Pilot study of a virtual weight management program for Duchenne muscular dystrophy

Young people with Duchenne muscular dystrophy (DMD) are at increased risk of obesity. Weight management is important to families; however, several barriers exist. This pilot study aimed to investigate the feasibility and acceptability of a co‐designed weight management program for DMD.

Results: Of nineteen eligible participants, eight were enrolled (median age 11.4 years, range 4.9-15.8),and seven completed the program.Visit attendance was high (88%-100%); most participants reported high satisfaction and that participation was easy.
Suggested changes included online and visual DMD-specific resources.At 6-weeks, median change in weight z-scores was À0.01 (IQR: À0.23, 0.17) indicating that on average, weight gain tracked as expected for age.Waist circumference measured by caregivers lacked accuracy and the completion rate of caregiver-reported secondary outcome measures (e.g., food diaries) was low.
Discussion: A co-designed, telehealth/phone weight management program appeared to be feasible and acceptable in a small group of boys with DMD.An adapted, hybrid telehealth and face-to-face program is recommended for efficacy testing.

| INTRODUCTION
2][3] When compared to typically developing children, body mass index (BMI) is 1.0 kg/m 2 higher in males with DMD; and weight velocity appears to increase from age 7 years. 4A complex range of factors contributes to weight gain including: long term steroid treatment and its effects on appetite and weight, reduced physical activity and consequently total energy expenditure due to progressive physical disability, 5 reduced resting energy expenditure, 6 and sleep disturbance and fatigue. 7In some young people with DMD, energy intake is above estimated requirements 8,9 and dietary quality is suboptimal. 9Parents and other carers ("caregivers") also report that fussy eating, sensory preferences, and time constraints are barriers to healthy eating and weight management. 10ight management is a priority for young people with DMD and their families, yet there is a lack of tailored nutrition and weight management information provided in healthcare settings. 10Health professionals working with individuals with neuromuscular conditions may also lack the confidence to address weight management. 11Weight management strategies tailored to DMD are needed to overcome these barriers and improve health professional confidence.However, literature on weight management approaches in DMD is sparse; two case studies have investigated weight management strategies in DMD. 12,13e further study found solution-focused coaching for physical activity and nutrition goal-setting may be an acceptable and feasible approach for young people with DMD. 14 A recent systematic review found that there are no other published weight management interventions for either children with DMD or other physical disabilities. 15e primary aim of this study was to investigate the feasibility and acceptability of a co-designed, lifestyle, weight management program for young people with DMD.The program design was informed by a survey of caregivers that identified preferences for a holistic, short, intensive program. 10Secondary outcome measures were changes in weight, waist circumference, physical function, quality of life, self-reported fatigue levels, dietary intake, and adverse events.

| Intervention co-design
The program was developed using a co-design approach with both families and health professionals to ensure that the program was family centered and evidence-based.6][17] Surveys were used to gather initial information about content, length, and style of delivery and program outcomes preferred by families (n = 27). 10Face-to-face interviews with a range of health professionals were used to gather opinions on program duration, delivery mode, and outcome measures.After this initial 'ideation' phase, a draft program of content, style, and delivery mode was developed as a prototype model.This prototype was further tested with families (n = 2) and with health professionals including neurologists (n = 2), physiotherapists (n = 2), dietitians (n = 2) and the research team.Health professionals provided additional feedback on outcome measures and changes to the prototype were made to reduce potential burden on families.The outcome of this co-design process was Supporting Nutrition and Optimizing Wellbeing Program (SNOW-P)-an intensive lifestyle weight management program delivered intensively over 6 weeks with 12 weeks total follow-up.

| Adaptation due to the COVID-19 pandemic
Due to the pandemic restrictions in Melbourne, Australia (2020-2021), all non-essential patient-facing clinical and research activities were required to adapt in order to mitigate infection risk and adhere to local restrictions.SNOW-P was adapted to enable an entirely virtual delivery via telehealth (video call) and phone, whilst maintaining all content as elucidated in the co-design process.See Table S1 for program outline.

