Background
Juvenile Idiopathic Arthritis (JIA) is a heterogeneous disease that is characterised by the occurrence of arthritis of unknown origin lasting for more than 6 weeks, with an onset before the age of 16 years. It is the most common chronic rheumatic disease in children [
1]. The health outcome for children and young adults with JIA has significantly improved, however unanswered questions still remain regarding the care of these patients [
1,
2]. Nevertheless, there is increasing evidence that the needs raised by patients with JIA, their caregivers and clinicians are not always reflected by the subjects as studied in research programmes [
3]. Importantly, various studies demonstrate the importance of involving the end-users of knowledge in setting priorities for research to formulate research questions that truly make a difference and are in tune with the needs of patients [
4,
5].
While the importance is widely acknowledged [
6], still little effort is made to include patients in the set-up of JIA research. This is probably also aggravated by the fact that it includes a paediatric population, and it is still not exactly clear how to best include their voice [
7‐
10]. A recent systematic review on research priority setting in paediatric chronic disease shows that only one in four studies reported parents/caregiver involvement, and only 5% included children directly [
7]. One of the studies that directly involves children/adolescents in research priority setting for rheumatic conditions was a study by Parsons et al. They successfully involved young people (11–24 years) in research prioritisation by organising focus groups [
11]. The domains ‘basic science’ and ‘psychosocial research’ were found to be most significant and have the highest priority for research. Notably, they showed that young people can discuss and prioritise scientific research, despite them being relatively research naïve. We aimed to interweave the advantages of such focus groups with the broader, well-established approach of setting research priorities of the James Lind Alliance (JLA).
The JLA is a non-profit initiative, founded in 2004 in the United Kingdom to bring patients, carers (caregivers, here: mostly parents), and clinicians together in Priority Setting Partnerships (PSPs) to set research priorities [
12,
13]. They have developed a methodological approach that has now been used worldwide to generate about a hundred research agendas. Following the JLA methodology, we aimed to conduct a nationwide prioritisation exercise for JIA in the Netherlands [
2]. The ultimate aim of this study is to guide future JIA research and funding to the issues that matter most to all directly involved.
Results
Initial survey
During the first phase of the process, a total of 604 questions were gathered. Of these 604, 506 were submitted by 278 respondents through the survey: 141 patients (50.7%), 88 carers (31.6%) and 49 health care professionals (17.6%). Demographic data are summarised in Table
1. Responses were gathered from all academic centres and geographic regions in the Netherlands. In the online survey, 25.8% of the carers had finished a lower and middle education. To lower the bar of participating, a hardcopy version of the survey was distributed at outpatient clinics. On this hardcopy version 59% of the respondents had lower and middle education (see Table
2), demonstrating the additional value of distributing hardcopy surveys. The underrepresentation of younger patients in the online survey – no more than 23% of responding patients were younger than 16 years – underscored the significance of the focus groups. In total 24 children (17 girls and 7 boys, 9–16 years of age, suffering from JIA between 4 months to 9 years), participated in two focus groups and six (group-) interviews, Together, they formulated 81 extra questions (Aussems et al., in preparation). Furthermore, in the Dutch JIA guideline 17 additional unanswered research questions were found [
15].
