Patient-preference disability assessment for disabling knee osteoarthritis: Validity and responsiveness of the McMaster-Toronto Arthritis Patient Preference Disability Questionnaire

Patient-preference disability assessment for disabling knee osteoarthritis: Validity and responsiveness of the McMaster-Toronto Arthritis Patient Preference Disability Questionnaire

Annals of Physical and Rehabilitation Medicine 59 (2016) 255–262 Available online at ScienceDirect www.sciencedirect.com Original article Patient-...

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Annals of Physical and Rehabilitation Medicine 59 (2016) 255–262

Available online at

ScienceDirect www.sciencedirect.com

Original article

Patient-preference disability assessment for disabling knee osteoarthritis: Validity and responsiveness of the McMaster-Toronto Arthritis Patient Preference Disability Questionnaire Katherine Sanchez a,b,c,d,*, Cle´mence Palazzo a,b,c,d, Ce´cile Escalas a,b,c,d, Franc¸ois Rannou a,b,c,d, Marie-Martine Lefe`vre-Colau a,b,c,d, Xavier Ayral e, Johann Beaudreuil f, Serge Poiraudeau a,b,c,d a

Inserm U1153, e´pide´miologie clinique des maladies oste´o-articulaires, 27, rue de Faubourg-Saint-Jacques, 75014 Paris, France Universite´ Paris Descartes, PRES Sorbonne Paris Cite´, 75004 Paris, France c Service de re´e´ducation et re´adaptation de l’appareil locomoteur et des pathologies du rachis, hoˆpital Cochin, AP–HP, 27, rue de Faubourg-Saint-Jacques, 75014 Paris, France d Institut fe´de´ratif de recherche sur le handicap, Institut national de la sante´ et de la recherche me´dicale, groupe hospitalier Pitie´-Salpeˆtrie`re, Secre´tariat ge´ne´ral, baˆtiment Pinel, 47, boulevard de l’Hoˆpital, 75651 Paris, France e Service de rhumatologie, hoˆpital Cochin, AP–HP, 27, rue de Faubourg-Saint-Jacques, 75014 Paris, France f Service de rhumatologie, hoˆpital Lariboisie`re, AP–HP, 2, rue Ambroise-Pare´, 75010 Paris, France b

A R T I C L E I N F O

A B S T R A C T

Article history: Received 18 February 2016 Accepted 31 May 2016

Background: The McMaster-Toronto Arthritis Patient Preference Disability Questionnaire (MACTAR) measurement of function may be more comprehensive and add useful information about disability than traditional fixed-item questionnaires, especially about issues that really matter to the patient, for developing personalized medicine. Objectives: We aimed to assess priorities in disability and restriction in participation in patients with disabling knee osteoarthritis (OA) by the MACTAR and evaluate its validity and responsiveness. Methods: We evaluated 127 in- and outpatients with knee OA in two tertiary care teaching hospitals between August 2010 and July 2012 by using the MACTAR, the Western Ontario and McMaster Universities Osteoarthritis Index, Lequesne scale, Fear Avoidance Beliefs Questionnaire, a life satisfaction score and pain, global assessment of disease activity and functional impairment scores on a numerical rating scale. Validity was assessed by Pearson correlation and responsiveness by the standardized response mean (SRM) and effect size (ES). Results: Patients ranked 35 different activities by the MACTAR; the 3 domains of the International Classification of Functioning, Disability and Health most often identified were mobility (cited 233 times, 52.3%); community, social and civic life (cited 122 times, 27.4%); and domestic life (cited 64 times, 14.4%). The MACTAR score was best correlated with functional impairment (r = 0.5). Convergent and divergent validity was as expected. In all, 108 patients completed a 6-month follow-up evaluation: 27 patients shifted their priorities at 6 months, for a decrease in SRM and ES. The SRM (0.64) and ES (0.92) for the MACTAR without shifts in priorities were the highest among the outcome measures tested; for patients considering their condition improved, the values were 0.85 and 1.17, respectively. Conclusions: For assessing priorities in disability and restriction in participation among patients with knee OA, the MACTAR has acceptable validity and responsiveness. ß 2016 Elsevier Masson SAS. All rights reserved.

Keywords: Knee Osteoarthritis Handicap Disability assessment Validity Responsiveness McMaster Toronto Arthritis Patient Preference Disability Questionnaire

* Corresponding author. Service de re´e´ducation orthope´dique de l’enfant, hoˆpitaux de Saint-Maurice, 12/14, rue de Val-d’Osne, 94410 Saint-Maurice, France. Tel.: +33143966975; fax: +33143966625. E-mail addresses: [email protected] (K. Sanchez), [email protected] (C. Palazzo), [email protected] (C. Escalas), [email protected] (F. Rannou), [email protected] (M.-M. Lefe`vre-Colau), [email protected] (X. Ayral), [email protected] (J. Beaudreuil), [email protected] (S. Poiraudeau). http://dx.doi.org/10.1016/j.rehab.2016.05.003 1877-0657/ß 2016 Elsevier Masson SAS. All rights reserved.