| Study design
This was a single-arm, feasibility, and acceptability pilot study of SNOW-P.All study procedures were approved by the Royal Children's Hospital (RCH) Research and Governance office (HREC/58876/RCHM-2019).Informed consent was obtained from a legal guardian.All boys participating in the study provided either written consent if able (depending on cognition and maturity determined by discussion with caregivers) or verbal assent.The study protocol was prospectively registered (ACTRN12620000167965 available from: www.anzctr.org.au).

| Setting
SNOW-P was delivered through a single pediatric neuromuscular clinic at RCH.This is one of Australia's largest pediatric neuromuscular clinics and provides care for children with a neuromuscular disease within Victoria and Tasmania and some children from the Northern Territory.All recruitment and documentation were conducted through RCH and the intervention was delivered and outcomes were measured virtually in the participants' homes.All study visits were conducted outside of routine neuromuscular clinic visits.

| Participants
Young people attending RCH who were 18 years or younger with a diagnosis of DMD confirmed by genetic testing or muscle biopsy and who had obesity were eligible to participate.Criteria for obesity was a BMI z-score ≥1.64 (equivalent to BMI ≥95th percentile) according to the Centers for Disease Control and Prevention (CDC) BMI-for-age male growth charts within the last 6 months. 18The BMI z-score value most recently recorded in the participant's electronic medical records was used to determine eligibility.Access to a device with an internet connection to enable telehealth and/or a phone was a requirement.
Exclusion criteria were: enrolment in another interventional study; documented significant illness that may contraindicate participation in a weight management program; enrolment in another formal weight management program; or attending a specialist weight management clinic.Due to the need for the intervention to be delivered via telehealth, the program could only be offered to families who could converse in English because interpreters were not available outside clinical appointments due to pandemic restrictions.Participants with neurodevelopmental disabilities were eligible for inclusion provided they met all other eligibility criteria.A pre-program phone call explored eating behaviors to screen for any characteristics that may contraindicate participation in a weight management program for example, obsession with counting kilojoules in a young person with obsessive-compulsive disorder.Medication use, including metformin was not an exclusion criterion.Potential participants were identified through neuromuscular patient lists or by referrals from treating clinicians.Additionally, caregivers who expressed interest in participating in the program during the co-design process were sent information and invited to discuss eligibility with a member of the research team.A simplified version of the feedback questionnaire was provided to some participants with DMD depending on maturity and level of cognition.For those who withdrew from the program, caregivers were asked to complete a questionnaire including reason for withdrawal; barriers to participating in the program; and recommended changes to the program to improve acceptability or feasibility.All families were also phoned by a researcher independent to the dietitian delivering the program (ZED) to provide an additional opportunity for feedback.

| Secondary outcome measures
Clinical measures were included as secondary outcomes to inform future trials.Secondary outcomes were measured at baseline, 6 and 12 weeks and included: anthropometry (self-reported weight and waist circumference), physical function (timed supine-to-stand for ambulatory participants and timed can stacking exercise for nonambulatory participants), quality of life (Pediatric Quality of Life Inventory DMD module, PedsQL DMD), fatigue (rating scale) and dietary intake (3 day food record).Adverse events were also recorded.A detailed description of these measures is provided in Table S2.

| Data analysis
Participant-level and descriptive data are reported where appropriate.
Participant-level data were reported due to the small sample size and due to the feasibility and acceptability study design.Feasibility and acceptability questions in the feedback questionnaire were scored on a scale of 1 (not satisfied at all/not very easy to participate) to 5 (very satisfied/very easy to participate).Continuous descriptive data were analyzed using SPSS Version 26.0 (IBM, Armonk, NY) and are reported as median and IQR.

| Participant characteristics
Nineteen participants were screened for eligibility.Of these, 10 families did not respond to the invitation to participate and one declined participation.The remaining eight participants met eligibility criteria and were enrolled in the program (Table 1).The median age of participants was 11.4 years (range 4.9-15.8)and all but one were treated with steroids.One participant was taking metformin to assist with weight management.

