Minimally Important Difference for Patient-reported Outcomes in Psoriatic Arthritis: Health Assessment Questionnaire and Pain, Fatigue, and Global Visual Analog Scales

2010 ◽  
Vol 37 (5) ◽  
pp. 1024-1028 ◽  
Author(s):  
TIFFANY KWOK ◽  
JANET E. POPE
Keyword(s):  
Psoriatic Arthritis ◽  
Health Status ◽  
Health Assessment Questionnaire ◽  
Patient Reported Outcomes ◽  
Health Assessment ◽  
Disease Status ◽  
Minimally Important Difference ◽  
Patient Reported ◽  
Assessment Questionnaire ◽  
Overall Health Status

Objective.To determine the minimally important difference (MID) for the Health Assessment Questionnaire-Disability Index (HAQ-DI), pain, fatigue, sleep, and global visual analog scale (VAS; 0–100 mm) in patients with psoriatic arthritis (PsA) using a patient-reported overall health status anchor. Patient-reported outcomes are often used to gauge the effect of PsA in clinical trials. There is currently no knowledge about the MID for patient-reported outcomes in PsA.Methods.Patients with a diagnosis of PsA who had answered questions about outcomes at 2 consecutive visits and an overall health status question (“How would you describe your overall status since your last visit: much better, better, the same, worse, much worse?”) were included. MID was calculated as the mean change between visits for those who rated their disease as “better” or “worse.”Results.Two hundred patients met inclusion criteria, of whom 17.5% rated their status as “better” and 25.0% rated their status as “worse” than the previous visit. MID estimates for improvement/worsening (SD) respectively were −0.131 (0.411)/0.131 (0.309) for HAQ-DI, −9.37 (24.37)/13.96 (22.05) for pain VAS, −8.15 (23.52)/3.63 (27.62) for fatigue VAS, −10.97 (29.74)/13.81 (27.32) for sleep VAS, and −8.41 (21.17)/11.53 (21.03) for global VAS. Spearman’s r correlation coefficients between the anchor and mean change were 0.374 (HAQ-DI), 0.448 (pain VAS), 0.239 (fatigue VAS), 0.326 (sleep VAS), 0.490 (global VAS); p < 0.01.Conclusion.This is the first study investigating MID of patient-reported outcomes in PsA. MID for HAQ-DI, pain, and global VAS were shown to be the best predictors for a patient’s perception of overall changes in disease status.

scholarly journals P158 Severity of hand vascular involvement as assessed by MRI Digital Artery Volume Index (DAVIX©) predicts worsening of clinical parameters and patient reported outcomes in scleroderma

Rheumatology ◽  
2020 ◽  
Vol 59 (Supplement_2) ◽  
Author(s):  
Fiammetta Danzo ◽  
Klodian Gjeloshi ◽  
Giovanni Lettieri ◽  
Giuseppina Abignano ◽  
Mark Hinton ◽  
...  
Keyword(s):  
Disease Activity ◽  
Health Assessment Questionnaire ◽  
Patient Reported Outcomes ◽  
Health Assessment ◽  
Volume Index ◽  
Vascular Involvement ◽  
Clinical Parameters ◽  
Digital Artery ◽  
Patient Reported ◽  
Assessment Questionnaire

