scholarly journals Assessment of disability in idiopathic inflammatory myopathy: a call for linearity

Rheumatology ◽  
2021 ◽  
Author(s):  
Minoesch Min ◽  
Anne W Walter ◽  
Johan Lim ◽  
Filip Eftimov ◽  
Camiel Verhamme ◽  
...  
Keyword(s):  
Construct Validity ◽  
Inflammatory Myopathy ◽  
Health Assessment ◽  
Idiopathic Inflammatory Myopathy ◽  
Change Score ◽  
Disability Score ◽  
Academic Medical Centre ◽  
Group Validity ◽  
Patient Reported

Abstract Objectives To evaluate the clinimetric properties of the Academic Medical Centre Disability Score (ALDS) in patients with idiopathic inflammatory myopathy (IIM). Methods We used prospectively collected data of IIM patients who completed a phase-2 study with first-line IVIg monotherapy. The ALDS is a patient reported questionnaire which contains 25 items relevant for disability in myositis. ALDS and all core set measures (CSMs) for myositis (including Health Assessment Questionnaire-Disability Index (HAQ-DI)) were evaluated at baseline and 9 weeks follow-up. In addition, the 2016 ACR/EULAR myositis response criteria outcome called Total Improvement Score (TIS) was evaluated at 9 weeks. We examined floor/ceiling effects, reliability and construct validity of the ALDS. To examine known-group validity, ALDS change scores over time were compared with TIS and physician impression of clinical response (PICR). Results Nineteen patients with IIM (median age 59 years, 12 (63%) female) were enrolled. At baseline, ALDS showed a median score of 65.4 (IQR 58.2–73.5), good Cronbach’s alpha (α = 0.84) and a small ceiling effect (11%). Construct validity was confirmed by moderate to strong correlations between ALDS and HAQ-DI (rs=-0.57 (baseline); -0.86 (follow-up)). ALDS change score correlated with TIS (rs=0.70), discriminated between responders and non-responders (TIS ≥ 40; p= 0.001), between groups based on PICR (p= 0.03), and detected deterioration. Conclusion The ALDS showed promising clinimetric properties and detected relevant changes in disability in patients with myositis. These results warrant further investigations.

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

2009 ◽  
Vol 36 (6) ◽  
pp. 1150-1157 ◽  
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.

scholarly journals The 27-Item Multiple Sclerosis Quality of Life Questionnaire: A New Brief Measure Including Treatment Burden and Work Life

2021 ◽  
Author(s):  
Helen Beckmann ◽  
Christoph Heesen ◽  
Matthias Augustin ◽  
Christine Blome
Keyword(s):  
Quality Of Life ◽  
Multiple Sclerosis ◽  
Construct Validity ◽  
Convergent Validity ◽  
Life Questionnaire ◽  
Quality Of Life Questionnaire ◽  
Patient Reported ◽  
Disease Specific

Abstract Background: Treatment- and work-related aspects have been neglected in health-related quality of life (HRQOL) measures in multiple sclerosis (MS). We aimed to develop a brief instrument covering all important impairment-, activity-, participation-, and treatment-related aspects for use in research and practice. Methods: The 27-item Multiple Sclerosis Quality of Life Questionnaire (MS-QLQ27) was developed using open item collection, a multidisciplinary expert panel, and cognitive pretesting. It was evaluated for reliability, construct validity, and responsiveness with 100 patients presenting with relapse (84 at follow-up ~14 days later). Construct validity was analyzed by correlating the MS-QLQ27 with the disease-specific Hamburg Quality of Life Questionnaire in MS (HAQUAMS) and generic HRQOL instuments. The Expanded Disability Status Scale (EDSS) was used to analyze known-groups validity. Responsiveness was determined as the correlation of changes in MS-QLQ27 scores with changes in validation criteria. Results: Internal consistency was high (Cronbach α = 0.94 at baseline and 0.93 at follow-up). Convergent validity was supported by direction and magnitude of associations with disease-specific and generic instruments. Correlations with change in convergent criteria were strong, indicating responsiveness. The HAQUAMS showed the strongest associations with the MSQLQ27. The MS-QLQ27 showed the highest effect size compared with other patient-reported outcomes and the EDSS. It successfully distinguished between levels of disease severity. Conclusions: These results indicate that the MS-QLQ27 is a reliable, valid, and highly responsive instrument for assessing HRQOL during relapse evolution in MS. Its advantages are that it is brief yet comprehensive, covering work- and treatment-related aspects not addressed in previous measures.

scholarly journals Responsiveness of EQ-5D in Patients With a Distal Radius Fracture