| Feasibility and acceptability
Of the eight participants enrolled, one withdrew and seven completed the intervention and follow-up period.Of those who completed the program, five attended 100% of the scheduled visits and two attended seven out of eight scheduled visits (one phone review was not attended).The extra education topics selected by caregivers were meal preparation and cooking (n = 4), followed by activities and interests, nutrition supplements (both n = 3), strategies for a healthy lunchbox and mindfulness (both n = 1).
Five caregivers, of whom one was a parent of two participants (participant 6 and 7) with DMD, and three young people with DMD completed the post-program questionnaire (see Tables 2 and S3).This represents six of the seven families who completed the program.Participants were mostly highly satisfied with the program and all recorded they would recommend it to another family with a son with DMD.The majority (7/8) of caregivers and participants rated ease of participation as either a four or five.All respondents liked participating in the program from home and the acceptability of telehealth visits emerged as a theme in the open-response feedback.Suggested T A B L E 1 SNOW-P baseline participant characteristics.
No. changes were having some face-to-face sessions, an online platform with DMD-specific nutrition information and more visual resources for young people with autism spectrum disorder.Beneficial aspects of the program were the weekly visits and speaking to someone knowledgeable in nutrition in DMD.

Age
One participant changed their mind about participating and withdrew after enrolment prior to the first session.The caregiver reported that issues around coping with COVID-19 restrictions including home schooling made it difficult for them and their son to participate in the program.
Response to the program in anthropometric outcomes is described in Table 3.Two participants lost weight and weight-z scores decreased in three participants.Waist circumference measurements T A B L E 2 Findings from the caregiver and participant post-program questionnaire.lacked accuracy suggested by the extreme changes in measurements.
Changes in the other secondary outcomes are outlined in Tables S4   and S5.
Three mild adverse events were recorded across two participants all of which were assessed as being unrelated to the intervention by the study Principal Investigator (ZED).One participant experienced an unwell episode with headaches, mild fever, pain, and fatigue with some symptoms likely related to change in steroid regimen.The second participant had two unwell episodes both due to a respiratory tract infection with symptoms including loss of appetite.

