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Development of obesity management medications (OMMs) for use in children and adolescents calls for patient-reported outcome (PRO) measures to evaluate treatment efficacy. Existing weight-specific symptom and impact PROs are limited to age ≥ 11 years and may not fully capture treatment benefit of new OMMs.
Methods
A targeted literature review was conducted to identify obesity symptom and impact outcomes and weight-specific PROs relevant to children and adolescents 6–17 years of age. Concept elicitation (CE) interviews were conducted by telephone with children and adolescents, including parents of children 6–11 years of age, to identify relevant weight-specific concepts. Key concepts relevant to three developmental age groups—6–7, 8–11, and 12–17 years—informed the creation of a pediatric weight-specific PRO to assess key symptoms and impacts of obesity: the Pediatric Weight Questionnaire (PWQ). Cognitive interviews (CI) were conducted by telephone to assess the comprehension, relevance, and comprehensiveness of the PWQ across the three age groups.
Results
Eight domains were identified from the literature as relevant to pediatric obesity: physical symptoms, physical function, psychological health, emotional behavior/function, family and social relationships, school functioning, and health-related quality of life. No existing weight-specific PRO encompassing these domains was identified. Twenty CE interview participants (mean age 11.4 years, 60% female, 50% Hispanic/Latino) confirmed that physical symptoms and physical, emotional, and social impacts were most relevant to their weight experience. The 23-item PWQ for adolescents 12–17 years of age and the 17-item PWQ for children 8–11 years of age are self-administered, while the 13-item PWQ for children 6–7 years of age is interviewer-administered. The three versions of the PWQ were evaluated with 34 CI participants (mean age 12 years, 64.7% female, 17.6% Hispanic/Latino), who found the PWQ easy to complete, relevant, and comprehensive.
Conclusion
The PWQ provides a means of assessing the impact of obesity and benefits of treatment in children and adolescents 6–17 years of age with obesity. Study findings support the content validity of this new measure for use in pediatric obesity clinical trials and observational research.
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Key Summary Points
In this study, researchers developed a questionnaire, the Pediatric Weight Questionnaire (PWQ), with input from children and adolescents aged 6–17 with obesity.
The PWQ is the first known weight-specific questionnaire to be developed for children as young as ages 6–7 years and up to 17 years of age.
The PWQ is designed to assess how young people with obesity feel and function in relation to their weight and can be used in research studies assessing obesity and obesity treatments.
Introduction
Obesity is a major public health concern due to its association with increased risk of complications including cardiovascular disease, high blood pressure, type 2 diabetes, respiratory disease, and numerous forms of cancer. It has been reported that children and adolescents with obesity are three times more likely to develop hypertension than children without obesity [1, 2]. Furthermore, obesity is correlated with poor quality of life and an increased prevalence of anxiety, depression, low self-esteem, and social stigma [3‐7].
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Childhood obesity can be prevented by adopting a healthy lifestyle, awareness among parents on proper nutritional requirements, and adequate exercise regimes. Despite these interventions, some children continue to experience difficulty in weight management and develop obesity-related comorbidities.
To evaluate effectiveness in future treatment trials and observational research with children and obesity, valid patient-reported outcome (PRO) measures are required. The FDA requires sponsors of treatment trials to provide evidence of a PRO’s fitness-for-purpose in the target patient population. According to this guidance, a PRO instrument is not considered a credible measure to support a labeling claim without evidence of its relevance to the intended target patient population. Existing weight-specific PRO measures are limited to certain age ranges and might not fully capture treatment benefit of new obesity management medications (OMMs). Furthermore, there is no current measure identified in pediatric obesity for use specifically among those aged 6–7 years.
To guide this research, the study followed the FDA PRO Guidance for Industry [8], Patient-Focused Drug Development Guidance documents [9, 10], and the Professional Society for Health Economics and Outcomes Research (ISPOR) Task Force Reports for PRO content validity [11, 12]. The FDA guidelines highlight the importance of providing evidence that a PRO instrument comprehensively reflects the perspectives of the target sample (i.e., evidence of content validity). We present findings from the development of a novel PRO instrument for assessing the impact of obesity in children 6–17 years of age: the Pediatric Weight Questionnaire (PWQ™).
Methods
Study Overview
This study included four steps: (1) targeted literature review (TLR); (2) concept elicitation (CE) interviews; (3) conceptual framework and development of the draft PWQ; and (4) cognitive interviews (CI). An overview of the project flow is presented in Fig. 1. The CE interviews and CI were noninterventional, cross-sectional, qualitative studies involving semi-structured interviews with children and adolescents 6–17 years of age and are detailed further below.
Fig. 1
Study steps. CE concept elicitation, CI cognitive interview, PRO patient-reported outcome, PWQ Pediatric Weight Questionnaire, TLR targeted literature review
The TLR explored qualitative and interventional studies for pediatric obesity published between January 2010 through December 2021 to capture symptom, impact, and/or health-related quality of life (HRQoL) concepts relevant to children 6–17 years of age with obesity. The key objectives to the TLR were: (1) to identify key concepts of interest (COIs) to children and adolescents living with obesity to gain a greater understanding into the patient perspective surrounding the characteristics of obesity (symptoms and impacts) and HRQoL (physical and emotional functioning, the impact on daily activities); and (2) identify relevant PRO measures used to assess symptoms, impacts, and HRQL in children and adolescents with obesity and to assess the appropriateness of these measures for the evaluation of symptoms and impacts in this target population.