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1. Introduction Rheumatic and musculoskeletal disorders are a major cause of disability worldwide. The number of years lived with disability due to knee and hip OA increased by 64% between 1990 and 2010, and OA is ranked 11th in the list of leading causes of years lived with disability [1]. In France, OA ranks first, followed by low back pain (LBP) for patient-perceived disability [2]. Accurately evaluating outcomes of treatments in patients with OA is a key issue in daily practice and clinical research. The Outcome measures in rheumatology clinical trials (OMERACT) group proposed a core set of outcome dimensions for phase 3 trials of knee and hip OA; 3 domains should be systematically included: pain, physical function and patient global assessment [3]. Disability and participation restriction, also called handicap, are negative aspects of functioning and are widely assessed in knee OA by many validated outcomes. The instruments most commonly used are the Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) [4,5], the Lequesne index [6,7] and more recently, the Intermittent and Constant Osteoarthritis Pain (ICOAP) [8] and the Knee disability and Osteoarthritis Outcome Score – Physical Function Short form (KOOS-PS) [9]. However, these tools do not take into account patient priorities. Previous research found that patients with rheumatoid arthritis (RA), healthy professionals, and healthy controls do not agree on the importance of disabilities [10]. Using a needs-based approach and accounting for patient priorities may help better understand what is important for patients and increase the content validity of scales assessing disability [11]. One functional scale that investigates patient priorities is the McMaster-Toronto Arthritis Patient Preference Disability Questionnaire (MACTAR) [12]. Its developers noted good responsiveness for patients with RA in a controlled trial that revealed a clinically important change, and the scale was found to have validity in a multicenter randomised trial of RA [13]. The MACTAR concept of function may be more comprehensive than that of traditional fixed-item questionnaires and may reveal issues that really matter to the patient. Thus, the MACTAR seems to be a better appropriate tool to develop a real personalized medicine. Some recent studies evaluating patient priorities in disability in knee and hip OA, chronic LBP and systemic sclerosis (SSc) suggested that the MACTAR adds useful information about disability [14–16]. In addition, the MACTAR seems to be a quick tool to complete. We aimed to assess priorities in disability and restriction in participation for patients with disabling knee OA by the MACTAR and evaluate the instrument’s validity and responsiveness in such patients.

2. Methods 2.1. Study design We asked 200 in- and outpatients admitted to the physical medicine and rehabilitation and rheumatology departments at Cochin and Lariboisie`re university hospitals in Paris for intensification of treatments of their knee OA between August 2010 and July 2012. The inclusion criteria were knee pain due to OA with pain duration of at least 3 months. The exclusion criteria were age < 35 years, etiology other than OA, inability to understand French or complete a self-administered written questionnaire, and uncontrolled mental disease. Patients had to complete self-administered questionnaires, undergo a 15min interview with a physician to check for unanswered

questions and gather clinical data. Six months later, they received the same questionnaire by mail for completion. This delay corresponds to the time used in daily practice and studies evaluating the effect of pharmacological and non-pharmacological treatment in knee OA. 2.2. Demographic and clinical variables Variables recorded at baseline were age, sex, knee pain duration, body mass index, Kellgren and Laurence (KL) radiologic score, educational level (baccalaureate degree or lower, higher than university degree), professional status, previous meniscectomy, pharmacological and non-pharmacological treatment. 2.3. Patient-reported outcome measures Patient priorities in disability were assessed by the MACTAR, developed to evaluate functional priorities in patients with RA [12]. We used the French version [15] and questions were adapted for knee pain (File S1). Patients were first asked about activities affected by chronic knee pain, then asked to rank these activities in order of importance by answering ‘‘Which of these activities would you consider most important to be able to do with minimal pain and difficulty?’’ We used a 3-item priority function. Each item is scored on an 11-point semiquantitative Likert scale (0–10), the global score ranging from 0 (no disability) to 30 (maximal disability) [13]. At follow-up, patients were reminded of the 3 baseline priorities they had identified and were asked to score them (0– 10). To assess possible shifts in priorities, participants were asked to define and score on a scale from 1 to 3 other activities that may have become more important to them since the baseline visit. So at 6 months, patients had 2 MACTAR scores, one maintaining baseline activities and another considering shifts in activity priorities. We classified the activities by the domains of the International Classification of Functioning, Disability, and Health (ICF) [17], considering the linking rules given by the World Health Assembly, in May 2001 [18]. The WOMAC is a 3-D measure. It contains 5 items related to pain, 2 to stiffness, and 17 to physical function [4]. The function subscale is widely used in clinical trials of hip and knee OA [5,19]. We used the short form of the function subscale, containing 8 questions, with scores ranging from 0 to 100 (worse status) and validated in knee and hip OA in French [20]. The Lequesne index is a composite French scale used to assess the concept of algofunctional disability induced by knee OA [6,7]. It includes 11 questions about pain, discomfort and function. The scores range from 0 to 24 (maximum pain and disability) [21]. Its responsiveness and construct validity have been assessed in French [22,23]. A numerical rating scale (NRS) was used to evaluate pain [24,25], global assessment of disease activity and function [20]. The NRS contained 11 points, with scores ranging from 0 to 10 (high level of symptoms). The Fear-Avoidance Beliefs Questionnaire for physical activity (FABQ-PA) was originally developed for LBP [26], and van Baar et al. [27] used it for patients with knee abnormalities. It consists of 4 items; each scored from 0 to 6. Higher scores represent greater fear-avoidance beliefs. The scale has adequate internal consistency in patients with knee OA [28]. Anxiety and depression were assessed by the Hospital Anxiety and Depression scale (HADa, for anxiety, and HADd, for depression) [29]. This scale has 7 questions for anxiety and 7 for depression. Each question is answered on a scale from 0 to 3. The total score ranges from 0 to 21 (maximal depression, maximal anxiety).