Abstract Background Neointima proliferation is a key pathologic feature of systemic sclerosis (SSc), causing arterial vessel narrowing and being the recognised culprit pathological lesion in digital ulcers (DUs), pulmonary artery hypertension and renal crisis. Nevertheless, there are no validated imaging techniques to assess the severity of vascular involvement in SSc. We have previously shown digital artery volume index (DAVIX©) assessed with time of right MRI angiography, is a reliable measure of neointima proliferation in the hands. The purpose of our study was to identify the value of DAVIX© in predicting worsening of patient reported outcomes (PROs) and clinical parameters in SSc. Methods Cross-sectional data were available for 91 patients and complete 12 months follow-up data for 68 patients. Data collected included: modfied Rodnan skin score (mRSS), pulmonary function tests (PFTs), echocardiography, nailfold capillaroscopy, Health Assessment Questionnaire Disability Index (HAQ-DI), and Scleroderma Health Assessment Questionnaire (sHAQ). DAVIX© of the dominant hand was calculated as the % mean of the 4 fingers, employing MeVisLab software. Following analysis of distribution, Spearman or Pearson test were used to determine correlation coefficients, as appropriate (Prism 7). Results 56/68 were female and median of disease duration was 4 years (IQR 1.91-9). As previously reported DAVIX© correlated with the presence of DUs (p = 0.0093). Considering all patients, DAVIX© correlated with mRSS (r=-0.258, p = 0.017), DLCO% (r = 0.338, p = 0.008) and the pattern of capillaroscopy (r=-0.388, p = 0.001). In patients with DUs, DAVIX© showed a stronger correlation with DLCO% (r = 0.786, p = 0.048). Most importantly, DAVIX© predicted the worsening of HAQ-DI (r=-0.295, p = 0.029), sHAQ (r =-0.333, p = 0.029) and VAS pain (r=-0.269, p = 0.038) independently of the presence of DUs. Conclusion The quantitative assessment of neointima proliferation in the hand by DAVIX© is a useful imaging biomarker of vascular disease activity. The value of DAVIX© in predicting the worsening of PROs and clinical parameters in overall patients, may offer insights on the role of vascular disease activity in the global progression of SSc. The validation of our data in an independent cohort and the sensitivity to change over time of DAVIX© may aid to the implementation of hand MRI as imaging outcome measure of vascular severity in SSc. Disclosures F. Danzo None. K. Gjeloshi None. G. Lettieri None. G. Abignano None. M. Hinton None. A. Dean None. G. Cuomo None. O. Kubassova None. F. del Galdo None.

What Factors Influence the Health Status of Patients with Rheumatoid Arthritis Measured by the SF-12v2 Health Survey and the Health Assessment Questionnaire?

2009 ◽  
Vol 36 (10) ◽  
pp. 2183-2189 ◽  
Author(s):  
LOUISE LINDE ◽  
JAN SØRENSEN ◽  
MIKKEL ØSTERGAARD ◽  
KIM HØRSLEV-PETERSEN ◽  
CLAUS RASMUSSEN ◽  
...  
Keyword(s):  
Rheumatoid Arthritis ◽  
Health Status ◽  
Health Survey ◽  
Health Assessment Questionnaire ◽  
Health Assessment ◽  
Physical Component Score ◽  
Related Factors ◽  
Component Score ◽  
Patient Reported ◽  
Assessment Questionnaire

Objective.The Health Assessment Questionnaire Disability Index (HAQ) is a widely used outcome measure in rheumatoid arthritis (RA), whereas the SF-12v2 Health Survey (SF-12) was introduced recently. We investigated how the HAQ and SF-12 were associated with socio-demographic, lifestyle, and disease- and treatment-related factors in patients with RA.Methods.In RA patients from 11 Danish centers, clinical and patient-reported data, including the HAQ and SF-12, were collected. Three multiple linear regression models were estimated, with the HAQ, SF-12 physical component score (PCS), and SF-12 mental component score (MCS) as outcome and sociodemographic, lifestyle, and RA-related treatment and comorbidity characteristics as explanatory variables.Results.In total, 3156 (85%) of 3704 invited patients participated — 75% women, 76% rheumatoid factor-positive, median age 61 years (range 15–93 yrs), disease duration 7 years (range 0–68 yrs), Disease Activity Score on 28 joints (DAS28) 2.97 (range 0.96–8.61), HAQ score 0.63 (range 0–3), SF-12 PCS 56 (range 6–99), and SF-12 MCS 57 (range 16–99). Variation in HAQ was associated with 12 of 15 possible variables (R2 0.41), in PCS and MCS with 6 of 15 variables (R2 0.02 and 0.05). Patients with moderate to high DAS28 and ≥ 3 comorbid conditions had consistently worse HAQ and SF-12 scores compared to the reference groups, while weekly exercise was associated with better scores compared to no exercise.Conclusion.The HAQ was more sensitive to differences in demographic, lifestyle, and disease- and treatment-related factors than the SF-12. The established clinical value and feasibility of the HAQ highlights its advantages over the SF-12 in describing health status in RA.