As a result of the TLR, a semi-structured interview discussion guide was developed for use in CE interviews with children 6–11 years of age and their parents, and adolescents 12–17 years of age.
Concept Elicitation Interviews
The CE interview protocol was approved by a US ethical central institutional review board (IRB), in accordance with local law (IRB study number WCG IRB# 20225362), and the methodology was developed in line with the FDA and the ISPOR guidance on best practice for eliciting concepts for a new PRO instrument [9, 11]. This study was also performed in accordance with the ethical principles that have their origin in the Declaration of Helsinki and that are consistent with good clinical practice and applicable regulatory requirements. Written informed consent was obtained from all participants to publish data and verbatim responses.
Pediatric Weight Questionnaire Development
Based on the TLR findings, a preliminary item concept pool was created and a preliminary conceptual model related to impacts of pediatric obesity were developed, which included the domains of physical symptoms, physical impacts, emotional/psychological impacts, school functioning, and social impacts. The COIs hypothesized to be most closely related to pediatric obesity experience were physical symptoms and physical impacts, followed by emotional and social impacts; the concepts posited to have less direct links to obesity experience were school function and school impacts. Also, school functioning and school impact concepts, such as bullying and lack of focus, were found to overlap with concepts in the emotional and social domains. After the CE interviews, the COIs reaching saturation were reviewed against the available weight-specific and generic PRO measures, and the PRO development team made a decision to pursue a de novo measure. Concepts that had evidence of saturation were proposed for item development [13]. This instrument is available for public use. To obtain a copy, contact copyright@lilly.com.
Cognitive Interviews
The protocol from the CE interview study was modified and approved by the same US IRB, and followed the FDA and the ISPOR on best practice for assessing respondent understanding of a new PRO instrument [10, 12].
Study Populations and Patient Recruitment
We conducted two cross-sectional, qualitative studies including one-on-one CE interviews and CIs with children ages 6–11 years of age and their parents, and adolescents 12–17 years of age with obesity. The CE and CI study participants were identified through clinical sites and a third-party vendor via patient associations, physician referrals, patient panels, and social media. A recruitment flyer and social media recruitment advertisement was provided to clinical sites and the vendor to assist recruitment efforts. Efforts were made to recruit equal numbers of participants in four distinct age groups—6–7, 8–11, 12–14, and 15–17 years—to capture concepts relevant across these ages.
Inclusion criteria for all populations were as follows: aged 6–17 years at enrollment, body mass index (BMI) of approximately 95th percentile age- and sex-specific Centers for Disease Control and Prevention percentile (if BMI was not provided, it was calculated at screening); clinician-confirmed weight and height or self-report of weight and height; and confirmation of one of the following as defined by: self-reported family history of obesity (e.g., parent, grandparent, siblings) or clinician-confirmation of obesity-related complications, such as hypertension, prediabetes, and metabolic dysfunction-associated steatotic liver disease, or self-reported current or past experience with obesity treatment, such as lifestyle or behavioral counseling, pharmacotherapy, or surgery; and parents or guardians of children 6–17 years of age able to provide consent for their child’s interview and consent to a parent interview for children ages 6–11. Additionally, prospective participants had to be able to participate in an approximate 90-min in-person or telephone interview and complete study questionnaires in English and be audio-recorded during the discussion.
Exclusion criteria included participation in a current clinical trial for investigational treatment of obesity, taking incretin-based medications at the time of screening; individuals with genetic or other medical conditions that may contribute to obesity development (e.g., Prader Willi syndrome, Down syndrome, Cushing syndrome) as well as any medical condition that, in the opinion of the investigator, would interfere with participation in an interview and/or completion of the study procedures.
Once eligibility was confirmed, parents of participants reviewed and electronically signed the informed consent form, and the child or adolescent signed the assent form. Telephone interviews were scheduled (in-person interviews were also offered) and, prior to the scheduled interview date, participants were sent a secured link to the sociodemographic questionnaire via email to complete prior to the interview.
Semi-structured CE interview guides were developed for children and adolescents 6–7, 8–11, and 12–17 years of age, and parents/guardians of children 6–11 years of age. The CE interview guides were developed based on the concepts identified in the TLR. The first section of the guide explored the experience of weight and weight management. Separate sections focused on eliciting the child’s or adolescent’s understanding of symptoms related to their weight, including the description, location, severity, duration, frequency, and degree of bother for each symptom, and perceived changes with weight loss. The final participant section aimed to understand physical, emotional, or social impacts experienced by adolescents and perception of change in these impacts with weight loss. Adolescents participated in a severity rating activity and a ranking exercise for the top three symptoms and impacts important for monitoring with weight loss. The last section of the guide for children 6–11 years of age gathered information about child-report of and parents’ observations of signs or impacts from obesity. Parents participated in a ranking exercise for the top three symptoms and the most important to change with weight loss.
Semi-structured Cognitive Interview Guides
Semi-structured CI guides reviewed the PWQ with children and adolescents 6–7, 8–11, and 12–17 years of age, and parent/guardians of children 6–11 years of age. After brief discussion about their weight symptoms and impacts, participants completed the PWQ for their age group. Subsequently, the interview focused on the child or adolescents’ general impressions, understanding, comprehension, and relevance of the PWQ to assess the clarity of the instructions, items, response scale, and recall period. The parent section of the CI discussion guide probed observations of their child’s comprehension of the PWQ questions and the relevance of the questions for their child’s age (6–11 years).
