Towards Person- Centred Outcome Measurement in Parkinson s Disease Peter Hagell, RN PhD School of Health and Society Kristianstad University, Kristianstad, Sweden
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Requirements To be meaningful and meet the purpose of their use in clinical trials, rating scales need to represent rigorous measures of the variables they intend to quantify the variables that therapies are intended to target the variables that are important to people with PD
the main problem was that we couldn t interpret the outcome measures from research studies (often scales of some sort). Although sufficient to secure drug marketing licenses, they had little practical meaning. Iain Chalmers National Institute of Clinical Exellence (NICE) R&D Advisory Committee BMJ 2007; 335: 400
A broader base of expercse, derived from a variety of realms and perspeccves, can lead to much beier research designs, tools and outcomes
Illustra(ng the Problem A clinical trial of an adjunct (L- dopa add- on) drug for Parkinson s disease with wearing- off Primary end- points: AcCviCes in Daily Living (ADL): UPDRS II Quality of Life (QoL): PDQ- 39
People with PD Participants (Possible score range, 0-52; 0=better) Pre-Rx: 24.7±12.3 Pre-Rx: 23.5±10 Post-Rx: -0.7±2.7 Post-Rx: -2.3±3.7 P<0.0001 good efficacy in terms of ADL
However What does a mean difference in scores of 1.6 on the UPDRS II mean? We do not know
What was measured? We do not know
Furthermore Results (ADL):? UPDRS II: P<0.0001 (favouring L- Dopa+) Schwab & England ADL scale: n.s. (no effect) PDQ- 39, ADL scale: n.s. (no effect) good efficacy in terms of ADL
What outcomes are important to assess in PD from an end- user perspeccve?
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Developing conceptual maps of prioritized areas of outcomes and determining their relative importance for outcome measurement in clinical PD trials from the perspectives of people with PD and health care professionals
Qualitative Concept Mapping Patients Clinicians Researchers Quantitative A concrete example of what is most important to assess when treacng PD, regardless of whether there is such a treatment available or not, is freezing medrörelser i armarna smärta överrörlighet kramper svårt att andas benskörhet talproblem (sluddrigare tal, svagare röst) förmåga att uppfatta vad folk säger värk viktförändring aspirationsbesvär stelhet ökad salivering (dregla) vända sig i sängen nedstämdhet lukt frustration inre darrningar psykiskt välbefinnande skrivsvårigheter motorisk långsamhet mardrömmar yrsel förstoppning somna mitt på dagen reaktionsförmåga oro håglöshet skamkänsla omdöme insikt ångest aggressivitet trötthet handlingsförmåga simultanförmåga organisera gångförmåga fall svängningar (motoriskt / mentalt off) svårt att äta (svälja och tugga ev finmotorik) stickningar (i fingrarna) rörlighet balans mimikfattigdom muntorrhet sväljförmåga tvångsmässiga upprepade handlingar lågt blodtryck kroppshållning muskelsvaghet uppresning inkontinens skakningar blodtrycksfall finmotorik erektion smak svettningar sömnproblem svindlingskänsla trängningar impotens känslomässig balans social förmåga depression matt/ orkeslös/fatigue planera distanslöshet stresskänslighet initiativförmåga företagsamhet socialt umgänge minne hitta strategier à 99 statements (outcomes)
People with PD vs. Health Care Professionals People with PD Health Care Professionals Heterogeneous and unidimensional Homogeneous and mulcdimensional
People with PD vs. Health Care Professionals People with PD Health Care Professionals Highest importance rated statements People with PD Clinicians Quality of life Quality of life Walking ability Depression Mobility Fluctuations Psychological well-being Walking ability Sleeping problems Sleeping problems Control over disease process Falling
Illustra(ng the Problem A clinical trial of an adjunct (L- dopa add- on) drug for Parkinson s disease with wearing- off Primary end- points: AcCviCes in Daily Living (ADL): UPDRS II Quality of Life (QoL): PDQ- 39
What about QoL? Outcome measure: 39-item Parkinson s Disease Questionnaire (PDQ-39) Results: No significant differences between treatments according to the PDQ-39 Difference score: 0.6 on a scale from 0-100 Implication: Adjunct therapy does not improve QoL
What does the PDQ- 39 measure? We do not know
Correlation between the PDQ-39 and perceived QoL: -0.4 Patient-prioritized QoL aspects not reflected in the PDQ-39 Patient uncertainty whether the questionnaire would be capable of reflecting their experiences
Hagell & McKenna. Parkinsonism & Related Disorders 2003; 10(2): 89-92. Kim et al. Acta Neurologica Scandinavica 2006; 113(2): 132-137. Hagell et al. Mov Disord 2003; 18(7): 773-783. The PDQ-39 from an end-user perspective Percentage 100 90 80 70 60 50 40 30 20 10 0 Irrelevant items Missing areas Extremely Relevant Relevance Ra3ng Not at all Relevant 10 9 8 7 6 5 4 3 2 1 Pa3ents Clinicians (MDs/RNs) 1 0,9 0,8 0,7 0,6 0,5 0,4 0,3 0,2 0,1 0 CorrelaCons with perceived QoL
How well does the PDQ-39 represent levels of health among people with PD?
Despite obvious similarices, pacent perspeccves differ from that of clinicans Lived experience vs. scholarly/biomedical perspeccve End- user prioriczed outcomes Quality of life, walking/mobility, sleep, facgue, perceived disease control; fluctuacons, depression, falls à Evidence from clinical trials that are more meaningful and interpretable for end- users Research implicacons Needs for new and refined outcome measures PrioriCes for therapeucc research