Measure Raphaèle of Function in Rheumatoid Arthritis: Individualized or Classical Scales?

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1 Measure Raphaèle of Function in Rheumatoid Arthritis: Individualized or Classical Scales? (1) Ravaud 1 Seror 1, Florence Tubach 1, Gabriel Baron 1, Francis Guillemin 2, and Philippe Clinique, de (2) AP-HP, Paris, Hôpital France; Bichat, INSERM Département U738, Paris, d Epidémiologie, France; Université Biostatistique Paris 7-Denis et Diderot, Recherche d épidémiologie médecine, INSERM, Paris, Centre France d Epidémiologie Clinique CIE6, ; CHU de Nancy, Service UFR les-nancy, et évaluation cliniques, Nancy ; Nancy-Université, EA 4003, Vandoeuvre- Correspondence France Dr Département Bichat, Raphaèle : 46 raphaele.se@gmail.com rue Seror d Epidémiologie, Henri and Huchard, reprint requests Biostatistique Paris, to: France. et Recherche Clinique, INSERM U738, Hôpital KEY index, WORD WORDS: Individualization, COUNT: Health 1999assessment Patient reported questionnaire, outcome, Rheumatoid Patient's perspective. arthritis, Patient-specific 1

2 widely of the ABSTRACT Objective. The (250 Health words) Patients treated patients HAQ-DI. used with and view leflunomide measure methods. in outcome of HAQ-DI Assessment function over assessment, a 6-month in data rheumatoid Questionnaire were this period. prospectively study arthritis At aimed Disability baseline (RA). to obtained evaluate and Index To final enhance from (HAQ-DI) individualized visits, 370 the RA patients incorporation is outpatients the forms had most of Results. rate select evaluated: added The the psychometric the to importance its 5 scales activities importance; preserving properties they and attached considered all of theses domains, to involving each the scales most activity which were for important. each compared addressed the patient score Different to by for only those the each the individualized 20 of item 5 HAQ-DI the most HAQ-DI. multiplied important items, scales and items. by were to Scores All HAQ-DI, response Conclusion. scales for For had all each a good HAQ-DI internal scales item, consistency were severity highly (Cronbach s and correlated importance alpha: with scores 0.87 the HAQ-DI were 0.88). weakly score Compared (rho>0.75). correlated. individualized measure mean: individualized Individualized questionnaires 0.64 to 0.69 scales vs. scales measuring 0.74). did have not importance have similar better properties gave sensitivity complementary as the to change HAQ-DI. information (standardized However, to to the assessment be clinically of disability. relevant all randomized for Therefore, individual controlled even RA patients. if trials, individualization the use of individualized is probably not questionnaires needed group could 2

3 reported However, recommended Questionnaire functional patients, Outcome outcomes symptoms assessment such such measures in pain, rheumatoid fatigue of disease and arthritis disability activity (RA) [1-3] are was relevant or primarily structural and based their progression on assessment physician- being status Disability RA evaluation Index patients.[7-10] (HAQ-DI) of response It a valid, a to standardized treatment.[6] reliable, and questionnaire, widely The used Health identical tool Assessment to measure for.[4, all 5] individuals. able investigating perform patients each ability activity. to perform However, some these activities, issues as may well vary as widely importance among of is noted address to involve as For each a major these Furthermore, patient s focus reasons, priorities for research.[6, enhancing patient-specific, outcome patients 11] assessment individualized, perspectives RA.[12-15] scales outcome have Some been have assessment developed been shown was developed each the be item more semi-structured is an sensitive weighted individualized to by interviews change the questionnaire importance than requiring classical it derived has 15 for instruments.[16-18] to 40 each from minutes patient. the modified to However, complete.[19] However, HAQ, responsiveness [20, most Hewlett 21] of where them al. of to properties from scale We has designed not been this evaluated. PATIENTS of patient-specific prospective scales, assessing study to the develop, level of validate disability and in evaluate RA patients, psychometric Study the HAQ-DI AND according METHODS to several methods of individualization. derived outpatients American physician visits population Measurement Data global Rheumatism treated were assessment obtained with Association leflunomide.[22] from of disease a 6-month, revised activity Patients prospective, criteria,[23] 2 a included 5-point open-label with Likert had active study RA scale disease (from involving defined 1 to 378 5). by by Two RA the At were scheduled, one baseline before initiation of leflunomide and one 6 months later. a items, a domain Dependence The baseline French and version final visits, of the patients HAQ-DI,[24] completed: the help referring and is the device on to help highest ability component. item device in performing score increases For of each daily that a item, termed lower domain, life activities, score the severity ranged unless to 2. involving The help from questionnaire. overall 0 or to 8 a domains 3. score device The It is score comprises and the required. includes mean of each 20 - answering by The a 84-point domain importance the Likert scores question questionnaire: scale, and In varies ranging your from patients daily from 0 to life, 0= not 3. had the to important following rate the importance activity to 3= extremely is. of each Importance important. HAQ-DI was item, rated by of 3