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Life satisfaction was assessed by the Satisfaction with Life Scale (SWLS), a 1-D structure [30] that consists of 5 items, each rated on a 7-point Likert scale ranging from 1 (completely disagree) to 7 (completely agree); the total scores range from 5 to 35. The SWLS has been validated in a Canadian French population [31]. At 6 months, patients were evaluated for the evolution of their status related to knee pain on a 6-point Likert scale, from aggravation to disappearance.

the change in MACTAR scores for patients considering their condition improved [37]. In most cases, missing data were < 10% and we used mean imputation to account for missing data. P < 0.05 was considered statistically significant.

2.4. Ethical statements

3.1. Demographic and clinical data

This survey was conducted in compliance with the protocol Good Clinical Practices and Declaration of Helsinki principles. In accordance with French national law in 2010, formal approval from an ethics committee was not required for non-interventional studies; patients gave their written consent to participate after being informed about the purpose of study.

Of the 200 patients at baseline, 60 patients did not meet inclusion criteria and 13 patients declined participation, so we had evaluable data for 127 patients (79 females, 62.2%) at baseline. In all, 108 patients (70 females, 65%) completed and returned the questionnaire at 6-month follow-up (2 declined participation, 10 returned incomplete surveys and 7 could not be reached) (Fig. 1). Patients lost to follow-up were younger than the other 108 participants and most were males, had less pain duration, were still working and had less than a baccalaureate level of education (Table 1). The mean baseline age for patients with knee OA, evaluated 2 times, was 65.3  10.9 [range: 36–92] years, mean pain duration

2.5. Statistical analysis The simple size was not less than 10 times the number of items [32] and we also had as a reference the sample size used in other validation studies of the MACTAR [14–16]. Analyses involved use of R 3.0.2 (R Foundation for Statistical Computing, Vienna, Austria). Quantitative variables are described with mean  SD and ranges. Qualitative variables are described with number (%). We analyzed priority items defined by the MACTAR by linking with the ICF domains. Correlation of the MACTAR score with other scores was assessed by the parametric Pearson rank coefficient (r), because all variables showed a normal distribution. Pearson correlation was interpreted as excellent (> 0.91), good (0.90–0.71), moderate (0.70–0.51), fair (0.50–0.31), and little or absent (< 0.30) [33]. Focused Principal Component Analysis was used to compare the MACTAR and the other measures. The paired P-values were added when we evaluated the difference between 2 measures on the same subjects at baseline and 6-month follow-up; they allowed for eliminating much of the inter-individual variability and improving the power of a test. Responsiveness may be considered an aspect of validity [34] and describes a scale’s ability to detect change over time that is clinically meaningful. Among different statistical approaches [35], we used the standardized response mean (SRM) and the effect size (ES). A high SRM or high ES indicates greater responsiveness; a negative value indicates that the mean score at baseline is smaller than the mean score at follow-up. The SRM and ES are considered small if < 0.2, moderate if near 0.5, and large if > 0.8 [36]. SRM values were also calculated for subgroups of patients who considered their condition improved (overall opinion of condition at 6-month follow-up considered disappeared, improved or slightly improved), their health status maintained (overall opinion of condition at follow-up considered identical) and their condition deteriorated (overall opinion of condition at follow-up considered worse). Then, these 3 groups were recoded into 2 groups, considering actual health status improved or deteriorated (overall opinion at follow-up considered identical or worse, respectively). The Student t-test was used to compare changes in scores in these 2 last groups of patients; validity conditions were previously verified. Stepwise logistic regression analysis was used to determine variables associated with health status at 6 months. Explanatory variables were introduced in the stepwise regression if on univariate analysis significant differences in scores were found between patients who considered their health condition improved and those deteriorated. The minimal clinically detectable improvement (MCDI) in condition was calculated for the MACTAR as the 75th percentile of

