Patient Characteristics, Treatment Patterns, and Health Outcomes Among
Chronic Obstructive Pulmonary Disease (COPD) Phenotypes
Felicia Allen-Ramey, PhD; Shaloo Gupta, MS; Marco DiBonaventura, PhD
Global Health Outcomes, Merck & Co., Inc., West Point, PA; Health Sciences Practice, Kantar Health, Princeton, NJ; Health Sciences Practice, Kantar Health, New York, NY
Rationale: Recent literature has suggested emphysema and chronic bronchitis,
traditionally considered overlapping entities within COPD, may be distinct conditions.
Although prior research has examined the extent of the overlap among COPD
phenotypes, few studies have examined the patient characteristic and health outcome
differences among them. The aim of this study was to address this gap using a large
nationally representative dataset.
• Out of the 75,000 participants, patients who did not report a diagnosis
Methods: Data were obtained from the 2010 US National Health and Wellness Survey
(NHWS). The NHWS is an annual Internet-based survey, that uses a stratified random
sample to ensure demographic representativeness to the adult US population. NHWS
respondents (N=75,000) were categorized into a COPD phenotype based on their
self-reported diagnosis: COPD only (n=970), emphysema only (n=399), and chronic
bronchitis only (n=2,071). Phenotypes were compared on demographics (e.g., age,
gender, ethnicity, employment, etc.), health characteristics (e.g., BMI, smoking,
alcohol use, exercise, comorbidities, etc.), treatment patterns, and health outcomes
(health-related quality of life using the SF-12v2, work productivity loss using the
Work Productivity and Activity Impairment questionnaire, and healthcare resource
use). All variables were compared using chi-square and ANOVA tests for categorical
and continuous outcomes, respectively. Health outcomes were also examined using
regression modeling controlling for demographic and health characteristic covariates.
Results: Patients with chronic bronchitis were significantly younger (51.38 years vs.
COPD=63.24 vs. emphysema=63.30, p<.05) and more likely to be employed (46.98%
vs. COPD=23.81% vs. emphysema=28.33%, p<.05). Relative to the other phenotypes,
patients with chronic bronchitis were also significantly more likely to be female, non-white,
and to currently exercise (all ps<.05); they were significantly less likely to be a current
or former smoker (all ps<.05). Controlling for demographic and health characteristics,
patients self-identified as COPD-only reported significantly worse physical quality of
life (adjusted mean=36.69) and health utilities (adjusted mean=0.65) and significantly
more absenteeism (adjusted mean=7.08%), presenteeism (adjusted mean=30.73%),
overall work impairment (adjusted mean=34.06%), and activity impairment (adjusted
mean=46.59%) than other phenotypes (all ps<.05).
Conclusions: These results suggest considerable heterogeneity in demographic and
health profiles among these self-reported phenotypes of COPD. Patients self-reporting
only COPD, rather than only chronic bronchitis or emphysema, may be more adversely
affected by their condition as indicated by worse health outcomes. Differences in the
characteristics and disease presentation of these phenotypes should be used to guide
treatment decision making.
Introduction
• Chronic obstructive pulmonary disease (COPD) refers to a progressive
condition that can result most frequently from emphysema, chronic
bronchitis, and a subset of asthma sufferers [1].
of COPD, emphysema, or chronic bronchitis were removed from
the analyses (n=70,345). Additionally, patients with more than one
COPD phenotype (those diagnosed with at least two of the following:
emphysema, chronic bronchitis, and COPD) were removed from the
analyses (n =1,215).
• However, these studies have assumed a degree of homogeneity
among different COPD phenotypes that may not exist. Research in
recent years has proposed that the heterogeneity associated with
the clinical presentation, disease course, and treatment of COPD is
sufficiently varied to warrant the identification and classification of
phenotypes to guide effective clinical care [4].
• The few research studies that have examined specific phenotype
characteristics and outcomes have reported significant differences,
thus establishing a need for further investigation.
Objective
• To investigate the patient characteristics, treatment patterns, and
health outcomes of those diagnosed with only chronic bronchitis,
emphysema, or COPD.