4 - items) The protein Methods The swollen preference they (CRP) considered and were tender questionnaire: also the joint collected most counts, important patients to erythrocyte had in the their to disease select sedimentation daily and activity lives. order score rate the (ESR), 5 (DAS28). activities level (i.e. of C-reactive HAQ-DI Several (1) The individualized of individualization importance domains. Scales - With based multiplicative, the scales importance were additive, obtained: obtained.[25] questionnaire (2) Scales score multiplied Individualized for scores each by, of domain or items added scale, was of to, with that the the multiplicative highest domain. importance methods: score The score. obtained method, total for score each preserving after was item, combining the the all mean severity 20 the items score severity score was of all also was (a) based Top 5 on HAQ: the preference involving only questionnaire. the 5 most important activities for each patient. and 8 Initial these (b) severity referred scales, Weighted to score the the resulting top most is weighted 5 HAQ: important items involving by are activity). rank the only of same the preference 5 for most all patients. important for this activity The activities, total (highest score whereby was weight obtained Because individualized sum of scores the the by scores, combining rating were weighted of obtained importance baseline by not, combining importance has of the not 5 most been data or preference important applied from the to items. ratings baseline the help and visit. and final device Final severity scores component, scores. were the transformed disability. Statistical to a scales of were 0 to developed 3; 0 indicating without no disability this component. and 3 indicating Each scale maximal was possible linearly were individualized involved All analysis patients scale the who were development had evaluated, completed of and individualized the compared original to HAQ-DI that forms. of the Psychometric with HAQ-DI: no missing properties data at baseline of each - Construct each validity measures DAS28). individualized using of validity disease Spearman was scale activity assessed correlation and (swollen that by between Spearman of and the tender HAQ-DI. scores rho joint of correlation individualized We count, also between ESR, examined scales CRP the level and divergent score other of comparisons 1000 Internal items), replications.[27] by consistency Cronbach s of Cronbach s was alpha assessed, alpha coefficient.[26] coefficients when estimable Estimation involved (i.e. of the for confidence bootstrap scales with method, intervals identical with and 4

