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Concordance of the Psoriasis Area and Severity Index (PASI) and patient-reported outcomes in psoriasis treatment


European Journal of Dermatology. Volume 20, Number 1, 62-7, January-February 2010, Investigative report

DOI : 10.1684/ejd.2010.0815

Summary  

Author(s) : Ines Schäfer, Jana Hacker, Stephan Jeff Rustenbach, Marc Radtke, Nadine Franzke, Matthias Augustin , CVderm – German Center for Health Services Research in Dermatology, Research Group Health Economics and Quality of Life Research, Department of Dermatology and Venereology, University Clinics of Hamburg, Martinistraße 54, D-20246 Hamburg, Germany.

Summary : A prospective observational study was conducted to analyze to what extent the Psoriasis Area and Severity Index (PASI) is correlated to patient reported outcomes (PRO). Data were collected on 93 outpatients with psoriasis prior to (t1) and after therapy (t2). A higher PASI score was associated with impaired Quality of Life (DLQI), however associations were only weak (R \= 0.24 on t1 and R \= 0.36 on t2). The average improvement in PASI score was correlated moderately highly with the change of DLQI (R \= 0.50) as well as with the PBI (R \= 0.45). The threshold values PASI-50 and PASI-75 were likewise positively associated with PRO, but they only incompletely reflect them: about half of the patients with PASI-50 did not see their expectations met (46.9%) or were not satisfied with the condition of the skin after treatment (56.3%). A quarter of these patients experienced no benefits from the therapy. Of the patients with PASI-75, 15.4% were not satisfied with the condition of their skin. In contrast, 36% of patients who did not attain PASI-50 specified a high level of treatment satisfaction. PASI and PRO are associated but based on different concepts. In addition to clinicial severity, patient-defined benefits should be implemented in the evaluation of psoriasis care.

Keywords : DLQI, PASI, patient benefit, patient-reported outcomes, psoriasis vulgaris

Pictures

ARTICLE

Auteur(s) : Ines Schäfer, Jana Hacker, Stephan Jeff Rustenbach, Marc Radtke, Nadine Franzke, Matthias Augustin

CVderm – German Center for Health Services Research in Dermatology, Research Group Health Economics and Quality of Life Research, Department of Dermatology and Venereology, University Clinics of Hamburg, Martinistraße 54, D-20246 Hamburg, Germany

accepté le 3 Septembre 2009

The therapeutic spectrum of psoriasis vulgaris is broad and management of the disease is varied [1, 2]. Over recent years, the need to improve the structure of psoriasis therapy, to define treatment goals and to measure treatment outcomes by means of parameters of clinical severity and Quality of Life has intensified [3, 4]. Of particular importance is the recording of outcomes of therapies with systemic drugs and biologics in accordance with valid treatment standards [5]. These essentially allow for the measurement of results by means of the Psoriasis Area and Severity Index (PASI) [6]. In particular, improvements in the PASI of about 50%, 75% or 90% in comparison to baseline are regarded as markers of effective treatment [7, 8].

It is yet to be shown that the PASI value fails to correlate strongly with the patient’s state of health as well as with her/his Quality of Life [9]. Subjective assessments of the severity of the disease can turn out in similar clinical findings rather differently. Additionally, the severity of the psoriasis [10] and the respective living circumstances, age [11], stress, and mental attributes [12, 13] have so far been identified as influence factors for the subjectively perceived burdens caused by the disease. The expectations and goals that patients associate with psoriasis treatments, as well as the assessment of treatment benefits, are appropriately complex and, to a great extent, unique to the individual [14].

Taking this into account, it is essential to supplement the assessment of a course of therapy via PASI with a patient-reported parameter [15]. Also, the PASI threshold value as a measure of “response” should exhibit sufficient relevance to patients. In preliminary studies, the correlation between a change in PASI (∆PASI) and patient-reported outcomes appeared inconsistent. Touw et al. [16] found only a moderate correlation between ∆PASI and the health-related Quality of Life (Dermatology Life Quality Index, DLQI); Revicki et al. [17] saw significant association of ∆PASI with the DLQI as well as with the Short Form 36 (SF-36 total score). However, a strong effect was found in an improvement of PASI-Scores from 90% or 100%. In contrast, the correlation between ∆PASI and the changes in various psychological scores was not significant [13].

