Including the voice of children Towards patient-centered care Lorynn Teela
Including the voice of children Towards patient-centered care Lorynn Teela
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Including the voice of children Towards patient-centered care ACADEMISCH PROEFSCHRIFT ter verkrijging van de graad van doctor aan de Universiteit van Amsterdam op gezag van de Rector Magnificus prof. dr. ir. P.P.C.C. Verbeek ten overstaan van een door het College voor Promoties ingestelde commissie, in het openbaar te verdedigen in de Agnietenkapel op vrijdag 22 december 2023, te 13.00 uur door Lorynn Teela geboren te Hengelo
Promotiecommissie Promotor: prof. dr. J.B. van Goudoever AMC-UvA Copromotores: dr. L. Haverman dr. H.A. van Oers AMC-UvA AMC-UvA Overige leden: prof. dr. F.B. Plötz prof. dr. A.M. Bosch prof. dr. E.M.W.J. Utens dr. M. de Wit prof. dr. W.B. de Vries prof. dr. A. Popma AMC-UvA AMC-UvA Universiteit van Amsterdam VUmc Vrije Universiteit Amsterdam Vrije Universiteit Amsterdam Faculteit der Geneeskunde
Table of contents Chapter 1 General introduction 6 Part 1: Pediatric patient engagement Chapter 2 Pediatric patient engagement in clinical care, research and intervention development: a scoping review 22 Chapter 3 Including the voice of paediatric patients: Cocreation of an engagement game 72 Part 2: Patient Reported Outcome Measures Chapter 4 Clinicians’ perspective on the implemented KLIK PROM portal in clinical practice 94 Chapter 5 Patients’ and parents’ perspective on the implementation of Patient Reported Outcome Measures in pediatric clinical practice using the KLIK PROM portal 122 Chapter 6 Implementation of the KLIK PROM portal using the Consolidated Framework for Implementation Research (CFIR) retrospectively 150 Chapter 7 Psychometrics of the patient-reported outcomes measurement information system measures in hemophilia; the applicability of the pediatric item banks 176 Part 3: Patient Reported Experience Measures Chapter 8 Use of Patient-Reported Experience Measures in pediatric care: a systematic review 200 Chapter 9 Discussion 254 Addendum Summary 284 Summary in Dutch – Nederlandse samenvatting 288 List of publications 294 List of contributing authors 297 Authors’ contributions per chapter 301 Financial support 303 PhD portfolio 304 Curriculum Vitae 308 Acknowledgements – dankwoord 309
1 General introduction
8 Chapter 1 Patient-centered care Patient-centered care1 (PCC), also referred to as person-centered care, is defined by the Institute of Medicine [1] as “providing care that is respectful of and responsive to individual patient preferences, needs and values, and ensuring that patient values guide all clinical decisions”. This approach puts the patient (and not the disease) at the center of health care and encourages a collaboration between patient, family, and clinicians to deliver care tailored to the unique wishes and needs of patients [2,3]. Although different models of PCC exist [1-4], the New England Journal of Medicine (NEJM) Catalyst [2] describes seven common elements of PCC: 1) The health care system’s mission, vision, and drivers for quality improvement are aligned with patient-centered goals. 2) Care is collaborative, coordinated, and accessible. The right care is provided at the right time and place. 3) Care is not only focused at physical comfort, but also on emotional well-being. 4) Care is respectful to patients’ and families preferences and values. 5) Patients and families are part of the care team and play a role in the decision making at both patient and system level. 6) Involvement of family in the health care is encouraged and facilitated. 7) Information is shared with patients and families so that they can make informed decisions. The most important reason for providing PCC is the improvement of individual health outcomes (i.e., medical outcomes, social well-being, and satisfaction with care) [2,5,6]. However, clinicians and health care organizations may also benefit from this approach as it results in enhanced patient satisfaction, greater job satisfaction, better productivity, and a reduction of health care costs (e.g., PCC is associated with a reduction of diagnostic tests and referrals) [2,5-8]. Despite the benefits of PCC, its implementation is challenging, as it calls for a different way of thinking about health care delivery and a changing role of patients and families, i.e., transitioning from a more passive role to an active member of the team [2,3]. This thesis focuses on the development of tools and scientific approaches on how to engage children and families in pediatric care, specifically (Figure 1): 1 In this thesis the term patient-centered care is used. However, we recognize that this term is broader in pediatrics and also means the engagement of parents and other family members.
