scholarly journals Disease Activity Measures in Paediatric Rheumatic Diseases

2013 ◽  
Vol 2013 ◽  
pp. 1-10 ◽  
Author(s):  
Nadia J. Luca ◽  
Brian M. Feldman

Disease activity refers to potentially reversible aspects of a disease. Measurement of disease activity in paediatric rheumatic diseases is a critical component of patient care and clinical research. Disease activity measures are developed systematically, often involving consensus methods. To be useful, a disease activity measure must be feasible, valid, and interpretable. There are several challenges in quantifying disease activity in paediatric rheumatology; namely, the conditions are multidimensional, the level of activity must be valuated in the context of treatment being received, there is no gold standard for disease activity, and it is often difficult to incorporate the patient’s perspective of their disease activity. To date, core sets of response variables are defined for juvenile idiopathic arthritis, juvenile systemic lupus erythematosus, and juvenile dermatomyositis, as well as definitions for improvement in response to therapy. Several specific absolute disease activity measures also exist for each condition. Further work is required to determine the optimal disease activity measures in paediatric rheumatology.

2021 ◽  
Vol 80 (Suppl 1) ◽  
pp. 812.1-812
Author(s):  
F. Oliveira Pinheiro ◽  
B. M. Fernandes ◽  
S. Garcia ◽  
M. Rato ◽  
D. Fonseca ◽  
...  

Background:There is growing evidence that vitamin D [25(OH)D]) plays an important role in maintaining skeletal health and modulating the immune system. Epidemiological data indicate that vitamin D deficiency is common in immune-mediated rheumatic diseases, especially in rheumatoid arthritis, but there is little data regarding its association with disease activity and response to therapy in patients with psoriatic arthritis (PsA) under bDMARD therapy.Objectives:We aimed to assess whether 25(OH)D basal levels correlate with disease activity and clinical response to the first bDMARD, at 6 and 12 months of therapy, in a monocentric cohort of patients with PsA.Methods:This retrospective study was carried out on PsA patients from a Rheumatology department of a tertiary hospital, fulfilling CASPAR criteria and registered in our national database (Reuma.pt), who started the first bDMARD since 2008. Demographic, clinical and laboratory criteria were evaluated at 0, 6 and 12 months of biologic therapy. Disease activity was assessed using CDAI, SDAI, DAS28(4V), BASDAI, ASDAS, DAPSA and the response was measured using the EULAR, BASDAI50, ASDAS, ASAS, ACR and PsARC responses. Correlations were made between absolute serum levels of 25(OH)D and continuous variables, as well as associations between different vitamin D cutoffs and disease activity measures and response criteria. Multiple linear and logistic regression analyses were performed to determine whether vitamin D is a predictor of disease activity and therapeutic response.Results:We included 81 patients, 41 (50.6%) females; with a mean age of 48.0±11.7 years, a mean disease duration of 9.5±7.4 years and a mean body mass index of 28.4±5.2 kg/m2. Thirteen (16.0%) were smokers. The mean 25(OH)D basal level was 25.5±13.2 ng/ml, 21 (25.9%) had 25(OH)D basal levels ≥30 ng/mL and 31 (38.3%) ≤20 ng/mL. Sixty-two patients (76.5%) were under csDMARD therapy. Golimumab (29, 35,8%), etanercept (28, 34.6%) and adalimumab (10, 12.3%) were the most frequently prescribed bDMARDs. There were only very weak, albeit positive, correlations between 25(OH)D levels and measures of disease activity. The BASDAI50 response at 6 months was associated with higher basal 25(OH)D levels (29.5±14.5 vs 21.5±10.2 ng/mL, p = 0.013); the ASAS20 (33.9±15.9 vs 24.2±12.8 ng/mL; p = 0.023), ASAS40 (31.9±14.6 vs 25.0±13.8 ng/mL; p = 0.023) and ASAS70 (47.0±4.2 vs 26.6±14.2; p = 0.027) responses at 12 months were associated with higher basal levels of 25(OH)D; basal 25(OH)D levels were ≥ 30ng/mL in a significantly higher proportion of patients who achieved CDAI (38.9% vs 10.5%; p = 0.027) and SDAI (38.9% vs 7.7%; p = 0.008) remission and ASDAS disease inactive (29.4% vs 7.3%; p = 0.040) at 1 year. In the regression models, basal levels of 25(OH)D were found to be predictors of good EULAR responders (OR 1.315, 1.017-1.213 95% CI; p = 0.037) at 6 months. Basal levels of 25(OH)D were not significantly different in patients who discontinued bDMARD and no significant correlations or associations were identified regarding more specific PsA activity measures, such as DAPSA and PsARC, nor were they predictive of these responses.Conclusion:We can conclude that there is a global trend for an association between higher levels of vitamin D and lower measures of disease activity and better therapeutic responses to the first biologic. It was possible to find statistically significant associations with some disease activity measures and response criteria that, although primarily designed for other rheumatic diseases, are often used in PsA.Disclosure of Interests:None declared.