In addition to Quality of Life, the patient-defined benefit of therapy is a further independent factor for external validity of ∆PASI, one that has not been analyzed in this context so far.

The patient-defined treatment benefits differ considerably in concept from the construct of Quality of Life. The Quality of Life instruments normally do not cover the broad and individual spectrum of patient-relevant benefits and provide no possibilities for preference formation [18]. Furthermore, Quality of Life parameters are based upon a global assessment of the areas of life, whereas the ascertainment of treatment benefits applies specifically to the effects of a defined therapy and thereby suggests a causal attribution. It is as yet unclear how relevant the improvements in PASI-scores are for the patient her/himself. In particular, there is a lack of data on the correlation between personal assessment of treatment benefits and the clinical outcomes PASI-50 and PASI-75.

Study questions

  • 1. To what degree does the cross-sectional PASI correlate with patient-reported outcomes?
  • 2. To what extent are PASI-50 and PASI-75 reflective of the health-related Quality of Life as well as the treatment benefits experienced by the patients?

Materials and methods

A prospective observational study with two collection dates was conducted. The study population was 100 consecutive patients who were treated as outpatients in the study period from April to September 2007 in the Department of Dermatology at the University Clinics of Hamburg. Inclusion criteria were age of at least 18 years, as well as a clinically confirmed diagnosis of psoriasis vulgaris. The treatments were carried out according to clinical standards and had no bearing on the patient recruitment of the study.

Data collection

The attending physicians and the patients filled out standardised questionnaires at the start (t1) and towards the end of treatment (t2). On the physician’s questionnaire the following clinical and treatment-related variables were recorded: attributes of the psoriasis, prior therapies and co-morbidity, and the existence or indication of psoriatic arthritis. In addition, the PASI was determined during both data collection dates. With the patient’s questionnaire at t1, data were obtained on socio-demographic characteristics, burden of disease, general state of health, and health care utilization. The DLQI was filled out as a parameter of health-related Quality of Life as well as the Patient Needs Questionnaire (PNQ) as the first part of the Patient Benefit Index (PBI) for assessment of patient-defined treatment needs. The patient questionnaire at the second collection date contained questions on the burden of psoriasis and its treatment, as well as the health scales and the DLQI. Additionally, a general evaluation of therapies, as well as the second part of the PBI, the Patient Benefits Questionnaire (PBQ) were recorded.

Measuring instruments

Psoriasis Area and Severity Index (PASI). The PASI score [19] is the most widely used index measure of psoriasis severity, evaluating the area, erythema, scaliness and thickness of the plaques. The PASI-score ranges from 0 to 72. The parameters PASI-50 and PASI-75 correspond to an improvement of the PASI of about 50% and 75%, respectively, compared to the baseline score.

Patient Benefit Index (PBI). For the assessment of patient-defined treatment benefits, the PBI was used. This is an instrument developed and validated specifically for the field of dermatology [20, 21]. The basic foundation step is the pre/post-data-collection. Prior to therapy, the individually perceived needs (Patient Needs Questionnaire, PNQ) are obtained. For each of the 25 standardized items the patient rates its importance by means of a 5-point Likert Scale ranging from 0 (not important at all) to 4 (very important). After therapy, the degree to which these benefits have been achieved is assessed by the same item list, on the Patient Benefit Questionnaire (PBQ), on a 5-point scale from 0 (did not help at all) to 4 (helped very much). As a single outcome parameter, the Patient Benefit Index (PBI) is computed by weighting the PBQ items with their respective PNQ items. The PBI ranges from 0 (no benefit) to 4 (maximum benefit). PBI ≥ 1 is adopted as a cut-off value for a “relevant treatment benefit”.

Single questions for treatment-evaluation. With a set of 8 items, patient satisfactions and the effects of the treatment in her/his living situation were obtained on t2. For the following analyses, the two questions which were generally formulated and are thus relevant to all forms of therapy, were used: 1) “Were your expectations met by therapy?”; and 2) “Is the condition of the skin achieved sufficient for you?”, Both questions were answered on a scale of 1 (completely) to 4 (not at all).