For the CI guide for children 6–7 years of age, two 5-point rating response option scales were explored to confirm understanding and scale preference. One response scale depicted a set of five speedometer gauges with increasing speed depicted and the second alternative response scale included a set of five drinking glasses showing increasing levels of fullness, corresponding to the 5-point rating scale.
Sociodemographic and Clinical Characteristics Questionnaire
An electronically administered questionnaire captured self-reported information about age, sex, race/ethnicity, living situation, weight perception, desired weight goal, doctor concern about weight or recommendation for weight management or weight loss, weight loss efforts, weight loss approaches, and comorbidities.
Adolescents 12–17 years of age completed the questionnaire unassisted. Parents of children 6–11 years of age were permitted to assist their child in completing the questionnaire.
Clinical Form
For participants recruited from clinical sites, physicians were asked to complete a clinical form that included questions about the child or adolescent’s weight, weight loss efforts, and comorbidities.
Interview Methods
Experienced qualitative interviewers trained in study-specific objectives and adverse event reporting requirements were trained. Interviewers were male and female and had a minimum of a master’s degree and had more than 5 years of qualitative research experience. A mock practice interview was conducted by each interviewer and feedback was provided.
Interviews were conducted over the telephone and followed a semi-structured interview guide and lasted approximately 90 min. CI study participants were mailed a study packet with a copy of the PWQ in a sealed envelope with instructions to only open when instructed at time of the interview for review during the interview. Post-interview, participants were compensated for their time according to fair market value rates. For CE interviews, symptom and impact concepts were examined for saturation by monitoring the first mention and percentage of new concepts identified in each interview. For CI interviews, comprehension issues arising during the review of the PWQ were tracked in an item tracking matrix by interviewers to monitor PWQ feedback and assess saturation.
Data Management and Analysis
For the CE interviews and CIs, interview audio files were transcribed verbatim, and transcripts were quality-checked to ensure interviews were conducted in compliance with the interview guide and consistently across different interviewers and remove any potential identifying information. A qualitative analysis plan and codebooks were developed for the CE interviews and CIs outlining the analyses to be conducted. The CE and CI codebooks were developed based on the semi-structured interview guides and were modified and updated throughout the coding process (as needed). Three trained qualitatively trained team members coded the transcripts. The coding was performed using ATLAS.ti version 22 qualitative analysis software. Once coding of all transcripts was complete, a second team member thoroughly reviewed a proportion of coded transcripts to ensure accuracy and consistency across transcripts, as well as adherence to the final codebook.
CE transcripts were coded for COIs related to weight experiences. Saturation of concepts was monitored within each age group. The definition for saturation was the point at which no new concepts emerged in successive CE interviews, with the aim of 100% saturation [14]. Concept saturation was assessed in the overall sample concurrent to the conduct of interviews and at the end of the study with coded data.
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CI transcripts were coded for comprehension, relevance of item concepts, instructions, response scale, recall period, and ease or difficulty of selecting a response for each PWQ item. The coding and analysis organized the interview data according to feedback specific to instrument instructions, item number, response options, recall period, any missing concepts, or suggested wording changes. For CI interviews, saturation was defined as the point at which there were no remaining issues or problems with comprehension of item meaning, or relevance or comprehension of understanding of clinical outcome assessment components [12].
A content analysis approach was used to analyze the coded data to allow for quantifying the number of participants endorsing concepts or assessing understanding, relevance, and comprehension of the features of the PWQ. Qualitative findings were summarized with frequencies, and percentages and exemplary quotes (as appropriate). Descriptive statistics (e.g., number/frequency, mean, SD) were used to summarize sample sociodemographic and clinical characteristics.
Results
TLR Findings
Key domain-level concepts identified from the TLR studies included physical symptoms, physical impacts, psychological health, emotional behavior/function, family and social relationships, school functioning, and HRQoL. A preliminary item concept pool was created. The disease-specific PRO measures identified in the TLR included: Impact of Weight on Quality of Life – Kids (IWQOL-Kids) (age range 11–19 years) [15], Weight-Bias Internalization Scale (age range 9–18 years) [16], Sizing Me Up (age range 5–13 years) [17], and Youth Quality of Life-Weight (age range 11–18 years) [18]. The PRO measures were mapped to the concepts identified from the pediatric literature. Those with the broadest concept coverage were the IWQOL-Kids and Sizing Me Up measures. However, neither had adequate concept coverage for the entire target pediatric population (ages 6–17 years). After reviewing the results from CE interviews, the item pool, conceptual disease model, and conceptual framework for measuring pediatric obesity were revised (Fig. 2).
Fig. 2
Conceptual framework for weight-specific patient-reported outcome for ages 12 to 17. *Blue = 6–7-year-old. *Red = 8–11-year-olds. Concepts shared across age groups. This instrument is available for free public use once copyright permission is obtained. To obtain a copy, contact copyright@lilly.com. PWQ Pediatric Weight Questionnaire
Qualitative CE interviews were conducted with 10 adolescents 12–17 years of age and 10 children 6–11 years of age each with one participating parent/guardian. Table 1 provides an overview of the demographics for CE interview participants aged 6–17. The mean age across the total sample was 11.4 years, and most participants were female (n = 12/20, 60%). There was an even split between those who were Hispanic or Latino and those who were not Hispanic or Latino (n = 10/20, 50%, respectively), and more than half of the participants were people of color (n = 11/20, 55%). Three-quarters of the sample lived with both parents (n = 15/20, 75%).
