Clinically relevant outcome measures for assessing disease activity, disease control and quality of life impairment in patients with chronic spontaneous urticaria and recurrent angioedema

Background: Chronic histaminergic angioedema (CHA) is defined as recurrent episodes of isolated angioedema (without hives) of unknown cause that respond to the same treatment as chronic spontaneous urticaria (CSU). Quality of life (QoL) studies have not been performed for CHA, except those carried out in the context of CSU associated with angioedema attacks (CSU-AE). Moreover, biomarkers for monitoring disease activity in CHA have not been identified. We aim to describe the burden of CHA and impact on patient QoL, compare the findings to those in CSU-AE patients, and investigate biomarker associations with disease severity and QoL parameters. Methods: We performed a prospective multicenter study that included 68 patients with CHA and 63 patients with CSU-AE. Demographic and clinical variables were collected. Validated patient-reported questionnaires were employed to analyze the quality of life and disease activity. Blood and serological parameters, including blood cell count, C-reactive protein, D-dimer and total IgE, were also analyzed. Results: Angioedema disease activity was significantly higher in CSU-AE patients (median AAS7, IQR: 1, [0–1]) than CHA patients (0, [0–1]; p= 0.022). A considerable impact on QoL was found in both groups, although significantly worse values were found for CSU-AE (median AEQoL, IQR: 37, [10–65]; p=0.005). CHA patients were older than CSU-AE patients, and female predominance was not observed. Conclusions: Angioedema severity and QoL impacts are significantly worse in CSU than in chronic histaminergic angioedema. Angioedema should be included in severity urticaria scores (UAS) as well as in specific quality of life urticaria scales.

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Outcome Measures in Multiple Sclerosis

10.1093/med/9780199341016.003.0011 ◽

2016 ◽

Author(s):

Robert M. Herndon

Keyword(s):

Quality Of Life ◽

Multiple Sclerosis ◽

Clinical Trials ◽

Disease Activity ◽

Outcome Measures

This chapter discusses the challenges involved in measuring disease activity and progression in multiple sclerosis and how different aspects of the disease are measured in clinical trials. Metrics have been developed to assess multiple aspects of the disease., These include, in addition to more global instruments, measures of specific functions such as, cognition, mobility, quality of life, fatigue, and so on. These scales are discussed along with their strengths and weaknesses.

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In Chronic Spontaneous Urticaria, Comorbid Depression Linked to Higher Disease Activity, and Substance P Levels

Frontiers in Psychiatry ◽

10.3389/fpsyt.2021.667978 ◽

2021 ◽

Vol 12 ◽

Author(s):

Bachar Memet ◽

Eren Vurgun ◽

Fatma Barlas ◽

Martin Metz ◽

Marcus Maurer ◽

...

Keyword(s):

Quality Of Life ◽

Substance P ◽

Disease Activity ◽

Chronic Spontaneous Urticaria ◽

Healthy Controls ◽

Comorbid Depression ◽

Healthy Control ◽

Severity Of Depression ◽

Urticaria Activity Score

Background: Patients with chronic spontaneous urticaria often exhibit psychiatric comorbidities including depression that contribute to the impairment of their quality of life. How CSU and depression are linked isn't well-understood. Substance P has been shown to be increased in patients with CSU and is held to contribute to the pathogenesis of depression.Methods: We measured disease activity in 30 CSU patients without depression and 30 CSU patients with depression by using the urticaria activity score. The severity of depression was assessed with the Beck Depression Inventory. We measured SP levels in these patients as well as in 30 healthy control subjects. In patients with comorbid depression, we correlated SP levels with CSU disease activity and the severity of depression.Results: In CSU patients, disease activity and the severity of depression were positively linked. UAS7 values were higher in CSU patients with comorbid depression as compared to those without (p < 0.05). SP levels were higher in CSU patients with depression than in those without (p < 0.001), but was similar in all CSU patients compared to healthy controls. SP levels weren't correlated with UAS7 values in CSU patients with depression, whereas they were weakly but significantly correlated with BDI scores (p < 0.05).Conclusion: Our results suggest that, in CSU patients with comorbid depression, CSU disease activity affects the severity of depression. CSU patients with high disease activity should be explored for comorbid depression.

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A new indicator to measure discordance between patient reported outcomes and traditional disease activity holds promise to advance care trajectories improve care in patients with early Rheumatoid Arthritis

10.1101/2021.12.01.21267120 ◽

2021 ◽

Author(s):

Sofia Pazmino ◽

Anikó Lovik ◽

Annelies Boonen ◽

Diederik De Cock ◽

Veerle Stouten ◽

...