DLQI. For the measurement of disease-specific (dermatological) Quality of Life, the 10-item-scale of the DLQI [22] was applied. The cumulative value of the DLQI ranges from 0 to 30. A higher DLQI indicates a more intense impairment due to the skin disease. On a score that is lower than 2, it is assumed that the QoL is not restricted.

EuroQol (EQ-5D). The general health-related Quality of Life was measured with the Visual Analog Scale (VAS) of the EQ-5D [23], in which the patient rates her/his current state of health on a scale ranging from 0 (“worst conceivable state of health”) to 100 (“best conceivable state of health”).

Statistical analyses

In the descriptive analyses, the absolute and percentage frequencies are presented for categorical data; for continuous variables, mean and standard deviation are given. The validated scales (PASI, PBI and DLQI) were each analyzed according to their standardised syntax. Changes in the PASI score between the collection dates t1 and t2 were calculated as continuous values (∆PASI in %) as well as threshold values PASI-50 and PASI-75. Computations were carried out (as with changes in DLQI and in the values of the health scale of the EQ-5D), as percentage changes of the baseline values (t1). For the analysis of the correlation between PASI and patient-reported outcomes (each with continuous variables), Spearman Rank Correlation Coefficients were calculated (level of significance α ≤ 0.05, two-sided test). The distribution of the parameters PASI<50, PASI-50 and PASI-75 were analyzed according to the dichotomous values of DLQI, PBI and the single questions on treatment-evaluation. The data analyses were carried out with SPSS 15.0 for Windows®.

Results

Available for the evaluation of the first collection date were data from 93 patients (38% female), of whom 80 (39% female) also took part in the second data collection (table 1). The mean time-lag from t1 to t2, and thus the period of treatment and observation, was 42.5 days (± 17.0; Median 38.5).

On average, the PASI score improved about 62% (± 24.9), and with the exception of one patient, the PASI of t2 was in each case lower than that of t1. 40% of all patients exhibited an improvement of at least 50%, and 32.5% of patients, an improvement of at least 75% in PASI. The general state of health represented by the visual analog scale of the EQ-5D increased on average from a value of 54.4 (max 100) at t1 to 69.0 at t2. With a DLQI total score of 5.6, the impairment of the Quality of Life of t2 was considerably lower than that of t1 (DLQI 9.7). On a scale of 0 (treatment did not help at all) to 4 (helped a lot), the mean value of PBI was 2.3 (± 1.3); 76.3% of the patients attained a PBI of ≥ 1.0.

On the general questions about treatment satisfaction, 61.3% of the patients specified that their expectations were met completely or predominantly. Slightly more than half of the patients were completely or predominantly satisfied with the achieved condition of the skin; 16.3% were not satisfied at all (table 2). In a cross-section, a minor correlation between PASI and the patient-defined outcomes existed in both collection dates (table 3): the correlation of PASI and DLQI was R = 0.24 on t1 and R = 0.36 on t2. A similar association appeared for the PASI and the health scales of the EQ-5D: here, the correlation coefficient in each case was about R = – 0.30. Thus, a higher PASI is associated with a more intense impairment of the dermatological Quality of Life and with a lower assessment of the general state of health; though the correlation was only moderate in each case.

The average improvement in PASI score (∆PASI) was correlated moderately highly with the PBI (R = 0.45), as well as with the change of DLQI (R = 0.50). There was no existing correlation with the increase on the general health scale of the EQ-5D (R = 0.10) (table 4).

The distribution of the three PASI groups, PASI<50, PASI-50 and PASI-75, on the patient-reported outcomes (table 5, figures 1, 2), shows that the percentage of patients who evaluated the outcome positively increased with improvements in PASI scores. Nevertheless, relevant percentages of unsatisfied patients in groups with high PASI improvement were found in each case, as well as a comparatively high percentage of satisfied patients in groups with low PASI improvement.

While most patients with PASI-75 specified that their expectations of the treatment were completely or predominantly met, this applied to only slightly more than half (53.1%) of the patients in the group with PASI-50. For the other half (46.9%), the expectations were not all met or somewhat met. In the group with the lowest clinical treatment success (PASI<50, however, 36.4% of the patients answered that their expectations of the treatment were met (figure 1). More than half of the patients with PASI-50 and 15.4% of the patients with PASI-75 were not satisfied with the achieved condition of the skin after therapy. On the other hand, 36.4% of the patients who did not reach PASI-50 were satisfied with their skin-condition (figure 2).

