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Patient-Reported Outcomes: Introducion and Overview. Pythia Nieuwkerk, PhD Department of Medical Psychology Academic Medical Center, Amsterdam. Outline presentation. What are patient–reported outcomes (PROs)? How do PROs complement traditional clinical outcome measures?

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patient reported outcomes introducion and overview

Patient-Reported Outcomes:Introducion and Overview

Pythia Nieuwkerk, PhD

Department of Medical Psychology

Academic Medical Center, Amsterdam

outline presentation
Outline presentation
  • What are patient–reported outcomes (PROs)?
    • How do PROs complement traditional clinical outcome measures?
  • How can we measure PROs?
    • Type of measures
  • How are PROs used in clinical research?
    • Examples
what is a patient reported outcome
What is a Patient-Reported Outcome?
  • A PRO is any report of the status of a patient’s health condition that comes directly from the patient
    • without interpretation of the patient’s response by a clinician or anyone else.1
  • The term PRO addresses the source of the report, and not the concept or content of the report.2

1. FDA, 2009, 2. Patrick et al. 2007

what concepts do pro instruments measure
What concepts do PRO instruments measure?
  • Concepts measured by PROs differ in their degree of complexity:
    • From simple
      • eg, presence of a symptom
    • To more complex concepts
      • eg, ability to carry out activities of daily living
    • To even more complex concepts
      • eg, health-related quality of life
what is health related quality of life
What is health-related quality of life?

Health:

A state of complete physical, social,

and mental well-being, not merely

the absence of disease or infirmity

WHO, 1948

who based consensus of quality of life
WHO-based consensus of “Quality of Life”

Multi-dimensional

Physical

Functioning

Social

Functioning

Mental

Functioning

Affected by disease/treatment

Subjective

subjectivity and objectivity
Subjectivity and Objectivity

HRQoL is not subjective in the usual sense of the term

It can be measured accurately in an individual, and in a group

It is “subjective” in that it:

derives from the individual patient.

represents what is important to the individual patient.

wilson cleary model of health outcomes
WILSON-CLEARY MODEL OF HEALTH OUTCOMES

Characteristics of Individual

Biological and Physiological Variables

Symptoms

Functional Status

General Health Perceptions

Quality of Life

Characteristics of Environment

Wilson & Cleary JAMA (1995)

motivations for pro qol research
Motivations for PRO/QOL research
  • Changing the concept of treatment model
    • Switching from biomedical model to patient-centered model
    • Living longer and comfortable, especially for cancer patients, elderly population, etc.
motivations for pro qol research1
Motivations for PRO/QOL research
  • Some treatment effects are known only to the patient
    • eg, pain intensity and fatigue
  • Capturing different aspects of health outcomes extended beyond biomedical / clinical indicators
    • eg, symptoms and functioning, comprehensive assessment of impact of disease and treatment
when are pros most relevant
When are PROs most relevant
  • When no survival gain is expected (e.g. palliative treatments)
  • When no significant differences in survival are expected
  • Where survival is gained at the expense of major toxicity and treatment burden
type of health outcomes instrument
Type of health outcomes instrument

HEALTH PROFILE

Health states and impact on

daily functioning and well-being

Generic

measure

Disease-specific

measure

SF-36

WHOQOL-100

MOS-HIV

EORTC QLQ C30

generic instrument sf 36
Generic instrument- SF-36

Health profile: 8 domains

  • Physical functioning (10 items)
  • Role limitations/physical (4 items)
  • Role limitations/emotional (3 items)
  • Social functioning (2 items)
  • Emotional well-being (5 items)
  • Energy/fatigue (4 items)
  • Pain (2 items)
  • General health perceptions (5 items)
does your health now limit you in walking more than a mile
Does your health now limit you in walking more than a mile?

(If so, how much?)

No, not limited at all

Yes, limited a little

Yes, limited a lot

how much of the time during the past 4 weeks have you been happy
How much of the time during the past 4 weeks have you been happy?

