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People living with moderate-to-severe COPD prefer improvement of daily symptoms over the improvement of exacerbations: a multicountry patient preference study

Nigel S. Cook, Gerard J. Criner, Pierre-Régis Burgel, Katie Mycock, Tom Gardner, Phil Mellor, Pam Hallworth, Kate Sully, Sophi Tatlock, Beyza Klein, Byron Jones, Olivier Le Rouzic, Kip Adams, Kirsten Phillips, Mike McKevitt, Kazuko Toyama, Florian S. Gutzwiller
ERJ Open Research 2022 8: 00686-2021; DOI: 10.1183/23120541.00686-2021
Nigel S. Cook
1Novartis Pharma AG, Basel, Switzerland
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Gerard J. Criner
2Temple University, Philadelphia, PA, USA
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Pierre-Régis Burgel
3Cochin Hospital, Assistance Publique Hôpitaux de Paris, Paris, France
4Université de Paris and Institut Cochin, Inserm U1016, Paris, France
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Katie Mycock
5Adelphi Real World, Macclesfield, UK
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Tom Gardner
6Adelphi Research, Macclesfield, UK
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Phil Mellor
6Adelphi Research, Macclesfield, UK
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Pam Hallworth
6Adelphi Research, Macclesfield, UK
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Kate Sully
7Adelphi Values, Macclesfield, UK
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Sophi Tatlock
7Adelphi Values, Macclesfield, UK
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Beyza Klein
1Novartis Pharma AG, Basel, Switzerland
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Byron Jones
1Novartis Pharma AG, Basel, Switzerland
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Olivier Le Rouzic
8La Fondation du Souffle, Paris, France
9Univ. Lille, CHU Lille, Inserm U1019, Lille, France
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Kip Adams
10COPD Foundation, Miami, FL, USA
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Kirsten Phillips
11Lung Foundation Australia, Brisbane, Australia
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Mike McKevitt
12British Lung Foundation, London, UK
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Kazuko Toyama
13J-Breath, Tokyo, Japan
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Florian S. Gutzwiller
1Novartis Pharma AG, Basel, Switzerland
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  • For correspondence: florian.gutzwiller@novartis.com
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  • FIGURE 1
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    FIGURE 1

    Design of quantitative patient preference study. PRO: patient-reported outcome; EQ-5D: European Quality of Life Questionnaire; CASA-Q: Cough and Sputum Assessment Questionnaire; DCE: discrete choice experiment; FDA: Food and Drug Administration; ISPOR: International Society for Pharmacoeconomics and Outcomes Research; NICE: National Institute for Health and Care Excellence; IRB: independent review board; A&L: attributes and levels. #: to assess existing evidence on patients’ preferences in terms of types and amount of evidence available and objectives and outcomes of earlier research; ¶: to gain more information on what affects and motivates patients, their questions, pains, experiences, concerns and the way they communicate about their disease; +: to explore the disease experience, understand the priorities, detect potentially hidden aspects and further understand the communication.

  • FIGURE 2
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    FIGURE 2

    Profile-matching showing average patient profile, selected by patients to best match themselves at time of completing the questionnaire (summating results over all countries). Shaded boxes show the median level selected by patients for each attribute as that matching their current health state. Percentages of selected levels by attribute (level 1/level 2/level 3/level 4 (for shortness of breath)): exacerbation 14%/64%/23%; sleep quality 17%/51%/32%; shortness of breath 31%/39%/26%/5%; urinary incontinence 46%/39%/14%; mucus clearance 22%/57%/21%; cough 29%/54%/17%.

  • FIGURE 3
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    FIGURE 3

    Preference weights. CI: confidence interval; sd: standard deviation.

  • FIGURE 4
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    FIGURE 4

    Impact of attributes improvement on patient preference (events of daily living). a) Improvement in cough and mucus versus improvement in shortness of breath (SOB); b) improvement in daily symptoms (excess mucus production, cough and SOB) versus improvement in exacerbations.