Methods
Sample
• Data were obtained from the 2010 US National Health and Wellness
Survey (NHWS). The NHWS is an annual Internet-based survey that
uses a stratified random sample framework to ensure demographic
representativeness to the adult US population. This cross-sectional
database includes responses from 75,000 adults (aged 18 and over).
• Of patients reporting one, and only one, diagnosis, most patients reported being diagnosed with chronic bronchitis
(n = 2,071; 60.20%), followed by COPD (n = 970; 28.20%) and emphysema (n = 399; 11.60%) (see Figure 1).
11.60%
(COPD only [n = 970], emphysema only [n = 399], and chronic
bronchitis only [n = 2,071]) and were the primary analysis groups
of interest.
Current medication use (for COPD)
Medication*
SABA only
LAMA only
ICS only
LAB A only
Oxygen only
28.20%
Diagnosed COPD only
Diagnosed chronic bronchitis only
Measures
• Demographics – Survey respondents reported their age, gender,
Diagnosed emphysema only
race/ethnicity, annual household income, education, and employment
status.
• Health characteristics – Body mass index (BMI), smoking status,
exercise behavior, and alcohol consumption were also assessed.
Self-reported comorbidity data were used to calculate a comorbidity
burden score using the Charlson comorbidity index.
60.20%
• Disease characteristics – Years diagnosed with their condition, their
self-reported disease severity (mild, moderate, or severe), current
medication use, and the frequency (on a five-point Likert-type scale
of “never” to “always”) of dyspnea, fits of coughing, infection, mucous
production, and wheezing were also measured.
• Health outcomes
––Health status - The mental component summary (MCS), physical
component summary (PCS), and health utility score (using the SF6D) from the SF-12v2 [5] were used in the analysis. Higher scores
on these measures indicate better health status.
––Work productivity - The Work Productivity and Activity Impairment
Questionnaire (General Health version; WPAI-GH) [6] was used to
calculate:
•
•
•
•
•
Absenteeism - percentage of missed work days due to health in
the past seven days
Presenteeism - percentage of impairment while at work due to
health in the past seven days
Overall work impairment - total percentage of missed days due to
absenteeism and presenteeism
Activity impairment - percentage of impairment during daily
activities in the past seven days.
Higher scores indicate greater impairment (range 0% to 100%)
• Healthcare resource use – All patients reported the number of
traditional healthcare provider visits, ER visits, and hospitalizations in
the past 6 months for their own medical condition.
Statistical Analysis
• Differences among phenotypes on demographics, health
characteristics, and treatment patterns were compared using chisquare and ANOVA tests for categorical and continuous outcomes,
respectively.
• Differences in health outcomes were examined using regression
modeling (linear regression for health status outcomes and
generalized linear models for work productivity and resource use
outcomes) controlling for demographic and health characteristic
covariates.
Table 2. Treatment and symptom differences among self-identified COPD phenotypes
Figure 1. Sample sizes for patient-reported COPD phenotypes
• The remaining patients were categorized based on their phenotype
• A number of studies have suggested that the presence of COPD
is associated with reduced quality of life, impaired functioning, and
greater direct and indirect costs [2-3].
Results
• Compared with COPD and emphysema patients, patients with chronic bronchitis were significantly younger and
more likely to be female, non-white, and a non-smoker (see Table 1).