Table 1
Concept elicitation: participant-reported sociodemographic and clinical characteristics (n = 20)
Variables
Total child/adolescent participants (n = 20)
6–7 years of age (n = 5)
8–11 years of age (n = 5)
12–17 years of age (n = 10)
Age (years)
n
20
5
5
10
Mean (SD)
11.4 (3.6)
6.6 (0.5)
10.0 (1.2)
14.4 (0.)
Median (range: min–max)
11.5 (6.0–17.0)
7.0 (6.0–7.0)
10.0 (8.0–11.0)
14.5 (12.0–17.0)
Sex, n (%)
Male
8 (40.0%)
2 (40.0%)
3 (60.0%)
3 (30.0%)
Female
12 (60.0%)
3 (60.0%)
2 (40.0%)
7 (70.0%)
Ethnic background, n (%)
Hispanic or Latino
10 (50.0%)
4 (80.0%)
1 (20.0%)
5 (50.0%)
Not Hispanic or Latino
10 (50.0%)
1 (20.0%)
4 (80.0%)
5 (50.0%)
Racial background, n (%)
White
9 (45.0%)
1 (20.0%)
3 (60.0%)
5 (50.0%)
Black or African American
7 (35.0%)
2 (40.0%)
2 (40.0%)
3 (30.0%)
Othera
4 (20.0%)
2 (40.0%)
0 (0.0%)
2 (20.0%)
Current living situation, n (%)
With both parents
15 (75.0%)
3 (60.0%)
4 (80.0%)
8 (80.0%)
With only mother
5 (25.0%)
2 (40.0%)
1 (20.0%)
2 (20.0%)
Weight perception
Overweight/obese
16 (80.0%)
3 (60.0%)
4 (80.0%)
9 (90.0%)
Too thin
1 (5.0%)
0 (0.0%)
0 (0.0%)
1 (10.0%)
About the right weight
3 (15.0%)
2 (40.0%)
1 (20.0%)
0 (0.0%)
BMI (kg/m2)
n
20
5
5
10
Mean (SD)
31.3 (8.0)
21.7 (2.6)
33.2 (8.9)
35.1 (4.2)
Median (range: min–max)
31.7 (19.8–44.4)
19.8 (19.8–25.2)
30.9 (22.9–43.3)
35.5 (24.9–44.4)
BMI category
Underweight (below 18.5)
0 (0.0%)
0 (0.0%)
0 (0.0%)
0 (0.0%)
Healthy weight (18.5–24.9)
6 (30.0%)
4 (80.0%)
1 (20.0%)
1 (10.0%)
Overweight (25.0–29.9)
2 (10.0%)
1 (20.0%)
1 (20.0%)
0 (0.0%)
Obesity (30.0 and above)
12 (60.0%)
0 (0.0%)
3 (60.0%)
9 (90.0%)
Desired weight goal
Less
16 (80.0%)
2 (40.0%)
5 (100%)
9 (90.0%)
About the same
4 (20.0%)
3 (60.0%)
0 (0.0%)
1 (10.0%)
Doctor concerned about weight, Yes
16 (80.0%)
2 (40.0%)
4 (80.0%)
10 (100%)
Doctor recommended weight management/loss, Yes
16 (80.0%)
2 (40.0%)
4 (80.0%)
10 (100%)
BMI body mass index
aOther race includes: American, Hispanic, Mexican, White; Black Or African American
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Most of the sample reported that their doctor was concerned about their weight (n = 16/20, 80%) and recommended the child/adolescent consider weight management/loss (n = 16, 80%). A small number of the study participants (n = 4/20, 20%) had sought help to lose weight in the past 12 months. The most common comorbidities reported were sleep problems/sleep apnea (n = 6/20, 30%), anxiety/depression (n = 5/20, 25%), and asthma (n = 4/20, 20%).
Symptom and impact concepts identified commonly across age groups are highlighted in Table 2. Evidence of concept saturation was assessed within each of the domains of physical symptoms, physical impact, emotional impact, and social impact for consideration in the PRO development.
