Is the ‘Child’s Voice’ from Diagnosis to End-of-Life

Trustworthy?

Pamela S. Hinds, PhD, RN, FAANDirector, Department of Nursing Research and

Quality OutcomesAssociate Director, Center for Translational

ResearchChildren’s National Health SystemProfessor, Department of PediatricsThe George Washington University

Children’s National Medical Center

Washington, D.C.

Disclosure

• I do not have any conflicts of interest to disclose.

• Funding sources for the data included in this presentation include the National Institute of Nursing Research , the National Cancer Institute, National Institute of Arthritis, Musculoskeletal and Skin Diseases, Association of Critical Care Nurses, the Oncology Nursing Foundation, and Alex’s Lemonade Foundation

Child Voice Considered as Part of the Family Voice

Most Excellent Co-Investigators

Judy Hicks, LCSWWayne Furman, MDLinda Oakes, MSNDeo Kumar Srivastava, PhD

Justin Baker, MDSheri Spunt, MDJohanna Menard, BSN

Chris Feudtner, MD

Nancy West, BSNBrent Powell, MDivShana Jacobs, MDScott Mauer, MDMichele Pritchard, PhD, RN

Jami Gattuso, MSNChris Feudtner, MDTessie October, MD

Most Excellent Co-Investigators

• Bryce Reeve, PhD• David Freyer, DO, MPH

• Lillian Sung, M.D., PhD

• Catriona Mowbray, PhD, RN

• Kathy Kelly, PhD, RN

• Steven Joffee, M.D.

• Janice Wythcombe, PhD, RN

• Jichuan Wang, PhD

• Changrong Yuan, PhD, RN

Overview of Comments

•Provide a description of the child’s voice–an overview of the evolution of child -reported outcomes in clinical investigations•describe the parallel science of parent and clinician reports of child treatment

Overview of Comments

•Describe research methods to solicit the child’s voice–Address using the child’s voice

•Describe future steps to document and apply the child’s voice in clinical investigations and care

The Child’s Voice -What is it? Why seek it? How do we seek it? When do we seek it? How do we use it?

The Child’s Voice

•What is ‘the child’s voice’?–Literature review–Reflection on completed pediatric studies

–Dialogue with pediatric and family researchers

–Review of federal policies

The Child’s Voice

• The direct reports of children using qualitative (e.g., words, colors, photos, depictions) , quantitative or both approaches to convey to others the quality of their illness experiences during a defined period of time.

The Child’s Voice

What it is not:

–Body and facial expressions not included: require interpretation

–Age not included: variation –Parent voice–Clinician voice

The Child’s Voice:

What is in the voice?

• Availability

• Intensity/ Degrees

• Meaning

The Child’s Voice

•What evidence do we have that the child’s voice is real?

The Child’s Voice: Is it Real?

• Symptoms– Pain - presence, location, intensity, change• Eland, 1975; 1978

• Awareness of Serious Illness– Not going to go home from the hospital– Not going to get better– Able to compare self to others

• Bluebond-Langer, 1978

Pediatric Self-Report Evolution of the Past Three Decades

Clinician Reports (CTCAE)

Use of

Adult Measures(HUI)

Proxy Reports (Play Performance

Scale)No Conceptual definitions for Pediatrics

Pediatric Definitions/Descriptions

Proxy Reports

Formatting changes to match Child Development (Rand)

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IQ Assessments

Can an Ill Child Report?

•Evidence for ability to report symptoms–>130 reports in past 32 years•Instrumentation studies•Theory testing•Intervention

Can an Ill Child Report?

•>500 children 5 to 18+ years of age

• Time of Diagnosis• Following the first course or 6 weeks of treatment

• 3 months into treatment• 6 months into treatment • End of treatment• Survivorship

Can an Ill Child Report?

•Age of child reports: QoL–3 and 4 year olds – quantitative - pain

–5 years of age and older - quantitative – quality of life•Up to 20% of 5 year olds uncomfortable with selecting a score

•Up to 15% of 6 year olds uncomfortable

•Up to 10% of 7 year olds uncomfortable

Can an Ill Child Report?

•Aspect of TIME in the description of ‘the Child’s Voice’–Ability to report changes over time•Past 7 days/Past 30 days

–Influence of maturation and of the moment

Can an Ill Child Report?

•Quality of Life•Symptoms•Satisfaction with Care•Preference for Decision Making

Evolution of Pediatric Self-Report Measures for the Past Four Decades

Clinician Reports (CTCAE)

Use of

Adult Measures(Hill)

Proxy Reports (Play Performance)

No Conceptual definitions for Pediatrics

Patient-Reported Outcomes as Primary

Pediatric Definitions/Descriptions

Government Role

Proxy Reports

Formatting changes to match Child Development (Rand)

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National Initiatives and PROsFood and Drug Administration Guidelines-1

• “Guidance for Industry—Patient-reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims” (December, 2009)

• Content Overview– Appropriate role of PRO in development of medical products

– Evaluation of PRO instruments (multiple criteria)– Clinical Trial Designs– Approaches to Data Analysis

• FDA accepts PRO endpoints in reviewing product applications

• Further highlights importance of PROs at national level

National Initiatives and PROsFood and Drug Administration Gludeliens-2

• Criteria for evaluating a PRO as a clinical trial endpoint

• Conceptual framework underlying instrument• Selection of concepts relevant to disease, population and treatment being evaluated

• Key properties of instrumentation– Reliability

• Test-retest• Internal consistency

– Validity • Content• Construct

– Clinical sensitivity (ability to detect change)