Table 1. Patient characteristics and self-reported health behaviors among COPD phenotypes
COPD only (n = 970)
Variable
Male
Female
White non-Hispanic
Black non-Hispanic
Hispanic
Other
High school graduate or less
Some college or higher
Income: $25K or less
Income: $25K to <$50K
Income: $50K to <$75K
Income: $75K or more
Income: Decline to answer
Full-time employed
Part-time employed
Self-employed
BMI: Underweight
BMI: Overweight
BMI: Normal
BMI: Obese
BMI: Decline to answer
Never smoked
Former smoker
Current smoker
Currently consume alcohol
Currently exercise
Age
Years diagnosed
CCI*
n
559
411
866
50
23
31
245
725
278
320
152
158
62
122
63
46
14
286
232
428
10
111
544
315
529
421
Mean
63.24
7.57
2.00
%
57.63%
42.37%
89.28%
5.15%
2.37%
3.20%
25.26%
74.74%
28.66%
32.99%
15.67%
16.29%
6.39%
12.58%
6.49%
4.74%
1.44%
29.48%
23.92%
44.12%
1.03%
11.44%
56.08%
32.47%
54.54%
43.40%
SD
10.90
7.43
1.47
Emphysema only
(n = 399)
n
%
248
62.16%
151
37.84%
346
86.72%
26
6.52%
9
2.26%
18
4.51%
109
27.32%
290
72.68%
120
30.08%
125
31.33%
66
16.54%
64
16.04%
24
6.02%
56
14.04%
32
8.02%
25
6.27%
12
3.01%
127
31.83%
128
32.08%
130
32.58%
2
0.50%
28
7.02%
223
55.89%
148
37.09%
247
61.90%
168
42.11%
Mean
SD
63.30
13.32
9.39
9.63
1.82
1.28
Chronic bronchitis only
(n = 2,071)
n
%
762
36.79%
1309
63.21%
1641
79.24%
189
9.13%
93
4.49%
148
7.15%
486
23.47%
1585
76.53%
538
25.98%
631
30.47%
377
18.20%
391
18.88%
134
6.47%
633
30.56%
198
9.56%
142
6.86%
28
1.35%
569
27.47%
440
21.25%
972
46.93%
62
2.99%
703
33.95%
698
33.70%
670
32.35%
1255
60.60%
1175
56.74%
Mean
SD
51.38
14.80
16.23
14.80
1.70
1.24
p
<.0001
<.0001
<.0001
.0001
.0002
<.0001
0.2140
0.2140
0.1239
0.3822
0.1982
0.1284
0.9412
<.0001
0.0110
0.0551
0.1799
0.1676
<.0001
<.0001
<.0001
<.0001
<.0001
0.1875
0.0034
<.0001
p
<.0001
<.0001
<.0001
*Charlson comorbidity index: index score that is calculated by weighting the presence of specific comorbidities based on their association with future mortality and summing the results.
• Those with chronic bronchitis had been diagnosed the longest, were the least likely to report the severity of
their condition as “moderate” or “severe”, and their hallmark symptoms, compared with those with COPD and
emphysema, were mucous production, coughing, and infection (see Table 2).
• A number of patients across all phenotypes remain untreated. Short-acting beta-agonist (SABA) use was the most
common monotherapy for all phenotypes but significantly more so (p<.05) for patients with chronic bronchitis.
Patients with chronic bronchitis were also significantly more likely to use inhaled corticosteroids (ICS) + a longacting beta agonist (LABA) (13.62%) and ICS+LABA+SABA (19.31%) therapies (ps<.05). Conversely, triple
therapy (ICS+LABA+long-acting muscarinic antagonist [LAMA]) was significantly more infrequent for patients with
chronic bronchitis whether it was with or without a SABA.
COPD only
(n = 970)
n
%
728
75.05
89
77
7
5
1
12.92
11.18
1.02
0.73
0.15
Emphysema only
(n = 399)
n
%
211
52.88
23
20
6
2
4
11.22
9.76
2.93
0.98
1.95
Chronic bronchitis
only (n = 2,071)
n
%
585
28.25
33.54
2.64
5.89
0.20
0.00
165
13
29
1
0
p
<.0001
90
78
15
9
6
4
13.06
11.32
2.18
1.31
0.87
0.58
28
21
5
1
1
2
13.66
10.24
2.44
0.49
0.49
0.98
67
36
39
2
2
1
13.62
7.32
7.93
0.41
0.41
0.20
0.5896
0.9528
<.0001
0.3919
0.0705
0.2114
ICS+LABA+SABA only
ICS+LAMA+LABA only
ICS+LAMA+SABA only
LAMA+LABA+SABA only
94
58
27
5
13.64
8.42
3.92
0.73
14
23
7
2
6.83
11.22
3.41
0.98
95
5
6
0
19.31
1.02
1.22
0.00
<.0001
0.0213
<.0001
0.1308
ICS+LAMA+LABA+SABA
116
16.84
44
21.46
31
6.30
<.0001
8
1.16
2
0.98
0
0.00
0.0602
412
434
124
42.47
44.74
12.
