1 Hearing the Voices of the Children Measuring Pediatric Health-Related Quality of Life from the Perspective of the Children and Their Parents Quantifying the Qualitative James W. Varni, Ph.D. Texas A&M University

2 World Health Organization Definition “Health as a state of complete physical, mental, and social well- being, and not merely the absence of disease or infirmity.” - World Health Organization (1948)

3 Quality of Life Measurements In recent years, Quality of Life (QOL) measurement has emerged as an important health outcome in clinical trials, clinical practice improvement strategies, and healthcare services research and evaluation

4 Use of QOL assessments Utilizing QOL assessments in the clinical setting can facilitate physician-patient communication and identify “hidden morbidities,” or physical and psychosocial functioning deficits in individual patients that might otherwise go under-identified. Results of such assessment may indicate problem areas that need to be further explored and can aid a physician or other healthcare professional in designing treatment interventions and/or making the appropriate referrals

5 Pediatric QOL measurement Prediction of pediatric risk and future resource utilization may assist in proactive targeting of healthcare resources to minimize morbidity and associated costs. Serial QOL screenings integrated into standard clinical practice may increase the probability that pediatric patients can be managed with optimal QOL.

6 The PedsQL™ Pediatric Quality of Life Inventory ™ Pediatric Health-Related Quality of Life Measurement Experiences with 18,792 Healthy and Ill Children and Adolescents and Their Parents: Child Self-Report (N = 11,355 ) Parent Proxy-Report (N = 18,617)

7 The PedsQL™ Measures Child’s and Parent’s Perceptions of Child’s Health-Related Quality of Life Child Self-Report: Ages 5-18 Parent Proxy-Report: Ages 2-18

8 The PedsQL™ Instructions (Child Report) Standard Version In the past ONE month, how much of a problem has this been for you … Acute Version In the past 7 days, how much of a problem has this been for you …

9 The PedsQL™ Instructions (Parent Report) Standard Version In the past ONE month, how much of a problem has your child had with … Acute Version In the past 7 days, how much of a problem has your child had with …

10 Scaling (0-4) Never Almost Never Sometimes Often Almost Always Reverse Scored to Scale Reverse Scored to Scale 100 Representing Higher HRQOL 100 Representing Higher HRQOL

11 23 Items Approx. 5 minutes to complete 4 domains: Physical Functioning (8 items) Emotional Functioning (5 items) Social Functioning (5 items) School Functioning (5 items)

12 Sample Subscale Items Physical Functioning It is hard for me to walk more than one block Emotional Functioning I feel sad or blue Social Functioning I have trouble getting along with other kids School Functioning I have trouble keeping up with my schoolwork

13 ID# ____________ Date:________ PedsQL ™ Pediatric Quality of Life Inventory Version 4.0 CHILD REPORT (ages 8-12) DIRECTIONS On the following page is a list of things that might be a problem for you. Please tell us how much of a problem each one has been for you during the past ONE month by circling: 0 if it is never a problem 1 if it is almost never a problem 2 if it is sometimes a problem 3 if it is often a problem 4 if it is almost always a problem There are no right or wrong answers. If you do not understand a question, please ask for help. In the past ONE month, how much of a problem has this been for you … In the past ONE month, how much of a problem has this been for you … About My Feelings (problems with…)Ne AlAl S O Al 1. I feel afraid or scared I feel sad or blue I feel angry I have trouble sleeping I worry about what will happen to me01234 How I Get Along with Others (problems with…)Ne AlAl S O Al 1. I have trouble getting along with other kids Other kids do not want to be my friend Other kids tease me I cannot do things that other kids my age can do It is hard to keep up when I play with other kids01234 About School (problems with…)Ne AlAl S O Al 1. It is hard to pay attention in class I forget things I have trouble keeping up with my schoolwork I miss school because of not feeling well I miss school to go to the doctor or hospital01234 About My Health and Activities (problems with…)Ne AlAl S O Al 1. It is hard for me to walk more than one block It is hard for me to run It is hard for me to do sports activity or exercise It is hard for me to lift something heavy It is hard for me to take a bath or shower by myself It is hard for me to do chores around the house I hurt or ache I have low energy01234 PedsQL™ Sample

14 The PedsQL™ Generic Core Total Scale Score Two Summary Scores Physical Health Psychosocial Health Three domains within Psychosocial Health Emotional Functioning Social Functioning School Functioning Total PhysicalPsychosocial EmotionalSocialSchool

15 PedsQL™ Disease and Condition Specific Modules Asthma Arthritis/Rheumatology Cancer Diabetes Cardiac Multidimensional Fatigue Scale Cerebral Palsy, Brain Tumor, End-Stage Renal Disease, Solid Organ Transplant and other modules under development

16 Internal Consistency Reliability: Healthy Children (N= 16,417) Reliability is strong for all age groups; 0.70 is standard for group comparisons * Includes only 5-18 data, as School Functioning scale for 2-4 has only 3 items Scale Total Child Self-Report TotalNA PhysicalNA PsychosocialNA Parent Proxy-Report Total * Physical Psychosocial *

17 Internal Consistency Reliability: Children with Chronic Health Conditions (N = 1,732) Scale Total Child Self-Report TotalNA PhysicalNA PsychosocialNA Parent Proxy-Report Total * Physical Psychosocial * Reliability is strong for all age groups; 0.70 is standard for group comparisons * Includes only 5-18 data, as School Functioning scale for 2-4 has only 3 items

