Formative work for the LEGEND (Low EnerGy diEt iN adolescents with type 2 Diabetes and obesity) study: example of PPIE in research

Abstract

This study used evidence-based guidance to report on patient and public involvement and engagement (PPIE) activities that informed the design of a feasibility trial to explore the use of a low-energy diet for children and young people (CYP) with type 2 diabetes. Overall, our PPIE work demonstrated the willingness of CYP to engage in these dietary interventions and the feasibility trial research processes. Engagement with stakeholders provided insights into factors influencing intervention participation and engagement, which will be considered in the design and implementation of subsequent trials.

Introduction

With escalating rates of childhood obesity,¹ the incidence of type 2 diabetes mellitus (T2DM) continues to increase in children and young people (CYP).^{2, 3} CYP with T2DM (CYP2D) have a higher risk of diabetes complications than those with adult-onset T2DM or type 1 diabetes mellitus (T1DM).⁴ T2DM with onset in childhood or adolescence is an aggressive form of the disease, with high rates of associated comorbidities, including obesity, hypertension, metabolic dysfunction–associated steatotic liver disease, albuminuria and neuropathy.⁵ It is associated with inequalities, being more prevalent in the most socioeconomically deprived areas and among ethnic minorities.^{5, 6} Furthermore, CYP2DM are less likely to receive all recommended health checks than those with T1DM.⁷ There is an urgent need for interventions to prevent and manage early-onset T2DM. Research reporting on interventions has mostly targeted adult populations. Low-energy diets (LEDs) have been shown to be effective for weight loss and acceptable to adults with obesity.^8-11 The Diabetes Remission Clinical Trial (DiReCT) found that almost half of the adults with T2DM who participate in LEDs as part of an intensive weight management intervention can achieve remission of T2DM (defined as glycated haemoglobin [HbA_1c] of <6.5% [48mmol/mol]).¹² In the DiRECT study, remission was sustained for 24 months for approximately one-third of adult participants with T2DM, linked to sustained weight loss.^{13, 14} However, no similar study has been conducted with CYP2D. Although research has shown that LED programmes can help CYP with obesity lose approximately 10kg,¹⁵ there is little research looking at the effects of LEDs on remission in CYP2D and obesity. Consequently, there is inconsistency and hesitancy regarding the use of LEDs for CYP2D in clinical practice. Recently, there have been calls for research into the effectiveness of an LED programme to treat paediatric obesity and T2DM.¹⁶ However, before the effectiveness of LEDs can be tested in CYP2D, the feasibility of LEDs for CYP2D needs to be established. Here, we report on the formative work that informed the design of a feasibility study on the use of LED in CYP2D.

Methods

Patient and public involvement and engagement (PPIE) is a crucial aspect of health services research and the implementation of behaviour change interventions; however, information on how members of the public are involved is often under-reported. We used evidence-based Guidance for Reporting Involvement of Patients and the Public Short Form(GRIPP2-SF),¹⁷ which includes five items on aims, methods, results, outcomes and critical perspective (Table 1).

Table 1. GRIPP2 short form

Section and topic	Item
1: Aim	The aim of the PPIE was to: Determine the study name, acronym and logo. Share views on appropriateness of study materials (eg questionnaire items). Explore views towards weight loss in T2DM. Explore perceived ability of CYP to cope with any adverse effects of LED. Explore views towards likelihood of CYP adherence to a 12-week diet programme. Explore willingness to receive text message reminders (either personally or via parent's phone). Explore willingness to use a wearable device for data collection (activity monitor). Explore willingness to undergo additional health tests to monitor safety and outcomes (blood tests/scans).
2: Methods	PPIE engagement included: Research team presentation to a YPAG (n=12) followed by group discussion and individual completion of a brief survey (with or without parent support at CYP preference). One-on-one discussions, on one occasion, between clinicians, CYP2D patients (n=5) and a parent (n=1) from one of two acute hospital sites.
3: Study results	All 17 CYP and CYP2D shared their views towards the proposed plan for the feasibility study (Supplementary File 1) and identified any barriers or enablers of participation in the dietary intervention.
4: Discussion and conclusions	PPIE contributions determined the final selection of the study name, acronym and logo. Minor revisions were made to questionnaire items to be used in the future feasibility study to allow free text comment. Participants all believed that weight loss was important in T2DM and understood the risks of diabetes. Approximately half of the participants believed that they could adhere to a 12-week LED and cope with side-effects. Some of the participants were unsure and perceived that how/whether they could cope with it may be dependent on addressing barriers to engagement and considering enabling factors that they raised. Most participants indicated that they would be happy to receive text message reminders (on their phone for older children and on their parent's phone or both for those aged ≤12 years) and wear a wrist-worn activity monitor. Participants were generally willing to undergo additional health tests for the research, although concerns were raised about timing (eg needing time out of school). This PPIE activity indicated that weight loss is perceived to be important to CYP2D, and the proposed approach for a study exploring the feasibility and acceptability of LED in CY2D is acceptable.
5: Reflections/critical perspective	This formative work gathered valuable insights from CYP and CYP2D that informed the design of a feasibility trial. Although our preference would be to have held a group event for CYP2D that was similar to YPAG for the CYP without diabetes, this was not practical for two reasons. First, the CYP2D were located in diverse geographical regions. Second, when peer-group events with CYP2D participants are planned, attendance and uptake are generally poor and potentially associated with guilt/stigma around the condition and its association with obesity. Therefore, one-on-one PPIE contacts were preferred by CYP2D. Our sample of CYP2D was small. Future PPIE should consider the influence of the interviewers on patient responses (ie participants may be more likely to agree with interview questions when the interviewer is their treating consultant). Finally, flexibility is required when gathering PPIE input in this population, as some of the younger CYP and CYP2D required parental support with completion of survey items.