Table 2
Concept elicitation interview findings: weight-related concepts of interest
Concept
Total (n = 20)
Age groups (n, %)
Illustrative quotes
Children and adolescents
6–7 (n = 5)
8–11 (n = 5)
12–17 (n = 10)
Physical symptoms, n, %
Body aches/pain
15, 75%
4, 80%
4, 80%
7, 70%
001-009_14yo_F: When I have body pain, I would say like it feels heavy, or it feels like aching
001-017_8yo_F: My feet hurt sometimes […] I can’t play for too long
Get tired easily
13, 65%
3, 60%
5, 60%
5, 50%
002-006_16yo_M: I would say I’m tired a lot.., I can get tired easily. Like sometimes I’m not tired and then I just get tired. But I mean sometimes I just stay tired the entire day and I just go through the day tired
003-012_6yo_M: I get tired really fast
Breathing difficulty
11, 55%
3, 60%
3, 60%
5, 50%
003-007_13yo_F: I feel most of the time fine. Just also going, like obviously in school, going to a lot of classes, l sometimes during the day I could get out of breath more than someone else who's not overweight
001-005_16yo_F: If I'm walking my pace, I can last of more than I would. If I'm walking a faster place pace than I normally would, I will lose my breath in a couple of minutes
Low energy
7, 35%
–
–
7, 70%
001-011_12yo_M: So, when I wake up, I just don’t have enough energy to like, you know, stand up after I wake up, because I’m really tired
002-006_16yo_M: Well, when I’m doing things, I can stay engaged but I do have little energy. Like I can go- when I wake up, I’m tired. I’ll go through the day tired, but I stay active. I would say I do have low energy though, throughout the day
Skin rashes/irritation
5, 25%
–
2, 40%
3, 30%
003-007_13yo_F: I have sometimes occasionally get rashes on like my legs or my arms. And I think it's like related to my weight because specifically like when I lost weight, I didn't really get it so much. Now I don't really have rashes anymore. But because I lost weight, so that's why I think like it's related to that
Physical impacts, n, %
Hard to run
14, 70%
3, 60%
4, 80%
7, 70%
Interviewer: Why do you think you’re a little bit heavy? 003-011_11yo_M: Because I have a hard time running
003-012_6yo_M: Like if I run like only for like 5 s, 10 s, then I already get tired
003-012_6yo_M: I don’t like being fat because I feel like being fat is the problem about my life because I want to be fast. I want to run fast like the Flash
Hard to walk
6, 30%
1, 20%
3, 60%
2, 20%
Interviewer: Okay. What about walking, do you feel like it’s different for you if you walk a short distance versus a long distance? If you walk a short distance, is it easy or hard?
003-011_11yo_M: Most distance are hard
003-007_13yo_F: Yeah, just walking, if it's like 15 to 20 min, then I could take just like a minute break and then I feel like a better
Doing sports or activities
10, 50%
1, 20%
4, 80%
5, 50%
003-012_6yo_M: I feel like my weight needs to go away so I can dance so I don’t fall or get hurt when I dance because of this weight
Difficulty standing
8, 40%
0, 0%
3, 60%
5, 50%
001-006_14yo_F: If I’m like standing a certain way also [back pain due to obesity]
Bending, kneeling
5, 25%
0, 0%
2, 40%
3, 30%
002-006_16yo_M: I would say like sometimes I just really can’t bend my knees very well. Sometimes I might be able to get down in a really deep squat. I would say staying in that position hurts a little bit more
Emotional impact, n, %
Worry about body/weight
8, 40%
2, 40%
2, 40%
4, 40%
001-009_14yo_F: I feel that sometimes I was kind of worried a little bit about being made fun of, but recently I don’t think that I look as curvy as I did, but, when I weigh myself, it’s not getting any smaller, and I was kind of saddened by that
Feel bad about weight
7, 35%
1, 20%
1, 20%
5, 50%
001-001_17yo_F: It’s like something I worry about pretty regularly, like if my clothes are like hugging anything too tightly or like normal
Feel embarrassed
7, 35%
2, 40%
0, 0%
5, 50%
005-013_15yo_M: Shy. And embarrassed sometimes because I’m 205 pounds in high school in ninth grade
Feel frustrated
7, 35%
1, 20%
0, 0%
6, 60%
002-003_10yo_M: Angry […] Because people keep calling me fat
Lack confidence
6, 30%
0, 0%
0, 0%
6, 60%
002-006_16yo_M: I would say it is, then it isn’t. So like sometimes my self-esteem would just be really low and it would be- it’s an issue and sometimes I just don’t care. I’m just going about my day and I’m just killing it, and I’m feeling good. But when in fact when it’s really bad my self-esteem would get really low or I’ll get like really just – I didn’t have any confidence at all
Loss of motivation
4, 20%
0, 0%
0, 0%
4, 40%
001-005_16yo_F: The loss of motivation. I would say it was like more of a mental thing for the weight. Because it just makes me overthink about it a lot. And it just drains it to the point where I just don't do anything about it
Hard to control eatinga
–
–
–
–
Participant 001-013_7yo_F: Because sometimes I get too hungry, and I want to change and eat a lot of food
Social impact, n, %
Stay to myself/feel alone
2, 10%
1, 20%
0, 0%
1, 10%
002-006_16yo_M: Maybe I just don’t feel like talking to people sometimes because I’m like, “Man, I’m feeling bad” because of my weight or something like that. Maybe this is – I would just go through my day and not speak to nobody sometimes. But other than that, maybe where I’d just be a really talking person. I feel like that is the effect it would have, like different things
Don’t feel like can participate
6, 30%
0, 0%
1, 20%
5, 50%
002-006_16yo_M: Yup, I mean I can’t do different things because I weigh a different amount. Like in football I can’t do this position because I weigh this much
Feel treated differently
6, 30%
0, 0%
1, 20%
5, 50%
002-006_16yo_M: [I worry] a little bit because the way people see you is different when you’re heavier or when you’re bigger. The people see you in a different way. You get treated differently and even now that I’m working on doing more activities and everything. The difference is apparent
Feel not included in activities
6, 30%
0, 0%
1, 20%
5, 50%
002-006_16yo_M: But like friends and just going to school, having social interactions, when you’re bigger, people- it’s just different. It’s not the same if I were to go to school and I weighed 80 pounds less
Do not want to participate in activities
6, 30%
0, 0%
1, 20%
5, 50%
005-015_13yo_M: Maybe participating because I can put it in my thoughts as it like maybe if I get bigger or something, I may not want to participate anymore
005-017_17yo_M: …I don’t participate in [races] because I know that I’m the slowest and I know that I’ll lose
Italicized cells refer to concepts reported > 30%
aHard to control eating item was identified as an important concept to include in the PWQ
Physical symptoms: Three primary physical symptoms were endorsed across the 20 participants 6–17 years of age: body aches/pain (n = 15/20, 75%), tiring easily (n = 11/20, 55%), and breathing difficulties (n = 11/20, 55%). Low energy (n = 7/20, 35%) and skin rashes (n = 5/20, 25%) were endorsed by adolescents 12–17 years of age.