18 Healthy Children and Children with Chronic Health Conditions Chronic < healthy (p<.001 on all scales) Child Self-Report

19 Healthy Children and Children with Chronic Health Conditions Parent Proxy-Report Chronic < healthy (p<.001 on all scales)

20 Comparisons Across Chronic Health Conditions- Child Report

21 Comparisons Across Chronic Health Conditions- Parent Report

22 Sensitivity of the PedsQL™: ESRD Treatment Type Child Self-Report p<.01 p<.05 Parent Proxy-Report p<.01

23 Sensitivity of the PedsQL™: Cancer Treatment Type Child Self-Report a<c** a<c*** Parent Proxy-Report a<c* * p<.05, **p<.01, *** p<.001 a<c**

24 Sensitivity of the PedsQL™: Rheumatic Diagnostic Groups Child Self-Report *p<.05, **p<.01,*** p<.001; FMS = Fibromyalgia, DMS = Dermatomyositis, JRA = Juvenile Rheumatoid Arthritis, SLE = Systemic Lupus Erythematosus a<b,c,d*** Parent Proxy-Report a<b** a<c,d*** a<b** a<c,d*** a<b* a<c,d***

25 Sensitivity of the PedsQL™: Diabetes Type Child Self-ReportParent Proxy-Report p<.05 p<.01

26 Sensitivity of the PedsQL™: Weight Status Child Self-ReportParent Proxy-Report P<.001 for Total & Physical Note: group differences do not include severely obese

27 Sensitivity of the PedsQL™: Cardiac Severity Child Self-Report Parent Proxy-Report 1> 2,4 * 1> 2,3 * 1> 4*** 2> 4* * p<.05, *** p<.001 1> 2,3 * 1> 4*** 2,3> 4* 1> 4 ***

28 Sensitivity of the PedsQL™: Cerebral Palsy Diagnostic Groups Child Self-Report Parent Proxy-Report c<a,b*** c<a*** c<b** c<a** c<b*** **p<.01, *** p<.001 c<a,b*** c<a,b*

29 Responsiveness of the PedsQL™: Rheumatology Sample Child Self-ReportParent Proxy-Report Time 1<Time 2**, Time 1<Time 3*** for all scalesTime 1<Time 2, Time 3** for all scales

30 Parent-Child Concordance SampleTotalPhysicalPsychosocial Healthy Chronic Asthma Cancer Cardiac Cerebral Palsy Diabetes ESRD Rheumatology (NS) All correlations are significant (p<.001) unless noted (NS = not significant)

31 Healthcare Costs by Chronicity Lowest Quintile (N = 51) 2 nd Quintile (N = 45) 3 rd Quintile (N = 48) 4 th /5 th Quintile (N = 99) Chronic Condition YesNoYesNoYesNoYesNo 6 months 37.43* months 59.16* months 61.74* % of cost * = high risk group; accounts for 62% of healthcare costs after 24 months, even though it represents only 8.7% (n = 21) of the total sample (n = 241) Percent of cost at each follow-up period, by PedsQL™ Physical Function quintile and chronic condition status

32 Parent proxy PedsQL & Costs Parent proxy-reported PedsQL™ scores prospectively accounted for significant variance in healthcare costs at 6, 12, and 24 months. Adjusted regression models that included both PedsQL™ scores and chronic health condition status accounted for 10%, 14%, and 21% of the variance in healthcare costs at 6, 12, and 24 months. Parent proxy-reported PedsQL™ scores and chronic health condition status together defined a ‘high risk’ group, constituting 8.7% of the sample and accounting for 37%, 59%, and 62% of healthcare costs at 6, 12, and 24 months. The high risk group’s per member per month healthcare costs were, on average, 12 times that of other enrollees’ at 24 months

33 Costs for High and Low-Risk Samples Time PeriodTotalPer MemberPer Member Per Month High RiskNot High Risk High RiskNot High Risk High RiskNot High Risk 6 months $54,493$91,484$2,595$396$432$66 12 months $203,875$168,022$9,708$727$809$61 24 months $363,822$330,846$17,325$1,432$722$60 Total costs, per member costs, and per member per month costs for high risk* (N = 21) and not high-risk (N = 231) samples * High risk defined as parent reported chronic condition and scoring in lowest PedsQL™ quintile

34 Parent proxy Peds QL ™ & Costs Parent proxy-reported PedsQL™ scores can be used to prospectively predict healthcare costs. When combined with chronic health condition status, parent proxy-reported PedsQL™ scores can identify an at risk group of children as candidates for proactive care coordination and cost containment.

35 Improved Quality of Life A standardized assessment of patients’ QOL is necessary… when combined with physician knowledge of treatment options and referral sources patient functioning and quality of life can improve.

36 Future Directions Integrating Generic and Condition/Disease Specific Instruments Item Response Theory Facilitated Items Computerized Adaptive Testing

37 The End Please proceed to the post test Download the post test Complete the post test Return the post test to Dr. Oliver 407I TAMUII

38 Post Test Question One 1. Child reported quality of life is lowest in which of the following diseases? 1. Diabetes 2. CP 3. Cancer 4. Asthma

39 Post Test Question Two 2. Parent-child concordance in PedsQL was significant in all diseases except: 1. Diabetes 2. CP 3. Cancer 4. Asthma

40 Post Test Question Three 3. Children with chronic health conditions self reported which of the following QOL functional subscale variables lowest? 1. Physical 2. Psychosocial

41 Thank you