Keyword(s):

Quality Of Life ◽

Disease Activity ◽

Disease Control ◽

Patient Reported Outcomes ◽

Sustained Remission ◽

List Type ◽

Factor Scores ◽

Patient Reported ◽

Disease Impact

ABSTRACTObjectiveTo unravel disease impact in early RA patients by separately quantifying patient reported (PRF), clinical (CF) and laboratory (LF) factors. We put forward a new indicator, the discordance score (DS), for early identification and prediction of unmet patient outcomes in terms of future achievement of sustained remission and RA-related quality of life (QoL).MethodsWe obtained factor scores via factor analysis in the CareRA trial, then calculated the DS between PRF and the mean of the other scores. We computed the improvement from baseline to week 104 (%) and area-under-the-curve (AUC) across time-points per factor score and compared these between patients achieving or not achieving sustained (week 16 to 104) remission (DAS28CRP<2.6) with ANOVA. Logistic and linear regressions respectively were used to predict SR based on previous factor and discordance scores, and QoL at year 1 and 2 based on DS at week 16.ResultsPRF, CF and LF scores improved rapidly within 8 weeks. In patients achieving SR; PRF improved 57%, CF 90% and LF 27%, compared to 32% PRF (p=0.13), 77% CF (p<0.001) and 9% LF (p=0.36) score improvement in patients not achieving SR. Patients achieving SR had an AUC of 15.7, 3.4 and 4.8 for PRF, CF and LF respectively, compared to 33.2, 10.1, and 7.2 in participants not achieving SR (p<0.001 for all). Early factor and discordance scores were associated with later stage factor scores as well as QoL and PRF scores predicted SR (p<0.005 for PRF and DS).ConclusionsAll factor scores improved rapidly, especially in patients achieving SR. Patient-reported burden improved less extensively. Discordance scores could help in predicting the need for additional non-pharmacological interventions to achieve SR and decrease disease impact.KEY MESSAGESWhat is already known about this subject?Early and intensive RA drug-treatment using disease activity as a target allows rapid disease control and prevents joint destruction.Including pain, fatigue and physical function when assessing patients with early RA broadens the evaluation of disease impact.What does this study add?Leveraging patient reported outcomes (pain, fatigue and physical function) and traditional disease activity measures, we introduce a new indicator (named discordance score) for unraveling disease impact and treatment efficacy.We show how the discordance score stands for current unmet patient reported outcomes and could be used to predict future sustained disease contol and quality of life (1 and 2 years after baseline).We demonstrate this effect both in patients with and without sustained remissionHow might this impact on clinical practice or future developments?The earlier detection of unmet needs despite good disease control could allow to perform timely interdisciplinary interventions other than medication adaptations and could promote psychosocial wellbeing for patients.

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OP141 Patient Relevant Outcome Measures As Predictors Of Healthcare Use In Multiple Sclerosis

International Journal of Technology Assessment in Health Care ◽

10.1017/s0266462317001970 ◽

2017 ◽

Vol 33 (S1) ◽

pp. 65-66

Author(s):

Michela Tinelli ◽

Olina Efthymiadou ◽

Jean Mossman ◽

Panos Kanavos

Keyword(s):

Quality Of Life ◽

Multiple Sclerosis ◽

Outcome Measures ◽

Economic Consequences ◽

Healthcare Resources ◽

Work Limitation ◽

The Impact ◽

Relevant Outcome

INTRODUCTION:Multiple-sclerosis (MS) is a highly disabling chronic disorder affecting young adults with long term economic consequences on society that escalate as MS disability increases (1,2). In the long-term, progression of MS results in increased level of disability and most patients will eventually experience some degree of functional impairment of the nervous system that impacts on mobility as well as sensory and coordination issues, bladder and sexual functioning, and mood and cognitionon (2). This is usually accompanied by a deterioration of their quality of life. Patient relevant outcome measures (PROMS) are largely used to measure individual disability, and quality of life in MS (2). International evidence from the International Multiple Sclerosis Study (IMPrESS) (2) was used to quantify the relationship between healthcare resources utilisation and disability, quality of life in individuals with MS.METHODS:Multivariable logistic regression was performed in order to identify patient-related variables reporting disability (Barthel) and utility (EQ-5D) that predict use of healthcare services (visits to GP, specialists, nurses, hospitalisation and treatment) and work limitation within the participants of the IMPrESS.RESULTS:Reponses were collected from 1,152 individuals across 21 countries of which 74.3 percent (856) were useful for analysis. Preliminary findings indicated that for the pooled data sets both EQ-5D and Barthel scores were predictors of healthcare resource use, across different categories (p<.05), except for nurse visits (Barthel only; p<.09). Overall the association between PROM data and use of healthcare resources appeared to be stronger with EQ-5D compared to Barthel. EQ-5D appeared to also predict the impact of MS on loss of productivity (in terms of work limitation; p<.05).CONCLUSIONS:PROMs can be used to predict the economic consequences of MS on healthcare providers and society, but more research is needed to confirm the robustness of the evidence and its validity across individual healthcare system settings.

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Disease Activity Only Moderately Correlates with Quality of Life Impairment in Patients with Chronic Spontaneous Urticaria

Dermatology ◽

10.1159/000351711 ◽

2013 ◽

Vol 226 (4) ◽

pp. 371-379 ◽

Cited By ~ 26

Author(s):

I. Koti ◽

K. Weller ◽

M. Makris ◽

E. Tiligada ◽

T. Psaltopoulou ◽

...