The Minimally Important Difference for the Health Assessment Questionnaire in Rheumatoid Arthritis Clinical Practice Is Smaller Than in Randomized Controlled Trials

2009 ◽  
Vol 36 (2) ◽  
pp. 254-259 ◽  
Author(s):  
JANET E. POPE ◽  
DINESH KHANNA ◽  
DEBORAH NORRIE ◽  
JANINE M. OUIMET
Keyword(s):  
Rheumatoid Arthritis ◽  
Clinical Practice ◽  
Health Assessment Questionnaire ◽  
Clinical Care ◽  
Damage Index ◽  
Health Assessment ◽  
Change Score ◽  
Minimally Important Difference ◽  
Patient Reported ◽  
Assessment Questionnaire

Objective.Patient-reported outcomes are used in clinical practice and trials. We studied a large clinical practice to determine the minimally important difference (MID) estimates for (1) the Health Assessment Questionnaire–Damage Index (HAQ-DI): improvement and worsening using patient global assessment anchor; and (2) pain using a patient-reported pain anchor.Methods.Patients with rheumatoid arthritis (RA; N = 225) had clinic visits at 2 timepoints within 1 year, completed the HAQ-DI and pain visual analog scale (VAS; 0–100 mm), and answered the question, “How would you describe your overall status/overall pain since the last visit?”, as much worsened, somewhat worsened, the same, somewhat improved, or much improved. If rated as somewhat improved or worsened, they were defined as the minimally changed subgroups.Results.Eighty-three percent were women, mean age 60 years, with disease duration 11.7 ± 10.7 years. The baseline HAQ-DI was 0.97 ± SD 0.76, and at followup 1.0 ± 0.77 (mean change +0.03 ± 0.40). The baseline pain VAS was 42.3 ± 28.8, and at followup 38.5 ± 27.9 (mean change −2.8 ± 25.9). The mean (SD) HAQ-DI change score was −0.09 (0.42) for somewhat improved and 0.15 (0.33) for somewhat worsened. The HAQ-DI change for somewhat/much better was −0.20 ± 0.52, and for somewhat/much worse +0.21 ± 0.33. For pain, somewhat improved changed by −11.9 mm on the VAS, and somewhat worsened by 6.8 mm. Estimates for HAQ-DI and pain were larger than the for no-change group, 0.03 (0.32) and −3.2 (20.9).Conclusion.The MID for HAQ-DI in clinical practice is smaller than it is in trials. This may have implications for observational studies and clinical care.

scholarly journals SAT0321 CURRENT PATIENT REPORTED OUTCOMES (PROS) POORLY REFLECT CHANGES IN LUNG FUNCTION IN PATIENTS WITH SYSTEMIC SCLEROSIS

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 1106.1-1106
Author(s):  
F. Danzo ◽  
K. Gjeloshi ◽  
G. Abignano ◽  
A. M. Dean ◽  
F. Masini ◽  
...  
Keyword(s):  
Systemic Sclerosis ◽  
Lung Function ◽  
Health Assessment Questionnaire ◽  
Patient Reported Outcomes ◽  
Health Assessment ◽  
Digital Ulcers ◽  
Strong Positive Correlation ◽  
Patient Reported ◽  
Assessment Questionnaire

