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

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 < 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. Disclosures F. Danzo: None. K. Gjeloshi: None. G. Abignano: None. A. Dean: None. F. Masini: None. G. Cuomo: None. F. del Galdo: None.

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

Annals of the Rheumatic Diseases ◽

10.1136/annrheumdis-2020-eular.6494 ◽

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

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FRI0241 INFLUENCE OF PATIENT REPORTED ‘’ARTHRITIS ACTIVITY’’ IN DETERMINING SHAQ, HAQ-DI AND COCHIN SCORES IN SYSTEMIC SCLEROSIS

Annals of the Rheumatic Diseases ◽

10.1136/annrheumdis-2020-eular.3886 ◽

2020 ◽

Vol 79 (Suppl 1) ◽

pp. 704.1-705

Author(s):

K. Gjeloshi ◽

F. Danzo ◽

G. Abignano ◽

A. M. Dean ◽

F. Masini ◽

...

Keyword(s):

Systemic Sclerosis ◽

Health Assessment Questionnaire ◽

Pulmonary Function Tests ◽

Health Assessment ◽

Joint Involvement ◽

Wide Range ◽

Patient Reported ◽

Hand Disability ◽

Assessment Questionnaire

Background:Arthritic involvement in systemic sclerosis (SSc) is a common manifestation impacting on the quality of life. A wide range of articular involvement is recognised including from arthralgia to inflammatory joint and tendon disease.Objectives:To assess the weight of patient reported ‘’arthritis activity’’ in determining Patient Reported Outcome Measures (PROMs) in an observational cohort of SSc patients.Methods:We conducted a retrospective study of 330 clinic episodes from 121 unselected patients diagnosed with SSc according to EULAR/ACR 2013 classification criteria, in annual follow-up (for a total of 165 patients/year) with Pulmonary Function Tests (PFTs), Health Assessment Questionnaire - Disability Index (HAQ-DI), Scleroderma Health Assessment Questionnaire (sHAQ) and Cochin Hand Function Score (CHFS). Hand disability index was assessed by CHFS and global disability index was assessed by HAQ and sHAQ. Patient reported arthritis activity was assessed by Visual Analogical Scale for Arthritis Activity (VAS3). Based on the median of VAS3, patients were classified in two groups and the evaluation of global and hand disability index was performed for each group. Furthermore, we assessed the correlation between the change of VAS3 and the modification of disability scores (ΔHAQ, ΔSHAQ, ΔCHFS) over 12 months 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 VAS3 was 35 (IQR 2 - 66). In patients with VAS3 <35 and VAS ≥35 the HAQ-DI medians were 0.625 (IQR 0.25 - 1.5) and 1.75 (IQR 1.125-2.25) respectively, (p<0.0001); the sHAQ medians were 0.628 (IQR 0.255 - 1.114) and 1.701 (IQR 1.234-2.059), respectively, (p<0.0001); and the CHFS medians were 4 (IQR 0 – 19) and 28 (IQR 10 – 46) respectively, (p<0.0001). A significant correlation was observed between VAS3 and HAQ (r= 0.463, p<0.0001), SHAQ (r=0.651, p<0.0001), CHFS (r=0.497, p<0.0001); between ΔVAS3 and ΔSHAQ (r=0.493, p<0.0001).Conclusion:This analysis of a monocentric non-selected population supports the key role of joint involvement in determining global patient reported functional and hand disability in SSc. Severity of musculoskeletal involvement should be carefully considered when interpreting PROs in patients with SSc.References:[1]Sandler RD, Matucci-Cerinic M, Hughes M. Musculoskeletal hand involvement in systemic sclerosis. Seminars in Arthritis and Rheumatism (2019)Disclosure of Interests:None declared

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P150 Influence of patient reported arthritis activity in determining sHAQ, HAQ-DI and Cochin scores in systemic sclerosis

Rheumatology ◽

10.1093/rheumatology/keaa111.145 ◽

2020 ◽

Vol 59 (Supplement_2) ◽

Author(s):

Klodian Gjeloshi ◽

Fiammetta Danzo ◽

Giuseppina Abignano ◽

Anne-Maree Dean ◽

Francesco Masini ◽

...

Keyword(s):