3. Results

Invited to participate (n=200) Excluded (n=73) Not meet inclusion criteria (n=60) Declined to participate (n=13) Agreed to participate at baseline (n=127)

Completed questionnaires (n=127)

6-month follow-up, invited to continue in the study (n=127) Excluded (n=9) Not reachable (n=7) Declined to participate (n=2)

Agreed to participate (n=118) Incomplete questionnaires* (n=10) Completed questionnaires (n=108) Fig. 1. Flow of patients in the study. *Questionnaires evaluating priorities in disability and restriction in participation among patients with knee OA.

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Table 1 Sociodemographic and clinical characteristics of 127 patients with disabling knee osteoarthritis (OA) at baseline visit.

Age, years, mean  SD [range] Female sex Pain duration, years, mean  SD [range] Pain, NRS (0–10), mean  SD [range] BMI, kg/m2, mean  SD [range] Kellgren & Laurence grade 2 3 4 Hypertension Diabetes mellitus Other OA Educational level < Baccalaureate level Baccalaureate level Higher university degree Professional status Employed Sick leave Retirement Unemployed Previous meniscectomy Treatment Non-steroidal anti-inflammatory drugs Analgesics Steroid injection Hyaluronic acid injection Physiotherapy Knee lavage Alternative medicine Diet Orthopaedic insole Walking stick Knee pad

Patients with knee OA n = 108

Patients lost to follow-up n = 19

65.3  10.9 [36–92] 70 (65) 6.7  7.8 [0.41–43]

59.3  9.2 [44–75] 9 (47) 5.4  5.9 [0.25–20]

5.8  2.2 [1–10]

6.7  2.0 [3–10]

27.8  4.6 [17.3–39.9]

29.1  5.4 [20.3–41]

6 (6) 47 (47) 48 (48) 52 (48) 14 (13) 62 (57)

2 (11) 6 (33) 10 (56) 7 (37) 3 (16) 7 (37)

41 (38) 14 (13) 53 (49)

14 (74) 1 (5) 4 (21)

27 (25) 11 (10.2) 68 (63) 2 (1.8) 31 (29)

8 3 7 1 3

75 (70)

13 (76)

95 (88) 67 (63) 53 (49) 61 (56) 27 (25) 9 (8) 30 (28) 35 (32) 19 (18) 32 (30)

16 (89) 12 (67) 6 (33) 7 (39) 6 (33) 1 (6) 3 (17) 7 (39) 4 (22) 6 (33)

(42.1) (15.8) (36.8) (5.3) (16)

Data are no. (%) of patients with available data, unless indicated. BMI: body mass index; NRS: numeric-rating scale.

6.7  7.8 [0.41–43] years and pain NRS score 5.8  2.2 [1–10]. For almost half of the patients (n = 48, 48%), the KL radiologic grade was 4. 3.2. Priority disabilities The priority activities differed among participants. At baseline, 7 (5.5%) patients cited only 2 activities instead of 3. Overall, patients cited 35 activities, 1 function and 4 environmental factors of the ICF (Table S1). Activities cited belonged to 6 domains: mobility (18 activities, cited 233 times, 52.3%); community, social and civic life (4 activities, cited 122 times, 27.4%) domestic life (6 activities, cited 64 times, 14.4%); major life areas (1 activity, cited 9 times, 2%); interpersonal interactions and relationships (4 activities, cited 8 times, 1.8%); and self-care (2 activities, cited 6 times, 1.4%). One function, sensory functions and pain, was cited 3 times (0.7%), and the 4 environmental factors cited included services, systems and polices (transportation services). The 5 activities most-often cited were sports (cited 61 times, 13.7%), unspecified recreation and leisure (cited 50 times, 11.2%), unspecified mobility (cited 43 times, 9.7%), unspecified walking (cited 37 times, 8.3%) and moving around outside the home and other buildings (cited 25 times, 5.6%). At 6-month follow-up, 81 patients (75%) maintained the same priorities as at baseline. For 27 patients (25%), at least 1 of the 3 priorities had changed: for 16, 1 priority had changed; for 9, 2 priorities had changed and for 2, all priorities had changed.

