Isolde A R Kuijlaars|Maarten G. van Emst|Janjaap van der Net|Merel A. Timmer|Kathelijn Fischer
Assessing the test–retest reliability and smallest detectable change of the Haemophilia Activities List
Utrecht University|University Medical Center Utrecht|Utrecht University|University Medical Center Utrecht|Utrecht University|University Medical Center Utrecht|Utrecht University|University Medical Center Utrecht|Utrecht University|University Medical Center Utrecht
haemophiliavolume 27 issue 1 p 108112 original articleopen access assessing testretest reliability smallest detectable change haemophilia activities list isolde r kuijlaars corresponding author isolde r kuijlaars iarkuijlaars2umcutrechtnl orcidorg0000000329202258 van creveldkliniek university medical center utrecht utrecht university utrecht netherlands correspondence isolde r kuijlaars van creveldkliniek university medical center utrecht utrecht university heidelberglaan 100 3584 cx utrecht netherlands email iarkuijlaars2umcutrechtnlsearch papers authormadelon van emst madelon van emst van creveldkliniek university medical center utrecht utrecht university utrecht netherlandssearch papers authorjanjaap van der net janjaap van der net orcidorg0000000326065104 center child development exercise physical literacy childrens hospital university medical center utrecht utrecht university utrecht netherlandssearch papers authormerel timmer merel timmer orcidorg000000031910999x van creveldkliniek university medical center utrecht utrecht university utrecht netherlandssearch papers authorkathelijn fischer kathelijn fischer orcidorg0000000171266613 van creveldkliniek university medical center utrecht utrecht university utrecht netherlandssearch papers author isolde r kuijlaars corresponding author isolde r kuijlaars iarkuijlaars2umcutrechtnl orcidorg0000000329202258 van creveldkliniek university medical center utrecht utrecht university utrecht netherlands correspondence isolde r kuijlaars van creveldkliniek university medical center utrecht utrecht university heidelberglaan 100 3584 cx utrecht netherlands email iarkuijlaars2umcutrechtnlsearch papers authormadelon van emst madelon van emst van creveldkliniek university medical center utrecht utrecht university utrecht netherlandssearch papers authorjanjaap van der net janjaap van der net orcidorg0000000326065104 center child development exercise physical literacy childrens hospital university medical center utrecht utrecht university utrecht netherlandssearch papers authormerel timmer merel timmer orcidorg000000031910999x van creveldkliniek university medical center utrecht utrecht university utrecht netherlandssearch papers authorkathelijn fischer kathelijn fischer orcidorg0000000171266613 van creveldkliniek university medical center utrecht utrecht university utrecht netherlandssearch papers author first published 13 december 2020 httpsdoiorg101111hae14226citations 4aboutsectionspdf toolsrequest permissionexport citationadd favoritestrack citation shareshare give accessshare full text accessshare fulltext accessplease review terms conditions use check box share fulltext version articlei read accept wiley online library terms conditions useshareable linkuse link share fulltext version article friends colleagues learn morecopy url share linkshare onfacebooktwitterlinked inredditwechat abstract introduction haemophilia activities list hal preferred instrument measure selfreported limitations activities persons haemophilia pwh information reliability interpretability hal scores lacking aim examine testretest reliability smallest detectable change sdc hal adult pwh methods fifty adult 18 years persons mild severe haemophilia completed hal 42 items 7 domains optimum 100 baseline t0 34 weeks later t1 intraclass correlation coefficient icc sdc calculated sum component scores results fifty persons haemophilia included median age 49 years 92 haemophilia 70 severe haemophilia median interquartile ranges hal sum score 77 62 99 t0 81 64 98 t1 reliability good iccs sum component scores 09 sdc sum score 102 upper extremity component score 92 basic lower extremity component score 167 complex lower extremity component score 134 conclusion hal good reliability sum component scores score changes normalized sum hal score greater sdc 102 indicate change result measurement error 1 introduction persons haemophilia pwh suffer recurrent joint bleeds lead synovial inflammation bloodrelated cartilage damage eventually resulting haemophilic arthropathy1 2 joint impairment result limitations functional abilities daily activities participation society reduction quality life1 developed countries treatment haemophilia greatly improved last decades life expectancy pwh almost normalized3 especially gene therapy promising next step haemophilia care4 appropriate clinimetric instruments essential assess effect new paramedical treatments monitor patients individual level besides reporting bleeding episodes joint assessment measurement impact haemophilia activities participation relation society important1 haemophilia activities list hal recommended measure selfreported activities participation5 hal developed patient interviews according world health organization international classification functioning disability health icf measures selfreported limitations activities participation due haemophilia previous month68 addition clinically relevant instrument valid reliable validity degree instrument measures construct aims measure reliability degree measurement free measurement error furthermore interpretability important measurement property degree one assign qualitative meaning instruments quantitative scores change scores9 10 hal developed according classical test theory ctt implies sum component scores sum individual ordinal items questionnaire8 recent