9 General introduction - Part 1: Pediatric patient engagement - Part 2: Patient Reported Outcome Measures - Part 3: Patient Reported Experience Measures Figure 1. An overview of the different part of this thesis in relation to the patient-centered care model of NEJM Catalyst 1. Pediatric patient engagement An essential aspect of providing PCC, according to the NEJM model, is to involve patients in the decision-making in both clinical care and research (common elements 5 and 7) [2]. This involvement of patients, with the aim of improving health (care), is referred to as patient engagement [9,10]. Patient engagement is an umbrella concept and can be explained using the Multidimensional Framework for Patient Engagement in Health Care developed by Carman et. al. [11] (Figure 2). This framework shows that patients can be involved in all areas of health care, including clinical care, policy, and research. In addition, it states that patient engagement is a continuum and patients can influence decision-making to a different extend, ranging from consultation (e.g., patients are asked for their opinion, but have limited influence on decision-making) to active partnership (e.g., patients cooperate as equal partners). For which type of patient engagement is chosen depends on factors such as patients’ knowledge, the culture of the organization, time and budget constraints, and societal norms and values regarding patient engagement [11,12]. Patient engagement is beneficial for both patients and organizations. Studies have shown that patient engagement leads to decisions better matching patients’ wishes and needs, resulting in improved health outcomes, higher quality of care, and increased inclusion rates in research [13-15]. Furthermore, engaging patients enables them to be more responsible for their own health, which enhances selfconfidence and sense of control [12,16]. While the added value of patient engagement is increasingly recognized, clinicians and researchers struggle with the engagement of patients [17,18]. Clinicians doubt 1
10 Chapter 1 whether patients are knowledgeable, involving patients is time consuming, and scheduling meetings with groups of patients is difficult [19-23]. Involving pediatric patients seems to be especially challenging for clinicians, as the competence of children is even more questioned than adults [20,21,24,25]. Also, pediatric patient engagement is complicated by the influence of parents and the lack of experiences by clinicians, policymakers, and researchers [20,25]. Since the United Nations Convention on the Rights of the Child (UNCRC) states that every child has the right to be heard and to have a say in matters that affect them [26], it is necessary to give clinicians more support to involve pediatric patients meaningfully and usefully [27]. However, it is unclear to what extent and in what way patients are engaged in clinical care and research. In addition, currently no tools are available to help clinicians with the engagement of pediatric patients. Figure 2. A multidimensional framework for patient engagement in health care, based on the model of Carman [11]
11 General introduction 2. Patient Reported Outcome Measures Common elements of providing PCC are to incorporate and respond to patient and families preferences, needs, and values (NEJM common element 4 and 6) and to also focus on emotional well-being (NEJM common element 3) [2,28]. Patient Reported Outcome Measures can be used to elicit information regarding the perceived impact of disease and/or treatment directly from the patients themselves and from their family members [28,29]. PROMs are thus useful to incorporate the patient perspective into decision-making in clinical care and are therefore seen as an effective way to provide PCC [28,29]. PROMs are standardized, validated questionnaires completed by patients regarding their health status, symptoms, or well-being [30-32]. PROMs can be disease-specific (i.e., applicable for patients with a specific disease) or generic (i.e., applicable for everyone, regardless of disease) and are available for different age-ranges [33]. PROMs can be used for several purposes. Originally, PROMs were developed for use in scientific research to measure the effect of healthcare interventions [34]. However, PROMs can also be used for quality registration of care, where aggregated PROM data is used to gain insight into the quality of care and opportunities for quality improvement [35]. Additionally, PROMs can be used on an individual level in clinical care enabling PCC [36]. The effects of using PROMs in clinical care have been widely studied. These studies showed that using PROMs increases awareness for patients’ problems and concerns, enhances patient-clinician communication, improves patient satisfaction with health care and is associated with improved treatment outcomes [28,37-42]. Given the added value of PROMs, it is beneficial for PCC to implement PROMs in clinical practice. Currently, there is a wide variety in how PROMs are implemented, which impacts the intended effects [43]. For example, PROMs are used in clinical practice to monitor symptoms, make diagnoses, decide whether patients’ needs an outpatient visit or to facilitate the communication between patients and clinicians [36,44]. The focus of this thesis is on the implementation of PROMs as part of the conversation with the clinician. There are several initiatives that provide guidelines about PROM implementation, such as the International Society for Quality of Life Research (ISOQOL) User’s Guide to implementing patient-reported outcomes in clinical practice [45] and the PROM-toolbox, developed by the Dutch National Healthcare Institute [46]. 1