Table 1
Demographics of participants of the first and second survey
Respondents (number; %) | Total | 278 (100%) | Total | 303 (100%) |
Patients | 141 (50.7%) | Patients | 125 (41.3%) |
Carers | 88 (31.6%) | Carers | 136 (44.9%) |
Clinicians | 49 (17.6%) | Clinicians | 42 (13.9%) |
Level of education of respondenta (number; %) | Patients | Primary school | 22 (15.6%) | Patients | Primary school | 3 (2.4%) |
PSE | 21 (14.9%) | PSE | 17 (13.6%) |
SGSE | 13 (9.2%) | SGSE | 22 (17.6%) |
PUE | 19 (13.5%) | PUE | 15 (12.0%) |
SVE | 26 (18.4%) | SVE | 25 (20.0%) |
HPE | 20 (14.2%) | HPE | 18 (14.4%) |
University | 13 (9.2%) | University | 20 (16.0% |
Other | 7 (5.0%) | Other | 5 (4.0%) |
Carers | Primary school | 5 (5.7%) | Carers | Primary school | 13 (9.6%) |
PSE | 5 (5.7%) | PSE | 6 (4.4%) |
SGSE | 4 (4.5%) | SGSE | 12 (8.8%) |
PUE | 2 (2.3%) | PUE | 2 (1.5%) |
SVE | 14 (15.9%) | SVE | 38 (27.9%) |
HPE | 43 (48.9%) | HPE | 47 (34.6%) |
University | 12 (13.6%) | University | 12 (8.8%) |
Other | 3 (3.4%) | Other | 6 (4.4%) |
Age of responding patient (mean; range) | mean 17.6 (range 4–55) years | mean 19..95 (range 10–52) years |
JIA subtype of patient (number; %) | Polyarticular JIA | 93 (40.6%) | Polyarticular JIA | 110 (42.3%) |
Oligoarticular JIA | 57 (24.8%) | Oligoarticular JIA | 50 (19.2%) |
Psoriatric arthritis | 10 (4.4%) | Psoriatric arthritis | 15 (5.7%) |
Enthesitis-related arthritis | 2 (0.9%) | Enthesitis-related arthritis | 5 (1.9%) |
Systemic JIA | 18 (7.9%) | Systemic JIA | 31 (11.9%) |
Don’t know | 49 (21.4%) | Don’t know | 50 (19.2%) |
Disease duration in years (mean; range) | 8.8 years (0–65 years) | 9.0 years (0–45 years) |
Uveitis (number; %) | Yes | 51 (22.2%) | Yes | 66 (25.3%) |
No | 174 (76.0%) | No | 189 (72.4%) |
Don’t know | 4 (1.7%) | Don’t know | 6 (2.3%) |
Location of treatment centre (number; %) | Groningen | 72 (25.9%) | Groningen | 48 (15.8%) |
Utrecht | 49 (17.6%) | Utrecht | 51 (16.8%) |
Nijmegen | 10 (3.6%) | Nijmegen | 5 (1.7%) |
Boxmeer | 29 (10.4%) | Boxmeer | 31 (10.2%) |
Amsterdam (Reade) | 15 (5.4%) | Amsterdam (Reade) | 13 (5.0%) |
Amsterdam (UMC) | 10 (3.6%) | Amsterdam (UMC) | 13 (5.0%) |
Leiden | 10 (3.6%) | Leiden | 18 (6.9%) |
Rotterdam | 79 (28.4%) | Rotterdam | 82 (27.1%) |
Maastricht | 1 (0.4%) | Maastricht | 5 (1.7%) |
Other | 3 (1.1%) | Other | 37 (12.2%) |
Table 2
Levels of education of carers responding to online vs. hardcopy versions of both surveys
FIRST SURVEY |
Education levela |
Lower (n, %) | 4 (6.1%) | 6 (27.2%) | 10 (11.4%) |
Middle (n, %) | 13 (19.7%) | 7 (31.8%) | 20 (22.7%) |
Higher (n, %) | 47 (71.2%) | 8 (36.3%) | 55 (62.5%) |
Other (n, %) | 2 (3.0%) | 1 (4.5%) | 3 (3.4%) |
Total (n) | 66 | 22 | 88 |
SECOND SURVEY |
Education levela |
Lower (n, %) | 16 (17.2%) | 3 (7.0%) | 19 (14.0%) |
Middle (n, %) | 31 (33.3%) | 21 (48.8%) | 52 (38.2%) |
Higher (n, %) | 43 (46.2%) | 16 (37.2%) | 59 (43.4%) |
Other (n, %) | 3 (3.2%) | 3 (7.0%) | 6 (4.4%) |
Total (n) | 93 | 43 | 136 |
Analysis of evidence uncertainties
Of the 604 submitted questions, 519 were determined to be in scope. Out of scope questions mostly related to logistics of care (e.g. “Why can’t blood be drawn at the GP’s office instead of at the hospital?”, or: “Do we have to pay for orthopaedic shoes ourselves or does the insurance cover it?”) or personal situations (e.g. “Which type of JIA do I have?”) and could not be answered by research. During a face-to-face meeting, the steering group summarized the remaining questions into 53 summary questions.
An elaborate search in PubMed and PsycInfo showed that none of the 53 questions was completely answered. A full list of all the submitted questions, the 53 summary questions, our search strategy for evidence checking and the critical appraisal of the studies has been published online [
16].