Rheumatology ◽  
2020 ◽  
Vol 59 (Supplement_2) ◽  
Author(s):  
Kieran Sandhu ◽  
Nadia Ahmad ◽  
Ayna Verdiyeva ◽  
Raashid Luqmani

Abstract Background Auto-immune rheumatic diseases are multisystem conditions with high morbidity and mortality, where co-ordinated multidisciplinary (MDT) care is necessary to ensure effective and timely treatment. An NHS England Commissioning for Quality and Innovation was implemented in 2016 to support development of MDTs to achieve earlier diagnosis and intervention and standardise quality of care and access to high-cost drugs. Methods A regional network for MDTs was established in one centre, led by Rheumatology with input from other specialities. MDT outcomes are recorded in a bespoke database. We analysed MDT outcomes over a two-year period from September 2017 to August 2019 for the following: disease activity assessments, investigations requested, referrals to other specialists, treatment changes and the reasons for these. Results During this period, 2,750 MDT discussions were recorded for 1,270 patients. The most common diagnoses were giant cell arteritis (30%), systemic lupus erythematosus (9%) and granulomatosis with polyangiitis (6%). Disease activity scores were recorded on 2,744 (99.8%) occasions with BVAS (n = 1,658, mean 0.82, range 0-21) and SLEDAI (n = 379, mean 2.04, range 0-20) used most. This underlines that decisions were consistently based on validated outcome measures. Investigations were ordered on 1165 (42%) occasions. Imaging was requested in 1,124 cases, biopsy in 37, functional tests in 221 and other tests in 18. Ultrasound was requested most often (614). Onward referrals were made in 237 (9%), most frequently to respiratory (32) and ENT (32). On 1550 occasions (56%) a treatment change was made: 1049 (38%) had a new change and 501 (18%) had a previously planned change. The most common reasons were good response to therapy (354 [23%]), inefficacy (345 [22%]) or starting additional therapy (214 [14%]). This shows that a significant number of treatment changes were approved by the MDT, both for treatment reduction as well as escalation. A biologic was added on 65 occasions (4% of treatment changes), most commonly in GCA (8 started on tocilizumab), SLE (5) and GPA (5). Biologics were repeated in 121 (8%) and stopped in 25 (2%). Where biologics were added, mean pre-treatment BVAS was 4.7 and post-treatment was 0.6. Mean reduction in BVAS after treatment was 5.2. The equivalent mean scores for SLEDAI were 7 (pre-treatment), 2 (post-treatment) and 7.7 (reduction). On average, the addition of biologics resulted in reductions in disease activity scores, which provides justification for their use. Conclusion Our data suggest that the large number of MDT reviews over this two-year period contributed significantly to management of systemic auto-immune rheumatic diseases by approving investigations, referrals and treatment changes. This facilitated timely intervention and avoidance of overtreatment. The auditable recording of these reviews and disease activity scores ensured decisions were guided by objective assessments and the use of high-cost drugs was standardised and justifiable. Disclosures K. Sandhu None. N. Ahmad None. A. Verdiyeva None. R. Luqmani None.