A DLQI score lower than 2 implies that the psoriasis does not impair patient’s Quality of Life. This applied for only 18.8% of the PASI-50 and for 34.6% of the PASI-75 group after therapy. Accordingly, 65% of the patients with an increase of the PASI of about 75% further experienced restrictions in their Quality of Life due to the psoriasis (table 5). In the groups with PASI-50 and PASI-75, 62.5% and 42.3% of the patients, respectively, attained no improvement of the DLQI of at least 5 score points. Slightly more than half of the patients in the group under PASI-50, 75.0% of the patients with PASI-50 and virtually all patients with PASI-75 had a substantial treatment benefit, defined by a PBI≥1. This implies that a quarter of the PASI-50 patients subjectively experienced no personally relevant treatment benefits.
Table 1 Characteristics of study population, PASI and patient reported outcomes t1 (before therapy) and t2 (after therapy)

t1

t2

Patients

n = 93

n = 80

Women

37.6%

38.8%

Age

49.3 (14.1)

50.1 (14.6)

PASI (range 0-72)

13.7 (9.5)

5.5 (5.8)

VAS EQ 5-d (range 0-100)

54.4 (26.3)

69.0 (22.7)

DLQI (range 0-30)

9.7 (6.5)

5.6 (5.2)

Duration of therapy (t1-t2)

-

42.5 (17.0)

PBI (range 0-4)

-

2.3 (1.3)

PBI ≥ 1

-

76.3%

PASI < 50 (%)

-

27.5

PASI-50 (%)

-

40.0

PASI-75 (%)

-

32.5

∆PASI (% diff. t1-t2)

-

62.0 (24.9)


Table 2 Distribution of answers to global questions on therapy satisfaction (n = 80 patients)

Expectations met by therapy

n

%

completely

18

22.5

predominantly

31

38.8

somewhat

25

31.3

not at all

6

7.7

Achieved conditions of the skin is sufficient

completely

5

6.3

predominantly

39

48.8

rather not

23

28.8

definitely not

13

16.3


Table 3 Cross-sectional correlations between PASI and patient-reported outcomes at t1 and t2

t1

t2

PASI

PASI

n

R

p

n

R

p

DLQI

80

0.24

0.03

80

0.36

0.00

Health-scale EQ-5D

80

-0.30

0.02

77

-0.31

0.01

PBI

-

-

-

80

0.41

0.00


Table 4 Correlations of improvement in PASI-Score from t1 to t2 (∆ PASI) with change in patient-reported outcomes

∆ PASI

n

R

p

∆ DLQI

73

0.50

0.00

PBI

80

0.45

0.00

∆ EQ-5D Health-scale

74

0.10

0.48


Table 5 PASI response groups by patient defined outcomes (% from PASI-group)

∆ PASI

< PASI-50

PASI-50

PASI-75

Expectations met completely/predominantly

36.4

53.1

92.3

Expectations met somewhat/not at all

63.6

46.9

7.7

Achieved conditions of the skin: completely/predominantly sufficient

36.4

43.8

84.6

Achieved conditions of the skin: rather not/definitely not sufficient:

63.6

56.3

15.4

DLQI < 2

18.2

18.8

34.6

DLQI ≥ 2

81.8

81.3

65.4

DLQI < 5

86.4

62.5

42.3

DLQI ≥ 5

13.6

37.5

57.7

PBI ≥1

54.5

75.0

96.2

PBI <1

45.5

25.0

3.8

Discussion

PASI-50 and PASI-75 are internationally the most important parameters for assessment of the severity of psoriasis. Shortcomings in their validity have previously been found in terms of inter-rater variability, construct and external validity [24] as well as with regard to their significance for treatment concepts [25]. This prospective study with ambulant psoriasis patients indicates a correlation between the objective clinical severity parameter PASI on the one hand, as well as the disease-specific Quality of Life and the patient-defined benefits in the psoriasis treatment on the other. There was no association with the general state of health. However, the correlations between PASI and the patient-relevant outcomes Quality of Life and treatment benefits were, on average, only mildly correlated. The extent of the changes in these items over the period of treatment indicates a somewhat more significant, but moderately high correlation with the PASI.