12 Chapter 1 KLIK PROM portal To facilitate the use of PROMs in clinical practice, the Emma Children’s hospital Amsterdam UMC developed the evidence-based KLIK PROM portal (www.hetklikt. nu) [47-50]. In the last 12 years, the KLIK PROM portal has been implemented in more than 40 hospitals in the Netherlands for pediatric and adult outpatient clinics [51]. In these hospitals, patients (children or adults) and/or caregivers complete online PROMs before an outpatient consultation to assess their physical, mental and/or social health (see Figure 3). Answers are converted into an electronic individual dashboard offering a broad range of feedback options customized to each specific PROM [52]. Clinicians discuss the KLIK dashboard with patients during the consultation with the aim to facilitate communication, monitor well-being over time, identify problems, and subsequently provide tailored advice and interventions. Figure 3. The KLIK workflow Implementation of the KLIK PROM portal The KLIK expertise center for PROMs and PREMs guides the implementation of PROMs in different multidisciplinary teams. Implementation strategies are crucial to reach the intended effects, but it remains challenging [53,54]. Implementation research can help to get insight into barriers and facilitators prior to implementation and can therefore ease the integration of PROMs in clinical care [55]. Implementation research is defined as the “scientific study of the use of strategies to adopt and integrate evidence-based health interventions into clinical settings in order to improve patient outcomes and benefit population health” [56]. Implementation research thus studies the mechanisms that influence implementation outcomes. Different implementation sciences models, theories and frameworks can be used to identify determinants (both barriers and facilitators) that influence the implementation outcome and provide implementation strategies as potential solution to barriers [56]. The Consolidated Framework for Implementation Research (CFIR) is a commonly used framework within PROM implementation [57]. This framework consists of five domains (intervention characteristics, outer setting, inner setting, characteristics of individuals, and implementation process), each with a
13 General introduction number of constructs focusing on different aspects of implementation (Table 1) [30,57]. With the increasing interest in PROM implementation, several barriers have been identified in the literature within the different CFIR domains [30]. Table 1 provides an overview of these barriers. Table 1. Barriers in the PROM implementation described using the CFIR framework Domain Description Barriers identified in literature Intervention characteristics For PROM implementation, the design of PROMs, PROM administration, and feedback, e.g., complexity - PROMs are perceived as burdensome - PROMs are not comparable due to different scoring methods Outer setting External factors that may impact the implementation, e.g., patients’ needs - Patients are not involved in the selection of PROMs - Patients do not have the ability to complete PROMs due to literacy issues Inner setting Internal factors that may impact the implementation, e.g., available resources - Clinicians experience a lack of time - Insufficient support from ICT Characteristics of individuals The views of individuals working with the intervention on implementation, e.g., knowledge about the intervention - Lack of knowledge on how to utilize and interpret PROMs Implementation process Factors related to the implementation process, e.g., planning and evaluation - Difficulties with embedding PROMs in the existing workflow Applying the CFIR model to the KLIK implementation process might yield valuable insights into barriers and facilitators, where after strategies can be identified to optimize the KLIK implementation. The involvement of patients/parents is of great importance in determining these factors. PROMIS To overcome the challenges associated with the burden of completing PROMs (i.e., long completion time, repetitive and irrelevant questions) [58,59], the National Institute of Health (NIH) developed the Patient-Reported Outcomes Measurement Information System (PROMIS®) [60,61]. PROMIS provides a set of person-centred, standardized instruments to measure a broad range of health domains (physical, mental, and social health) in both adults and children [60,62]. In contrast to legacy instruments, based on Classical Test Theory, PROMIS measures were developed according to Item Response Theory (IRT) [63,64]. An important advantage of IRT is the option of using Computerized Adaptive Testing (CAT) [62,63]. With CAT, questions are presented to patients based on their previous responses. In this way, patients answer a few questions per construct to get a reliable score. Consequently PROMIS measures are shorter, items are more tailored to the patients’ situation, and 1
