Setting the international standard for longitudinal follow-up of patients with systemic sclerosis: a Delphi-based expert consensus on core clinical features

Hoffmann‐Vold, Anna‐Maria; Distler, Oliver; Baron, Murray; Kowal‐Bielecka, Otylia; Khanna, Dinesh; Allanore, Yannick

doi:10.1136/rmdopen-2018-000826

Cited by 40 publications

(26 citation statements)

References 22 publications

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“…Lately, there has been an increasing effort to establish expert consensus, including diagnosis and monitoring of ILD in SSc patients, to provide standardized clinical guidance for the identification and management of systemic sclerosis -interstitial lung disease (SSc-ILD). 5,6 There was agreement that the severity at time of ILD diagnosis in patients with SSc should be assessed using high-resolution computed tomography (HRCT) pattern and extent. 7 However, more than one measure should be used to determine ILD severity including respiratory symptoms, exercise-induced oxygen desaturation and quality of life.…”

Section: Introductionmentioning

confidence: 99%

Circulating biomarkers of systemic sclerosis – interstitial lung disease

Hoffmann‐Vold

Fretheim

Meier

et al. 2020

Journal of Scleroderma and Related Disorders

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Interstitial lung disease is a frequent organ manifestation in systemic sclerosis and is associated with high mortality. It is crucial to diagnose interstitial lung disease in systemic sclerosis and to assess severity and identify patients prone to progression at an early stage to ultimately decrease organ damage and improve outcome. Circulating anti-topoisomerase-I autoantibodies have long been associated with the presence and development of systemic sclerosis – interstitial lung disease, evidence on their potential to further predict the clinical course of systemic sclerosis is however conflicting. C-reactive protein is a marker of infection and systemic inflammation with widespread clinical application and is elevated in systemic sclerosis with a tendency towards higher abundancy in patients with early disease. The role of other circulating biomarkers is promising but hampered by the lack of standardized criteria and guidelines for sample/data collection, analyses, reporting and validation and has not reached prime time for clinical application. However, epithelial markers including Krebs von den Lungen-6 and surfactant protein D and several cytokines and chemokines including CCL2 and CCL18 for severity assessment of systemic sclerosis – interstitial lung disease patients at the time of interstitial lung disease diagnosis and to predict interstitial lung disease progression have been reported and seem to be promising candidate biomarkers in the future.

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Section: Introductionmentioning

confidence: 99%

Circulating biomarkers of systemic sclerosis – interstitial lung disease

Hoffmann‐Vold

Fretheim

Meier

et al. 2020

Journal of Scleroderma and Related Disorders

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“…This results in differences between centres, such as the varying frequency of GAVE screening by upper endoscopy noted across the Nor-SSc cohort. With the aim of standardizing the follow-up of patients with SSc, a Delphi-based expert consensus was recently published that will hopefully lead to an adequate standard of care for all patients with SSc and enhance the standardization and homogenization of the practices worldwide [ 35 ].…”

Section: Discussionmentioning

confidence: 99%

Multidimensional tracking of phenotypes and organ involvement in a complete nationwide systemic sclerosis cohort

et al. 2020

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Objective SSc is a severe, heterogeneous multi-organ disease where population-based estimates on phenotypic spectrum, overall disease burden and societal impact are largely missing. Here the objective was to provide the first-ever complete national-level data on phenotype and major organ afflictions in SSc. Methods A stepwise strategy was applied to find and characterize every SSc patient resident in Norway from 2000 to 2012. First we identified every case in the country registered with an International Classification of Diseases, Tenth Revision code for SSc (M34). Next we manually reviewed all cases coded as M34 to determine whether they met the 1980 ACR and/or 2013 ACR/EULAR classification criteria for SSc and could be included in the Norwegian SSc cohort (Nor-SSc). Finally, all disease features from SSc onset to study end were reviewed. Results The Nor-SSc cohort included 815 SSc patients. The mean age at diagnosis was 53 years, with 84% females and 77% limited cutaneous SSc. The estimated incidence increased from 4 per million in 2000 to 13 per million in 2012. We identified high cumulative frequencies of internal organ involvement, coexistence of multiple organ afflictions across disease subsets and autoantibody status and stable frequencies of pulmonary arterial hypertension across haemodynamic definitions, but indications of referral-related differences in pulmonary hypertension detection rates across the study area. Conclusion This nationwide cohort study provides new, unbiased evidence for a high disease burden in SSc patients of Caucasian descent and indicates the existence of hurdles preventing equality of assessment across the SSc population.

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“…The included predictors in the Machine-Learning-Assisted model were selected based on the predictors identified by experts, 8 additional predictors were selected based on clinical expertise and current literature. In order to prevent exclusion of too many patients due to missingness, we dropped four variables (out of 94) with a missingness percentage >5% (nailfold videocapillaroscopy, of which annual collection started in 2013, and three variables derived from the UCLA GIT questionnaire, namely faecal soiling, diarrhoea and distension/bloating, of which annual collection started in 2013).…”

Section: Methodsmentioning

confidence: 99%

“…Overall, 55 items were identified including clinical assessments, laboratory measurements, imaging and functional investigations. 8 Whether the identified items are sufficient to identify disease progression timely in all patients is yet to be determined. Moreover, in some patients with mild disease, annual follow-up might even be more concise and assessing 55 tools on an annual basis might not be necessary.…”

Section: Introductionmentioning

confidence: 99%

New risk model is able to identify patients with a low risk of progression in systemic sclerosis

et al. 2021

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ObjectivesTo develop a prediction model to guide annual assessment of systemic sclerosis (SSc) patients tailored in accordance to disease activity.MethodsA machine learning approach was used to develop a model that can identify patients without disease progression. SSc patients included in the prospective Leiden SSc cohort and fulfilling the ACR/EULAR 2013 criteria were included. Disease progression was defined as progression in ≥1 organ system, and/or start of immunosuppression or death. Using elastic-net-regularisation, and including 90 independent clinical variables (100% complete), we trained the model on 75% and validated it on 25% of the patients, optimising on negative predictive value (NPV) to minimise the likelihood of missing progression. Probability cutoffs were identified for low and high risk for disease progression by expert assessment.ResultsOf the 492 SSc patients (follow-up range: 2–10 years), disease progression during follow-up was observed in 52% (median time 4.9 years). Performance of the model in the test set showed an AUC-ROC of 0.66. Probability score cutoffs were defined: low risk for disease progression (<0.197, NPV:1.0; 29% of patients), intermediate risk (0.197–0.223, NPV:0.82; 27%) and high risk (>0.223, NPV:0.78; 44%). The relevant variables for the model were: previous use of cyclophosphamide or corticosteroids, start with immunosuppressive drugs, previous gastrointestinal progression, previous cardiovascular event, pulmonary arterial hypertension, modified Rodnan Skin Score, creatine kinase and diffusing capacity for carbon monoxide.ConclusionOur machine-learning-assisted model for progression enabled us to classify 29% of SSc patients as ‘low risk’. In this group, annual assessment programmes could be less extensive than indicated by international guidelines.

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Setting the international standard for longitudinal follow-up of patients with systemic sclerosis: a Delphi-based expert consensus on core clinical features

Cited by 40 publications

References 22 publications

Circulating biomarkers of systemic sclerosis – interstitial lung disease

Circulating biomarkers of systemic sclerosis – interstitial lung disease

Multidimensional tracking of phenotypes and organ involvement in a complete nationwide systemic sclerosis cohort

New risk model is able to identify patients with a low risk of progression in systemic sclerosis

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