2020 ◽  
Vol 7 (1) ◽  
pp. e000419
Author(s):  
Manuel Francisco Ugarte-Gil ◽  
Rocio Violeta Gamboa-Cardenas ◽  
Cristina Reátegui-Sokolova ◽  
Victor Román Pimentel-Quiroz ◽  
Paola Zeña-Huancas ◽  
...  

ObjectiveThe Lupus Foundation of America Rapid Evaluation of Activity in Lupus (LFA-REAL) clinician-reported outcome (ClinRO) and the LFA-REAL patient-reported outcome (PRO) were developed in order to capture manifestations of SLE from the perspective of both the clinician and the patient. The aim of this study is to compare the LFA-REAL ClinRO and PRO with other lupus disease activity measures.MethodsA cross-sectional analysis of patients from a single-centre cohort was performed using Spearman’s correlation. Disease activity measures included were LFA-REAL ClinRO (range 0–1400), LFA-REAL PRO (range 0–1200), Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K), clinical SLEDAI-2K and Physician Global Assessment (PGA, range 0–100).ResultsTwo hundred and twenty-seven patients with SLE were studied. The mean age was 46.3 (SD: 13.8); 212 (93.4%) were female. The mean (SD) LFA-REAL ClinRO was 25.4 (34.7), LFA-REAL PRO was 241.1 (187.6), PGA was 11.9 (15.4), SLEDAI-2K was 2.3 (3.3) and clinical SLEDAI-2K was 1.6 (2.9). The LFA-REAL ClinRO correlated with PGA (r=0.758, p<0.001), SLEDAI-2K (r=0.608, p<0.001) and clinical SLEDAI-2K (r=0.697, p<0.001); the LFA-REAL PRO correlated modestly with PGA (r=0.160, p=0.016), SLEDAI-2K (r=0.121, p=0.069), clinical SLEDAI-2K (r=0.143, p=0.031) and LFA-REAL ClinRO (r=0.161, p=0.015).ConclusionsThe LFA-REAL ClinRO and the LFA-REAL PRO had good and weak correlations, respectively, with several physician-based disease activity measures in a cross-sectional study, suggesting their potential usefulness in establishing disease severity. Longitudinal studies will be required to determine their value in monitoring patients with SLE.


2021 ◽  
Vol 10 (3) ◽  
pp. 382
Author(s):  
Jesús A. Valero-Jaimes ◽  
Ruth López-González ◽  
María A. Martín-Martínez ◽  
Carmen García-Gómez ◽  
Fernando Sánchez-Alonso ◽  
...  

Objective: Since obesity has been associated with a higher inflammatory burden and worse response to therapy in patients with chronic inflammatory rheumatic diseases (CIRD), we aimed to confirm the potential association between body mass index (BMI) and disease activity in a large series of patients with CIRDs included in the Spanish CARdiovascular in rheuMAtology (CARMA) registry. Methods: Baseline data analysis of patients included from the CARMA project, a 10-year prospective study of patients with rheumatoid arthritis (RA), ankylosing spondylitis (AS), and psoriatic arthritis (PsA) attending outpatient rheumatology clinics from 67 Spanish hospitals. Obesity was defined when BMI (kg/m2) was >30 according to the WHO criteria. Scores used to evaluate disease activity were Disease Activity Score of 28 joints (DAS28) in RA, Bath Ankylosing Spondylitis Disease Activity Index (BASDAI) in AS, and modified DAS for PsA. Results: Data from 2234 patients (775 RA, 738 AS, and 721 PsA) were assessed. The mean ± SD BMI at the baseline visit were: 26.9 ± 4.8 in RA, 27.4 ± 4.4 in AS, and 28.2 ± 4.7 in PsA. A positive association between BMI and disease activity in patients with RA (β = 0.029; 95%CI (0.01–0.05); p = 0.007) and PsA (β = 0.036; 95%CI (0.015–0.058); p = 0.001) but not in those with AS (β = 0.001; 95%CI (−0.03–0.03); p = 0.926) was found. Disease activity was associated with female sex and rheumatoid factor in RA and with Psoriasis Area Severity Index and enthesitis in PsA. Conclusions: BMI is associated with disease activity in RA and PsA, but not in AS. Given that obesity is a potentially modifiable factor, adequate control of body weight can improve the outcome of patients with CIRD and, therefore, weight control should be included in the management strategy of these patients.