14 Chapter 1 the measurement is more reliable in comparison to legacy instruments resulting in a reduced burden for patients [62-64]. 3. Patient Reported Experience Measures According to NEJM Catalyst, delivering PCC is only possible if care is in line with patient-centered goals and provided at the right time and place (common element 1 and 2) [2]. Therefore, it is necessary to gain patients’ feedback about the received care and the extent to which care is experienced as patient-centered. Patient Reported Experience Measures (PREMs) are questionnaires asking patients about their experience with the care received, and are commonly used to gain insight into the quality of care [55,65,66]. PREMs thus offer patients the opportunity to provide information, with the aim to improve care [67]. Contrary to PROMs, PREMs do not measure outcomes of care, but assess the impact of process of care such as communication between clinician and patient, information sharing, and involvement of patients in decision-making. PREMs and satisfaction questionnaires are often used interchangeably, although there are important differences between these instruments [65,66]. PREMs assess whether something that should happen in the health care setting actually happened or how often it happened (i.e., objective experiences), while satisfaction questionnaires assess whether the patient’s expectation about the care received were met (i.e., subjective views). PREMs are increasingly used to pursue PCC. However, guidelines regarding the use of PREMs are lacking, resulting in a wide variety of PREMs and PREM use in pediatrics. Aim and outline of this thesis The ultimate goal of this thesis is to enhance pediatric PCC in clinical care and research. Therefore, this thesis focuses on the development of tools and scientific approaches to provide PCC in pediatric care. This thesis is divided into three parts that discuss different parts of PCC. Part 1: Pediatric patient engagement Pediatric patient engagement is developing in recent years, and pediatric patients are more often involved in health care (projects). However, clinicians and researchers still struggle with the engagement of pediatric patients [17,18]. To gain insight into the extend of pediatric patients engagement and the methods used, Chapter 2 provides a comprehensive overview of the literature about pediatric patient
15 General introduction engagement in clinical care, policy, and research. To support clinicians with a tool to involve pediatric patients meaningfully and usefully, we developed a pediatric patient engagement tool with adolescents, as described in Chapter 3. Part 2: Patient Reported Outcome Measures As PROM implementation is a challenging process, insight into barriers and facilitating factors that influence implementation outcomes and stakeholder involvement is required. Understanding the wishes and needs of clinicians, patients, and parents (all stakeholders) is crucial. Therefore, the perspective of clinicians on the implementation of PROMs is investigated in Chapter 4. In Chapter 5, patients’ and parents’ perspective on the implementation of PROMs in clinical care is described. Chapter 6 retrospectively describes the implementation of the KLIK PROM portal using the CFIR framework and identifies implementation strategies to address the identified barriers. Insights into determinants of successful PROM implementation can both improve the implementation of the KLIK PROM portal and help others working on PROM implementation. PROMIS CATs can offer a promising solution to the burdensomeness of PROMs. Several steps are necessary to use these new instruments in clinical practice. As the PROMIS measures were initially developed in the United States [61], the first step was translation into Dutch [68] and validating the PROMIS instruments in the general population in the Netherlands [69,70]. A second step is to investigate the applicability and feasibility of the PROMIS CATs in a clinical population. Therefore, in Chapter 7 the psychometric properties of the PROMIS item banks were assessed in boys with hemophilia. Part 3: Patient Reported Experience Measures PREMs gather patients’ views of their experience with the care received and are commonly used to measure the quality of care, with the goal to make care more patient-centered [55,65,66]. With the growing adoption of PREMs in pediatric care, it is relevant to identify suitable PREMs. Chapter 8 therefore provides a systematic review of the available PREMs that can be used in pediatric care. This thesis ends with Chapter 9; a general discussion including a reflection on the main findings, clinical implications, methodological considerations, and future perspectives to promote PCC in clinical care and research. 1