Interim survey
Three hundred and three people chose their top 10 from the 53 questions: 125 patients (41.3%), 136 carers (44.9%) and 42 clinicians (13.9%). Two focus groups with seven and nine children respectively (in total 9 girls and 7 boys, 10–15 years), and a subsequent group priority setting discussion attended by six girls and five boys from the focus groups, resulted in a top 5 of their most important questions (Aussems et al. in preparation).
The 35 questions in the respective top 10s of patients, carers, clinicians, and the top 5 of younger children showed considerable overlap. One question – on fatigue – was prioritised by all 4 groups. Four questions were ranked in three top 10s. This resulted in a combined top 21. Table
3 shows how these 21 questions were ranked in the different groups. Interestingly, the question “How can JIA be cured?” was highly prioritised by patients and carers, and ranked very low by the clinicians. The clinicians may have been less optimistic about the chances to find a cure in the near future. Three questions in the childrens’ top 5 – numbered 14, 16 and 19 in Table
3 – were ranked much lower by the other three groups. This illustrates the added value of the focus groups.
Table 3
Interim rankings of the Top 21 research questions and the rankings per group
1. | Pain and fatigue are often present when the disease is in remission. How does this happen, what can one do about it, and can one predict which patients will suffer from them? | 3 | 10 | 1a | |
2. | What is the best treatment plan for each individual patient? (e.g. start a biological directly, which one, and what to do when the first one does not work and how can medication best be tapered off? b) | 42 | 28 | 7a | |
3. | What is the best treatment plan for uveitis in JIA, and are there factors that predict its effectiveness? | 36 | 25 | 5 | |
4. | Why are children with JIA fatigued more quickly, what can be done about it, and how can one best cope with the fatigue in daily life? | 6a | 6 | 1a | 2 |
5. | How does JIA develop and which factors influence this? | 6a | 5 | 24 | |
6. | How can the course (flares, extensions, cure) of JIA be better explained and predicted? | 15 | 9 | 6 | |
7. | What is the influence of nutrition on JIA, and can a diet help? | 2 | 2 | 7a | |
8. | What are the short and long term side effects/consequences of the drugs taken for JIA? | 8 | 1 | 10a | |
9. | What is the influence of JIA on future opportunities regarding school results, work and relationships? | 9 | 11 | 20 | 1 |
10. | What is the influence of sports and exercise on JIA and vice versa? | 24 | 37 | 7a | |
11. | What are the long term physical consequences of JIA? | 1 | 3 | 10a | |
12. | How can JIA be cured? | 4 | 4 | 42a | |
13. | Is there an association between JIA and other (autoimmune) diseases, and if yes, how can one better understand this? | 10a | 8 | 42a | |
14. | How can pain best be recognised and be treated (with medication), and what action can a patient take him/herself? | 32 | 30 | 29a | 3 |
15. | Which knowledge and skills are needed for patients and parents to achieve a healthy and active lifestyle? | 38 | 24 | 4 | |
16. | How can pills be manufactured in such a way that they are easy to take? (i.e. shape, color, taste) | 29 | 40 | 29a | 4 |
17. | How can children/adolescents with JIA can best be supervised regarding school/education in order to minimize drop-out rates and absenteism? | 10 | 15 | 10a | |
18. | Is JIA inheritable, and if yes, in what way? | 5 | 13 | 51 | |
19. | What is the best way to practice your favorite sport safely? | 43 | 49 | 17 | 5 |
20. | Are there any strategies in alternative medicine that can help alleviate health complaints of JIA? | 21 | 7 | 42a | |
21.b | When and how can medication for JIA best be tapered off? | 37 | 21 | 3 | |
Final workshop
During the final priority setting workshop, on February 7, 2020, the top 10 was chosen (see Table
3). The workshop was attended by five patients, five parents of patients, and ten clinicians (paediatric rheumatologists (
n = 3), an ophthalmologist (
n = 1), physical therapists (
n = 2), nurses (
n = 3), and a psychologist (
n = 1)). Clinicians were recruited from all academic centres in the Netherlands. The results of two rounds of discussions and ranking in three mixed groups were combined, and discussed further in a final session. The participants chose to merge the two summary questions on personalised medicine and strategies to taper off medication (see Table
3). All 20 attendees unanimously agreed on the final Top 10.
