were used, a hospital (HC) control and a sibling control (SC) group. RESULTS: 40% of the Kawasaki disease group showed elevated internalising scores in the clinical or borderline-clinical range. This compared with 18% of hospital controls and 13% of sibling controls. Additionally, the Kawasaki disease (KD) group were shown to be experiencing greater overall total difficulties when compared with the controls (KD 13.7, HC 8.6, SC 8.9). The KD group attained higher behavioural scores within the internalising sub-categories of somatic problems (KD 61, HC 57, SC 54) and withdrawn traits (KD 56, HC 53, SC 51). The KD group were also shown to be suffering more thought problems (KD 57, HC 53, SC 50) compared with the controls. Further difficulties relating to conduct (KD 3.3, HC 1.4) and social interactions (KD 6.7, HC 8.3) are also highlighted for the KD group compared with hospital controls. Positron emission tomograms were performed on nine patients to investigate severe behavioural problems. Three showed minor changes, possibly a resolving cerebral vasculopathy. CONCLUSION: Kawasaki disease can be associated with significant behavioural sequelae. This is an important consideration in the long-term follow up and referral to a clinical psychologist may be necessary in selected patients.

Physical and psychosocial health in children who have had Kawasaki disease.

OBJECTIVE: The purpose of this study was to examine the physical and psychosocial well-being of children who have had Kawasaki disease (KD), including the influence of coronary artery status on health and health perceptions. METHODS: The Child Health Questionnaire (CHQ) measures overall physical and psychosocial well-being in children 5 to 18 years. To study the long-term impact of KD on overall health status, we mailed the CHQ to patients without a history of coronary artery abnormalities (normal group), with regressed aneurysms (regressed group), with current coronary aneurysms <8 mm (mild-moderate aneurysm group), and with giant aneurysms >or=8 mm (giant group). RESULTS: Of 201 questionnaires mailed, 174 were delivered and 110 (63%) were completed. Median age (range) at completion was 10.5 years (5.1-17.9 years) and at illness onset was 3.1 years (0.2-12 years). There were no significant differences in psychosocial summary scores in any of the Kawasaki groups when compared with the US population sample. Physical summary scores were also similar to the US population sample in the normal coronary, mild-moderate aneurysm, and regressed aneurysm groups. However, the giant aneurysm group had significantly lower physical summary scores compared with the US population sample. Among subscales, general health perceptions in the KD groups were lower than in the US population sample, reaching statistical significance in all but the mild to moderate aneurysm group. In addition, parents whose children have had KD reported a higher proportion of anxiety issues, allergies, and orthopedic/bone/joint issues in their children than did the general US population sample. We did not find any difference in the incidence of attentional, behavioral, or learning issues when compared with the US population sample. CONCLUSIONS: KD patients without coronary artery aneurysms were similar to the general population in their general physical and psychosocial health. However, the parents of children in all KD groups reported lower general health perceptions than parents in the US population sample, suggesting that long-term concerns about their children's health exist regardless of overall health status. In addition, children with giant coronary artery aneurysms had lower overall physical summary scores.

The effect of Kawasaki disease on cognition and behavior.

OBJECTIVE: To determine whether there are associated long-term deficits in the cognitive, academic, or behavioral outcomes of children with a previous episode of Kawasaki disease. DESIGN: Cohort analytic study. SETTING: A tertiary care pediatric hospital in Ottawa, Ontario. PARTICIPANTS: Thirty-two patients with a past diagnosis of Kawasaki disease. Siblings of the patients with Kawasaki disease were eligible to be controls. MEASURES: A blinded psychometrist (Y.K.) assessed cognition by the appropriate Wechsler Intelligence scale, academic achievement by the Wechsler Individual Achievement Test, and behavior by the Achenbach Child Behavior Checklist. RESULTS: No differences were found in cognitive or academic measures and the mean scores corresponded closely to national norms. Parents rated their children who had Kawasaki disease as having significantly more internalizing (P<.03) and attentional (P<.02) behavior problems than controls; the risk of a clinically significant behavioral score was 3.3 times greater (P<.03; 95% confidence interval, 1.1-9.9) than for sibling controls. CONCLUSIONS: While no effect on cognitive development or academic performance was demonstrated, these results provide preliminary indication of a post-Kawasaki disease deficit in internalizing and attentional behavior.