None of the time

A little of the time

Some of the time

Most of the time

All of the time

slide20
Physical Health

Physical function

Role function-physical

Pain

General Health

SF-36 Physical Health

sf 36 mental health
Mental Health

Emotional Well-Being

Role function-emotional

Energy

Social function

SF-36 Mental Health
generic instrument whoqol 100
Generic instrument – WHOQOL-100

Health profile: 6 domains

  • Physical health (12 items)
  • Psychological health (20 items)
  • Level of independence (16 items)
  • Social relationship (12 items)
  • Environment (32 items)
  • Spirituality, religiousness & personal beliefs (4 items)
same domain different content
Same domain, different content

Social domain:

Social functioning versus social well being

  • Social functioning: limitations due to disease/treatment (SF36, EORTC-QLQ-C30)
    • More likely to respond to medical treatment
  • Social wellbeing: closeness with family and friends (FACT-G)
    • More likely to respond to psychosocial interventions
generic versus disease specific pros
Generic versus Disease specific PROs

Generic PRO

  • Intended for use across broad chronic disease populations
  • Allow comparisons across these groups
  • Disadvantage: may not permit adequate disease-specific focus
        • Disease caused symptoms
        • Treated related symptoms
relative disease burden generic pros allow for cross disease comparison of disease impact
Type-2 Diabetes

Depression

Congestive Heart Failure

Average Adult

Average Well Adult

Chronic Lung Disease

Asthma

30

40

50

34

36

55

SF-36’s Physical Component Summary (PCS)

RELATIVE DISEASE BURDEN: Generic PROs allow for cross-disease comparison of disease impact

Ware & Kosinski, 2001

generic versus disease specific pros1
Generic versus Disease specific PROs

Disease specific PRO

  • Focus on the impact of a particular condition on the patient’s functioning and experience
  • Responsive to disease-related changes
  • Cannot be used across populations with other diseases
wilson cleary model of health outcomes1
WILSON-CLEARY MODEL OF HEALTH OUTCOMES

Characteristics of Individual

Biological and Physiological Variables

Symptoms

Functional Status

General Health Perceptions

Quality of Life

Characteristics of Environment

Wilson & Cleary JAMA (1995)

combining pro measures
Combining PRO measures

Disease-specific and Generic PROs are complementary:

  • When both are included in a study, it is possible to capture:
    • Disease-specific concepts
    • Generic concepts, compare to norm: (relative) burden of illness / benefit of treatment
measuring pros hrql
Measuring PROs/HRQL
  • No standard scale, need to specify what we want to measure
    • What is your research question?
    • Who are your patients?
    • What do you anticipate what will happen?
  • Appropriateness of the measure to the question or issue of concern.
  • Correspondence between the content of the measure and goals of the study.
study goals
Study Goals
  • Characterizing the burden of disease and treatment
  • Characterizing treatment-specific outcomes for use in shared decision making
  • Predicting patient outcomes
  • Evaluating the effectiveness of interventions
slide33
The EORTC QLQ-C30

Physical functioning

Role functioning

Functional scales

Cognitive functioning

Emotional functioning

Social functioning

slide34
The EORTC QLQ-C30

Physical functioning

Role functioning

Functional scales

Cognitive functioning

Emotional functioning

Social functioning

Fatigue

Nausea andVomiting

Pain

Symptoms

Dyspnea

Insomnia

Appetiteloss

Constipation

Diarrhea

Financial difficulties

slide35
The EORTC QLQ-C30

Physical functioning

Role functioning

Functional scales

Cognitive functioning

Emotional functioning

Social functioning

Fatigue

Nausea and Vomiting

Pain

Symptoms

Dyspnea

Insomnia

Appetite loss

Constipation

Diarrhea

Financial difficulties

Global health

status scale

Global health status

Overall QoL

slide36
The EORTC QLQ-C30

Standardized score

Physical functioning

Role functioning

Range 0 - 100

Functional scales

Cognitive functioning

Emotional functioning

Social functioning

Fatigue

Nausea and Vomiting

Pain

Symptoms

Dyspnea

Insomnia

Appetite loss

Constipation

Diarrhea

Financial difficulties

Global health

status scale

Global health status

Overall QoL

slide37
The EORTC QLQ-C30

Standardized score

Physical functioning

Role functioning

Range 0 - 100

A higher score

indicates a higher

level of functioning

Functional scales

Cognitive functioning

Emotional functioning

Social functioning

Fatigue

Nausea and Vomiting

Pain

A higher score

indicates a higher

level of symptoms

Symptoms

Dyspnea

Insomnia

Appetite loss

Constipation

Diarrhea

Financial difficulties

A higher score

indicates a

higher

level of QoL

Global health

status scale

Global health status

Overall QoL

slide38
Profiles

100 = Good QOL

0 = Poor QOL

71

96

Physical functioning

63

93

Role functioning

83

94

Functional scales

Cognitive functioning

62

Emotional functioning

77

Social functioning

71

91

Global health status

64

71

O= No symptoms

100 = Many symptoms

14

38

Fatigue

2

10

Nausea and Vomiting

14

Pain

31

6

Dyspnea

28

Symptoms scales

Insomnia

34

14

4

20

Appetite loss

2

11

Constipation

4

Diarrhea

7

Healthy women (50-59 years) (Schwarz et al. Eur J Cancer, 2001)

