Patient-acceptable Symptom State as an Outcome Measure in the Daily Care of Patients with Ankylosing Spondylitis

2012 ◽  
Vol 39 (7) ◽  
pp. 1424-1432 ◽  
Author(s):  
CARLOS RODRÍGUEZ-LOZANO ◽  
MARÍA-ÁNGELES GANTES ◽  
BEATRIZ GONZÁLEZ ◽  
JOSÉ A. HERNÁNDEZ-BERIAIN ◽  
ANTONIO NARANJO ◽  
...  

Objective.We assessed the prevalence of patients with ankylosing spondylitis (AS), rating their state as acceptable (patient-acceptable symptom state; PASS), among 190 patients with AS seen in daily practice. Factors associated with PASS status and PASS thresholds for outcome measures were also analyzed.Methods.The characteristics of patients with affirmative and negative assignment to PASS were compared. Associated factors were estimated by logistic regression models and PASS thresholds by the 75th percentile and receiver-operating characteristic curve methods.Results.A total of 77% of patients rated their state as acceptable (95% CI 62–91). These patients were taking fewer nonsteroidal antiinflammatory drugs and corticosteroids, practiced more exercise, had less anxiety and depression, and had lower values of all patient-reported outcome measures, physicians’ assessment, AS Disease Activity Score (ASDAS) and C-reactive protein. Lower values of Bath AS Disease Activity Index and physician’s global assessment were independent factors associated with acceptable symptom state. High rates of anxiety and depression were found in patients not in PASS. The thresholds with the 75th percentile approach were 4.55 for the BASDAI and 2.84 for the ASDAS. Fifty-three percent of patients in PASS had a high or very high disease activity state according to ASDAS cutoff values.Conclusion.A high percentage of patients with AS in daily practice declared that their symptom state was acceptable. PASS status correlated with physician global assessment and BASDAI. PASS thresholds for common recommended outcome measures were relatively high and many patients in PASS had unacceptably high disease activity states according to ASDAS. Other factors such as psychological problems may influence a negative PASS state.

Rheumatology ◽  
2020 ◽  
Author(s):  
Augusta Ortolan ◽  
Sofia Ramiro ◽  
Floris van Gaalen ◽  
Tore K Kvien ◽  
Robert B M Landewe ◽  
...  

Abstract Objective To develop an alternative Ankylosing Spondylitis Disease Activity Score (ASDAS) to be used in research settings in axial SpA (axSpA) when Patient Global Assessment (PGA) is unavailable in databases. Methods Longitudinal data from four axSpA cohorts and two randomized controlled trials were combined. Observations were randomly split in a development (N = 1026) and a validation cohort (N = 1059). Substitutes of PGA by BASDAI total score, single or combined individual BASDAI questions, and a constant value, were established in the development cohort. Conversion factors for each substitute were defined by Generalized Estimating Equations, obtaining seven ‘alternative’ formulae. Validation was performed in the validation cohort according to the OMERACT filter, taking into consideration: (i) truth (agreement with original-ASDAS in the continuous score, by intraclass correlation coefficient and in disease activity states, by weighted kappa); (ii) discrimination [standardized mean difference of ASDAS scores between high/low disease activity states defined by external anchors, e.g. Patient Acceptable Symptom State; agreement (kappa) in the percentage of patients reaching ASDAS improvement criteria according to alternative vs original formulae]; and (iii) feasibility. Results Comparing various options, alternative-ASDAS using BASDAI total as PGA replacement proved to be: truthful (intraclass correlation coefficient = 0.98, kappa = 0.90), discriminative [ASDAS scores between Patient Acceptable Symptom State no/yes: standardized mean difference = 1.37 (original-ASDAS standardized mean difference = 1.43); agreement with original-ASDAS in major improvement/clinically important improvement criteria: kappa = 0.93/0.88] and feasible (BASDAI total often available, as questions required for the ASDAS; conversion coefficient ≈ 1). Conclusion Alternative-ASDAS using BASDAI total score as PGA replacement is the most truthful, discriminative and feasible instrument.


