Chapter 8 Quality of Life Assessment

1. Chapter 8Quality of Life Assessment

2. News • 乳癌的標靶治療用藥有了新突破！國內台大、榮總、三總、馬偕、奇美、高醫、彰基等醫學中心現正著手進行乳癌新藥Lapatinib的臨床試驗，對於國內每年新增近五百人晚期患者來說，可說是最後一線希望。 • 只要是HER-2成陽性反應，且屬於轉移性第四期，嘗試過各種藥物都無效，就可能參加這項人體試驗。

3. Why Are We Interested in the Quality of Life? The United States Food and Drug Administration has stated that efficacy with respect to overall survival and/or improvements in QOL might provide the basis for drug approval “Live longer – feel better” Shaughnessy JA, Wittes RE, Burke G et al. Commentary concerning demonstration of safety and efficacy of investigational anticancer agents in clinical trials. J Clin Oncol 1991; 9:2225-32.

4. Outline for Quality of Life • General background • Data collection considerations

5. Your Quality of Life How are you feeling this afternoon? Mood Happy Miserable

6. What is Quality of Life? WHO: “Health is not only the absence of infirmity and disease, but also a state of physical, mental and social well being Multiple domains include: physical, cognitive, emotional and social functioning, pain, sexual functioning, health perceptions, and symptoms such as nausea and fatigue Fundamental principle: quality of life is assessed by the patient

7. Quality of Life (1) • Definition depends on context Cancer vs. MI vs. hypertension • Some instruments are disease specific • Others are "general health status" instruments • POMS = Profile of Mood • SIP = Sickness Impact Profile • Difficulties with concept • No agreement on definitions • Lack of standardized measures

8. Quality of Life (2) • One definition (Levine & Croog) Two Components - Functioning 1. Social (Major component) - Get along with family & friends | sense of worth(家庭價值) 2. Physical - Perform daily life activities 3. Emotional - Stability and self control 4. Intellectual - Decision making ability - Perceptions 1. Life Satisfaction - Sense of well being(幸福感覺) 2. Health Status - Compared to others

9. Factors Which Influence Quality of Life 1. Intervention 2. Disease Process 3. "Labeling" Need a control group - Diagnosis brings on change 4. Concomitant Care 5. Non-related life events (e.g. death in family)

10. Rationale in Clinical Trials • Quality of life may assess effect of intervention • primary response (treatment less toxic?) • side effects (treatment toxic?) • economic aspects (low risk/cost treatment but benefit high?)

11. How To Assess Quality of Life • Determine your QoL Objective • Choose an instrument • Reliable, valid, responsive, feasible • Global measures, disease-specific measures, symptom checklists • Select your assessment time points and administration format • Develop an analysis plan

12. Data Collection Considerations (1) • Mode • Self-administered • glasses, reading skills, fine-motor skills • Personal interview • training/background of interviewer • sensitivity to gender/ethnicity/age • hearing impairment

13. Data Collection Considerations (2) • Content • instrument validity, sensitivity & specificity • sensitivity of questions • frame of reference for answers (cognitive skills, privacy, cultural background) • Source(s) • participant, family or support network, health care providers

14. Off the Shelf Instruments • Off-the-shelf instruments • Designed to distinguish sickness from wellness • May not be sensitive to particular aspect of a given trial • May not be validated or "normed" in population being tested • May ask ridiculous questions for trial pop. • May take hours to complete • May impact negatively on compliance

15. Off-the-Shelf Instruments(Example-NOTT) • Design • Advanced Chronic Obstructive Pulmonary Disease • 24 vs. 12 hours of O2 • Quality of life 10 outcome (No norms in this pop!) • Quality of Life Results  • Patients were sick • Patients got worse • No treatment difference BUT • Mortality ratio was 2/1 (p<.01)

16. More Perspectives • "Tailor Made" Instruments • Can be Quick and simple • Standardized but targeted to disease • Must be Validated for trial population • Select subsets of off-the-shelf instruments

17. More Perspectives • “Home-made" Instruments • Often designed by a graduate student • Often too long • Often not validated or field tested on your patient population

18. Quality of Life Instruments • Can be simple and short • Classic examples for • Cancer • Congestive Heart Failure

22. Quality of Life Analysis • Analytic methods likely to be based on a scoring system • Methods often rank based • Challenging to design/ compute sample size

23. Statistical Consideration • Multi-trait scaling and other construct examination • Reliability (internal consistency and test-retest reliability) • Correlation analyses with other instruments • Clinical validity (known group comparisons (sensitivity)) • Responsiveness to change over time

24. Multi-trait Scaling (1) • Multi-trait scaling analysis will be employed to examine whether the individual items in the instrument can be aggregated into hypothesised multi-item scales • Evidence of item convergent validity is defined as correlation of 0.40 or greater between an item and its own scale (corrected for overlap).