5 Statistical - Responsiveness mean deviation moderate SRM comparisons change of ( ) the was change score involved assessed small between the score.[28] (<0.5).[29, by bootstrap the baseline standardized SRM 30] method SRM values final with confidence response can 1000 visit be mean replications.[27] divided considered interval (SRM), by estimations the large which standard (>0.8), is the RESULTS DI (The Of the analysis R Foundation 378 outpatients involved for Statistical use with of RA SAS, Computing, enrolled release 9.1 the Vienna, (SAS study, Austria). Inst., 370 (98%) Cary, completed NC), and R, the release patients correlated revealed without For correctly (rho each missing ranging completed item, data from severity the baseline 0.01 importance and to 0.29) importance (table and (table 1). preference Among 2). scores Plots them, were questionnaires, of severity weakly 360 (95%) by or respectively. importance not and significantly 281 scores (74%) HAQitems consider (124/175) important 3 walk as that important the of items extremely patients the not as and with not disabled important. patients the highest with patients At (figure the severity baseline, lowest (severity 1). scores As severity based for for item on score items of the get 0) for did preference considered items not and necessarily off did questionnaire, the this not toilet, necessarily activity grade 71% the showed more items most than outdoors important that 75% each of on items of the flat these patients. ground were 3 dress activities At (55%). final yourself was visit, Data considered collected no (86%), change wash by important in selection and importance dry entire of extremely the body 5 most (63%), important also and by as selected Construct All table swollen individualized 3). was Lower observed validity least 4 and scale for 3, 17% respectively, scores of patients, were of highly whereas the initial correlated 34% 5 measures items (61/182) with they of the chose. and disease HAQ-DI 66% (121/182) activity: score tender (rho 0.75; of patients 0.47). as Internal For ESR The and joint lowest CRP counts level correlations (rho (rho ranging were from observed with to 0.39) 0.18). biological and DAS28 features (rho of ranging disease activity, from 0.38 such and Scales scales consistency with 8 domains, Cronbach s alpha coefficient ranged from 0.87 to 0.88 (table 3). to Responsiveness The (p<0.05). preserving SRM preserving SRMs of all the 20 of items HAQ-DI all individualized 20 was items was had 0.74, higher scales compared Cronbach s ranged to from 0.69 alpha 0.64 without coefficients to 0.69 help (table and (alpha=0.94). 3). device SRM component of scales 5

6 Table Age 1. Baseline characteristics of patients Rheumatoid Disease Sex (female) (years) duration factor (years) positive Patients for 9.84 with N=370 HAQ-DI (66.85%) (78.92%) ±11.93 ±8.49 complete data Patients for 9.34 with N=281 all (80.07%) (61.92%) complete scores data Tender Prior Patient DMARD global intake of disease (87.84%) (86.48%) ESR Physician Swollen (mm) joint global count assessment (/28) of disease activity Data CRP DAS28 HAQ-DI level (mg/l) Range [ ± ± ] Range ± [ ± DAS: questionnaire are disease presented disability activity as mean score; index; ± DMARD: SD CRP: or n C-reactive (%) disease-modifying protein anti-rheumatic drug; ESR: erythrocyte ] sedimentation rate; HAQ-DI: health assessment 6

7 Table 2. Importance of each activity of the HAQ-DI from the patient s perspective Domain Dressing Item Mean Questionnaire disability Severity (n=370) baseline Mean Questionnaire Importance importance (n=360) baseline severity Correlation importance between and at Items baseline (n=274) selected visit Questionnaire Items Preference Arising and grooming Shampoo Dress yourself (14%) (86%) at and both 124 (n=182) final 7 (4%) selected (68%) baseline visits Identical baseline at selected (n=182) final (78%) (86%) visit items and at Eating Walking Hygiene Get Lift Open Stand Cut in your a full from and new meat cup a carton chair or of glass bed of milk to mouth (27%) (31%) (21%) (6%) (13%) (14%) (2%) (9%) (79%) (77%) (73%) Reach Climb Walk outdoors 5 steps flat ground (12%) (55%) 66 (36%) (80%) (92%) Grip Take Get Wash on a bath dry off entire the toilet body (27%) (63%) (53%) (18%) (68%) (85%) (74%) Other Bend Reach down car doors get and down pick a up 5-lb clothing object (16%) (7%) 13 6 (3%) (7%) (73%) (81%) Data activities are presented as Open Turn Get Do Run mean chores in errands taps jars ± on (previously out SD and of or shop off car n opened) (%) ± ± (9%) (12%) (11%) (32%) (20%) (23%) (4%) (2%) (85%) (63%) (87%) (83%) (91%) 7

8 Table 3. Psychometric properties of classical HAQ-DI Importance and individualized questionnaire HAQ scales Construct Spearman s HAQ-DI Individualized multiplicative Individualized additive HAQ Preference 5-item HAQ questionnaire with Mean baseline change validity correlation in score ± ± ± ± Weighted 5-item HAQ ± 0.66 Sensitivity Internal Cronbach s HAQ-DI consistency to alpha change score [ ] [ ] 0.88 [ ] NE 0.76 NE 0.61 Each Mean [95% For SRM 1Data HAQ-DI: Spearman s CI]: score are changes CI] 95% presented health was confidence correlations, in linearly assessment scores mean transformed were intervals all questionnaire ± p-values calculated SD. were [ ] to were estimated a 0.74 between score < disability by from the index; 0 bootstrap baseline to [ ] 3. NE: 0.69 visit method not at estimable; week with (W0) [ ] SRM: replications and standardized the final visit response [ ] at 0.65 week mean. 24 (W24):[ ] W24-W