Rheumatology ◽  
2019 ◽  
Vol 59 (1) ◽  
pp. 69-76 ◽  
Author(s):  
Jeanie Z Fei ◽  
Anthony V Perruccio ◽  
Justine Y Ye ◽  
Dafna D Gladman ◽  
Vinod Chandran

Abstract Objectives The Psoriatic Arthritis Disease Activity Score (PASDAS) and Disease Activity Index for Psoriatic Arthritis (DAPSA) are composite PsA disease activity measures. We sought to identify the PASDAS and DAPSA cut-off points consistent with patient acceptable symptom state (PASS), the threshold of symptoms beyond which patients consider themselves well, and examine PASS across published PASDAS and DAPSA thresholds for low, moderate and high disease activity. Methods We used a standard protocol including physician assessment and patient-reported outcomes to prospectively record measures required to calculate PASDAS and DAPSA. We identified PASS thresholds for the PASDAS and DAPSA using receiver operating characteristics curve analyses. We assessed the frequency of reporting acceptable symptom state across disease activity thresholds for PASDAS and DAPSA scores. Results A total of 229 patients (58.5% male, mean age 55.5 years, mean disease duration 17.1 years) were recruited. The PASS threshold for the PASDAS was 3.79 [area under the curve (AUC) 0.86, sensitivity 0.75, specificity 0.82] and for the DAPSA was 11.10 (AUC 0.91, sensitivity 0.89, specificity 0.82). With the PASDAS, 90% of patients defined as having low disease activity considered their symptom state acceptable, compared with 55% and 17% among those with moderate and high disease activity, respectively. With the DAPSA, 98% of patients in disease remission considered their symptom state acceptable compared with 85, 22 and 18% among those with low, moderate and high disease activity, respectively. Conclusion We have defined PASS thresholds for PASDAS and DAPSA. The PASDAS target for low disease activity and DAPSA targets of low disease activity or remission align well with PASS.


2012 ◽  
Vol 72 (7) ◽  
pp. 1221-1224 ◽  
Author(s):  
Irene E van der Horst-Bruinsma ◽  
Debra Jeske Zack ◽  
Annette Szumski ◽  
Andrew S Koenig

ObjectivesTo examine the impact (if any) of gender on the clinical, functional and patient-reported outcomes of treatment using data pooled from four controlled clinical trials.MethodsStudy data were pooled from four clinical control trials in which 1283 adult patients with active ankylosing spondylitis (AS) were treated with etanercept, sulfasalazine or placebo. Patients were stratified by gender and analysed for differences/similarities in baseline demographics, disease characteristics, and efficacy in AS outcome measures and safety and discontinuation rates after 12 weeks of therapy.ResultsSignificant baseline differences were observed between 326 female patients compared with 957 male patients. Female patients had an older mean age of disease onset (35.0 vs 31.2 years; p<0.001), shorter mean time of disease duration (7.4 vs 9.5 years; p<0.001) and lower mean baseline C-reactive protein (13.1 vs 20.9 mg/l; p<0.001); a lower proportion was HLA-B27 positive (76.3% vs 85.2%; p<0.001) compared with male patients. Women had significantly (p<0.001) smaller differences in all week 12 efficacy assessments including AS disease activity score (0.87 vs −1.08), Bath AS disease activity index (−19.22 vs −23.41) and Bath AS functional index (−13.89 vs −16.88) relative to men. A similar relationship was observed between women and men in the adjusted mean difference of nocturnal back pain (4.04, 95% CI 0.77 to 7.32; p<0.05), total back pain (3.80, 95% CI 0.77 to 7.32; p<0.05) and patient global assessment (4.79, 95% CI 1.51 to 8.08; p<0.01).ConclusionsWomen had a higher burden of disease and less improvement in AS outcome measures compared with men. This was observed despite women having a later disease onset of shorter duration; the mechanism behind this observation is unclear. Additional research is necessary to better understand female patients with AS and the burden of disease in this population.


2017 ◽  
Vol 45 (1) ◽  
pp. 62-69 ◽  
Author(s):  
Linda E. Dean ◽  
Gary J. Macfarlane ◽  
Gareth T. Jones