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18 Chapter 1 43. Hsiao, C.-J., Dymek, C., Kim, B., & Russell, B. (2019). Advancing the use of patient-reported outcomes in practice: understanding challenges, opportunities, and the potential of health information technology. Quality of life Research, 28, 1575-1583. 44. Schougaard, L. M. V., Larsen, L. P., Jessen, A., Sidenius, P., Dorflinger, L., de Thurah, A., et al. (2016). AmbuFlex: tele-patient-reported outcomes (telePRO) as the basis for follow-up in chronic and malignant diseases. Quality of life Research, 25, 525-534. 45. Aaronson, N., Choucair, A., Elliott, T., Greenhalgh, J., Halyard, M., Hess, R., et al. (2015). User’s guide to implementing patient-reported outcomes assessment in clinical practice. International Society for Quality of Life Research, 2, 1-47. 46. van der Wees, P. J., Verkerk, E. W., Verbiest, M. E., Zuidgeest, M., Bakker, C., Braspenning, J., et al. (2019). Development of a framework with tools to support the selection and implementation of patient-reported outcome measures. Journal of patient-reported outcomes, 3(1), 1-10. 47. Engelen, V., Haverman, L., Koopman, H., Schouten - van Meeteren, N., Meijer-van den Bergh, E., VrijmoetWiersma, J., et al. (2010). Development and implementation of a patient reported outcome intervention (QLIC-ON PROfile) in clinical paediatric oncology practice. Patient Education and Counseling, 81(2), 235244, doi:doi:10.1016/j.pec.2010.02.003. 48. Haverman, L., Engelen, V., Van Rossum, M. A., Heymans, H. S., & Grootenhuis, M. A. (2011). Monitoring health-related quality of life in paediatric practice: development of an innovative web-based application. BMC Pediatrics, 11, 3-10, doi:doi:10.1186/1471-2431-11-3. 49. Engelen, V., Detmar, S., Koopman, H., Maurice-Stam, H., Caron, H., Hoogerbrugge, P., et al. (2011). Reporting health-related quality of life scores to physicians during routine follow-up visits of pediatric oncology patients: Is it effective? Pediatric Blood & Cancer, 58(5), 766-774, doi:doi:10.1002/pbc.23158. 50. Haverman, L., Van Rossum, M. A., Van Veenendaal, M., van den Berg, J. M., Dolman, K. M., Swart, J., et al. (2013). Effectiveness of a web-based application to monitor health-related quality of life. Pediatrics, 131(2), 533-543, doi:doi:10.1542/peds.2012-0958. 51. Haverman, L., van Oers, H. A., Limperg, P. F., Hijmans, C. T., Schepers, S. A., Sint Nicolaas, S. M., et al. (2014). Implementation of electronic Patient Reported Outcomes in pediatric daily clinical practice: The KLIK experience. Clin Pract Pediatr Psychol, 2(1), 50-67, doi:https://doi.org/10.1037/cpp0000043. 52. Haverman, L., van Oers, H. A., van Muilekom, M. M., & Grootenhuis, M. A. (2019). Options for the Interpretation of and Recommendations for Acting on Different PROMs in Daily Clinical Practice Using KLIK. Med Care, 57 Suppl 5 Suppl 1, S52-S58, doi:10.1097/MLR.0000000000001061. 53. Proctor, E., Silmere, H., Raghavan, R., Hovmand, P., Aarons, G., Bunger, A., et al. (2011). Outcomes for implementation research: conceptual distinctions, measurement challenges, and research agenda. Adm Policy Ment Health, 38(2), 65-76, doi:10.1007/s10488-010-0319-7. 54. Proctor, E. K., Landsverk, J., Aarons, G., Chambers, D., Glisson, C., & Mittman, B. (2009). Implementation research in mental health services: an emerging science with conceptual, methodological, and training challenges. Adm Policy Ment Health, 36(1), 24-34, doi:10.1007/s10488-008-0197-4. 55. McCabe, E., Rabi, S., Bele, S., Zwicker, J. D., & Santana, M. J. (2023). Factors affecting implementation of patient-reported outcome and experience measures in a pediatric health system. Journal of patientreported outcomes, 7(1), 1-12. 56. Nilsen, P. (2015). Making sense of implementation theories, models and frameworks. Implement Sci, 10, 53, doi:10.1186/s13012-015-0242-0. 57. Damschroder, L. J., Aron, D. C., Keith, R. E., Kirsh, S. R., Alexander, J. A., & Lowery, J. C. (2009). Fostering implementation of health services research findings into practice: a consolidated framework for advancing implementation science. Implement Sci, 4, 50, doi:10.1186/1748-5908-4-50. 58. Antunes, B., Harding, R., Higginson, I. J., & Euroimpact (2014). Implementing patient-reported outcome measures in palliative care clinical practice: a systematic review of facilitators and barriers. Palliative medicine, 28(2), 158-175. 59. Philpot, L. M., Barnes, S. A., Brown, R. M., Austin, J. A., James, C. S., Stanford, R. H., et al. (2018). Barriers and benefits to the use of patient-reported outcome measures in routine clinical care: a qualitative study. American Journal of Medical Quality, 33(4), 359-364.