• CYP, children and young people; CYP2D, children and young people with type 2 diabetes; GRIPP2, Guidance for Reporting Involvement of Patients and the Public, Version 2; LED, low-energy diet; PPIE, patient and public involvement and engagement; T2DM, type 2 diabetes mellitus; VLED, very-low-energy diet; YPAG, Young Person's Advisory Group.

Research ethics approval is not needed for PPIE activity as patients are stakeholders in the study design rather than research participants. Our PPIE involved 17 CYP completing surveys with the assistance of a PPIE coordinator (treating clinician) or the CYP's parent, where required. Surveys were completed through one of two routes: (1) a Young Person's Advisory Group (YPAG; n=12) or (2) an individual approach from the treating clinician with a one-on-one discussion (five CYP and one parent).

The YPAG event was held in a meeting room at an acute hospital trust, which lasted approximately one hour and included an oral presentation by a medical doctor with expertise in paediatric diabetes and endocrinology that lasted approximately 15 minutes. The aim of the presentation was to outline the research and discuss views regarding the research plans and any barriers to or enablers of participation in the dietary intervention. The focus of the presentation was on four areas: (1) what is T2DM, (2) T2DM in CYP, (3) previous research on weight loss in people with T2DM (adults and CYP) and (4) research plans for the Low EnerGy diEt iN adolescents with type 2 Diabetes and obesity (LEGEND) feasibility trial. The YPAG group had 13 attendees, including 12 CYP and one parent of a CYP. The CYP were 9 to 18 years old (mean age: 14.5 years) and identified as girls (n=7) and boys (n=5), White (n=8), Asian (n=3) and Black (n=1). Of the 12 CYP, nine had lived experience of a chronic health condition, and three had previously participated in research. Five CYP2D and one parent completed the questions individually over the telephone with their treating clinician. One CYP2D had previously tried LED under clinical supervision. The CYP2D were 11 to 19 years old (mean age: 14.2 years) and identified as girls (n=3), boys (n=2), Asian (n=4) and Black African (n=1), broadly reflecting the higher prevalence of early-onset T2DM in CYP of Black and minority ethnic origin.¹⁸ They were diagnosed with T2DM at <18 years of age, with a duration since diagnosis between 12 and 36 months (mean age: 20 months). All except one CYP2D completed the questions together with a parent. On a scale of 1 (not at all ready) to 10 (completely ready), their ‘readiness to lose weight’ ranged from 5 to 10 (median; 8.50; interquartile range [IQR]: 4.25). On a scale of 1 (not at all confident) to 10 (extremely confident), their ‘confidence in being able to lose weight’ ranged from 4.5 to 8.5 (median: 6; IQR; 3). One parent of CYP2D (aged 12 years) reported a need for building children's confidence in engaging in LED and providing support from the clinical team in transitioning to a food plan at the end of the intervention. Summarised findings are shown in Figure 1 (closed-response items) and Table 2 (open-ended items relating to uptake, engagement, barriers and context).