systematic review performed according ctt reported hal good content validity reflects daily activities based interviews pwh conflicting evidence construct validity5 example hal discriminated well patients intensive less intensive prophylaxis patients stopped continued prophylaxis5 however information reliability including testretest reliability interpretability scores lacking necessary interpret hal scores clinical practice research5 aim study examine testretest reliability smallest detectable change sdc hal adult pwh furthermore measurement error needs considered determine sdc 2 materials methods 21 study design study population study singlecentre prospective psychometric study adult 18 years persons mild severe haemophilia visited van creveldkliniek utrecht netherlands routine assessment asked participate study first hal t0 completed clinic visit second hal sent mail three weeks later t1 pwh asked complete questionnaire within one week time interval t0 t1 considered sufficiently long prevent recall bias data collected september 2017 september 2018 pwh excluded recent bleed synovitis joint surgery atbetween t0 t1 aimed inclusion 50 pwh according consensusbased standards development measurement instruments cosmin guidelines11 medical research ethical committee mrec university medical center utrecht reviewed approved study 17591c 22 measurements hal contains 42 items across 7 domains lying downsittingkneelingstanding functions legs functions arms use transportation selfcare household tasks leisure activities sports items scored 6point likert scale impossible always usually sometimes almost never never applicable option items summary score well component scores upper extremity basic lower extremity complex lower extremity calculated using official scoring tool available wwwvancreveldklinieknl12 scores converted normalized score 0 100 higher scores represent better functional status half items missing scored applicable valid domain component sum score calculated6 8 patient characteristics included age baseline hal assessment type haemophilia b severity disease mild factor viiiix activity 006040 iuml moderatefactor viiiix activity 001005 iuml severe factor viiiix activity 001 iuml use aids time test retest 23 statistical analyses patient characteristics time t0 t1 presented proportions medians interquartile ranges iqrp25p75 descriptive analyses median iqr range performed hal sum score component scores t0 t1 addition assess effect delayed response 34 weeks time t0 t1 plotted change hal sum score t0 t1 linear regression analysis performed analyses performed using ibm spss statistics software version 26 reliability measurement error interpretability evaluated interpreted according definitions cosmin9 10 development hal analyses present study performed according ctt using ctt standard error measurement sem assumed stable total scale9 sem sdc calculated present study interpreted average sem sdc values hal scores 231 reliability reliability defined degree measurement free measurement error expresses well patients distinguished despite presence measurement error9 intraclass correlation coefficient icc iccagreementp2p2 m2 r2 calculated testretest reliability twoway random effects model agreement term refers variance component 2 p patient measurement r residual9 13 icc represents part variance scores attributed true differences patients icc expressed value 0 1 value 070 considered acceptable9 232 measurement error measurement error defined systematic random error patients score attributed true changes construct measured10 standard error measurement sem agreement semagreement 2 2r calculated9 addition bland altman plot shown hal sum score illustrate measurement error relation mean hal score 95 limits agreement loa loa mean difference t0t1 196 sd difference t0t1 illustrates variation scores stable patients9 14 233 interpretability interpretability defined degree one assign qualitative meaning instruments quantitative scores change scores10 sdcagreement sdcagreement 196 2 semagreement calculated measure interpretability smallest change score detect measurement error9 iccagreement semagreement sdcagreement calculated sum component scores 3 results 31 patient characteristics sixtynine pwh invited fifteen participants excluded due recent bleeding synovitis four participants return hal t1 eventually 50 pwh included analysed table 1 sum threecomponent scores available patients t0 t1 scoring missing 94200 items total median age 490 years range 20 79 700 severe haemophilia 100 moderate haemophilia 200 mild haemophilia nine pwh 180 used aids performing certain activities median iqr time measurement t0 t1 34 weeks 30 52 range 14 104 weeks table 1 patient characteristics baseline n 50 patient characteristics n 50 median iqr n age years 490 368 613 haemophilia 46 920 severity haemophilia mild 10 200 moderate 5 100 severe 35 700 using aids performing certain activitiesbb three persons used two different aids 7 140 one crutchcane 4 80 two crutches 3 60 wheelchair 1 20 aidsaa aids ie scooter modified bicycle 2 40 time weeks t0 t1 34 30 52 aids ie scooter modified bicycle b three persons used two different aids 32 hal sum component scores table 2 presents sum component scores t0 t1 group level median iqr hal sum score 771 625 986 t0 812 636 985 t1 median iqr absolute difference sum component scores varied 22 00 45 44 00 67 pwh scored highest upper extremity component lowest complex lower extremity component scores highest domain selfcare lowest domain functions legs table s4 maximum scores t0 t1 occurred frequently maximum hal sum 18 maximum component scores 2032 sum component scores leftskewed distributions difference hal sum component scores t0 t1 increased increasing time assessments b 018 p 0001 34 pwh filled retest 50 days 71 weeks scores