19 General introduction 60. Cella, D., Riley, W., Stone, A., Rothrock, N., Reeve, B., Yount, S., et al. (2010). The Patient-Reported Outcomes Measurement Information System (PROMIS) developed and tested its first wave of adult self-reported health outcome item banks: 2005-2008. J Clin Epidemiol, 63(11), 1179-1194, doi:10.1016/j. jclinepi.2010.04.011. 61. Cella, D., Yount, S., Rothrock, N., Gershon, R., Cook, K., Reeve, B., et al. (2007). The Patient-Reported Outcomes Measurement Information System (PROMIS): progress of an NIH Roadmap cooperative group during its first two years. Medical Care, 45(5 Suppl 1), S3-s11, doi:10.1097/01.mlr.0000258615.42478.55. 62. Broderick, J. E., DeWitt, E. M., Rothrock, N., Crane, P. K., & Forrest, C. B. (2013). Advances in patientreported outcomes: the NIH PROMIS® measures. Egems, 1(1). 63. Cella, D., Gershon, R., Lai, J.-S., & Choi, S. (2007). The future of outcomes measurement: Item banking, tailored short-forms, and computerized adaptive assessment. Quality of Life Research, 16(SUPPL. 1), 133-141. 64. Fries, J. F., Witter, J., Rose, M., Cella, D., Khanna, D., & Morgan-DeWitt, E. (2014). Item response theory, computerized adaptive testing, and PROMIS: assessment of physical function. The Journal of Rheumatology, 41(1), 153-158, doi:jrheum.130813 [pii];10.3899/jrheum.130813 [doi]. 65. Kingsley, C., & Patel, S. (2017). Patient-reported outcome measures and patient-reported experience measures. Bja Education, 17(4), 137-144. 66. Beattie, M., Murphy, D. J., Atherton, I., & Lauder, W. (2015). Instruments to measure patient experience of healthcare quality in hospitals: a systematic review. Systematic reviews, 4(1), 1-21. 67. Nwaru, B. I., Friedman, C., Halamka, J., & Sheikh, A. (2017). Can learning health systems help organisations deliver personalised care? BMC medicine, 15(1), 1-8. 68. Haverman, L., Grootenhuis, M. A., Raat, H., van Rossum, M. A., van Dulmen-den Broeder, E., Hoppenbrouwers, K., et al. (2016). Dutch–Flemish translation of nine pediatric item banks from the patientreported outcomes measurement information system (PROMIS)®. Quality of life Research, 25, 761-765. 69. Klaufus, L., Luijten, M., Verlinden, E., Van der Wal, M., Haverman, L., Cuijpers, P., et al. (2021). Psychometric properties of the Dutch-Flemish PROMIS® pediatric item banks Anxiety and Depressive Symptoms in a general population. Quality of life Research, 30(9), 2683-2695. 70. Luijten, M. A., van Litsenburg, R. R., Terwee, C. B., Grootenhuis, M. A., & Haverman, L. (2021). Psychometric properties of the Patient-Reported Outcomes Measurement Information System (PROMIS®) pediatric item bank peer relationships in the Dutch general population. Quality of life Research, 30, 2061-2070. 1
Part 1 Pediatric patient engagement
2 Pediatric patient engagement in clinical care, research and intervention development: a scoping review Lorynn Teela, Lieke E. Verhagen, Hedy A. van Oers, Esmée E.W. Kramer, Joost G. Daams, Mariken P. Gruppen, Maria J. Santana, Martha A. Grootenhuis, Lotte Haverman Journal of Patient-Reported Outcomes. 2023; 7,32.
24 Chapter 2 Abstract Background In the last decades, pediatric patient engagement has received growing attention and its importance is increasingly acknowledged. Pediatric patient engagement in health care can be defined as the involvement of children and adolescents in the decision-making of daily clinical care, research and intervention development. Although more attention is paid to pediatric patient engagement, a comprehensive overview of the activities that have been done regarding pediatric patient engagement and the changes over time is lacking. Therefore, the aim of this study is to provide an overview of the literature about pediatric patient engagement. Methods The methodological framework of Arksey & O’Malley was used to conduct this scoping review. The bibliographic databases Medline, Embase, and PsycINFO were searched for eligible articles. All retrieved articles were screened by at least two researchers in two steps. Articles were included if they focused on pediatric patient engagement, were carried out in the context of clinical care in pediatrics, and were published as full text original article in English or Dutch. Data (year of publication, country in which the study was conducted, disease group of the participants, setting of pediatric patient engagement, used methods, and age of participants) were extracted, synthesized, and tabulated. Results A total of 288 articles out of the 10,714 initial hits met the inclusion criteria. Over the years, there has been an increase in the number of studies that engage pediatric patients. Pediatric patients, especially patients with multiple conditions or oncology patients, were most involved in studies in the United States, United Kingdom, and Canada. Pediatric patients were most often asked to express their views on questions from daily clinical care and the individual interview was the most used method. In general, the extent to which pediatric patients are engaged in health care increases with age. Discussion This scoping review shows that there is an increasing interest in pediatric patient engagement. However, lack of uniformity about the definition of pediatric patient engagement and clear information for clinicians hinders engagement. This overview can inform clinicians and researchers about the different ways in which pediatric patient engagement can be shaped and can guide them to engage pediatric patients meaningfully in their projects.