The parameters PASI-50 and PASI-75, established as threshold values of psoriasis-severity measurement, were likewise positively associated with patient-defined outcomes, but they reflected them incompletely. Thus, about half of the patients with PASI-50 did not see their expectations of the treatment met, or were not satisfied with the condition of their skin after treatment. A quarter of these patients experienced no benefits from the therapy. Of the patients with PASI-75, 15% were not satisfied with the condition of their skin, and a clinically relevant restriction in Quality of Life due to the psoriasis still existed for more than half of them. In contrast, a relevant percentage in each case of patients who did not attain PASI-75 or PASI-50 reported a high level of satisfaction or a subjectively significant treatment benefit.

The relatively short average observation time (42.5 days) in this study was chosen intentionally in order to give more variance in the outcome parameters. The idea was that after a longer observation period under routine conditions, too many patients might have reached PASI 75 and only a few would have remained in a moderate-to-poor skin condition. As intended, a fair distribution of delta PASI has resulted (table 1). With this approach, however, we cannot rule out that after a longer treatment period a response shift may have occurred.

Limitations in terms of the interpretation of the results might have arisen from the small sample size of this pilot study. Thus, stratification, for example by current therapies, was not possible. Moreover, only a few cases were found in several subgroups, particularly in the bottom area of the scale of the general questions for treatment and therapy satisfaction. For this reason, further differentiation of the PASI grouping, such as the representation of PASI-90, could not be carried out. Response statistics could not be conducted during the recruitment of patients which was carried out consecutively over a definite time period. Moreover, there was no available information about non-participants, so that a selection bias (internal validity) or a restriction of the generalizability of results (external validity), due to selective participation of patient groups, could not be excluded.

As desired, there was in fact a fair distribution of delta PASI (table 1). As a reference for the evaluation of results, the national psoriasis study PsoHealth [26] can be applied. In that survey, representative data on the health-care situation of n = 2,009 psoriasis patients were collected within a network of 130 dermatological practices and outpatient clinics. The age and sex structure of this extensive sample conforms largely to the age and sex distribution of our study. In PsoHealth, average PASI and DLQI lie in-between the determined values for t1 and t2 (PsoHealth: PASI = 10.1; DLQI = 7.5; health scale EQ-5D = 64.5, PBI = 2.5). In this respect, this study conforms to expectations, since PsoHealth was a cross-sectional study with patients in different stages of treatment, whereas the data presented here were collected before and at the end of a therapy. The comparison of these results with the extensive study supports the assumption that adequate validity is given for this pilot-study.

The findings for the correlation of PASI and patient-reported outcomes are verified by the PsoHealth data, which also show moderate to weak associations: the correlation coefficient of PASI and DLQI was R = 0.39, R = – 0.24 for PASI and PBI and R = – 0.34 for PASI and EQ-5D in PsoHealth. Other longitudinal studies have resulted in similarly low associations between ∆PASI and ∆DLQI (R = 0.4) [16] such as in the study presented here (R = 0.50). Effects were similar when the two parameters, Body Surface Area (BSA) as clinical measurement, and Willingness to Pay, as patient-reported outcome, were used instead of the PASI [27], where no relevant correlation appeared for these two parameters. Studies on other dermatological diagnoses point out that the moderate to weak correlation between the clinical measures of disease severity and patient-relevant outcomes exists not only for psoriasis, but also for atopic dermatitis [28] and acne [29].

The finding, that the correlation between PASI and disease-specific Quality of Life (DLQI), was stronger than that between PASI and general state of health (EQ-5D), conforms to expectations and is identical to the results of Revicki et al. [17] and other studies [30]. Clinical severity of psoriasis, disease-specific Quality of Life, as well as patient-reported treatment-needs and -benefits are parameters that are associated with each other, but are based on differing concepts and reflect specific aspects of treatment outcomes. In each case, they should be considered in the evaluation of psoriasis care. The results of this pilot study confirm the need and the feasibility of further research. For this purpose, a comprehensive multi-center study on ambulant and hospital-patients is projected.

Acknowledgements

No financial support was given for this study. The authors declare no conflict of interest.

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