The result of the discussions in the final workshop broadly reflected the interim rankings. For both patients and carers, 6 of the interim top 10 questions (60%) were selected. For clinicians it was 80%. For the focus groups 2 out of 5 questions (40%) were part of the final top 10. Importantly, question 9 of the final top 10 made it into the list because a young adult patient advocated for it during the final discussion, using the argument that it was the children’s top priority. Evaluations from the participants of the final workshop were very positive: patients, carers and clinicians felt like their voice was heard, that all voices mattered equally, and that the atmosphere was very positive.
Discussion
This project has brought patients, caregivers and clinicians together in creating a research agenda for JIA using the JLA method. This is one of the first times a PSP has integrated the JLA approach with additional focus groups with children to ensure involvement of paediatric patients of all age categories. We found that the number one research priority involved the cause and care of pain and fatigue when the disease was in remission. This is in line with a qualitative study by Bromberg et al. that finds self-reported pain and fatigue are highly common in children with JIA despite advances in treatment strategies [
17]. It is also highlighted to be an important research area by Palman et al. [
18], along with defining better predictors of remission states, which was also part of our Top 10. The fact that these studies all underline the same top research priority demonstrates the importance of future research focusing on this. Next to pain and fatigue, the aetiology of the disease remains an important topic that was featured high in our Top 10 and also in a recent priority setting exercise in the United States [
19].
Moreover, our study exposed other important research areas such as personalised treatment strategies, JIA-associated uveitis, nutrition, long-term effects of drugs taken for JIA and sports/exercise that were not directly featured in other studies. This may be due to the wide scope we defined for this project, as well as the easily accessible nature of the JLA method that may have led to the collection of more widespread themes. The fact that the questions do not only cover the healthcare setting but also comprise more (psycho) social and educational issues suggests funding for these topics can also be sought from psychological, social and educational funding bodies.
One major strength of our study is the use of the systematic and transparent JLA method, which enhances the validity of our results. Furthermore, our efforts to include younger children is a strength. We conducted focus groups during the two phases of the process where information was gathered, such that they truly had their say in determining the most important topics. Two questions of the children’s Top 5 made it to the final Top 10 (see Table
3), which indicates the significant influence they had in the PSP.
A challenge in this study was the inclusiveness of people with all educational backgrounds and age groups throughout the survey phases. The input of patients and parents in the steering group was very valuable. For example, they thought of using a cartoon on the flyers and in social media coverage to capture people’s attention. A parent proposed the use of hardcopy questionnaires in these phases, which improved the inclusion of respondents with regard to education level, especially in the first phase. For people with low digital skills, it might be more difficult to formulate their own question, than to choose from a list of questions.
In a subgroup analysis of the parent responses in the prioritising phase, we found that the online and hardcopy groups of parents showed overlap in their priorities. The parents responding with paper and pencil did not rank the question on alternative medicine that high. Interestingly, they did prioritize two questions on school and education (nrs 9 and 17), that were not prioritized in the online parent group. This may have been related to their own educational background.
In this light, it is important to realise that participants of the final workshop consisted of a group of (predominantly) white, articulate, higher educated people, despite explicit efforts to include people with lower educational backgrounds and other ethnicities. This may have influenced which questions made it to the Top 10. This is a typical tendency in survey research and was also observed in other PSPs [
20]. Future PSPs may want to come up with and employ alternative strategies to ensure equal representation of all members of society. We also observed that more questions from the clinicians initial shortlist made it into the final Top 10 (see Table
3). This may be due to the fact that clinicians are more trained to give their opinion about these matters than young people and parents. Nevertheless, all attendees at the final workshop in the end unanimously agreed on the final Top 10 as a list of shared priorities.
Formally, this Top 10 was generated in the Netherlands, but we expect it also holds for other western countries. Now that the research agenda has been set, it is important that it is now indeed implemented with research funders and researchers. To achieve this, we attempted to inform the whole JIA research community in the Netherlands of this prioritisation exercise from the start, and some of the research groups made the commitment to truly incorporate the Top 10 in the long-term vision of their research lines. In addition, we have organised workshops with researchers, patients, parents and clinicians to turn the relatively broad questions into research proposals that can be presented to funding agencies. These workshops took place in the fall of 2020. With these workshops, we hope to gather a group of people that is truly committed to finding answers to the research questions of the Top 10.
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