Systemic Sclerosis ◽

Health Assessment Questionnaire ◽

Pulmonary Function Tests ◽

Hand Function ◽

Health Assessment ◽

Joint Involvement ◽

Patient Reported ◽

Hand Disability ◽

Assessment Questionnaire

Abstract Background Arthritic involvement is a common manifestation in systemic sclerosis (SSc). The aim of this study is to assess the weight of patient reported arthritis activity in determining Patient Reported Outcome Measures (PROMs) in an observational cohort of SSc patients. Methods We conducted a retrospective study of 330 clinic episodes from 121 unselected patients diagnosed with SSc according to EULAR/ACR 2013 classification criteria, in annual follow-up (for a total of 165 patients/year) with Pulmonary Function Tests (PFTs), Health Assessment Questionnaire - Disability Index (HAQ-DI), Scleroderma Health Assessment Questionnaire (sHAQ) and Cochin Hand Function Score (CHFS). Hand disability index was assessed by CHFS and global disability index was assessed by HAQ and sHAQ. Patient reported arthritis activity was assessed by Visual Analogical Scale for Arthritis Activity (VAS3). Based on the median of VAS3, patients were classified in two groups and the evaluation of global and hand disability index was performed for each group. Furthermore, we assessed the correlation between the change of VAS3 and the modification of disability scores (ΔHAQ, ΔSHAQ, ΔCHFS) over 12 months 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 VAS3 was 35 (IQR 2 - 66). In patients with VAS3 <35 and VAS ≥35 the HAQ-DI medians were 0.625 (IQR 0.25 - 1.5) and 1.75 (IQR 1.125-2.25) respectively, (p < 0.0001); the sHAQ medians were 0.628 (IQR 0.255 - 1.114) and 1.701 (IQR 1.234-2.059), respectively, (p < 0.0001); and the CHFS medians were 4 (IQR 0 - 19) and 28 (IQR 10 - 46) respectively, (p < 0.0001). A significant correlation was observed between VAS3 and HAQ (r = 0.463, p < 0.0001), SHAQ (r = 0.651, p < 0.0001), CHFS (r = 0.497, p < 0.0001); between ΔVAS3 and ΔSHAQ (r = 0.493, p < 0.0001). Conclusion This analysis of a monocentric non-selected population supports the key role of joint involvement in determining global patient reported functional and hand disability in SSc. Severity of musculoskeletal involvement should be carefully considered when interpreting PROs in patients with SSc. Disclosures K. Gjeloshi: None. F. Danzo: None. G. Abignano: None. A. Dean: None. F. Masini: None. G. Cuomo: None. F. del Galdo: None.

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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 ◽

10.1093/rheumatology/keaa111.153 ◽

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.

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

The Journal of Rheumatology ◽

10.3899/jrheum.090832 ◽

2010 ◽

Vol 37 (5) ◽

pp. 1024-1028 ◽

Cited By ~ 34

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.

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

The Journal of Rheumatology ◽

10.3899/jrheum.090086 ◽

2010 ◽

Vol 37 (4) ◽

pp. 816-822 ◽

Cited By ~ 18

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.

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POS0319 PERFORMANCE OF PATIENT REPORTED OUTCOMES (PROs) IN SCLERODERMA PATIENTS WITH REDUCED LUNG FUNCTION IN AN OBSERVATIONAL COHORT

Annals of the Rheumatic Diseases ◽

10.1136/annrheumdis-2021-eular.3505 ◽

2021 ◽

Vol 80 (Suppl 1) ◽

pp. 387.1-387

Author(s):

M. G. Lazzaroni ◽

G. Abignano ◽

M. Wilson ◽

V. Kakkar ◽

F. Del Galdo

Keyword(s):