Metastatic breast cancer baseline (Bottomley et al 2003)

Metastatic breast cancer at cycle 2 of doxorubicin/cyclophosphamide

study goals1
Study Goals
  • Characterizing the burden of disease and treatment
  • Characterizing treatment-specific outcomes for use in shared decision making
  • Predicting patient outcomes
  • Evaluating the effectiveness of interventions
changes in hrql from start to 18 months of antiretroviral therapy for hiv infection
Changes in HRQL from start to 18 months of antiretroviral therapy for HIV-infection

Cognitive function

Physical function

Health distress

Social function

General health

Role function

Mental health

Overall QoL

Vitality

Pain

study goals2
Study Goals
  • Ccharacterizing the burden of disease and treatment
  • Characterizing treatment-specific outcomes for use in shared decision making
  • Predicting patient outcomes
  • Evaluating the effectiveness of interventions
predicting survival in hiv infection
Predicting survival in HIV infection
  • 560 HIV infected patients starting HAART.
  • Completed the MOS HIV between 1998-2000.
  • All cause mortality established in March 2008.
  • 66 patients (11.8%) died during follow-up.
  • Physical Health Summary score (MOS HIV) significant predictor of survival, independent of other (clinical) parameters.

de Boer-van der Kolk: CID 2010

physical health summary score mos hiv
Physical Health summary score (MOS-HIV)

de Boer-van der Kolk: CID 2010

predicting outcomes
Predicting Outcomes
  • Baseline HRQL has been shown to be an independent predictor for overall survival
    • Overview of 36 trials that assessed baseline PROs and mortality (Gotay, JCO 26:1355, 2009)
  • PRO is a complex biomarker that can be highly predictive
    • Help signal those patients who are in need of medical attention
    • Can be an early warning useful for clinical decision making
    • Can be used as a stratification variable in research
study goals3
Study Goals
  • Characterizing the burden of disease and treatment
  • Characterizing treatment-specific outcomes for use in shared decision making
  • Predicting patient outcomes
  • Evaluating the effectiveness of interventions
vital study prevention of coronary heart disease

VITAL study Prevention of Coronary Heart Disease

Intervention to enhance adherence to statin therapy and life-style recommendations

r isk c ounseling
Risk counseling
  • Protocolized (nurse practitioner).
  • Identification individual risk factors.
  • Calculation Absolute Cardiovascular Risk (Framingham risk score)
  • Graphical presentation personal risk

 Risk Passport.

  • Life style counseling (stop smoking, weight reduction)
slide49
Inclusion Criteria

> 18 yrs

Indication for statin therapy- primary prevention- secondary prevention

Subjects

(n = 201, from outpatient clinics)

study endpoints
Study endpoints
  • Primary endpoints
    • LDL cholesterol levels
    • Adherence to statins
    • Anxiety
  • Secondary endpoint
    • Quality of Life (QOL)
slide51
PROs
  • Adherence to statins:

Please estimate the percentage of prescribed lipid lowering medication that you have taken during the last month

    • 9 point scale (<30% to 100%)
  • Anxiety (HADS)
  • Quality of Life (SF-12)
slide52
Routine care

Questionnaire

Weight, RR

LDL cholesterol

Questionnaire

Weight, RR

LDL cholesterol

Questionnaire

Weight, RR

LDL cholesterol

Questionnaire

Weight, RR

LLDL cholesterol

0 3 9 18(month)

Questionnaire

Weight, RR

LDL cholesterol

risk counseling

risk calculation

Questionnaire

Weight, RR

LDL cholesterol

risk counseling

risk calculation

Questionnaire

Weight, RR

LDL cholesterol

risk counseling

risk calculation

Questionnaire

Weight, RR

LLDL cholesterol

risk counseling

risk calculation

Extended care

result ldl cholesterol
Extended care

Routine care

Result: LDL cholesterol

Secondary prevention

Primary prevention

summary
Summary
  • PROs can be used to assess the impact of disease and treatment from the patient perspective.
  • Various PRO measures are available from which you can choose depending on your study goals.
  • PROs can complement traditional clinical outcome measures when applied in clinical research.
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