At 6-month evaluation, the shift in priorities did not modify the order of domains when considering the 3 priorities cited. Two body functions and 4 environmental factors were identified (Table S2). Body functions were mental function (sleep), sensory functions and pain (knee pain); environmental factors included products and technology (walk without a stick). The 5 activities most-often cited at baseline were maintained at 6 months. For the 27 patients who shifted at least 1 priority, the order of domains remained the same; nevertheless, self-care was not cited, and a new function was added (Table S3). Concerning activities, their frequency changed and the first activity cited was climbing stairs. 3.3. Outcome measure scores 3.3.1. Correlation of the MACTAR score with other scores The MACTAR total score at baseline was best correlated with the NRS score for functional impairment (r = 0.5) (Table 2, Fig. 2). Other correlations with MACTAR score were the WOMAC function subscale score (r = 0.4), Lequesne index (r = 0.3) and global assessment score (r = 0.4). All correlations were statistically significant (P < 0.05) and only life satisfaction score was negatively correlated with the MACTAR score. 3.3.2. Comparison between patients maintaining baseline priorities and those shifting priorities At 6-month evaluation, the mean MACTAR score was 14.9  8.2 [range: 0–29] when considering the same priorities defined at baseline but 16.9  7.9 [0–30] when considering shifts in priorities (Table 3). For the 27 patients who shifted at least one priority, the mean MACTAR score was 19.4  5.6 [4.3–30] at baseline (Table 4), and the score was 10.1  6.3 [0–24.1] at 6-month follow-up when considering priorities defined at baseline and 18.2  6.9 [2.9– 30] when considering shifts in priorities. Patients who shifted priorities and those who maintained their baseline choice did not differ in baseline characteristics; nevertheless, the change in MACTAR score between baseline and followup was greater for patients shifting than not shifting priorities, as was the frequency of considering their health status improved (19 [66.7%] vs 47 [58%]; Table 4). 3.3.3. Sensitivity to change Among all measures tested, the MACTAR score, when not considering shifts in priority, had the highest values for SRM (0.64) and ES (0.92) (Table 3). The other measures had moderate responsiveness: MACTAR considering shifts (0.39 and 0.55, respectively), functional impairment (0.48 and 0.68, respectively),

Table 2 Correlationa of scores of other measures with baseline McMaster-Toronto Arthritis Patient Preference Disability Questionnaire (MACTAR) global score. Measure (score range)

MACTAR global score

[95% CI]

P-value

WOMAC function (0–100) Pain, NRS (0–10) Global assessment, NRS (0–10) Functional impairment, NRS (0–10) Lequesne (0–24) HADa (0–21) HADd (0–21) FABQ-PA (0–24) Life satisfaction (5–35)

0.4 0.4 0.4 0.5 0.3 0.2 0.3 0.1 0.3

[0.2–0.5] [0.2–0.5] [0.2–0.5] [0.3–0.6] [0.1–0.5] [0.03–0.4] [0.2–0.5] [ 0.1–0.3] [ 0.5–0.1]

< 0.01 < 0.01 < 0.01 < 0.01 < 0.01 0.02 < 0.01 0.3 < 0.01

FABQ-PA: Fear-Avoidance Beliefs Questionnaire for physical activity; HADa: Hospital Anxiety and Depression Scale for anxiety; HADd: HAD for depression; NRS: numerical rating scale; WOMAC: Western Ontario and McMaster Universities Osteoarthritis Index; 95% CI: 95% confidence interval. a Pearson correlation coefficient (r).

K. Sanchez et al. / Annals of Physical and Rehabilitation Medicine 59 (2016) 255–262

between means of individual changes in the 2 groups with shifts in priorities). The MCDI value for the MACTAR score was affected by shifts in priorities: it was 12 with baseline priorities retained and 10.1 with priorities shifted (Table S4).

r=0

r = 0.2

r = 0.4

Life_Satisfaction r = 0.6

FABQ P GA FI

Lequesne Womac_function

259

r = 0.8

MACTAR

3.3.4. Predictors of improved health status at 6 months On univariate analysis, scores for all scales except the HAD for depression were associated with evolution of health status (improved or deteriorated) at 6-month follow-up. However, on stepwise logistic regression, 3 variables were associated with patients’ opinion of their health status: changes in WOMAC function subscale score (odds ratio [OR]: 1.09, 95% CI: 1.05–1.14), MACTAR score retaining baseline priorities at 6 months (OR: 1.09; 95% CI: 1.01–1.18) and life satisfaction score (OR: 1.3; 95% CI: 1.1– 1.5). 4. Discussion

Depression Anxiety

Fig. 2. Focused principal components analysis: correlations between scores for McMaster-Toronto Arthritis Patient Preference Disability Questionnaire (MACTAR) and explanatory variables (FI: functional impairment; GA: global assessment; P: pain, life satisfaction, WOMAC function, Lequesne, depression, anxiety and FABQ). Green variables are positively correlated with the MACTAR score and yellow variables are negatively correlated. r: Pearson rank coefficient; WOMAC: Western Ontario and McMaster Universities Osteoarthritis Index; FABQ: fear-avoidance beliefs questionnaire.