Lung Function ◽

Patient Reported Outcomes ◽

Hand Function ◽

Health Assessment ◽

Digital Ulcers ◽

General Function ◽

Research Support ◽

Cross Sectional ◽

Patient Reported ◽

Spearman Test

Background:Patient Reported Outcomes (PROs) are used to capture disease impact on patients’ Health Related Quality of Life (HRQoL) and they have been increasingly used as endpoints in clinical trials for Systemic Sclerosis (SSc). The DeSScipher project within the EUSTAR group highlighted dyspnea as one of the factors more strongly related with the highest SHAQ scores (1). Nevertheless, the SENSCIS trial in SSc-ILD (2) showed the efficacy of Nintedanib in reducing the annual rate of FVC loss, as compared to placebo, without significant changes in the PROs used as secondary endpoints (St. George’s Respiratory Questionnaire and the FACIT-Dyspnoea questionnaire). Since patient’s perspective is a crucial determinant to define the overall relevance of an intervention, the performance of PROs in reflecting different lung functional stages is a relevant issue in SSc.Objectives:To analyse in a prospective SSc cohort the inference of reduced lung function as measured by FVC on median PRO scores and the correlation among distinct commonly used PROs.Methods:A cross-sectional study was performed on data exported from the STRIKE database regarding SSc patients followed in Leeds Scleroderma Programme for SSc. Data included records of periodical visits with scores of different PROs commonly used in SSc (Scleroderma-Health Assessment Questionnaire (SHAQ), Cochin Hand Function Scale (CHFS) and Borg dyspnoea scale) and FVC%-predicted (%pFVC) and DLCO%-predicted (%pDLCO). SHAQ score was calculated as the mean value of HAQ (0-3) with the average of the 7-VAS scores divided by 3.33. The 7-VAS (score 1-10) were 1: pain, 2: general function, 3: arthritis, 4: gastro-intestinal, 5: dyspnoea, 6: Raynaud Phenomena; 7: digital ulcers. The correlation of FVC with distinct PROs, and the inter-PRO correlation, were analysed through the non-parametric Spearman test.Results:Complete data were available from 182 visits of 87 SSc patients (41 with diffuse and 46 with limited cutaneous involvement). Mean %pFVC was 95.16 ±24.93 (median 95) and mean %pDLCO was 59.31±16.51 (median 59). Overall, FVC and DLCO showed a moderate correlation with SHAQ (r=-0.36, p<0.001 and r=-0.24, p:0.001 respectively), while Borg score showed a stronger negative correlation with FVC and DLCO (r:-0.42 and r-0.38, p<0.001 for both). In a sub-analysis of patients grouped by FVC, patients with FVC 50-70% showed a significant correlation of FVC with SHAQ (r =-0.47, p:0.012), which was not present in patients with FVC 70-90% (r:-0.23, p:0.13). VAS-5 dyspnoea and Borg were not associated with FVC in these two subgroups of patients.Inter PROs analysis showed that CHFS score had a stronger correlation with SHAQ than Borg dyspnoea score in the overall population (r:0.86 vs. r:0.57, both p<0.001).Conclusion:The analysis of a single centre prospective cohort of SSc patients, suggests a small inference of lung function in the overall SHAQ. The stronger correlation of SHAQ with CHFS, than with Borg score, suggests a higher weight of hand function on SHAQ in this population with relatively conserved lung function. In patients with %pFVC <70%, the correlation with SHAQ was stronger than in patients with %pFVC >70%, suggesting that mild reductions in FVC might not be perceived by the patients, or at least they might not modify HrQoL as measured by SHAQ.References:[1]Jaeger VK, et al. Rheumatology 2017;57(3):441-50.[2]Distler O, et al. N Engl J Med 2019; 380:2518-2528.Disclosure of Interests:Maria Grazia Lazzaroni Grant/research support from: Boehringer-Ingelheim, Giuseppina Abignano: None declared, Michelle Wilson: None declared, Vishal Kakkar: None declared, Francesco Del Galdo Consultant of: Astra-Zeneca, Boehringer-Ingelheim, Mitsubishi-Tanabe, Capella Biosciences, Kymab, Actelion, Grant/research support from: Astra-Zeneca, Boehringer-Ingelheim, Mitsubishi-Tanabe, Capella Biosciences, Kymab, Actelion

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

Annals of the Rheumatic Diseases ◽

10.1136/annrheumdis-2020-eular.1440 ◽

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

The Journal of Rheumatology ◽

10.3899/jrheum.090134 ◽

2009 ◽

Vol 36 (10) ◽

pp. 2183-2189 ◽

Cited By ~ 30

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.

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Validating and Assessing the Sensitivity of the Health Assessment Questionnaire-Disability Index-derived Short Form-6D in Patients with Early Aggressive Rheumatoid Arthritis

The Journal of Rheumatology ◽

10.3899/jrheum.080959 ◽

2009 ◽

Vol 36 (6) ◽

pp. 1150-1157 ◽

Cited By ~ 8

Author(s):

SOGOL S. AMJADI ◽

PAUL M. MARANIAN ◽

HAROLD E. PAULUS ◽

ROBERT M. KAPLAN ◽

VEENA K. RANGANATH ◽

...

Keyword(s):

Rheumatoid Arthritis ◽

Construct Validity ◽

Health Assessment Questionnaire ◽

Short Form ◽

Health Assessment ◽

Correlation Coefficients ◽

Patient Reported ◽

Clinical Measures ◽

Assessment Questionnaire ◽

Questionnaire Disability

Objective.New methodologies allow the scores for the Health Assessment Questionnaire-Disability Index (HAQ-DI) to be translated into preferences/utility scores. We evaluated the construct validity of the HAQ-DI-derived Short Form-6D (SF-6D) score and assessed its responsiveness to change over 6- and 12-month followup periods in patients with early aggressive rheumatoid arthritis (RA).Methods.Patients (n = 277) participating in an RA observational study completed self-reported measures of symptoms and the HAQ-DI at baseline and at 6 and 12 months. Total Sharp scores, C-reactive protein, and erythrocyte sedimentation rate were assessed along with clinical data. Construct validity was assessed by examining the association between SF-6D score and patient-reported and clinical measures using Spearman correlation coefficients. The responsiveness of SF-6D to change was assessed using patient and physician assessments of the disease as clinical anchors. The magnitude of responsiveness was calculated using SF-6D effect size (ES).Result.Mean SF-6D scores were 0.690, 0.720, and 0.723 at baseline and 6 and 12-month followup, respectively. Baseline patient-reported measures had moderate to high correlations with baseline SF-6D (r = 0.43 to 0.52); whereas clinical measures had negligible to low correlations with SF-6D (r = 0.001 to 0.32). ES was moderate for the groups that were deemed to have improved (ES 0.63–0.75) but negligible to small for those that did not (ES 0.13–0.46).Conclusion.Our data support the validity and responsiveness of the HAQ-DI derived SF-6D score in an early RA cohort. These results support the use of the HAQ-DI derived SF-6D in RA cohorts and clinical trials lacking preference-based measures.

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