Background:Lung involvement is very common is systemic sclerosis (SSc). Approximately one quarter of patients develops pulmonary problems within the first 3 years of diagnosis and still represents the leading cause of death in these patients. In a recent clinical trail, the reduction of FVC was not accompanied by a benefit with respect to health-related quality of life and patient-reported outcomes (PROs).Objectives:To assess how the change in Pulmonary Function Test (PFTs) parameters correlates with the Patient Reported Outcomes (PROs) in an observational cohort of patients with Systemic Sclerosis (SSc).Methods:We conducted a retrospective study of 330 clinic episodes from 121 unselected patients diagnosed with systemic sclerosis according to EULAR/ACR 2013 criteria, in annual follow-up (for a total of 165 patients/year) with PFTs, Health Assessment Questionnaire Disability Index (HAQ-DI), Scleroderma Health Assessment Questionnaire (sHAQ), Modified Borg Dyspnea Scale (Borg) and Cochin Hand Function Score (CHFS). We assessed the correlation between the HAQ and the Visual Analogical Scale 1-7 at baseline (VAS1 pain, VAS2 disease severity, VAS3 arthritis activity, VAS4 intestinal problems, VAS5 dyspnea, VAS6 Raynaud’s phenomenon, VAS7 digital ulcers). We evaluated the correlation of PFTs with PROs at every time period and the correlation between the change of PFTs parameters (δFVC, δDLCO) with the change of the PROs over a year of follow-up. Following analysis of distribution, Spearman or Pearson Test were used to determine correlation coefficients, as appropiate (Prism 7).Results:The median disease duration was 5 years (IQR 3-10). The median of 12 months δFVC% and δDLCO% were 0 (IQR -5.81 to 3.28) and -2.439 (IQR -8.76 to 5.98), respectively. The analysis evidenced a strong positive correlation between VAS1-7 and HAQ. We observed also significant correlation between FVC%, DLCO% and HAQ-DI (r= - 0.355 and -0.266, respectively; p<0.0001 for both), Borg (r= -0.403 and -0.379, respectively; p<0.0001) and CHFS (r = -0.355 and -0.256, respectively; p<0.0001). Nevertheless, in longitudinal setting there was no significant correlation between δPROs and changes lung function, as continuous variables, neither there was any significant PROs difference in patients that did or did not lose more than 10% of FVC and DLCO over a year of follow-up.Conclusion:This analysis of a monocentric non-selected population evidenced that the current commonly used PROs in SSc while showing a good correlation with lung function are poorly sensitive to change or to reflect changes in lung function over 12 months. In this sense, prudent interpretation of the lack of correlation between FVC and patient-reported outcomes in studies of phase 3 is warranted.References:[1]Rahimi S., Nintedanib for systemic sclerosis-associated interstitial lung disease, Lancet Respiratory Medicine (2020)Disclosure of Interests:None declared

The Minimally Important Difference for Patient-reported Outcomes in Spondyloarthropathies including Pain, Fatigue, Sleep, and Health Assessment Questionnaire

2010 ◽  
Vol 37 (4) ◽  
pp. 816-822 ◽  
Author(s):  
LAURA WHEATON ◽  
JANET POPE
Keyword(s):  
Health Assessment Questionnaire ◽  
Patient Reported Outcomes ◽  
Disease Duration ◽  
Health Assessment ◽  
Analog Scale ◽  
Patient Reported ◽  
The Mean ◽  
Minimal Important Differences ◽  
Assessment Questionnaire ◽  
Previous Visit

Objective.To study minimal important differences (MID) in spondyloarthropathies (SpA). MID are important in determining clinically relevant changes and for interpretation of trials and treating patients. MID have been widely studied in rheumatoid arthritis, but less so in SpA.Methods.Patients with SpA had to be seen for 2 consecutive visits and have completed the Health Assessment Questionnaire (HAQ) and 100 mm visual analog scale on both visits for fatigue, pain, sleep, and global assessment. At the second visit they had to answer a question regarding any change in their overall health (from last visit), responding with much better, better, same, worse, or much worse. The MID were the mean changes for those who were either better or worse.Results.Our study involved 140 eligible patients with a SpA: 69% were men, the mean age was 45 years, and the mean disease duration was 14.5 years. Almost half the patients rated themselves as unchanged from the previous visit but the remainder were either better or worse, with a minority rating themselves as much better or much worse. The MID for better and worse outcomes were HAQ (−0.136; 0.220), pain (−6.93; 18.97), fatigue (−1.43; 14.42), and sleep (−2.23; 10.76). No gender differences were observed.Conclusion.Our results demonstrate that the MID vary depending on better versus worse (bidirectionally different). MID may be smaller in clinical practice than what is observed in trials.

The Minimally Important Difference in Clinical Practice for Patient-centered Outcomes Including Health Assessment Questionnaire, Fatigue, Pain, Sleep, Global Visual Analog Scale, and SF-36 in Scleroderma

2010 ◽  
Vol 37 (3) ◽  
pp. 591-598 ◽  
Author(s):  
SUNEET SEKHON ◽  
JANET POPE ◽  
MURRAY BARON ◽  
Keyword(s):  
Clinical Practice ◽  
Visual Analog Scale ◽  
Health Assessment Questionnaire ◽  
Health Assessment ◽  
Minimally Important Difference ◽  
Analog Scale ◽  
Sf 36 ◽  
Patient Reported ◽  
Global Status ◽  
Assessment Questionnaire