varied 100 points table 2 characteristics test t0 retest t1 hal sum component scores hal score t0 t1 absolute difference t0t1 median iqr min max median iqr min max median iqr sum 771 625 986 234 1000 812 636 985 195 1000 24 05 50 upper extremity 956 828 1000 378 1000 956 833 1000 289 1000 22 00 45 basic lower extremity 733 550 1000 67 1000 783 533 1000 33 1000 33 00 67 complex lower extremity 578 306 961 67 1000 567 328 1000 67 1000 44 00 67 33 reliability measurement error interpretability table 3 presents iccagreement semagreement sdcagreement sum component scores icc values exceeded 090 hal sum score sem 37 sdc 102 basic lower extremity component score highest variation sem 60 sdc value 167 upper extremity component score lowest variation sem 33 sdc value 92 figure 1 shows bland altman plot hal sum score loa 092 1014 differences scores t0 t1 change increasing mean hal values graphically checked table 3 standard error measurement sem smallest detectable change sdc intraclass correlation coefficient icc haemophilia activities list n 50 hal score semagreement sdcagreement iccagreement 95 ci sum 368 1020 097 095 098 upper extremity 333 923 097 094 098 basic lower extremity 602 1669 095 091 097 complex lower extremity 485 1345 098 096 099 figure 1open figure viewerpowerpoint bland altman plot hal sum score limits agreement 092 1014 exclusion pwh time t0 t1 50 days icc values increased sem sdc values smaller basic lower extremity component score highest variation sem 57 sdc value 158 hal sum score lowest variation sem 28 sdc value 78 see table s5 4 discussion present study aimed determine testretest reliability sdc hal hal demonstrates good testretest reliability sum components score icc value 090 average sdc value normalized hal sum score 102 implies change score 102 signifies true change one patient due measurement error upper extremity component score change score 93 signifies true change basic lower extremity component score change 167 complex lower extremity component score change 135 sdc values smaller excluding patients delayed response 50 days sdc sum score 78 upper extremity component score 88 basic lower extremity component score 158 complex lower extremity component score 117 41 comparison studies studies examining measurement properties hal limited icc values present study similar previously reported icc values 087097 adult pwh usa n 158162 reported two questionnaires completed within 2 hours15 icc values 066090 lower brazilian pwh n 52 completed hal interviews interval 15 days different individually completing paper questionnaire present study16 sem sdc values published 42 strengths limitations strength study followup time median 34 weeks sufficiently long prevent recall bias addition sample size 50 patients according recommendation cosmin guideline disadvantage ctt approach calculated sem sdc values stable whole continuum score present study hal sum scores high comparable scores studies united kingdom uk united states america usa17 18 indicating ceiling effect hal western countries therefore sem sdc values calculated present study best reflect measurement error sdc upper end hal score better functional status furthermore hal sum component scores 0100 sum ordinal items corrected difficulty separate items example scoring impossible easy item like sitting weight sum score scoring impossible difficult item like running addition skewed distribution affects precision calculation iccs sems sdcs based variance components analysis variance anova anova test assumes data normally distributed case study finally outliers delayed response time t0 t1 50 days increased sem sdc implies assumption patients change time study design fully met 43 clinical implications future research change scores normalized sum hal score greater sdc 102 indicate change result measurement error sdc hal helps pick real changes activities participation clinical practice patients monitored hal intervention annual routine visit addition sdc score compared minimal important change mic smallest change score perceived important patients9 however mic hal still needs established 5 conclusion hal reliable selfreported outcome measure limitations activities participation pwh average sdc values 102 normalized hal sum score 92 upper extremity component score 167 basic lower extremity component score 134 complex lower extremity component score signifies true change score due measurement error difference hal scores test retest increases larger time intervals tests acknowledgements authors would like thank p de kleijn ep mauserbunschoten pr van de valk help data collection addition authors would like p de kleijn contribution writing manuscript conflict interest none authors reported conflict interest regarding manuscript membership group developed hal author contribution iar kuijlaars j van der net k fischer contributed design study iar kuijlaars timmer performed data collection van emst performed initial statistical analyses wrote first draft paper authors contributed interpretation data writing manuscript supporting information filename description hae14226sup0001supinfodocxword document 482 kb supplementary material please note publisher responsible content functionality supporting information supplied authors queries missing content directed corresponding author article references 1srivastava santagostino e dougall et al wfh guidelines management hemophilia 3rd edition haemophilia 2020 google scholar 2van vulpen lfd holstein c martinoli k joint disease haemophilia pathophysiology pain imaging haemophilia 2018 24 44 49 wiley online librarypubmedweb sciencegoogle scholar 3shapiro makris haemophilia ageing br j haematol 2019 184 712 720 wiley online librarypubmedweb sciencegoogle scholar 4miesbach w 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