199
162
38
49.87
40.60
9.52
1354
621
96
65.38
29.99
4.64
<.0001
<.0001
<.0001
3.74
2.78
1.99
2.97
2.97
1.07
1.06
0.93
1.15
1.08
3.59
2.65
1.78
2.86
2.71
1.16
1.07
0.92
1.23
1.16
2.95
2.83
2.31
3.05
2.76
1.18
1.02
0.97
1.12
1.10
<.0001
0.0040
<.0001
0.0053
<.0001
*Percentages based on only those using a prescription medication. ** Symptoms were reported on a five-point Likert-type response scale (1=never to 5=always).
LAMA: ipratropium, tiotropium LABA+ICS (fixed dose combination): budesonide/formoterol, fluticasone/salmeterol; LAMA+SABA: albuterol/ipratropium; ICS: flunisolide, mometasone,
triamcinolone, fluticasone, fluticasone propionate, budesonide; LABA: arformoterol, formoterol, salmeterol; SABA: albuterol, pirbuterol, albuterol sulfate, levabuterol;
p-values test whether the percentages (or means) are significantly different across different phenotypes.
• Adjusting for demographics and health characteristics, patients with COPD reported the lowest levels of PCS
(adjusted mean = 36.69) and health utilities (adjusted mean = 0.65) relative to other phenotypes (see Figure 2).
Figure 2. Adjusted differences on physical and mental component summary scores among
COPD phenotypes
*
50
46.59%
45%
*
41.30%
40.40%
40%
35%
30.73%
30%
*
34.06%
26.60%
24.64%
23.50%
22.10%
25%
*
20%
15%
10%
5%
7.08%
5.64%
2.96%
0%
Absenteeism
COPD only
Presenteeism
Overall Work
Productivity
Chronic bronchitis only
Activity
Impairment
Emphysema only
*p <0.05 emphysema only vs. COPD only.
Absenteeism, presenteeism, and overall work productivityloss only consist of the employed sample.
• No significant differences were observed among the groups with respect to healthcare resource utilization.
Conclusions
• These
results suggest considerable heterogeneity in demographic and health profiles among
these self-reported phenotypes of COPD.
• Drug
use reflected a different approach to treatment across the phenotypes, with ICS/LABA use
most prominent among COPD and chronic bronchitis patients and triple therapy (both with and
without a SABA) most commonly prescribed for emphysema patients. As emphasized in the
most recent GOLD guidelines [7], these patterns highlight chronic bronchitis and emphysema
as distinct from COPD despite their clinical associations.
• Patients
self-reporting only COPD, rather than only chronic bronchitis or emphysema, may be
more adversely affected by their condition as indicated by worse health outcomes.
• Research
45.52 44.97 46.98
36.69
40
50%
<.0001
<.0001
0.3623
<.0001
0.0002
ICS+LABA only
LAMA+SABA only
ICS+SABA only
LABA+SABA only
ICS+LAMA only
LAMA+LABA only
Oxygen + other treatments
Disease severity
Mild
Moderate
Severe
Symptom frequency**
Dyspnea (Mean, SD)
Coughing (Mean, SD)
Infection (Mean, SD)
Mucous (Mean, SD)
Wheezing (Mean, SD)
Figure 3. Adjusted differences on work productivity and activity impairment among
COPD phenotypes
Adjusted impairment
Participants self-reported information on demographics, healthcare
attitudes and behaviors, disease status, and outcomes.
Adjusted health status
Abstract
40.45
*
aimed at understanding the differences in patient characteristics and disease
presentation of these phenotypes could be used to guide treatment recommendations.
38.27
30
Limitations
• All
data from the NHWS is self-reported so clinical verification of diagnoses, treatments, and
lung function were unavailable. Diagnostic reasons for patients to receive one diagnosis or
another were not known and could be a combination of patient and physician factors.
20
• This
study focused on “pure” diagnoses (COPD, chronic bronchitis, or emphysema) and it is
unclear the extent to which the differences observed here manifest when multiple diagnoses
are present.
10
0
MCS LSMEAN
COPD only
Chronic bronchitis only
PCS LSMEAN
Emphysema only
*p <0.05 emphysema only vs. COPD only & chronic bronchitis only.
• Patients with COPD reported the highest level of work impairment. Patients with COPD reported greater levels of
both work and activity impairment relative to both those with chronic bronchitis and emphysema (see Figure 3).
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