Measuring Health-Related Quality of Life in Children and Adolescents

1. Measuring Health-Related Quality of Life in Children and Adolescents Anne Klassen Veronica Schiariti Jill Zwicker

2. Workshop Presenters Anne Klassen, Associate Professor of Pediatrics at McMaster University. Interests include developing, validating and using HRQOL instruments in clinical and population-based studies. Veronica Schiariti, Paediatrician & PhD student working as a Research Associate in the CYDiS research unit at BC Children’s Hospital. Interests are health services research and measuring HRQOL of high risk populations of infants and children. Jill Zwicker, OT & PhD student in Rehabilitation Sciences at UBC & CIHR Quality of Life Strategic Training Fellow. Her interests include measuring HRQOL of children with developmental coordination disorder

3. Today’s Workshop 1) Issues specific to understanding and measuring HRQOL in children and adolescents 2) HRQOL of preschoolers admitted at birth in 1996–1997 to a tertiary NICU in BC 3) Systematic review of HRQOL studies of children, adolescents and young adults born preterm and/or at very low birth weight

4. Measuring HRQOL in Children and Adolescents Anne Klassen, DPhil Associate Professor, Pediatrics McMaster University May 2008

5. Child Welfare Officials to Make Sure Boy They Seized Gets Chemo – CBCnews.ca, Friday May 9, 2008 Child welfare officials have taken temporary custody of an 11-year-old Ontario boy to ensure he undergoes chemotherapy after his father decided to take him off treatment for his aggressive form of leukemia. A father who cannot be identified says his son is being treated 'like a prisoner' at the hospital where he is being treated for leukemia. His father … told CBC News on Friday that the boy didn't want to continue with the treatments. "I think about the first time around, what it did to him and how it almost killed him, and when he told me he doesn't want it anymore. He doesn't want to die this way. He would rather die at home in a peaceful, comfortable way.“ The dad, who lives in Hamilton, was briefly shackled by security when he arrived at McMaster Children's Hospital on Thursday with his son for what he believed was a routine appointment. Local Children's Aid Society officials then took custody of the boy due to the father's refusal to admit the son for another round of chemotherapy. "He had a 50 per cent chance of survival if the treatment was carried out. If the treatment was not carried out, then in fact his chance of survival would be not good. In fact they estimated it would be fatal in six months.“ But the father said doctors told him the boy had a 20 per cent chance of making it through his chemotherapy treatments, then a 50 per cent chance after that, once he undergoes full body radiation and a bone marrow transplant.

6. … quality > quantity “I watched a friend be tortured by the cancer treatment. It was the treatment, not the cancer, that was the torture. If the issue comes up for me, I'll refuse treatment. There are worse things than death.”

7. … quantity > quality “I lost a cousin at age 28 to leukemia but I also have a friend who survived a year of intensive treatments. Yes, it almost killed him but he is doing OK now and has travelled the world since. He will never be 100% but he is very happy to be alive and able to carry on.”

8. As people today survive what used to be primarily fatal diseases, and learn to live with complex chronic conditions, the impact of treatment and disease on QOL has become increasingly important to clinicians, researchers and patients

9. Background • Measurement of QOL emerged in the 1970s when the focus of health care evaluation moved from traditional clinical outcomes (i.e. mortality, morbidity) to the measurement of broader outcomes, such as function (i.e. ability to perform daily activities of life). • Traditional clinical outcomes were seen to not capture the whole range of ways in which a patient is affected by disease and/or treatment • The inclusion of more holistic outcomes, such as quality-of-life (QOL) began to gain increasing interest • QOL is conceptualized as a broad assessment of well-being across various domains • HRQOL is considered to be a subdomain of QOL

10. Moons et al 2006 Adult QOL publications

11. Pediatric QOL publications Klassen et al 2007

12. Definitional and conceptual issues • Despite increasing interest in QOL, there is no consensus on the definition and/or conceptualization of QOL • A wide spectrum of QOL definitions and conceptualizations exist in the literature • People use the term QOL to mean many different things – this can make comparison of findings difficult or impossible • QOL is often used as a generic label for an assortment of physical functioning and psychosocial variables • There is often no distinction made between measures of QOL and measures of HRQOL

13. WHO definition of QOL (1994) “an individual’s perception of his/her position in life in the context of the culture and value systems in which he/she lives, and in relation to his/her goals, expectations, standards and concerns. It is a broad-ranging concept, incorporating in a complex way the person’s physical health, psychological state, level of independence, social relationships, and their relationship to salient features of their environment.”