2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 327.1-328
Author(s):  
A. Kavanaugh ◽  
M. H. Buch ◽  
B. Combe ◽  
L. Bessette ◽  
I. H. Song ◽  
...  

Background:The primary treatment goal for patients (pts) with rheumatoid arthritis (RA) is a state of sustained clinical remission (REM) or low disease activity (LDA).1,2Objectives:To assess the long-term sustainability of responses to upadacitinib (UPA), a JAK inhibitor, with or without background csDMARD(s) in pts with RA.Methods:Data are from two phase 3 randomized, controlled trials of UPA in RA pts with roughly similar baseline disease characteristics: SELECT-NEXT enrolled pts with an inadequate response (IR) to csDMARD(s) on background stable csDMARD(s) receiving UPA 15 mg or 30 mg once daily or placebo for 12 weeks (wks); SELECT-MONOTHERAPY enrolled methotrexate (MTX)-IR pts receiving UPA 15 mg or 30 mg monotherapy or blinded MTX for 14 wks. After 12/14 wks, pts could enter a blinded long-term extension and receive UPA 15 mg or 30 mg for up to 5 years. This post hoc analysis evaluated clinical REM (CDAI ≤2.8; SDAI ≤3.3), LDA (CDAI≤10; SDAI≤11), and DAS28(CRP) <2.6/≤3.2 at first occurrence before Wk 84; additionally, these measures were evaluated at 3, 6, and 12 months after the first occurrence for the total number of pts randomized to UPA 15 mg. Sustainability of response was evaluated by Kaplan-Meier only for those pts who achieved REM/LDA and was defined as time to the earliest date of losing response at two consecutive visits or discontinuation of study drug. The predictive ability of time to clinical REM/LDA was assessed using Harrell’s concordance (c)-index (for reference, an index ~ 0.5, indicates no ability to predict; an index of 1 or -1 would be a perfect prediction). The last follow up dates were 22 March, 2018 (SELECT-NEXT) and 25 May, 2019 (SELECT-MONOTHERAPY), when all pts had reached the Wk 84 visit.Results:Through Wk 84, the percent of treated pts achieving CDAI REM/LDA was 43%/79% for those receiving UPA 15 mg with background csDMARD(s) (SELECT-NEXT) and 37%/76% for those receiving UPA 15 mg without background csDMARD(s) (SELECT-MONOTHERAPY). 35%/25% of pts randomized to UPA 15 mg with background csDMARD(s) and 27%/23% of pts randomized to UPA 15 mg without background csDMARD(s) achieved sustained CDAI REM through 6/12 months after the first occurrence. 