WOMAC function (0.42 and 0.48, respectively) and FABQ-PA (0.43 and 0.55, respectively). Table 5 summarizes individual changes in disability scores for patients who considered their condition improved (65 patients), identical (22 patients) and deteriorated (21 patients) at 6 months. The MACTAR scale, whether considering baseline priorities retained or shifted, discriminated well between patients who considered their condition improved or deteriorated (SRM = 0.85 and 0.11, respectively; 95% confidence interval [95% CI] 8.0, 2.5 between means of individual changes in the 2 groups with same priorities defined at baseline and SRM = 0.60 and 0.17, respectively; 95% CI: 7.9, 2.7

This study suggests that the MACTAR scale has acceptable validity and is as responsive to change as other outcome measures widely used for knee OA. However, patients could shift priorities in disabling activities over time, which has implications for the responsiveness of the measure. The MACTAR questionnaire identified activities and body functions according to the ICF classification but also environmental factors. These environmental factors are usually not captured by fixed-item tools. All items of the WOMAC function subscale and the Lequesne Index, except for morning stiffness, were identified by the MACTAR, whereas as much as 69% of the activities captured by the MACTAR were not represented in the WOMAC or the Lequesne scales. This finding is particularly true for domains such as domestic life, interpersonal interactions and relationships, major life areas, and community, social and civic life. Our results agree with those by Barten et al. [14], who studied a mixed cohort of hip and knee OA. The 2 domains most often cited in the previous work were mobility, and community, social and civic life. Overall, for patients with knee and hip OA [14], chronic LBP [16] and SSc [38], the first impaired domain of activities classified by the ICF is mobility. The MACTAR score was poorly correlated with the WOMAC function subscale and Lequesne scores, which suggests that these instruments are not redundant and that disability priorities are not well-reflected by global disability assessed with predetermined items tools. This weak correlation is not surprising, because many

Table 3 Scores for pain, disability, handicap, fear-avoidance beliefs, anxiety and depression for 108 patients with chronic knee OA at baseline and 6-month follow-up, differences in scores and sensitivity to change.

MACTAR (0–30)a MACTAR (0–30)b WOMAC function (0–100) Pain, NRS (0–10) Global assessment, NRS (0–10) Functional impairment, NRS (0–10) Lequesne (0–24) HADa (0–21) HADd (0–21) FABQ-PA (0–24) Life satisfaction (5–35)

Baseline

6 months

Difference

Mean  SD [range]

Mean  SD [range]

Mean  SD [range]

SRM/ES

19.9  5.5 [0.8–30] 19.9  5.5 [0.8–30] 50.4  18 [3.1–84.4] 5.8  2.2 [1–10] 6.4  2 [1–10] 6.6  1.9 [1–10] 10.7  4 [1–24] 9.1  3.8 [2–17] 6.6  3.5 [0–16] 17.1  5.4 [3–24] 22.8  6.2 [6–35]

14.9  8.2 [0–29] 16.9  7.9 [0–30] 41.8  21.8 [0–87.5] 5  2.6 [0–10] 5.6  2.7 [0–10] 5.3  2.6 [0–10] 9.4  4.8 [0–22.5] 8.2  3.8 [1–21] 6.1  3.4 [0–16] 14.1  6.6 [0–24] 21.6  5.4 [7–32]

5.1  7.9 [ 15.2–30]* 3  7.7 [ 15.2–30]* 8.6  20.4 [ 43.8–59.4]* 0.8  2.7 [0,5–10]* 0.8  2.7 [0,5–10]* 1.3  2.7 [0,8–10]* 1.2  4.8 [ 11.5–18.5]* 0.9  3.1 [–9–8]* 0.5  2.9 [–7]* 3  6.9 [0,17–20]* 1.3  4.6 [0,15]*

0.64/0.92 0.39/0.55 0.42/0.48 0.31/0.38 0.30/0.41 0.48/0.68 0.26/0.31 0.29/0.23 0.17/0.14 0.43/0.55 0.28/0.21

ES: effect size; SRM: standardized response mean. a Considering priorities defined at baseline and retained at 6 months. b Considering shifts in priorities at 6 months. * P < 0.05 comparing baseline and 6-month follow-up.

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Table 4 Scores for pain, disability, handicap, fear-avoidance beliefs, anxiety and depression for 108 patients with knee OA at baseline, differences at follow-up and patients’ actual evaluation of health by MACTAR score considering or not considering shifting priorities in activities of disability.