25 Scoping review pediatric patient engagement Introduction In 1989, over 190 countries, including the Netherlands, signed the United Nations Convention on the Rights of the Child (UNCRC) [1]. The UNCRC describes the human rights for every child, such as self-determination, freedom of thoughts and religion, and the right to have a say in matters that affect them. It is with this convention that the engagement of children in health care, research and intervention development became more important [2,3]. From that moment on, clinicians, researchers and policymakers more often tried to carry out their health care projects and decision-making together with pediatric patients rather than about or for pediatric patients [3]. Involving children in decision-making about daily clinical care, research and intervention development is referred to as ‘pediatric patient engagement’ [4,5]. The extent to which children influence the decision-making processes can vary from consultation (e.g., patients are asked for their opinion, but have limited influences on decision-making) to active partnership (e.g., patients cooperate as equal partners with other stakeholders and share responsibility) [6,7]. Notwithstanding the extent of involvement, pediatric patient engagement has important value for health care. Previous research shows that pediatric patient engagement increases children’s self-confidence and sense of control, which results in better treatment outcomes [8]. Moreover, pediatric patient engagement leads to higher inclusion rates in research and improves the translation from research to clinical practice [9]. Although the importance of pediatric patient engagement is acknowledged, pediatric patients are not always involved in the decision-making process in health care [8,10]. Clinicians, researchers, and policymakers are, for example, reserved in involving pediatric patients in health care as they doubt the capacity of children required for participating, and they lack experience in engaging children [8,11,12]. In addition, pediatric patient engagement is complicated by the tendency of adults to protect children from making difficult decisions [8,11]. Professionals therefore need more support to involve pediatric patients meaningfully and usefully [13]. In the last years, a few systematic reviews on pediatric patient engagement in clinical care have been conducted [2,3,14]. These systematic reviews are relatively outdated (over 10 years old), given the fact that pediatric participation is a developing practice. The focus of the conducted systematic reviews were only on engagement in the decision-making process in the consultation room and the challenges involved [2,14]. Also, in one paper, the included articles are only summarized and interpreted by 2
26 Chapter 2 one author [2] , as opposed to systematically collating, summarizing, and reporting the results. A recent scoping review describes the involvement of adolescents and young adults (12-25 years) with a chronic condition in health and social care [3]. This review, only including 23 studies, provides a synopsis of the used definitions of patient engagement, goals, methods, and impact of the involvement of youth in research and implementation projects. However, a comprehensive overview of the activities that have been done in the past regarding pediatric patient engagement, also including primary school-aged children (4-18 years) in health care is lacking, as well as insights into how patient engagement takes place in clinical care, research, and intervention development. In addition, we want to know how pediatric patient engagement has developed in recent years to learn more about the different ways pediatric patients can be involved in health care. Therefore, the aim of this study is to provide an overview of the literature about pediatric patient engagement in clinical care, research, and intervention development. Methods Due to the broad nature of the study aim, a scoping review was conducted. Scoping reviews can be used to provide an overview and map the available evidence around a certain topic [15,16]. The methodological framework of Arksey & O’Malley [16] was used to guide this scoping review. This framework consisted of the following 5 stages: Stage 1: Identifying the research question The research question of this scoping review was: What is known from the literature about pediatric patient engagement in clinical care, research and intervention development? A comprehensive approach was chosen to examine the extent and nature of pediatric patient engagement in the broad field of pediatrics. Key parameters were patient engagement (defined as: actively involving children in the clinical care, medical research, and intervention development. This means that children were asked for their opinion on certain topics or that they played a role in the decision-making process), children and adolescents (defined as people aged 4-18 years), and pediatrics (defined as the medical care of children and adolescents in a hospital/clinical setting and the associated science).