| DISCUSSION
This study demonstrates that a co-designed, six-week, intensive lifestyle program delivered virtually may be a feasible and acceptable approach to weight management for some young people with DMD and their caregivers.Most participants who completed SNOW-P were highly satisfied with the program and all would recommend it to other individuals with DMD.Most families reported it was easy to participate and the virtual mode of delivery was acceptable.There were high rates of attendance for the scheduled sessions and low withdrawal rates.There was variability in weight responses to the program and collection of several secondary outcome measures was not feasible in a virtual environment.Outcomes with incomplete data were those requested to be completed outside of schedule visits including food diaries, electronic surveys measuring quality of life and global rating of change in fatigue questionnaire.
Family-centered interventions delivered weekly that utilize goalsetting and motivational interviewing techniques may be a promising solution to improving weight status, dietary intake, and physical activity in DMD.An earlier study found a solution-focused coaching intervention (SFC-Peds) delivered using a combination of face-to-face and video call visits, may be a feasible and acceptable approach for diet and physical activity goal setting in young people with DMD (n = 5). 14 SNOW-P, the dietitian delivering the intervention also assumed the role of a coach, for example by prompting participants and/or caregivers to identify their own goals, using affirmation and problem solving to overcome barriers to implementing nutrition, activity, and behavioral strategies.Evidence from the prior SFC-Peds study and from the current SNOW-P study consistently demonstrated high rates of completion, attendance, reported satisfaction and perceived benefits among young people with DMD and their caregivers. 14spite being an adaptation due to pandemic restrictions, telehealth visits were considered a favorable component of the program by families to receive weight management advice.During the pandemic, telehealth was the status quo for many healthcare appointments; this may have contributed to the ease of utilizing this mode of delivery.Telehealth weight management interventions have also shown to be acceptable, feasible and efficacious in typically developing young people with obesity. 19,20Telehealth visits can provide unique insights into the home food environment; for example families can share food brands and labels.For young people with complex needs such as DMD, telehealth may be preferred to avoid additional time and burden attending healthcare appointments in-person. 21wever, this study demonstrates that outcome measures conducted within the participants' homes may be unreliable, inaccurate and lead to incomplete data.To understand the efficacy on weight and health outcomes in DMD, hybrid models should be considered with flexible telehealth intervention delivery and face-to-face outcome measures.
The regular weekly visits also emerged as an accepted aspect of SNOW-P.Intensive programs (e.g., weekly visits) are a key aspect of efficacious weight management programs for both typically developing young people and those with chronic healthcare needs. 15,22terventions delivered at a high intensity allow time for small, achievable goals to be set without compromising on effectiveness and this model has been utilized by up-scaled community-based weight management programs. 23ilst the median weight z-score at 12-week follow up was À0.01, participant-level analysis of weight outcomes (n = 6) demonstrated variability in response to the program.Four out of 6 participants gained weight, but for two of these boys, weight z score remained relatively unchanged.This suggests that whilst weight is increasing, weight gain is tracking as expected for age and gender as opposed to accelerated weight gain velocity.Accelerated weight gain was still observed in two boys which aligns with observational studies. 4We were unable to accurately obtain height measures from participants so we could not consider changes in BMI z-score.This is an important limitation of this study.For young people who are overweight or obese, weight maintenance is recommended during linear growth while gradual weight loss is indicated for post-pubertal adolescents. 24Although height growth may be slow in DMD, 25 weight or weight z-score maintenance are both preferred over an increasing weight trajectory.Some waist circumference measures did not appear to be accurately measured in participant's home.If waist circumference is to be measured by caregivers, further detailed instructions are required to improve accuracy of measurements.
Completing secondary outcome measures outside of scheduled visits was not feasible for some families.These outcomes included dietary intake measured using food diaries and quality of life and fatigue measured with questionnaires.Whilst there is some evidence to support the validity of food diaries to measure dietary energy intake in DMD, 26 anecdotally families find food diaries burdensome.
In future research, it is worth considering quicker methods of dietary assessment that can be completed in a single phone call or visit, such as a food frequency questionnaire or 24 h recall.Alternatively, a combination of telehealth delivery of content but with face-to-face visits for outcomes measures, particularly weight, height and waist circumference, may minimize missing data and overcome the limitations associated with self-reported anthropometric measures.
There are several limitations of this pilot study.The outcomes need to be considered in the context of the small sample size and high risk of bias inherent in the study design.The program would be strengthened if delivered by a multidisciplinary team, such as a dietitian, physiotherapist, and mental health clinician.Generalizability is limited because only families fluent in English language were included.

2. 6 |
Intervention delivery SNOW-P was delivered by one dietitian (NB) over eight virtual visits.Table S1 provides a detailed overview of the program including preprogram procedures.This included 6 weeks of intensive intervention delivery and an additional 6 weeks of follow-up.The telehealth sessions involved education and goal setting with participants with DMD and their caregivers.During the first telehealth session (week 0) families were provided a report with detailed written suggestions based on their pre-completed food and hunger diary.Motivational interviewing techniques were used throughout telehealth and phone sessions.The final two visits delivered at weeks 6 and 12 involved planning for goals and weight management strategies for the following 6 weeks and post-program periods, respectively.The final session included recommendations for future support.

Feasibility
and acceptability were measured with attrition and program uptake data including: participants referred, approached or who expressed interest; reason for ineligibility; participants enrolled; withdrawals and reasons; program completers; and lost to follow up.Caregivers who completed the program were asked to complete a feedback questionnaire via REDCap that included: overall satisfaction with the program; likelihood of recommending the program; perceived success of the program; ease and feasibility of participating; best aspects and recommendations for changes to the program; and the impact of the COVID-19 pandemic on the feasibility of participating.
Anthropometric measures at baseline, week 6 and week 12.