Baseline values MACTAR WOMAC function Pain, NRS Global assessment, NRS Functional impairment, NRS Lequesne HADa HADd FABQ-PA Life satisfaction Differences: baseline vs. 6-month evaluation MACTARa MACTARb WOMAC function Pain, NRS Global assessment, NRS Functional impairment, NRS Lequesne HADa HADd FABQ-PA Life satisfaction Actual health status, n (%) Improved Deteriorated

Patients with priority shift, n = 27

Patients without priority shift, n = 81

Mean  SD [range]

Mean  SD [range]

19.4  5.6 [4.3–30] 49.5  16.3 [3.1–71.9] 6.2  2 [2–10] 6.7  1.7 [2.5–10] 6.9  1.6 [3–10] 12.2  4.7 [1–24] 8.7  4.4 [2–17] 7.4  4 [2–16] 17.3  6 [3–24] 21.9  7 [9–34]

20.1  5.6 [0.8–30] 50.7  18.7 [3.1–84.4] 5.7  2.3 [1–10] 6.3  2.1 [1–10] 6.6  2 [1–10] 10.2  3.6 [1–18] 9.2  3.6 [3–17] 6.4  3.3 [0–15] 17  5.2 [6–24] 23.2  6 [6–35]

9.3  6.8 [ 1.1–21.4]* 1.2  7.3 [ 15–15.2] 3.5  19.5 [ 37.5–32.6] 0.7  2.7 [0,5,6] 0.4  2.1 [0.4–6] 0.8  2.2 [0.4–6] 1.8  5.6 [ 8–18.5] 1.4  3.1 [0.5–8]* 0.9  3.2 [0.7] 3.3  8.8 [0.17–19] 1  3.6 [ 11–6]

3.7  7.8 [ 15.2–30]* NA 10.3  20.6 [ 43.8–59.4]* 0.9  2.7 [0.5–10]* 1  2.9 [0.5–10]* 1.5  2.9 [0.8–10]* 1.1  4.5 [ 11.5–14]* 0.7  3 [ 9–8]* 0.4  2.8 [ 7] 2.9  6.1 [0.12–20]* 1.4  4.9 [0.15]*

18 (66.7) 9 (33.3)

47 (58) 34 (42)

NA: not applicable. a Considering priorities defined at baseline and retained at 6 months. b Considering shifts in priorities at 6 months. * P < 0.05 comparing baseline and 6-month follow-up for patients with and without priority shifts.

of the 35 activities identified in the MACTAR are not represented among the activities assessed by the WOMAC function subscale or Lequesne index. A poor correlation between MACTAR and WOMAC scores was also reported in a Dutch cohort [14]. The correlation between functional impairment scores, globally assessed by NRS, and MACTAR scores was higher than that between the MACTAR score and WOMAC or Lequesne score. This correlation was observed in chronic LBP [16], which suggests that global rating of disability probably reflects patient priorities better than do fixed-items tools. As expected, we found weak or fair correlations between the MACTAR score and other tool scores assessing concepts different from disability. Sensitivity to change of the MACTAR at follow-up when reassessing priorities selected at baseline was higher than that observed for functional impairment (NRS), the WOMAC subscale

score and Lequesne index, for which good sensitivity to change was previously reported [20,22,23]. Therefore, the MACTAR may be a responsive outcome measure. Moreover, the SRM and ES for the MACTAR score, considering or not changes in priorities, was increased in patients who felt better, did not change for patients who felt unchanged and was worse for patients who felt worse, which characterizes a clinically meaningful sensitivity to change. In all, 25% of patients shifted their priorities at follow-up. Although this shift did not modify the profile of ICF domains for disability covered, except self-care, the specific activities and their frequencies in domains were changed. Therefore, despite having a chronic pain condition, some patients changed their expectations over time, and one advantage of using the MACTAR is that it probably captures well what is most important to the patient at one moment in time. Two-thirds of priorities were new at 1-year

Table 5 Changes in MACTAR scores for patients with knee OA who considered their condition improved, not changed or deteriorated at 6-month follow-up.

MACTARa MACTARb WOMAC Lequesne Functional impairment, NRS

Condition improved

Condition not changed

Condition deteriorated

n = 65

n = 22

n = 21

Mean  SD [range]

SRM/ES

Mean  SD [range]

7.2  8.5 [ 15.2–30] 5.1  8.5 [ 15.2–30] 17.6  17.3 [ 18.8–59.4] 2.6  5 [ 11.5–18.5] 2.1  3 [0,8–10]

0.85/1.17 0.60/0.84 1.02/0.98 0.51/0.62 0.70/1.02

3.1  5.8 [ 4–17.6] 0.41  5.4 [ 15–9.6] 4.4  14.6 [ 31.3–21.6] 0.05  3.7 [ 3.5–7.5] 0.4  2.1 [0,4,5]