Tables

  • Figures
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  • TABLE 1

    Example of choice task for COPD disease state profiles as seen by the patients: patients are asked to select their preference between the disease states of patient A or B

    Patient APatient B
    When waking up on a typical morning:You feel restedYou do not feel rested at all
    On a typical morning:It is not at all difficult to bring up mucusIt is a little difficult to bring up mucus
    On a typical day:You experience shortness of breath when washing (e.g. taking a shower) or dressingYou experience shortness of breath when sitting or lying down
    Your cough does not interrupt/disturb any of your usual activitiesYour cough interrupts/disturbs most of your usual activities
    COPD symptoms are causing a few drops of urine leakageCOPD symptoms are causing a few drops of urine leakage
    During a typical year:Never experience any COPD flare-ups/exacerbationsYou experience one or more COPD flare-ups/exacerbations that require a hospital stay or visit
  • TABLE 2

    Demographics and clinical characteristics of participants who completed the online survey

    ParametersQuantitative phase II (n=1050)
    Age, years, mean±sd
     At survey60.5±11.0
     At diagnosis53.6±11.3
    Time since diagnosis, years, mean±sd7.2±7.0
    Age groups %
     40–59 years45
     60–79 years50
     >80 years5
    Sex, male %51
    Country n (%)
     USA400 (38.1)
     UK200 (19.0)
     France150 (14.3)
     Australia150 (14.3)
     Japan150 (14.3)
    Body mass index % (n=954)
     Underweight (<18.5 kg·m−2)4
     Healthy (18.5–24.9 kg·m−2)44
     Overweight (25.0–29.9 kg·m−2)28
     Obese (>30 kg·m−2)24
    Smoking status %
     Current smokers29
     Ex-smokers57
     Never-smokers14
    Comorbidities %
     Asthma44
     Hypertension40
     Allergies34
     Depression/anxiety25
     Gastro-oesophageal reflux disorder22
     Sleep disturbance31
     Diabetes17
     Obesity16
     Osteoporosis16
     Urinary incontinence15
     Rheumatology disease15
    COPD severity (self-perceived) %
     Mild11
     Moderate54
     Severe/very severe35
    Severity derived from PRO instruments#, mean±sd
     CAT score25.0±6.67
     CASA-Q score50.3±20.9
    Exacerbations in the past 12 months¶%
     029
     124
     222
     ≥325
    Amount of mucus when coughing in the past 7 days %
     A great/very great deal35
     Some/a little62
     None at all3
    Asthma/respiratory allergy %
     Since childhood23
     Since adulthood42
     No allergy35
    Educational qualification (higher college or university degrees) %
     USA68
     UK48
     France72
     Australia59
     Japan86
    Employment status %
     Retired42
     Currently full time34
     Other (working part time, home maker, on sick leave, student and unemployment)24

    PRO: patient-reported outcome; CAT: COPD Assessment Test; CASA-Q: Cough and Sputum Assessment Questionnaire. #: CAT scores range from 0 to 40; higher scores denote a more severe impact of COPD on a patient's life; and CASA-Q domain scores range from 0 to 100, with higher scores associated with fewer symptoms/less impact due to cough or sputum. ¶: that required hospitalisation; higher scores denote a more severe impact of COPD on a patient's life.

    Supplementary Materials

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      Supplementary material 00686-2021.SUPPLEMENT

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    People living with moderate-to-severe COPD prefer improvement of daily symptoms over the improvement of exacerbations: a multicountry patient preference study
    Nigel S. Cook, Gerard J. Criner, Pierre-Régis Burgel, Katie Mycock, Tom Gardner, Phil Mellor, Pam Hallworth, Kate Sully, Sophi Tatlock, Beyza Klein, Byron Jones, Olivier Le Rouzic, Kip Adams, Kirsten Phillips, Mike McKevitt, Kazuko Toyama, Florian S. Gutzwiller
    ERJ Open Research Apr 2022, 8 (2) 00686-2021; DOI: 10.1183/23120541.00686-2021

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    People living with moderate-to-severe COPD prefer improvement of daily symptoms over the improvement of exacerbations: a multicountry patient preference study
    Nigel S. Cook, Gerard J. Criner, Pierre-Régis Burgel, Katie Mycock, Tom Gardner, Phil Mellor, Pam Hallworth, Kate Sully, Sophi Tatlock, Beyza Klein, Byron Jones, Olivier Le Rouzic, Kip Adams, Kirsten Phillips, Mike McKevitt, Kazuko Toyama, Florian S. Gutzwiller
    ERJ Open Research Apr 2022, 8 (2) 00686-2021; DOI: 10.1183/23120541.00686-2021
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