27 Scoping review pediatric patient engagement Stage 2: Identifying relevant studies A comprehensive search strategy was developed and carried out in collaboration with a medical research librarian (JGD). To obtain a clear description of the construct, both published and unpublished literature about engagement of children and adolescents was collected and reviewed by at least two research-psychologists (FW, MV, LH). Subsequently, a visualization of similarities (VOS) analysis [17] was carried out with the software tool VOSviewer® to remove irrelevant terms from the search strategy by NOTing [18]. Medline, Embase and PsycINFO were searched for eligible articles from inception (May 2017). The construct of the search strategy can be summarized as follows: ([hospitalized patient] AND [patient participation]) NOT [irrelevant terms identified by VOS analysis]. See Additional file 1 for full search details. In February 2021, an update of the literature search was done. The same search strategy was applied. The bibliographic databases were searched for eligible articles in the period January 2017 until February 2021. For practical reasons, duplicate articles from the period January 2017 – May 2017 were removed in the last step of the study selection. Stage 3: Study selection Title and abstract of the articles retrieved were assessed by at least two members of the research team (LT, LEV, EEWK, FW, MV, LH) using the software tool Rayyan [19]. To reduce individual bias during the screening process and to refine inclusion and exclusion criteria, consultation took place between the members of the research team after screening the first 300 articles. The full text of potentially relevant articles was obtained and assessed by at least two members of the team (LT, LEV, EEWK). If necessary, a third member (LH) made the decision regarding inclusion of an article. An article was included if the study described all following inclusion criteria: · Focused on engagement of children and adolescents (4-18 years). Studies that included pediatric patients in a broader age range or studies that included both pediatric patients and young adults were also included. · Participants were asked for their opinion regarding clinical care, research, policy and/or intervention development. · Carried out in the context of clinical care/pediatrics. · Published as a full text original article (i.e. not an abstract, review, commentary, dissertation or study protocol). · Published in English or Dutch. 2
28 Chapter 2 Studies that reported only on the engagement of representatives of pediatric patients (i.e., caregivers, family members) or studies that did not clearly distinguish pediatric patients as a subgroup were excluded. In addition, studies that were conducted in the field of dentistry or psychiatry or studies that described the engagement of pediatric patients in a school or home setting were excluded. Also, studies that explored the experiences of children living with a medical condition in general (e.g., experiences of children living with HIV) were excluded, unless the studies reported on the life-experiences of these children with the aim to improve a medical treatment or to develop an intervention/tool. Furthermore, studies describing the involvement of pediatric patients in developing measurements using cognitive interviews for checking the understanding of questions or icons were excluded. The research team does not consider using cognitive interviews for this purpose to be part of pediatric patient engagement. The opinion of children and adolescents is thus not being asked in these cognitive interviews. Finally, studies that only described the importance of pediatric patient engagement, but did not discuss the application of pediatric patient engagement, were also excluded. Stage 4: Charting the data A data extraction form was developed by the team, and data were extracted from the included articles by one members of the team (LT, LEV, or EEWK). A second member of the team (LT, LEV, or EEWK) cross-checked a selection of the extracted data. The following data were extracted from the articles: year of publication, country in which the study was conducted, disease group of the participants, number of participants, setting of pediatric patient engagement (health care, research, or development of interventions or tools), method used for patient engagement, and age of participants. Stage 5: Collating, summarizing, and reporting the results Extracted data were analyzed quantitatively with the use of the Statistical Package for Social Sciences (SPSS) version 28. This quantitative data provided an overview of the nature and extent of pediatric patient engagement. To learn more about the goals of pediatric patient engagement, the data were screened by the research team and examples were cited.
29 Scoping review pediatric patient engagement Results Search and selection results The study selection process is presented in the PRISMA flow diagram of Fig 1. The literature search yielded 10,365 (2017) and 3249 (2021) articles. After removing duplicates, title and abstracts of 11,071 (2017) and 3190 (2021) articles were assessed. Of these, 519 (2017) and 205 articles (2021) were eligible for full-text review. A total of 288 articles met the inclusion criteria and were included. An overview of the characteristics of included studies can be found in Additional file 2. Figure 1. Flow chart of the identification and selection process of studies [20] 2
30 Chapter 2 Pediatric patient engagement through the years The included articles are published between 1983 and February 2021, as shown in Fig 2. Over the years there has been an increase in the number of studies that include pediatric patient engagement. * Range is less than 5 years Figure 2. Overview of the included articles (number) per 5 years Pediatric patient engagement per country Figure 3 shows the number of studies in which pediatric patient engagement is included per country. Most studies involving pediatric patient engagement are performed in the United States of America, followed by the United Kingdom, and Canada. Pediatric patient engagement per disease group The largest group of studied patients encompasses pediatric patients from different disease groups (26%) in their clinical care, research or development of intervention, and 10% of the studies concerned children being admitted to the hospital for various reasons. When looking at individual disease groups, pediatric oncology patients (22%) are most often engaged about their opinion, followed by pediatric patients undergoing surgery (7%), diabetes patients (5%), asthma patients (4%), transplant patients (4%), patients with Juvenile Idiopathic Arthritis (2%), and pediatric patients in palliative care (2%).
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