SRM/ES 0.54/0.75 0.08/0.10 0.30/ 0.24 0.02/0.01 0.41/0.23

Mean  SD [range] 0.7  5.8 [ 6.9–16] 0.7  4.4 [ 6.9–11] 5.7  14.7 [ 43.8–25] 1.6  4.2 [ 8.5–5.5] 0.1  1.3 [ 3–2]

[95% CI]

P-value

[ 8–2.5] [ 7.9–2.7] [ 29.3–5.9] [ 5.1–1.6] [ 2.9–0.9]

< 0.01 < 0.01 < 0.01 < 0.01 < 0.01

SRM/ES 0.11/0.17 0.17/ 0.19 0.29/ 0.32 0.38/ 0.51 0.07/ 0.06

P-value comparing improved vs. deteriorated by t-test, after recoding the actual status of health in 2 groups: condition considered improved or deteriorated (identical and worse). a Considering priorities defined at baseline and retained at 6 months. 95% CI for means of individual changes in the 2 groups with same priorities defined at baseline. b Considering shifts in priorities at 6 months. P-value comparing improved vs. deteriorated. Conditions ‘‘not changed’’ and ‘‘deteriorated’’ were recoded as deteriorated. 95% CI for means of individual changes in the 2 groups with shifts in priorities.

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follow-up for RA patients [13], about 50% for chronic LBP patients at 6-month evaluation [39] and almost all (93.9%) for SSc patients at 1.7  0.8 years of follow-up [38]. Shifts in priorities affected the MACTAR global score, sensitivity to change and MCDI value. The MACTAR global score at 6-month follow-up was lower when considering baseline activities maintained from baseline than when considering shifted priorities. Taking into account shifts in priorities to calculate the MACTAR global score led to decreased sensitivity to change (SRM and MCDI values) for patients who considered their condition improved and increased sensitivity for those who considered their condition deteriorated. This finding is not surprising because defining a new priority at follow-up means that one activity has become more difficult than the one chosen at baseline and omitted at follow-up. This observation raises the question of how the MACTAR should be used in clinical research and practice. Changes in the MACTAR score at 6-month evaluation when considering priorities defined at baseline reflect the evolution of patient-perceived handicap in 3 specific priorities but which may have become less important to the patient at follow-up. Changes in MACTAR score considering shifted priorities at 6 months reflect the evolution of patientperceived global priority handicap; it reflects a more pragmatic approach to capturing how a ‘‘global priority burden’’ has changed over time. These 2 ways of measuring changes in MACTAR global score over time are probably complementary. Considering shifts in priority in calculating the MACTAR global score would probably lead to the instrument’s lack of sensitivity to change in clinical trials. Moreover, the aim at baseline is to reduce limitations in participation in activities defined as a priority at this time, and therefore, priorities at baseline should be considered for calculating the MACTAR global score at follow-up. Our sensitivity analysis confirmed this suggestion. Regardless, the validity of comparing 2 sets of global scores calculated from different items at follow-up and calculating change scores should be further analyzed. Although the MACTAR approach could closely reflect real-life limitations in participation and may help in clinical decisionmaking, it may not be an easy, cost-effective instrument. This point should be assessed in further studies, because the MACTAR seems to be a quick questionnaire to complete as compared with other tools evaluating disability in knee OA. Moreover, whether the instrument measures change rather than just unrealistic desires is unclear. However, the strength of the MACTAR is that the concept of measuring priorities in disabilities may apply to all clinical situations inducing disability. The proportion of patients lost to follow-up in our study (15%) could be explained by the low education level (74%) in this group, which would explain the difficulty in completing the questionnaire. Other limitations of the study were the recruitment in tertiary care hospitals, where most patients had a moderate to severe knee OA, and the lack of test–retest reliability evaluation. In conclusion, the MACTAR is an outcome measure that has acceptable validity and is sensitive to change. For epidemiological surveys, recording shifts in priority of activities that cause trouble for OA patients can provide a qualitative analysis of limitations in participation that probably gives accurate information on what matters most to patients. However, clinical investigators must be aware that considering shifts in priority activities to calculate the MACTAR global score probably leads to the instrument’s lack of sensitivity to change when measuring health evolution.

Disclosure of interest F.R. received consultant fees, speaking fees and/or honoraria (less than $10,000 each) from Pierre Fabre, MSD, Servier and Expanscience; S.P. from MSD, Abbvie, Pfizer and BMS.

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K.S., C.P., C.E., M.-M.L., X.A. and J.B. declare that they have no competing interest. Acknowledgments The authors thank the patients from the rehabilitation and rheumatology departments at Cochin and Lariboisie`re hospitals for their participation. No funding was received for the study.

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