64%/56% of pts randomized to UPA 15 mg with background csDMARD(s) and 61%/56% of pts randomized to UPA 15 mg without background csDMARD(s) achieved sustained CDAI LDA through 6/12 months after the first occurrence (Figure 1). Time to initial clinical REM/LDA did not appear to be associated with sustained disease control. The c-indices (95%CI) for CDAI REM in the UPA 15 mg with background csDMARD(s) and UPA 15 mg without background csDMARD(s) groups were 0.541 (0.47, 0.62) and 0.568 (0.49, 0.65) and that of LDA were 0.521 (0.46, 0.58) and 0.498 (0.43, 0.56), respectively. Through last follow-up visit, 55% of pts receiving UPA 15 mg with background csDMARD(s) and 62% of pts receiving UPA 15 mg without background csDMARD(s) remained in CDAI REM while 72% and 70% of pts remained in CDAI LDA, respectively (Figure 2). Similar results were observed across other disease activity measures (SDAI REM/LDA and DAS28(CRP) <2.6/≤3.2).Conclusion:More than a quarter and more than a half of pts with RA and prior IR to csDMARD(s) receiving UPA with or without background csDMARD therapy achieved sustained clinical REM and LDA, respectively, across disease activity measures. Sustainability of responses appeared comparable among pts receiving UPA with or without background csDMARDs through up to 84 wks.References:[1]EULAR: Smolen JS, et al. Ann Rheum Dis 2017;76:960–977.[2]ACR: Singh et al. Arthritis & Rheumatology Vol. 68, No. 1, January 2016, pp 1–26.Disclosure of Interests: :Arthur Kavanaugh Grant/research support from: Abbott, Amgen, AstraZeneca, BMS, Celgene Corporation, Centocor-Janssen, Pfizer, Roche, UCB – grant/research support, Maya H Buch Grant/research support from: Pfizer, Roche, and UCB, Consultant of: Pfizer; AbbVie; Eli Lilly; Gilead Sciences, Inc.; Merck-Serono; Sandoz; and Sanofi, Bernard Combe Grant/research support from: Novartis, Pfizer, Roche-Chugai, Consultant of: AbbVie; Gilead Sciences, Inc.; Janssen; Eli Lilly and Company; Pfizer; Roche-Chugai; Sanofi, Speakers bureau: Bristol-Myers Squibb; Gilead Sciences, Inc.; Eli Lilly and Company; Merck Sharp & Dohme; Pfizer; Roche-Chugai; UCB, Louis Bessette Grant/research support from: AbbVie, Amgen, Bristol-Myers Squibb, Celgene, Eli Lilly, Janssen, Merck, Novartis, Pfizer, Roche, Sanofi, UCB Pharma, Consultant of: AbbVie, Amgen, Bristol-Myers Squibb, Celgene, Eli Lilly, Janssen, Merck, Novartis, Pfizer, Roche, Sanofi, UCB Pharma, Speakers bureau: AbbVie, Amgen, Bristol-Myers Squibb, Celgene, Eli Lilly, Janssen, Merck, Novartis, Pfizer, Sanofi, In-Ho Song Shareholder of: AbbVie Inc., Employee of: AbbVie Inc., Yanna Song Shareholder of: AbbVie Inc., Employee of: AbbVie Inc., Jessica Suboticki Shareholder of: AbbVie Inc., Employee of: AbbVie Inc., Peter Nash Grant/research support from: AbbVie, Bristol-Myers Squibb, Celgene, Eli Lilly and Company, Gilead, Janssen, MSD, Novartis, Pfizer Inc, Roche, Sanofi, UCB, Consultant of: AbbVie, Bristol-Myers Squibb, Celgene, Eli Lilly, Gilead, Janssen, MSD, Novartis, Pfizer Inc, Roche, Sanofi, UCB, Speakers bureau: AbbVie, Bristol-Myers Squibb, Celgene, Eli Lilly, Gilead, Janssen, MSD, Novartis, Pfizer Inc, Roche, Sanofi, UCB