9 HAQ-DI, importance information DISCUSSION RA This patients. whatever study Individualized evaluated the individualization the interest scales have of individualizing method similar used. psychometric the However, HAQ-DI properties adding to measure a measure the disability clinicians.[31-33] to a measure of severity for each activity provided complementary parent opinions Many that studies could So have far, be clinically shown the use divergent useful of in views decision of making a patient s for individual health and inclusion between patients. patients of patients and of patient, To proposed. patients the opinion their importance in development of each has of gradually patient-reported and to weight increased.[34] that outcomes item One with is usual to the determine, approach mean importance to in instrument group facilitate level. rating Two patient However, of key practices input such into are a the group addition recommended assessment approach to severity for process, assumes individualization for each concordance individualized item, and/or :[35] of measured all (ii) (i) patients to to augment incorporate a has group views. score been the of recommendation, severity the study items, importance he unfortunately, considered and with importance. of supplemental and these did used not most 2 methods. offer In important. items the patients second that The Moreover, items. first method, the patients opportunity method Regarding both each can combined, methods add. patient the to Our provide second selected were for study each supplemental combined, recommendation, a relies limited item, the on by number rating the relevant ranking first our of performing be of ratings not Arguing contained that in importance the HAQ-DI.[31] disability, individualized importance sufficient.[36, of but the that were and importance activity, 37] level also Against of similar some disability were this authors among extremely of hypothesis, were patients wonder activity weakly variable our whether with is correlated. resulted closely diverse among assessment showed related levels Moreover, patients that of to of disability. with level for as previously most similar of [20, items, disability alone levels shown, rating could So, of on subscale, furthermore, sensitive what In ratings our really the approach previous meaningful of individualized the importance may study to be each evaluating useful scale patient. and retaining severity clinical individualized of practice each 5 most item to forms help important of physician strongly the activities WOMAC to correlated;[38] prioritize was function more care that different, they wording, but not were had no to and the change difficulty patients most than performing had need the to parent of define improvement. them. scale. importance As In shown that In study, of the for activities present item patients get of study, were on their and the asked daily of wording the lives, to toilet, define even was activities discrepancies perhaps it easier likely to that do. Therefore, important items if these for items a patient are initially may be close those to that normal, are relevant improvement with slightly to though him, this devices rating possible. of As component, importance previously Items between are but was proposed,[25, results therefore found not applied of it these less less sensitive to 39] studies. sensitive the we help to also to and change change. used devices than the This component, the HAQ-DI difference parent HAQ-DI. without individualized might the Because explain help scales and the 9

10 did individualized than questionnaire method not the Individualized include parent that scales HAQ-DI. this did had scales component. not lower focus This derived responsiveness could This activities from be omission explained the than HAQ-DI need the could by parent of did the have improvement, not wording HAQ-DI. explained have of better the in by responsiveness individualized part the why scoring the activity individualization trials, for the for of gave use the which of patients. complementary HAQ-DI, individualized is the probably patient somewhat not information is questionnaires most needed already disabled. for to individualized group could the However, measure assessment be clinically the considering of disability. measure relevant all randomized for of in Therefore, importance each decision domain controlled making even of the an if Acknowledgements: physicians Competing who interests: participated This None. study to this was study. funded Aventis Pharma, Paris, France. We thank all the The behalf worldwide to published Corresponding exploit of all basis authors, Annals to all the Author an BMJ of exclusive subsidiary the has Publishing Rheumatic the licence right rights, Group to Diseases (or grant non-exclusive Ltd as editions and behalf its set Licensees and of for all out government any authors other to in permit BMJPGL and employees) our does this article products grant licence on on to a 10