Objective.A chronic inflammatory condition manifesting in young adulthood, ankylosing spondylitis (AS) affects both physical and emotional quality of life (QOL). To inform future intervention strategies, this study aimed to (1) assess the QOL of patients with AS, and (2) identify potentially modifiable factors associated with reporting poor QOL.Methods.The Scotland Registry for Ankylosing Spondylitis collects clinical and patient-reported data on clinically diagnosed patients with AS across Scotland. QOL is measured using the ASQoL questionnaire [range: 0 (high) to 18 (poor)]. Potentially modifiable factors associated with reporting poor QOL (score 12–18) were examined using Poisson regression models, adjusted for a variety of demographic characteristics, plus various nonmodifiable factors. Results are given as risk ratios (RR) with 95% CI.Results.Data were available on 959 patients: 74% male, mean age 52 years (SD 13), median ASQoL 7.0 (interquartile range 2–12). Although many factors were univariately associated with poor QOL, 5 were identified as independent predictors: reporting moderate/severe fatigue (RR 1.60, 95% CI 1.13–2.28), poor physical function [Bath Ankylosing Spondylitis Functional Index (BASFI) ≥ 4: 3.46, 1.76–6.82], chronic widespread pain (CWP; 1.92, 1.33–2.75), high disease activity [Bath Ankylosing Spondylitis Disease Activity Index (BASDAI) ≥ 4: 1.52, 1.09–2.12], and poor spinal mobility [Bath Ankylosing Spondylitis Metrology Index (BASMI) ≥ 4: 1.52, 0.93–2.50]. For these factors, population-attributable risks ranged between 20% (disease activity) and 56% (physical function).Conclusion.We have identified 5 potentially modifiable factors independently associated with poor QOL. These findings provide evidence that in addition to traditional clinical targets (BASDAI, BASFI, and BASMI), focus on nonspecific symptoms (CWP and fatigue), perhaps with nonpharmacological therapies, may yield important improvements in QOL.


2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 1173.1-1174
Author(s):  
A. Ogdie ◽  
M. E. Husni ◽  
J. Scher ◽  
E. Craig ◽  
S. Reddy ◽  
...  

Background:While several outcome measures have been studied for use in clinical studies of psoriatic arthritis, little is known about thresholds of meaning such as minimal clinically important improvement (MCII).Objectives:To investigate the distribution of scores for candidate outcome measures for pragmatic trials in PsA and to calculate the MCII for each outcome measure.Methods:We performed a longitudinal cohort study within the Psoriatic Arthritis Research Consortium (PARC), a multi-center study based in the US. Patients completed validated PROs (patient reported outcomes) and rheumatologists completed skin, joint, enthesis and dactylitis scores at therapy initiation and follow up 12-16 weeks later. In addition, patients completed a global assessment of response at the follow up visit, categorizing their status as improved, stayed the same, or worsened and then ratied the importance of the change on a scale from 0-7.1We then calculated and plotted the change in each of the following measures: Routine Assessment of Patient Index Data (RAPID3), clinical Disease Activity of Psoriatic Arthritis (cDAPSA), Patient Reported Outcome Measure Information System (PROMIS) Global Health short form (10a) physical health (PH) subscore, patient pain assessment, patient global assessment (0-10 NRS), and physician global assessments (0-10 NRS) of the joints and overall. We calculated the MCII as the mean change in score (with 95% confidence interval) among patients who reported improvement and rated the level of improvement as “almost none/hardly at all” or “a little important.” Additionally, we calculated Spearman’s correlation coefficients between the measures and the global assessment of response.Results:Among 148 unique patients, 233 therapy change visits were eligible for analysis. The average age was 52.5 years, 52% were female and mean BMI was 29.6. Baseline RAPID3 was 11.1 (SD 6), cDAPSA 17.9 (SD 13.9), PROMIS PH 42 (SD 8), patient global 4.2 (SD 2.5), TJC 5.9 (SD 7.5), and SJC 2.9 (SD 4.5). TNFi comprised 61% of drug initiations, 21% were IL17i and the remainder were other biologics and oral systemic therapies. At follow up, 63 (27%) patients rated themselves as improved whereas 103 (44%) stayed the same and 67 (29%) reported worsening. The mean change in each measure by patient-reported response (improved, stayed the same, or worsened) are shown in Figures 1A & B. In general, the mean score increased from ‘improved’ to ‘worsened’ as expected (with the exception of PROMIS PH which declines given a different direction of scoring). The MCII for each measure was as follows: RAPID3 -1.8 (-4.1 to 0.5), Patient Global -0.6 (-1.6 to 0.4), Physician Global -1 (-1.9 to -0.1), cDAPSA -5.7 (-9.8 to -1.7), and PROMIS PH 1.9 (-2.1 to 5.8). Correlation for each measure with the global assessment of response were: RAPID3 0.48, Patient Global 0.37, Physician Global 0.39, cDAPSA 0.51, and PROMIS PH 0.39.Figure 1A. Distribution of change (median, IQR) in RAPID3, Physician Global, Patient Global, PROMIS10a physical therapy by patient reported response.Conclusion:This is the first study to test thresholds of meaning for these particular measures in PsA. The MCII values are relatively low for all outcome measures. This may be related to the relatively low disease activity at baseline but is consistent with patients seen in clinical practice initiating therapy.2References:[1]Ward MM et al. J Clinical Epi 2014;2Ward MM et al. J Clinical Epi 2015Figure 2B. Distribution of change (median, IQR) in clinical DAPSA by patient reported response.Disclosure of Interests:Alexis Ogdie Grant/research support from: Pfizer, Novartis, Consultant of: Abbvie, Amgen, BMS, Celgene, Corrona, Janssen, Lilly, Pfizer, Novartis, M Elaine Husni Grant/research support from: Pfizer, Consultant of: AbbVie, Bristol-Myers Squibb, Eli Lilly, Janssen, Novartis, Regeneron, and UCB, Jose Scher Consultant of: Novartis, Janssen, UCB, Sanofi., Ethan Craig: None declared, Soumya Reddy Grant/research support from: AmgenCelgeneAbbvie, Consultant of: AmgenPfizerNovartisJaansenUCB, Jessica A. Walsh Grant/research support from: AbbVie, Pfizer, Janssen, Consultant of: AbbVie, Novartis, Eli Lilly and Company, UCB