Physical impacts: Four primary physical impact concepts were endorsed by the 20 children and adolescents: difficulty running (n = 14/20, 70%), doing sports or activities (n = 8/20, 40%), walking longer distances (n = 6/20, 30%), and bending/kneeling (dressing, tying shoes) (n = 9/20, 45%). Standing (n = 8/20, 40%) was endorsed for children and adolescents 8–17 years of age, but not by children ages 6–7.
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Emotional/psychological impact: Two emotional/psychological impact concepts were endorsed by the 20 participants: worry/afraid/fear (n = 11/20, 55%) and feeling bad about weight (n = 7/20, 35%). Two additional concepts were experienced among children and adolescents 8–17 years old: feeling embarrassed (n = 6/20, 30%) and finding it hard to control eating (n = 7/20, 35%). Three concepts—feel frustrated (n = 6/20, 30%), lack of confidence (n = 6/20, 30%), and loss of motivation (n = 4/20, 20%)—were only reported by adolescents 12–17 years of age. The hard to control eating concept was introduced from an eating measure as it was considered important for the PWQ.
Social impacts: Three social impact concepts were endorsed among the 20 participants: feeling treated differently (n = 6/20, 30%), feeling not included in activities (n = 6/20, 30%), and feeling lonely (n = 2/20, 10%). Additionally, children and adolescents 8–17 years of age also experienced not wanting to participate in activities (n = 6/20, 30%). Only one concept, getting upset at how people treated me (n = 6/20, 30%), was only reported by adolescents 12–17 years of age.
Developing the Pediatric Weight Questionnaire
COIs identified from the CE interviews were mapped to item concepts of existing weight-specific and generic PRO measures. A decision was made to pursue a de novo measure given existing weight-specific PRO measures did not include all key COIs, were not validated across the 6–17 age range, and/or had lengthy recall periods. Consequently, these measures were not considered fit-for-purpose to capture the treatment benefit of new OMMs in children and adolescent ages 6–17 years.
Results of the CE interviews were used to develop the draft PRO measure. Age-specific concepts endorsed by a minimum of 30% of participants within each age group were considered for item development. Four domains were identified: physical symptoms, physical impacts, emotional impacts, and social impacts. Physical symptom evaluates participants’ perspectives of their physical symptoms experienced due to obesity over the past 7 days. Physical impact evaluates participants’ perspectives of their physical impacts experienced due to obesity over the past 7 days. Emotional impact evaluates participants’ perspectives of their emotional impacts experienced due to obesity over the past 7 days. Social impact evaluates participants’ perspectives of their social impacts experienced due to obesity over the past 7 days. The conceptual framework for concepts relevant to each age group are pictorially represented for the PWQ (Fig. 2).
An iterative process was used to review and discuss potential item concepts. Key item writing criteria were determined that focused primarily on creating items conveying a single unidimensional concept, were easy to understand (consisted of patient language), and measured concepts amenable to change within clinical trial timeframes. Concept frequencies were examined for each age group and reviewed against the item writing criteria. Language used by children and adolescents to describe the physical symptoms, and physical, emotional, and social impacts was retained for the draft items. A draft common set of items was developed that was relevant across age groups, as well as additional items that were age specific.
During the PRO development, revisions were made to the instructions, item content, recall period, and response option wording following consultations with clinical outcome assessment, obesity, pediatric, and translation experts. All pertinent feedback or recommendations were considered, and the PWQ items and conceptual frameworks were revised accordingly. Once the drafting of the items had been completed and reviewed, a translation expert conducted a translatability assessment. The translatability assessment reported that most items had a difficulty rating of 0 (no issues) to 1 (minor difficulty to translate). A Flesh–Kincaid readability assessment was conducted to assess grade level and reading ease. The results of the readability assessment showed that the majority of the 23 items, 18 (78%) were understood with ease at a 5th grade reading level or less. Two items (9%) were understood at 7th grade reading level or less (Item 5: My skin got irritated from rubbing; Item 20: I felt others did not include me in activities). Three items (13%) were understood at or above 9th grade level (Item 17: I lacked confidence; Item 19: I felt others treated me differently; Item 22: I did not feel like participating in activities).
The PWQ evaluates the symptoms and impacts of pediatric obesity using a frequency scale (never, rarely, sometimes, often, always) and 7-day recall period. The three age-specific versions included the same four domains—physical symptoms, physical impacts, emotional impacts, and social impacts. The PWQ for adolescents 12–17 years of age includes 23 items and is self-administered. The versions for children 6–7 and 8–11 years of age are a subset of age-appropriate items of the version for adolescents 12–17 years of age. The PWQ for children 8–11 years of age includes 17 items and is self-administered. The PWQ for children 6–7 years of age includes 13 items and is interviewer-administered (Supplementary material).