2011 ◽  
Vol 2011 ◽  
pp. 1-7 ◽  
Author(s):  
Andrew Taylor ◽  
Hanish Bagga

Objectives. To investigate which rheumatoid arthritis (RA) disease activity measures are being collected in patients receiving glucocorticoids, non-biologic or biologic disease-modifying antirheumatic drugs (DMARDs) in Australian rheumatology practice. Methods. A retrospective audit of medical records was conducted from eight rheumatology practices around Australia. Each rheumatologist recruited 30 consecutive eligible patients into the review, 10 of whom must have been receiving a biological agent for rheumatoid arthritis. Disease activity measures and radiographic assessments were collected from each patient's last consultation. For biologic patients, disease activity measures were also collected from when the patient was first initiated on the biological agent. Results. At last consultation, the disease measures that were recorded most often were ESR (89.2%), haemoglobin (87.5%), and CRP (84.2%). DAS28 was infrequently recorded (16.3%). The rate of recording disease activity measures for patients receiving biologic DMARDs decreased over time (mean 27 months). Conclusion. This review has shown inconsistency of RA activity measures being recorded in Australian rheumatology clinical practice. An accurate assessment of the disease process is necessary to effectively target rheumatoid arthritis patients to treat in order to achieve optimal outcomes.


2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 954.1-954
Author(s):  
M. Movahedi ◽  
D. Weber ◽  
P. Akhavan ◽  
E. Keystone

Background:Progressive rheumatoid arthritis (RA) is responsible for joint damage causing disabilities with no agreement on which disease measures best predict radiographic progressionObjectives:We aimed to determine which disease activity measures including disease activity score (DAS), modified (M) DAS28 (CRP), clinical disease activity index (CDAI), and health assessment questionnaire disability index (HAQ-DI) best predict rapid radiographic progression (RRP) in early RA patients at baseline (BL) and 3 months.Methods:PREMIER data, a 2-year, multicenter, double-blind active comparator–controlled study with methotrexate (MTX) naïve RA patients and active disease <3 years, were used. Only patients in the MTX arm were analyzed. RRP was defined as change in modified total Sharp (mTSS) > 3.5 at month 12. Logistic regression analysis assessed impact of measures at BL and 3 months on RRP at 12 months. Best cut-off points of M-DAS28(CRP) was also estimated using area under the receiver operating characteristic curve.Results:149 patients were included: female (n=113; 75.8%), positive RF (n=127; 85.2%), mean (SD) age 52.9 (13.3) years, disease duration 0.8 (0.9) year, DAS28(CRP) 6.3 (0.9). After adjusting for potential confounders, only M-DAS28(CRP) at BL (adjOR=3.29; 95% CI: 1.70-6.36) and 3 months (adjOR=2.56; 95% CI: 1.43-4.56) strongly predicted RRP at 12 months. M-DAS28(CRP) 4.5 and 2.6 at BL and 3 months maximized sensitivity and specificity for prediction of RRP.Conclusion:M-DAS28(CRP) was a stronger predictor at BL and 3 months for RRP compared with other disease activity measures. Removing tender joint count and patient global assessment from DAS28(CRP) improves prediction of RRP.References:[1] Breedveld FC, Weisman MH, Kavanaugh AF, Cohen SB, Pavelka K, van Vollenhoven R, et al. The PREMIER study: A multicenter, randomized, double-blind clinical trial of combination therapy with adalimumab plus methotrexate versus methotrexate alone or adalimumab alone in patients with early, aggressive rheumatoid arthritis who had not had previous methotrexate treatment. Arthritis and rheumatism. 2006;54(1):26-37.Acknowledgments :The authors wish to knowledge AbbVie Canada Inc. for providing patients data.Disclosure of Interests:Mohammad Movahedi Consultant of: Allergan, Deborah Weber: None declared, Pooneh Akhavan: None declared, Edward Keystone Grant/research support from: AbbVie; Amgen; Gilead Sciences, Inc; Lilly Pharmaceuticals; Merck; Pfizer Pharmaceuticals; PuraPharm; Sanofi, Consultant of: AbbVie; Amgen; AstraZeneca Pharma; Bristol-Myers Squibb Company; Celltrion; F. Hoffman-La Roche Ltd.; Genentech, Inc; Gilead Sciences, Inc.; Janssen, Inc; Lilly Pharmaceuticals; Merck; Myriad Autoimmune; Pfizer Pharmaceuticals, Sandoz, Sanofi-Genzyme, Samsung Bioepsis., Speakers bureau: AbbVie; Amgen; Bristol-Myers Squibb; Celltrion; F. Hoffman-La Roche Ltd, Janssen, Inc; Merck; Pfizer Pharmaceuticals; Sanofi-Genzyme; UCB


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