14. QOL: The degree to which a person enjoys the important possibilities of his or her life -- Ctr for Health Promotion, U of Toronto Being: who one is • Physical Being: physical health; personal hygiene; nutrition; exercise; grooming/clothing; physical appearance • Psychological Being: psychological health & adjustment; cognitions; feelings; self-esteem/self-concept/self-control • Spiritual Being: personal values ; personal standards of conduct; spiritual beliefs Belonging: connections with one's environments • Physical Belonging: home; workplace/school; neighbourhood; community • Social Belonging: intimate others’; family; friends ; co-workers; neighbourhood and community • Community Belonging: adequate income; health & social services; employment; educational programs; etc Becoming: achieving personal goals, hopes, and aspirations • Practical Becoming: domestic activities; paid work; school or volunteer activities; seeing to health or social needs. • Leisure Becoming: activities that promote relaxation and stress reduction • Growth Becoming: activities that promote the maintenance or improvement of knowledge/skills; adapting to change

15. What about pediatric QOL? Many pediatric QOL measures have now been developed and several reviews have been published.

17. Review of Child QOL measures • Reviewed QOL measures for children aged 0 to 12 yr • Looked at conceptual frameworks & definitions • 14 generic and 25 condition-specific identified • Reliability and validity tested for most • Only 3 based on a conceptual model/theory

19. Health-related QOL • Health is consistently included as an important aspect of QOL • HRQOL is seen as a subset of the overall concept of QOL and includes those parts of QOL that can directly relate to an individual’s health. • HRQOL, Health Status, Functional Status are terms that are often used interchangeably but they measure different things

21. HRQOL Based on the view that health is multidimensional and that it is subjective and therefore should be evaluated by asking a person directly

22. HRQOL / Health Status • HRQOL and HS were distinct constructs in a meta-analysis of 12 chronic disease studies (Smith 1999) • When rating HRQOL patients gave greater emphasis to mental health • When rating HS patients gave greater emphasis to physical health • QOL was only modestly correlated with HS in study of 203 adolescents with CP (Rosenbaum 2008)

23. Does a healthy life = a high QOL? • People with significant HS and functional status problems do not necessarily have low QOL score • “Disability paradox” -- physically disabled persons unexpectedly experience a good QOL even though most external observers may assume that these people live an undesirable life (Albrecht & Devlieger, 1999)

24. What measures are available to measure HRQOL in children?

26. Review findings • 30 generic & 64 disease/condition-specific identified • Number & name of HRQOL domains varies substantially • Lower age limit for child-report measures = 5-6 yrs • Many instruments meet accepted psychometric standards

27. Generic vs. disease-specific • Generic questionnaires allow direct comparisons across disease groups or between sick and healthy groups • Disease or condition-specific instruments address problems specific to only one illness or disease. • Such instruments, when developed through in-depth qualitative interviews, can help to identify QOL issues of importance to them.

31. 16 generic HRQOL measures identified

33. Research example HRQOL of Children with ADHD

35. Background • ADHD is a common psychiatric disorder, affecting approximately 3-5% of children • The aim of treatment is to decrease symptoms, enhance functionality and improve well-being for the child and his/her close contacts • Measurement of treatment response is often limited to measuring symptoms using behavior rating scales

36. Methods • Subjects: all referrals to the ADHD clinic at BC Children’s Hospital, Vancouver between Nov ’01 and Oct ’02 • Data collection: CHQ included in package of symptom rating scales sent prior to outpatient appointment • Clinical data: psychiatric diagnoses extracted from the hospital charts

37. Measures • Child (CSI), Adolescent (ASI) & Youth (YSI) Symptom Inventory • Measure of symptoms for a wide range of DSM-IV diagnoses including ADHD subtypes • Conners Rating Scale Hyperactivity Index • Measure of overall psychopathology for children presenting with ADHD • Child Health Questionnaire (50-item parent form) • Multidimensional HRQOL measure for children 5-18

38. Interpretation of low and high scores for each CHQPF-50 concept

39. Response Rate 335 mailed 79 children ineligible 165 returned 64.5% response rate

40. Child characteristics

41. Mean CHQ-PF50 item and domain scores comparing Australian and USA population norms with ADHD children

42. Effect sizes comparing ADHD sample with USA population norms

43. Comparisons with other ADHD samples and population norms

44. Proxy-reported HRQOL • Patient self-report is considered the gold standard in HRQOL assessment • Riley (2004) reviewed research from a range of areas: • studies of cognitive abilities • cognitive interviewing studies of children’s ability to respond to questionnaires and influences on their responses • psychometric studies of child-report questionnaires • longitudinal research on the value of child report • Children as young as 6 years are able to understand questions about their QOL and to give valid and reliable answers

45. Complementary perspectives • Self-report is not always feasible or possible for children • may be too ill, unwilling, lacking the necessary language skills, attention, or cognitive abilities • Proxy may need to report HRQOL scores for child • Even when children are able to participate, proxy ratings may provide a different perspective that is complementary • What is important is what each reporter contributes to the overall understanding of child HRQOL rather than who is more accurate

46. Eiser & Morse review of 14 studies • Agreement was generally good for more observable domains of HRQOL (e.g., physical function, behavior) • Agreement generally poor for less observable domains of HRQOL (e.g., social function, emotional function)

47. Research example Agreement between parent- and self-report of HRQOL in children with ADHD

49. 58 children diagnosed with ADHD and their parents participated • CHQ permitted comparisons on 8 HRQOL domains and one single item

50. Sample characteristics