National Initiatives and PROsFood and Drug Administration Guidelines-3

Considerations for Children and Adolescents• In general, PRO review criteria similar to adults

• Additional age-related criteria– Age-related vocabulary– Language comprehension– Comprehension of health concept being measured– Duration of recall

• Proxy-reported outcome measures are discouraged– Observer reports should include only those events or behaviors that are directly observable

Evolution of Pediatric Self-Report Measures for the Past Four Decades

Clinician Reports (CTCAE)

Use of

Adult Measures(HUI)

Proxy Reports (Play Performance

Scale)No Conceptual definitions for Pediatrics

Patient-Reported Outcomes as Primary

Pediatric Definitions/Descriptions

Government Role

Proxy Reports

Formatting changes to match Child Development (Rand)

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Child Voice

Incorporated into research and care

Evolution: Practice Guidelines

Research Priority Recommendations

Measuring the Child’s Voice

• Evolution from disease-specific instruments to document ill child’s voice

• Comparability across clinical contexts– Core instruments i.e., Pediatric PROMIS measures

– Pediatric PROMIS use with children diagnosed with cancer, sickle cell disease, asthma, kidney disease

Soliciting Pediatric Reported Outcomes

•Future Next Steps

Can Ill Children Report Treatment Toxicity?

• Common Terminology Criteria for Adverse Events (CTCAE), v. 4.03, 2010– Mandatory report by clinicians (DHHS/NIH/NCI)

• 147 clinicians identified AEs that children 7 to 20 years of age could report (subjective experiences)

• RO1 to create and validate the Pediatric CTCAE

Cognitive Interviewing

Age Group PediatricForm A (x items)

PediatricForm B(x items)

PediatricForm C(x items)

PediatricForm D(x items)

Totals

7-8 years 6 children6 proxies

6 children6 proxies

6 children6 proxies

6 children6 proxies

48 English speaking children and proxies (24 dyads)

9-12 years 6 children6 proxies

6 children6 proxies

6 children6 proxies

6 children6 proxies

48 English speaking children and proxies (24 dyads)

Age Group Adult Form A(x items)

AdultForm B(x items)

AdultForm C(x items)

  Totals

13-15 years 8 children8 proxies

8 children8 proxies

8 children8 proxies

  48 English speaking youth and proxies (24 dyads)

16-20 years 8 children8 proxies

8 children8 proxies

8 children8 proxies

  48 English speaking youth and proxies (24 dyads)

Cognitive Interviewing - Spanish

Age Group PediatricForm A (x items)

PediatricForm B(x items)

PediatricForm C(x items)

PediatricForm D(x items)

Totals

7-13 years

5 children5 proxies

5 children5 proxies

5 children5 proxies

5 children5 proxies

40 Spanish speaking children and proxies (20 dyads)

Soliciting the Child’s Voice

•‘Please tell me what you are hoping for now.’

When Would a Child’s Voice be Silent?

• Able but not invited to speak• Able but not wanting to report

– Lack of confidence in self– Lack of confidence in those asking– Fear of outcomes– Protecting others

• Not Able to speak– Not understanding the method– Too ill

Soliciting the Child’s Voice

•Future Next Steps

Can Ill Children Report Treatment Toxicity?

• Common Terminology Criteria for Adverse Events (CTCAE), v. 4.03, 2010– Mandatory report by clinicians (DHHS/NIH/NCI)

• 147 clinicians identified AEs that children 7 to 20 years of age could report (subjective experiences)

• RO1 to create and validate the Pediatric CTCAE

How Do We Validate the Ill Child’s Voice?

Can a Very Ill Child Report?

•Evidence for ability to report symptoms–>130 reports in past 15 years; <5% at end of life

Ill Child Reports at End of Life: 2007

Ill Child Reports at End of Life: 2012

Timing of Soliciting the Very Ill Child’s Voice

• Before the child loses his/her voice

• Soliciting child preferences– Method flexibility– Method blending

• Ill child and parent agreement

– Measuring agreement over time• Challenging current approaches to ‘agreement’

– Estimation of change, concern – Effect of training to report– Other voices other than parent/guardian?

Can There be Only One Voice?

Child Voice/ Family Voice

ILL CHILD

THE PARENT

THE WELL SIBLING

Child and Parent Voices at End of Life

• Parent: – Doing as my child would prefer

• Child: – Thinking about my relationships with others

Single Unit of Analysis and Interpretation

• Child Reports

• Exclusionary• Informed• Collaborative• Delegation: Allowed to choose

• Parent Reports

• Exclusionary• Informed• Collaborative• Delegation: Allowed to choose

Combining Voices: Dyad, Triad and More

Family Level of Analysis

Family and Clinician Level of Analysis

Primary Points

• Ill children can self-report during illness and end-of-life care

• Parent s and clinicians add important voices to the ill child’s treatment and end-of-life experiences

• Single unit analyses and interpretations (ill child, parent, clinician) are informative but insufficient

The Child’s Voice -What is it? Why seek it? How do we seek it? When do we seek it? How do we use it?

Of What Benefit is the Child’s Voice in Research and Care?• Accurate symptom and quality of life assessments

• Impact of therapy

• Impact of care

• Patient and parent trust of clinicians and satisfaction with care– Relationship with treatment decision- making

Do We Trust the Child’s Voice?

• If we do not trust the child’s voice, we will not ask

• If we do not trust the child’s voice, we will not act on the voice

• If we ask, it is like a promise to the child to listen, to act in respect of the child’s voice