11 Figure items. 1. Patterns of plots of scores of severity by importance for 3 of the 20 HAQ-DI These area the Reach not on and highest necessarily of graphics off each and the severity get point toilet ). consider represent down is scores proportional a 5 the patterns lb for items object ) items as of to did not and the plots not important number patients of necessarily severity of with (item patients. scores the grade 1 and lowest the Plots against 12: items severity "Dress showed importance as important score yourself" that patients for scores. items (item and "Get with The did 13: 11

12 2 and Comparison Organization/International 3 References activity study 1 Rheumatology. van Prevoo Felson validation Gestel scores ML, DT, AM, with van that Anderson Preliminary of Prevoo 't include the Hof the European MA, JJ, ML, League preliminary twenty-eight-joint definition Boers Kuper van Against League 't M, Hof HH, improvement American Bombardier MA, Rheumatism van Against counts. Leeuwen van Rijswijk College Rheumatism Development C, in Criteria. rheumatoid MA, Furst MH, van of D, Arthritis van de response Rheumatology Goldsmith and arthritis. Putte de validation Putte LB, criteria Arthritis C, LB, van 1996;39: et and van Riel a al. for Rheum prospective Riel American PL. rheumatoid the PL. 1995;38: Modified World Development longitudinal College arthritis. disease Health of : Dis clinical Sharp van Aletaha 2008;67: of der trials JT. patients Heijde D, Radiologic Landewe of with patients DM. rheumatoid Joint assessment R, with Karonitsch erosions rheumatoid arthritis. as and T, outcome patients Bathon Arthritis arthritis: J, with measure Boers Rheum EULAR/ACR early M, in 1995;38:44-8. rheumatoid Bombardier collaborative arthritis. C, et al. Br recommendations. Arthritis Reporting J Rheumatol disease 1995; ;32:221- Ann activity Suppl in 1996: disability 1980;23: Bruce Fries JF, B, and Fries Spitz pain JF. P, PW, scales. The Kraines Young Stanford J Rheumatol RG, DY. Health Holman The 1982;9: dimensions HR. Measurement Questionnaire: of health of outcomes: patient a review outcome the of health its history, assessment arthritis. issues, Arthritis questionnaire, progress, Rheum 11 Perspective documentation. 10 editor. Ramey Kirwan Quality D, J, Fries Heiberg J of Rheumatol life JF, Sing and T, Hewlett 2003;30: G. pharmacoeconomics The S, Health Hughes Assessment R, Kvien clinical T, trials. Ahlmen 2nd - M, Status ed. et Philadelphia: al. and Outcomes review. In: Lippincott-Raven, from In: Spikler the B and e, 14 investigations 12 Preference 13 rheumatoid 15 Tugwell Workshop P, OMERACT C, Buchanan 6. J Rheumatol WW, Goldsmith 2003;30: and physical Ripat Bell Meenan the Health MJ, Etcheverry Disability disability arthritis. RF, Bombardier of Assessment a Gertman health Arthritis in Questionnaire--an E, clinical status Cooper Questionnaire. C, PM, Rheum Tugwell measure. trials J, Mason Tate in 1990;33: rheumatoid P. individualized RB. Arthritis Can JH, Measurement A J Dunaif comparison Occup Rheum arthritis. R. functional Ther 1982;25: of The of J 2001;68: CH, functional Rheumatol the arthritis Canadian priority Grace status, 1987;14: impact E, approach Occupational Hanna quality measurement B. for The assessing of Performance life, MACTAR scales. and improvement utility Measure Further Patient in rheumatoid reflected compared 16 Tugwell with P, measures methotrexate Wells G, of Strand function in patients V, and Maetzel with health-related rheumatoid A, Bombardier quality arthritis: C, of Crawford sensitivity life following B, et relative al. treatment Clinical efficiency with improvement leflunomide to detect preference 18 treatment Group. 17 Verhoeven Tugwell Arthritis effect arthritis. P, AC, Bombardier Rheum in Boers a clinical Impact twelve-month, 2000;43: M, trial. van C, quality Buchanan The der Liden placebo-controlled McMaster of life WW, S. Validity assessed Toronto Goldsmith of by Arthritis. trial. the traditional MACTAR C, Leflunomide Grace J Rheumatol standard-item E, questionnaire Bennett Rheumatoid 2000;27: KJ, and as et a Arthritis individualized functional al. Methotrexate Investigators index patient in in disorders. Jolles BM, J health Clin Buchbinder Epidemiol status questionnaires. 2005;58: R, Beaton DE. Arch A Intern study Med compared 1990;150: nine patient-specific indices for musculoskeletal a Association Impact 22 rheumatoid 1983;26: activities Arnett Hewlett Nguyen Pincus Health FC, of T, arthritis S, M, 1987 daily Summey Assessment Smith Edworthy Kabir revised in living AP, M, everyday JA, SM, criteria Kirwan Ravaud Questionnaire using Soraci Bloch clinical for JR. a P. SA, the DA, modified Short-term Measuring use Jr., classification (PI McShane : Wallston HAQ). open-label, Stanford efficacy the DJ, Ann meaning of KA, Fries rheumatoid Rheum prospective Health and Hummon JF, of safety Dis disability Cooper Assessment arthritis. 2002;61: study. NP. of leflunomide NS, Assessment Clin Arthritis et rheumatoid Questionnaire. Drug al. The in Investig Rheum of the American patient arthritis: treatment 1988;31: ;24: Arthritis satisfaction Rheumatism the of Personal active in 12