Objective.We studied a large clinical practice and multicenter database to estimate the minimally important difference (MID) in systemic sclerosis (SSc) using global rating of change anchors for the Health Assessment Questionnaire-Disability Index (HAQ-DI) and visual analog scale (VAS) in pain, fatigue, sleep, global status, and the Medical Outcomes Study Short-Form 36 (SF-36) in clinical practice.Methods.Longitudinal data were collected from a scleroderma clinic on patients with scleroderma (n = 109) who had completed the HAQ-DI and pain/fatigue/sleep/global status VAS (0 to 100 mm) questionnaires at 2 consecutive visits, and rated their change in overall status since the last visit as much better, better, same, worse, or much worse. Data were extracted from the Canadian Scleroderma Research Group (CSRG) database (n = 341) for 2 consecutive annual visits where the patients had completed HAQ-DI and SF-36, and the SF-36 “change in health” item.Results.For the single site, the mean baseline HAQ-DI was 0.895 and 0.911 at followup, with a mean change of 0.016. The MID estimates for improvement and worsening respectively were –0.0125 (0.125, 75th percentile)/0.042 (0.217, 75th percentile) for HAQ-DI, –8.00/3.61 for pain, –10.00/3.79 (25.32) for fatigue, –18.50/5.92 for sleep, and –6.70/4.05 for global VAS. In the CSRG, baseline scores were 0.787 for HAQ-DI, 37.20 for the Physical Component Summary (PCS) of SF-36, and 48.57 for the Mental Component Summary (MCS). The MID estimates for improvement and worsening were –0.037 (0.250, 75th percentile)/0.140 (0.375, 75th percentile) for HAQ-DI, 2.18/–1.74 for PCS, and 1.33/–2.61 for MCS.Conclusion.This study provides MID estimates in SSc from 2 large databases for commonly used patient-reported outcomes in a clinical practice setting, which could differ from MID in trials.

scholarly journals P159 Current patient reported outcomes (PROs) poorly reflect changes in lung function in patients with systemic sclerosis

Rheumatology ◽  
2020 ◽  
Vol 59 (Supplement_2) ◽  
Author(s):  
Fiammetta Danzo ◽  
Klodian Gjeloshi ◽  
Giuseppina Abignano ◽  
Anne-Maree Dean ◽  
Francesco Masini ◽  
...  
Keyword(s):  
Systemic Sclerosis ◽  
Lung Function ◽  
Health Assessment Questionnaire ◽  
Patient Reported Outcomes ◽  
Health Assessment ◽  
Digital Ulcers ◽  
Strong Positive Correlation ◽  
Patient Reported ◽  
Assessment Questionnaire

Abstract Background In a recent clinical trail the reduction of FVC was not accompanied by a benefit with respect to health-related quality of life and patient-reported outcomes (PROs). The aim of this study is to assess how the change in Pulmonary Function Test (PFTs) parameters correlates with the patient reported outcomes (PROs) in an observational cohort of patients with systemic sclerosis (SSc). Methods We conducted a retrospective study of 330 clinic episodes from 121 unselected patients diagnosed with systemic sclerosis according to EULAR/ACR 2013 criteria, in annual follow-up (for a total of 165 patients/year) with PFTs, Health Assessment Questionnaire Disability Index (HAQ-DI), Scleroderma Health Assessment Questionnaire (sHAQ), Modified Borg Dyspnea Scale (Borg) and Cochin Hand Function Score (CHFS). We assessed the correlation between the HAQ and the Visual Analogical Scale 1-7 at baseline (VAS1 pain, VAS2 disease severity, VAS3 arthritis activity, VAS4 intestinal problems, VAS5 dyspnea, VAS6 Raynaud’s phenomenon, VAS7 digital ulcers). We evaluated the correlation of PFTs with PROs at every time period and the correlation between the change of PFTs parameters (δFVC, δDLCO) with the change of the PROs over a year of follow-up. Following analysis of distribution, Spearman or Pearson Test were used to determine correlation coefficients, as appropriate (Prism 7). Results The median disease duration was 5 years (IQR 3-10). The median of 12 months δFVC% and δDLCO% were 0 (IQR -5.81 to 3.28) and -2.439 (IQR -8.76 to 5.98), respectively. The analysis evidenced a strong positive correlation between VAS1-7 and HAQ. We observed also significant correlation between FVC%, DLCO% and HAQ-DI (r= -0.355 and -0.266, respectively; p &lt; 0.0001 for both), Borg (r= -0.403 and -0.379, respectively; p &lt; 0.0001) and CHFS (r = -0.355 and -0.256, respectively; p &lt; 0.0001). Nevertheless, in longitudinal setting there was no significant correlation between δPROs and changes lung function, as continuous variables, neither there was any significant PROs difference in patients that did or did not lose more than 10% of FVC and DLCO over a year of follow-up. Conclusion This analysis of a monocentric non-selected population evidenced that the current commonly used PROs in SSc while showing a good correlation with lung function are poorly sensitive to change or to reflect changes in lung function over 12 months. In this sense, prudent interpretation of the lack of correlation between FVC and patient-reported outcomes in studies of phase 3 is warranted. Disclosures F. Danzo: None. K. Gjeloshi: None. G. Abignano: None. A. Dean: None. F. Masini: None. G. Cuomo: None. F. del Galdo: None.