25. Multi-trait Scaling (2) • Evidence of item discriminant validity will be based on a comparison of correlation of an item with its own scale and with other scales • Scaling success for any item is defined as an item correlated significantly higher with its own scale (corrected for overlap) than with another scale

26. Reliability • The internal consistency of the multi-item questionnaire scales will be assessed by Cronbach’s alpha coefficient • A magnitude of >0.70 is considered acceptable for group comparisons • The test-retest reliability of scales will be assessed using intraclass correlations between the second and retest assessments in the patients

27. Clinical Validity • Known-group comparison will be used to assess whether the questionnaire scores can discriminate between subgroups of patients of clinical status

28. Responsiveness Over Time • Mixed effects regression model or generalized estimating equations (GEE) will be used to test for the significance of changes in QL scores before and after treatment

29. Sample Size Requirements • The sample size is based on the recommendation of Tabachnik and Fidell that for multivariate analysis techniques to obtain reliable estimates the number of observations should be 10 times the number of variables in the model

30. EORTC QLQ-C30 (version 3) 台灣中文版 (1) • 您從事一些費力的活動，如攜帶重物或手提箱，是否有困難？完全沒有,有一點,相當多,非常多 • 您從事長距離步行，是否有困難？完全沒有,有一點,相當多,非常多 • 您在戶外從事短距離步行，是否有困難？完全沒有,有一點,相當多,非常多 • 您在白天是否需要待在床上或椅子上？完全沒有,有一點,相當多,非常多 • 您進食、穿衣、洗澡或上廁所需要別人幫助嗎？完全沒有,有一點,相當多,非常多

31. EORTC QLQ-C30 (version 3) 台灣中文版 (2) 在過去一星期內（過去七天內）： 6. 您在從事工作或日常生活上是否受到限制？完全沒有,有一點,相當多,非常多 7. 您在從事嗜好或休閒生活上是否受到限制？完全沒有,有一點,相當多,非常多 8. 您呼吸會喘嗎？完全沒有,有一點,相當多,非常多 9. 您曾感到頭痛嗎？完全沒有,有一點,相當多,非常多 10. 您需要休息嗎？完全沒有,有一點,相當多,非常多

32. EORTC QLQ-C30 (version 3) 台灣中文版 (3) 11. 您曾難以入睡嗎？完全沒有,有一點,相當多,非常多 12. 您曾感到虛弱嗎？完全沒有,有一點,相當多,非常多 13. 您曾缺乏食慾嗎？完全沒有,有一點,相當多,非常多 14. 您曾感到噁心嗎？完全沒有,有一點,相當多,非常多 15. 您曾嘔吐嗎？完全沒有,有一點,相當多,非常多 16. 您曾便秘嗎？完全沒有,有一點,相當多,非常多 17. 您曾腹瀉嗎？完全沒有,有一點,相當多,非常多 18. 您疲倦嗎？完全沒有,有一點,相當多,非常多 19. 疼痛干擾您日常生活嗎？完全沒有,有一點,相當多,非 常多

33. EORTC QLQ-C30 (version 3) 台灣中文版 (4) 20. 您曾否難將注意力集中在一些事情上，如看報紙或看 電視？完全沒有,有一點,相當多,非常多 21. 您覺得緊張嗎？完全沒有,有一點,相當多,非常多 22. 您感到憂慮嗎？完全沒有,有一點,相當多,非常多 23. 您覺得容易發怒嗎？完全沒有,有一點,相當多,非常多 24. 您覺得情緒低落嗎？完全沒有,有一點,相當多,非常多 25. 您曾感到記憶困難嗎？完全沒有,有一點,相當多,非常 多 26. 您的身體狀況或醫療過程是否曾干擾您的家庭生活？完全沒有, 有一點,相當多,非常多 27. 您的身體狀況或醫療過程是否曾干擾您的社交生活？完全沒有, 有一點,相當多,非常多 28. 您的身體狀況或醫療過程是否曾造成您財務上的困難？完全沒有, 有一點,相當多,非常多

34. EORTC QLQ-C30 (version 3) 台灣中文版 (5) 29. 您如何評定過去一星期內（過去七天內 ）您整體的健康？1(非常差)234567( 極好)  30. 您如何評定過去一星期內（過去七天內 ）您整體的生活品質？1(非常差)2345 67(極好) 

35. FACT-P (第四版) (1)

36. FACT-P (第四版) (2)

37. FACT-P (第四版) (3)

38. FACT-P (第四版) (4)

39. FACT-P (第四版) (5)