2021 ◽  
Vol 80 (Suppl 1) ◽  
pp. 900.1-900
Author(s):  
L. Diebold ◽  
T. Wirth ◽  
V. Pradel ◽  
N. Balandraud ◽  
E. Fockens ◽  
...  

Background:Among therapeutics used to treat rheumatoid arthritis (RA), Tocilizumab (TCZ) and Abatacept (ABA) are both biologic agents that can be delivered subcutaneously (SC) or intravenously (IV). During the first COVID-19 lockdown in France, all patients treated with IV TCZ or IV ABA were offered the option to switch to SC administration.Objectives:The primary aim was to assess the impact of changing the route of administration on the disease activity. The second aim was to assess whether the return to IV route at the patient’s request was associated with disease activity variation, flares, anxiety, depression and low physical activity during the lockdown.Methods:We conducted a prospective monocentric observational study. Eligibility criteria: Adult ≥ 18 years old, RA treated with IV TCZ or IV ABA with a stable dose ≥3 months, change in administration route (from IV to SC) between March 16, 2020, and April 17, 2020. The following data were collected at baseline and 6 months later (M6): demographics, RA characteristics, treatment, history of previous SC treatment, disease activity (DAS28), self-administered questionnaires on flares, RA life repercussions, physical activity, anxiety and depression (FLARE, RAID, Ricci &Gagnon, HAD).The primary outcome was the proportion of patients with a DAS28 variation>1.2 at M6. Analyses: Chi2-test for quantitative variables and Mann-Whitney test for qualitative variables. Factors associated with return to IV route identification was performed with univariate and multivariate analysis.Results:Among the 84 patients who were offered to switch their treatment route of administration, 13 refused to change their treatment. Among the 71 who switched (48 TCZ, 23 ABA), 58 had a M6 follow-up visit (13 lost of follow-up) and DAS28 was available for 49 patients at M6. Main baseline characteristics: female 81%, mean age 62.7, mean disease duration: 16.0, ACPA positive: 72.4%, mean DAS28: 2.01, previously treated with SC TCZ or ABA: 17%.At M6, the mean DAS28 variation was 0.18 ± 0.15. Ten (12.2%) patients had a DAS28 worsening>1.2 (ABA: 5/17 [29.4%] and TCZ: 5/32 [15.6%], p= 0.152) and 19 patients (32.8%) had a DAS28 worsening>0.6 (ABA: 11/17 [64.7%] and TCZ: 8/32 [25.0%], p= 0.007).At M6, 41 patients (77.4%) were back to IV route (26 TCZ, 15 ABA) at their request. The proportion of patients with a DAS28 worsening>1.2 and>0.6 in the groups return to IV versus SC maintenance were 22.5%, 42.5% versus 11.1% and 22.2% (p=0.4), respectively. The univariate analysis identified the following factors associated with the return to IV route: HAD depression score (12 vs 41, p=0.009), HAS anxiety score (12 vs 41, p=0.047) and corticosteroid use (70% vs 100%, p=0.021), in the SC maintenance vs return to IV, respectively.Conclusion:The change of administration route of TCZ and ABA during the first COVID-19 lockdown was infrequently associated with a worsening of RA disease. However, the great majority of the patients (77.4%) request to return to IV route, even without disease activity worsening. This nocebo effect was associated with higher anxiety and depression scores.Disclosure of Interests:None declared


2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 6-6
Author(s):  
A. Ortolan ◽  
S. Ramiro ◽  
F. A. Van Gaalen ◽  
T. K. Kvien ◽  
R. B. M. Landewé ◽  
...  