Cognitive Interviews
Thirty-four children and adolescents were enrolled in CIs, with the following participant breakdown by age group: nine (26.5%) participants aged 15–17, 11 (32.4%) participants aged 12–14, nine (26.5%) participants ages 8–11, and five (14.7%) participants ages 6–7. Most of the sample were female (n = 22/34, 64.7%), not Hispanic or Latino (n = 28/34, 82.4%), and white (n = 21/34, 61.8%) or Black or African American (n = 17/34, 50.0%). Most children lived with both parents (n = 24/34, 70.6%). Most participants (n = 32/34, 94.1%) had a BMI ≥ 95th percentile. Nearly all children reported that they had overweight/obesity (n = 32/34, 94.1%) and desired to weigh less (n = 31/34, 91.2%) and stated that their doctor was concerned about their weight (n = 30/34, 88.2%) and had recommended weight management/loss (n = 27/34, 79.4%). Eight (n = 8/34, 23.5%) participants reported asthma, six (n = 6/34, 17.6%, each) reported anxiety/depression or gastroesophageal reflux, and four reported sleep problems (n = 4/34, 11.8%) as a comorbidity.
Adolescents 12–17 Years of Age
The average time it took for adolescents 12–17 years of age to complete the 23-item PWQ was approximately 2 min. The 5-point response scale was well utilized across all 23 items. Item tracking of the PWQ measure for adolescents 12–17 years of age confirmed that no comprehension issues or problems were noted with the PWQ. Participants demonstrated a clear understanding of the item meaning and understanding of the PWQ instructions, response scale, and recall period.
Children 8–11 Years of Age
Nine children 8–11 years of age took an average time of 2 min to complete the 17-item PWQ. The 5-point response scale was less well utilized across all 17 items for PWQ responses; notably, more than half (n = 10/17, 59%) of the PWQ item responses showed floor effects (i.e., > 30% “never” responses). Item tracking of the PWQ measure for adolescents 8–11 years of age confirmed that no significant issues or problems with comprehension of the PWQ were noted.
Overall, most (n = 8/9, 89%) children 8–11 years of age liked the PWQ, describing the questions as good” and “pretty understandable.” All (n = 9/9, 100%) children 8–11 years of age understood the PWQ’s instructions and found them clear and easy to follow. All (n = 5/5, 100%) participants asked about the “because of my weight” attribution felt that it was helpful for understanding the items. Overall, participants considered the physical symptom, physical impact, and emotional impact domains to be the most relevant or important to their weight experience (n = 3/9, 33%, respectively).
Participants did not identify any missing items in the physical symptom, physical impact, emotional impact, and social impact domains in the PWQ. All participants (n = 9/9, 100%) were able to describe PWQ’s recall period of the “past seven days.” All (n = 9/9, 100%) children 8–11 years of age understood the PWQ’s response scale.
Caregivers of Children 8–11 Years of Age
All parents/caregivers (n = 8/8, 100%) mentioned that the PWQ instructions were easy for their child to understand when completing the PWQ. All (n = 7/7, 100%) parents/caregivers who were asked felt that their child understood the PWQ items. One (n = 1/7, 14.2%) parent/caregiver felt there may be some items that their child did not fully understand because they had not experienced some of the symptoms or impacts. Caregivers did not identify any missing items in the physical symptom, physical impact, emotional impact, and social impact domains in the PWQ. All (n = 8/8, 100%) parents/caregivers reported that the 7-day recall period was suitable for their child. Most parents/caregivers (n = 5/8, 63%) suggested rewording to “the past week” to make it more plain language for their child to understand. All parents/caregivers asked (n = 6/8, 75%) thought the response options were “good,” “fair,” and that they “liked that scale.”
Children 6–7 Years of Age
The time for the administration of the interview for children 6–7 years of age was approximately 5 min. In terms of overall impressions, participants thought the PWQ questions were understandable. None of the 13 items had ceiling effects (i.e., > 30% “always” responses). Floor effects > 30% were noted for several items in the physical impact and emotional impact domains and all items in the social impact domain.
All (n = 5/5, 100%) children 6–7 years of age understood the PWQ’s overall instructions and preferred the speedometer dial over the drinking glass response scale. Two (n = 2/5, 40%) of the participants reported they could answer the questions on their own without help from a parent/caregiver.
Participants did not identify any missing items in the physical symptom, physical impact, emotional impact, and social impact domains in the PWQ. The children 6–7 years of age tended to understand time more broadly and were unfamiliar with counting events or occurrences back in time. However, one (n = 1/5, 20%) participant, with help from their parent/caregiver, seemed to understand the recall period. The interviewer reviewed the response scale and the response options with the children 6–7 years of age and asked questions to assess their comprehension. Three (n = 3/5, 60%) participants understood the PWQ’s response scale using the speedometer figures as a guide. Two (n = 2/5, 40%) participants did not fully demonstrate their understanding of the response scale. One (n = 1/5, 20%) of these participants seemed to initially understand the response scale, but only responded to the items with “Never,” while the second (n = 1/5, 20%) participant needed assistance from their parent/caregiver, as they seemed confused as to how to answer at times.