Hand ◽  
2017 ◽  
Vol 13 (5) ◽  
pp. 572-580 ◽  
Author(s):  
Johanna Rundgren ◽  
Anders Enocson ◽  
Cecilia Mellstrand Navarro ◽  
Gunnar Bergström
Keyword(s):  
Distal Radius Fracture ◽  
Distal Radius ◽  
Controlled Trial ◽  
Roc Curves ◽  
Change Score ◽  
Radius Fracture ◽  
External Criterion ◽  
Patient Reported ◽  
External Responsiveness

Background: The EuroQol Group 5-Dimension (EQ-5D) questionnaire is frequently used as an outcome measure of general patient-reported health-related quality of life (HRQoL). We evaluated the internal and external responsiveness of EQ-5D (specifically EQ-5Dindex score) in patients with a surgically treated distal radius fracture. Methods: Within the context of a randomized controlled trial (RCT), 132 patients with a surgically treated distal radius fracture filled out EQ-5D and Patient-Rated Wrist Evaluation (PRWE-Swe) at baseline (preinjury state), and at 3 and 12 months. Internal responsiveness was evaluated by calculating mean change score and standardized response mean (SRM) of the EQ-5Dindex scores. External responsiveness was evaluated with PRWE-Swe as the external criterion. PRWE-Swe was used to define 4 subgroups of patients with different clinical outcomes. The ability of EQ-5Dindex change score to discriminate between these subgroups was analyzed with logistic regression, receiver operating characteristic (ROC) curves, and area under the ROC curves (AUROCs). Correlation analysis was made with Spearman’s ρ test. Results: The mean change in EQ-5Dindex score from baseline to the 3-month follow-up was −16.1 and from the 3- to the 12-month follow-up was 7.6. The corresponding SRMs were large (0.93) and small to moderate (0.47). Analysis of external responsiveness displayed odds ratios >1 and AUROCs between 0.70 and 0.76 in all 4 subgroups. The proportion of patients correctly classified into the 4 subgroups by the EQ-5Dindex change scores was 78% to 94%. Spearman’s ρ was 0.35. Conclusion: EQ-5D displayed an overall acceptable to good responsiveness in patients with a distal radius fracture. It may thus be used as a measure of HRQoL in this patient group.

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.

Education, Gender, and the Compression of Morbidity

1994 ◽  
Vol 39 (3) ◽  
pp. 233-246 ◽  
Author(s):  
J. Paul Leigh ◽  
James F. Fries
Keyword(s):  
Health Assessment ◽  
Good Health ◽  
Compression Of Morbidity ◽  
Disability Score ◽  
Cross Sectional ◽  
Health And Nutrition ◽  
Healthy Habits ◽  
Stanford Health Assessment Questionnaire ◽  
Independent Variable

According to the Compression of Morbidity (CM) hypothesis, people who exercise, eat nutritiously, do not smoke, and maintain good weight, i.e., people who practice healthy habits, will be more likely to live free of disabling diseases and injuries up until the last few months or years of life. The Increasing Misery (IM) hypothesis, on the other hand, holds that preventive health measures will extend life expectancy but will also increase the number of infirm years. The CM theory implies that curves of morbidity or disability with age should become increasingly “rectangular” for groups who practice healthy habits in the broadest sense. The IM theory does not. This Rectangularization hypothesis is examined with cross-sectional data measuring disability from the Epidemiological Follow-up to the National Health and Nutrition Examination Survey, I (NHEFS), using years of schooling as the independent variable proxy representing favored health status, and examining interactions with age. A modified version of the Disability Index (DI) from the Stanford Health Assessment Questionnaire (HAQ) is used to measure disability. In some analyses, deceased subjects were assigned the worst disability score. Four subsamples of women and men, fifty years old and over, alive and deceased in 1982–84, were analyzed. Female, and especially male, subsamples which included the deceased provided evidence for the CM hypothesis. Results for the subsamples of those remaining alive in 1982–84 were ambiguous. However, lifetime (over age 50) cumulative disability was 21 to 60 percent less for the more educated than the less educated, depending upon whether deceased were included or excluded. If higher education level is an appropriate surrogate for the effect of good health practices, then extending such practices will result in less, rather than more, lifetime disability.

Wrist-worn accelerometer as innovative tool for longitudinal follow-up of idiopathic inflammatory myopathy patients: A pilot study

2015 ◽  
Vol 25 ◽  
pp. S310 ◽  
Author(s):  
Y. Allenbach ◽  
A. Foucher ◽  
N. Champtiaux ◽  
L. Gilardin ◽  
B. Hervier ◽  
...  
Keyword(s):  
Pilot Study ◽  
Inflammatory Myopathy ◽  
Idiopathic Inflammatory Myopathy ◽  
Innovative Tool

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

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

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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