Minimally Important Difference of Health Assessment Questionnaire in Psoriatic Arthritis: Relating Thresholds of Improvement in Functional Ability to Patient-rated Importance and Satisfaction

2011 ◽  
Vol 38 (11) ◽  
pp. 2461-2465 ◽  
Author(s):  
PHILIP J. MEASE ◽  
J. MICHAEL WOOLLEY ◽  
BOJENA BITMAN ◽  
BRIAN C. WANG ◽  
DENISE R. GLOBE ◽  
...  
Keyword(s):  
Psoriatic Arthritis ◽  
Health Assessment Questionnaire ◽  
Mixed Model ◽  
Linear Mixed Model ◽  
Health Assessment ◽  
Minimally Important Difference ◽  
Double Blind Study ◽  
Double Blind ◽  
Link Type ◽  
Assessment Questionnaire

Objective.To evaluate changes in function as measured by Health Assessment Questionnaire Disability Index (HAQ-DI) and the meaningfulness of the changes, in importance and satisfaction, in patients with psoriatic arthritis (PsA).Methods.HAQ-DI was assessed at baseline and at Weeks 4, 12, and 24 in a randomized double-blind study of 205 patients with active PsA receiving etanercept 25 mg twice weekly or placebo. Concurrently, patients rated the importance of and satisfaction with their change in function on a 7-point scale (1 = not at all important/satisfied; 7 = extremely important/satisfied). Mean HAQ-DI improvement corresponding to ratings of minimally (2–3) or very (6–7) important or satisfied was determined using a posthoc linear mixed-model analysis. Patient importance ratings were used as an anchor to estimate minimally important difference (MID) for HAQ-DI; distribution-based estimates were also calculated.Results.A total of 161 patients (69 placebo; 92 etanercept) had ≥ 1 HAQ-DI scores showing improvement from baseline and a corresponding importance or satisfaction rating. HAQ-DI improvements corresponding to importance scale ratings of 2 or 3 were 0.335 (95% CI 0.214, 0.455) and 0.360 (95% CI 0.263, 0.456), respectively, suggesting an MID of about 0.35. HAQ-DI improvements corresponding to satisfaction scale ratings of 2 and 3 were 0.293 (95% CI 0.230, 0.357) and 0.360 (95% CI 0.307, 0.413). For a given change in HAQ-DI, nearly two-thirds of patients indicated a lower rating for satisfaction than for importance. This trial was registered in the ClinicalTrials.gov registry (NCT00317499).Conclusion.Our study suggests the MID for HAQ-DI in PsA is about 0.35. The results may also provide insight into patient satisfaction with changes in function and expectations for therapy.

scholarly journals FRI0048 NUMBER NEEDED TO TREAT TO ACHIEVE MINIMUM CLINICALLY SIGNIFICANT DIFFERENCES IN PATIENT-REPORTED OUTCOMES IN PATIENTS TREATED WITH BARICITINIB

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 599.1-600
Author(s):  
V. Strand ◽  
L. Sun ◽  
J. Ross Terres ◽  
C. L. Kannowski
Keyword(s):  
Physical Function ◽  
Patient Reported Outcomes ◽  
Bodily Pain ◽  
Health Assessment ◽  
Number Needed To Treat ◽  
Chronic Illness Therapy ◽  
Sf 36 ◽  
Patient Reported ◽  
Assessment Questionnaire ◽  
Questionnaire Disability