Caregivers of Children 6–7 Years of Age
Overall parent/caregiver feedback was positive, with parents/caregivers noting that the PWQ questions were “pretty well understood” and “has good coverage.” Three (n = 3/5, 60%) parents/caregivers reported the social impact domain as being most important to their child. Four (n = 4/5, 80%) parents/caregivers felt their child understood the instructions, while one (n = 1/5, 20%) parent/caregiver recognized their child needed extra help to complete the questionnaire. Parents/caregivers were asked whether they observed that their child understood the 7-day recall period. Three (n = 3/5, 60%) parents/caregivers felt their child did not understand the recall period. Two (n = 2/5, 40%) parents/caregivers thought five response options were too many options.
Discussion
There is increased interest in and availability of OMMs, including those FDA-approved for use by adolescents aged 12 and above: Xenical (orlistat), Qsymia (phentermine/topiramate), Victoza (liraglutide), and Wegovy (semaglutide). Additionally, phentermine hydrochloride (Adipex) is FDA-approved for the treatment of obesity among adolescents aged 16 years and above for short-term use (e.g., 12 weeks or less). To date, there is no US-approved drug to treat obesity in children under 12 years of age. Understanding the patient perspective of the impact of OMM on how they feel or function can be enhanced through the use of a content valid measure like the PWQ and can be important for assessing treatment effectiveness.
The results from the TLR and CE interviews were used to develop the initial version of the PWQ for three age groups: 6–7, 8–11, and 12–17 years. Concept identification and CE work contributed critical information to assist in the understanding of the symptoms and impacts experienced by children and adolescents with obesity. The literature review explored concepts relevant to this patient population, resulting in a comprehensive item pool, conceptual disease model, and information for the development of CE interview guides.
The de novo instruments were designed to evaluate the symptoms and impacts of pediatric obesity across four domains: physical symptoms, and physical, emotional, and social impacts. Overall feedback on the PWQ found the measure to be relevant, clear, concise, and easy to understand. The domains and items in the physical symptom, physical impact, emotional impact, and social impact domains were found to be relevant to experience with obesity. Key components of the PWQ measures were clearly understood by adolescents 12–17 years of age and children 8–11 years of age, including the recall period, items, and response options, and was demonstrated throughout the interviews. No changes were made to the PWQ, demonstrating that the PWQ development process was rigorous and benefitted from following best practices.
Overall feedback on the PWQ for children 6–7 years of age identified that children generally understood the PWQ instructions and items content. Parents/caregivers confirmed the relevance of the item content to their child’s pediatric obesity experience. However, findings from the interviews identified potential issues with the 7-day recall period and the 5-point frequency response scale. In response to the findings from interviews with children 6–7 years of age, an instruction sheet has been developed to provide further interviewer instruction to be reviewed with the child specific to the PWQ response scale and recall period.
This study utilized a purposive sampling approach to identify children and adolescents with pediatric obesity across the designated age groups. This sampling approach is suitable for research designed to explore people’s experiences and attitudes [19]. The authors acknowledge certain limitations of this approach that may introduce selection bias, and the study results may not be fully representative of children and adolescents with pediatric obesity and may not be generalizable.
The sample size for this qualitative interview study was relatively small within the age subgroups (n = 10 for the 6–7 and 8–11 age groups for CE and CI interviews); however, the sample was demographically and geographically diverse sample of children and adolescents in the US; and saturation was reached for concepts included in the PWQ measure and for obtaining feedback on the measure. The study followed referenced recommendations for qualitative studies of this type. Measurement properties, construct validity, reliability, responsiveness to change, meaningful change of the PWQ, and health literacy assessment will be examined in a larger sample of children and adolescents participating in OMM treatment.
Conclusion
The results from the CI study support the content validity of the PWQ for use as a clinical trial endpoint among adolescents (12–17 years of age) and children (8–11 years of age) with obesity. Challenges were noted with the use of the 7-day recall period and 5-point response scale with children 6–7 years of age that may require further testing.
The PWQ provides a means of assessing the impact of obesity and benefit of treatment in children 6–17 years of age. Study findings support the initial content validity for the use of this new measure in pediatric obesity clinical trials. Further assessment of the measurement properties, including understanding of the factor structure, and the reliability, validity, responsiveness, and ability to detect change of the PWQ measures will need to be confirmed in future studies.
Acknowledgements
We would like to thank the following Evidera team members who helped with the recruitment, data collection, and analysis: Hoda Fotovvat, Mary-Claire Miller, Belle Pattara, and Yipin Han.
Medical Writing/Editorial Assistance
Editorial support was provided by Michael Grossi and Kawthar Nakayima.
Declarations
Conflicts of Interest
Anne M. Skalicky and Amy Clark are employees of Evidera, which received funding from Eli Lilly to conduct the study. Kristina Secnik Boye, Chisom Kanu, and Lisa M. Neff are employees and shareholders of Eli Lilly and Company.
Ethical Approval
The CE interview protocol was approved by a US ethical central institutional review board (IRB), in accordance with local law (IRB study number WCG IRB# 20225362), and the methodology was developed in line with the FDA and the ISPOR guidance on best practice for eliciting concepts for a new PRO instrument [9, 11]. This study was also performed in accordance with the ethical principles that have their origin in the Declaration of Helsinki and that are consistent with good clinical practice and applicable regulatory requirements. Written informed consent was obtained from all participants to publish data and verbatim responses. Consent was obtained from parents and assent obtained from child and adolescent participants to publish verbatim responses.
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