13 29 recommendations Med French Husted Efron Cronbach Bruce Liang Guillemin Care adaptation B, MH, 1990;28: JA, Tibshirani Fries LJ. F, Cook Fossel Coefficient Braincon JF. of RJ, the The AH, RJ. Farewell Health Larson An S, alpha Pourel Introduction Assessment VT, MG. and J. Gladman the Comparisons [Measurement internal to Questionnaire the DD. Bootstrap. structure Methods of of five the (HAQ)]. of (HAQ). New health functional for tests. York: assessing status Rev Clin Psychometrika Chapman Rhum Exp capacity instruments responsiveness: Rheumatol Mal and Osteoartic 1951;16: rheumatoid Hall, for 2005;23:S14-8. orthopedic a critical 1991;58: polyarthritis: review evaluation. a 30 2nd 31 public. 32 between Streiner Hewlett Kwoh ed. Ann clinician CK, New DL, S, Smith O'Connor York: Norman and J Dis Clin AP, patient Oxford 2001;60: GR. Epidemiol GT, Kirwan assessment Health University Regan-Smith JR. 2000;53: Measurement Values Press, physical MG, for function Olmstead Scales: and mental A in EM, Practical rheumatoid health Brown status. Guide LA, arthritis: J to Rheumatol Burnett Their patients, Developpement JB, 1992;19: et professionals, al. Concordance and Use. and functioning. Gossec Berkanovic L, Arthritis Dougados E, Hurwicz Care M, Res Rincheval ML, 1995;8: Lachenbruch N, Balanescu PA. A, Concordant Boumpas DT, and Canadelo discrepant S, views al. The of patients' elaboration physical pain Rheumatol 1994;272: preliminary Heiberg Kvien Gill TM, TK, 2003;30: T, Rheumatoid Kvien Feinstein Heiberg TK. T. Preferences AR. Patient A Impact critical perspective for of improved appraisal Disease in health (RAID) outcome of the examined score: assessments--perceptions quality a EULAR in of 1,024 quality-of-life initiative. patients with Ann measurements. rheumatoid something Rheum Dis more? arthritis: of Jama the and improvement 39 (DHAQ), Seror Wolfe McMaster has highest R, F. Tubach Which to Universities measure priority. F, HAQ Baron Arthritis functional is osteoarthritis best? G, Falissard Rheum A impairment comparison index 2002;47: B, Logeart (WOMAC) in hip of the I, or Dougados knee HAQ, function osteoarthritis. MHAQ M, subscale: et and al. Individualising Ann RA-HAQ, incorporating Rheum a Dis difficult the 2008;67: Western 8 priorities item Ontario HAQ for J leflunomide and initiation. a rescored J Rheumatol 20 item HAQ 2001;28: (HAQ20): analyses 2,491 rheumatoid arthritis patients following 13