Background:Baricitinib (BARI) provided rapid and sustained improvements in patient-reported outcomes (PROs) in randomized, controlled trials (RCTs) in patients (pts) with active rheumatoid arthritis (RA) and inadequate responses (IR) to methotrexate (MTX) (RA-BEAM;NCT01710358)1,2and biologic DMARDs (bDMARD-IR; RA-BEACON;NCT01721044)3,4.Objectives:To determine the number needed to treat (NNT) to report improvements ≥minimum clinically important differences (MCIDs) in multiple PROs at Week (Wk) 12 after treatment with BARI 4-mg in RA-BEAM and BARI 2-mg or BARI 4-mg in RA-BEACON. NNTs ≤10 vs placebo (PBO) are considered clinically meaningful.Methods:Evaluated PROs with respective MCID definitions included Patient Global Assessment of Disease Activity (PtGA, 0-100 mm visual analog scale [VAS], MCID ≥10 mm), pain (0-100 mm VAS, MCID ≥10 mm), physical function (Health Assessment Questionnaire-Disability Index, MCID ≥0.22 points), fatigue (Functional Assessment of Chronic Illness Therapy-Fatigue [FACIT-F], MCID≥4.0), and health-related quality of life (SF-36 physical component summary [PCS: MCID ≥2.5] and domain scores: physical function [PF], role physical [RP], bodily pain [BP], general health [GH], vitality [VT], social functioning [SF], role emotional [RE], mental health [MH], MCID ≥5.0).5The percentages of pts reporting improvements ≥MCID were determined at Wk 12. NNTs were calculated as 1/difference in response rates between BARI 2-mg or 4-mg and PBO.Results:At Wk 12, percentages of pts reporting clinically meaningful improvements were greater and statistically different from PBO (p<0.01) with BARI 2-mg and 4-mg across most PROs in both RCTs. Most NNTs were ≤10. (Figure)Conclusion:Across different populations, MTX-IR and bDMARD-IR pts with active RA reported clinically meaningful improvements in PROs after BARI treatment. The NNTs in these analyses indicate that <10 pts need to be treated with BARI 2- or 4-mg to report a clinically meaningful benefit.References:[1]Taylor et al. NEJM, 2017;376: 652-62[2]Keystone et al. Ann Rheum Dis, 2017;76:1853-61[3]Genovese et al. NEJM, 2016; 374: 1243-52[4]Smolen et al. Ann Rheum Dis, 2017; 76: 694-700[5]Strand et al. J Rheumatol, 2011; 38: 1720-27Figure.Percentages of patients reporting improvements ≥MCID with baricitinib vs placebo and associated NNTs for baricitinib in RA-BEAM and RA-BEACON. *p<0.05; **p<0.01; ***p<0.001. Abbreviations: BP, bodily pain; FACIT-F, Functional Assessment of Chronic Illness Therapy-Fatigue; GH, general health; HAQ-DI, Health Assessment Questionnaire-Disability Index; MCID, minimum clinically important difference; MH, mental health; NA, not applicable (ie, difference between treatment and placebo is not statistically significant, confidence interval of NNT is not calculated); NNT, numbers needed to treat; Pain, Patient’s assessment of pain; PCS, physical component score; PF, physical function; PtGA, Patient’s Global Assessment of Disease Activity; RE, role emotional; RP, role physical; SF-36, Short Form-36; SF, social functioning; VT, vitalityDisclosure of Interests:Vibeke Strand Consultant of: AbbVie, Amgen, Biogen, Celltrion, Consortium of Rheumatology Researchers of North America, Crescendo Bioscience, Eli Lilly, Genentech/Roche, GlaxoSmithKline, Hospira, Janssen, Merck, Novartis, Pfizer, Regeneron Pharmaceuticals, Inc., Sanofi, UCB, Luna Sun Shareholder of: Eli Lilly and Company, Employee of: Eli Lilly and Company, Jorge Ross Terres Shareholder of: Eli Lilly and Company, Employee of: Eli Lilly and Company, Carol L. Kannowski Shareholder of: Eli Lilly and Company, Employee of: Eli Lilly and Company

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