Background:Ankylosing Spondylitis Disease Activity Score (ASDAS) is a composite index measuring disease activity in axial spondyloarthritis (axSpA). It includes questions from the Bath Ankylosing Spondylitis Disease Activity Index (BASDAI), Patient Global Assessment (PGA), and inflammation biomarkers. However, ASDAS calculation is not always possible because PGA is sometimes not collected.Objectives:To develop an alternative ASDAS to be used in research settings when PGA is unavailable.Methods:Longitudinal data from 4 axSpA cohorts and 2 RCTs were combined. Observations were randomly split in a development (N=1026) and a validation cohort (N=1059). Substitutes of PGA by BASDAI total score, single or combined individual BASDAI questions, and a constant value, were considered. In the development cohort, conversion factors for each substitute were defined by Generalized Estimating Equations. Validation was performed in the validation cohort according to the OMERACT filter, taking into consideration: 1) Truth (agreement with original-ASDAS in the continuous score, by intraclass correlation coefficient -ICC- and in disease activity states, by weighted kappa) 2) Discrimination (standardized mean difference –SMD- of ASDAS scores between high/low disease activity states defined by external anchors e.g Patient Acceptable Symptom State –PASS-; agreement -kappa- in the % of patients reaching ASDAS improvement criteria according to alternative vs. original formulae) 3) Feasibility.Results:Taking all psychometric properties into account and comparing the different formulae (Table), alternative-ASDAS using BASDAI total as PGA replacement proved to be: 1) truthful (agreement with original-ASDAS: ICC=0.98, kappa=0.90); 2) discriminative: it could discriminate between high/low disease activity states (e.g. scores between PASS no/yes: SMD=1.37 versus original-ASDAS SMD=1.43) and was sensitive to change (agreement with original-ASDAS in major improvement/clinically important improvement criteria: kappa=0.93/0.88; 3) feasible (BASDAI total often available; conversion coefficient≈1).Table.Psychometric properties of alternative ASDAS formulaeConclusion:Alternative-ASDAS using BASDAI total score as PGA replacement is the most truthful, discriminative and feasible instrument. This index enables ASDAS calculation in existing cohorts without PGA.Disclosure of Interests:Augusta Ortolan: None declared, Sofia Ramiro: None declared, Floris A. van Gaalen: None declared, Tore K. Kvien Grant/research support from: Received grants from Abbvie, Hospira/Pfizer, MSD and Roche (not relevant for this abstract)., Consultant of: Have received personal fees from Abbvie, Biogen, BMS, Celltrion, Eli Lily, Hospira/Pfizer, MSD, Novartis, Orion Pharma, Roche, Sandoz, UCB, Sanofi and Mylan (not relevant for this abstract)., Paid instructor for: Have received personal fees from Abbvie, Biogen, BMS, Celltrion, Eli Lily, Hospira/Pfizer, MSD, Novartis, Orion Pharma, Roche, Sandoz, UCB, Sanofi and Mylan (not relevant for this abstract)., Speakers bureau: Have received personal fees from Abbvie, Biogen, BMS, Celltrion, Eli Lily, Hospira/Pfizer, MSD, Novartis, Orion Pharma, Roche, Sandoz, UCB, Sanofi and Mylan (not relevant for this abstract)., Robert B.M. Landewé Consultant of: AbbVie; AstraZeneca; Bristol-Myers Squibb; Eli Lilly & Co.; Galapagos NV; Novartis; Pfizer; UCB Pharma, Pedro M Machado Consultant of: PMM: Abbvie, Celgene, Janssen, Lilly, MSD, Novartis, Pfizer, Roche and UCB, Speakers bureau: PMM: Abbvie, BMS, Lilly, MSD, Novartis, Pfizer, Roche and UCB, Adeline Ruyssen-Witrand Grant/research support from: Abbvie, Pfizer, Consultant of: Abbvie, BMS, Lilly, Mylan, Novartis, Pfizer, Sandoz, Sanofi-Genzyme, Astrid van Tubergen Consultant of: Novartis, Caroline Bastiaenen: None declared, Désirée van der Heijde Consultant of: AbbVie, Amgen, Astellas, AstraZeneca, BMS, Boehringer Ingelheim, Celgene, Cyxone, Daiichi, Eisai, Eli-Lilly, Galapagos, Gilead Sciences, Inc., Glaxo-Smith-Kline, Janssen, Merck, Novartis, Pfizer, Regeneron, Roche, Sanofi, Takeda, UCB Pharma; Director of Imaging Rheumatology BV


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