Keyword(s):

Quality Of Life ◽

Disease Activity ◽

Chronic Spontaneous Urticaria

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Is There a Symmetry in Disease Control and Quality of Life of Patients with Rheumatoid Arthritis Treated with Biological Therapy?

Symmetry ◽

10.3390/sym13040538 ◽

2021 ◽

Vol 13 (4) ◽

pp. 538

Author(s):

Konstantin Tachkov ◽

Vladimira Boyadzhieva ◽

Nikolay Stoilov ◽

Konstantin Mitov ◽

Guenka Petrova

Keyword(s):

Quality Of Life ◽

Rheumatoid Arthritis ◽

Disease Activity ◽

Disease Control ◽

Biological Therapy ◽

Activity Index ◽

Real Life ◽

Janus Kinase ◽

Low Level

This study aims to analyze and compare the disease activity control and quality of life of patients with rheumatoid arthritis (RA) who were treated with biological products in real-life settings. We tried to determine whether there is a symmetry in the performance of the biological molecules between each other and with the first Janus kinase (JAK) inhibitor. This is an observational, longitudinal, real-life study performed in the biggest rheumatology clinic during the period 2012–2020 comparing quality of life, cost of therapy, and disease control via different clinical measures. In all three disease activities measurement instruments, we observed an improvement for all biologic and target synthetic medicines. The disease activity score (DAS28) score decreased from 5.06 to 3.01, on average, for all INNs, suggesting that the majority of patients move away from moderate to low disease activity. The clinical disease activity index (CDAI) score decreased from 25.9 to 9.4, also indicating that patients with moderate disease activity reached a low level of activity. Similar results are reflected in the score, which fell from 27.7 to 10.3, again confirming the improvement to a low level of disease activity for patients treated with all INNs. Logically, with the successful control of disease activity, the quality of life (QoL) of the observed patients improved from 0.77 to 0.83 after a one-year follow up, as measured with the EuroQuol 5D-3L (EQ5D). Based on these results, we can consider that the observed biological INNs perform symmetrically in terms of the control of disease activity and improvement in the QoL of the observed patients. Biological therapy improves the disease control and quality of life of suitable patients with RA in real-life settings. All available biological therapies could be used interchangeably.

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From Clinically Relevant Outcome Measures To Quality of Life in Epilepsy

Value in Health ◽

10.1016/j.jval.2014.08.1924 ◽

2014 ◽

Vol 17 (7) ◽

pp. A573

Author(s):

Kinderen R.J.A. de ◽

B.F.M. Wijnen ◽

D. Postulart ◽

M.H. Majoie ◽

A.P. Aldenkamp ◽

...

Keyword(s):

Quality Of Life ◽

Outcome Measures ◽

Relevant Outcome

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Systematic Review of Development and Content Validity of Patient-reported Outcome Measures in Inflammatory Bowel Disease: Do We Measure What We Measure?

Journal of Crohn s and Colitis ◽

10.1093/ecco-jcc/jjaa057 ◽

2020 ◽

Vol 14 (9) ◽

pp. 1299-1315 ◽

Cited By ~ 3

Author(s):

Emma M van Andel ◽

Brechtje D M Koopmann ◽

Femke Crouwel ◽

Casper G Noomen ◽

Nanne K H de Boer ◽

...

Keyword(s):

Quality Of Life ◽

Disease Activity ◽

Outcome Measures ◽

Content Validity ◽

Patient Reported Outcome Measures ◽

Patient Reported Outcome ◽

Self Report ◽

Patient Reported ◽

Validity Studies

Abstract Background and Aims Patient-reported outcome measures are increasingly important in daily care and research in inflammatory bowel disease [IBD]. This study provides an overview of the content and content validity of IBD-specific patient-reported outcome measures on three selected constructs. Methods Databases were searched up to May 2019 for development and/or content validity studies on IBD-specific self-report measures on health-related quality of life, disability, and self-report disease activity in adults. Evidence was synthesised on content validity in three aspects: relevance, comprehensiveness, and comprehensibility following the COnsensus-based Standards for the selection of health Measurement INstruments methodology. Questionnaire items were organised in themes to provide an overview of important aspects of these constructs. Results For 14/44 instruments, 25 content validity studies were identified and 25/44 measures had sufficient content validity, the strongest evidence being of moderate quality, though most evidence is of low or very low quality. The Crohn’s Life Impact Questionnaire and IBD questionnaire-32 on quality of life, the IBD-Control on disease activity, and the IBD Disability Index Self-Report and its 8-item version on disability, have the strongest evidence of sufficient relevance, comprehensiveness, and comprehensibility, ranging from moderate to very low quality. A fair number of recurring items themes, possibly important for the selected constructs, was identified. Conclusions The body of evidence for content validity of IBD-specific health-related quality of life, self-report disease activity, and disability self-report measures is limited. More content validity studies should be performed after reaching consensus on the constructs of interest for IBD, and studies should involve patients.

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