Figure 1

Survey responses (12 in CYP and 5 in CYP2D)

Table 2. Uptake, engagement, barriers and context

PPIE findings
Factors encouraging uptake	Understanding of and concerns about the risks of diabetes and obesity Understanding the advantages of weight loss (ie link between weight loss and T2DM remission) Appeal of the diet plan (eg ‘flavours’) Evidence-based diet (‘research behind it’) Potential to come off medication (‘I would do anything to make sure I do not need to be on medication’) Helping others
Facilitators of engagement with LED	A positive mindset A clear diet plan Timing (eg easier during school holidays, ideally at home the first few days to adjust) Good prevention and management of side effects (eg relaxation, hydration and use of paracetamol when needed) Support from family and friends Support from schools Various options (eg food items and flavours) Potential for flexibility (eg weekly small treat, split portions and break in the middle) Regular checkups (eg telephone, video call and in person) Reminders (eg of the purpose and to engage in diet) Countdowns, rewards and celebration
Barriers to engagement with LED	Limited options (eg flavours and food types) and boredom Lack of support from family or friends Opinions of other people (ie feeling embarrassed, ‘hiding it’) Family meals or personal events (eg birthdays) Effect on socialising Perceived stress of dieting Conflicting information (eg inaccurate information on social media) Regular time-out of school for clinic appointments No flexibility on timings (eg around examinations and schoolwork) Long duration of LED (12 wk with no break)
Contextual challenges	For some CYP, the COVID-19 pandemic has negatively affected weight, dietary and exercise behaviour, mood (ie loneliness or isolation) and motivation to lose weight.

• CYP, children and young people; LED, low energy diet; PPIE, patient and public involvement and engagement; T2DM, type 2 diabetes mellitus.

Conclusion

CYP understood the risks of diabetes and the benefits of weight loss for diabetes control and potential remission. Most respondents expressed a willingness to engage with an LED programme and were satisfied with the proposed research approaches related to health monitoring (ie additional scans and tests), data collection (questionnaire items and wearable devices for activity monitoring) and reminders (text messaging). Participants shared insights into the factors that might influence their engagement in LED, any barriers to and enablers of participation in LED and ways to cope with any side-effects. These insights informed the development of the LEGEND study, which will test the feasibility, acceptability and safety of this behavioural intervention for CYP2D aged 12 to 17 years and their families.

Declaration of interests

The authors report no conflict of interest.

Author contributions

P.S., T.B., J.M.L. and H.B. conceived the study. P.S. and H.B. prepared patient and public involvement and engagement (PPIE) materials. P.S. and E.P. delivered PPIE activity and gathered responses. H.B. drafted the manuscript. All authors reviewed and approved the final manuscript.

Funding: The Young Person's Advisory Group activity was supported by the National Institute for Health and Care Research Nottingham Biomedical Research Centre and National University Hospital Research and Innovation. The Low EnerGy diEt iN adolescents with type 2 Diabetes and obesity study is funded by Diabetes UK (reference number: 21/0006341).

Key points

• This study used the GRIPP2-SF evidence-based guidance to report on patient and public involvement and engagement (PPIE) activity for the Low EnerGy diEt iN adolescents with type 2 Diabetes and obesity (LEGEND) feasibility trial.
• Overall, our PPIE work demonstrated the willingness of children and young people to engage in a low-energy diet and LEGEND trial research processes.
• PPIE work provided insights into factors influencing intervention participation and engagement, which will be considered in the LEGEND trial.

Supplementary File 1: Study information

The Low EnerGy diEt iN adolescents with type 2 Diabetes and obesity (LEGEND) study

We are trying to collect information from young people with type 2 diabetes mellitus (T2DM) to design a research study. Please, can you tell us if this research study is something you would like to participate in?

For most young people, a normal diet should consist of around 2000 calories a day. A low-energy (low-calorie) diet is a special diet that provides between 800 and 1200 calories a day. It usually involves having low-calorie meal replacement products (such as soups, shakes or bars) instead of normal food. This diet usually lasts between 8 and 12 weeks and is called total diet replacement.

After this, there is a period of 2 to 8 weeks when normal food is introduced back into the diet. During this period, you will have both meal replacement and healthy meals. This process is performed slowly to help you get used to eating again, allowing you to return to healthy eating and a balanced lifestyle.

In adults, this type of diet helps people lose weight and achieve remission with T2DM. This means discontinuing all medicines and maintaining normal glucose levels, as if you did not have diabetes anymore. However, there is still a risk of it returning. Therefore, it is necessary to continue regular monitoring and a healthy diet and lifestyle.

This study aimed to determine whether this low-energy diet can help young people with T2DM achieve remission. You do not have to take part in this test, and you can change your mind at any time. In this case, you will continue to receive the usual care from their paediatric diabetes team.

Some adults on a low-energy diet reported constipation during the diet and weight gain after returning to the diet, and other risks might include gallstone formation (because of reduced fat in the diet) or the effect of significant weight loss on bone strength. However, the adult study did not show more side-effects in those who followed the low-calorie diet than in those who did not.

Importantly, the long-term risks of T2DM in young people are much greater than the risks discussed above, and regular visits will ensure that you are monitored carefully by your hospital team for any health problems.